Transforming Clinical Trials: Establishing the Rare Disease Advisory Committee

Authored:  Scott Schliebner, SVP, Centers for Rare Diseases & Digital Therapeutics,
PRA Health Sciences

According to Global Genes, the 7,000 distinct types of rare and genetic diseases impact more people than cancer and aids combined.  PRA Health Sciences understands that rare disease clinical development programs are unique, and to be successful, should be approached from an equally innovative perspective.  PRA Health Sciences’ Center for Rare Diseases believes that by working directly with patient advocates offers us the opportunity to better understand the needs of the rare disease community and how we can transform clinical trials to be more effective and accessible for patients.  We are passionate about the role that patients can play to help better design, enhance, and optimize rare disease clinical trials.

The Center for Rare Diseases believes that by partnering with patients, clinical trials are designed and executed more efficiently and with a focus on the items that are most important to patients.  To help achieve this, we formed the Rare Disease Advisory Committee (RDAC), an independent, autonomous group of rare disease patient advocates who are committed to advising and working with PRA to improve the way patients are integrated into the clinical development lifecycle.  The RDAC will examine and explore ways to optimize the drug development process by involving patients, thereby accelerating clinical development and bringing new therapies to patients faster.  Central to this effort will be a focus on how patients and patient advocacy organizations can be impactful partners throughout the entire drug development lifecycle.

The Committee’s initial two focus areas are on (1) developing a Rare Disease Drug Development Patient Engagement Roadmap, and (2) developing a Rare Disease Clinical Trial Engagement Burden Assessment.  We will also be focusing on how patients, and patient advocacy organizations can be made partners throughout this entire process.  We are excited about the efforts of the RDAC and are committed to bringing new therapies to patients faster.

To learn more about the Rare Disease Advisory Committee (RDAC), please visit 

Members have an opportunity to share educational information and new initiatives being introduced about clinical research to patients, caregivers, advocacy organizations and industry. To submit a brief article for consideration, please contact Joan Chambers,

Medical Hero Spotlight: Sandy Morris, ALS Advocate

On January 6, 2018, at the age of 51, Sandy Morris was diagnosed with Amyotrophic Lateral Sclerosis, also known as ALSALS is a progressive nervous system disease that affects the nerve cells in your brain and spinal cord. The disease causes individuals to lose control of the muscles needed to move, speak, eat and breathe. According to the ALS Association, every 90 minutes someone is diagnosed with ALS and someone passes as a result of the disease. There is currently no cure for this debilitating disease. 

Sandy spoke candidly about the effects of ALS, “No one wants their body to trap them.” A mother of 3, she was determined not to give up hope. Following the advice of her best friend, she got involved in a clinical trial at the California Pacific Medical CenterAs part of the trial, she had to undergo bone marrow extraction in order to retrieve stem cells which was followed by a series of infusions and lumbar puncturesAlthough the clinical trial involved invasive procedures, she describes her experience as an overall positive one. Regarding her participation Sandy stressed, “I would rather die trying. I don’t want to just accept my fate.” 

ALS affects everyone differently. Sandy explained, “How we progress is totally unique. I have some friends who can’t talk but can walk, and others who can walk but can’t talk.” For individuals considering participating in a trial, she stressed the importance of being well informed by researching the treatment being tested. Sandy shared her willingness to participate in future clinical trials and encourages others to educate themselves until ALS treatments and cures are found. She encourages others to get involved noting, “That’s the only way we’re going to move forward.” 

 There are currently four medications available to treat ALS. But more research remains to be done to find a cureDespite ALS being a fatal disease, Sandy remains hopeful, “We just need a happy ending and we don’t have one yet.” Her advice for other ALS patients and their loved ones is to remain as positive as possible while learning to live with their disease, something she acknowledges is not an easy thing to do.  

Sandy discussed ways she wants to continue to make a difference including attending FDA meetings to share her experience and influence more efficient, humane clinical trial design. She also stressed the importance of pharmaceutical companies and patients working together to create clinical trials. Her goal is to create hope for the future. Sandy stressed, “I want to make sure that the next 51-year-old mother of three doesn’t have to hear she has ALS period. I want a comma, nothing finalized.” 

