Medical Hero Spotlight: Nicole & Chuck Johnson, Navigating ALS

Chuck and Nicole Johnson were out celebrating her 37th birthday in June 2023, when Chuck mentioned his hands had been shaking during dinner. Nicole was somewhat concerned, but Chuck reassured her that it ran in his family and wasn’t anything serious.


Married for five years and parents to two toddlers, the couple was excited about expanding their family. Their hopes turned into joy when Nicole discovered she was pregnant that September. However, only a month later, Chuck began to realize that something was seriously wrong.


One morning, while singing “The Wheels on the Bus” with their two-year-old daughter, he struggled to lift her in the air—something that had never been difficult for the 6’5” former collegiate football player. This marked the beginning of a series of troubling health issues.

A Search for Answers
“I remember I had just finished a Teams call for work when he came into my office to talk,” Nicole recalled. “He told me he had lost 30 pounds since the summer without trying, felt weak, and was experiencing more frequent and noticeable muscle fasciculations.”

That moment set them on an intense diagnostic journey. Chuck couldn’t get an appointment with his primary care physician for several months, so they turned to a CVS MinuteClinic for initial bloodwork. The results showed abnormal liver enzymes. Concerned, Nicole urged Chuck to send the results to his doctor, who then found an earlier appointment for him. Further bloodwork led his doctor to suspect a liver issue, but that didn’t explain the muscle fasciculations. Eventually, the Johnsons were referred to a neurologist at Beth Israel Deaconess Medical Center in Boston. After an evaluation, the general neurologist suspected Chuck was in the early stages of ALS, though more testing was needed for confirmation.


Through the holiday season and into the new year, Chuck endured eight weeks of extensive testing to rule out other conditions with a neuromuscular specialist. On February 14, 2024, he was officially diagnosed with limb-onset, sporadic ALS.


From that moment, Chuck and Nicole’s daily lives changed drastically as they adjusted to his evolving health and treatment needs.

A Life-Altering Diagnosis

The next six months brought immense changes and challenges. In the final months of her pregnancy, Nicole juggled processing Chuck’s diagnosis, scheduling his care and treatments, acquiring necessary medical equipment, working full-time, coordinating childcare for their two kids, and preparing for their newest arrival.


“It was overwhelming,” Nicole admitted. “I barely had time to stop and process everything—there was just so much to do.”


Hope for treatment was quickly met with roadblocks. Chuck was immediately given the standard of care treatment approved for ALS, but the Johnsons had also started looking into innovative clinical trials. The HEALEY ALS Platform trial at Massachusetts General Hospital (MGH) where Chuck was receiving care, had already reached enrollment capacity. Other local trials were unavailable due to Chuck’s elevated liver enzymes. The couple considered traveling for other trial opportunities, but with Nicole’s pregnancy, it wasn’t a feasible option.


In May, they welcomed their youngest daughter, Jenna, into the family. By that time, Chuck was using a motorized chair, and his health continued to decline. His breathing difficulties eventually led to a stay in the ICU at the end of the summer, and his lung capacity dropped below the threshold for any clinical trial eligibility.

“Unfortunately, this situation is common for those with a fast-progressing disease,” Nicole shares. “Many people become ineligible for traditional clinical trials, and it can cause families to feel hopeless.”

However, in October 2024, Chuck was granted access to an investigational drug through the Expanded Access Program. Unlike standard clinical trials where participants may receive a placebo, this program ensured he received the actual treatment. Though not a cure, the oral medication is helping to slow the decline of his ability to speak and eat, providing more stability for the family in the short-term.

Finding Community Support

Since Chuck’s diagnosis, the Johnson family have relied on their network of friends, family, healthcare aids, and their community to provide the support needed to manage each day. The family has hired a caregiver who is with them full-time, as well as an au pair from Colombia who joined them this past year to help with any childcare needs. There’s nearly always another member of the family staying over the house as well to help with preschool drop-off/pick-up so Nicole can get some sleep.


For Nicole, coordinating the different care needed for her family involves a lot of planning.

“Sometimes I feel like a cruise director or the family CEO, with the level of project management that goes into planning our lives,” she laughs. “Luckily, organization is one of my strong suits.”

The Johnsons have also found community and support from local advocacy groups. Organizations like the Pete Frates Foundation, ALS One, and Compassionate Care ALS have offered grants, equipment, and emotional support. These groups have provided essential resources like a shower chair and Hoyer transfer lift, as well as opportunities for the family to participate in community events, raising awareness and funds for ALS research.


Nicole and Chuck have also become actively involved in advocacy, sharing their story at events like the ALS One Snowball gala in March 2025.

Treasuring Every Moment

Today, Chuck remains a dedicated father to his three children, despite his challenging journey with ALS. The kids love spending quality time with their dad by riding on his wheelchair, helping to feed him Oreos (while sneaking some for themselves), finding ramps in public places for him to use, and taking fun trips in the family’s new wheelchair accessible van.

“This was a huge change in all our lives, but they’ve adapted so well. It’s really showed Chuck and I that time is the most precious thing in the world, worth more than any material things,” Nicole says. “Just enjoying time together with my kids and husband is something I’ll never take for granted.”

To keep up with updates from the Johnson family, visit their website: https://makewayforchucklings.com/

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

Written by Lindsey Elliott, Marketing & Communications Manager, CISCRP | lelliott@ciscrp.org

Input Needed On Using AI To Create Lay Summaries Of Trial Results

Lay summaries (LS) of clinical trial results play a critical role in improving transparency and accessibility in medical research by transforming complex scientific information into understandable content for the public and patients. Under the European Clinical Trial Regulation (EU CTR) 536/2014, clinical trial sponsors must publicly provide an LS within one year of a trial’s completion (within six months for pediatric trials). With the EU CTR now in effect, there is increased demand to create LS as efficiently as possible.

AI has emerged as a promising tool to increase the efficiency of LS creation and reduce resourcing, particularly through the use of large language models (LLMs). However, the application of AI in LS requires careful oversight and thoughtful integration to ensure that patient-facing content remains accurate, unbiased, and culturally sensitive.

Background And Development Of The Considerations Document

AI offers a range of opportunities for improving LS development:

  • Efficiency: AI can reduce the time required to draft summaries, allowing organizations to deliver clear, patient-friendly content more quickly.
  • Consistency: Automated processes can help ensure uniformity in tone and structure across summaries.
  • Accessibility: AI models trained in plain language principles can support health literacy.

Despite these benefits, the challenges of using AI in LS cannot be overlooked:

  • Accuracy: AI may misinterpret complex clinical data or introduce factual errors (e.g., AI hallucinations). Proper human oversight is necessary.
  • Bias: AI systems can replicate and amplify biases present in their training data.
  • Transparency: Undisclosed AI involvement in creating LS could erode public trust.
  • Ethics and Compliance: Ensuring compliance with privacy regulations and ethical standards is essential when deploying AI in patient-facing communications.

To address these challenges, the considerations document emphasizes a balanced approach: AI should complement, not replace, human expertise. Every AI-generated LS should undergo thorough review and validation by qualified professionals to ensure accuracy and relevance.

