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,

Making It Clear: Lay Language Summaries

When a patient has invested their time and energy into participating in a clinical trial, it’s of vital importance to provide information about the results in an easy-to-read format. CISCRP’s Health Communication Services (HCS) group specializes in the “translation” of clinical research documents into plain language to help patients understand complex clinical trial information. Among the most common of these is the trial results summary. With the impending implementation of EU CTR (536/2014) and as sponsors increasingly recognize the importance of patient engagement, many companies are beginning to prioritize the development of these summaries, sometimes referred to as “lay language summaries.” These documents play a critical role in the effort to keep patients and the public informed about clinical research and to demystify the process.

Producing a plain language trial results summary is a multi-step process. At CISCRP, the first step is the development of a robust summary template that can be customized from study to study. The CISCRP template is structured to meet EU CTR requirements and incorporate plain language best practices. Yet, it is flexible enough that it can be structured to align with a sponsor’s company requirements and preferences. To keep templates up to date and incorporate lessons learned, CISCRP updates our own template and our clients’ customized templates annually.

When creating content, a patient-centric focus is key. Summaries are most effective when written at a 6th to 8th grade reading level, and it is vital that the content be unbiased, clearly written, and easy-to-understand. During the development process, CISCRP engages volunteer Editorial Panel members to review and comment on a draft of the summary to ensure readability. These panels consist of members of the public (whether or not they are patients), health professionals, and patient advocates. Their feedback is crucial to ensuring that the summary is understandable and strikes the proper tone.

Sponsors working with CISCRP have their option of two different service models: full-service, or Editorial Review only. In the latter model, the sponsor writes the summary and engages CISCRP to perform an editorial review of the draft. This includes feedback regarding plain language best practices and EU CTR requirements. This may also include the implementation of requested graphic design elements.

Some sponsors prefer to pilot their initial summaries using the Editorial Review only approach. This allows them to utilize CISCRP’s plain language expertise and volunteers, while still “owning” the writing and stylistic elements of the summary.

The full-service model, however, engages our plain-language trained staff to assume a more active project management role. This gives valuable time back to an organization’s internal medical writers and creates efficiencies by allowing the writer and editor to communicate more directly. In the full-service model, CISCRP is responsible for the authoring, plain-language editing, content and editorial QC, and graphic design of the summaries. The sponsor has two opportunities to review and comment on the document to ensure it is scientifically accurate. This approach allows each party to focus on their respective areas of expertise, collaborating to produce a document that is plain language friendly and informative.

Producing patient-facing collateral that is transparent and direct is an essential part of the conduct of clinical research. Learn more about CISCRP’s patient engagement and education materials.

Supporter Spotlight: CSL Behring

Although the public acknowledges the importance of clinical research in developing new medicines, general awareness of clinical trials and understanding of what it means to participate in them remain low. In a global study of more than 12,500 people, The Center for Information and Study of Clinical Research Participation  (CISCRP), found that while the majority of people consider clinical research to be very important, most do not regularly discuss clinical trials with their healthcare provider. And nearly two-thirds (65%) believe they could convince their doctor that they should participate if a relevant clinical trial was identified.

Most people consider clinical research studies to be relatively safe.  Access to clinical trials, however, is challenging for certain patients based on socio-economic status and ethnicity. Rodney Winley, Senior Director, Patient-Focused Clinical Trials at CSL Behring, sees awareness-building as only half the battle. “The other challenge,” he says, “is understanding the actual barriers to clinical trial participation and helping patients navigate through those barriers. It’s our obligation as the developers of treatments for patients to do our part in making clinical trials available to everyone.”

Many initiatives are underway to help address health disparities and improve health equity. While there has been a significant increase in patient-facing clinical trial information online, most efforts are tied to company-specific programs and attempt to channel patients into specific trials.

CSL Behring was the first of ten companies to sign on to CISCRP’s 5-year AWARE Industry Consortium. Rodney sees this consortium as a way for the biotechnology industry to present a “unified front” to improve clinical research awareness, accessibility, and ultimately participation levels. “We believe that taking the message into communities is a much more proactive and personable approach which will increase the likelihood that we will be able to positively engage patients and caregivers.”

CSL Behring is committed to ensuring that the patient voice is incorporated across all aspects of the clinical trial lifecycle. Their goal for 2020 is to continue to listen to patients’ and caregivers’ needs in order to create a positive and supportive clinical trial experience, co-create a study experience that recognizes patients as partners and an extension of their clinical study teams, as well as improve clinical trial transparency by sharing information that is meaningful and understandable to patients and caregivers before, during, and after clinical trial participation.

In addition to their participation in the AWARE Industry Consortium, CSL Behring assembles a team to participate in CISCRP’s Medical Heroes Appreciation 5K events annually, works with CISCRP to translate clinical trial documents into plain language summaries, sponsors projects with our Research Services team, and collaborates with us to host patient-facing educational content for their MyTrials Hub. They also collaborate with other non-profits to further drive awareness about clinical research and recognize study volunteers by donating flu vaccines to underserved communities on behalf of their clinical trial participants.