New Patient Engagement Insights from the 2019 CISCRP Perceptions & Insights Study

CISCRP hosted a webinar on October 29, 2019 to share the results of the latest 2019 Perceptions & Insights Study. Over 12,450 people (including 3,600 prior study volunteers) from around the world provided their opinions on clinical research. This latest study reveals significant new insights on patient engagement preferences in particular – such as preferences for learning about clinical trials and services which reduce burden the most, the impact of physician recommendations and involvement in clinical trials, and receptivity to emerging models of clinical trials and new technologies.  Critical differences in sentiments by region, race/ethnicity and other demographic variables will also be explored, along with trends and comparisons to prior studies.   

Topics Discussed:

  • How perceptions of clinical research have changed over the years 
  • Preferred patient engagement initiatives
  • Ways to improve clinical research experiences 

Key Takeaways:

  • Continued acknowledgment of the importance of clinical research, but awareness and trust less among those that have never participated  
  • Healthcare provider recommendations continue to be critical to increasing participation in trials 
  • No one size fits all when it comes to various clinical trial models (traditional vs de-centralized) 
  • Study summary and individual results most wanted post-participation
  • Electronic consent forms viewed as easier to understand compared to paper 


  • Ken Getz, Founder and Board Chair at CISCRP and Deputy Director and Professor at Tufts CSDD Tufts University School of Medicine
  • Annick Anderson, Director of Research Services at CISCRP
  • Jasmine Benger, Senior Project Manager of Research Services at CISCRP

To receive invitations to future CISCRP webinars subscribe to our email list.

To collaborate or host a webinar with CISCRP, please email or call the marketing team at 617-725-2750.

Patient Engagement in Clinical Trials

CISCRP and World Courier collaborated to host a webinar on September 24, 2019. This webinar examines the role of patient engagement in recruitment, retention and patient satisfaction in clinical trials. 

Topics Discussed:

  • Patient Feedback on Clinical Trial Participation
  • What do Patients Want and how can we Support Them?
  • Value of Investing in Patient Engagement
  • What is Direct-to-Patient (DtP) Logistics?
  • DtP Case Study: Collaborative Project Setup
  • What are Organizations Doing Now? What are the Next Steps?

Key Takeaways:

  • There is no ‘one-size fits all’ approach to patient engagement – options are key!
  • Helping advance science and treatment, the ability to access information about their health and condition, and the amount of care and attention they received while enrolled are some of the top ‘likes’ among clinical trial volunteers


  • Jasmine Benger, Senior Project Manager, Research Services, CISCRP
  • Alex Guite, Vice President, Strategy and Services, World Courier
  • Mike Sweeney, Senior Director, Patient Centric Logistics, World Courier

To receive invitations to future CISCRP webinars subscribe to our email list.

To collaborate or host a webinar with CISCRP, please email or call the marketing team at 617-725-2750.

Supporter Spotlight: Biogen

Biogen has played, and continues to play, an active role in supporting CISCRP’s mission to educate patient communities about clinical trials. A host sponsor of our upcoming AWARE for All programs in Atlanta and Phoenix, their support has further demonstrated their commitment to improving disparities in clinical research literacy among diverse and underserved communities and building a foundation of support and access among minority health care providers.

Biogen’s work with CISCRP also extends to our Perceptions & Insights study, Health Communication Services, as well as Patient Advisory Boards (PABs) which further showcases their patient-focused culture and mission to deliver innovative therapies for people living with neurological and neurodegenerative diseases.

We met with Murray Abramson, MD, Vice President of Global Clinical Operations at Biogen and a major champion of these engagement efforts. Murray highlighted the importance of “stepping into the shoes of patients” to gain a better understanding of what matters to them. Murray shared one of his motivations with us, “It is absolutely imperative to reach out to people, particularly in disease areas where there aren’t good treatment options or perhaps not any treatment options at all.”

