AWARE for All – Atlanta Event Overview

On November 16-18, 2021, CISCRP hosted the AWARE for All – Atlanta virtual event, the fifth and final event in 2021. With a different format from previous events, AWARE for All – Atlanta was held over several nights as a series of webinars to offer more clinical research information and resources. This event was city-based, tailored to the Atlanta, Georgia community and featured clinical trial participants, healthcare professionals, and exhibiting organizations.

 

The event’s first night focused on sharing an overview of the clinical trial process with attendees. Rabih Dahdouh, Clinical Research Associate of the Center for Health Equity Transformation, explained what clinical trials are, how trials are conducted, why people join, the important role of clinical trials, and how clinical trial participants help to advance medical treatments for everyone. As Rabih explains, “Our ability to live long healthy lives is due to the participation of clinical trial participants.” 

 

The second night, attendees had the opportunity to hear from a panel of clinical trial participants who shared their experiences and perspectives in a lively discussion. The panel included: 

  • Tina Berry, Oncology Trial Participant and Project Manager,  Northside Hospital Cancer Institute 
  • Terp Vairin, Mental Health/Depression Trial Participant 
  • Melissa Hardman, Trial Participant, Founder & CEO of Faces of Research, LLC 
  • Ashley Nealy, COVID-19 Trial Participant 
  • Glenn Bachmann, Lymphoma Trial Participant 

The panelists shared their own clinical trial stories and discussed important topics like informed consent, compensation, and the obstacles that can prevent people from joining a trial. Melissa Hardman, who has experience as both a trial participant and  healthcare professional, noted the importance of making clinical trials accessible to all, sharing, “The ability to participate in clinical research should be the next step in the healthcare process for patients, not a daunting process. Access to clinical trials is ultimately access to healthcare.”

 

The AWARE for All – Atlanta  event concluded with a final panel discussion with healthcare and research professionals who shared their experiences. The panel included:   

  • Phyllis Kaplan, Diabetes Trial Participant, Sr. Manager of Events & Community Engagement, CISCRP 
  • Meghan Mckenzie, Principal Inclusion, Internal Partnering in Chief Diversity Office, Genentech 
  • Leah Szumita, Director of the Clinical Trial Support Center for the Leukemia & Lymphoma Society 
  • Zach Mitchell, Director of Business Operations & Recruitment, iResearch 
  • Tamara Wakhisi, Medical-Surgical Research Supervisor, Oncology, Northside Hospital – Central Research Department 
  • Yolanda Little, Diverse Patient Recruitment and Retention, Novartis 
  • Mary Slomkowski, Director, Clinical Management CNS, Otsuka 

 

The panelists shared their passion for clinical research and how they work with community members, discussing important topics like the importance of diverse representation and how the industry is working to overcome barriers to participating. Leah Szumita, explained the importance of building awareness and education at the community level, noting, “Clinical trials are the key step to advancing treatment. We need to increase awareness and participation across the board.”  

 

Throughout the event, attendees were able to navigate to the Informational Exhibit Center, a virtual exhibit hall that offers resources and information from over 30 health and wellness organizations in the Atlanta community and across the country. Exhibitors include Pfizer, Biogen, Janssen, Otsuka, IQVIA, CSL Behring, EMD Serono, WCG, Novartis, and Genentech.  

 

Visitors can also connect with Atlanta-based organizations such as SisterLove, RedMoon Project, iResearch Atlanta, Georgia Ovarian Cancer Alliance, Diabetes Association of Atlanta, and many more.  

 

If you missed the AWARE for All – Atlanta event or would like to tune in again, the recorded webinar and Informational Exhibit Center remain accessible here.

View more AWARE for All events here.

To stay informed about clinical trials, visit our Resources: https://www.ciscrp.org/education-center/resources/

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

The Importance of Clinical Research: Medical Heroes Appreci-a-thon Article Featured by USA Wire

CISCRP’s 4th annual Medical Heroes Appreci-a-thon event promotes staying active while learning about the importance of clinical research participation.

This is a free, virtual activity challenge that is open to the public and will be held January 3rd-31st. The Medical Heroes Appreci-a-thon brings the public and global clinical research community together to express their appreciation for all “Medical Heroes” — clinical trial volunteers, medical and healthcare professionals, and researchers. 

Racers can log their activity throughout the month of January, with each measurement of activity converted into miles that will pin-point teams on the virtual course. This year’s virtual racecourse will wrap around the coastline of Oahu, with scenic views for participants to enjoy every mile. 

Written by: Lindsey Elliott | lelliott@ciscrp.org

Read the full article on USA Wire here.

 Visit the 2022 Medical Heroes Appreci-a-thon page for more information.

