PDUFA/FDASIA Implementation Update
PDUFA/FDASIA Implementation Update
06.13.13 | By Stephanie Fischer
As you may know from reading past Catalyst posts , the Prescription Drug User Fee Act (PDUFA ) was reauthorized last July when the Food and Drug Safety and Innovation Act (FDASIA ) was signed into law.
PhRMA remains deeply concerned about the impact of sequestration on FDA’s ability to fulfill its critical public health mission by fostering timely patient access to safe and effective new medicines and advancing regulatory science - the two key goals of PDUFA.
Patient-Focused Drug Development
One of the elements of PDUFA V which has attracted a lot of attention in the patient community and from industry is the FDA’s commitment to Patient-Focused Drug Development, an initiative to gather the patient perspective on the severity of their condition and their unmet medical needs. FDA officials have stated the intention to incorporate the patient perspective into their review of new medicines.
In the PDUFA V agreement, FDA committed to hold 20 Patient-Focused Drug Development meetings in five years. After receiving feedback at a public meeting  and through comments, FDA finalized the list of diseases to be the focus of the first  three years of meetings. The first meeting was held in April and focused on chronic fatigue syndrome and myalgic encephalomyelitis. Later this month, FDA will host meetings on HIV  and lung cancer. A date has not yet been set for the final meeting of the year, which will be on narcolepsy.
Benefit-Risk Assessment Framework
The Patient-Focused Drug Development initiative is part of FDA’s commitment in PDUFA V to the development of a structured Benefit-Risk Assessment Framework  to be used in the human drug and biologic review processes.
In our comments , PhRMA stressed the importance of fully integrating use of the structured benefit-risk framework within FDA’s regulatory review to advance the Agency’s efforts to achieve greater consistency in the review and decision-making process while supporting systematic consideration of the patient perspective.
Enhanced Communication and Transparency during NME Review
A review program  for new molecular entity (NME) new drug applications (NDA) and original biologic license applications (BLA) was established under PDUFA V to promote greater transparency and provide more opportunities for communication between FDA and biopharmaceutical companies. The goal is to increase the efficiency and effectiveness of the first review cycle of a new medicine, which will translate into more timely access for patients to safe, effective, and high quality new medicines.
Last month, FDA announced  that it had approved the first new medicines under the NME review program, a new therapy for advanced prostate cancer, more than three months ahead of its prescription drug user fee date. We hope this announcement is the first of many on timely review and approvals.
While not part of the PDUFA V agreement, the creation of a Breakthrough Therapy designation  was included in FDASIA and is available only for therapies to treat serious or life-threatening conditions. FDASIA also enhanced the existing Accelerated Approval Program .
As I wrote in a previous post , FDA has indicated that there is more engagement with senior officials during the review process for products that have been designated as Breakthrough Therapies . Of the 59 requests for Breakthrough Therapy designation  received as of May 31st, 20 were approved, 20 denied and the remaining 19 were pending.
PhRMA looks forward to the anticipated release by FDA of an integrated draft guidance for industry on the Breakthrough Therapy, Accelerated Approval, enhanced Accelerated Approval and Fast Track programs. We remain committed to working with all stakeholders to explore ways of continuously improving the efficiency and effectiveness of the regulatory review and approval process to bring new medicines to patients in a timely manner.