Biogen has also developed a number of meaningful, impactful, internal programs to promote patient engagement among staff. The team’s Tree of Hope, dedicated to Carol Seider’s memory, was prominently displayed with placards representing staff motivations on ways in which they can support patients around the world. Carol Seider lived her life dedicated to finding treatments and cures for patients in her role as Senior Director of Global Clinical Operations. She helped inspire Biogen to seek new ways to amplify the voice of patients in clinical research.

In addition, Biogen hosts an annual Patient Engagement Day to bring employees together across R&D to raise awareness about Biogen’s patient engagement efforts and ways to implement meaningful initiatives as a part of department-wide activities. Ken Getz shared CISCRP’s Perceptions & Insights data at a recent Patient Engagement Day that focused on building a sustainable and stronger engagement with underrepresented patient populations for clinical trials.

CISCRP is grateful for Biogen’s support of numerous patient engagement activities, which help to raise awareness, empower individuals and their families with education to allow for informed decision-making processes, and improve overall clinical trial experiences.

Written by Ellyn Getz, Associate Director of Development & Community Engagement

From the Editor

Dear Readers, 

Autumn is here and in New England, there is a crisp coolness in the air along with the leaves starting to turn colors of red, yellow and orange.  A beautiful time of year and a sign that 2019 is fast approaching its close.  Like many organizations, CISCRP is focusing and spear-heading several different initiatives with an eye towards 2020 planning.   

In this edition, Leah Crocker, our Medical Hero Spotlight, shares her journey with Lupus and Raynaud’s phenomenon, a common symptom of Lupus. Leah’s clinical trial journey began when relocating to a warmer climate, Georgia, and connected with a Rheumatologist, who encouraged her to participate in a clinical trial. 

In addition to Leah’s inspiring story, our Q3 newsletter features a variety of exciting articles covering key programs, events and initiatives supporting our mission of building awareness and education among diverse communities. 

The initiatives are in full motion with the AWARE for ALL events being hosted in Atlanta and Phoenix, the Journey to Better Health mobile, interactive exhibit ‘on wheels,’ to the 2019 Perceptions and Insights study where results are in and analyzed.  We are continually working with industry professionals on producing Trial Result Summaries to offer to patients who participated in clinical studies to help them understand the study’s outcome.  

A new initiative underway that we are very excited about is expanding clinical trial awareness and the importance of participation to diverse and underserved minority communities.  This new media awareness campaign is in addition to our annual National Clinical Trial Outreach and Awareness Initiative with the USA Today Supplement.   

I invite you to read the brief articles to learn moreshare your comments and to participate in the media awareness campaign.  

If you are in search of educational materials about clinical trial participation, please visit the CISCRP Store for brochures, posters and videos that can be used to educate patients about clinical research. Brochures may also be co-branded for use at research sites, healthcare forums, educational workshops or other events. Please contact me,, for details.  

As we enjoy Autumn, and all that it brings, we welcome the opportunity to assist you in further building educational and awareness programs around clinical trials to all communities.  Please contact us as you work on developing your end of year 2019 and 2020 plans.  

We look forward to seeing you at the upcoming industry events.  Thank you for your continued support.  

Warm regards, 

Joan A. Chambers 
Senior Director, Marketing & Outreach 

Spreading Clinical Research Awareness to Diverse Communities

The Center for Information and Study on Clinical Research Participation (CISCRP) is launching a new educational campaign focusing on the importance of clinical research participation and diversity in clinical trials.  

As an extension of our ongoing semi-annual national media outreach program to recognize Medical Heroes and spread clinical research awareness in the USA Today newspaper, CISCRP has established relationships with five leading newspaper publishers serving diverse minority communities.  The new campaign will reach nearly one million people in African American and Hispanic communities in major cities throughout the United States during the month of November.  These major cities include Atlanta, Georgia (Atlanta Voice Newspaper), Chicago, Illinois (Chicago Citizen Newspaper), Detroit, Michigan (Michigan Chronicle), Los Angeles, California (Excélsior Newspaper), and New York City, New York (New York Amsterdam News). 

Throughout the month of November, CISCRP will place a compelling and inspiring full-page, 4-color ad with thought-provoking copy in each of the newspapers—both print and digital.  The ad will highlight the need for diverse patient participation in clinical research in order to discover new therapies that are effective for all individuals.  

