Patients, PDUFA, Pandemics to Shape R&D in 2016

Patient perspectives on treatment preferences and risk-benefit tolerances will continue to dominate discussions about developing new drugs and medical products, as scientists increasingly look to tap genetic information to identify personalized therapies for critical diseases. FDA approved 41 new drugs and 10 biologics in 2014, and this pace is expected to continue in the coming months. Regulatory flexibility and the application of expedited review programs are key to speeding access to new treatments for serious conditions, said FDA acting commissioner Stephen Ostroff, in a speech to the RAPS Regulatory Convergence conference in October. He emphasized the importance of patient input in accelerating drug development, and how such considerations will continue to be examined through FDA’s Patient-Focused Drug Development (PFDD) initiative.

More than 20 PFDD meetings have been or will be scheduled through next year by the Center for Drug Evaluation and Research (CDER) to more formally incorporate the “patient voice” into the process for developing new treatments for specific conditions, ranging from narcolepsy and sickle cell disease to heritable bleeding disorders and Huntington’s disease. Input from this program will further help FDA and sponsors better understand the nature of a condition, current treatment options, areas of unmet medical need, and strategies for developing rigorous, objective methods for reliably measuring patients’ experiences and perspectives.

Ostroff also highlighted initiatives to advance genomic sequencing and to establish more predictable pathways for developing combination products. These programs, along with the  development of biomarkers and surrogate endpoints, he noted, can stimulate biomedical innovation and shorten drug development timeframes. And these efforts support President Obama’s Precision Medicine Initiative, which aims to generate greater understanding of the underlying causes of disease and lead to the development of more targeted breakthrough therapies to treat cancer and other critical conditions.

Patient engagement will remain an important topic as FDA and biopharmaceutical companies negotiate the next round of the Prescription Drug User Fee Act (PDUFA VI) in the coming months, as well as fees for medical devices, generic drugs, and biosimilars. The PFDD is funded by the current PDUFA V program, and both FDA and industry aim to continue integrating patient perspectives into drug development and regulatory decision-making. The user fee negotiators also are examining strategies to ensure the long-term stability of the fee programs, to make funding more predictable for FDA, and to enhance investment in FDA information technology and the agency’s workforce.

Ethics & integrity

The emergence of more complex and targeted biopharmaceuticals, the globalization of drug development and production, and pressure on sponsors to bring new therapies to market more quickly all raise concerns about how well investigators and sponsors ensure adherence to study protocols, protection of research subjects, and generation of accurate and reliable data. FDA is reorganizing its Bioresearch Monitoring program (BIMO) for overseeing and inspecting clinical research sites to provide more expertise in evaluating research operations and more consistent oversight of sites in different regions.

An agency-wide working group is examining ways to restructure BIMO as part of FDA’s Program Alignment initiative for transforming how its field force ensures compliance with agency rules and regulations. FDA officials look to move forward in 2016 with a proposal to form a specialized BIMO group of inspectors with special training in compliance with good clinical practices (GCPs) for all medical product areas and in issues related to ensuring the integrity of data filed to support product registration in the U.S.

These initiatives also stand to support increased transparency in biomedical research involving human subjects, even for studies that fail to support new drug applications filed with FDA and other regulatory authorities. In the U.S., the government will move forward with a major proposal for updating the “Common Rule” governing all human subject research funded or regulated by federal agencies. New policies governing the privacy of patient information, future use of clinical specimens, and data access and disclosure will have a broad impact on clinical research operations.

In the wake of the Ebola outbreak, the research community is examining policies and practices for dealing with emerging infectious disease. These include methods for rapidly evaluating investigational products during a public health emergency and the challenges in providing early access to therapies before they are fully characterized and evaluated. Important issues include the use of randomized and controlled clinical trials to treat lethal diseases, and strategies for the rapid design and conduct of studies that can provide valid scientific information in critical situations, for both preventive and therapeutic treatments. Scientists continue to emphasize the importance of adhering to ethical standards in conducting research in response to disease outbreaks, and are working to better prepare public and private researchers to deal with future epidemics.