In the current era of health care reform, Congress has expanded its financing of, and commitment to, the development of comparative-effectiveness research (CER) through its passage of the Patient Protection and Affordable Care Act (PPACA) this past March. The American Recovery and Reinvestment Act (ARRA) targeted $1.1 billion towards CER in 2009, and now PPACA earmarks an additional $1.26 billion in its direction over the next ten years. PPACA creates a Patient-Centered Outcomes Research Institute (PCORI, or “the Institute”), which will “assist patients, clinicians, purchasers, and policy-makers in making informed health decisions…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.” In addition, the Institute is charged to direct “the dissemination of research findings with respect to the relative health outcomes, clinical effectiveness, and appropriateness of…medical treatments [and] services.”PPACA defines CER as “research evaluating and comparing health outcomes and the clinical effectiveness, risks, and benefits of 2 or more…health care interventions, protocols for treatment, care management, and delivery, procedures, medical devices, diagnostic tools, pharmaceuticals (including drugs and biologicals), integrative health practices, and any other strategies or items being used in the treatment, management, and diagnosis of, or prevention of illness or injury in, individuals.” The Institute will spearhead Federal efforts to guide future CER. This report discusses the establishment and charge of PCORI, as well as the subsequent financing and dissemination of appropriated funds. Following this examination, recent publications concerning CER will be considered and speculations addressed.
The Patient-Centered Outcomes Research Institute. PCORI terminates, and subsequently expands upon, the powers of the Federal Coordinating Council for Comparative-Effectiveness Research (FCCCER), which was created by ARRA as a CER advisory group to the President and to Congress. The Institute is a private, nonprofit, tax-exempt corporation. Its first charge will be to identify national research priorities for CER, “taking into account factors of disease incidence, prevalence, and burden in the United States (with emphasis on chronic conditions).” Recently, the Institute of Medicine (IOM) published its own recommendations for CER research needs, listing its top 100 priorities. Once PCORI’s research agenda is established, it is mandated to include 45-60 days of public comment to ensure transparency and public access. PCORI will then undertake these priorities using the following research methods:(i) Systematic reviews and assessments of existing and future research and evidence including original research conducted subsequent to the date of the enactment of this section.(ii) Primary research, such as randomized clinical trials, molecularly informed trials, and observational studies.(iii) Any other methodologies recommended by the methodology committee.
PCORI will be able to contract with other agencies of the Federal Government, academic institutions, and private sector research groups, but must give “preference” to the Agency for Health Care Research and Quality (AHRQ) and the National Institutes of Health (NIH) if identified research is within the scope of these agencies. AHRQ will create grant programs for training, and contracts will be provided if the contracting partner abides by proper transparency and conflict of interest requirements as set forth by PPACA, consults with PCORI’s established expert advisory panels, and complies with all methodological and ethical standards of the Institute, as administered by the Board of Directors. This Board will contain the following members:
(A) The Director of Agency for Healthcare Research and Quality (or the Director’s designee).
(B) The Director of the National Institutes of Health (or the Director’s designee).
(C) Seventeen members appointed, not later than 6 months after the date of enactment of this section, by the Comptroller General of the United States as follows:
(i) 3 members representing patients and health care consumers.
(ii) 7 members representing physicians and providers, including 4 members representing physicians (at least 1 of whom is a surgeon), 1 nurse, 1 State licensed integrative health care practitioner, and 1 representative of a hospital.
(iii) 3 members representing private payers, of whom at least 1 member shall represent health insurance issuers and at least 1 member shall represent employers who self-insure employee benefits.
(iv) 3 members representing pharmaceutical, device, and diagnostic manufacturers or developers.
(v) 1 member representing quality improvement or independent health service researchers.
(vi) 2 members representing the Federal Government or the States, including at least 1 member representing a Federal health program or agency.
The Comptroller General of the United States will then appoint a Chairperson and Vice Chairperson from this Board for three year terms.
PCORI must then create a “standing methodology committee.” No more than 15 members, including the Directors of AHRQ and the NIH, or their designated representatives, will be appointed by the Comptroller General for the committee. Appointees must be experts in fields “such as health services research, clinical research, comparative clinical effectiveness research, biostatistics, genomics, and research methodologies.” Within 18 months of the Institute’s establishment, the methodology committee must issue methods standards for CER, with which all contractors will be required to comply in order to receive federal funds. Given the broad range of research that is included in CER (systematic reviews, double-blind randomized clinical trials, etc.), there is some question as to the scope of these future standards.