Working Towards A More Inclusive Environment: Transgender & Non-Binary Participants in Clinical Research

Members of the transgender and non-binary community face myriad challenges during healthcare interactions, including unequal treatment and inappropriate curiosity from healthcare staff. Additionally, there is a widespread lack of understanding of specific transgender health needs and difficulties assessing approaches to healthcare by medical professionals. As a result, 37% of transgender people and 33% of non-binary people responding to a recent survey, reported having avoided medical treatment for fear of discrimination. 62% of transgender people said they have experienced a lack of understanding of specific trans health needs by healthcare staff. (1) A report published by the UCLA School of Law Williams Institute reports that 1.4 million adults in the United States identify as transgender. (2) Colleagues at Parexel International recently conducted a comprehensive review of current medical literature to assess the barriers that transgender and non-binary individuals face to medical care and clinical trial participation, in an effort to enhance the clinical research environment and promote inclusivity.

Gender-Identity-Sexual-Orientation

“We spent quite a lot of time speaking with members of the transgender community and we had a patient advisory council meeting at Parexel where we invited members of the community to speak with us, so that we could listen and learn how we can do better as an industry,” says Rosamund (Roz) Round, Vice President, Patient Innovation Center & Decentralized Trials, Parexel International. Recommendations included strategies for clinical site staff and transgender and non-binary individuals seeking health care in general, as well as through participation in clinical research.

Respecting the Individual
“It’s all about the lived experience of the patient. When we think about health professionals and health professionals who run clinical trials, they have to have an understanding of the transgender community, including things such as pronoun use, life experiences, health practices- all of those things are a foundation for making it a more inclusive environment for all transgender patients,” says Liam Paschall, Global Business Training Partner at Parexel, a transgender man who knows how critical and life-altering inclusive healthcare and clinical trials can be to members of the LGBTQ+ community.

“I think for anyone who is new, (to providing medical care to members of the transgender and non-binary community) the important thing is to know that you might not get it right every time, but it’s important that you learn,” says Roz. “If you use an incorrect pronoun, for example, the important thing is to apologize, move on and do better next time.”

Providing Education & Training for Medical Staff
Liam points to the importance of providing education and training for clinical research and other medical staff. Key topics to cover include the differences between biological sex, sexual orientation, gender identity and gender expression.

“We can do all the work that we can on getting the intake forms right and our systems correct, but if every member of a particular facility, hospital – wherever it is – is not fully educated and trained on how to work with trans patients, it’s not going to change,” says Liam. “It comes down to the individual. Every single person who comes into contact with that patient has to know how to talk to that patient, has to know the right questions to ask, has to understand that they can’t react a certain way, for example, if there is a transgender female, who they feel doesn’t necessarily look the role of being female. It’s all about respect.”

Dr. Sebastian Barr, a licensed psychologist and transgender man who specializes in helping institutions and healthcare providers adopt affirming, inclusive and evidence-based practices in both clinical work and research with members of the transgender community, underlines the importance of cultural humility training and education for staff at every level.

“There is true complexity in the diversity of different biological components that transgender people bring, but what I would remind people of is that there is huge diversity in non-transgender people’s bodies and biologies, as well,” says Sebastian.

Bridging the Trust Gap
There are historical and current reasons and examples as to why there is mistrust of the health care system by transgender and non-binary community members. To bridge this trust gap, Sebastian advises recognizing the diversity within the communities.

“There is a lot more marginalization and oppression of trans women, trans people of color, trans women of color, and non-binary folks,” says Sebastian. “How do we tell the trans community that we have their best interests at heart? We make sure that we are talking to everyone.”

Sebastian also recommends building relationships with community organizations and community members who can facilitate recruitment for clinical research and coordinate advisory boards so that “…you’re not just telling people that you’re trustworthy, but you’re demonstrating that you are and that you have actively considered their participation.”

Modifying Intake Forms
The majority of intake forms in medical settings that Liam has viewed are not representative of the transgender community. “It’s hard to get people to share, in terms of their identity and sexual orientation, when it doesn’t seem like the intake forms and the questions being asked are inclusive of who we are,” says Liam.

“In medical systems, we do a lot of gendering. Everything from records, lab results, the way that we talk about anatomy, in the way that we talk, even casually, about patients. Forms are a really important starting point,” says Sebastian. “Actually integrating what people are telling you about themselves and integrating that with how you and your colleagues are discussing who you are working with is a critical next step.”

Reviewing Protocols
Roz recommends that clinical research professionals conduct protocol review sessions to ensure that there are not items built into the protocol that are inadvertently exclusionary. In a conversation with a member of the scientific community, Roz learned that historically, there had not been transgender and non-binary participation in clinical research because it was not written in the protocol.

“Things evolve and the world changes. The scientific review of protocols to see what is really necessary and the need to enable diversity within protocols, where possible, is really important,” says Roz. “Just because it never has doesn’t mean that it can’t.”

