Harmonization Of Health Technology Assessment Across The European Union: Lessons For The United States

publisher’s Note

This post is part of the Health matters Blog short series, “Value assessment: where are we going after COVID?”The series explores what we learned about value assessment during the coronavirus pandemic and related issues, how we might think about value in healthcare going forward and how those ideas might translate into policy. The series is produced with the support of Innovation and values initiative (IVI) and grew from a Group of webinars jointly organized by IVI and ISPOR – The specialist society for health economics and outcome research. Contributions included are checked and edited by Health matters Blog contributor; the opinions expressed are those of the authors.

Public insurance programs and private insurance plans need to develop criteria for deciding which drugs are covered for which patients and under what conditions. To do this, they need to evaluate the incremental clinical and social contribution of new products and compare them with products already on the market. These evaluations must then be weighed against the likely additional expenses. Implicitly, if not explicitly, insurers must carry out a Health Technology Assessment (HTA).

In the US pluralism system, each public program, private insurer, and self-insured employer conducts its own assessments. Most are done without explicitly considering the quality of the evidence and without providing the documentation necessary for those involved to understand what was decided and why. Neither is coordinated with the other, which leads to duplication of work and an increased administrative burden for doctors and patients.

European nations have developed a much more formal approach to HTA, with government-administered or mandated bodies evaluating new drugs and making recommendations for insurance coverage and pricing. While national ratings tend to pay more attention to the quality of evidence than their US counterparts, they too suffer from a lack of coordination, duplication, opacity for doctors, and uneven access to patient care.

In recognition of the problems caused by the diversity of technology impact assessments, the European Union has launched a 20-year harmonization process in the hope of achieving a uniform process and result for every innovative therapy. These efforts have met with fierce opinions about methodology and jealous consideration for local privileges. The voluntary harmonization initiative called EUnetHTA has a framework (ie HTA core model) and some common reviews for individual drugs, but never achieved standardization. This year the European Union took voluntary efforts out of their misery and prescribed a new approach.

Heterogeneity in HTA principles and methods

All efforts to harmonize HTA must deal with the numerous dimensions in which assessment methods can and often do differ. A partial list would include:

  • Can the incremental clinical benefit be measured using a cardinal index (e.g. Quality Adjusted Life Years) or an ordinal index (e.g. no incremental benefit, minor improvement, major improvement)?
  • What should be chosen as a comparative product against which each innovation is evaluated? What if the standard of care differs depending on the region or population?
  • Should the analysis include a formal cost-benefit analysis or should it be limited to a comparative clinical performance?
  • Should costs be measured from the payer’s point of view or from a broader perspective that takes into account the stresses on family members, productivity, and other non-medical factors?
  • What role should key stakeholders play, including payers, manufacturers, physician organizations and patient advocacy organizations?
  • How transparent should the assessment process be and how extensive should the documentation of its conclusions be? How should potential conflicts of interest be dealt with?
  • How should the principle of data protection be balanced against the principle that data must be representative of subpopulations and across clinical contexts?

Navigating between centralization and fragmentation

The European efforts can be traced back to 2006 and the Creation of the EUnetHTA collaboration, with the original aim of harmonization and the endeavor to create a single European assessment body. EUnetHTA prepared white papers on methodological issues but the payers in every nation have been reluctant to accept joint assessments, Appreciation of national autonomy compared to a Europe-wide harmonization and continued to insist local assessments. Frustration grew with slow progress. In 2018 the European Commission proposed a to the European Parliament mandatory procedure that will come into force at the end of this year.

The approach chosen by the European Parliament aims to steer between the two dangers of excessive centralization and excessive fragmentation. The new procedure does not strive for a supranational organization and uniform evaluation criteria, but is no longer based on voluntary cooperation. Under the new legislation, a European level coordination group with technical representatives from each of the 27 Member States will select teams to evaluate new drugs and devices. The teams will likely consist of employees from the national HTA institutions (e.g. AIFA in Italy, IQWIG in Germany, HAS in France), depending on which institution has already developed expertise in the therapeutic area. The individual nations must take the joint assessments into account, but can add additional data or methods to them.