The considerations document outlines six key points to guide responsible AI use in LS development:

  1. Humans Must Be in the Loop: AI should augment, not replace, standard LS processes. Human involvement is crucial in areas such as model development, review and revision, and health literacy refinement. Existing personnel roles remain vital, with AI experts playing a newly integrated role.
  2. Researcher Involvement: Collaborating with researchers ensures accurate interpretation and plain language communication of results. While AI can expedite LS creation, expert oversight mitigates risks of misinformation or misinterpretation.
  3. Data Privacy: AI tools must respect data privacy by using aggregated rather than individual data, adhering to established reporting standards.
  4. Disclosure of AI Use: Transparency about AI involvement is essential to maintain public trust. Clear guidelines outline where, when, and how AI use should be disclosed.
  5. Trust: Ensuring accuracy, consistency, and the absence of bias is critical to maintaining credibility. Misinformation or biased outputs can harm public confidence.
  6. Technology Considerations: AI systems evolve rapidly, necessitating governance through internal standards, testing, monitoring, and continuous improvement to align with ethical and regulatory standards.
Public Comment Period: An Opportunity To Shape Best Practices

The public comment period is a vital step in refining the considerations document. Your feedback as clinical research professionals, medical writers, patient advocates, or technology experts is crucial to ensuring that the recommendations are comprehensive, actionable, and applicable across diverse contexts.

Comments are invited from a wide range of stakeholders, including research professionals, patients, and advocacy groups. Reviewers can provide input via an online survey hosted on the CISCRP website. The survey allows for detailed comments on specific sections of the document as well as general feedback. Respondents are also encouraged to identify their stakeholder group(s) to provide valuable context for their input.

How To Participate

The public comment period runs until February 18.

  1. Review the Document: The draft document is available on the CISCRP website for download.
  2. Provide Feedback: Use the online survey to submit your comments. The survey includes fields for line-specific feedback, proposed revisions, and general observations.
  3. Share the Opportunity: Spread the word within your networks to ensure broad participation from diverse perspectives.
Following the close of this period, all feedback will be reviewed and adjudicated by the working group. Insights from the comments will inform revisions, and the final document will be publicly released as a resource for the clinical research community.
Looking Forward

AI holds significant promise for advancing the development of lay summaries, but its use must be guided by principles of accuracy, transparency, and ethical responsibility. The considerations document represents a collaborative effort to establish a framework for responsible AI integration in LS processes. Your input is invaluable to ensuring this resource meets the needs of all stakeholders and supports the ultimate goal of improving patient and public understanding of clinical research results.

We encourage you to participate in this public comment period and contribute your expertise to this important initiative. Together, we can ensure that the use of AI in lay summary creation is both innovative and responsible, fostering greater trust and transparency in clinical research.

Note: Generative AI was used to assist with the creation of the first draft of this article.

Acknowledgements:

The authors are members of the working group developing the “Considerations for the Use of Artificial Intelligence in the Creation of Lay Summaries of Clinical Trial Results.” This article represents the current thinking of the group and the outcomes of the group’s work. The authors thank and acknowledge the respective contributions of all work group members. 

View Considerations Document
About the Authors

Behtash Bahador, MS is the director of health literacy at the nonprofit organization CISCRP and holds a Master of Science in health communication from the Tufts University School of Medicine. Since 2014, he has collaborated with a range of stakeholder groups to establish and implement patient- and community-centric initiatives across the life cycle of clinical research.

Behtash Bahador

Kim Edwards, Ph.D. is the senior director of health communication services at CISCRP, where she oversees the creation of easy-to-understand trial resources for patients, participants, and the public. Kim earned her BS in neuroscience from Trinity College and her Ph.D. in developmental and brain sciences from the University of Massachusetts Boston.

View Editorial on Clinical Leader.com here.

Medical Hero Spotlight: Kimberly Dorris, Graves’ Disease Patient Advocate

Diagnosed with Graves’ Disease

In 2007, during a routine checkup, Kimberly Dorris received abnormal bloodwork results indicating lower-than-average levels of thyroid stimulating hormone (TSH). She was referred to an endocrinologist who, after conducting further tests including an ultrasound and radioactive iodine uptake and scan, diagnosed her with Graves’ disease.

“At the time, the diagnosis was surprising,” Kimberly recalls. “But looking back, I realize I had many of the classic symptoms but attributed them to other causes.”

At the time, Kimberly had been struggling with insomnia and tremors, which she chalked up to stress from her fast-paced job and too much caffeine consumption. She also experienced muscle weakness and weight loss but had not thought they were caused by an underlying illness.

Later, Kimberly would learn that this is a common experience for people living with undiagnosed Graves’ disease. Many mistake the symptoms for stress, menopause, or other illnesses – while others are misdiagnosed with depression or anxiety.

Choosing a Treatment Plan

Graves’ disease is an autoimmune condition where the immune system mistakenly attacks the thyroid’s TSH receptors. This causes an overproduction of the TSH hormones, resulting in hyperthyroidism and its associated symptoms.

Treatment options for hyperthyroidism caused by Graves’ disease include medication to regulate thyroid hormones, surgical removal of the thyroid, or radioactive iodine treatment to destroy thyroid tissue. At the time of Kimberly’s diagnosis, antithyroid medication and radioactive iodine were the two primary treatments. “Nobody was talking about thyroidectomy as a front-line treatment option back then.”

“Neither option sounded great,” Kimberly says. “The antithyroid medication came with potential side effects. My doctors made radioactive iodine sound easy: ‘destroy your thyroid, so you’ll have hypothyroidism instead, and then take another pill to balance out your levels.’ However, the reality is more complex for patients. The connection between RAI and thyroid eye disease wasn’t widely known back in 2007.”

When it came time to choose between medication and radioactive iodine treatment, an unlikely factor influenced the decision: Kimberly was about to leave on a vacation. Before scheduling an RAIU test or radioactive iodine treatment, Kimberly’s doctor required a low-iodine diet (LID) for two weeks. This diet is standard for differentiated thyroid cancer patients prior to treatment with RAI, but is not as commonly recommended for Graves’. The diet deprives the thyroid of iodine so that it is more receptive to treatment when it begins. For two weeks prior to the RAIU test, Kimberly prepared all her meals from scratch to ensure minimal iodine consumption. Kimberly told the doctor that she wouldn’t be able to start the LID until she returned home. She was given a prescription for a generic antithyroid medication for the duration of the trip to stabilize her levels, which gave her time to consider long-term treatment options.

“The methimazole started as a temporary treatment, but it turned out to be a really good thing,” Kimberly recalls. “The medication got me to a place where I felt better and could think more clearly about my treatment. I was doing very well on the medication, so I chose to continue with it after coming home and stayed on the drug for seven years.”

By 2014, Kimberly was successfully weaned off treatment, achieving normal thyroid levels. However, this stability only lasted for two years before she began experiencing symptoms of hypothyroidism.

“I have been having a long, slow slide into hypothyroidism for the last several years, which happens to about 15% of patients,” Kimberly explains. “Managing Graves’ disease becomes a balancing act where we are constantly being monitored to ensure we stay between hyperthyroidism and hypothyroidism without sliding into either.”
Becoming A Full-Time Advocate

About a year after her diagnosis, Kimberly attended a large patient advocacy conference from the National Graves’ Disease Foundation (now the Graves’ Disease & Thyroid Foundation) to learn more about Graves’ disease and connect with others in the community. From there, she became an active member of an online message board, sparking her interest in deeper involvement. She eventually became a support group leader for the GDATF and established a local group in her home state of Arizona.

In 2010, Kimberly took on an opportunity to do contract work for the foundation and later transitioned into the full-time role. Today, she serves as the Executive Director & CEO of the GDATF.

“The foundation has really grown since its start in 1990, now serving as a supportive community for newly diagnosed patients and as a credible resource of information,” Kimberly says. “Many people who find out they have Graves’ disease or thyroid eye disease will immediately turn to Google for answers, but wading through so much information can be overwhelming – and in many cases, can be dangerously inaccurate. GDATF ensures all the information shared with patients is vetted and recommended by doctors.”