Collectively, we can raise public and patient awareness and education with the full-page ad and its powerful message, “Diversity in Clinical Trials Brings New Treatments to Everyone.”  

The campaign’s goal is aimed at building trust within diverse and underserved minority communities, breaking down the barriers, informing individuals about clinical research participation and the importance of diversity in clinical trials.  

CISCRP will be receiving support from various stakeholders in the clinical research industry who share the same passion and commitment to improving clinical trials and extending opportunities to participate in studies to underrepresented communities.  This exciting campaign is just the beginning.  OvertimeCISCRP plans to increase the scope and frequency of this diversity-focused initiative.  We are hopeful that future participating sponsors of this important campaign will contribute their own educational content and utilize our media channels to share valuable information with underserved communities.    

To learn more or participate in this important initiative, please contact Kat Marriott at 

Written by Kat Marriott, Marketing Program Manager 

Leah Crocker: Lupus Advocate Shares Her Journey

Lupus is an autoimmune disease that causes your body’s immune system to attack your tissues and organs. Difficult to diagnose, Lupus can remain dormant in an individual’s body, only to be “woken up” by an illness or major surgery. This was the case for Leah Crocker. In 2000, Leah had just undergone carpal tunnel surgery on both hands but wasn’t healing properly. At this time, her orthopedic surgeon suggested she consult a rheumatologist. Upon seeing a rheumatologist her blood work came back positive for Lupus.

A disease with no cure, Lupus is managed through treatments that help patients control their symptoms. Soon after her diagnosis Leah began experiencing numbness in her fingers. This issue was identified as Raynaud’s phenomenon, a common symptom of Lupus. Raynaud’s is a condition that results in the discoloration of the fingers and toes in response to cold temperature or stress. During this time, Leah was prescribed an anti-malaria drug to help. However, her condition would soon grow worse when she discovered that gangrene had set into two of her fingers. At the time she had a choice between amputation or chemotherapy. Leah chose to undergo chemotherapy, which she continued for several years.

Despite undergoing treatment for Raynaud’s, because of the severity of her condition her rheumatologist strongly recommended that she relocate to a warmer climate to reduce the chances of her condition worsening. For Lupus patients in similar situations Leah advises, “Know your limitations and live within them.” So, in 2007 she made the move from her home state of New Jersey to start a new life in Georgia. After relocating to Georgia Leah connected with a new rheumatologist Sam Lim at Emory University School of Medicine.

This is when her clinical trial journey began. Leah credits Dr. Lim for encouraging her to get involved in clinical trials. The first study she joined was The Georgians Organized Against Lupus study, led by Dr. Lim at Emory University in 2013. The goal of this study is to better understand the burdens of Lupus. Leah has been involved in this study for the past several years, participating in in-person visits once a year and completing an online questionnaire twice a year. Leah also participated in another trial for Raynaud’s in 2015. This trial was studying the effects of Botox on individuals with Raynaud’s. As part of the trial she was asked to hold her hands under cold water and receive Botox injections. Despite the challenging experience, Leah is happy to have participated in this trial.

An experienced clinical trial volunteer, Leah’s advice to those thinking of participating is, “Just do it.” At the time of her diagnosis there were fewer medications available for individuals like Leah. Today, thanks to clinical trial volunteers, there is newfound hope for the Lupus community. To find trials for Lupus in your area visit CISCRP’s Search Clinical Trials page. For more information about Lupus, treatments, clinical trials, community forum and other educational information, please visit

Written by Leslie Perez, Marketing and Communications Coordinator

Traveling Exhibit Tour Begins: CISCRP’s Journey to Better Health

A first-of-its kind educational and recreational vehicle equipped with interactive tools, healthcare information and relevant resources, insightful study data and knowledgeable experts well versed in the clinical research industry 

Starting this month, September, the Journey to Better Healthmobile exhibit on wheels’ is traveling to diverse communities across Atlanta, Chicago, New York, Newark and Long Island, stopping at public schools, museums, libraries, community and senior centers, places of worship and free public-health expo’s 

Community members are invited to walk through our educational exhibit and learn about the clinical research process through a variety of stations focusing on: 

  • Why diversity is important in clinical research 
  • A timeline of advancement in public health 
  • Study volunteers and their experiences 
  • Resources & take-aways for individuals interested in clinical research participation  
  • Questions about eligibility criteria and the informed consent process in order for individuals to make informed decisions about clinical research participation 
  • Potential risks and benefits 

The CISCRP team trains representatives from each host community – our clinical research navigators — to answer questions, address concerns and serve as personal guides throughout the exhibit. This vehicle continues to pick up momentum as a customize, engaging experience that not only educates but also demonstrates the value we place on the public as partners in the clinical research process. 

