First Technology Pilot Program Shares Perspective of Sickle Cell Disease Community with FDA

Incorporation of Patient Perspective on Disease and Treatment Options Critical in Drug Development and Review

06.18.14 | By Kristin Van Goor

In the guest blog below, Michele Lloyd-Puryear, Special Consultant to Genetic Alliance, discusses key findings from a patient-driven data collection effort intended to complement the Food and Drug Administration’s (FDA) patient-focused drug development initiative which was part of the most recent reauthorization of the Prescription Drug User Fee Act.  While initial findings were submitted to the FDA docket (with the consent of survey respondents), the survey remains open for individuals with sickle cell disease.

Use of technology-enabled tools, such as Genetic Alliance’s Platform for Engaging Everyone Responsibly (PEER), to gather patient input is an important first step in trying to understand the patient perspective about disease and treatment options in a more systematic, science-based manner.  To achieve the goal of patient-focused drug development, we must continue to learn about the impact of diseases and conditions on daily life from those living with the disease or condition and all stakeholders must come together to translate these learnings and knowledge into tangible drug development tools that can bring innovative and life-changing medicines to patients in need.

This pilot is the first of three in a joint initiative between Genetic Alliance and PhRMA. Future surveys will focus on idiopathic pulmonary fibrosis and irritable bowel syndrome, gastroparesis and gastroesophageal relux.

For the first Patient-Focused Drug Development pilot project, Genetic Alliance, in collaboration with PhRMA, partnered with four community-based organizations concerned with patient experience and their needs and treatment goals for sickle cell disease: North Alabama Sickle Cell Foundation, Inc.; Sickle Cell Disease Association of America Southern Connecticut; William E. Proudford Sickle Cell Fund; and Citizens for Quality Sickle Cell Care

Sickle cell disease, a genetic disease, affects approximately 100,000 Americans and millions of people throughout the world.  Sickle cell disease is a major public health concern and the need for innovative medicines to treat this disease is great.  It is hoped that the information provided by patients and their families and gathered by Genetic Alliance, in partnership with the community-based groups, will further the understanding of sickle cell disease, the daily experience of and challenges for patients with the disease, and the patient perspective on the need for new treatment options.

Using the PEER system, we were able to engage a diverse group of individuals with sickle cell disease and their families.  The PEER system allows each participant to select who can access their data and allows them to modify the access over time as their preferences change.   In addition, participants were able to not only address the questions put forward by the Food and Drug Administration (FDA) but also able to propose new questions and issues that are important to the community.

The information gathering process engaged more than 120 individuals across the country.  The top two symptoms experienced by the participants included pain and fatigue or tiredness.  More than half of those surveyed reported that symptoms affected their ability to sleep through the night, to participate in physical activity, and to withstand weather changes.  Symptoms also impacted the ability to complete tasks at home, enjoy life, and/or attend work or school.  On an individual’s worst days, any type of physical activity or activity related to cognition was affected.  Of note, because individuals do not see themselves as sickle cell patients per se, respondents focused more on how treatments affect them, rather than how the disease affects them.

Nearly half of participants reported fatigue as a side effect of their treatment plan that negatively impacts their daily life the most, followed by pain, constipation and insomnia.  Participants indicated that being treated like a drug seeker was the most common problem when trying to comply with their prescribed treatment plan.  Individuals also noted the frequent clinic visits and blood draws as problems. 

A great majority (89%) of individuals would either agree or possibly agree to participate in a clinical trial even if the trial would not directly benefit themselves.  However, if participation in the clinical trial required the individual to stop their current treatment, the willingness to join a clinical trial drops by approximately ten per cent.  Factors that would improve clinical trial participation included being matched with another trial participant for support (87%), a clear explanation of the value of the trial (77%), receiving a summary of the outcome (65%), and frequent communications about trial milestones (56%).

The sickle cell disease community is passionate about finding efficacious treatments for sickle cell disease. They are committed to working with industry and federal agencies, such as FDA and the National Institutes of Health, to achieve that goal within a participatory and patient–centered framework achieved through partnerships with individuals affected by the specific disease being targeted for drug development. Toward that end, they are willing to share clinical data about themselves and their disease.  However, our work shows that they want to participate fully in the data collection in a dynamic manner that is mindful of privacy.



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