Washington Report: Hard Evidence

The push for more useful information on medication effectiveness is shaping drug development and reimbursement.
Jun 30, 2006


Jill Wechsler
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.

Conditional Coverage

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.


Off the Label
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 facilities.

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.