The program was recently recognized with SCOPE’s Participant Engagement Award and nominated with CARE’s Medidata Clinical Partnership of the Year as a new benchmark in partnering through collaborative clinical trial activity.  

We’re grateful for Janssen Research & Development, Pharmaceutical Companies of Johnson & Johnson’s continued support for expansion as we visit 15 events this fall and bring the traveling exhibit vehicle to five new cities per year over the next five years as part of our Journey to Better Health consortium. 

Over the fall season we traveled to and continue to travel educating and spreading clinical trial awareness: 



New York 

Mark your calendars and share these dates with colleagues, friends, and families.  We look forward to seeing you. For more information on these events, please contact Ellyn Getz at 

Written by Ellyn Getz, Associate Director of Development & Community Engagement 

CISCRP’s 2019 Perceptions and Insights Study

It is hard to believe two years have passed since CISCRP’s last Perceptions & Insights Study. We are excited to announce the 2019 study results are in! Over 12,450 people from around the world provided their opinions on various aspects of clinical research this year revealing significant new insights on patient engagement preferences.

Since 2013, CISCRP has conducted the Perceptions & Insights study on a biannual basis in order to monitor trends and identify opportunities to better inform and engage the public and patients as partners in the clinical research enterprise. Annick Anderson, Director of Research Services, notes, “The 2019 Perceptions & Insights Study offers the most comprehensive look to date at the ideal clinical trial experience from the global public and patient perspective – allowing stakeholders to model their patient engagement strategies most effectively around these patient needs.”

The 2019 study captured the experiences of over 3,600 study volunteers across diverse therapeutic areas, a significant jump from the 2,000 responses collected in 2017. The results continue to support that study volunteers generally have positive experiences, with 45% rating the care and attention they received during the trial as better than the care they would have otherwise received.

Did you receive any reports or updates on the results of the study 
once you completed the clinical research study?

Year Yes No

Sample size: 
1,828 in 2017; 3,054 in 2019.   
Base: respondents who participated in clinical trial, excludes “don’t remember” 

Similar to 2017, a significant percentage (96%) would be willing to participate in another study in the future – which is another strong indicator of overall satisfaction. However, areas of improvement remain, for example – a large proportion (39%) of study volunteers reported not ever hearing anything back from the site or the sponsor after their participation ended – demonstrating that post-participation engagement activity should be an area of focus for stakeholders. The study additionally takes an in-depth look at the burden of participation and identifying areas where attention is needed – for instance, close to 50% of clinical trial participants reported traveling at least 30 minutes or more one-way to the study clinic for their visits.

A preliminary review of specific sub-groups—such as minority populations and their motivations to participate in a clinical trial– shows that populations of African descent were significantly more likely to find it “very important” (73%) for their primary care physician or specialist to be aware of clinical research studies being conducted in their communities compared to their Caucasian counterparts (67%). This finding highlights the importance of enabling local health care providers to discuss clinical trial opportunities with their patients and driving inclusion of minority populations in clinical trials. Potential costs and reimbursements associated with clinical trial participation, as well as compensation for time-off from work were also particularly important to this patient population.

To access insights and findings from this recent study, visit CISCRP’s Perceptions and Insights webpage. The 2019 reports will be posted and available for download this Fall. These are available free of charge, however, interested parties are encouraged to provide a donation for the reports. CISCRP will also be presenting the results of the Perceptions & Insights study at industry conferences, company meetings, and other forums in the coming months.

Written by Jasmine Benger, Senior Project Manager of Research Services