Exciting projects are underway at the GDATF, including the completion of a 24-page print newsletter, intended to share with community members and distribute at conferences. Kimberly recently co-hosted a two-part webinar with the foundation’s founder, Nancy Hord Patterson, focusing on patient survey results about thyroid eye disease. Funded by Amgen (formerly Horizon Therapeutics), the survey explored patient experiences with various treatment options and the emotional impact of the disease.

“The survey findings shed light on the emotional challenges faced by thyroid eye disease patients, which is something physicians must acknowledge and address,” Kimberly notes. “For many, the physical changes caused by the disease can make social interactions stressful and exacerbate mental health issues like anxiety and depression.”

Advice for Patients

Drawing from her personal experiences navigating the healthcare system and her ongoing work supporting the Graves’ disease and thyroid community, Kimberly offers valuable advice to patients:

Know your family history.

“After my diagnosis, I learned my grandmother had a thyroid issue earlier in her life,” Kimberly shares. “I also had a cousin diagnosed with thyroid cancer. Patients struggling to receive a diagnosis should share as much information as they can about family history, even if it seems unrelated. I didn’t know then, but Graves’ disease and other autoimmune disorders tend to cluster in families, so if there is a history of different autoimmune disorders like rheumatoid arthritis, multiple sclerosis, and lupus among your family members, that is important to tell doctors.”

Understand your treatment options, including the risk and benefits of each option.

“Make sure your doctor is thoroughly explaining all treatment options, including their risks and benefits,” Kimberly advises. “Patients who educate themselves early on about their condition and treatment options are better equipped to understand their doctors’ recommendations and to make the decision that is right for them.”

Ensure your information comes from a credible source.

“With countless opinions and alternative treatments promoted online, newly diagnosed patients should consult their doctor or reputable organizations like the GDATF or the American Thyroid Association for reliable information,” Kimberly recommends.

Don’t be afraid to seek help for mental health issues.

“A Graves’ diagnosis can be emotionally challenging. Symptoms often mimic anxiety, depression, or panic disorder,” Kimberly acknowledges. “It’s essential for individuals living with Graves’ disease to seek mental health support if needed, rather than attributing all symptoms to their thyroid. Whether through an experienced counselor or participation in support groups, patients should not hesitate to seek help.”

Resources:

https://gdatf.org/ 

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

For volunteer opportunities with CISCRP, visit our Volunteer page.

Written by Lindsey Elliott, Marketing & Communications Manager, CISCRP | lelliott@ciscrp.org

Medical Hero Spotlight: Noa Greenwood, Canavan Disease Clinical Trial Participant

Canavan Disease Diagnosis

Lee and Lori Greenwood welcomed their daughter Noa in August 2020. Born full-term and seemingly healthy, Noa’s first weeks of life passed without issue. However, at around 8 weeks old, Noa began crying much more than the average baby. Concerned she might have colic or a reflux problem, the Greenwoods took her to the pediatrician. Noa’s doctor determined she did not have colic but couldn’t identify the underlying issue.

Soon after, Lee and Lori noticed that Noa wasn’t responding to lights or movements and worried she had vision loss. “She wasn’t looking at us — she was almost looking through us,” Lee recalls. Around the same time, Noa stopped gaining weight and dropped from the 89th percentile to the 26th percentile in her age group.

Referred to an ophthalmologist, they again found no clear answer to Noa’s symptoms, and she continued to miss developmental milestones.

When she began struggling to swallow, the Greenwoods pushed for a consultation with a neurologist, initially meeting via Zoom. “Noa’s neurologist was wonderful,” Lori says. “She originally suspected hydrocephalus and recommended we bring her into the office. However, I could tell from her reaction to Noa that something else was going on.”

Noa was then scheduled for an MRI, which unfortunately took several months of waiting due to the pandemic. In the meantime, she received early intervention care via Zoom calls with different specialists.

In August 2021, a few weeks before her first birthday, Noa’s MRI appointment revealed that she had Canavan disease, a progressive, fatal, genetic disorder affecting the central nervous system.

“We received the news on Zoom,” Lee recalls. “Lori and I joined a video call having no idea that doctors were about to tell us our beautiful little daughter had a fatal disease with a life expectancy of around 10 years.”
Finding Community Support

After Noa was diagnosed, Lee and Lori began researching the disease, seeking information and support online. They found a patient advocacy organization based in Boston that supports children with Canavan and related diseases. This organization helped connect them to relevant Facebook groups and the Canavan community.

“Everyone has been incredibly supportive. They have all experienced the same diagnosis and understand what it’s like. Being involved in this community and patient advocacy has been immensely helpful for us,” Lori says.
Clinical Trial Referral

For most patients, clinical trial participation is only mentioned by doctors after standard treatment options are exhausted. However, there is currently no cure for Canavan disease and the only treatment is palliative care. For such an ultra-rare disease affecting only 1,000 children across the U.S. and the EU, research studies are hard to come by.

The day Noa was diagnosed with Canavan, a new clinical trial became available at Massachusetts General Hospital in Boston, only 30 minutes from the Greenwood’s home. Noa was immediately referred by her neurologist, and within weeks the family was in contact with the trial coordinator.

“Everything came together in a serendipitous way. The timing was right, the location was so close to us, and Noa met all the eligibility criteria to qualify for the trial, which many kids don’t,” Lee says. “It’s funny to think of yourself as fortunate when facing a disease like Canavan, but we really are.”

Since clinical research was one of the only ways Noa might be successfully treated for Canavan disease, Lee and Lori decided that joining a trial for gene therapy was the best choice for their family.

Starting Gene Therapy

Gene therapy corrects genetic information that causes illness by replacing a faulty or missing gene with a genetically engineered ‘vector’. In recent years, gene therapy has been transformative across many diseases.

Before starting treatment, Noa and her family spent a lot of time at doctors’ appointments and in the hospital for the screening process. Noa had multiple MRIs under anesthesia to ensure she would be suitable to receive this type of treatment.

In June of 2022, when Noa was almost 2 years old, she received treatment in the Canavan trial using gene therapy. Noa spent about a week in the hospital afterward and then took steroids for about six months. She continues to be monitored and has regular check-ins with her care team.

Patient Advocacy Work

Before Noa began the trial treatment for Canavan disease, Lee and Lori already understood the complexities of navigating clinical trials and the healthcare system through research and their backgrounds.

“Clinical trials can be complicated and intimidating for patients and their families, especially when there’s a power dynamic between patients and doctors that can make asking questions difficult. Many people, particularly in underserved communities, face additional barriers like language and cultural differences that we don’t,” Lori explains.

Since Noa’s participation in clinical research began, the Greenwoods have made it their mission to raise awareness for Canavan disease and empower other patients to ask questions and advocate for themselves.

“By sharing our experiences, we hope to help others feel more confident in navigating the medical system. We’re fortunate to have a supportive care team and feel a responsibility to share our journey to benefit others, not just Canavan families but anyone involved in the medical field. By sharing our story, we aim to remind those working in research of the real people they are helping,” Lee says.

“It’s important to remind researchers that when they are conducting trials, they are changing the lives of real patients. You’re not just working to treat Canavan disease; you’re working to help kids like Noa.”
Life Today for Noa & Her Family

Today, Noa is currently in the second year of the five-year clinical trial, and for the Greenwood family, the results have been incredible.

“When we found out Noa had Canavan, we never imagined that her life could be the way it is now,” Lori says. “We are so proud of her!”

