111^th Congress

Comparative Effectiveness Research Legislation

H.R. 684, S. 330, H.R. 1321, H.R. 2502, H.R. 2520, S. 1099, S. 1142, S. 1213, H.R. 2824, S. 1259, H.R. 3001, H.R. 3002, H.R. 3090, H.R. 3200, H.R. 3361, H.R. 3400

Background

Comparative effectiveness research (CER), simply put, is “what works best.” It is research that compares drugs and other therapies that are used to treat the same illness or condition for a particular set of patients. CER results can be used to improve health care because the information that CER produces can inform the decisionmaking process for patients and health care providers regarding the best treatment options. Receiving the best treatment option can also potentially reduce the cost of health care. A sizable amount of CER is currently underway, supported and conducted by numerous respected organizations, both outside and within the Federal Government. The National Institutes of Health (NIH) has conducted CER for years and invested hundreds of millions of dollars in this area. Other agencies that conduct CER include the Agency for Healthcare Research and Quality (AHRQ), U.S. Department of Veterans Affairs (VA), Centers for Disease Control and Prevention, and Centers for Medicare and Medicaid Services (CMS).

Examples of landmark studies that have been stimulated, overseen, supported, and disseminated by NIH and that have left substantial positive impacts on the Nation’s quality of health include:

• Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack Trial: Cheap diuretics are just as effective as more expensive agents for first-line treatment of hypertension.
• Sudden Cardiac Death Heart Failure Trial: In patients with heart failure, defibrillators save lives, while the medication amiodarone does not.
• Diabetes Prevention Program: Lifestyle changes are superior to medication in preventing or slowing the onset of diabetes.
• Clinical Antipsychotic Trials of Intervention Effectiveness: The older antipsychotic medication perphenazine is less expensive and no less effective than newer medications.

In order to accelerate investment in this area of research, the American Recovery and Reinvestment Act (ARRA) of 2009 (P.L. 111-5) included a $1.1 billion appropriation for CER. ARRA allocated $400 million to the Secretary of Health and Human Services (HHS), $400 million to NIH, and $300 million to AHRQ. ARRA also established the Federal Coordinating Council for Comparative Effectiveness Research to foster coordination among Federal agencies that conduct such research, with the goal of reducing duplication of effort. The Council is also charged with advising the President and Congress on strategies related to the infrastructure needs of CER within the Federal Government and organizational expenditures for CER by relevant Federal departments and agencies.

In June 2009, the Council submitted a report (available for download here) required by ARRA containing information describing current Federal activities on CER and research recommendations for the $400 million allocated to the Secretary of HHS. The report includes the following definition of CER for the purposes of ARRA funding: “Comparative effectiveness research is the conduct and synthesis of research comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat and monitor health conditions in ‘real world’ settings. The purpose of this research is to improve health outcomes by developing and disseminating evidence-based information to patients, clinicians, and other decision-makers, responding to their expressed needs, about which interventions are most effective for which patients under specific circumstances.” As mandated by ARRA, the Institute of Medicine also released a report the same month that includes recommendations on national priorities for CER to be conducted or supported with funds provided to the Secretary.

While some Members of Congress and stakeholders view CER as an important approach to improving health care outcomes and slowing the growth of health care costs (“bending the curve”), other Members see this area of research as a mechanism by which the Federal Government will eventually ration or deny health care based on cost. Although provisions are included in ARRA to allay this concern, this language is not a sufficient safeguard for some Members, and legislation has been introduced to more explicitly prohibit the results of CER to deny coverage or ration care. Others are critical of the structure of the Council and have introduced legislation to terminate it or amend the statute to add representatives from outside the Government. Other legislation regarding CER is directed toward ensuring personalized medicine, including certain populations in the research, including comparative effectiveness information on drug and device labeling, and including CER as a condition for inclusion of drugs in Medicare formularies. In addition, legislation solely dedicated to CER was introduced. This legislation would establish an independent institute to contract with appropriate Federal agencies or the private sector to conduct such research and would establish a trust fund to pay for the research.

CER is also included in health care reform legislation. Both the House and Senate versions establish a center within AHRQ and an entity to oversee the center. Similarities also include provisions requiring that the research be insulated from political or stakeholder influence, that CER have stakeholder input, that the public have access to CER information, and that dissemination of findings take place.

