One of the key tasks of the incoming Congress is going to be reauthorization of PDUFA—the Prescription Drug User Fee Act. This Act provides key resources for the FDA to review and approve safe and effective new products. PDUFA expires in September in 2012, but the heavy lifting is going to begin in 2011, as the new GOP Congress selects Chairmen, picks keys staff, and starts setting its agenda for the 112th Congress.
A new report by Price Waterhouse Cooper and BIOCOM reveals that there is a great deal that needs to be done to fix the FDA, and the PDUFA reauthorization process gives Congress an important mechanism for making the necessary fixes. The PWC report took the form of an anonymous survey of life science executives, which is important because industry people are often reluctant to go on the record with concerns about FDA—for understandable reasons. According to the report, in fact, 58 percent of executives think that political considerations have too much influence on the FDA approval process, which explains why the best way to get their input is in this anonymous fashion.
The report highlights some of the biggest challenges the life science faces with respect to the FDA. According to the report, getting approval for a new product is a 12-year, billion dollar process, which, coupled with the uncertainty of getting approval, is an enormous disincentive to investing in the creation of new products. This problem is widely recognized on both sides of the aisle—even HHS Secretary Kathleen Sebelius has acknowledged the difficulty posed by “bottlenecks” in the life sciences development process. Other complaints voiced in the report were that the FDA changes its position during reviews, and that the FDA has failed to approve products because of a lack of resources. In addition, 92 percent of industry execs surveyed felt that FDA was not doing enough to advance new frontiers such as personalized medicine, and over half felt that FDA is incapable of implementing the Critical Path initiative to expedite the review and approval of high priority products. Despite these serious concerns, the industry is often timid in its PDUFA requests, preferring to ask for a “clean bill” in order to expedite the legislation rather than proposing substantive changes to improve the pharmaceutical development process.
Of course, the FDA does not bear responsibility for all of the problems. Despite statistical and anecdotal evidence that having pre-review meetings with FDA improves the batting average of products up for review, companies do not always take advantage of having these meetings. This is a mistake, since, according to the study, 64 percent of companies who had meetings with the FDA before giving FDA materials for review found that the meetings improved the applications, and 87 percent found that having meetings expedited the review. Unfortunately, the study found that the industry was too inconsistent in pursuing these meetings, and only 53 percent felt that the FDA consistently encouraged these kinds of helpful meetings.
These findings point to a serious need to make the FDA process clearer and more transparent. The PDUFA reauthorization should also redirect user fee dollars so they fulfill their original purpose of expediting reviews, fund the Critical Path initiative to expedite the highest priority products, and reduce the influence of politics on a process that should be based on sound science. While these sound like relatively simple goals, they would go along way to improving the industry’s relations with the FDA, and towards expediting the approval process.
The politics of this reauthorization process should be interesting to watch. For years, Republicans have been the defenders of the life science industry, often at great political cost, while Democrats have consistently bashed the industry. In the recent health care reform debate, however, the Pharmaceutical Research and Manufacturers of America (PhRMA) backed the Obama health plan and even ran favorable ads about it, much to the chagrin of the Republicans in Congress, including incoming Speaker John Boehner. It is doubtful that the Democrats—even with the PhRMA support for the Obama bill—will back the industry’s requests during the PDUFA debate, and it is now an open question whether the spurned Republicans will want to help PhRMA either.
Politics aside, though, the reauthorization of PDUFA and improved relations between the FDA and the industry are crucial for the continued creation of new life-saving and life-extending medicines. The compelling but anonymous voices from industry as laid out in the new report will be extremely valuable for policy-makers as they enter the PDUFA reauthorization season – if they are willing to listen.