Today, Noa’s parents describe her as a sweet and affectionate toddler who can walk independently, is learning her ABCs and how to count, and goes to public school every day with other children her age. Just two years ago, Canavan disease would have made these milestones unreachable for Noa.

The Greenwoods share that they are proud of Noa’s participation in the research that may one day cure Canavan’s disease, even if her treatment today is only the first step.

“We have had an amazing experience in the clinical trial with Noa so far, but we understand the nature of this disease. There is so much uncertainty —  her progress could reverse unexpectedly at any time,” Lee notes.
“But through this experience, we have gotten so much better at learning to embrace the current moment as parents. Regardless of what happens in the future, no one can take away the joy I’ve had seeing Noa grow and thrive the last  two years, which wouldn’t have been possible without the trial.”

Additional Resources:

https://www.umassmed.edu/news/news-archives/2023/07/family-connects-with-researchers-behind-canavan-gene-therapy/

https://www.canavan.org/

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

For volunteer opportunities with CISCRP, visit our Volunteer page.

Written by Lindsey Elliott, Marketing & Communications Manager, CISCRP | lelliott@ciscrp.org

Medical Hero Spotlight: Michael Herman, Multiple Myeloma Clinical Trial Participant & Patient Advocate

Abruptly Diagnosed: Facing Multiple Myeloma

In the Fall of 2012, Mike Herman and his wife Angela were taking their daily walk with their dogs through the neighborhood. When Mike dropped his glasses and bent down to pick them up, he felt a sharp pain, much like walking into the corner of a counter. He assumed it may have been a pulled muscle, and tentatively moved on until it happened again a couple weeks later. This time, Mike was at work and had gone to pick some files up off the floor when the same pain started again. Although he didn’t know it at the time, the pain was caused by multiple micro-fractures.

At the doctor’s office, Mike was referred to a hematologist who believed he had a vitamin B12 deficiency and recommended he go home and take some supplements. “I was so lucky that day to have my wife with me who is a nurse and has a lot of experience navigating the healthcare system,” Mike recalls. “Angela insisted it was not a B12 deficiency and said we would not be leaving with that diagnosis, which was what finally pushed them to order a bone survey. That same afternoon, I was X-rayed from head to toe, which is how the microfractures were found.”

About a week later, Mike noticed an email in his medical portal with test results. He assumed this was good news about his health issues and never imagined he would receive a diagnosis without a phone call.

“I opened the message and must have read it ten times. I thought I must have been missing something, so I called my wife and read it to her,” Mike says. “When she hesitated, I realized that this was real and that I had cancer.”
Finding the Right Doctor

After receiving his diagnosis for multiple myeloma, Mike’s doctors started him on the standard treatment of chemotherapy.

“At that time, the only thought in my head was the fear of dying. I had been told that the life expectancy with this cancer was four years, and only three months prior my first granddaughter had been born. I was devastated that I wouldn’t be there to see her grow up,” Mike says. “Other options for treatment or clinical trials weren’t brought up by my doctors, so I didn’t know it was even possible for me.”

During the early days of treatment, Mike had the opportunity to change health insurance providers, which gave him much more flexibility in finding specialists for his cancer. Through research, the couple found Dr. Bart Barlogie, who had founded the UAMS Myeloma Center in Arkansas. Mike made the difficult decision to temporarily move to Arkansas and receive treatment at the center.

“I was down there for nine months and during that time I had two stem cell transplants and tried a couple different treatments,” Mike says. “My cancer wasn’t going into remission yet, but I was starting to feel a lot better, which is when I started becoming close with my doctor and appreciating his way of treating patients.”

Mike’s doctor believed in an individualized treatment approach, something that had been missing from his care when he was first diagnosed. “I highly value his feedback and we developed a great friendship. He welcomed me to ask questions and get second opinions from other doctors, which is so important when you are dealing with cancer. Any doctor who discourages you from getting another opinion does not have your best interest in mind,” Mike says.

Mike and Dr. Barlogie discussed his treatment, and Dr. Barlogie encouraged Mike to consider clinical trial participation. After Dr. Barlogie retired, Mike, having tried nearly every publicly available myeloma drug, sought new options, and opted for clinical research.

“I knew from my wife’s work in clinical research that these studies are essential to developing new medicines. If I could, I wanted give back to other people with myeloma who might need this medicine in the future. For me, clinical trials felt like the right thing to do.”
Clinical Trial Participation & Success

Mike has been in two clinical trials at the University of Pennsylvania, both with incredible results. The first study was in 2020 for a treatment called Teclistamab, sponsored by Johnson & Johnson Innovative Medicine. The trial tested two different ways of administering the treatment, and Mike received his subcutaneously, as a shot into his stomach.

“My doctors said results may take a bit longer this way, so I wasn’t expecting anything immediate. It was only the first mini dose,” Mike explains. However, within 48 hours, Mike was in excruciating pain to the point where he could barely speak. During this, he received a phone call from his doctor who let him know that his cancer levels had dropped 99%. The pain Mike had been in was caused by something called cytokine release syndrome, and while difficult to endure, meant that the drug was effective for him.

“I wish I had known about it before. Maybe anticipating the pain would have made it easier to get through,” Mike reflects.

After finishing up with Teclistamab, Mike’s cancer was undetectable for 18 months. However, because there is currently no cure for multiple myeloma, the cancer tends to have a recurrence. In 2022 when his cancer returned, Mike signed up for another clinical trial run by Genentech for a treatment called Cevostamab. Within two weeks of starting the trial, Mike’s cancer was in total remission and has remained undetectable since.

Patient Advocacy Work

Recently, the drug Teclistamab was approved by the FDA and is now available to multiple myeloma patients as Tecvayli. “It’s incredible knowing that my participation in a study contributed to a new treatment for cancer,” Mike notes. “My experience has driven me to become more involved with patient advocacy, so I can share my story with others and be the impetus for significant change in our broken healthcare system.”

Over the last decade, Mike has spoken at various events and conferences about his experience and the importance of clinical research participation.

“In your lifetime, you will likely either be diagnosed with cancer, or have a loved one who is diagnosed. I don’t want people to feel frozen by fear when it happens like I did at first. Having the resources and knowledge you need ahead of time can change that experience and outcome entirely,” Mike says. 

Together, Mike and Angela founded Speaking on Cancer Patient Advocacy (SoCPA), a non-profit with a mission to empower cancer patients and improve their treatment outcomes. The website includes helpful resources for individuals who have just been diagnosed, including guides to reading lab results, and education about different types of cancer.

Looking ahead, Mike is excited about some upcoming projects SoCPA has planned to help promote cancer awareness and education in larger companies and health care systems. His aim is to encourage companies to invest more in employee health and education, ultimately creating an environment where colleagues are comfortable navigating a cancer diagnosis in the workplace and know what their treatment options are.

“Being involved in patient advocacy and meeting such wonderful people has kept me motivated to work even harder.”

Additional Resources:

https://www.socpanow.com/index

https://themmrf.org/multiple-myeloma/

https.myeloma.org  

 

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

For volunteer opportunities with CISCRP, visit our Volunteer page.

