The question of value of rare disease therapies is a question of ongoing debate. There is not agreement on how to determine value in part because what a drug is worth depends on where you stand.

The world of science may rely on objective standards and measurements, but questions of value are subjective. The value of a therapy may appear different to a patient, than it does to an insurance company, a drug company, or society.

Health economists have focused on the number of QALYs—quality adjusted life years—as a way to quantify a drug’s benefit by considering how it may increase healthy year of life for a patient. When the financial burden of a disease is measured, people tend to focus on direct healthcare costs. 

A new study in the Orphanet Journal of Rare Diseases argues for a broader view for governments and the public to consider when it comes to determining the value of rare disease therapies. It uses hereditary transthyretin-mediated amyloidosis (hATTR), a rare a progressive condition, for its model, as well as taxes and benefits in the Netherlands.

Alnylam Pharmaceuticals, which produces a treatment for hATTR, sponsored the work of Global Market Access Solutions’ Managing Director Mark Connolly and Senior Health Economics and Market Access Consultant Saswat Panda, the first and second authors on the paper, but had no editorial input into the work according to disclosures in the paper.

hATTR is a genetic condition that is chronically debilitating and causes increased mortality. It leads to the accumulation of amyloid fibrils in organs throughout the body and affects the nerves, heart, eyes, and gastrointestinal track. It causes sensory, motor, and autonomic neuropathy, as well as cardiomyopathy. The authors report that of the one-third of people with the condition who are employed, nearly 22 percent report missing work because of the disease and more than 40 percent report some impairment at work.

For their analysis the authors used a public economic framework model to consider such things as income support, disability support, pensions, and healthcare in determining the financial consequences of hATTR patients on the government. Though the study focused on hATTR, the authors argue that similar estimates could be made for other rare diseases applying their approach. The main variation lies in the age of disease onset and the rate of disease progression that causes someone to become disabled.

Because disease progression and mortality vary widely among hATTR patients, the authors created four different patient scenarios to capture the variation: early onset with a median progression, early onset with a slow progression, late onset with a median progression, and late onset and late diagnosis. The different scenarios consider the patient’s lifetime earnings, their taxes paid, and the spending by the government on the patient including healthcare, pensions, and other direct support. The authors compared these scenarios to someone from the general population, the authors compared the amount an individual pays in taxes to what they receive in other direct support, such as disability payments. While there was great variance among the scenarios, they show the financial impact interventions could have on the financial benefit to the government if they could keep someone working.

“This would suggest that the ability to prevent health events or halt disease progression early in the disease course may enable people to remain productive: this will have benefits on earnings and wealth accumulation, as well as reducing demand for public benefits now and in the future,” the authors write.

One downside to this approach is the fact that patients who die at an early age can save governments money, a problematic reality for anyone who wants to rely on a cold economic approach. Nevertheless, the authors provide a way for people to consider the public costs of rare disease on society, and that may lead to a more nuanced discussion of the value of rare disease therapies.

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