Provisions of the Legislation/Impact on NIH

H.R. 684/S. 330, the Medicare Prescription Drug Savings and Choice Act of 2009: This bill would create a Medicare-operated drug plan to compete with private drug plans. As a part of this plan, the Secretary of HHS would have the authority to negotiate with drug companies. AHRQ would have the authority to assess the clinical benefit of a covered drug and make recommendations regarding which drugs to include in the formulary. As part of the assessment, AHRQ would be required to examine comparative effectiveness. The Secretary would be required to establish an advisory committee to review petitions from drug manufacturers and others regarding which drugs to include in the formulary. As part of the petition process, the advisory committee would be authorized to request an assessment of comparative effectiveness.

H.R. 1321, the Healthy Americans Act: This bill would amend Section 1013 of the Medicare Prescription Drug, Improvement, and Modernization Act of 2003 to expand CER. It would:

• Rename the stakeholders group required to consult with the Secretary of HHS in developing research priorities as the Comparative Effectiveness Advisory Board
• Establish a trust fund in the U.S. Department of the Treasury known as the Health Care Comparative Effectiveness Research Trust Fund to carry out research under Section 1013
• Require the Secretary of HHS to establish a permanent council to assist DHHS agencies, VA, the U.S. Department of Defense (DOD), and any other department or agency in coordinating the conduct or support of health services research. NIH is named as a member of the council.

S. 1099/H.R. 2520, the Patient’s Choice Act: Section 903 would terminate the Federal Coordinating Council for CER.

S. 1142, the Informed Health Care Decision Making Act: This legislation would require inclusion of effectiveness information in drug and device labeling and advertising.

S. 1213, the Patient-Centered Outcomes Research Act of 2009 and H.R. 2502, the Comparative Effectiveness Research Act of 2009: S. 1213 would establish a nonprofit corporation called the Patient-Centered Outcomes Research Institute to contract with appropriate Federal agencies or the private sector to conduct CER. H.R. 2502 would name this entity the Health Care Comparative Effectiveness Research Institute. A Board of Governors (including NIH representatives) would be established to oversee the Institute. The Institute would be responsible for:

• Establishing and carrying out a research project agenda. The Institute would be authorized to enter into contracts with Federal agencies with experience in conducting CER.
• Establishing a methodology committee to develop scientifically based methodological standards for comparative clinical effectiveness research
• Ensuring that there is a process for peer review of the research

S. 1213 would create the Patient-Centered Outcomes Research Trust Fund in the Treasury Department. H.R. 2502 would name this fund the Comparative Effectiveness Research Trust Fund. The trust fund would be financed through fees on Medicare and private health insurance plans. S. 1213 would transfer $10 million of CER funds designated to the Secretary of HHS in ARRA, and H.R. 2502 would transfer CER ARRA funds not already obligated or expended.

H.R. 2824, the Doctor-Patient Relationship and Research Protection Act: H.R. 2824 would amend the ARRA statute establishing the Council in the following manner:

• AHRQ would be provided full authority to direct and coordinate all Federal funding of CER.
• The following entities would no longer be represented on the Council: the Office of the National Coordinator for Health Information Technology, the Food and Drug Administration, VA, and DOD. NIH would continue to be represented, and the Secretary of HHS would be added.
• The following representatives would be added to the Council: 20 members, 9 of whom are not full-time Government employees, appointed by the Comptroller General. Of the 20 new representatives, there would be 3 members representing patients and health care consumers; 3 members representing practicing physicians, including surgeons; 3 members representing agencies that administer public programs, specifically 2 from CMS and 1 from agencies that administer other Federal health programs (such as DOD, VA, or medical care program of the Indian Health Service); 3 members representing pharmaceutical, device, and technology manufacturers or developers; 1 member representing nonprofit organizations involved in health services research; 1 member representing organizations that focus on quality measurement and improvement or decision support; 1 member representing independent health services research; 1 member representing research in differences in treatment outcomes along the lines of racial and ethnic background, gender, and geography; and 1 member representing research in treating rural populations.
• The “rule of construction” provisions would be amended to add language stating that nothing in Section 804 (regarding the Council) of Division A of ARRA shall be construed as preventing the Secretary of HHS from covering the routine costs of clinical care received by an individual entitled to or enrolled for benefits under Title XVIII, XIX, or XXI (Medicare, Medicaid, and the State Children’s Health Insurance Program) of the Social Security Act in the case where the individual is participating in a clinical trial and the costs would otherwise be covered.
• The rule of construction would further be amended to state that no reports submitted under this section or research findings submitted by the Council can be construed as mandates, guidelines, or recommendations for payment, coverage, or treatment.
• A new provision would be added to require the Council to establish procedures to ensure transparency, credibility, and access.