Written by Lindsey Elliott, Marketing & Communications Manager, CISCRP | lelliott@ciscrp.org

Latest CISCRP Patient Survey Reveals Diversity Gaps, Yields 5 Tips For Improvement

Despite the FDA introducing a draft guidance document two years ago that outlined strategies to improve minority representation in clinical trials,4 recent studies suggest that racial and ethnic minority populations continue to remain underrepresented across the board, from ophthalmology to oncology studies.5-7 More effort is needed to understand the views of underserved groups, such as ethnic and racial minorities, toward clinical research to improve their enrollment and participation.1

Because it is also important to periodically reassess the values, perceptions, and barriers that may preclude clinical trial participation among potential trial volunteers, the Center for Information and Study on Clinical Research Participation (CISCRP), an independent nonprofit organization in Boston, conducts a biennial global survey called the “Perceptions & Insights” study, assessing public and patient views and experiences in clinical research. The CISCRP 2023 study8 offered valuable information on participant recruitment, improvement of trust and diversity, and optimized technology use as well as adaptations to post-COVID changes in clinical research. By analyzing these findings, we help provide pharmaceutical companies, CROs, regulators, and other clinical leaders with insights and strategies to improve clinical research inclusivity, efficiency, and engagement.

Here, we outline five such opportunities to improve clinical trial recruitment and participation, especially among ethnic and racial minority groups:

1. Increase trial access through HCPS and other trusted information sources.

The foundation of successful clinical trial recruitment and retention lies in the trust, reliability, and accessibility of information sources. For pharmaceutical companies, this underscores the critical importance of prioritizing transparency and actively engaging with patients to make it easier to participate in clinical research studies. By openly sharing information on the risks and benefits of treatment in a language accessible and understandable to patients, study sponsors can significantly enhance public trust.

The outcomes of our 2023 survey further underscored the critical role of physicians and HCPs in this area. We found that HCPs continue to be the most trusted source of information regarding clinical trials. In 2023, an encouraging 23% of potential participants (20% among Hispanic respondents compared to 23% among non-Hispanic respondents; 16% among Black or African Americans compared to 24% among White respondents) reported being asked by their HCPs to consider trial participation, a significant increase from 14% in 2021. While this trend highlights the pivotal role of HCPs, it is merely a starting point toward a more personalized, trust-based approach to engaging with potential trial participants, as our study indicated that the majority (66%) still do not consider clinical research as an option when discussing treatments with their doctor.

Pharmaceutical companies and stakeholders should prioritize leveraging trusted sources of information to build trust and facilitate access to clinical trials. This might mean collaborating more closely with physicians and HCPs — particularly those practicing in minority community settings — to actively strengthen educational efforts, initiatives, and feedback loops between HCPs and trial sponsors. For example, this could include creating clear, accessible content that HCPs can easily review and share with potential participants, providing HCPs with e-learning modules on the latest trial protocols, or easy-to-access online portals that could feature regular updates on upcoming trial recruitment, progress, and outcomes on ongoing or completed trials.

Likewise, patient advocacy and support groups also play a pivotal role, especially among minority communities, with email invitations proving to be the most effective recruitment method. Patient advocacy groups proved to be especially successful in increasing clinical research engagement among Black respondents (12% reported being asked to participate by an advocacy group) compared to White respondents (6% reporting the same), highlighting the importance of leveraging these groups for broader, culturally sensitive trial outreach.

2. Increasing diversity and building trust with patients is mutually beneficial.

The diversity of trial participants is a critical factor that directly influences the trust and relevance of pharmaceutical companies among diverse populations, particularly in light of historical and current injustices in clinical research. The CISCRP study showed that trust in pharmaceutical companies among Black respondents (32%) was more likely to be increased if a company demonstrated workforce diversity. This proportion further increased to 52% when trials included a diverse set of participants.

These findings further reinforce the importance of sponsors to consider targeted and inclusive strategies, which may involve a combination of:

  • implementing policies and practices aimed at recruiting a diverse workforce,
  • ensuring clinical trial participant pools reflect the demographic diversity of the population affected by the condition, and
  • actively engaging with ethnic and racial minority groups and community leaders through direct outreach programs, employing culturally sensitive and language-appropriate materials and communication strategies to effectively engage potential participants.
3. Expand accessibility through alternative site models.

Venturing beyond the traditional clinical trial setting also can offer unique opportunities to enhance the convenience of participation and significantly improve the diversity of trial participants at the same time. According to our study, 46% of participants overall were very willing and 38% were somewhat willing to go to a local pharmacy for their study visits. This trend also was observed among various demographic groups (percent responses of “very willing”: Black: 53%, White: 48%, Hispanic: 44%, Asian 34%).

A separate CISCRP survey performed 10 years ago10 highlighted the benefits of pharmacists directly engaging with patients and providing education about clinical trials. The concept of using pharmacies as alternative sites for studies gained traction, especially when CVS and Walgreens launched their clinical trials initiatives in the early 2020s.11 Despite the noted participant openness, however, 15% of our study respondents were still not very, or not at all, willing to consider a pharmacy site, citing concerns with the facilities/lack of privacy, confidentiality of information, quality of care, and quality of answers to questions.

Outside of the traditional clinical site model, respondents were receptive to three different clinical study models (i.e., hybrid, mobile nurse, and remote) and provided high marks for “very willing to participate” — 43%, 44%, and 43% respectively for each model. Furthermore, having access to some trial site visits closer to home or having some visits conducted virtually were both highly chosen factors for long-term retention, at 66% and 58%, respectively.

CROs and pharmaceutical companies are encouraged to form partnerships with pharmacies and other local health facilities to develop innovative alternative trial sites and clinical study models. Sponsors, however, should carefully establish standardized protocols for alternative clinical sites that prioritize participant convenience and care, ensure appropriate staffing and staff training, and communicate where participant information is securely managed.

 

4. Leverage technology for optimized recruitment, retention, and enhanced participant experience.

The integration of technology in clinical trials is yet another important factor for improving recruitment and diversity. In our study, 34% noted it was very important to use mobile applications, while 32% expressed interest in using eConsent. Technology adoption was notably higher among Hispanic and Black respondent subgroups, and both groups were more likely to cite the importance of mobile app availability and the ability to review and sign documents in an electronic format compared to non-Hispanic and White subgroups. Additionally, 66% of respondents were comfortable using their personal computers for clinical trial data entry, 61% with wearable devices and clinical apps, and 58% with video conferencing with physicians. Text messaging emerged as another helpful tool, with 65% of respondents indicating its usefulness. The use of text messaging in patient communications also has increased since 2021 (22% compared to 29% in 2023). While technology offers significant benefits, some concerns were again noted, with 18% of respondents indicating that smartphone or tablet use during studies disrupted their daily routines.

Pharmaceutical companies might consider focusing on creating secure, intuitive, and user-friendly mobile applications that serve as one-stop portals for trial information, consent, and data collection. Seeking direct input from trial participants and pilot testing the initiative with ethnic and racially diverse user groups before launching these programs will help ensure that these technologies meet the needs of diverse communities. Using wearable devices, text messaging, smartphone apps, and video conferencing may also help to maintain continuous engagement with trial participants and help improve remote monitoring and communication.

5. Retain post-COVID-19 progress made in clinical trials.

Despite its challenges, the COVID-19 pandemic significantly accelerated the use of technology and innovative trial models between 2020 and 2022, and many industry professionals were hopeful about the lasting positive effects of these technological advancements. However, when we compared our 2023 study findings with the data from 2019 and 2021, we noticed a shift to pre-pandemic practices, including lesser use of eConsent forms and reduced participant communication during and after the trial. For example, from 2021 to 2023, there was a decrease of 12% for eConsent usage and 9% for video consent usage.

Pharmaceutical companies should continue to stay agile and adaptive in response to evolving trends and challenges while maintaining the momentum toward more flexible, technology-driven trial models. One such strategy may involve commitment to the #NoGoingBack initiative12, ensuring that innovations and efficiencies gained during the pandemic are not lost but, rather, built upon. Emphasizing clear communication during and post-trial, timely feedback, and quality of care in clinical trials may perhaps help. This includes continuing to leverage virtual visits, remote monitoring, and digital consent processes, and ensuring these practices solidify in the clinical trial landscape.

