Executive Summary 

• Breakthrough therapies for rare diseases often face long delays in coverage by single-payer systems due to strict cost-effectiveness evaluations. 
• The disparity in treatment of rare diseases between single-payer systems and the United States is pronounced; treatments for rare diseases often fail health technology assessments in single-payer systems because they are inherently expensive to develop and serve very small patient populations. 
• When governments must choose between funding a high-cost therapy for a handful of patients or allocating those resources for broader public health initiatives (e.g., vaccinations, diabetes care), rare disease treatments often lose out. 

Introduction 

As this series has documented, on most metrics of success, single-payer health care systems fall behind the U.S. system. This disparity is especially pronounced on access to drugs treating rare diseases. A rare disease is defined as a disease, disorder, illness or condition that affects fewer than 200,000 Americans. Despite the low prevalence of individual rare diseases, more than 30 million Americans (1 in 10) are affected by 10,000 known rare conditions. 

Single-payer systems typically employ health technology assessments (HTAs) and cost-benefit analyses to decide which treatments to fund. Agencies such as the United Kingdom’s (UK) National Institute for Health and Care Excellence (NICE) assess drugs based on their cost per quality-adjusted life year (or QALY – see the American Action Forum’s piece here) gained. Treatments for rare diseases, also known as orphan drugs, often fail these evaluations because they are inherently expensive to develop and serve very small patient populations. 

For example, single-payer systems initially rejected several innovative treatments for cystic fibrosis (CF) and spinal muscular atrophy (SMA) because they exceeded acceptable cost thresholds, despite their life-transforming potential. A 2020 review found that only about 50 percent of orphan drugs approved by the European Medicines Agency (EMA) were reimbursed through the UK’s National Health Service (NHS) within two years of approval. Patients suffering from rare conditions frequently experience years-long waits for access to therapies that, in the United States, are often available soon after Food and Drug Administration (FDA) approval. 

In contrast, single-payer countries often impose strict price caps and aggressive price negotiations on new drugs. These government price controls could reduce private-sector incentives to develop new treatments. A 2019 study by the University of Chicago estimated that drug price controls could reduce global research and development spending by up to $2 trillion, or a 29–60 percent reduction in research and development from 2021–2039, translating into 167–342 fewer new drug approvals during that period. Without a strong, receptive market, pharmaceutical firms may deprioritize research on rare diseases in favor of therapies for more common conditions.  

Single-payer systems aim to balance the greatest good with the greatest value. This goal inherently introduces a tension between equity and individualized care. When governments must choose between funding a high-cost therapy for a handful of patients or allocating those resources for broader public health initiatives (e.g., vaccinations, diabetes care), rare disease treatments often lose out. 

Cystic Fibrosis in the UK 

Orkambi is a medication developed for the treatment of CF in patients aged two years and older. This combination therapy addresses the underlying cause of CF, offering significant improvements in lung function and quality of life for eligible patients.​ 

In 2015, both the FDA and the EMA approved Orkambi. In the UK, however, NICE initially rejected the drug for use in the NHS in 2016, citing concerns over its cost-effectiveness. At the time, the annual cost per patient was approximately £104,000, which NICE deemed too high relative to the clinical benefits provided. 

This decision led to a prolonged period of negotiations between the manufacturer and NHS England, during which patients in England were unable to access Orkambi through the NHS. The lack of access prompted significant public outcry, including protests and campaigns by patients, families, and advocacy groups. The situation was further exacerbated by reports that thousands of packs of Orkambi had expired and were destroyed during the impasse, highlighting the human cost of the delay.​ 

After nearly four years, a breakthrough occurred in October 2019 when NHS England announced a deal with the manufacturer. The deal was hailed as a significant victory for the CF community, with approximately 5,000 patients in England expected to benefit from access to these life-changing treatments. But one can’t help but note that this was a problem that a single-payer system inflicted on its own citizens and patients. Even the then-health secretary trumpeted that value – not access – was the main reason the NHS held out on covering this treatment. 

The Orkambi case underscores the complexities inherent in a health care system where decisions about drug availability are influenced by cost-effectiveness assessments and budgetary constraints. While single-payer systems aim to provide equitable health care access, they can also lead to delays in the adoption of innovative therapies, particularly for rare diseases where treatment options are limited and often expensive.​ 

Spinal Muscular Atrophy in Canada 

Spinal muscular atrophy (SMA) is a rare genetic disorder that affects muscle control, leading to severe physical disability and, in some cases (Type 1), early death. There were no effective treatments for SMA until the development of Spinraza, a breakthrough for patients with this disease. 

The FDA approved Spinraza in December 2016, making it the first-ever treatment for SMA. Private insurers quickly began covering the drug, ensuring access to SMA patients nationwide. In contrast, Health Canada did not approve Spinraza until June 2017 – six months after U.S. approval – but even still the Canadian public health care system did not cover it due to its high cost. Eventually, provinces announced they would cover treatment for one type of SMA, but not for other types of the disease. As a result, many Canadian SMA patients were denied coverage unless they paid out of pocket or sought treatment elsewhere. As late as 2022, Canadian Agency for Drugs and Technologies in Health recommends that Spinraza should not be reimbursed by public drug plans for the treatment of patients with Type 2 and Type 3 SMA, despite Spinraza being covered in more than 40 countries around the world with no limitations on patient age or disease type. 

Canadians with SMA had to wait years for access to treatment, leaving many to suffer irreversible nerve damage that could have been prevented with early intervention, and some had aged out of reimbursement eligibility when it was finally approved. Reporting at the time showed some families considered moving to get treated, an unfortunate solution to a debilitating disease, highlighting the disparity in centrally controlled health care access. 

Rare Disease Treatments Would Become Rarer 

Breakthrough therapies often face long delays in coverage by single-payer systems due to strict cost-effectiveness evaluations. The U.S. system, despite its imperfections and slightly higher overall costs than other Organization for Economic Co-operation and Development countries, often provides faster access to life-saving treatments. Transitioning to a single-payer system could make patients face extended wait times for new therapies – if they are developed at all.