The Prescription Drug User Fee Act (PDUFA) is one of the single most important pieces of legislation affecting the life sciences industry, and in particular innovative pharmaceutical and biopharmaceutical companies.
It is, at its most basic element, a trade-off between pharmaceutical companies and the US Food and Drug Administration (FDA). In the 1990s, an industry frustrated with the slow pace of drug approvals got together with FDA to float a proposal: If FDA would agree to hire more staff, adhere to timelines for reviewing drug products and make other improvements to regulatory capacity, the industry would fund the entire expansion through user fees.
In the ensuing decades, PDUFA - now on its fifth iteration as passed in the 2012 FDA Safety and Innovation Act (FDASIA) - has become the very model of success, acting as the basis for subsequent user fee programs for devices, generic drugs, biosimilars, animal drugs and other regulated products.
Keeping Track of Performance
But despite its successes, the user fee program has not been without its setbacks as well. A recurring question for the pharmaceutical industry is whether the program is working as well as it could be - a question spurred largely by the time it takes FDA to review various applications.
Now, however, the industry's two largest trade groups, the Pharmaceutical Research and Manufacturers of America (PhRMA) and the Biotechnology Industry Organization (BIO), have formally announced the launch of a new real-time database tracking system intended to allow them to recommend adjustments to the PDUFA program much sooner.
That database, known as the Industry PDUFA Tracking Database, is meant to be a response to a difficult problem. Because PDUFA is renewed on a five-year cycle, the opportunities to correct deficiencies in the process are few. In past years, explained Sascha Haverfield, vice president of scientific and regulatory affairs at PhRMA, both BIO and PhRMA queried their members for feedback on the application review process on an irregular basis.
But that left the industry reacting far more slowly to trends in FDA review times, leaving PhRMA and BIO less able to offer specific feedback to FDA on how to make improvements.
The new system is set to make some major changes to this paradigm, said Haverfield and Sara Radcliffe, executive vice president of health at BIO.
"In thinking about that reauthorization process, we realized that we, as stakeholders, had kind of gone through this approach of focusing on the reauthorization process of focusing on the last two years, but not focusing on a sustained engagement throughout the PDUFA V cycle … to make sure our aspirations are in fact being realized, and to think over the longer period about how we want to approach the next reauthorization cycle," said Radcliffe.
With the huge number of new commitments of both industry and FDA enacted under the newest PDUFA legislation, Haverfield noted that there are a huge number of unknown variables, and that FDA may or may not perform as anticipated.
"We can't just wait for five years until the next reauthorization cycle to make adjustments," he said. "We want the latest information and technology to advance biomedical information and to strengthen FDA's review process."
At the heart of BIO and PhRMA's interest is the review of new molecular entities (NMEs) and new drug applications (NDAs), both of which form the bulk of their member companies' profits. Those companies have a vested interest in making sure that FDA is able to complete as many decisions during a first-cycle review as possible, avoiding costly delays stretching into second-, third- and even fourth-cycle reviews.
The PDUFA Tracking Database is essentially a collection of information submitted from companies (not FDA) on their experience going through FDA's drug review process. As a drug goes through FDA's review program, companies will enter information into the database, a process that will take between one and three hours to complete.
Information will essentially include data about all relevant dates of action, including filings, meetings, information required, communication, interim responses and final decisions.
The database will result in two primary benefits for industry, said Haverfield. In regards to FDA's review process, Haverfield said that PhRMA and BIO will be able to assess - based on several years' worth of data-whether certain programs are working. If they aren't, PhRMA and BIO will then have the data necessary to sit down with FDA and make recommendations on how to improve the implementation of a particular program.
Second, industry will be able to determine if it is falling short on expectations in certain areas, such as not including proper information, allowing it to recommend corrections to member companies' behavior.
"The goal is to make sure that we are all doing a better job of providing the next PDUFA reauthorization with greater consensus and greater clarity about what's really needed to improve the process," added Radcliffe.
BIO and PhRMA said they have "a 100% commitment from all member companies" to participate in the database, which officially launched on 1 April 2013 and is being managed jointly by both industry associations.
Information from a 25 June 2013 interview with PhRMA's Haverfield and BIO's Radcliffe.