Things That Affect YOU: PDUFA and AERS

The href="" rel="tag">New England
Journal of Medicine
has a set of three
early-release editorials, all pertaining to prescription drug safety,
and all openly accessible:

  1. href="">Paying
    for Drug Approvals — Who's Using Whom?

    Jerry Avorn, M.D.
  2. face="Helvetica, Arial, sans-serif"> href="">Drug
    Safety Reform at the FDA — Pendulum Swing or Systematic

    Mark McClellan, M.D., Ph.D.
  3. href="">PDUFA
    Reauthorization — Drug Safety's Golden Moment of Opportunity?

    Sean Hennessy, Pharm.D., Ph.D., and Brian L. Strom, M.D., M.P.H.

As I've mentioned before, when NEJM publishes things in the
early-release/open-access format, it means that the editors believe the
issues are pertinent to a high-priority public debate.  In
this case, it is because the PDUFA ( href="">Prescription Drug User
Fee Act) is up for reauthorization.  Some medical
policy folks are concerned that changes are needed, but do not appear
to be forthcoming.  This issue has made it into the mainstream
news media, but the number of articles is small. This is a
disappointing response by the media, to an issue of critical importance.

The media are quick to write volumes when problems are found with
pharmaceutical products.  But when we have a golden
opportunity to prevent such problems, they all but ignore it.

This post will cover the main points from the first article.
 The other two will be covered in subsequent posts.

for Drug Approvals — Who's Using Whom? Jerry Avorn,

Years ago, an administrator at a community hospital explained to me how
well his institution's grand-rounds program worked. "The drug companies
find the speakers, pay their honoraria, and provide free food for the
doctors, which helps a lot with attendance," he said. "It works well
for us, especially with our budgets so tight." Yet those lunches were
actually quite costly for the hospital: attendees at such events
predictably go on to prescribe the products promoted there —
which is precisely why the drug companies so willingly pay for these

This penetration of commerce into the province of science isn't limited
to continuing medical education. Since 1992, the United States has
relied heavily on the pharmaceutical industry to pay the salaries of
Food and Drug Administration (FDA) scientists who review new drug
applications. The Prescription Drug User Fee Act (PDUFA) is now up for
its periodic 5-year renewal, and Congress seems ready to reauthorize it
with the same short-sightedness that afflicted that naive hospital

This puts the entire controversy into context.  Although in
some ways it eems fiscally appropriate to have pharmaceutical companies
pay fees for the service provided by the FDA, some would say that it is
like having foxes pay the guards at the henhouse.  

Indeed, the FDA does provide services to the pharmaceutical companies.
 Specifically, it reviews the data pertaining to safety and
effectiveness, and issues approvals.  Only after the approval
is granted, can the drug be marketed in the USA.  

Of course, some may say that this is not a service to the
pharmaceutical industry; rather, it is a service to the citizens of the
USA.  But our citizenry does not like to pay for services.
 They prefer to have the companies pay.  So now, the
pharmaceutical companies pay about 40% of the operating costs of the
division of the FDA that assesses new drug applications.  

This has had some potential benefits.  The time required for
drug approval has been decreased, to the point that it among the
shortest in the world.  However, there is some evidence that
this has compromised safety:

analyzed by Daniel Carpenter, a professor of government at Harvard
University, suggest that drugs approved just before PDUFA deadlines are
far more likely than those approved at other points in the review cycle
to cause safety problems after they are in widespread use.

Another huge issue is this:

key problem may not be fixed by Congress either. The FDA currently
lacks the authority to require companies to conduct follow-up studies
of suspected safety problems. Most such studies are therefore not
performed, even when they are requested ( href="">see
pie charts). But the requisite legislation to give the agency
this vital authority also seems unlikely to emerge from this Congress.

The FDA can recommend follow-up, post-marketing safety studies.
 But it cannot compel anyone to do them.  Out of 1259
studies requested, only 11% have been completed.  Another 15%
are on schedule; 71% have not even been started.

The author (Dr. Avorn) suggests that the PDUFA be renewed for only 6-12
months, so that there is more time to re-evaluate the legislation and
introduce reforms.

More like this

The first two posts in this series are href="">here and href="">here.   The final editorial in the NEJM's three-part series…
By Susan Wood Next week both the Senate and House are moving forward on legislation to reauthorize the Prescription Drug User Fee Act (PDUFA), along with other key FDA legislation.  The Senate will be âmarking upâ a large omnibus piece of legislation that combines PDUFA with drug safety…
By David Michaels In the wake of the debacles involving Vioxx, Ketek, and other dangerous drugs that should never have been approved in the first place, Congress is about to take up reauthorization of the user fee system that funds the Food and Drug Administrationâs drug review process. The system…
This is a continuation of the first post, href="">Things That Affect YOU: PDUFA and AERS.  PDUFA is the Prescription Drug User Fee Act.  AERS is the href="">Adverse…