More than a year after the pharmaceutical and biotech trade groups began voicing their opinions and crafting the next iteration of the US Food and Drug Administration’s (FDA) Prescription Drug User Fee Act (PDUFA), and both PhRMA and BIO have said they are pleased with the performance goals outlined by and for the agency from 2018 to 2022.
On 15 July, the US Food and Drug Administration (FDA) released for comment a 46-page document outlining how the agency will use the user fees from industry, breaking down not only the planned deadlines for new guidance documents and pilot projects but also recurring themes of recent importance, including patient input to the regulatory process, use of real-world evidence, biomarker qualification and increased pharmacovigilance.
Last week, FDA and industry stakeholders met for the first time to discuss these performance and procedural goals for PDUFA VI, with some new buzzwords thrown around, like, “Advancing Model-Informed Drug Development,” and changes to FDA-industry meetings.
Drugmaker AstraZeneca has already raised a couple of concerns and requested additional commitments from FDA on combination product reviews and for sponsors to gain more access to data in FDA’s surveillance system known as Sentinel.
Unlike AstraZeneca, the trade group PhRMA is not seeking any additions or revisions to the proposed performance goals, according to its comment on the performance goals dated Monday.
“Implementation of the proposed PDUFA VI performance goals will enhance FDA’s access to the tools, processes, and expertise necessary to keep pace with the latest scientific advances in drug development and regulation, potentially accelerating clinical development and reducing the time needed to bring new medicines to patients,” Sashca Haverfield, PhRMA’s SVP of science and regulatory advocacy, writes.
PDUFA VI also will continue to facilitate 21st-century approaches to drug development, PhRMA says, noting increases in FDA staff capacity and resources for the qualification of biomarkers; increased support for the appropriate use of model-informed drug development (MIDD) to reduce drug development and review times; and expanding the use of real-world evidence for regulatory decision-making – including from studies based on electronic medical records and patient registries.
PhRMA also says that implementation of the proposed PDUFA VI goals will streamline the review of combination products and improve coordination among FDA’s review centers.
“PDUFA VI will also enhance the Breakthrough Therapy Program by investing resources to appropriately prioritize the designation, development, and regulatory review of breakthrough medicines for patients with serious and life-threatening diseases,” PhRMA says. “The focus on rare diseases in past PDUFA performance goals has yielded significant advances in the development, review and approval of new treatments for rare diseases. For example, in 2015, 90% of all Breakthrough therapies approved by the Agency were for Orphan indications. Building upon the success of the Rare Disease Program under PDUFA V, implementation of PDUFA VI will continue to advance the development and timely approval of innovative medicines for rare diseases, including rare diseases in children.”
The biotech trade group, similarly to PhRMA, also offered no adjustments or new recommendations to the goals set forth by FDA and praised the agency for agreeing to evaluate the use of new trial designs, as well as for FDA’s “receptiveness to the use of biomarkers as surrogate endpoints,” which it says, “exemplifies the combination of communication and flexibility that is a hallmark of the PDUFA VI goals.”
Kay Holcombe, senior VP for science policy at BIO, also writes that the PDUFA VI commitments “build on the activities and successes achieved under PDUFA V, through further refinement of approaches to obtaining scientifically valid patient perspective information and data that are sufficiently rigorous to inform regulatory decisions and to be included as appropriate in the product label.”