We thank CSL Behring for their support and dedication to CISCRP’s mission, for applying patient-focused principles at each stage of the drug development process, for recognizing study volunteers as team mates in the drug development process, and for improving access to information and study opportunities among diverse communities around the world.

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

CSL Behring is a global biotechnology company driven by its promise to save lives. The company discovers, develops and delivers innovative therapies to treat individuals with serious and life-threatening medical conditions. The organization collaborates with patient and biomedical communities to improve access to therapies, advance scientific knowledge and support medical research.

CISCRP’s AWARE Industry Consortium builds on CISCRP’s 18-year experience producing and hosting internationally recognized live educational events – called AWARE for All programs, throughout the United States and parts of Europe. Each event engages a local planning committee comprised of members of the public, patients, advocacy groups, healthcare and clinical research professionals, offers educational presentations and discussions, as well as free health screenings and information about local clinical trial activity. Learn more.


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.” 

Thank You for Your Generous Support

Dear Readers,

I want to take a moment to thank you on behalf of CISCRP for your generous support last year.  Your time, sponsorship and donations, ideas and encouragement enabled us to achieve unprecedented growth and momentum.  A few highlights — in 2019, CISCRP:

  • Produced four (4) AWARE-for-All educational events in diverse cities throughout the U.S.
  • Facilitated thirty-three (33) patient input panels to solicit feedback on draft protocol designs, clinical research documents, communications, and new clinical trial technologies and solutions
  • Provided over 700 clinical trial result summaries and thousands of other communications — in plain easy language — for patients and their families
  • Raised clinical trial awareness with the Journey to Better Health traveling exhibit in three (3) major urban communities – Atlanta, Chicago and New York
  • Reached millions of people through our National Media Awareness campaign and expanded our focus to include a special patient diversity initiative
  • Assisted more than 2,500 patients and their families in search of information on relevant clinical trials underway

Thank you for collaborating with us to continue our efforts to raise clinical research literacy and improve patient engagement.  Together we make a difference!


Joan A. Chambers

Senior Director, Marketing & Outreach

PS – please give me a call or visit to learn more about supporting our programs and initiatives

From the Editor

Dear Readers,

It’s official.  The February 2nd Groundhog Day report has forecasted an early Spring!  And we’re excited to share updates on our upcoming 2020 initiatives.  We’ve also added a new feature for our circle of supporters to announce their new patient education and engagement initiatives.

First, a quick reflection on some 2019 highlights.  CISCRP:

·        Held its first Patient Advisory Board in Asia and initiated its capabilities in conducting patient input panels throughout the region

·        Collaborated with ASH Research Collaborative to engage adult and pediatric sickle cell disease communities in a series of national interactive and educational workshops

·        Collaborated with NCI, The Hope Foundation for Cancer Research to develop a plain language clinical trials summary template

·        Reached millions of people through its National Media Awareness campaign and expanded its

clinical research education channel into minority and underserved patient communities

·        Organized and launched the Aware Industry Consortium among 10 industry leaders to produce AWARE for All educational events and the Journey to Better Health traveling exhibit in major cities throughout the US

In this edition, we are honored to profile Sandy Morris in our Medical Hero Spotlight. Sandy shares her story and her clinical trial journey with ALS.  We also highlight our Health Communication Services team now collaborating with several dozen sponsors and CROs to provide clinical trial result summaries and many other communications — in plain, easy language and in print and digital formats — for patients and their families.

If you have not yet had an opportunity to review the results of our 2019 Global Perceptions & Insights Study results, we have included two articles—one summarizing key findings and the other elaborating on patient preferences and experiences with convenience-enhancing technologies designed to minimize study volunteer burden.

Our 2020 calendar of events is expanding rapidly. Our first AWARE-for-All event is scheduled for April 2nd in Miami and a second on May 20th in Chicago. Later in the year we will be in Philadelphia, Raleigh Durham and Los Angeles.  Our annual Medical Hero 5K run/walk, in collaboration with DIA, is scheduled for June 15th in Washington, DC.  Stay tuned for our 2nd annual Appreci-a-thon, a virtual fitness challenge scheduled for the full month of April. I invite and encourage you to visit our event and conference pages on our web site at for more information.

Lastly, a huge thank you for your ongoing support.  Our impact and growth would not be possible without your time, sponsorship and donations, ideas and encouragement. Together, we are making a difference.

We look forward to keeping you informed on our progress in 2020.

Warm regards,

Joan A. Chambers

Convenience Enhancing Solutions for Clinical Trials

In ongoing efforts to minimize study volunteer burden, sponsors and other stakeholders have invested heavily in the implementation of various convenience enhancing solutions over the past several years – ranging from the use of technologies such as wearables, smartphone apps and electronic tablets to supporting study volunteers with transportation needs and the provision of child care.  This investment is reflected in the results of CISCRP’s latest Perceptions & Insights (P&I) study, a global online survey of over 12,450 individuals, including over 3,650 prior study volunteers.

The 2019 P&I study shows evidence of growth in the use of certain initiatives in clinical trials since 2017:

·        the use of text messaging has grown from 18% in 2017 to 21% in 2019

·        the use of smartphone apps increased from 10% to 15%

·        wearable device use grew from 8% to 13%

·        concierge services (e.g., transportation to/from study clinic) increased from 7% to 11% in 2019 (see Chart 1).