Upon completion of CER studies, research must then be made available to the general public within 90 days, in a “comprehensible and useful” manner. Here, PCORI must discuss findings as they relate to specific subpopulations, risk factors, and co-morbidities, acknowledge research limitations, and may not include “practice guidelines, coverage recommendations, [and] payment or policy recommendations.” Public comment, availability, and access are ensured per mandatory guidelines. The Institute is then required to report its findings and all other administrative activities annually to Congress and the President.
Dissemination and Use of Data. AHRQ and the NIH will be charged to broadly disseminate PCORI research findings to “physicians, health care providers, patients, payers, and policy makers,” including “a description of considerations for specific subpopulations, the research methodology, and the limitations of the research, and the names of the entities, agencies, instrumentalities, and individuals who conducted any research which was published by the Institute.” These publications may not be construed as mandates or recommendations for payment, coverage, or treatment decisions.
In fact, PPACA, as amended by the Health Care and Education Reconciliation Act of 2010, specifically defines how CER may not be used by the Secretary of Health and Human Services (HHS) in regards to changes in coverage of items and services, reimbursement rates, or incentive programs. The Secretary “may only use evidence and findings from research…to make a determination regarding coverage…if such use is through an iterative and transparent process which includes public comment and considers the effect on subpopulations.” The Secretary also may not “deny coverage of items or services under such title solely on the basis of comparative clinical effectiveness research.”
The Secretary may not use CER “in a manner that treats extending the life of an elderly, disabled, or terminally ill individual as of lower value than extending the life of an individual who is younger, nondisabled, or not terminally ill.” However, if CER finds relevant evidence or alternative therapies regarding extending life due to age, disability, or terminal illness, the Secretary may make coverage and reimbursement decisions based on this evidence. The Secretary also “shall not use evidence or findings…in a manner that precludes, or [intends] to discourage, an individual from choosing a health care treatment based on how the individual values the tradeoff between extending the length of their life and the risk of disability.”
In addition, the Institute “may not develop or employ a dollars per-quality adjusted life year (or similar measure that discounts the value of a life because of an individual’s disability) as a threshold to establish what type of health care is cost effective or recommended. The Secretary shall not utilize such an adjusted life year (or such a similar measure) as a threshold to determine coverage, reimbursement, or incentive programs.”
Financing of the Patient-Centered Outcomes Research Trust Fund. The Institute will be funded by a trust fund (PCORTF) that is a private corporation and tax-exempt. Over the fiscal years 2010 through 2019, PCORTF will receive $1.26 billion from the Federal Government. Ten million will be appropriated in 2010, $50 million in 2011, and $150 million each year subsequently from 2012 to 2019.
A new fee will be imposed on health insurance policies and self-insured health plans beginning in 2013 and ending in 2019. The net revenues of this levied fee will be directed to the Trust Fund. The fee is “imposed on each specified health insurance policy for each policy year ending after September 30, 2012, a fee equal to the product of $2 ($1 in the case of policy years ending during fiscal year 2013) multiplied by the average number of lives covered under the policy.” After 2013, this rate will be updated annually to correct to medical inflation. In the same manner, Medicare Trust Funds will transfer $2 multiplied by the average number of Part A and Part B beneficiaries in the fiscal years 2014-2019 ($1 for the fiscal year 2013), and this rate will also be updated annually for inflation. This per-capita charge is expected to raise approximately $2.6 billion through 2019, according to the Congressional Budget Office, placing PCORI’s annual funding at approximately $500 million.
These fees will be terminated on September 30, 2019, and all unused and remaining funds in the Trust Fund will be relinquished to the general Treasury on this date if the agency is not reauthorized by Congress.
Implications of PCORI and the Future of Comparative-Effectiveness. The establishment of the Institute and funding for CER is not without controversy. Comparative-effectiveness has been implicated as a first step to government rationing, i.e. denying coverage for more expensive treatments. There is also concern that CER ignores certain subpopulations (such as minorities) and that Federal oversight will misuse its findings to deny “life-saving treatments to seniors and disabled people.” These concerns, whether based on fact or fiction, are highlighted and addressed by specific language within PPACA that limit the authority of the Secretary of HSS to make policy decisions that would deny the coverage of treatments that would in any way be construed as devaluing the life of an elderly, disabled, or terminally ill person. However, researchers note that “at some point…we will have to confront the problem of cost-effectiveness at the level of the patient.” How PCORI’s research findings are interpreted, and whether they will significantly affect coverage and policy decisions, remains to be seen. Certainly, however, the language of the new health bill allows for ample speculation and debate regarding potential policy implications and changes.
