Genetic Alliance Pilot to Advance Patient-Focused Drug Development

Technological Approach to Increase Patient Engagement in FDA Initiative

01.28.14 | By Kristin Van Goor

When considering the benefits and risks of medicines, patients provide a unique perspective of unparalleled value.  A deep understanding of the patient perspective on benefits and risks throughout the drug development process has the potential to significantly advance the biopharmaceutical industry’s efforts to bring to patients new medicines that extend and improve the quality of their lives.  

Careful consideration of the patient perspective in regulatory assessment of the benefit-risk balance of new medicines is another critical component to more efficient and patient-focused drug development. 

Patient-Focused Drug Development

As part of the five year implementation of the Prescription Drug User Fee Act (PDUFA V), the Food and Drug Administration (FDA) is conducting quarterly, disease-specific meetings to gather patient input on the severity of the disease and the unmet medical need.  Systematic consideration of the views of patients – their perception of the acceptable balance of known and possible risks and benefits – has the potential to add crucial perspective when regulators are faced with difficult benefit-risk decisions.  

Genetic Alliance & PhRMA Collaboration to Utilize Technology to Gain Patient Perspective

Last year, PhRMA provided an unrestricted grant to Genetic Alliance to utilize the PEER platform, which was developed by Genetic Alliance in partnership with Private Access, to pilot a technology-enabled, crowd-sourcing approach to the collection of patient perspective information for purposes of informing benefit-risk assessments of new medicines.  Genetic Alliance selected advocacy organizations representing three disease areas that will be the focus of upcoming FDA Patient-Focused Drug Development meetings in 2014 and 2015 for the pilot so that collected information can complement the FDA’s ongoing initiative.

One of the primary goals of the pilot is to reach a broad and diverse group of patients, particularly those who traditionally have not engaged in the drug development and regulatory review process. 

Engagement in this process can be challenging, especially for patients who are very sick, as it requires awareness of FDA’s public meetings, travel to FDA’s offices in Maryland to participate in public meetings, and effort to provide input through formal comments to the FDA. 

Mobile Technology & Bilingual Content to Increase Diversity of Patient Participation

In order to increase the number and diversity of patients who are able to participate in patient-focused drug development efforts, Genetic Alliance and PhRMA  will enable the organizations participating in the pilot to utilize mobile technology on computer tablets to bring the PEER survey directly to patients in health care clinics where they receive treatment and translate the PEER surveys and associated content into Spanish to enable Spanish-speaking patients to participate in their preferred language. 

Dr. Bill Chin, Executive Vice President of Scientific and Regulatory Affairs at PhRMA, joined Sharon Terry, President and CEO of Genetic Alliance, and Teresa Barnes, Vice President of Patient Outreach and Advocacy at the Coalition for Pulmonary Fibrosis, on BioCenturyTV last month to discuss the importance of patient perspective and the goals of this pilot program. You can view the full discussion here.

Hearing from a wide array of patients about their range of experiences with a disease or condition is essential to gaining a robust and accurate understanding of the patient experience with a particular disease or condition.  


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