With medicare now spending millions on prescription drugs, the drive for more comparative drug information is taking off.
Everyone agrees that preapproval clinical trials tell practitioners and payers little about the value of new medicines. In
addition to basic safety and efficacy data, payers want more evidence on how a drug compares to existing (and often cheaper)
therapies. Evidence-based medicine (EBM) has become the Holy Grail for ensuring appropriate patient treatment—and for reducing
inappropriate spending on less effective therapies and procedures.
The Centers for Medicare and Medicaid Services (CMS) is carving out a lead role in assessing real-world drug safety and effectiveness.
One prominent and controversial strategy is its Coverage with Evidence Development (CED) policy, which authorizes Medicare
reimbursement for a drug or medical procedure while the sponsor collects additional effectiveness information through clinical
trials, patient registries or other assessments (see "Cross-Agency Collaboration," Pharm Exec, February 2006). While CMS officials paint this approach as expanding payment for "promising but not persuasive" drugs and
treatments, pharma companies are wary that Medicare will routinely require post-approval studies in order to gain reimbursement
for new drugs.
For example, Medicare announced last year that it would greatly expand the use of PET (positron emission tomography) imaging
for diagnosis and staging of a broader range of cancers. At the same time, the agency wanted physicians to submit data to
a PET imaging registry to show how the diagnostic informed treatment decisions. This expansion of the CED program was placed
on hold for several months, though, due to questions about insufficient protections for seniors, who would be required to
provide treatment information in order to obtain coverage. CMS has revised the program to avoid any appearance of coercing
disclosure of private health information; now there is an alternative process for collecting treatment data on non-consenting
patients, while also avoiding extensive informed-consent and institutional-review-board procedures at all 800 participating
Off the Label
Another CED program involves an ICD registry to track the effectiveness of implantable cardioverter defibrillators in preventing
fatal heart attacks. In deciding in January 2005 to cover ICD use more broadly, CMS required patients receiving these devices
to enroll in the National Cardiovascular Data Registry developed by the American College of Cardiology and the Heart Rhythm
Society, which is gathering data on all patients (not just Medicare beneficiaries) receiving ICDs at 1,300 hospitals. With
more than 125,000 ICDs implanted annually in the United States, the registry expects to collect considerable information on
these products and treatment outcomes.
CMS anticipates that the ICD registry will be the "poster child" for its CED policy, said CMS special advisor Steven Pearson,
at a recent EBM conference. Registration of patients at time of service will ensure that treatment is targeted to individuals
most likely to benefit; data review will point out key differences in outcomes for different providers, devices, drugs and
patients; and all this information would be useful in developing pay-for-performance programs that reward physicians who are
able to provide higher quality care.
Push for Registries
These initiatives reflect increased interest in using patient registries to obtain more real-world outcomes information on
innovative treatments. This approach may gain wider acceptance with the development of a Web-based reference guide on patient
registries by the Effective Healthcare Program at the Agency for Healthcare Research and Quality (AHRQ). The guide aims to
help payers and research organizations establish, maintain, and evaluate registries that collect information on patient outcomes
following medical treatment. While a main customer is Medicare's CED program, it should be useful to pharma companies devising
strategies for collecting additional safety and efficacy information as part of post-approval study commitments.
AHRQ has posted a draft outline for the guide on its Web site, and a final version is expected by the end of the year from
Boston-based consultant Outcome Sciences, which is producing the guide for AHRQ. It will include a series of case studies
on effective registries that address many of the ethical patient-protection issues that surfaced with the PET scan registry.