Pushing for comparative-effectiveness analysis
Efforts to determine the appropriate prescribing and pricing of prescription drugs stand to benefit from comparative-effectiveness
reviews of medical procedures and products. Better information on what medical treatments work best for certain patients could
reduce inappropriate drug use, avoid unnecessary or harmful procedures, and support a more efficient and less costly healthcare
system. These possibilities are boosting support for a larger government role in assessing the effectiveness of medical treatment,
while health plans and payers also are demanding more evidence that medical products are cost-effective as well as medically
safe and effective.
An important issue is what kind of data will support studies comparing the effectiveness of different treatments. Experts
anticipate a greater reliance on econometric analysis and observational data, in addition to randomized controlled clinical
trials. Such a shift, though, will require clear standards and agreement on analytical approaches for assessing product value.
The challenge for drug and medical product manufacturers is to focus such analysis on how drugs compare to alternative medical
and surgical procedures, and to avoid using comparative assessments to control costs or to deny coverage for innovative products.
Even if a study indicates that a drug may be less effective for many patients, doctors should have room to prescribe that
drug for a smaller patient population that may benefit.
Revising patent policy
Congressional leaders seek to enact sweeping patent-reform legislation that they believe will better protect intellectual
property and bring US policy more in line with that of other nations. Software and computer firms support the legislation
as likely to reduce their exposure to endless litigation. Biotechnology and pharmaceutical companies, however, fear a serious
erosion of patent protections and reduced value for patent awards.
A main issue is a proposed postgrant review system that allows patents to be re-evaluated after they are awarded. High-tech
companies believe this strategy could curb patent disputes, but pharmaceutical companies fear that leaving the door open to
later challenges will create too much uncertainty about patent value and expose the system to abuse. The proposed bill also
aims to prevent "forum shopping" for friendly judges to hear patent-infringement suits by requiring patent holders to file
cases locally. Another change makes it harder to challenge a patent based on "inequitable conduct" (i.e., a manufacturer's
failure to provide all valid information when filing a patent). This issue often figures into patent disputes between generic
and innovator drug companies. Difficulties in reaching a compromise on these issues has delayed legislative action, but key
members of Congress are working to finalize the bill this year.
To bring new drugs to market more efficiently and with less cost, FDA is collaborating with industry to develop methods for
testing and assessing drug safety and efficacy. The agency's Critical Path Initiative encourages public–private partnerships
to develop new biomarkers, assess drug-quality issues, and explore new approaches for ensuring product safety. An important
project aims to improve methods for assessing liver toxicity in preclinical testing. FDA recently launched a collaboration
with Duke University Medical Center to streamline clinical-trial operations through electronic data standards, updated review-board
policies, and accreditation programs for clinical investigators and research sites. Other initiatives aim to improve the assessment
of medical-product bioequivalence and complex drug-product characterization.
FDA also continues to promote new approaches for ensuring drug quality in the US and abroad. The International Conference
on Harmonization is working to finalize guidelines to help manufacturers establish quality systems based on an integrated
quality risk-management approach. CDER staffers have been attempting to streamline the manufacturing-supplement submission
and review process to greatly reduce this burden on the agency as well as industry. Companies that choose to adopt quality-by-design
approaches and good risk-management strategies stand to gain benefits. They could enjoy reduced postmarketing oversight of
production changes and less intensive plant inspections as rewards for their efforts.
Curbing conflicts of interest
Some members of Congress and consumer advocates believe it is important to prevent scientists with industry ties from serving
on FDA advisory boards. An important FDAAA compromise emphasized the need to reduce conflicts of interest (COIs) among advisors
but allowed some waivers so that valuable experts will be able to provide advice when needed. FDA has issued new policies
to make COI waiver policies more transparent. The agency is also taking steps to meet Congressional requirements for reducing