Putting It All Together

By enhancing trust and diversity, expanding access to alternative clinical sites, and leveraging technology use, the pharmaceutical industry and stakeholders may drive significant advancements in clinical research and move toward a more inclusive and participant-friendly future. We propose several strategies that, if adopted, would help improve access and deepen engagement in clinical trials, especially among ethnic and racially underrepresented communities.

About the CISCRP 2023 Perceptions & Insights Study

The CISCRP 2023 Perceptions & Insights Study provides a global overview of current trends, challenges, and opportunities within the clinical trial industry from a patient and public perspective. The questionnaire was developed with input from a cross-functional workgroup — including representatives from pharmaceutical and biotechnology companies, patient advocacy groups, and CROs. It was distributed online from April – June 2023 with support from Clariness, James Lind Care, Benchmark Research, and Rare Patient Voice and resulted in responses from 12,017 participants. This article does not capture in totality the insights derived from the study; however, you may access additional resources here: https://www.ciscrp.org/results-from-ciscrps-2023-perception-insights-study/. Also of note, a peer-reviewed article that takes a deeper dive into the diversity insights captured by the survey respondents is forthcoming.

By Annick de Bruin, CISCRP

References:

  1. Raven-Gregg T, Shepherd V. Exploring the inclusion of under-served groups in trials methodology research: an example from ethnic minority populations’ views on deferred consent. Trials. 2021;22(1):589. doi:10.1186/s13063-021-05568-z
  2. MacLennan DL, Plahovinsak JL, MacLennan RJ, Jones CT. Clinical Trial Site Perspectives and Practices on Study Participant Diversity and Inclusion. Clin Pharmacol Ther. 2023;113(3):670-679. doi:10.1002/cpt.2817
  3. Corneli A, Hanlen-Rosado E, McKenna K, et al. Enhancing Diversity and Inclusion in Clinical Trials. Clin Pharmacol Ther. 2023;113(3):489-499. doi:10.1002/cpt.2819
  4. Russell ES, Aubrun E, Moga DC, et al. FDA draft guidance to improve clinical trial diversity: Opportunities for pharmacoepidemiology. J Clin Transl Sci. 7(1):e101. doi:10.1017/cts.2023.515
  5. Bains A, Osathanugrah P, Sanjiv N, et al. Diverse Research Teams and Underrepresented Groups in Clinical Studies. JAMA Ophthalmol. 2023;141(11):1037-1044. doi:10.1001/jamaophthalmol.2023.4638
  6. Aldrighetti CM, Niemierko A, Van Allen E, Willers H, Kamran SC. Racial and Ethnic Disparities Among Participants in Precision Oncology Clinical Studies. JAMA Netw Open. 2021;4(11):e2133205. doi:10.1001/jamanetworkopen.2021.33205
  7. O’Donoghue J, Luther J, Hoque S, et al. Strategies to improve the recruitment and retention of underserved children and families in clinical trials: A case example of a school-supervised asthma therapy pilot. Contemp Clin Trials. 2022;120:106884. doi:10.1016/j.cct.2022.106884
  8. CISCRP 2023 Perceptions and Insights Study. Accessed February 2024. https://www.ciscrp.org/services/research-services/perceptions-and-insights-study/.
  9. CISCRP. Journey to Better Health Mobile Exhibit. Bringing Clinical Research Information to Local Communities. Accessed February 2024. https://www.ciscrp.org/mobileexhibit2023/
  10. Getz, Kenneth; Leveraging Pharmacists as a Channel to Raise Clinical Research Literacy Among Patient Communities. Applied Clinical Trials, Vol. 22, Issue 10. October 2013. Accessed February 2024. https://www.appliedclinicaltrialsonline.com/view/leveraging-pharmacists-channel-raise-clinical-research-literacy-among-patient-communities
  11. Natalia Mesa. “Walgreens Launches Partnership with Prothena to Compete with CVS, Walmart” May 01, 2023. Biospace. Accessed February 2024. https://www.biospace.com/article/walgreens-launches-partnership-with-prothena-to-compete-with-cvs-walmart/
  12. #NoGoingBack. Accessed February 2024. https://www.nogoingback.health/.

Round-Up: Highlighting Recent Health Literacy Educational Materials

What is a placebo, Eligibility criteria, and How to find a clinical trial 

CISCRP is dedicated to providing educational resources and empowering people through all steps of clinical research participation. This includes everything from general awareness to what to expect during a trial. Or, as with one of our newer brochures, how to actually find a trial. We recently published three resources to help people understand certain parts about trials. 

Especially during Health Literacy Month in October (but also all the time), we can’t emphasize enough the importance of providing health-related education and tools to patients and the public. We’re constantly reviewing our materials and getting feedback from the public so we can fix any gaps in our resources. The ultimate goal is to provide guidance to help with decision making. To do this, we provide details, such as potential risks and benefits, clear action items, and a summary of possible options. And we strive to do this without actively encouraging participation and encouraging the reader to make the best decision for themselves and their loved ones. 

Each resource CISCRP creates, such as brochures, goes through a thorough review process to ensure the readers’ needs and the above goals are met. This includes writing, several editing steps, getting feedback from a Review Panel and surveys, getting internal feedback, and approval from an IRB. 

What is a Placebo?

If you asked a member of the public to pick the first word that comes to mind when they think “clinical research,” there’s a good chance they would say placebo. And then if you asked them to define it, they may say something like, “It’s just a sugar pill.” Since there’s a lack of awareness about clinical trials and how they work outside of CISCRP, that’s a fair guess. But it’s much more complicated than that. To help clear up the confusion for patients and the public, we updated our What is a Placebo? brochure.

The idea of a placebo has many parts. There’s why it’s used, how it’s used, and the “placebo effect,” for starters. There’s also the concern of not knowing whether or not a participant might receive a placebo. The brochure explains why it’s important that participants don’t know whether or not they receive a placebo, and why they can’t choose their treatment. It also explains how to know whether or not a placebo is even a possibility.

Then there’s the issue of randomization and blinding. How do researchers determine who receives a placebo? How does anyone know who gets what? Because many readers prefer visual mediums, we created a graphic structured a bit like a flow chart that shows treatment being randomized and that the participants and trial doctors usually don’t know who’s receiving what.

Eligibility Criteria for Clinical Trials

 

Our team—specifically, our events team that directly engages with different communities—heard from many different people at events that patients who want to join a trial are confused if they are told that they cannot join. Some people feel offended or upset, thinking that they were denied because of personal reasons. However, they may not be aware of eligibility criteria. So, we created an Eligibility Criteria for Clinical Trials infographic explaining the basics.

The immediate question many readers might have is why does it even matter? As mentioned in our graphic, eligibility criteria are necessary to protect participants’ safety and to get the most accurate results possible. Hopefully this explanation helps readers understand why researchers can allow only certain participants to join.

It’s vague to simply say “there are specific reasons why you can or can’t join,” so we provided some examples of both inclusion and exclusion criteria. This ranges from the most obvious, such as having the condition a treatment is designed to help, to other factors someone may not even think about, like needing to be a certain weight to join, or not being able to join because of having previous treatments that would affect how the trial treatment would work.

The infographic also provides patients who do not qualify or who did not want to participate with other ways to get involved and contribute to research. For example, participating in an observational study, joining an advisory board, or even simply discussing with friends and family.

And, of course, there may be other trials they can join.

