Economic Evaluation and Market Access for Medical Devices in the U.S.: Implications of a U.S. Health Technology Assessment Agency

June 15, 2021 - 3:14 pm, by Avalon Health Economics LLC

Economic Evaluation and Market Access for Medical Devices in the U.S.: Implications of a U.S. Health Technology Assessment Agency

John Schneider, PhD and Hailey Crawford

June 15, 2021

The Biden Administration has indicated an intent to explore the creation of a government-operated health technology assessment (HTA) agency to engage in some form of review of biopharmaceuticals and other medical technologies.[1, 2] While there has been and continues to be considerable debate over what such a US HTA agency might look like,[1-5] were it to be implemented it may resemble at least in part the HTA agencies of Europe, such as the UK’s National Institute for Clinical Excellence (NICE) and Germany’s Institute for Quality and Efficiency in Health Care (IQWiG). It may also resemble in part the U.S.’s Institute for Clinical and Economic Review (ICER), which operates essentially like an HTA but in a non-governmental advisory capacity.

In this report, we briefly explore the implications of a US HTA for medical device manufacturers. Although it is currently difficult to determine the likelihood of the passage of HTA enabling legislation in the US in the remaining years of the Biden Administration, we suggest that increased reliance on economic evidence by commercial payers, government payers, and hospital and health system purchasers is inevitable. Thus, the time is right for health technology makers to prepare for an increased level of economic scrutiny more aggressively in the years to come.

Economic Evaluation in the US

Again, HTA is already undertaken in the US, and has been for many years, albeit in a fragmented, decentralized way. While there have been occasional calls for more aggressive assessment of health technology in the US over the past four decades,[6-8] there is mounting evidence that in recent years US payers, mostly commercial payers but also government payers, are increasingly relying on economic evidence in coverage decisions.[9-15]. For example, one survey of payers showed that 90% of payers believe that comparative cost-effectiveness analysis between treatment alternatives would be extremely or very impactful when guiding healthcare decision making.[16]

The same has been true for hospital and health system purchasers, which for many medical device makers constitute the primary target market. These organizations increasingly rely on value assessment committees (VACs) and supply chain management departments to serve as de facto HTA agencies within the organization, providing recommendations on technology adoption decisions.[17] In a survey of US VAC and supply chain members, 63% of respondents strongly agreed that purchasing decisions are driven by whether the product represents “good value for the cost.”[18]

HTA Models

Briefly, in theory the basic goals of most HTAs are four-fold: (1) measure, assess and report the clinical effectiveness of novel drugs and devices; (2) ensure that patients receive access to clinically effective treatments; (3) ensure that the costs of such treatments are (more or less) aligned with clinical value; and (4) maintain incentives for manufacturers to continue to innovate and bring new high-value treatments to market. Of course, HTA agencies generally cannot perfectly balance these four objectives; thus, the effectiveness of the HTA mission has generally been viewed from the perspective of optimization and tradeoffs.[3-5, 14, 15, 19-30]

The most readily available HTA models are the UK’s NICE and the US’s ICER. There are some other potentially useful predicates, such as Germany’s IQWiG and the US’s Agency for Healthcare Research and Quality (AHRQ). However, NICE is generally considered more rigorous and more transparent than IQWiG,[31-33] and AHRQ does not employ a regular HTA agenda, instead conducting comparative effectiveness reviews only in an ad hoc manner.

The UK’s National Health Service (NHS) relies on NICE to conduct appraisals of health technologies available to its beneficiaries. Technology appraisals consist of one of three types: (1) single technology appraisal (STA), which covers a single technology for a single indication and is the most common; (2) fast-track appraisal (FTA), which also covers a single technology for a single indication but with a shorter process time to speed up access to the most cost-effective new treatments; and (3) multiple technology appraisal (MTA), which typically cover more than one technology, or one technology for more than one indication.[34] NICE uses a cost-effectiveness “willingness to pay” (WTP) threshold of roughly $35,000 per quality-adjusted life year (QALY), meaning that if incremental cost-effectiveness ratio (ICER) associated with the new technology is at or below this WTP threshold, the technology will generally be considered cost effective and will be recommended for coverage.[35] A higher threshold is applied to health care interventions considered “highly specialized,” such as treatments for rare diseases.