Developing a strategy that includes working with patient advocacy groups, patients and caregivers in the actual design of the clinical trial protocol is key.

Removing Barriers to Participation in Clinical Research
In order to remove barriers to participation in clinical trials for the transgender and non-binary community, Liam advises that clinical research professionals recognize “…things like accessibility, language, confidentiality concerns, make sure we’re not ‘outing’ people, cultural humility training for the staff, sexual orientation and gender identity inclusive intake forms, strategies for public outreach and education, financial reimbursement for expenses, and trans-specific resources in general would be a big help.”

Another step is providing non-gendered spaces in medical facilities. “Creating gender neutral bathrooms is really critical and not challenging, operationally, to do, for example. And it can be a really big difference about how comfortable someone feels or their fear of being revealed,” says Roz.

Involving Community Members as Part of the Health Care Team
“I would recommend that the transgender community not be involved in clinical research where there isn’t an identifiable transgender person involved on the team,” says Sebastian. “I know that sounds severe, but there’s so much risk of harm. Look to see who’s guiding the project and if it will be safe for your involvement.”

Liam adds “There’s a lot of drugs that behave differently from one population to another. So whether there’s a transgender person at the medical facility or not, (medical professionals need to) really understand the differences, at the clinical trial stage, that could leave transgender patients at risk of an avoidable safety issue. Having a diverse and inclusive study population does help to ensure an adequate understanding of how the drugs work and how safe it is across the target population.”

In order to design clinical trials that are truly inclusive, clinical research professionals need to have an understanding of the transgender and non-binary community as a whole. To learn more, access the webinar “Improving Access to and Experiences of Transgender and Non-Binary Patients in Clinical Research” here.

Written by Melissa E. Daley, Communications & Marketing Manager, CISCRP

Access a post webinar Q&A educational resource here.

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.

Sources:

(1) LGBT in Britain; Health Report. Stonewell, 2018 and 2015 US Transgender Survey

(2) Report: How Many Adults Identify as Transgender in the United States?, UCLA School of Law, Williams Institute

Clinical Trial Participants’ Selfless Gift to Medicine

By volunteering to participate in clinical trials of new and experimental treatments, medical heroes give society valuable knowledge about the nature of disease, its progression, and how — and how not — to treat it. Participation always carries some risk, offers some hope for a treatment, and even offers some hope for a cure, but it is likely to bring no direct personal benefit to those who volunteer. As such, medical heroes ultimately give a gift that will benefit future generations of patients.

Facing the challenge

For the vast majority of people, clinical trials are an abstract concept that have little to no personal relevance. Most people take a hard look at clinical trials for the first time when facing the sudden and often unexpected prospect of a serious and debilitating illness for which no medication is available or adequate. Typically, patients, their families, their friends, and their healthcare providers must gather information quickly to understand how the clinical trial process works, the requirements of participation as defined by the study protocol, and whether participation is appropriate. This rush to navigate and master the unfamiliar terrain of clinical trials invariably feels overwhelming and confusing, and once in a clinical trial, many volunteers find the requirements and demands of participation difficult to manage.

Raising awareness

In 2003, the Center for Information and Study on Clinical Research Participation (CISCRP) was founded to provide outreach and education to those individuals considering participation in clinical trials and their support network. Based in the Boston area, this nonprofit organization serves the global community of patients and the public by raising awareness and providing education about clinical trials and by enhancing study volunteer experiences during and after clinical trial participation. Our many events and services are designed to improve public and patient literacy; to engender feelings of empowerment and control; to ensure more informed decision-making, improved convenience, and accessibility; and to recognize and appreciate the medical heroes that inspire us. This special USA Today supplement also plays an important part in raising awareness and literacy. It is a reference resource offering an introduction to clinical trials and providing valuable information to help you and your support network navigate the participation process.

At the present time, nearly 6,000 experimental drugs and therapies are in active clinical trials, and that number continues to grow as improvements are made in detecting disease, discovering new medical innovations, and understanding and addressing the root cause of acute and chronic illnesses. At the very heart of all of this promising, lifesaving, and life-altering activity are medical heroes to whom we owe our deepest appreciation for their profound gift of participation. 

Written by: Ken Getz | Founder and Chairman, CISCRP

Article from 2021 Clinical Trials Supplement, USA Today. View Supplement Here >

An Inside Look at Advocating for Yourself or Your Child in a Clinical Trial

Dr. Mark Sorrentino, MD, MS, Vice President for Pediatric Development, ICON and Dr. Joanna L. Perkins, MD, MS, Director of Medical Affairs, Hematology-Oncology, Americas, ICON, discussed how to advocate for yourself or your child in a clinical trial in a CISCRP 15-minute Flash webinar. Dr. Sorrentino shared his perspective as a clinical trial participant and Dr. Perkins expanded on her experience from a principal investigator’s lens. You can view the webinar here.

