Engaging Patients in Drug Development

To further the possibility of FDA guidance to encourage product sponsors to engage patients throughout the drug research and development lifecycle, the National Health Council and the Genetic Alliance convened a 12/9/15 meeting with 36 stakeholders to inform the scope and content of a proposed FDA guidance document. The meeting followed one in 3/2015 that brought together a stakeholder group including FDA to identify barriers hindering progress on the issue.

The two groups say that participants in that first meeting identified the lack of guidance from FDA on patient engagement efforts, particularly how regulations governing pre-approval promotion apply to pre-approval patient engagement to collect patient-provided information, as a fundamental barrier to patient involvement in research and development. “Participants indicated that the risk, perceived or real, of violating these regulations may discourage product sponsors from proactively and meaningfully engaging patients in innovative ways and to the fullest extent possible,” the groups say. “They agreed that clarity from FDA could alleviate uncertainty and unpredictability, facilitate meaningful patient engagement, allow sponsors to more accurately estimate the return on investing in patient engagement, and reduce skepticism regarding patient engagement benefits. In addition, stakeholders also welcome FDA guidance regarding how the agency will evaluate and use submitted patient-provided information.

A report on the 12/2015 meeting issued 5/4 says that key findings on where FDA guidance is needed, in order of priority, included: 

•          gaining alignment and clarity on key terminology;
•          demonstrating that FDA encourages collection and use of patient-provided information in drug development;
•          providing clarity for stakeholders on characteristics of appropriate and inappropriate patient interactions for collecting patient-provided information, with examples provided in the guidance;
•          providing clarity on acceptable methods and presentation of data and analyses for FDA review of patient-provided information;
•          emphasizing that the context in which a particular interaction with a patient occurs is important to assessing the appropriateness of the interaction and, thus. Context must be considered when determining methods and approaches to interacting with patients;
•          promoting transparency and communication across all aspects of patient-focused drug development to increase information-sharing and collaboration among patients, stakeholders, and FDA, including how FDA interacts and shares collected data with patients and other stakeholders; and
•          enhancing clarity in the agency’s approach to evaluating and using patient-provided information in regulatory decision-making and product-labeling.

 Participants suggested that the themes likely capture more content than what could be included in one guidance document and that multiple draft guidances might be appropriate. The report includes an outline for an initial draft on the first three themes.