As stated previously, there are numerous stipulations regarding the use, interpretation, and implementation of CER. In particular, there are two clauses within the bill that seem to grant the Secretary of HSS some leeway in determining how CER studies may be used to affect coverage. Quoted above, the Secretary “may only use evidence and findings from research…to make a determination regarding coverage…if such use is through an iterative and transparent process.” In the very next sentence, it states that nothing shall be construed as “superceding or modifying the coverage of items or services…that the Secretary determines are reasonable and necessary.” These two statements suggest that the Secretary ultimately has the power to make some policy changes based on new CER, as long as the process is transparent (within certain limits). On the other hand, she may ignore these findings if she believes existing coverage is “reasonable and necessary.”
In essence, despite the dissemination of CER that has met rigorous peer-review and methodology requirements, the Secretary (a Presidential appointee) controls Federal acceptance of this research. CER will arguably, in the end, be influenced by politics, and Congress may micro-manage its use through amendments to this law. PCORI began as an attempt from lawmakers to remove politics from CER process, yet clearly CER will remain impotent in regards to Federal coverage and reimbursement decisions without significant political support. This statement is not meant to diminish its potentially invaluable impact on the medical community and improvements in patient care. But it is clear that Congress is willing to regulate the use of CER data, as seen through the negation of the November 2009 U.S. Preventive Services Task Force’s recommendations concerning mammography. In this case, PPACA itself was used as a means of requiring specific coverage for services despite the latest comparative-effectiveness research.
Many other questions still remain. AHRQ and the NIH have been given “preference” for PCORI funding, but there is no language in the bill suggesting how the Institute will choose agencies to contract with and disseminate funds. Federal agencies such as the Center for Medicare and Medicaid Services (CMS) and the Food and Drug Administration (FDA), as well as private interest groups, have been been reportedly vying for their share of influence. PCORI is set up to be a private entity, and despite the influence of the Directors of AHRQ and the NIH on the Board – and their belief that AHRQ and NIH will be first in line for all funding within the agencies’ scope – ultimately the Institute will be able to make its own decisions regarding CER financing. There is no telling how quickly the methodology committee will be formed, exactly who its members will be, and how soon they will be able to release their methods standards. There also remains concern about the scope of their guidelines, and whether these could restrict methodological innovation. Ultimately it could be up to two years before initial contracts are developed, and much longer before academia and, more importantly, patients begin to reap the benefits of federally sponsored CER under PPACA.
The Patient-Centered Outcomes Research Institute. PCORI terminates, and subsequently expands upon, the powers of the Federal Coordinating Council for Comparative-Effectiveness Research (FCCCER), which was created by ARRA as a CER advisory group to the President and to Congress. The Institute is a private, nonprofit, tax-exempt corporation. Its first charge will be to identify national research priorities for CER, “taking into account factors of disease incidence, prevalence, and burden in the United States (with emphasis on chronic conditions).” Recently, the Institute of Medicine (IOM) published its own recommendations for CER research needs, listing its top 100 priorities. Once PCORI’s research agenda is established, it is mandated to include 45-60 days of public comment to ensure transparency and public access. PCORI will then undertake these priorities using the following research methods:(i) Systematic reviews and assessments of existing and future research and evidence including original research conducted subsequent to the date of the enactment of this section.(ii) Primary research, such as randomized clinical trials, molecularly informed trials, and observational studies.(iii) Any other methodologies recommended by the methodology committee.
PCORI will be able to contract with other agencies of the Federal Government, academic institutions, and private sector research groups, but must give “preference” to the Agency for Health Care Research and Quality (AHRQ) and the National Institutes of Health (NIH) if identified research is within the scope of these agencies. AHRQ will create grant programs for training, and contracts will be provided if the contracting partner abides by proper transparency and conflict of interest requirements as set forth by PPACA, consults with PCORI’s established expert advisory panels, and complies with all methodological and ethical standards of the Institute, as administered by the Board of Directors. This Board will contain the following members:
(A) The Director of Agency for Healthcare Research and Quality (or the Director’s designee).