It was near Thanksgiving in 2020, and Dr. Mark Sorrentino noticed a small lump in his neck which was diagnosed as head and neck cancer. “I found out I had cancer that required surgery, radiation and chemotherapy,” Dr. Sorrentino recalls. “At the time of my diagnosis, my care team told me there was a clinical trial that I could participate in. Given that I had been in clinical research for over 20 years, it was the first time I had the opportunity to participate in a clinical trial – I was both nervous and excited about seeing what it would be like from the other side.” The experience reinforced the importance of being an advocate for oneself or one’s child in Dr. Sorrentino’s mind.

Reviewing Informed Consent & Schedule of Assessments

Starting from the beginning of Dr. Sorrentino’s journey, he  received an informed consent document that was 15 pages long. While it was not the longest document of this type that he had seen during his career in clinical trials, it was still somewhat daunting. Dr. Sorrentino thought about how this same document would need to be translated into an assent document for a child. (An assent form differs from an informed consent form and is designed to indicate that a child is willing to participate in a clinical study and understands what he or she will need to do. You can learn more here).

“It was filled with all sorts of legalese, and one thing that I picked out that I thought was very challenging, was the schedule of assessments,” says Dr. Sorrentino. The schedule outlines the treatment, tests and other procedures that may occur during the course of the clinical trial. The documents are not always presented in an easy-to-follow format. Because of his experience in clinical research, Dr. Sorrentino knew to ask for a version of the document that outlined the details in a table.

“Informed consent documents can be completely overwhelming,” says Dr. Joanna L. Perkins. “From a regulatory standpoint, there is a lot of information that has to be included.” And, at the beginning of the study, it’s important to find out how the results of the clinical study will be shared with participants.

It is also important to understand before deciding to participate, what is particularly being done for the trial and what is above and beyond that is specific to that clinical trial and not part of standard of care.  This is not always clear for the participant and important to feel confident to ask the study team questions early on. For example, a weekly blood draw that is outlined in the schedule of assessments may feel burdensome, but it may also be part of the regular standard of care for the disease or indication.

Coordinating Care & Providing Support

“One time I went in for my standard of care blood draw, which was in one laboratory area in the hospital. Later that day, when I went to the clinic to do the schedule of assessments for the clinical trial, and I had to have another blood draw,” said Dr. Sorrentino. He asked the clinical study coordinator why a single blood draw had not been scheduled and coordinated, to save time and physical discomfort. “This was an example of the health care teams not communicating with each other,” says Dr. Sorrentino.

“Depending on the clinical trial that you are in, and the center you are at, there may be two totally different teams of medical professionals who are overseeing your clinical care and your study assessments,” says Dr. Perkins. “It’s really important to advocate for yourself or your child to make a point of understanding what is being done on each of the days you go for your visit. Painful procedures are often a part of treatment. Make a point to ask what will be done to support your child through those painful procedures – and adults, as well.” Many clinical research centers have dedicated child-life staff to ensure that pediatric participants are cared for in the most sensitive way to minimize pain and fear. This can include play-based distractions, medical procedures such as sedation, or meetings with pediatric mental health professionals.

Parents may have to coordinate childcare options for siblings, in tandem with scheduling medical treatment for the child participating in clinical research. “Most large pediatric clinical centers have sibling play areas,” adds Dr. Perkins. Other services may include programs that address how to cope when one’s brother or sister is going through treatment for a chronic illness, such as cancer. “Parents should ask about programs to support their other children,” advises Dr. Perkins. Sometimes, there is financial support available for childcare and transportation.

Coordinating visits to the clinical research center with standard of care visits, when possible, can greatly reduce the burden of clinical research participation for both adults and children. It is important to advocate for yourself and/or child when scheduling clinical trial visits with the study team. “Take a really good look at when you have to be at the clinic,” says Dr. Sorrentino.

Bringing the Clinical Trial to You

Remote capabilities are being scaled in clinical research, due, in part, to the advent of the COVID-19 pandemic. Some tests may be able to be performed at home with the use of portable medical devices and apps, coordinated through a laptop, tablet or mobile phone. Clinical research staff, including nurses, can often make home visits. Dr. Sorrentino was able to be evaluated at home, outside in his yard, during the COVID-19 restrictions in his hometown of Los Angeles. “It was fantastic, it kept me from having to go down to the hospital,” says Dr. Sorrentino. “The nurse took my vitals and did my lab work.”

Asking Questions About the Placebo

Another point of advocacy is around the use of placebos. Inquire what happens at the end of the study. If your child was on the placebo, ask if they will be able to receive the therapeutic drug or agent that was being studied at that point of time. “Many times, that is the case, but you have to remember to ask about it,” says Dr. Sorrentino.

