What review improvements are of concern to the FDA in initiating the PDUFA revision process?

The U.S. Food and Drug Administration (FDA) has launched a re-approval process for the Prescription Drug Manufacturers Payment Act (PDUFA) at a public meeting to prepare for the monthly discussions with industry that will begin in the fall, as well as the FDA's regular meetings with patient, consumer and health professionals.
PDUFA provides for periodic repeal clauses, which are re-approved by Congress every five years and are revised in the face of implementation, regulatory environment and changes in the pharmaceutical industry. The current scheme, PDUFAV., expires in September 2017, which means that PDUFAVI. needs to be revised by the middle of next year. Similarly, the FDA has held amendments to the Generic Drug Payer Act (GDUFA) and the Medical Device Use And Modernization Act (MDUFMA), both of which follow a similar revision schedule.to retain review talent
because the user-paid re-approval process coincides with the U.S. presidential election, this time the time is very tight. The new U.S. administration and the new Congress will begin operations in January 2017, and these user-pay agreements must be prepared to be reviewed by both current and future administration officials before being submitted to Congress.
the importance of the PDUFA program to the FDA need not be stressed, according to Stephen Ostroff, acting director of the FDA, and Robert Califf, deputy director. For the public meeting, Janet Woodcock, director of the FDA's Drug Review and Research Center (CDER), concluded by highlighting the importance of patient input into drug development and review, expanding the FDA's Sentinel program to strengthen post-market safety oversight and further strengthen regulatory science. The FDA's ability to attract top scientists is widely recognized in the industry as key to a more stable and efficient drug development and approval process. In response, Woodcock said CDER would remain committed to the recruitment and retention of key staff.
, director of CDER's Strategic Projects Office, is tasked with keeping PDUFA negotiations on track. She noted that PDUFAV. focuses on strengthening the approval process for important new drugs and increasing the number of approvals approved in the first cycle by enhancing communication with sponsors. She said high-level priorities would be retained to strengthen control and predictability of the drug review process and to maintain a reasonable and efficient cost structure that uses funds to hire and retain top talent for the FDA.Focus on approved evidence discovery
In addition, a range of initiatives by patient groups and professional organizations deserve PDUFA support: more validation of biomarkers and patient reporting outputs (PROs), development of paediatric and neonatal drugs, expanded use of registration data, data transparency measures, and greater consistency in the acceptance of fast-track review channels by CDER review authorities. Some commentators argued that the reasonableness of the 60-day waiting period should be further assessed, a provision in PDUFAV. designed to ensure that new drug applications are complete before they begin their review and whether they are eligible to accelerate or slow down their eligibility for review.
, expanding the use of real-world evidence to accelerate drug development is one of the most important topics of discussion in this PDUFA revision. Greg Daniel of the Brookings Institution advises on how PDUFAVI. should move from focusing on streamlining application reviews to focusing on uncovering clinical evidence and other data to support approval decisions and documenting post-market security. Allan Coukell of the Pew Charitable Trusts also stressed the value of better access to observations from databases of health needs outside clinical trials.
Kay Holcombe, of biotech industry group biotech, has set the goal of further integrating patient perspectives into drug development and regulatory decision-making by transforming an empirical review process into a data-oriented and systematic review process based on a structured benefit risk framework and clear FDA guidelines. Applicants also expect the FDA to make improvements in communication and practice across CDER review departments: a BIO survey found that half of organization members reported that interactions with the FDA were useful and productive, while the other half said "there is room for improvement."
FDA officials stressed that the user-pays program deals only with institutional processes related to drug development and review, and not with policy issues that require legislative or regulatory action. However, proposals to expand the use of real-world evidence and other data sources could be caught in a policy trap.
Holcomb raised another clear legislative issue: Industry wants Congress to make it clear that user payments will not be subject to future budget cuts to ensure the long-term stability of the PDUFA. During the automatic spending cuts three years ago, a series of federal spending cuts that automatically came into effect, some industries were unable to pay for them by the FDA, delaying the start-up and development of important regulatory science. "We can't let this happen again," Mr Holcomb insisted. (Biological Exploration)