(B) The Director of the National Institutes of Health (or the Director’s designee).
(C) Seventeen members appointed, not later than 6 months after the date of enactment of this section, by the Comptroller General of the United States as follows:
(i) 3 members representing patients and health care consumers.
(ii) 7 members representing physicians and providers, including 4 members representing physicians (at least 1 of whom is a surgeon), 1 nurse, 1 State licensed integrative health care practitioner, and 1 representative of a hospital.
(iii) 3 members representing private payers, of whom at least 1 member shall represent health insurance issuers and at least 1 member shall represent employers who self-insure employee benefits.
(iv) 3 members representing pharmaceutical, device, and diagnostic manufacturers or developers.
(v) 1 member representing quality improvement or independent health service researchers.
(vi) 2 members representing the Federal Government or the States, including at least 1 member representing a Federal health program or agency.
The Comptroller General of the United States will then appoint a Chairperson and Vice Chairperson from this Board for three year terms.
PCORI must then create a “standing methodology committee.” No more than 15 members, including the Directors of AHRQ and the NIH, or their designated representatives, will be appointed by the Comptroller General for the committee. Appointees must be experts in fields “such as health services research, clinical research, comparative clinical effectiveness research, biostatistics, genomics, and research methodologies.” Within 18 months of the Institute’s establishment, the methodology committee must issue methods standards for CER, with which all contractors will be required to comply in order to receive federal funds. Given the broad range of research that is included in CER (systematic reviews, double-blind randomized clinical trials, etc.), there is some question as to the scope of these future standards.
Upon completion of CER studies, research must then be made available to the general public within 90 days, in a “comprehensible and useful” manner. Here, PCORI must discuss findings as they relate to specific subpopulations, risk factors, and co-morbidities, acknowledge research limitations, and may not include “practice guidelines, coverage recommendations, [and] payment or policy recommendations.” Public comment, availability, and access are ensured per mandatory guidelines. The Institute is then required to report its findings and all other administrative activities annually to Congress and the President.
Dissemination and Use of Data. AHRQ and the NIH will be charged to broadly disseminate PCORI research findings to “physicians, health care providers, patients, payers, and policy makers,” including “a description of considerations for specific subpopulations, the research methodology, and the limitations of the research, and the names of the entities, agencies, instrumentalities, and individuals who conducted any research which was published by the Institute.” These publications may not be construed as mandates or recommendations for payment, coverage, or treatment decisions.
In fact, PPACA, as amended by the Health Care and Education Reconciliation Act of 2010, specifically defines how CER may not be used by the Secretary of Health and Human Services (HHS) in regards to changes in coverage of items and services, reimbursement rates, or incentive programs. The Secretary “may only use evidence and findings from research…to make a determination regarding coverage…if such use is through an iterative and transparent process which includes public comment and considers the effect on subpopulations.” The Secretary also may not “deny coverage of items or services under such title solely on the basis of comparative clinical effectiveness research.”
The Secretary may not use CER “in a manner that treats extending the life of an elderly, disabled, or terminally ill individual as of lower value than extending the life of an individual who is younger, nondisabled, or not terminally ill.” However, if CER finds relevant evidence or alternative therapies regarding extending life due to age, disability, or terminal illness, the Secretary may make coverage and reimbursement decisions based on this evidence. The Secretary also “shall not use evidence or findings…in a manner that precludes, or [intends] to discourage, an individual from choosing a health care treatment based on how the individual values the tradeoff between extending the length of their life and the risk of disability.”
In addition, the Institute “may not develop or employ a dollars per-quality adjusted life year (or similar measure that discounts the value of a life because of an individual’s disability) as a threshold to establish what type of health care is cost effective or recommended. The Secretary shall not utilize such an adjusted life year (or such a similar measure) as a threshold to determine coverage, reimbursement, or incentive programs.”
Financing of the Patient-Centered Outcomes Research Trust Fund. The Institute will be funded by a trust fund (PCORTF) that is a private corporation and tax-exempt. Over the fiscal years 2010 through 2019, PCORTF will receive $1.26 billion from the Federal Government. Ten million will be appropriated in 2010, $50 million in 2011, and $150 million each year subsequently from 2012 to 2019.