S. 1259/H.R. 3002, the Preserving Access to Targeted, Individualized, and Effective New Treatments and Services (PATIENTS) Act of 2009: This legislation would prohibit the Secretary of HHS from using data obtained from CER research, including CER funded by ARRA, to deny coverage under a Federal health care program. The Secretary would also be tasked with ensuring that CER conducted or supported by the Federal Government accounts for factors contributing to differences in the treatment response and treatment preferences of patients, including patient-reported outcomes, genomics and personalized medicine, the unique needs of health disparity populations, and indirect patient benefits.

H.R. 3001, the Ending LGBT (Lesbian, Gay, Bisexual, Transgender) Health Disparities Act: Provisions that contain CER language include a requirement that the Secretary of HHS, in collaboration with the Deputy Assistant Secretary for LGBT Health, award contracts or grants to support demonstration projects to improve the health and health care of sexual or gender minorities. These demonstration projects must use funds to include eligible patients from sexual or gender minority groups in CER research. The Deputy Assistant Secretary would also be responsible for coordinating with existing Federal research initiatives, including CER.

H.R. 3090, the Health Equity and Accountability Act of 2009: Section 313 would prohibit the Secretary of HHS from making available any Federal funds for comparative effectiveness health care research unless the recipient of the funds agrees to ensure that the research data will be disaggregated by race, ethnicity, and gender to detect and measure differences among subpopulations.

H.R. 3200, America’s Affordable Health Choices Act of 2009: Division B, Title IV, Subtitle A of the House health care reform bill contains the following CER provisions that would:

• Establish a Center for CER within AHRQ to conduct, support, and synthesize research (including research conducted or supported under section 1013 of the Medicare Prescription Drug, Improvement, and Modernization Act of 2003) with respect to the outcomes, effectiveness, and appropriateness of health care services and procedures in order to identify the manner in which diseases, disorders, and other health conditions can most effectively and appropriately be prevented, diagnosed, treated, and managed clinically
• Establish a CER Commission to oversee and evaluate the Center’s activities. The Secretary of HHS would be required to establish an independent CER Commission to oversee and evaluate the activities carried out by the Center.
• Prohibit the Center and Commission from mandating coverage, reimbursement, or other policies for any public or private payer
• Establish the Health Care CER Trust Fund, which would receive contributions from Medicare and private health insurance plans to fund the Center and the research it would conduct

During the House Committee on Energy and Commerce markup sessions, amendments were adopted that would prohibit CMS from using federally funded CER data to make coverage determinations for medical treatments on the basis of cost; prohibit the research conducted, supported, or developed by the Center, Commission, or the Council established by ARRA from being used by the Federal Government to deny or ration care; and require that the work performed by the Center or Commission be based on consultation with “specialty colleges and academies of medicine” regarding best practices.

Draft Senate Bill Reported Out by the Committee on Health, Education, Labor and Pensions (HELP): CER provisions in the Senate HELP Committee’s draft version, which was marked up in July, are somewhat different from and not as extensive as the House version. Section 219 of the draft would establish a Center for Health Outcomes Research and Evaluation within AHRQ. While the duties of the Center are more extensive than in the House bill, language establishing an “advisory council” (rather than a commission) is very brief. Furthermore, the draft does not establish a trust fund to pay for the research. The following amendments by Senator Michael B. Enzi (R-WY) were adopted during the markup sessions:

• Require consideration of the genetic makeup and coexisting conditions of individuals in CER
• Provide special safeguards for CER on rare diseases
• Allow expert panels to advise the government on how to conduct CER
• Add to the representation of the advisory council to include members representing the practice of medicine
• Add that reports to the Center from a grantee or contractor must assess whether the research demonstrates a benefit of the therapy to a specific subpopulation

The Senate HELP Committee rejected amendments to explicitly prohibit the use of data obtained from CER to deny or ration care.

H.R. 3361, the Medicare Patient Safeguards Act of 2009: The purpose of the bill is to provide a process for public comment and Medicare Evidence Development and Coverage Advisory Committee review of certain Medicare national coverage determinations. The findings section of the bill references funding for CER in ARRA. The section also notes concern regarding the use of CER to deny care. The bill contains a Sense of Congress that application of CER with regard to the Medicare program should not be used to deprive patients of medically necessary care solely based on cost.