In the U.S., the Institute for Clinical and Economic Review (which, as we indicated above, is unfortunately also referred to as “ICER”) is an independent U.S.-focused non-profit organization that conducts value assessments of health technologies, including pharmaceuticals and medical devices. The US’s ICER is somewhat like NICE in its approach to technology appraisal. Value is determined based on clinical effectiveness as well as long-term economic value and short-term affordability within the U.S. healthcare system.[12] Clinical effectiveness is established through a systematic review of clinical evidence presented in peer-reviewed publications, and cost-effectiveness and budget impact models are then constructed to estimate the economic value, affordability, and comparable value-based price benchmarks of the technology. There are three important differences between NICE and ICER. First, ICER functions only in an advisory capacity; no US payer is obligated to use its appraisals. Second, ICER does not undertake any studies of allocative efficiency; that is, it does not explicitly consider the opportunity costs of paying for one technology versus another (though implicitly cost-effectiveness analysis does this). Third, ICER has not adopted an explicit WTP threshold below which technologies are considered cost effective. In the U.S., based on technologies currently covered by payers, the implicit WTP per QALY threshold ranges from about $50,000 to $150,000 and more.[12, 36-38]

Options for US HTA

One recent study found that more than half (53%) of payers agreed (and another 40% strongly agreed) that there was a need for an independent HTA body in the US.[39] However, given its long history of resisting legislative attempts at forming an effective HTA agency, and its recent history of coming close to overturning the Affordable Care Act (ACA), it is unlikely that a US HTA would take the form of a rigorous and systematic HTA, akin to the UK’s NICE. Instead, a US HTA is likely to look somewhat more like ICER, which most US payers are familiar with and already to some extent rely upon when making coverage decisions. For example, according to a survey of 100 payer representatives, 46% indicated that they intended to use ICER reports as part of their evaluation process and 59% indicated that they had used ICER reports in the past.[40] In another survey of payers, more than three-quarters (77%) of respondents expected ICER to become more influential in the near future.[39]

Implications for Device Makers

The main implication of the “threat” of an emerging HTA body in the US for medical device makers (and biopharmaceutical makers as well) is that, regardless of the form that it takes, its arrival will signal an overall increase in reliance on economic evidence for payer coverage decisions. Moreover, there will likely be spillovers to hospital and health system purchasers, who will certainly consider HTA appraisals alongside the evidence they currently use in making purchasing decisions. Depending on the scope and rigorousness of a US HTA, it is possible that some proportion of the existing ad hoc assessments employed by payers and purchasers will be “crowded out,” resulting in an overall greater reliance on HTA agency appraisals.

But the overarching implication is that the need to provide strong clinical and economic evidence will only grow. If the Biden Administration does not succeed in creating a US HTA, all indicators point to an intensifying of private-sector efforts in conducting HTA-like assessments to fill the void. On the other hand, if the Administration does succeed in creating an HTA entity, this will result in even greater economic scrutiny of novel health interventions in the US.

Thus, it is more important than ever for medical device makers to begin planning for economic evidence generation early in the commercialization process. This includes, for example, the following: (1) assuring that clinical study protocols include the collection of data elements that can support economic studies; (2) building in-house conceptual models or “early” models of economic value, based on simple cost-benefit or cost-offset calculations; (3) in some cases, considering the value of more rigorous economic models, like cost effectiveness analyses; and (4) building cost-impact and budget-impact models that show the net economic effects of the intervention to purchasers (in the case of hospitals and health systems) and payer (in the case of coverage decisions). Armed with these types of economic evidence, medical device makers can be better prepared to bring their products to market in a more resource-conscious environment.