Developing New Treatments & Optimizing Care

Overall, Dr. Sorrentino views his experience as a clinical trial participant in a positive light. “Clinical trials need you and they need your children, if we’re going to get new and innovative therapies, particularly for children, since we have lagged behind on that in the past,” says Dr. Sorrentino. It’s important to remember there is a whole team of medical people watching your disease and diagnosis.

“The overall cure rate for pediatric cancers in the 1960s was about 20%,” says Dr. Perkins. “And now, today, it’s higher than 80%. The biggest, most important reason for that is the sharing of data and participation in clinical trials. With standardized clinical trials, we can learn much more quickly how to optimize care and increase survival rates for diseases like cancer,” says Dr. Perkins.

Authored by: Melissa E. Daley, Communications & Marketing Manager, CISCRP

View CISCRP’s library of webinars and podcasts here.

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.

Supporting Caregivers in Clinical Research During the Pandemic

The COVID-19 pandemic has escalated the social isolation, medical uncertainty and disruptions in home/work life that rare disease patients and caregivers already experience on a daily basis. “We’re 18 months into a chronic grind of events that might not be impacting us dramatically, day to day, but there’s this cumulative, chronic, wearing-downness,” observes Scott Schliebner, former Senior Vice President, Scientific Affairs and Therapeutic Expertise Head, Center for Rare Diseases, ICON. Participants in clinical studies are surrounded by a support system of caregivers that help facilitate their participation. In the rare disease space, this often means parents. Great strides have been made in the clinical research enterprise to scale patient-centric approaches. This spectrum of support is also being extended to caregivers of participants in clinical research.

“Half the patient population in rare disease is children. The caregiver support is incredibly important. These are the individuals making decisions on behalf of their child, or their patient-adult child, it’s not all under 18 minors. Many of these patients have grown into adulthood and need ongoing support. Parents are involved in clinical trial decisions, advocacy decisions, and treatment decisions,” says Cristol Barrett O’Loughlin, Founder and CEO of Angel Aid Cares, a non-profit that provides mental health and wellness services to rare families,

particularly caregivers, with a focus on rare disease mothers. Offering assistance at different levels to caregivers is essential, particularly when it comes to emotional support.

In a study published by Health Psychology, rare disease patients were recently surveyed and interviewed about the types of support they receive, stress levels and their overall satisfaction with life. Four types of support were evaluated by the respondents:

  • Emotional support where the individual feels understood and validated by others
  • Informational support where individuals receive advice from others
  • Tangible support which provides practical assistance to address day to day tasks
  • Companionship support where individuals develop a sense of belonging by participating in leisure activities with others

Emotional and companionship support were the strongest predictors for life satisfaction, regardless of stress levels, with companionship being rated as the most significant factor. (1)

Cristol explains that these types of support can also be extended to the caregiver experience.

“Informational and tangible support are the triage list of what has to get done. What is this disease? What are the symptoms? What doctor do I go see to treat those symptoms?” says Cristol. “Are there any clinical trials? How do I get enrolled? How do I get insurance coverage for my claims? What medical devices do I need to have at home to manage this experience for my patient-child or patient-adult? Emotional support and companionship is where that caregiver or patient feels that someone understands their feelings about what’s happening to them. Someone is really, truly listening and engaged at a heart level, with what they’re experiencing.” This emotional and companionship level is enhanced further if the other person is currently undergoing or has undergone the same experience, providing a sense of relief for the patient or caregiver.

Cristol knows from personal experience the toll that stress takes on caregivers. She assisted with caring for her three older brothers who were born with a rare genetic disease and passed away at ages 12, 18 and 19 years old. “For decades I witnessed this chronic stress within the family, with caring for healthy individuals in the family and medically fragile individuals within the family,” says Cristol. “This isn’t a stressful event, as in PTSD, and now we’re recovering from it. This is ongoing, chronic stress.”

Cristol shares her insights into what she terms as CTSD, or chronic traumatic stress disorder.

“In a stressful event, your cortisol spikes and you go into a heightened awareness state and then you go into recovery (PTSD). (2) But when you are a caregiver, you elevate into this stressful state and now you’re buzzing at that higher level. You are in this for the long haul. The relief that you bring to CTSD or to a caregiver is different than trying to recover from a situational traumatic experience. That relief comes in the form of daily micro-moments of self-care,” says Cristol. This moment can be as simple as implementing slow breathing techniques to lower the heart rate. “Breathwork is the number one thing we can do,” says Cristol. Other wellness methodologies to implement include healthy eating, sleeping and exercise habits, all of which can be incredibly challenging for caregivers to navigate.

