We need to talk about values: a proposed framework for the articulation of normative reasoning in health technology assessment.

It is acknowledged that health technology assessment (HTA) is an inherently value-based activity that makes use of normative reasoning alongside empirical evidence. But the language used to conceptualise and articulate HTA's normative aspects is demonstrably unnuanced, imprecise, and inconsistently employed, undermining transparency and preventing proper scrutiny of the rationales on which decisions are based. This paper - developed through a cross-disciplinary collaboration of 24 researchers with expertise in healthcare priority-setting - seeks to address this problem by offering a clear definition of key terms and distinguishing between the types of normative commitment invoked during HTA, thus providing a novel conceptual framework for the articulation of reasoning. Through application to a hypothetical case, it is illustrated how this framework can operate as a practical tool through which HTA practitioners and policymakers can enhance the transparency and coherence of their decision-making, while enabling others to hold them more easily to account. The framework is offered as a starting point for further discussion amongst those with a desire to enhance the legitimacy and fairness of HTA by facilitating practical public reasoning, in which decisions are made on behalf of the public, in public view, through a chain of reasoning that withstands ethical scrutiny.

Alternatives to the quality-adjusted life year: How well do they address common criticisms?

OBJECTIVE: To analyze whether other outcome measures used in health technology assessment (HTA) address the criticisms of quality-adjusted life years (QALYs). DATA SOURCES AND STUDY SETTING: HTA methods guidance from 11 US comparator countries (the G10 and Australia) and six value frameworks from US organizations were reviewed to identify health outcome measures currently used to evaluate the benefits of a drug. STUDY DESIGN: The study involved a documentary analysis of guidelines to identify outcome measures used by the sampled HTA organizations. Similar outcomes were grouped together into outcome types. Each type was analyzed to determine the extent to which it replicates key advantages and responds to criticisms of QALYs extracted from the literature. EXTRACTION METHODS: Outcomes were included if guidance from at least one HTA organization identified the outcome as acceptable for HTA. Outcomes measuring or evaluating the benefit, clinical effect, or impact of a drug or health technology was included; methods of calculating costs were excluded. PRINCIPAL FINDINGS: Seven types of outcome measures were identified falling into three groups: preference-based, single-dimension outcomes, and outcomes using non-health perspectives. Among the seven QALY alternative outcome measures currently used for HTA by the sampled countries, no one outcome measure addresses all the QALY criticisms while retaining the advantageous features of the QALY. CONCLUSIONS: Proposals to adopt health technology assessment (HTA) to support value-based pricing of prescription drugs in the US have faced pushback over the use of the QALY. There is no single "right" outcome measure, and the criticisms of QALYs apply to other outcome measures used to evaluate health. The measures identified have different features and strengths, which may be appropriate for specific decision making goals, but the QALY remains the best option for decision making that requires comparisons of the overall societal value of health gains.

What Should US Policymakers Learn From International Drug Pricing Transparency Strategies?

This article analyzes differences in prescription drug pricing transparency practices among 3 Organisation for Economic Co-operation and Development member nations: the United Kingdom, Germany, and Canada. Specifically, this article compares these countries' policies on list and net price disclosures and on how international reference pricing is used to evaluate merits and drawbacks of different pricing transparency approaches. Finally, the article summarizes what policymakers in the United States should learn from these comparisons.

Getting the Price Right: Lessons for Medicare Price Negotiation from Peer Countries.

The USA pays more for brand-name prescription drugs than any other country and new legislation from August 2022 gives Medicare the authority to directly negotiate certain drug prices with manufacturers starting in 2026-something the federal insurer had been prohibited from doing for its prior history. As the USA prepares for negotiations, we therefore surveyed how comparable industrialized countries use statutory requirements and procedures to negotiate brand-name drug prices. Guidance documents, regulations, government and academic publications were reviewed to identify the process of negotiating drug prices in peer countries that have been cited as potential examples for US payment reform: Australia, Canada, France, Germany, and the UK. Processes for arriving at a final price for a drug generally fall under three approaches: statutory rebates, setting a maximum price, and arbitration between national (public) insurers and manufacturers. Each approach to price negotiation could be adopted by Medicare and reduce spending even if Medicare does not adopt an exclusionary or closed formulary. Much remains to be determined about how the new price negotiation authority in the USA will be implemented, and policymakers can learn from comparator countries' statutory and regulatory strategies for price negotiation.

High-Priced Sickle Cell Gene Therapies Threaten to Exacerbate US Health Disparities and Establish New Pricing Precedents for Molecular Medicine.

Gene therapies to treat sickle cell disease are in development and are expected to have high costs. The large eligible population size - by far, the largest for a gene therapy - poses daunting budget challenges and threatens to exacerbate health disparities for Black patients, who make up the vast majority of American sickle cell patients.

