What is at stake with the Congressional reauthorization of the
Prescription Drug User Fee Act (PDUFA)?
Congress must reauthorize PDUFA this year in order to
keep the program operational. The current user fee relationship
between the biopharma industry and the FDA under PDUFA has allowed
the FDA to hire a significant number of new employees to work on
drug application review and new drug safety. The implementation of
PDUFA initially led to a drop in review times for new drug
applications and allowed new medicines to reach patients more quickly.
However, over recent years, the original successes of PDUFA have
slipped, partly due to additional requirements placed on the FDA
during prior PDUFA reauthorizations.
The campaign believes that without the proper regulatory
environment in place, America will lose the innovation race and will
fail to realize all of the critical health and economic benefits
that new medicine research, manufacturing, and delivery can
bring.
The campaign will be advocating to Congress that the PDUFA
reauthorization is an opportunity to advance innovation from
U.S. companies and bring new medicines to patients faster.
What happens when PDUFA works?
When PDUFA works, it creates a regulatory environment that is
predictable, transparent, and institutes accountability for the
FDA. This allows the pharmaceutical industry to deliver safe and
innovative cures and treatments that will help people live longer,
healthier, and more productive lives. It also facilitates the
expansion of America's position as the global leader in drug
innovation.
Since PDUFA was implemented in 1992, we’ve seen how it can work,
and its 18-year history has paralleled our country's most productive
and innovative generation of new drug development. Congress needs to
be reminded how well PDUFA can work if it’s structured right.
For example:
- More than 2,900 medicines are in clinical trials today or
being reviewed by FDA, up from 1,800 in 1999.
- More than 50% of new drugs are launched in the U.S. now,
compared to 8% pre-PDUFA.
- America currently has more potential treatments in clinical
trials than the rest of the world combined.
- The U.S. accounts for 80% of the world's biotechnology.
- Since 1993, over 1,000 new drugs have been approved, including
90 new cancer drugs, 139 drugs for metabolic and endocrine
disorders, 125 anti-infective drugs, 138 drugs for neurological
and psychiatric disorders, and 106 new drugs to treat
cardiovascular and renal disease.
More specifically:
- PDUFA user fees have enabled the FDA to significantly increase
scientific review staffing to work on drug application review and
new drug safety.
- Median review times for priority review drugs has been cut in
half and for standard applications has been cut by 37%.
- PDUFA has decreased review times while not reducing patient
safety.
- Previous PDUFA reauthorizations have increased drug and
patient safety.
What happens when PDUFA doesn't work correctly?
With Congress reauthorizing PDUFA every five years since 1992,
changes have been made to the program that have unintentionally
reduced its effectiveness and impaired the FDA's ability to review
new drug applications quickly and efficiently. This ultimately means
unnecessary delays in getting new, safe and innovative medicines to the
patients who need them. It also means the United States risks its
position as a leader in drug discovery and innovation as other
countries might bring equivalent medicines to market before the
U.S.
As Congress looks to reauthorize PDUFA this year, it needs to
know that previous changes to PDUFA have helped slow down the
regulatory process at the FDA. Right now new medicines are taking
longer to get to patients and fewer new drugs are being approved by
the FDA than in past years.
For example:
- The last PDUFA reauthorization (PDUFA IV) mandated many new
performance commitments and process improvements that have placed
new demands on the FDA. These have hindered the FDA's ability to
meet review goals. In FY 2008, the FDA only met 1/3 of review
performance goals as outlined in PDUFA IV.
- Median review times for new drug applications have increased
for the first time in five years.
- Median review times for priority drugs almost doubled from 6
months to 11 months between FY 2007 and FY 2008, partly due to
new requirements included in the Food and Drug Administration
Amendment Act (FDAAA).
- New drug review times, which decreased by 63% in PDUFA I & II,
began to rise under PDUFA III. Under PDUFA IV (in FY2008) median
review times rose further.
- PDUFA fees – which are intended to supplement FDA
appropriations – not replace them, have almost doubled between
FY2007 and FY2011, and made up 65% of FDA's drug review budget in
FY2010.
How Congress gets the PDUFA reauthorization right
When Congress votes to reauthorize PDUFA this year, they need to remember that PDUFA has brought about tremendous advances to the FDA and has helped spur new drugs to patients faster. However, Congress must show caution when reauthorizing PDUFA. It should not be a legislative vehicle for unnecessary and costly new mandates on the FDA. The focus of PDUFA V is on enhancing the FDA's ability to safely review and approve innovative new drugs; burdening PDUFA with changes that don't focus on this mission would only slow the pace of innovation in the U.S. and cause unnecessary delays in getting medicines to the patients who need them.
To us, a clean PDUFA reauthorization is one where Congress does not alter the technical letter already endorsed by patients, health care professionals, and industry groups, and does not add unrelated provisions that will only hinder the FDA from approving and delivering safe, innovative medicines to patients in a timely manner.
The reauthorization of PDUFA gives Congress the opportunity to fix problems with the new drug review process at the FDA. The CMM has formed to educate Congress on how best to do that.
- Streamline the regulatory review process for new drugs and make it predictable.
- Expand the transparency of the science-based review process for new drugs.
- Increase the FDA's accountability.
- Reduce conflict with the FDA's mission of ensuring the safety and efficacy of medicine.
- Ensure people's confidence in what their government, and in this case, the FDA can accomplish in helping provide safe and effective medicines.
- Focus on PDUFA. During previous reauthorizations, PDUFA has acted as a vehicle for other measures that actually undermine and impair the FDA's ability to safely review and approve innovative new drugs. Burdening PDUFA with changes that don't focus on this mission would only slow the pace of innovation in the U.S. and cause unnecessary delays in getting medicines to the patients who need them.
How can you help make sure Congress gets the PDUFA reauthorization right?
Join the Campaign for Modern Medicines and help us urge Congress to reauthorize a clean PDUFA, free of extraneous additions that bring new costs and burdens to the FDA. A clean PDUFA V will enable the FDA fulfill its mission of speeding safe and effective medicines to patients, and enhance America's position as the global leader in developing innovative new treatments and cures.
The Campaign for Modern Medicines is a group of individuals, advocacy organizations, and businesses who are working together towards these goals.