The joint assessments are written and published in English. In contrast, national HTA ratings are usually written in the local language and many are not published. This transparency will allow for direct comparisons and hopefully stimulate discussion of good practice. National HTA units will likely be required to Explain and justify deviations from the joint reports.

An example of a possible deviation from the joint ratings is the choice between cardinal measures of clinical impact (e.g. quality-adjusted years of life) and ordinal measures (e.g. minor versus large impact). Nations that have used QALYs as part of their local assessments can convert ordinal measures of impact into cardinal measures if they are not already used in the joint assessment. It is unlikely that QALYs will be used in the joint assessments as the UK, the main proponent of the metric, has left the Union while France and Germany, the largest remaining members, do not favor QALYs.

Another example of a deviation from joint assessments could occur in countries in which the standard of care and thus the relevant comparative therapy deviate from the team selected by the coordination group. These deviations in the comparative values often occur due to different national guidelines with regard to the introduction of generics and biosimilars and in Insurance coverage for expensive orphan drugs.

Each country retains its independence in deciding on insurance policy and pricing, and thus on patient access to novel therapies in accordance with their budgetary capacities. In the long run, coverage and pricing decisions are likely to converge across countries. Citizens are unlikely to accept unequal access policies between nations. The experience with the population’s insistence on access to COVID vaccines and willingness to travel across borders can serve as a lesson.

Impact on the United States

The status quo of non-transparent, non-scientific and non-accountable technology assessment in the USA is not sustainable. Doctors are burdened with complex and contradicting requirements for prior admission and tiered therapy. Consumers can’t determine whether the medication you need is covered by the health plan by considering enrollment. Patients can be forced to switch treatments when they change plans or when their plan changes its formulas. The system emphasizes All parties involved distrust.

It may seem logical for US payers to agree on a single set of assessment criteria and a single process for translating technology assessments into coverage, prior approval, and pricing decisions. But the European experience calls for caution. Payers have different budgets, cultures and forms of stakeholder engagement. No one is eager to give up their self-developed process, no matter how flawed it may be, in order to have one developed elsewhere. This is especially true if the prospective agency is viewed as a dominant government bureaucracy, a self-serving private company, or an unaccountable non-profit organization.

In the European context, historical rivalries hamper the transfer of powers from national to supranational entities. In the US context, ideological perspectives on an appropriate division of labor between the public and private sectors make it difficult to establish a variant of the former Congress Bureau for Technology Assessmentlet alone a full-blown imitation of a European agency. In the private sector, cooperation in technology assessment is hampered by rivalries between competitors, the privileges of self-insured employers and legal prohibitions against anything that could be construed as collusion.

The US path to health technology assessment could be analogous to European efforts. The first step would be to be more transparent about the inputs into the process, about what evidence is used and how it is weighted, and about the results in relation to the collection criteria chosen for each product. This would allow the process of each payer to be shared with the others and with the methods of medical societies, government agencies such as the Institute for patient-centered results research, academic initiatives like the Second panel on cost efficiency in health and medicine, and independent third parties like the Institute for Clinical and Economic Review. The direct comparability of evidence and methods could encourage the adoption of good practice. Publication of coverage decisions, including the inclusion of forms, pre-approval procedures, and tiered therapy requirements, could better support medical prescription and patient adherence.

No one will force the public Medicare program, private Medicare Advantage plans, commercial insurers, pharmacy benefit managers, government Medicaid agencies, and the Veterans Administration to take a unified approach to technology assessment, coverage, and pricing. But with transparency, a coalition of the willing, and pressure from stakeholders to reduce complexities and inequalities, the US could still find a method in the madness of health technology assessment.