If a Part D rebate plan does not end up in Medicare cost-cutting proposals as part of broad deficit-reduction legislation
slated to come before Congress, it will be on the list of pharma-related reforms included in next year's legislation to reauthorize
the Prescription Drug User Fee Act (PDUFA V). After a year of consultation with industry and other stakeholders, FDA officials
and manufacturers reached a new PDUFA agreement this past spring; it faces further public discussion before it is formally
transmitted to Congress in January 2012. The current user-fee program expires on Sept. 30, 2012, and a new program has to
be in place before then in order for FDA to be able to continue collecting industry payments, which now support more than
60% of the cost of the approval process for new drugs and biologics.
Members of Congress already are gearing up to add a range of pet programs to the user-fee legislation. A number of drug-related
policies calculated to generate savings for government health programs will include bills to legitimize drug reimportation,
reduce exclusivity for biosimilars, and curb brand–generic patent settlements. There will be a strong push to extend steep
340B discounts on drugs used by safety-net hospitals to include inpatient treatment and orphan medicines.
The basic PDUFA proposal is not radically different from the current user-fee program and by itself should win Congressional
support fairly easily. FDA is looking to boost user-fee revenue by about $40 million per year, according to FDA Director
of the Center for Drug Evaluation and Research (CDER) Janet Woodcock's testimony to the Energy and Commerce Committee—that's
a "modest" 6% annual increase that will bring in nearly $700 million in fiscal year 2013. The most notable change gives FDA
an extra two months to review applications, here in the guise of a 60-day "administrative filing period" after submission
of an application; this timeframe will permit agency reviewers to fully vet the filing before starting the official review
More meetings will take place between reviewers and sponsors, with a new cadre of FDA liaison officials to enhance communications.
FDA will assess meta-analysis methods, biomarkers, and patient-reported outcomes measures to improve clinical studies. The
plan also calls for standardizing risk evaluation and mitigation strategies (REMS); boosting FDA's Sentinel system to improve
postmarket drug-safety surveillance; and implementing an electronic submissions system for clinical data, something that has
been in the works for years.
Along with PDUFA, FDA and manufacturers are working on a new user-fee program for generic drugs, while also discussing a formula
for fees for new biosimilar-product applications. Support for renewing pediatric-drug development and research incentives
is strong, preferably on a permanent basis. The aim is to move away from the five-year sunset-and-reauthorization process
linked to PDUFA for the Best Pharmaceutical for Children Act and the Pediatric Research Equity Act to encourage more pediatric
The Medical Device User Fee Act (MDUFA) also is up for renewal, and negotiations on that program have been much more contentious
than those for drugs. FDA is embroiled in evaluating proposals for overhauling its 510k regulatory process for low-risk devices,
which has device makers up in arms. A recent Institute of Medicine (IOM) report advises FDA to find a better way to assess
the safety and efficacy of these products, an approach that industry feels will stymie medical device innovation and product
development. FDA has to reduce fees for 2012 because it collected too much from industry this year. This situation, along
with the larger device regulatory issues, has not smoothed the process for negotiating future fees.
As the PDUFA legislation emerges, pharmaceutical companies are looking to line up behind a number of drug-related measures,
such as proposals to boost incentives for the development of new antibiotics and to update FDA's orphan-drug program. Industry
backs efforts led by Rep. John Dingell (D-MI) to strengthen oversight of imported drugs and active ingredients, with added
FDA authority for recalls and import controls and required registration and fees on foreign manufacturing facilities to cover
the costs of overseas inspections. Companies also support Republican-backed efforts to make REMS requirements less burdensome
and to revise FDA conflict-of-interest policies that exclude many qualified experts from seats on FDA advisory committees.
The Biotechnology Industry Organization (BIO) recently unveiled its own wish-list of policy changes to spur biotech R&D. BIO
wants to make FDA an independent agency, more like the Federal Trade Commission, and to depoliticize it by appointing the
FDA commissioner to a fixed six-year term, separate from the presidential election cycle. BIO wants to require FDA to tell
sponsors specifically why it's not approving an application, and also proposes a conditional or "progressive" drug-approval
procedure that permits therapies for diseases that lack any effective treatment to come to market on a limited basis while
the sponsor completes clinical studies. The list also includes reviving the Reagan–Udall Foundation, an independent research
organization established by the FDA Amendments Act of 2007, but never funded by legislators.