A new fee will be imposed on health insurance policies and self-insured health plans beginning in 2013 and ending in 2019. The net revenues of this levied fee will be directed to the Trust Fund. The fee is “imposed on each specified health insurance policy for each policy year ending after September 30, 2012, a fee equal to the product of $2 ($1 in the case of policy years ending during fiscal year 2013) multiplied by the average number of lives covered under the policy.” After 2013, this rate will be updated annually to correct to medical inflation. In the same manner, Medicare Trust Funds will transfer $2 multiplied by the average number of Part A and Part B beneficiaries in the fiscal years 2014-2019 ($1 for the fiscal year 2013), and this rate will also be updated annually for inflation. This per-capita charge is expected to raise approximately $2.6 billion through 2019, according to the Congressional Budget Office, placing PCORI’s annual funding at approximately $500 million.
These fees will be terminated on September 30, 2019, and all unused and remaining funds in the Trust Fund will be relinquished to the general Treasury on this date if the agency is not reauthorized by Congress.
Implications of PCORI and the Future of Comparative-Effectiveness. The establishment of the Institute and funding for CER is not without controversy. Comparative-effectiveness has been implicated as a first step to government rationing, i.e. denying coverage for more expensive treatments. There is also concern that CER ignores certain subpopulations (such as minorities) and that Federal oversight will misuse its findings to deny “life-saving treatments to seniors and disabled people.” These concerns, whether based on fact or fiction, are highlighted and addressed by specific language within PPACA that limit the authority of the Secretary of HSS to make policy decisions that would deny the coverage of treatments that would in any way be construed as devaluing the life of an elderly, disabled, or terminally ill person. However, researchers note that “at some point…we will have to confront the problem of cost-effectiveness at the level of the patient.” How PCORI’s research findings are interpreted, and whether they will significantly affect coverage and policy decisions, remains to be seen. Certainly, however, the language of the new health bill allows for ample speculation and debate regarding potential policy implications and changes.
As stated previously, there are numerous stipulations regarding the use, interpretation, and implementation of CER. In particular, there are two clauses within the bill that seem to grant the Secretary of HSS some leeway in determining how CER studies may be used to affect coverage. Quoted above, the Secretary “may only use evidence and findings from research…to make a determination regarding coverage…if such use is through an iterative and transparent process.” In the very next sentence, it states that nothing shall be construed as “superceding or modifying the coverage of items or services…that the Secretary determines are reasonable and necessary.” These two statements suggest that the Secretary ultimately has the power to make some policy changes based on new CER, as long as the process is transparent (within certain limits). On the other hand, she may ignore these findings if she believes existing coverage is “reasonable and necessary.”
In essence, despite the dissemination of CER that has met rigorous peer-review and methodology requirements, the Secretary (a Presidential appointee) controls Federal acceptance of this research. CER will arguably, in the end, be influenced by politics, and Congress may micro-manage its use through amendments to this law. PCORI began as an attempt from lawmakers to remove politics from CER process, yet clearly CER will remain impotent in regards to Federal coverage and reimbursement decisions without significant political support. This statement is not meant to diminish its potentially invaluable impact on the medical community and improvements in patient care. But it is clear that Congress is willing to regulate the use of CER data, as seen through the negation of the November 2009 U.S. Preventive Services Task Force’s recommendations concerning mammography. In this case, PPACA itself was used as a means of requiring specific coverage for services despite the latest comparative-effectiveness research.
Many other questions still remain. AHRQ and the NIH have been given “preference” for PCORI funding, but there is no language in the bill suggesting how the Institute will choose agencies to contract with and disseminate funds. Federal agencies such as the Center for Medicare and Medicaid Services (CMS) and the Food and Drug Administration (FDA), as well as private interest groups, have been been reportedly vying for their share of influence. PCORI is set up to be a private entity, and despite the influence of the Directors of AHRQ and the NIH on the Board – and their belief that AHRQ and NIH will be first in line for all funding within the agencies’ scope – ultimately the Institute will be able to make its own decisions regarding CER financing. There is no telling how quickly the methodology committee will be formed, exactly who its members will be, and how soon they will be able to release their methods standards. There also remains concern about the scope of their guidelines, and whether these could restrict methodological innovation. Ultimately it could be up to two years before initial contracts are developed, and much longer before academia and, more importantly, patients begin to reap the benefits of federally sponsored CER under PPACA.
(sources available upon request)
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