The schedule for renewing the Prescription Drug User Fee Act (PDUFA) usually sets the stage for Congress to enact overarching
Food and Drug Administration legislation every five years. Last June the legislators did just that, approving the FDA Safety
& Innovation Act (FDASIA) with PDUFA V before user fee authority expired October 1, 2012. As usual, the bill contained a host
of new requirements and programs, including stronger drug shortage reporting rules, added FDA enforcement authorities, incentives
for developing orphan drugs and antibiotics, and more "transparency" in the FDA approval process.
Now it seems that Congress may enact important FDA legislation every year. The legislators approved a sweeping food safety
and oversight law in 2010, and this year there's a big push on Capitol Hill to strengthen FDA authority over certain drug
compounding operations. Congress needs to reauthorize the Animal Drug User Fee Act (ADUFA) by this September to continue collection
of fees to support FDA's Center for Veterinary Medicine; this is usually a simple matter, but could become overloaded.
CME Avoids Sunshine
In addition to drug compounding legislation, Congress may consider a bill to mandate a national drug track-and-trace program.
Some pharma companies would like action before a California requirement kicks in in 2015, and FDA gets support for adding
unique identifiers to individual products from a new Institute of Medicine report on countering fake drugs. There's also talk
of legislation to encourage development of abuse-deterrent opioid drugs or to limit access to certain opioid combination products.
Some members of Congress want to prohibit "pay-for-delay" brand-generic deals, and medical device makers continue to lobby
for repeal of the 2.3% excise tax on sales imposed by the Affordable Care Act.
FDA legislation currently is on the back burner, as Congress struggles to address a host of critical issues, starting with
the federal budget crisis and the threat of across-the-board cuts in government funding. Congressional health committees,
moreover, are focused on Obamacare implementation and funding for Medicare and Medicaid. But a groundswell for doing something
to limit illegal drug compounding could prompt action this summer.
More limited approvals
Another candidate for legislative action is a measure authorizing a streamlined development and approval pathway for new antibiotics
and other life-saving medicines. A February FDA public hearing and a seminar sponsored by the Pew Charitable Trusts discussed
a proposal from the Infectious Diseases Society of America for a Limited Population Antibacterial Drug approval mechanism.
This approach also was recommended in a September 2012 report from the President's Council of Advisors on Science and Technology
on "propelling innovation in drug discovery, development, and evaluation."
The main idea is to permit FDA approval of a drug for a very narrow indication based on smaller, faster clinical trials for
targeted patient populations. Specific labels would limit prescribing to the designated uses, and approval would require postmarket
studies and monitoring. Some researchers and advocates say the new pathway is not necessary, given FDASIA provisions that
authorized speedier FDA approval for "breakthrough" drugs and added exclusivity for new orphan drugs and antibiotics. FDA
officials say they already have considerable authority to approve critical treatments based on streamlined trials and limited
evidence. But patient advocates consider the additional incentives provided by LPAD important for developing new treatments
for Parkinson's, leukemia, tuberculosis, transplant patients, and various rare conditions, as well as infectious diseases.
It's not clear whether FDA needs additional legislation to implement such a modified approval process, but there is a sense
that congressional approval would highlight its importance to the medical community. To move forward, FDA and sponsors need
to address a number of issues, starting with what evidence FDA would require for limited population approvals and whether
special labels or "limited use" identifiers would discourage inappropriate prescribing. Pharma companies want LPAD to be voluntary
and not imposed by FDA, and there's strong opposition to a ban on off-label prescribing from both industry and physicians,
who want flexibility to prescribe a new drug for, say, a brain infection, even without studies for that condition.
The role of payers in spurring development of new antibiotics was examined more closely at the Pew meeting. Even with a less
costly testing and development process, the limited markets for these therapies means premium prices—high above those for
generic antibiotics. Payers said that they will look for data from sponsors on how well the new treatments prevent deaths,
shorten hospital stays or reduce readmissions, along with evidence on the severity of the condition and lack of alternative
treatments. Yet Pew's Nicole Mahoney noted that payers have less impact on patient management of drug use in hospitals, compared
to outpatient settings, and thus may not play a major role in influencing prescribing of limited patient antibiotics.
"Antimicrobial stewardship" programs may do more to discourage inappropriate uses of limited use antibiotics, and monitoring
and mining electronic health records and other databases could help track how the drugs are prescribed and their impact. As
Paul Huckle, head of regulatory affairs at GlaxoSmithKline, summarized at the FDA meeting, a combination of appropriate FDA
labeling, appropriate promotion and education by the sponsor, and underlying stewardship at institutions and by the community
should provide "a pretty good way of managing use of these products in the antibiotic space."
Jill Wechsler is Pharm Exec's Washington correspondent. She can be reached at