Trust in the Food and Drug Administration: A National Survey Study.

Building trust in public health agencies like the US Food and Drug Administration (FDA) has become a key government priority. Understanding the roots of FDA mistrust is important if the agency is to develop targeted messaging and reforms aimed at building confidence in the agency. We conducted a survey of 2,021 respondents in the US probing attitudes toward the FDA. The primary outcome was FDA trust, defined as the mean score that each respondent assigned to the FDA across four prespecified axes: (1) competence and effectiveness; (2) commitment to acting in the best interests of the American public; (3) abiding by the rules and regulations set forth by policy or law; and (4) expertise in health, science, and medicine. On multivariable ordinal logistic regression, FDA mistrust was associated with female gender (odds ratio [OR] = 0.74, 95% confidence interval [CI] 0.62-0.88), rural community (OR 0.85, 95% CI 0.75-0.96), conservative political views (OR 0.77, 95% CI 0.74-0.81), worse self-reported health (OR 0.89, 95% CI 0.80-0.98), lower satisfaction with health care received (OR 0.63, 95% CI 0.56-0.71), less attention to health and science news (OR 0.72, 95% CI 0.64-0.80), and not having children under the age of 18 (OR 0.72, 95% CI 0.60-0.86). These findings underscore the challenges faced by US political leaders in convincing a heterogeneous American public to trust the FDA. The FDA should develop and deploy targeted outreach strategies to populations with lower levels of trust and strengthen internal processes that minimize biases and ensure sound decision-making.

Leah Z. Rand, Daniel P. Carpenter, Aaron S. Kesselheim, Anushka Bhaskar, Jonathan J. Darrow, and William B. Feldman Reply.

The authors respond to a letter by Mitchell Berger in the March-April 2024 issue of the Hastings Center Report concerning their essay "Securing the Trustworthiness of the FDA to Build Public Trust in Vaccines."

Securing the Trustworthiness of the FDA to Build Public Trust in Vaccines.

The Covid-19 pandemic highlighted the need to examine public trust in the U.S. Food and Drug Administration (FDA) vaccine approval process and the role of political influence in the FDA's decisions. Ensuring that the FDA is itself trustworthy is important for justifying public trust in its actions, like vaccine approvals, thereby promoting public health. We propose five conditions of trustworthiness that the FDA should meet when it reviews vaccines, even during emergencies: consistency with rules, proper expert or political decision-makers, proper decision-making and noninterference, connection to public preference, and transparency of both reasons and procedures. The five conditions provide a road map of procedural and substantive requirements, which the FDA has variably implemented, focused on ensuring appropriate influence of political interests. While being a trustworthy agency cannot guarantee the public's trust, implementing these conditions builds a groundwork for public trust.

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.

Congress' Misguided Plan to Ban QALYs.

This Viewpoint discusses the flawed assumptions and potential negative impacts of a proposed federal bill that would ban government health care programs from using the quality-adjusted life-year (QALY) and "similar measures" when determining insurance coverage or negotiating prices.

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.

Payments for research participation: Don't tax the Guinea pig.

Under current US statute, payments to research participants are taxable income. This means that even though institutional review boards and researchers agree to specific payment amounts to account for the burden of research, participants are paid less than anticipated, and participants' net payment will vary depending on their home state. Unlike other entities in the research enterprise, who receive incentivizing tax exemptions and credits, research participation is tax dis-incentivized. In addition, incentives and rewards for other socially valuable activities are not taxed. Given these differences and the restrictions on research payments, it is unfair to tax participants on their payments and the statue should be revised.

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.