The high cost of rare disease therapies is often discussed in isolation. When a new drug is approved for an orphan indication and an eye-popping price tag is attached drugmakers, and often patients, will make the case that the price is justified because of the benefits it provides.

In cases where no alternatives are available, drugmakers and patients will hold out even limited evidence that a drug provides benefit to justify its use despite great expense.

Thanks to advances in science, incentives from regulators, the development of drugs that target specific subpopulations of cancer patients, and changing business models of biopharmaceutical companies, the number of approved orphan drugs continues to rise. Orphan drugs represented 40 percent of new drugs approved in the United States and Europe in 2016.

ISPOR—The Professional Society for Health Economics and Outcomes Research devoted the entire May 2018 issue of its journal Value in Health to a themed series of articles that address the challenges in rare disease diagnosis, drug approval, and patient access. The findings of one of those articles should be of concern to rare disease patients because of what it says about public attitudes towards healthcare spending choices when it comes to rare diseases.

The paper “Societal Preferences for Funding Orphan Drugs in the United Kingdom: An Application of Person Trade-Off and Discrete Choice Experiment Methods,” authored by Siobhan Bourke, Catrin Plumpton, and Dyfrig Hughes of the Center for Health Economics and Medicines Evaluation at Bangor University, has a dry, academic title that might suggest it will put all but the wonkiest readers to sleep. Nevertheless, it delivers what should be a wake-up call to drugmakers and rare disease patients and their advocates about the fights ahead about access to orphan drugs.

The researchers wanted to see if the public in the United Kingdom supported existing policies of the National Health System that justify the funding of rare disease therapies at the expense of deploying that funding to treat more common diseases. They also sought to determine whether the public would approve the use of a sample of recently approved orphan drugs based on preferences.

The researchers took two different approaches to determine the preferences of a representative sample of the U.K. adult population. About half of the 3,940 respondents participated in a person trade-off experiment that asked them to select the number of patients for whom they would prefer the NHS to allocate resources. They were asked to allocate resources by choosing between  different populations and scenarios. The other group of respondents participated in a discrete choice experiment that asked them to choose between hypothetical but realistic medicines for rare and common disease by their characteristics and associated benefits.

Because the researchers believe most people would not be familiar with rare diseases and the high cost of medicines used to treat these conditions, they asked participants to watch an animation overview to provide them with a basic understanding. The video can be found online here.

Based on equal costs, the majority of respondents to the person trade-off experiment (54 percent), chose to allocate funds equally between patients treated for rare diseases and those treated for common diseases, with 32 percent favoring treating rare disease over treating common diseases. That dropped to 23 percent when rare disease treatments were 10 times more expensive. When framed differently, more respondents prioritized not increasing waiting list size than to treat rare disease patients.

The discrete choice experiment indicated a greater preference for treating a common disease over a rare disease. Respondents agreed with five of 12 positive appraisal recommendations for orphan drugs, even if the list price of the drug was higher, but preferred if the NHS would not fund the others.

The authors note that today the NHS provides preferential treatment to rare disease therapies. Last year NHS implemented new parameters for evaluating orphan drugs that fall within its Highly Specialized Technologies program. These include increasing the threshold value to $135,000 (£100,000) per QALY (the quality-adjusted life year-a widely used measure of the value of a drug),  and where transformational improvement to health to $405,000 (£300,000) per QALY. They note this would place the value of health gains in rare diseases up 15 times higher than equivalent gains in common diseases.

The researchers say their findings indicate that there is no societal preference for higher valuation of orphan drugs, whose sole distinctive attribute is rarity. The authors conclude that the study shows that the UK public does not consider rarity of a disease alone as being sufficient to justify special preferential NHS funding. They note their findings are not unique and say researchers have found similar results in Norway, Sweden, Canada, and Australia.

“Policymakers should therefore be cautious when determining special funding status for orphan drugs, especially given the increase in their numbers, cost, and the profitability of their manufacturers,” they write.

While there are intensifying debates about drug pricing and value in the United States, orphan drugs will increasingly be put in the crosshairs, particularly as they represent a significant share of new drug approvals. While regulators have demonstrated a willingness to approve drugs for rare diseases in the absence of strong evidence of efficacy or dramatic impact on a disease, payers threaten to be a greater barrier.

The debate about the price of a single drug is a different discussion than the broader discussion about healthcare spending. When treating patients with a specific disease with an expensive new drug, there may be public compassion for the need. But as healthcare becomes viewed as a finite resource, the discussion shifts from ensuring access to a medication for a group of patients to the need to balance competing demands in a system and how to get the most out of a limited budget. As the number of high-priced therapies proliferate, that’s the discussion rare disease advocates will need to prepare to have.

May 18, 2018