“Mothers are the primary caregiver in 82% of families in the rare disease space,” says Cristol. “We know (from support provided by Angel Aid Cares) that focusing on a mother, a grandmother, or an aunt, within a family, we can uplift a whole family. It’s never an exclusion of any other member of the family, rare fathers included.” Cristol also makes note that there is much to be learned from the mature caregiving models of the veterans, military, senior and dementia care spaces.

“If you want patients to enroll in a clinical trial, we need to make things easier for them, and part of this, is, of course, just extending this concept of patients to caregivers. What else can we do with that care circle to help that whole family unit or care circle group participate in a clinical trial? There are a lot of practical things,” says Scott.

To learn more, watch the 15-minute Flash webinar “Supporting Caregivers in Clinical Research: Navigating the Pandemic” here.

Authored by: Melissa E. Daley, Communications & Marketing Manager, CISCRP

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.

Sources:

(1)  https://www.psychologytoday.com/us/blog/disability-is-diversity/202010/people-rare-diseases-need-better-social-support

(2)   https://www.psychiatry.org/patients-families/ptsd/what-is-ptsd

Participating in a Clinical Trial: Building A Support Network

From "The Gift of Participation" by Ken Getz, Founder & Board Chair, CISCRP

Another important part of the human side of clinical trials is the support network that you create. Study volunteers are not alone in their clinical trial participation. Your decision to participate in a clinical trial is best made with input from the people you know and trust. Your network should include your family physician or specialist who has previously been treating your disease or condition. Your primary-care and specialty nurses may be very helpful in sorting out your identification of a clinical trial and the risks and benefits of participating in one.

Perhaps no one has a greater interest in your well-being as a potential study subject than your family and friends. They will want to be actively involved in the decision-making process. Young children rely on their parents or guardians for support and guidance. Parents in their later years may well depend on their adult children. Whatever your support network, you need to draw comfort, assistance, and resolve from your family, friends, and advocates in order to determine if a clinical trial is right for you. And once you’ve enrolled in a trial, you may need to tap that support network for aid, all the way from ongoing encouragement and advice
down to transportation to and from the research center.

Many medical conditions have special support groups and communities that can help assist in evaluating clinical trials as treatment options. You can find information about local and national patient advocacy and support groups online, in primary care centers, hospitals and in public libraries. There are also a growing number of online self-help groups that provide up-to-the-minute information on new drugs and treatments, as well as electronic bulletin boards and chat rooms where patients can share their personal stories and experiences with both standard and investigational therapies.

For more information on clinical trials and making informed decisions about volunteering for clinical research, read “The Gift of Participation” by Ken Getz, Founder and Board Chair, CISCRP.

You can find the book here.


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.

Rare Disease Clinical Trials: How to Prepare for When the Clinical Trial Ends

CISCRP hosted a 3-part webinar series titled “Navigating Rare Disease and Clinical Research: Every Patient Matters”. This article brief is based on the second episode, “Rare Disease Clinical Trials: How to Prepare for When the Clinical Trial Ends”. Moderated by Joan Chambers, Senior Director, Marketing and Outreach, CISCRP and Marsha Lanes, Genetic Counselor/Medical Editor, NORD (National Organization for Rare Disorders), panelists included Dr. Tracy Dixon-Salazar, Executive Director, Lennox-Gastaut Syndrome (LGS) Foundation, Richie Kahn, Senior Director, Patient Engagement, Medable, Inc., and Dana Holinka, Operating Board Member, Tuberous Sclerosis Complex (TSC) Alliance. You can access the webinar recording here.

You or a loved one is participating in a clinical trial, which at some point, will come to completion. There are several considerations to address before the end of the clinical trial, and conversations to have with the PI (Principal Investigator) and clinical site staff. These include how medical care will be transitioned, who will be the primary physician moving forward, post-study access to medical records and study staff, access to study medication, how you will be informed about the outcome of the clinical trial and potential options to enroll in an upcoming clinical trial. In rare disease clinical research, participants are often minors, so caregivers and patient advocates may have additional questions.

“Don’t think that any question is silly, stupid or dumb, because you’re going to go into this transition phase feeling like you have no control, after you’ve been taken care of and guided along,” says Dana Holinka, whose daughter is a rare disease clinical trial participant.

Dr. Tracy Dixon-Salazar’s daughter has participated in 26 rare disease clinical trials for medications, treatments, alternative therapies, and diet. Tracy advises to ask questions about access and care. If the participant received the study medication and not the placebo, and was benefitting from it, ask if they will still have access to it. 

In a clinical trial, the participant is cared for by the physician investigator and research staff, in addition to the standard of care that is concurrently being provided by their primary care physician. Once the trial is completed, ask how care will be transitioned, and if there is an option available to remain in the care of the study staff.