How to Find a Clinical Trial

We have lots of information about clinical research participation, including helping to decide if participation is right for you (see our brochure, Should I Participate?). But to even get to that stage, a person needs to know How to Find a Clinical Trial. Some patients may think that their doctors will tell them about any trials they might be able to join, but that is not always true.

The process is not as simple as just finding a trial, though. As we explain in the brochure, there are multiple steps beyond simply finding a trial and joining. Before someone even starts looking, a patient should understand their condition to help identify which trials might be best for them.

As with anything in today’s world, looking online is a great place to start looking for a trial, so we provided three main clinical trial search sites based on location. We recommend putting together a list of trials that interest a potential participant. Then we suggest putting together a list of questions for trial staff, and then contacting them.

This is only the first step of deciding whether to join or not.

Conclusion

Clinical research participation is a complicated process, from thinking about joining, to figuring out if you should join, and then actual participation. We’ve created the three resources listed above to help people in all steps of the process, but we have a lot more in our Resource center that support patients, caregivers, and others across all parts of research.

One of CISCRP’s missions is to partner with patients and the public. This includes working together with patients, community members, and healthcare professionals to create our brochures, infographics, and other materials. We are always open to feedback on our materials or any other resources we should make. Feel free to contact us!

Written by: Scott Finger

Community Trust: The Foundation for Fostering Diversity in Clinical Trials

Featured Article in our October 2023 Patient Diversity Campaign

As an industry, we must recognize and address a complex problem: racial and ethnic minority populations have historically been underrepresented in clinical trials. Over time, it has become widely recognized that this issue is a systemic problem, not a participant one.

What does that mean? It means there have been limitations in clinical research that aren’t isolated, but deeply embedded in our processes and systems. These limitations can include anything from protocol complexity to a lack of diversity in the clinical research field, which can create barriers to ensuring participation. This can have long-term implications for equitable access to medicines.

We’re making progress. In the past few years, Merck has increased participant diversity in our trials. Much of our progress is thanks to our community collaborators, whose engagement is vital in helping us understand and solve the complex issues at play.

Written by: LaShanda Gordon

Defining community collaborations

For me, community is synonymous with home. I grew up in Selma, Alabama. Many consider it the seat of the Civil Rights movement, but it is also the small southern town that taught me the power and importance of community.

At Merck, I’m a Diversity Program Lead for Clinical Trials, and part of my job is working directly with community-based organizations and leaders to provide valuable resources about clinical research. I also help our company and communities make connections to try to improve trial access. An important step for increasing clinical trial participant diversity is helping to ensure everyone can make informed decisions about their health, no matter who they are or where they live. That starts with education.

Over my years in the industry, I’ve learned something valuable from every person, project, and community. Some of my takeaways include:

  • Learning never ends. Every engagement helps improve our understanding of how an organization operates, the unique needs of a particular community, or what approach would be most impactful. We need to listen.
  • Start with the community-based organizations. They are the most knowledgeable about what their community needs. In addition, they are connectors and can be a bridge to valuable resources, insights, and additional partners.
  • Each community is unique, with its own set of challenges and strengths. For example, the needs of an African American community in the Bronx are different from one in rural Alabama – we cannot retrofit strategies from one to the next. It is important to listen and learn about the particular needs of the community.

Building trust

Being a good collaborator starts with trust. Without that, we can’t build a path forward. It’s the foundation to everything else, but it can be the hardest part to get right.

I’ve found the best way to earn trust is by listening. We want to ensure our partners feel heard – especially because we use insights from them to build our approach. It’s also important to ensure we’re not guided by preconceived ideas about what they need. If you ask what needs to be done, the community will tell you.


At Merck, we recently established a U.S. Community Advisory Panel, with patients, caregivers, health care providers, and community members from diverse backgrounds who – along with our existing Patient Advisory Panel – share insights that help us incorporate the patient perspective in our site and patient engagement methods. We listen to them, and we learn a lot.


The best part is that we are seeking their guidance early, when it can have the greatest impact. For example, they are helping us design our protocols to be patient-friendly by reviewing the number of in-person site visits and inclusion/exclusion criteria for trials. That way, we’re able to address potential barriers as early as possible in the process.

Putting people back into collaborations

Too often, we forget that collaborations are about relationships, and those are built between people who trust each other. People are also the focus of our trials.

When we make our efforts more about people, we must also think bigger than trials and focus on wraparound care like disease education and screenings. There are still many who are not aware that they may be eligible for a clinical trial. It’s important that we prioritize education and awareness, long before there is a study to enroll in.
We can only earn trust as a collaborator in the continuum of a community’s health care. Our support can’t be exclusive to one-off projects. We must be present and active in what we help build.
Most importantly, we must remember the thread that runs through it all, the real definition of the word community: people.

Authored by: LaShanda Gordon

A “Day in the Life” of a Medical Writer at CISCRP

It is Monday morning at the CISCRP office, and I open my computer a few minutes before 9 AM and prepare myself for a new week. I normally work from home on Mondays, but a few colleagues who also often work from home were planning to come into the office in downtown Boston today, and I wanted to join them.

As a Medical Writer, I write about clinical trials. Every clinical trial is an incredible achievement, and one that involves collaboration between experts in many areas. They also could not happen without the curiosity and bravery of clinical trial participants, and their steadfast belief in the medical breakthroughs of the future. My job is to tell the story of clinical trials in plain language, with a focus on the big picture. What happened during the trial, and why? What did we learn from the trial? What do we still need to learn?

Our mission at CISCRP is to empower people to make informed decisions about their health and about their participation in clinical research. And this mission is personal. In addition to a Medical Writer, I am also sometimes a patient. As a patient, I know first-hand that it is very difficult to access, let alone interpret the results of clinical research. While doctors and other trusted people can help, navigating the flood of health information online, in news stories, and in advertising is overwhelming. It can be tempting to tune it all out. Yet, it is vital to understand how the newest clinical research impacts our own health care, and to be able to weigh the benefits and risks of participating in clinical research. To do so, we need access to clear, accurate, and unbiased information about clinical trials.

As I ease into the day, I check the stage of each project I am working on. One of my favorite things about this job is the opportunity to learn about many different areas of clinical research through the range of projects I work on. I check my inbox and see an email from one of our editors, who has just reviewed a Plain Language Summary, or PLS, that I had previously drafted. A PLS is a summary of clinical trial results that the trial sponsors distribute to the trial participants and sometimes publish on their website. It takes me about half an hour to go through the editor’s comments in the PLS and make the necessary changes. I send it back to the editor so they can review it. If the editor decides the document is ready, they will give their approval to send it to our graphic designer. During this step, the graphic designer creates visualizations that incorporate details about the trial design and results. This helps make the information easier to read and understand.

The writers, editors, and graphic designers at CISCRP work together to maintain consistency across different summaries, avoid unnecessary jargon that might be confusing, and keep the language and visuals engaging and approachable. We have honed our craft in large part due to feedback from Review Panels on our materials. Review Panels are composed of volunteer patients, patient advocates, and members of the public who give feedback on every clinical trial communication that CISCRP works on. Review Panels continually give us ideas and suggestions for how to make our materials as clear and engaging as possible. With everything we create, the perspectives of patients are always top-of-mind.