H.R. 3400, the Empowering Patients First Act: Section 801 would:

• Prohibit the Secretary of HHS from using data obtained from CER, including research conducted or supported using funds appropriated under ARRA, to deny coverage of an item or service under a Federal health care program
• Require the Secretary to ensure that CER conducted or supported by the Federal Government accounts for factors contributing to differences in the treatment response and treatment preferences of patients, including patient-reported outcomes, genomics and personalized medicine, the unique needs of health disparity populations, and indirect patient benefits
• Require consultation with and approval by relevant physician specialty organizations before findings by the Council established by ARRA can be released in final form

Status and Outlook

H.R. 684 was introduced by Representative Marion Berry (D-AR) on January 26, 2009, and was jointly referred to the House Committees on Energy and Commerce and on Ways and Means. No further action has occurred on this legislation.

S. 330 was introduced by Senator Richard J. Durban (D-IL) on January 27, 2009, and was referred to the Senate Committee on Finance. No further action has occurred on this legislation.

H.R. 1321 was introduced by Representative Anna Eschoo (D-CA) on March 5, 2009, and was jointly referred to the House Committees on Energy and Commerce, on Ways and Means, on Education and Labor, and on Oversight and Government Reform. No further action has occurred on this legislation.

H.R. 2502 was introduced by Representative Kurt Schrader (D-OR) on May 19, 2009, and was jointly referred to the House Committees on Ways and Means and on Energy and Commerce. No further action has occurred on this legislation.

H.R. 2520 was introduced by Representative Paul D. Ryan (R-WI) on May 20, 2009, and was jointly referred to the House Committees on Ways and Means and on Energy and Commerce. No further action has occurred on this legislation.

S. 1099 was introduced by Senator Tom Coburn (R-OK) on May 20, 2009, and was referred to the Senate Committee on Finance. No further action has occurred on this legislation.

S. 1142 was introduced by Senator Jack Reed (D-RI) on May 21, 2009, and was referred to the Senate HELP Committee. No further action has occurred on this legislation.

S. 1213 was introduced by Senator Max Baucus (D-MT) on June 9, 2009, and was referred to the Senate Committee on Finance. No further action has occurred on this legislation.

H.R. 2824 was introduced by Representative Donna M. Christensen (D-VI) on June 11, 2009, and was jointly referred to the House Committees on Energy and Commerce, on Ways and Means, on Armed Services, and on Veterans’ Affairs. No further action has occurred on this legislation.

S. 1259 was introduced by Senator Jon L. Kyl (R-AZ) on June 15, 2009, and was referred to the Senate HELP Committee. No further action has occurred on this legislation.

H.R. 3001 was introduced by Representative Tammy Baldwin (D-WI) on June 23, 2009, and was jointly referred to the House Committees on Energy and Commerce, on Armed Services, on the Judiciary, on Ways and Means, on Oversight and Government Reform, on House Administration, on Education and Labor, on Veterans’ Affairs, on Transportation and Infrastructure, on Intelligence (Permanent Select), and on Foreign Affairs. No further action has occurred on this legislation.

H.R. 3002 was introduced by Representative John A. Boehner (R-OH) on June 23, 2009, and was jointly referred to the House Committees on Energy and Commerce and on Ways and Means. No further action has occurred on this legislation.

H.R. 3090 was introduced by Representative Christensen on June 26, 2009, and was jointly referred to the House Committees on Energy and Commerce, on Ways and Means, on Education and Labor, on the Judiciary, on Natural Resources, on Armed Services, on Veterans’ Affairs, and on Agriculture. No further action has occurred on this legislation.

H.R. 3200 was introduced by Representative John D. Dingell (D-MI) on July 14, 2009, and was jointly referred to the House Committees on Energy and Commerce, on Ways and Means, on Education and Labor, on Oversight and Government Reform, and on the Budget. On July 17, the House Committee on Education and Labor ordered the bill reported out by a vote of 26 to 22. The House Committee on Ways and Means ordered the bill reported out by a vote of 23 to 18 on the same day. On July 31, the House Committee on Energy and Commerce ordered the bill reported out by a vote of 31 to 28. The House Committee on Rules will create the final version of the bill and adopt procedures for floor debate. The Senate HELP Committee completed a markup of a draft bill on July 15 by a vote of 13 to 10. The Senate Committee on Finance is working on its own legislation, which has not yet been made public. No further action has occurred on this legislation.

H.R. 3361 was introduced by Representative Charles W. Boustany, Jr. (R-LA) on July 28, 2009, and was jointly referred to the House Committees on Energy and Commerce and on Ways and Means. No further action has occurred on this legislation.

H.R. 3400 was introduced by Representative Tom Price (R-GA) on July 30, 2009, and was jointly referred to the House Committees on Energy and Commerce, on Ways and Means, on Education and Labor, on Oversight and Government Reform, on the Judiciary, on Rules, on the Budget, and on Appropriations. No further action has occurred on this legislation.

August 2009