References

Cohen, J.T., What Could Biden’s Possible Bid For A National Health Technology Assessment Entity Look Like? Forbes, 2021. Mar 2 2021.
Frakt, A., Drug Prices by Committee: One Way Biden Could Lower Costs. The New York Times, 2021. Feb. 11, 2021.
Garrison, L.P., Jr., et al., A Health Economics Approach to US Value Assessment Frameworks-Summary and Recommendations of the ISPOR Special Task Force Report [7]. Value Health, 2018. 21(2): p. 161-165.
Mulligan, K., et al., Health Technology Assessment for the U.S. Health Care System. 2020, Aspen Institute and USC Schaeffer Center: Los Angeles, CA.
Pearson, S.D., et al., Cornerstones of “Fair” Drug Coverage: Appropriate Cost-Sharing and Utilization Management Policies for Pharmaceuticals 2020, Institute for Clinical and Economic Review (ICER) and UK Office of Health Economics (OHE).
Aiken, L.H. and H.E. Freeman, An essay and selective review on technology and contemporary medical care. Health Med Care Serv Rev, 1979. 2(4): p. 1-11.
Banta, H.D., Health care technology as a policy issue. Health Policy, 1994. 30(1-3): p. 1-21.
Neumann, P.J., Challenges ahead for federal technology assessment. Health Aff (Millwood), 2007. 26(2): p. w150-2.
Chambers, J.D., et al., Medicare is scrutinizing evidence more tightly for national coverage determinations. Health Aff (Millwood), 2015. 34(2): p. 253-60.
Chambers, J.D., M.D. Chenoweth, and P.J. Neumann, Mapping US commercial payers’ coverage policies for medical interventions. Am J Manag Care, 2016. 22(9): p. e323-8.
Chambers, J.D., et al., Factors predicting Medicare national coverage: an empirical analysis. Med Care, 2012. 50(3): p. 249-56.
Chambers, J.D., P.J. Neumann, and M.J. Buxton, Does Medicare have an implicit cost-effectiveness threshold? Med Decis Making, 2010. 30(4): p. E14-27.
Neumann, P.J., Yes, Improve the US Value Frameworks, But Recognize They Are Already in Prime Time. Value Health, 2019. 22(9): p. 975-976.
Sanders, G.D., et al., Recommendations for Conduct, Methodological Practices, and Reporting of Cost-effectiveness Analyses: Second Panel on Cost-Effectiveness in Health and Medicine. Jama, 2016. 316(10): p. 1093-103.
Willke, R.J., et al., Review of Recent US Value Frameworks-A Health Economics Approach: An ISPOR Special Task Force Report [6]. Value Health, 2018. 21(2): p. 155-160.
Xcenda/AmerisourceBergen. and T.N.P. Council., Information Wanted: Finding the Balance in Pharmaceutical Evidence Exchange With Payers and Providers. 2017.
Hartford, J., Strategies for Medical Device Manufacturers to Address Hospital Value Analysis. 2013, IVD.
HFMA, Strategies for Supply Chain Success. 2015, Health Care Financial Management Association.
Briggs, A., A View from the Bridge: Health Economic Evaluation – A Value-Based Framework? Health Econ, 2016. 25(12): p. 1499-1502.
Drummond, M., Evaluation of health technology: economic issues for health policy and policy issues for economic appraisal. Soc Sci Med, 1994. 38(12): p. 1593-600.
Frønsdal, K.B., et al., Health technology assessment to optimize health technology utilization: using implementation initiatives and monitoring processes. Int J Technol Assess Health Care, 2010. 26(3): p. 309-16.
Godman, B., et al., Barriers for Access to New Medicines: Searching for the Balance Between Rising Costs and Limited Budgets. Front Public Health, 2018. 6: p. 328.
Levy, A.R., et al., International comparison of comparative effectiveness research in five jurisdictions: insights for the US. Pharmacoeconomics, 2010. 28(10): p. 813-30.
Neumann, P.J. and J.T. Cohen, Measuring the Value of Prescription Drugs. N Engl J Med, 2015. 373(27): p. 2595-7.
Neumann, P.J. and J.T. Cohen, ICER’s Revised Value Assessment Framework for 2017-2019: A Critique. Pharmacoeconomics, 2017. 35(10): p. 977-980.
Neumann, P.J., R.J. Willke, and L.P. Garrison, Jr., A Health Economics Approach to US Value Assessment Frameworks-Introduction: An ISPOR Special Task Force Report [1]. Value Health, 2018. 21(2): p. 119-123.
Robinson, J.C., Value and payment for oncology in the United States. Ann Pharm Fr, 2013. 71(5): p. 285-90.
Robinson, J.C. and S. Howell, Specialty pharmaceuticals: policy initiatives to improve assessment, pricing, prescription, and use. Health Aff (Millwood), 2014. 33(10): p. 1745-50.
Wilkinson, T., et al., The International Decision Support Initiative Reference Case for Economic Evaluation: An Aid to Thought. Value Health, 2016. 19(8): p. 921-928.
Zettler, P.J. and E.C. Fuse Brown, The challenge of paying for cost-effective cures. Am J Manag Care, 2017. 23(1): p. 62-64.
Dintsios, C.M., et al., Different interpretation of additional evidence for HTA by the commissioned HTA body and the commissioning decision maker in Germany: whenever IQWiG and Federal Joint Committee disagree. Health Econ Rev, 2019. 9(1): p. 35.
Herpers, M. and C.M. Dintsios, Methodological problems in the method used by IQWiG within early benefit assessment of new pharmaceuticals in Germany. Eur J Health Econ, 2019. 20(1): p. 45-57.
Peinemann, F. and A. Labeit, Varying results of early benefit assessment of newly approved pharmaceutical drugs in Germany from 2011 to 2017: A study based on federal joint committee data. J Evid Based Med, 2019. 12(1): p. 9-15.
NICE., NICE Technology Appraisal Guidance. 2021.
Mccabe, C., K. Claxton, and A.J. Culyer, The NICE Cost-Effectiveness Threshold. PharmacoEconomics, 2008. 26(9): p. 733-744.
McDougall, J.A., et al., Understanding the global measurement of willingness to pay in health. J Mark Access Health Policy, 2020. 8(1): p. 1717030.
Schwarzer, R., et al., Systematic overview of cost-effectiveness thresholds in ten countries across four continents. J Comp Eff Res, 2015. 4(5): p. 485-504.
Ye, Z., et al., A systematic review and meta-regression of studies eliciting willingness-to-pay per quality-adjusted life year in the general population. Expert Rev Pharmacoecon Outcomes Res, 2021: p. 1-9.
Svoboda, K., et al., ICER’s Impact on Payer Decision Making. 2020.
Lising, A., et al., Payers’ Use of Independent Reports in Decision Making – Will There Be an ICER Effect? 2017, ISPOR Value & Outcomes Spotlight.