Controversy Over Using Quality-Adjusted Life-Years In Cost-Effectiveness Analyses: A Systematic Literature Review.

Researchers and policy makers in the US are exploring the implementation of health technology assessment and value-based pricing to negotiate drug prices and limit spending. Objections made to the quality-adjusted life-year (QALY), the most frequently used health economic outcome for such assessments, are a barrier to the adoption of these tools. This literature review identifies and addresses the range of criticisms made against QALYs. Methods-based criticisms require attention from stakeholders to address well-known shortcomings of the QALY and ensure consistency. Ethical criticisms, however, do not apply only to the QALY and require political decisions about societal values. Understanding and overcoming criticisms of the QALY to enable its use as part of health technology assessment and value-based pricing will be crucial as US policy makers seek to address high drug costs and health care spending.

International reference pricing for prescription drugs: a landscape analysis.

During the Trump administration, members of Congress and the administration proposed the introduction of international reference pricing (IRP) to Medicare in order to reduce US drug spending by benchmarking prices to those in other countries. Many other countries currently use IRP. We examined how US policy proposals compare with the implementation of IRP in the countries that would be referenced by the United States. Nearly two-thirds of comparator countries use IRP but also use other price negotiation strategies. The congressional proposal was most like the approach used by other countries, while the Trump administration's proposals took an uncommon approach to IRP by not adopting additional pricing strategies. DISCLOSURES: This work was supported by Arnold Ventures, which provided overall funding but was not involved in conception, design, or conduct of this work. Kesselheim provides guidance to the Massachusetts Health Policy Commission on its prescription drug price review process under a contract to Brigham and Women's Hospital but does not receive personal funding for this work. Rand has nothing to disclose.

International Reference Pricing for Prescription Drugs in the United States: Administrative Limitations and Collateral Effects.

OBJECTIVES: Many countries use international (or external) reference pricing-benchmarking prices against those in other countries-to manage spending on prescription drugs. By contrast, the United States (US) allows manufacturers to set drug prices freely. In December 2019, a major bill passed the House of Representatives that would introduce international reference pricing to reduce US drug spending. In September 2020, President Trump issued an executive order to apply international reference pricing for drugs purchased under Medicare. As US policymakers consider adopting reference pricing, it is important to recognize four key administrative issues that have complicated other countries' experiences. METHODS: We analyzed the US policy proposals and literature on international experience with international reference pricing to identify implementation challenges and potential effects of US adoption of international reference pricing. RESULTS: Four key administrative issues were identified: lack of price transparency, delays in market approvals, the frequency of price revisions, and the prevalence of cross-referencing. CONCLUSIONS: Failure to account for the key issues in the emerging US approach will lead to overspending from overestimation of prices. Policymakers also need to recognize the collateral effects that the US adoption of international reference pricing may have on other countries' prices. Given the size of the pharmaceutical market in the US and other market issues, US reference pricing will likely increase drug list and net prices in other countries. Because of limitations in implementation and collateral effects, US policymakers should consider international reference pricing as a supportive tool alongside other cost containment policies, such as value-based pricing or volume agreements. International reference pricing could limit drug spending in the US but faces implementation challenges and will negatively affect other countries.

The ethics of grandfather clauses in healthcare resource allocation.

A grandfather clause is a provision whereby an old rule continues to apply to some existing situation while a new rule applies to all future cases. This paper focuses on the use of grandfather clauses in health technology appraisals (HTAs) issued by the National Institute for Health and Care Excellence (NICE) in the United Kingdom. NICE provides evidence-based guidance on healthcare technologies and public health interventions that influence resource allocation decisions in the National Health Service (NHS) and the broader public sector in England and Wales. In this context, a grandfather clause is included when NICE does not recommend treatment with a given technology. The grandfather clause provides an exemption from the general recommendation for patients who have already started treatment with the technology in question, before the publication of the NICE guidance. In this paper we first lay out the contexts in which grandfather clauses occur in NICE guidance, and then consider ethical arguments against and in support of grandfather clauses and the continuation of treatment. We argue that NICE's current practice of automatic inclusion of a grandfather clause is ethically problematic and unfair. While the inclusion of a grandfather clause may be appropriate and justified in specific cases, we argue that inclusion of such a clause should be considered as part and parcel of the decision making process on a case by case basis, rather than adopted as the default.

An International Review of Health Technology Assessment Approaches to Prescription Drugs and Their Ethical Principles.

