PhRMA Statement Regarding Reauthorization of the Prescription Drug User Fee Act (PDUFA-V)

The Pharmaceutical Research and Manufacturers of America (PhRMA) appreciates the opportunity to participate in this public meeting, and share its views on the reauthorization of the Prescription Drug User Fee Act (PDUFA-V) as outlined in the PDUFA-V performance goals letter that was published on September 1.  The PDUFA-V performance goals letter is the result of extensive, technical negotiations between the US Food and Drug Administration (FDA) and the innovative biopharmaceutical industry.  FDA’s process included unprecedented transparency and input from other stakeholders, including patient advocates, healthcare professionals, consumers and academia.

 

PhRMA represents the country’s leading pharmaceutical research and biotechnology companies, which are devoted to inventing medicines that allow patients to live longer, healthier and more productive lives. PhRMA companies are leading the way in the search for new cures. PhRMA members alone invested an estimated $49.4 billion in 2010 in discovering and developing new medicines.  Industry-wide research and investment reached a record $67.4 billion in 2010.

 

PhRMA strongly supports the original goals of PDUFA, which are to provide patients with faster access to innovative medicines, to preserve and strengthen FDA’s high standards for safety, efficacy and quality, and to advance the scientific basis for the Agency’s regulatory oversight.  PhRMA also strongly endorses the recommendations of the PDUFA-V performance goals letter.  This agreement – as drafted – will provide FDA with the resources and tools required to further enhance the timeliness, completeness, and efficiency of the drug review process.

 

PhRMA urges Congress to act expeditiously to reauthorize PDUFA, passing a bill that includes the carefully negotiated PDUFA-V performance goals letter.  PhRMA further urges Congress to refrain from adding additional provisions that could create excessive regulatory burdens or uncertainty for the Agency as well as industry and other stakeholders, or that could delay the legislative process.  Failure to reauthorize PDUFA in a timely manner would have an extraordinarily disruptive effect on the Agency, and impede patients’ access to new treatments.

 

PhRMA was an original participant in and supporter of PDUFA, beginning in 1992, when Congress first authorized user fees to help address what was then an untenable delay in regulatory review and approval of life-saving new medicines.  PDUFA has been a great success for patients – the tens of millions of Americans who rely on innovative drugs and biologics to treat disease and to extend and improve the quality of their lives.  The PDUFA user fee program provided FDA with the additional staffing and resources it needed to significantly reduce the timeframe for review of new medicines, while protecting public health by assuring the safety of these products.

 

Unfortunately, during the last reauthorization of PDUFA, Congress introduced substantial policy changes and provisions that significantly expanded FDA’s responsibilities and activities.  Certain provisions in the Food and Drug Administration Amendments Act of 2007 (FDAAA), while passed with good intentions, increased the Agency’s regulatory burden to the detriment of the efficiency and effectiveness of the drug review process.  As a result, the percentage of missed user fee goals has risen sharply in recent years.  Recommendations in the PDUFA-V performance goals letter seek to address some of these issues, as well as provide FDA with additional resources and scientific tools to hasten availability of new drugs and biologics, while maintaining the thoroughness and completeness of the review process.  

 

There are a number of important new commitments in the carefully negotiated PDUFA-V performance goals letter, including provisions to make the regulatory review of new medicines more efficient and timely, to advance regulatory science and modernize drug development, to improve benefit/risk decision-making, and to further strengthen FDA’s focus on patient safety.

 

Enhanced NME Review Program.  A new review program for new molecular entity (NME) drug and biologic applications is particularly significant for patients, as NMEs are novel compounds that have the potential to address unmet medical needs and advance patient care.  The NME review model acknowledges the increasing complexity of reviewing new drug applications (NDAs) and biologic license applications (BLAs), and provides for increased communication between FDA and drug sponsors prior to and during the drug review process.  A 60-day validation period will help FDA plan activities such as inspections and advisory committee meetings, and will accommodate iterative interactions between sponsors and the Agency.  As a result, the NME review program should improve the efficiency of the review process and reduce the overall time until new medicines become available to patients.  Specifically, it is anticipated that earlier and more comprehensive communication between the Agency and drug sponsors will improve the rate of “on-time, first-cycle” successes – that is, the number of new medicines that are fully reviewed and approved within the target timeframe following initial submission.  The success of the new review program and of the Agency’s ability to achieve its drug review goals will be independently assessed and publicly reported in 2015 and 2017.

 

Advancements in Regulatory Science.  Several new provisions in the PDUFA-V performance goals letter afford FDA with appropriate staffing and resources to develop, through public input, new tools and methods to integrate emerging scientific data and techniques into the drug development and review process.  These advancements in regulatory science will rely on engagement with industry, academia and other stakeholders to identify best practices so the Agency can provide appropriate guidance to stakeholders involved in drug development.  Provisions to enhance FDA’s regulatory review capabilities include:

 

Systematic Approach to Benefit-Risk Assessment.  A key provision in the PDUFA-V performance goals letter recognizes that the drug review process could be improved by a more systematic approach to benefit-risk assessment that fairly considers disease severity and unmet medical needs.  During PDUFA-V, the Agency will implement a structured benefit-risk framework, and hold public meetings to assess the application of such frameworks in the regulatory environment.  In addition, over the course of PDUFA-V the Agency will hold 20 public meetings with the patient advocacy community to identify disease states that – from the patient perspective – have considerable unmet needs.  Development and implementation of a patient-focused, structured framework for evaluating benefits and risks of new treatments will help inform the drug development process as well as ensure that regulatory decisions are consistent, appropriately balanced, and based on best science.

 

Modernizing Drug Safety System.  Finally, further enhancement and modernization of the FDA drug safety system under PDUFA-V will ensure that patient safety remains paramount.  The PDUFA-V performance goals letter provides for a public process to help standardize risk evaluation and mitigation strategies (REMS), with the intent to assess and reduce burden on healthcare providers and patients.  Additionally, FDA will continue to evaluate the feasibility of using the Agency’s Sentinel Initiative to actively evaluate post-marketing drug safety issues.

 

In summary, PhRMA fully supports the reauthorization of PDUFA, a program that has advanced public health by accelerating the availability of innovative medicines to patients while helping to ensure patient safety.  Additionally, PDUFA has strengthened the scientific basis of FDA’s regulatory review process through the development and application of new tools, standards, and approaches that facilitate assessment of the safety and efficacy of innovative drugs and biologics.  PDUFA-V will continue to provide FDA with the resources and tools that are essential to support patient safety and promote medical innovation through increased timeliness, completeness, and efficiency of the drug review process.

 

PhRMA encourages Congress to act responsibly to reauthorize PDUFA based on the negotiated PDUFA-V performance goals, and to resist encumbering the legislation with provisions unrelated to the Act’s original intent.