“How are you going to work with our primary care provider to make sure there is no gap, if the medication is something we want to stay on?” says Tracy. Other considerations are whether the study medication will be covered by the participant’s medical insurance and the length of access to it.

“People who are conducting clinical trials are the best of the best,” says Tracy. “They’re going to know very much about the research, very much about the treatment you’re on, and they’re going to know a lot about the process. Our experience, in the multiple trials that we have done, was that we could stay, if our insurance (in the United States) was now going to cover the medication. We had to work that out.”

Richie says another key for a successful transition is to “Prepare yourself to advocate, either as a patient or a caregiver, for a friend or loved one participating, and do it early.” Conduct research on clinical trials and the informed consent process, to make sure that all your questions get answered at the onset of the study, and that you understand exactly what is involved.

“Sponsors aren’t necessarily bound to continue providing access (to a treatment) after a trial ends. But when you’re having that conversation during the informed consent process, within that informed consent form, itself, all of this should be outlined. It’s good to check. If you are not sure, you can always reach out to your site coordinator, your PI (Physician Investigator). Often times, there are open label follow ups, long term extensions, and a couple of other possibilities, as well, where sometimes an investigational product will be made available after the trial wraps up. That is especially common after early Phase and first-in-human trials,” says Richie.

Biopharmaceutical companies and drug sponsors may have programs to assist families with the extra cost of the medication, after the clinical trial ends. This is dependent upon whether the product is already approved and marketed. There are other tools available, including state-level medication assistance programs. For investigational products that are not yet approved, there are mechanisms where a pharmaceutical company may be able to intervene to continue providing the investigational product after the clinical study concludes. And there may be the possibility of participating in another clinical trial. If a participant has not yet received the investigational medicine during the clinical trial, there may be an open access arm available at the end of the trial.

“Sponsors really do try to make that happen, as much as possible,” says Tracy.

Dana emphasizes the importance of networking with other patients, patient advocates, caregivers and advocacy organizations.

“Parents, and having those connections in the community, have helped us immensely. Connecting with other families and other people who are experiencing what you’re experiencing is huge. You can get some of the best information,” says Dana. This can include medical articles about the latest developments, or resources to help navigate day to day details. For example, Dana learned about a financial assistance resource called needhelppayingbills.com from another rare disease parent.

Dana recommends connecting with a local representative from the pharmaceutical company that can help with questions or concerns if there is one in your area.

“I think it’s imperative that you make those kinds of connections,” says Dana. “You can make them at the beginning, when you’re in the trial, or with some of the questions towards the end. Find out who that person is locally. They’ve had the experience of interacting with other parents, caregivers and the people involved in the studies.” The representative may be able to offer resources, such as discount coupons for medications or assistance with grassroots fundraising efforts.

“Generally, this is a really collegial industry. I think for just about everyone, it’s all about making those connections for the good of the patient. If you can get to the right person, you’re going to have a warm reception,” says Richie.

How information is shared with clinical research participants at the conclusion of the study is outlined during the informed consent process. If there are multiple sites and institutions involved in the clinical trial, enrollment rates and timetables may impact when the results of the clinical study are shared. The information has to be collected and analyzed. You can find information on clinicaltrials.gov and from advocacy groups and non-profits in your respective indications and therapeutic areas.

“Many sponsors will share this information directly with patients, if you ask,” says Richie.

“I think no matter where you live, if you are a patient in the trial or a caregiver, we all tend to have the same exact questions,” says Tracy. “Thinking about transitioning is a really anxious time for patients and families. Having those conversations start early, about the questions we’re all going to have, is really important.”

To learn more, you can view the webinar here. Access the companion infographic here.

You can view part-one of the series, “Rare Disease Clinical Trials: Being Informed About Clinical Research” here and part-three, “Rare Disease Clinical Trials: After Participation, Paying It Forward” here.

Access the companion infographics:

  • Clinical Research Participation Roadmap | Part-one here.
  • A Garden of Questions to Ask Before the Clinical Trial Ends | Part-two here.
  • Paying it Forward in Clinical Research | Part-three here.

Authored by: Melissa E. Daley, Communications & Marketing Manager, CISCRP

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.

What Does a Caregiver Need?

Sponsored by: Bristol-Myers Squibb

Dealing with significant health challenges requires expert medical care and the support of committed caregivers who always have the needs of the patient in mind. But what do caregivers need to do this effectively? When Don Selmon’s wife, Carla, was diagnosed with cancer, he found his sudden role as her caregiver overwhelming. “There is so much information to process and keep track of,” he says. “There is simply too much data to try and remember not to use some sort of system. I personally created Excel spreadsheets so I could keep track of drugs she was taking for side effects, daily vitals, upcoming scans and appointments, question sets when meeting with our trial doc, etc.”