In many countries, health technology assessment (HTA) organizations determine the economic value of new drugs and make recommendations regarding appropriate pricing and coverage in national health systems. In the US, recent policy proposals aimed at reducing drug costs would link drug prices to six countries: Australia, Canada, France, Germany, Japan, and the UK. We reviewed these countries' methods of HTA and guidance on price and coverage recommendations, analyzing methods and guidance documents for differences in (1) the methodologies HTA organizations use to conduct their evaluations and (2) considerations they use when making recommendations. We found important differences in the methods, interpretations of HTA findings, and condition-specific carve-outs that HTA organizations use to conduct evaluations and make recommendations. These variations have ethical implications because they influence the recommendations of HTA organizations, which affect access to the drug through national insurance and price negotiations with manufacturers. The differences in HTA approaches result from the distinct political, social, and cultural contexts of each organization and its value judgments. New cost-containment policies in the US should consider the ethical implications of the HTA reviews that they are considering relying on to negotiate drug prices and what values should be included in US pricing policy.

Public involvement in the governance of population-level biomedical research: unresolved questions and future directions.

Population-level biomedical research offers new opportunities to improve population health, but also raises new challenges to traditional systems of research governance and ethical oversight. Partly in response to these challenges, various models of public involvement in research are being introduced. Yet, the ways in which public involvement should meet governance challenges are not well understood. We conducted a qualitative study with 36 experts and stakeholders using the World Café method to identify key governance challenges and explore how public involvement can meet these challenges. This brief report discusses four cross-cutting themes from the study: the need to move beyond individual consent; issues in benefit and data sharing; the challenge of delineating and understanding publics; and the goal of clarifying justifications for public involvement. The report aims to provide a starting point for making sense of the relationship between public involvement and the governance of population-level biomedical research, showing connections, potential solutions and issues arising at their intersection. We suggest that, in population-level biomedical research, there is a pressing need for a shift away from conventional governance frameworks focused on the individual and towards a focus on collectives, as well as to foreground ethical issues around social justice and develop ways to address cultural diversity, value pluralism and competing stakeholder interests. There are many unresolved questions around how this shift could be realised, but these unresolved questions should form the basis for developing justificatory accounts and frameworks for suitable collective models of public involvement in population-level biomedical research governance.

Are Medicaid Closed Formularies Unethical?

State Medicaid programs have proposed closed formularies to limit spending on drugs. Closed formularies can be justified when they enable spending on other socially valuable aims. However, it is still necessary to justify guidelines informing formulary design, which can be done through a process of decision making that includes the public. This article examines criticisms that Medicaid closed formularies limit deliberation about decisions that affect drug access and unfairly disadvantage poor patients. Although unfairness to poor patients is a risk, it is not a problem unique to Medicaid, since private insurance programs have also implemented closed formularies.

National Standards for Public Involvement in Research: missing the forest for the trees.

Biomedical research funding bodies across Europe and North America increasingly encourage-and, in some cases, require-investigators to involve members of the public in funded research. Yet there remains a striking lack of clarity about what 'good' or 'successful' public involvement looks like. In an effort to provide guidance to investigators and research organisations, representatives of several key research funding bodies in the UK recently came together to develop the National Standards for Public Involvement in Research. The Standards have critical implications for the future of biomedical research in the UK and in other countries as researchers and funders abroad look to the Standards as a model for their own policy development. We assess the Standards and find that despite offering useful suggestions for dealing with practical challenges associated with public involvement, the Standards fail to address fundamental questions about when, why and with whom public involvement should be undertaken in the first place. We show that presented without this justificatory context, many of the recommendations in the Standards are, at best, fragments that require substantial elaboration by those looking to apply the Standards in their own work and, at worst, subject to potentially harmful misapplication by well-meaning investigators. As funding bodies increasingly push for public involvement in research, the key lesson of our analysis is that future recommendations about how public involvement should be conducted cannot be coherently formulated without a clear sense of the underlying goals and rationales for public involvement.

Prior Authorization as a Potential Support of Patient-Centered Care.

We discuss the role of prior authorization (PA) in supporting patient-centered care (PCC) by directing health system resources and thus the ability to better meet the needs of individual patients. We begin with an account of PCC as a standard that should be aimed for in patient care. In order to achieve widespread PCC, appropriate resource management is essential in a healthcare system. This brings us to PA, and we present an idealized view of PA in order to argue how at its best, it can contribute to the provision of PCC. PA is a means of cost saving and as such it has mixed success. The example of the US demonstrates how implementation of PA has increased health inequalities whereas best practice has the potential to reduce them. In contrast, systems of universal coverage, like those in Europe, may use the cost savings of PA to better address individuals' care and PCC. The conclusion we offer therefore is an optimistic one, pointing towards areas of supportive overlap between PCC and PA where usually the incongruities are most evident.