FDA Commissioner Scott Gottlieb, M.D., released a statement on June 12, 2018 discussing new agency efforts. Throughout the statement, Gottlieb acknowledges how biological advancements have led to the development and FDA approval of targeted treatments for patients with specific molecular sub-types. Highly targeted treatments can address unmet medical needs and are primarily a result of scientific advancement. However, Gottlieb recognizes that these individualized treatments are “also a consequence of a bipartisan consensus that has supported investments in medical research and a modern regulatory process to advance safe and effective therapies.”1
For example, the Breakthrough Therapy designation helps accelerate treatments that show significant clinical benefits over the current standard of care. This designation exemplified FDA’s recognition that as science for drug development advanced, FDA needed the capability to work closer with sponsors. In the treatment of diseases like cancer, the benefits of the more efficient approaches can be seen. 1
Gottlieb states that the FDA learns, “through scientific advances, but also by listening to patients.” Gottlieb continues, “our work demands that we must continue to reflect on how we can make the science of drug development and review more modern and more patient-centered, so that approved products impact the metrics that real-world patients and families value most.”1He believes that ongoing engagement with the patient community is key to creating products that impact real-world patients.
FDA has made it a priority to partner with companies on gathering information from patients about their views and needs. Furthermore, FDA is focusing on building the tools that are needed to collect patient input and create meaningful data.
According to the statement, FDA has held Patient Focused Drug Development (PFDD) meetings in more than 20 disease areas to date, including “opioid use disorder, autism, HIV, Parkinson’s disease and various conditions involving pain.”1Gottlieb mentions that the meetings have helped FDA’s professional staff develop a deeper understanding of patient and caregiver experiences. Another part of the effort involves educating companies about rigorous approaches to obtain and incorporate the input into product development.
The FDA draft guidance issued on June 12th informs patients and product developers on those principles, notes Gottlieb. Gottlieb explains that this “set of guidances describes how patient experience data and other relevant information from patients and caregivers can be collected and used for medical product development and regulatory decision-making.”1The guidance issued also discusses methods on how to operationalize and standardize the collection, analysis, and distribution of patient experience data.
To conclude the statement, Gottlieb notes that the agency will continue to build upon the efforts, especially as the nature of drug development becomes more targeted. He also looks forward to receiving public comment on the draft guidance.
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1https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm610509.htm