The drug approval process is flawed — in some cases,
fatally. Recent weeks have seen several reports of potentially serious
problems that put life, and, by extension, the industry, at risk. The
New England Journal of Medicine recently filed an amicus brief with
the Supreme Court that asserts Merck, Wyeth, and Bayer Healthcare
intentionally withheld drug-safety information from the Food and Drug
Administration (FDA). The brief raises questions about the effectiveness
of clinical trials by shedding light on the drug-approval process.
While the drug companies have not yet publicly
responded to the accusations, the FDA’s approval process is certainly
not an industry secret. The FDA does not conduct independent tests or
compile independent data. Instead, the agency relies on clinical trials
provided by the drug maker to determine drug safety. This is, arguably,
a flawed process. It is clear to see that the party that benefits most
by bringing the product to market — the drug company — is the party that
is funding, conducting, and controlling the trials.
Certainly the FDA’s medical researchers and ethics
committees review applications to determine whether trials are well
controlled, whether data reflect the product’s effectiveness, and
whether data show the product is safe. But here is the rub: The FDA
relies on available data but not necessarily adequate data.
This is why the FDA’s stamp of approval, alone, does
not indicate whether a drug, device, or biologic is suitable for
adoption. Until the FDA’s approval process requires
comparative-effectiveness studies, approval cannot be relied upon for
determining crucial issues, such as whether a product will improve
patient outcomes, work better for intended clinical conditions than
established treatments, or be more cost effective than existing
Certainly, comparative-effectiveness studies are not
usually in the best interest of device makers and drug companies. Most
companies prefer to compete solely in the marketing arena and would not
choose to fund comparative-effectiveness studies. The risk is too great.
Such studies could find that a new drug or device is inferior to
existing offerings, killing their chances in the marketplace and sending
millions in research and development up in smoke.
Legislators and healthcare industry leaders, however,
increasingly recognize the magnitude of the problem. Sens. Max Baucus
(D-MT) and Kent Conrad (D-ND) have introduced the Comparative
Effectiveness Research Act of 2008 for the development and use of
comparative clinical-evidence studies in healthcare decision making. The
bill would implement government-supported research centered on patient
and physician needs, and improved healthcare quality.
The U.S. Agency for Healthcare Research and Quality
now has a $30 million budget to conduct clinical-effectiveness research.
The Drug Effectiveness Review Project in Oregon launched an initiative
to provide analyses of comparative research on efficacy and safety. The
Institute of Medicine (IOM) in Washington, DC, is urging Congress to
ensure comparative-effectiveness information by designating an entity
overseen by a clinical-effectiveness advisory board. And presidential
candidates Sens. John McCain (R-AZ) and Barack Obama (D-IL) include
provisions on comparative-effectiveness research in their health-reform
But here is the rub: The FDA relies on available
data but not necessarily adequate data.
This expanding interest in the need for
clinical-effectiveness research is, of course, positive. But as
clinicians, we do not have to sit idle while advocates, politicians, and
lobbyists debate the issue. Healthcare providers can effect change today
by implementing a technology-assessment committee to evaluate new
technologies. An evidence-based technology-acquisition process can
create a disciplined framework for reliably analyzing new technologies
and accelerating the movement towards sound healthcare decision making.
Such a committee would address questions, such as:
- Does this technology really work better than what
- Is the technology safe?
- For which patients might this technology be
- What are the advantages and disadvantages
compared to other options?
The technology-assessment committee would determine,
through a thorough review of clinical evidence, the relative medical
benefits and risks of new, controversial, or emerging health
technologies. They might also use comparative-effectiveness research to
weigh quality and costs. In the absence of that data, hospitals should
challenge the claims of “superior” products by manufacturers and
pharmaceutical companies, insisting on reliable data prior to paying
more for the “new” technology. And they might review product
applications submitted to the FDA to independently evaluate whether
clinical trials were structured and recorded appropriately.
This could be accomplished through a first-hand
internal analysis of published clinical trials or by utilizing research
provided by independent health-technology assessment organizations. An
evidence-based health-technology acquisition process is not influenced
by pharmaceutical or medical-device manufacturers and avoids conflicts
Changes to the FDA-approval process, and establishment of funding
mechanisms for clinical-effectiveness trials, will change clinical care
for the better in the long term. Meanwhile, we have patients to serve.
Establishing a technology-assessment committee is key to sound decision
making today, and provides the foundation for improved quality, safety,
and cost containment that all providers seek.
Winifred S. Hayes, MS, PhD, RN, president and CEO, founded Hayes Inc., an international pioneer in providing unbiased, timely, clinically focused, evidence-based reports to health plans, hospitals, managed-care organizations, government agencies, and healthcare systems since 1989.