Baucus Bill Takes “Patient-Centered” Focus For Comparative Effectiveness

The latest comparative effectiveness legislation from Sen. Max Baucus suggests an approach that might be more welcome to the pharma industry.

Among the differences between the Finance Committee chairman's Patient-Centered Outcomes Research Act of 2009, S. 1213, and his Comparative Effectiveness Research Act of 2008 are the focus on patient/physician decision-making and the removal of the lone reference to cost-effectiveness research.

PCORA, introduced June 9, keeps the idea of a public/private institute to serve at the center of comparative effectiveness research coordination, but the revised language describing the entity's purpose sets the stage for the new focus on informed decision-making Baucus is taking.

The Montana Democrat expects PCORA to be part of the Finance Committee's health care reform legislation. However, Sens. Orrin Hatch, R-Utah, and Mike Enzi, R-Wyo., have so far kept the Baucus approach out of the committee's bill.

PCORA states the purpose of the institute "is to assist patients, clinicians, purchasers and policy makers in making informed health decisions by advancing the quality and relevance of evidence concerning the manner in which diseases, disorders, and other health conditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed through research and evidence synthesis that considers variations in patient subpopulations, and the dissemination of research findings with respect to relative clinical outcomes, clinical effectiveness, and appropriateness of the medical treatment services."

In contrast, the previous bill said the institute was "to improve health care ... by advancing the quality and thoroughness of evidence concerning the manner in which diseases, disorders, and other health conditions can effectively and appropriately be prevented, diagnosed, treated, and managed clinically through research and evidence synthesis, and the dissemination of research findings with respect to the relative outcomes, effectiveness, and appropriateness of the medical treatments, services and items."

PCORA's patient-centered approach is also evident in its list of factors to be considered for setting the research agenda (see chart: " ¹ Factors In Setting Research Priorities' following page: 'Factors In Setting Research Priorities ").

Cost-Effectiveness Reference Dropped

Another key change in PCORA is the removal of the only direct reference to cost-effectiveness. CERA called for a report within five years on methods "by which cost-effectiveness and value could be assessed in a scientifically valid way."

In contrast, that lone reference to cost-effectiveness was replaced in PCORA with a report due in three years examining methods "by which efficiency and value (including the full range of harms and benefits, such as quality of life) could be assessed in a scientifically valid and standardized way."

The move to drop the reference to cost-effectiveness puts the emphasis on the comparative clinical research. The bill maintains the use of "comparative clinical effectiveness research" throughout the text, as it did in the bill's previous iteration.

PCORA, cosponsored by Budget Committee Chairman Kent Conrad, D-N.D., also includes safeguards to ensure that a single comparative effectiveness research study does not become the sole basis for a coverage decision (² (Also see "Baucus Maintains Public/Private Comparative Effectiveness Vision" - Pink Sheet, 16 Mar, 2009.)).

Funding Doubled

PCORA follows CERA's lead in funding the institute through a combination of public and private funds. However, PCORA would create a trust fund to support the Patient-Centered Outcomes Research Institute.

For fiscal 2010, the government will allocate $10 million to the Patient-Centered Outcomes Research Trust Fund. In fiscal 2011, the allocation jumps to $50 million. For fiscal 2013-2019, the government portion of funding becomes $150 million annually.

There also are provisions to draw $10 million of the $400 million allocated to the Department of Health and Human Services for comparative effectiveness research in the American Recovery and Reinvestment Act for transfer to the trust fund.

On the private side, funds again will come through a fee assessed on insurance companies based on the number of covered lives, at $2 per covered life through 2019.

A Bigger Role For Pharma

Another key addition to S. 1213 is a potential larger role for pharmaceutical and other medical manufacturers to have input in the research process.

According to the bill, the institute "shall appoint expert advisory panels in carrying out the research project agenda. ... Such expert advisory panels shall, upon request, advise the institute and the agency, instrumentality, or entity conducting the research on the research question involved and the research design or protocol, including the appropriate comparator technologies, important patient subgroups, and other parameters of the research, as necessary."

The bill states that an expert advisory panel "may include a representative of each manufacturer of each medical technology that is included under the relevant topic, project, or category for which the panel is established." Other panel members are to include practicing and research clinicians, patients and patient representatives, and experts in scientific and health services research, health services delivery and evidence-based medicine.

HELP Proposal Also Patient-Focused

A comparative effectiveness proposal also surfaced in comprehensive reform legislation released by the Health, Education, Labor and Pensions Committee, but the proposal is less detailed than Baucus' plan, and its organizational structure is within HHS.

Sen. Barbara Mikulski, D-Md., said during a HELP Committee hearing June 12 that research results should not lead to clinical practice mandates or standards, because the goal is not to interfere with physician judgment and tying outcomes to coverage could suppress innovation.

The HELP reform bill creates the Center for Health Outcomes Research and Evaluation within HHS to "coordinate, conduct, support, and synthesize research relevant to the comparative health outcomes and effectiveness of the full spectrum of health care treatments, including pharmaceuticals, medical devices, medical and surgical procedures, screening and diagnostics, behavioral health care, and other health interventions."

CHOR would disseminate findings to providers, patients, and public and private payers, and would "develop a publicly available resource database that collects and contains high-quality independent evidence to inform health care decision-makers, which shall include reliable evidence from government and non-government sources." The center also would encourage development and use of clinical registries as well as outcomes research data networks from electronic health records.

The center would take recommendations from an advisory committee consisting of representatives from HHS, CMS and a wide range of stakeholders, but not manufacturers.

Like the Baucus proposal, any recommendations for CHOR "shall not be construed as mandates for payment, coverage or treatment."

Mikulski, who authored the provision, is a cosponsor of a recently introduced bill that would put comparative effectiveness information on drug labeling ( ³ (Also see "Comparative Effectiveness Bill Allows Industry Input Into Study Selection" - Pink Sheet, 8 Jun, 2009.) ).

- Gregory Twachtman ([email protected])