Around the World in HTAs: United States – Making HTA Great Again

A primer on US health care

The US’s health care system is notable for its contrast with that of other high-income countries. Most notably, the US lacks universal health coverage. The system is made up of a complex mix of private and public insurance and is perceived by many to prioritise high-quality services and advanced technologies at the expense of equitable access to affordable care. Indeed, the US pays the highest prices for drugs, devices, procedures, and other health services but performs relatively poorly on major indicators of population health.

Despite its Cinderella status in recent years – which we’ll get to – the US was once an innovator and leading light in health technology assessment (HTA). Beginning with the Office of Technology Assessment (OTA) in 1972, and continuing with the National Center for Health Care Technology (NCHCT) in 1978, HTA was employed to advise the Federal government on new technologies and decisions about coverage of health services, tackling prioritisation challenges that would be familiar to any HTA practitioner in 2024. Both the OTA and NCHCT were de-prioritised due to political infighting in the 1980s and eventually eliminated, though their raison d’être was not. Decentralised HTA decision-making processes persisted within both government and private entities, with the Agency for Healthcare Research and Quality (AHRQ) becoming a key player from the early 1990s.

More recently, and especially since the introduction of the Affordable Care Act (ACA) in 2010, and the acceleration in healthcare costs that accompanied its passage, HTA has gained renewed vigour in the US. The need for prioritising resource allocation has become a matter for public discussion.

The role of HTA

Today, HTA in the US reflects the health care system as a whole; it is characterised by a complex and decentralised landscape of public and private operators, including government agencies, independent organisations, health care providers, and payers. Within government, the AHRQ continues to support health research that informs technology assessments. The Centers for Medicare & Medicaid Services (CMS) uses HTA methods to inform coverage decisions, and the Food and Drug Administration (FDA) may consider health economic data as part of its evidence reviews.

Arguably, independent organisations play a more important role. The Institute for Clinical and Economic Review (ICER) was founded in 2006 as a non-profit organisation conducting HTA and providing recommendations for payers about the cost-effectiveness of new technologies. ICER is now the most visible body addressing questions of both clinical evidence and affordability for US health systems. The Patient-Centered Outcomes Research Institute (PCORI) was established by the government as a non-governmental funder of research that supports HTA decision-making. There is also an array of health insurance companies and health care providers who conduct HTA, largely behind closed doors, to determine coverage and reimbursement decisions and inform clinical guidance.

There are no two ways about it: HTA plays an important role in the US. But it’s different to most other countries, because much of it takes place in the shadows. It’s difficult for us to understand the basis of many coverage decisions in the US. Increasingly, however, the likes of ICER, AHRQ, and PCORI are becoming influential, and we can observe their influence.

ICER, in particular, has been a game-changer. ICER’s recommendations are not binding, but its reports are closely watched by payers and other decision-makers and are used as an input to price negotiations and coverage decisions. Perhaps more importantly, ICER’s determinations feed into the public discourse around drug pricing and the affordability of health care more broadly.

Clinical (or comparative) effectiveness research is now accepted in the US as a basis for prioritising the allocation of resources, and both independent organisations like PCORI, and government agencies like AHRQ, make use of it. Cost-effectiveness analyses and budget impact assessments, though employed widely, remain controversial. The quality-adjusted life year (QALY) has been famously ‘banned’ in US government decision-making (including at PCORI), preventing meaningful comparisons being made across treatments or clinical areas. There is growing interest, however, in assessing the health equity impact of resource allocation decisions, given the stark and persistent disparities in treatment and access to care experienced by disadvantaged populations in the US.

Current challenges

A more effective HTA system could moderate some of the most important challenges faced by US health care, including high costs, poor access to care, and disparities in the quality of care. The persistence of some elements of high expenditure in the US may at least partly be due to the lack of centralised HTA decision-making that helps direct resources most effectively. Progress in HTA for the US faces several interconnected challenges.

A fragmented and decentralised health care system means that there are many conflicting priorities and perspectives that HTA processes must consider. The value of HTA in most jurisdictions lies in its consistency in process, and such consistency is a challenge for the US.

Methodological and politically-charged disputes impede progress. Arguments about the suitability of widely accepted metrics and methods, such as the measurement of QALYs, hinder evidence-based decision-making. This sets the US apart from many other jurisdictions around the world, where metrics such as the QALY are accepted and used routinely in decision-making. More broadly, there is a lack of societal acceptance of the need to make difficult resource allocation decisions; ‘rationing’ is seen as a dirty word by many in the US, despite its reality for disadvantaged populations who cannot access care.

Significant power and influence is aligned in contradiction to HTA’s objectives, serving to undermine its progress in the US. As a result, policy barriers have been created that undermine HTA, such as Medicare being prohibited from relying on cost-effectiveness evidence as a primary factor in its coverage decisions, and attempts to kill off the QALY in other parts of government.

The future of HTA

Yet there are reasons for hope. Despite challenges to the QALY and cost-effectiveness analysis in general, the recent Inflation Reduction Act has given the Medicare programme the ability to negotiate prescription drug prices for the first time. It remains to be seen, however, whether value-based assessments and HTA evidence and processes will form part of the negotiations, as one of us has recommended. There may also be an increasing willingness to price new drugs within or close to ICER’s target range, as suggested by recent reviews of therapies for sickle cell disease and metachromatic leukodystrophy.

There is still space for HTA innovation in the US. In 2023, ICER worked with multiple stakeholders to develop a plan for integrating health equity into its methods and process, which is a challenge being confronted by HTA agencies globally. Some of these new methods are still in development, but certain changes have already been introduced, including advanced signalling for topics with significant disparities and a novel rating system for clinical trial diversity.

Promising initiatives are also ongoing. ICER is an active participant in several international collaborations to advance HTA methods, including working groups focused on considering surrogate endpoints in economic models and developing analyses at the whole disease level rather than for individual treatments. In 2024, ICER will embark on an ambitious agenda to explore new HTA methods and processes, including key elements of simulation modelling (e.g., novel value elements, discount rate) and the deliberative process.

HTA will always have a role in the US, with health care rationing an unavoidable fact of life. Current developments provide hope for the return of a more joined-up system to support evidence-based coverage decisions and fair pricing in the US. It’ll require a lot of work, but the gains for patients are potentially huge (and measurable in QALYs).