The most frequently reported initiative was patient experience surveys at 29%.  Notably, almost 1 out of every 3 people indicated not using any of these initiatives, although that proportion has decreased since 2017 when 40% reported not using or not being offered any of these services during their clinical trial.  Usage varied among different subgroups of respondents. The use of electronic solutions such as smartphone apps and wearable devices, for example,  was more prevalent among younger populations, whereas older adults were more likely to report not using any of these services.

Chart 1

Stakeholders have been particulalry interested in better understanding the impact of these various initiatives on the patient experience.  The latest P&I study findings reveal that these implementations are indeed positively influencing clinical research participants’ medical journeys.  Among study volunteers that used these services, a significantly higher proportion reported that their clinical research study met or greatly exceeded their expectations, compared to individuals that did not use any of these services. 

The provision of childcare had the greatest impact on patient perception, with 40% indicating their experience exceeded expectations, compared to just 14% among those that did not use any service (see Chart 2).  These individuals also rated the care and attention they received during the study higher.


The expansion of these service offerings will assist in reducing the burdens of clinical trial participation.

Chart 2

The 2019 Perceptions & Insights executive summaries are available free of charge on the CISCRP website under Research Services.  CISCRP will also be presenting the aggregate results of the Perceptions & Insights study at industry conferences, company meetings, and other forums in the coming months.


For a more detailed, in-depth report with a facilitated on-site or webinar meeting, please contact Annick de Bruin at or 617-725-2750 x400.

CISCRP Releases 2019 Perceptions & Insights Study

CISCRP is pleased to announce that executive summaries featuring the results of the 2019 Perceptions & Insights (P&I) study are now available on the CISCRP website.  Since 2013, CISCRP has conducted the Perceptions & Insights study biannually in order to monitor trends and identify opportunities to better inform and engage the public and patients as partners in the clinical research enterprise.

The latest 2019 study contains insights from over 12,450 individuals around the world, including over 3,650 prior study volunteers.  The 2019 executive summaries are organized around 4 key topic areas:

 1) General Perceptions & Awareness

2) Deciding to Participate

3) Patient Engagement Preferences

4) Participation Experiences

General Perceptions & Awareness

While the public continues to acknowledge the importance of clinical research in identifying new medicines, general awareness of and participation in clinical research studies remain low for a variety of reasons. Many initiatives are underway to help change this paradigm.  In recent years, patients and their loved ones have played a more active role in helping to identify the outcomes most relevant to them and by suggesting ways to minimize the burden of clinical trial participation for study volunteers.  Patient advocacy groups and local communities, in partnership with other industry stakeholders, are also developing innovative campaigns and programs to raise awareness.  The hope is that these initiatives can help accelerate the pace at which new treatments are developed and become available to patients. 

CISCRP provides an overview of the analyzed results of the latest global survey of general public and patient perceptions about clinical research.

Deciding to Participate

The decision to participate in a clinical trial is highly personal and each pathway to that decision is different.  Patients and intimately acquainted individuals who provide assistance and support to them often turn to their personal doctors for advice, therefore gaining a better understanding of the role healthcare providers play in the decision-making process. A majority consult the wealth of information that is available in the public domain.  Oftentimes, this content can often be unreliable and overwhelming.  Consequently, providing prospective study volunteers and their loved ones with  comprehensive information from a trusted source has become increasingly important.

CISCRP details a high-level overview of the results of the latest global survey of the general public and patient perceptions about clinical research – including valuable insights on the decision-making process.  The findings illustrate what matters most to patients and offer ways to best support prospective volunteers during this process.

Patient Engagement Preferences

Patients and their loved ones often have different needs and preferences when it comes to clinical trial participation. Each patient’s clinical trial journey is unique – with each person balancing varying degrees personal commitments and work responsibilities. While myriad new patient engagement initiatives are now being offered as part of clinical research studies, what may work for one person may not work for another. The ability to have various options capable of accommodating different lifestyles is critical.

CISCRP provides an overview of the analyzed results of the latest global survey of the general public and patient perceptions about clinical research – including valuable insights on patient engagement preferences among various sub-groups of patient populations, as well as receptivity to new clinical trial models.

Participation Experiences

The advent of emerging technologies and convenience enhancing solutions is profoundly changing the conduct of clinical research and increasing access to clinical trials among broader patient populations.  Despite these new solutions, the traditional burdens of participation – such as travel to the study clinic and the duration of study visits, persist.

CISCRP provides an overview of valuable insights from over 3,650 prior study participants.  The findings will help with the development of Informed Consent strategies and identify other ways to minimize study participation burden, as well as highlight opportunities to maintain engagement post-participation.

The 2019 Perceptions & Insights executive summaries are available free of charge on the CISCRP website under Research Services.  CISCRP will also be presenting the aggregate results of the Perceptions & Insights study at industry conferences, company meetings, and other forums in the coming months.

For a more detailed, in-depth report with a facilitated on-site or webinar meeting, please contact Annick de Bruin at or 617-725-2750 x400. 

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