Selmon’s experience as his wife’s caregiver quickly showed him how vital that role is. “We can’t sit still with an enemy like cancer,” he says, noting that as a caregiver his roles included “cheerleader, confidant, shoulder to vent on — always doing my best to have her back in meetings and in treatment.”

Another crucial role caregivers play is often overlooked: helping patients discover clinical trials that can offer hope, relief, and, possibly, the greatest gift of all: more time.

Finding clinical trials
“Caregivers play a critical role in helping patients process the information that they are hearing from their treatment team and help them weigh the options,” says Sharon Hanlon, head of clinical trial engagement and enrollment at Bristol-Myers Squibb (BMS). “They may play a role in the decision to participate in a clinical trial, doing some home-work on behalf of the patient to help with their decision.”

Caregivers often start with a simple online search to find out about clinical trials. Web resources like clinicaltrials.gov can be easy to find, but trying to use because the trial descriptions can be difficult to read, and often assume a medical background that many caregivers lack. To help caregivers more easily access this crucial information, many pharmaceutical companies have created patient-focused websites that make it easier to identify appropriate trials.
“Ours is BMS Study Connect,” says Hanlon, “which utilizes patient-friendly language to help simplify the process and help make the journey easier. We also provide informational resources such as patient stories, such as the one here of the experience of a patient and caregiver during their clinical trial.”

At Study Connect, patients or their caregivers can use straight-forward tools to find potential clinical trials they can participate in. Even though the site is run by BMS, Study Connect searches all possible trials, even those not sponsored by BMS, something similar websites don’t offer.

Supporting caregivers
Despite their vital importance, the tremendous — and sometimes exhausting — efforts caregivers contribute is not always visible. “I often say that caregivers are the forgotten member of the patient experience,” Hanlon says, noting that they often experience feelings of powerlessness and stress around the experience. Caregivers are just as much in need of emotional and practical support as the patients they’re looking out for, and companies like BMS have also set up online resources where caregivers can exchange information and support each other.

“Web communities present an outlet for caregivers,” Hanlon says, “providing access to information.” For example, BMS participates in the Inspire Community making it available for patients, caregivers, and healthcare professionals. Members of the forum can support each other emotionally, compare notes, and discuss trials they have learned about or are participating in. 

Inspire surveyed patient and caregiver members about clinical trial perceptions in late 2018, and found that almost a third — 31 percent — of all respondents had tried to sign up for a clinical trial, and 21 percent agreed with the statement “clinical trial information is too confusing.” As a result of this information, now many Inspire communities have sections that are devoted specifically to clinical trial discussions to provide support and further information.

However, different people use Inspire in different ways. For Don Selmon, it was a vital connection to shared experience. “I utilized Inspire to learn more about the reported impacts of various treatments,” he says. “It was of great benefit to me as it helped me pre-pare as a caregiver for what might come from my wife’s treatment and how best to help her.”

Sometimes that connection is the best help a caregiver can receive. As Hanlon notes, “Sometimes the simplest connection is often the most impactful!”

Article from 2019 Clinical Trials Supplement, USA Today. View Supplement Here >

Expanding Clinical Trial Access for Women Living With HIV

Written by: Jan Nissen, BSN, MBA, MS Pop Health, VP Patient Innovation & Engagement, Merck & Co., Inc

For over 30 years, Merck has been committed to improving the lives of people living with HIV. In those three decades, there have been remarkable advances in therapies to treat and monitor the disease. Today, however, the people who are infected with HIV is changing; there is a silent epidemic of women living with HIV, requiring new ways of understanding the needs of people living with HIV and developing more effective options for treatment and care.

Globally, there are 18.8 million women and girls living with HIV and approximately 870,000 new cases annually. That means every 80 seconds, a young woman becomes infected. Designing clinical trials for these women presents challenges given their unique barriers. From a cultural, social, and economic perspective, many women with HIV are not aware of or willing to participate in clinical trials. We want to change that.

Connecting through the community
Merck is working with community outreach groups and women living with HIV to understand the burden of the disease and what solutions might help. Partnering with female patient advocates (patients who publicly support or make recommendations for a disease) has helped us to listen and learn about known barriers to clinical trial engagement and to develop a female-focused recruitment strategy for our clinical trials. A community advisory board also helps generate solutions that address patients’ questions and helps them overcome challenges to participating in a clinical trial such as transportation or even child care.

Kathleen Squires, M.D., Merck Scientific Affairs, reiterates the importance of evolving our approach to better meet the needs of women with HIV. “Less than 20 percent of HIV clinical trial participants are women. Women may be at higher risk for complications, and we need to understand these risks and address their concerns and needs if we want to design effective trials and medicines.” Bringing meaningful input from women living with HIV into the design of clinical trials helps us partner with patients to make trials more women-friendly.

Article from 2019 Clinical Trials Supplement, USA Today. View Supplement Here >