Earlier in the week of September 24th, the Food and Drug Administration (FDA) posted Federal Register notices to implement two provisions of the recently enacted user fee law that seeks to enhance the role of the patient voice in the therapy development process. The notices represent initial but important milestones, particularly for patient advocacy organizations and other stakeholders that fought hard to secure these provisions in the law.

In one notice, FDA is seeking public comment as it develops a list of 20 disease areas that would be the focus of meetings designed to enhance the benefit/risk assessment used to evaluate a potential therapy. The effort, part of the agency's larger patient-focused drug development initiative, recognizes that patients – particularly those suffering from serious and life threatening diseases that lack adequate treatment options – bring a critically important perspective to shaping the benefit/risk paradigm that FDA employs in considering new approvals.

The initial notice seeks feedback on a list of nearly 40 disease areas nominated by FDA to be the subject of one of the 20 meetings over the next five years. It also seeks nominations of additional diseases not on the FDA's list and invites comment on the criteria used to select disease areas. Comments are due November 1, and advocates can also choose to voice their comments at a public meeting in late October.

In the second notice, FDA is seeking patients and patient advocacy organizations willing to participate in periodic consultation meetings focused on important process issues to help maximize the patient voice in a systemic manner. Patients interested in participating in such process meetings must inform the FDA of their interest by early October.

In the coming months, FDA is expected to issue other notices, including draft guidance to implement provisions of the user fee law intended to accelerate the review process, particularly of potential therapies to treat serious and life-threatening and rare diseases. Stakeholders, including industry, academia and patient advocacy organizations, will want to closely follow this process and provide FDA with thoughtful comments throughout to shape the final provisions.

FaegreBD Consulting's Health and Biosciences Practice Team, having advocated on behalf of a number of these provisions within the recent user fee debates, has deep policy, program development and regulatory expertise, particularly in the rare disease space. Our team will be engaged throughout the PDUFA guidance and implementation process on these key issues and can support stakeholders of all types to help shape key regulations and guidelines and navigate the process within FDA.

For more on our rare disease therapy development services, visit our rare disease expertise page.