Next on my schedule is a “kick-off” meeting with a team from the sponsor of the trial about another PLS project that is getting started. This team from the sponsor may consist of patient engagement officers, trial physicians, and statisticians, who will all help in the planning and drafting process. A Project Manager from CISCRP leads off the meeting, which involves explaining and planning the drafting process and timeline. As we move forward with drafting, the Project Manager will ensure that everyone is moving together and that the project is on time. Next, I present an outline of the PLS itself that I previously prepared for the meeting. Preparing the outline involved going through the Clinical Study Report and other source documents provided by the sponsor, familiarizing myself with the trial, and identifying key pieces of efficacy and safety data to include in the PLS. In the meeting, we discuss the outline, go over any questions, and ensure that everyone is happy with the plan for drafting.

Many of the clinical trial materials CISCRP creates are done in collaboration with, and funded by, the trial sponsors. Sponsors, which are often pharmaceutical companies, are realizing increasingly that patient engagement is important for their success. In addition, the European Union Clinical Trials Regulation Annex V will require that a PLS be written for each clinical trial done within European Union (EU) nations. The EU is also requesting plain-language Protocol Synopses to be written at the beginning of trials, which CISCRP also helps to prepare. This shift towards patient engagement and plain language communications is a positive step forward towards improving literacy about health and clinical research, and I am proud to be a part of it.

After the kick-off meeting, I write down some notes about our discussion while it is still fresh on my mind. Next, I switch gears to another PLS. Another writer drafted this one, and I will be performing quality control, or QC. Given the complexity of clinical trials and the volume of clinical trial documents, it is essential to have multiple sets of eyes reviewing the draft throughout the process. My job now will be to verify every piece of data and every detail about the study design, and to otherwise “fact-check” the document. QC is a rewarding process for all involved. I enjoy getting a “crash-course” in a specific clinical trial while performing a QC. The results and design of clinical trials can be fascinating. From the other perspective, I am always grateful to receive QC results from another writer, as it provides additional confidence that we are creating accurate and high-quality materials.

I finish the QC right at lunch time. The weather is warm, so we head outside to a nearby plaza to enjoy some lunch, get to know our new colleagues, and hear stories about each other’s weekends.

Once back inside, I decide to spend the rest of the afternoon drafting a Plain Language Summary of Publication, or PLSP. Like PLSs, PLSPs are summaries of clinical trial results. However, rather than using the lengthy documents from the trial sponsors as the source material, they use trial results that have been published in an academic journal. Like the publication it is based on, the PLSP will ultimately go through a peer review process and be published in a journal. Compared to PLSs, PLSPs are often heavier on graphics, which means they typically involve close collaboration between the writer and graphic designer. I take time during the afternoon to experiment with different ways of explaining key concepts and presenting the trial results. I use PowerPoint to make “sketches” that our graphic designer will, in turn, use as inspiration to create the visualizations. There are no government regulations to define the scope of PLSPs like there are for PLSs, which leaves more space for creativity. I enjoy this freedom, and believe it is necessary to continue improving and innovating in our work.

By 5 PM, I am happy with my progress on the PLSP draft. I pack up my laptop and say goodbye to my colleagues. I reflect on the day as I head for the elevators.

Written by: Sam Entwisle

Plain Language Protocol Synopsis 101

What is a Plain Language Protocol Synopsis (PLPS)?

A clinical study protocol (CSP) is a comprehensive document that outlines important aspects of a clinical trial including the goals of a trial, the trial design, and more. A protocol synopsis is a summary of the CSP, but it still contains highly complex scientific terms, acronyms, and processes not readily understandable to a non-expert. A protocol synopsis written in plain language that can be understood by a wider, non-expert audience can be a beneficial resource for ethics committees, site staff, and potential trial participants. 

Additionally, Annex 1, D.24 of the European Union’s Clinical Trials Regulation (EU CTR) 536/2014 outlines that for future clinical trial applications, “The protocol shall be accompanied by a synopsis of the protocol.” The specific content to be included in the protocol synopsis is further outlined in Question 5.8 of the EU CTR Questions & Answers document. Some of this information is included below as a reference (taken from version 6.4, dated February 2023).

5.8 Question: What should be included in the protocol synopsis described in Annex I, D.24 ? 238.

Answer: Sponsors should include the information below in the protocol synopsis (maximum two pages) to be submitted with the clinical trial application according to Annex I D24 … Sponsors should consider making the synopsis understandable to a layperson. 

Further specified are the nine requirements from the clinical study protocol (CSP) that must be included in this two-page protocol synopsis. These nine requirements are:

  1. Trial title
  2. Rationale
  3. Objectives
  4. Primary Endpoints
  5. Secondary Endpoints
  6. Population
  7. Trial design
  8. Interventions
  9. Risks and Benefits

CISCRP’s Health Communications team was thrilled to learn about this new type of deliverable because we specialize in, and are passionate about, creating easy-to-understand plain language documents. With the implementation of the EU CTR in January 2022, sponsors have increasingly shown interest in developing plain language protocol synopses with CISCRP to be included as part of their clinical trial applications.

When conceptualizing our version of the plain language protocol synopsis, a primary goal became to make a graphically designed template that would include all the nine required elements from the regulation, be written in plain language, and remain under the two-page limit. After we had the template, we were able to put it to work for several trials.

What We’ve Learned from Writing Plain Language Protocol Synopses

Some challenges become immediately apparent when writing a plain language protocol synopsis under the guidelines set out by EU CTR. The first challenge is the two-page limit. Especially since plain language often requires more room to explain complex terms, and health literacy best practices require ample white space.

Additionally, from a more technical standpoint, tables have proven to be an essential asset for presenting the objectives, endpoints, and trial design. A table helps improve the readability in specific scenarios such as aligning a singular objective with multiple endpoints. For trial design, having a table that outlines the different treatment arms, medications, doses, administrations, and duration serves as a great alternative to piling all that information into a paragraph.

We have also compiled a robust glossary of plain language terms from our years of experience writing and user-testing plain language summaries. When writing a PLPS, we can utilize terms from this glossary to increase efficiency when drafting and increase consistency among all plain language documents for a specific sponsor. This consistency helps patients who may read a PLPS, informed consent form, and/or a trial results summary over the course of a given trial. It also helps sponsors by speeding up the process and the amount of work needed to create these documents.

The second and more elusive challenge is that, as a new deliverable, the target audience has not yet been clearly defined. In the meantime, we are trying to give it the broadest appeal possible. We take special care to explain as much as possible for a layperson audience while still making a document that can be utilized efficiently in an industry or medical setting.  

Another tip that we recommend is utilizing graphics and color coding as much as possible. Applying colors to different treatment groups in a table can clear up what specific treatments, doses, or activities apply to that group, without adding any words. Also, using icons can increase readability and provide a visual break in an otherwise text-heavy document.

One final consideration is an optional third page glossary to define any number of the complex terms that were included in the two-page PLPS. This would not necessarily be for submission purposes but could be used by the clinical trial staff to facilitate conversations with potential patients. The PLPS has the potential to be a multi-purpose document and provide value beyond the clinical trial application process.

Should Sponsors Add a Plain Language Protocol Synopsis to Future Clinical Trial Applications?

Based on the regulation, it seems that some form of a protocol synopsis is required. As far as the plain language part, it seems some sponsors are waiting to see what others are doing and how this rule is enforced, while others are making plain language protocol synopses a priority.

Here at CISCRP, we think the more plain language, the better. We believe engaging a wider audience in the clinical research process is an important and worthwhile endeavor. So, when the time comes to start writing your protocol synopses, why not take the next step and make them as accessible as possible to all audiences?

Written by: Zack Fey

References:

EU CTR 536/2014 Q&A p. 54-55

EU CTR 536/2014 p. 58

https://health.ec.europa.eu/medicinal-products/clinical-trials/clinical-trials-regulation-eu-no-5362014_en