February 26, 2024

How should funds be allocated for rare diseases?

Brian Wallach and Sandra Abrevaya are a power couple in Chicago. They met during Obama’s campaign for President and both worked in the White House under Obama. They have impressive connections in the Biden Administration, as well.

But a lot has happened since their marriage in 2013. In 2017 Wallach was diagnosed with amyotrophic lateral sclerosis (ALS), one of the most terrifying of many rare diseases. It leads to gradual paralysis and death within three to five years from diagnosis. Wallach is still alive, but severely handicapped – at this stage he can barely communicate.

But this has not stopped him and his wife from using their impressive professional skillset to lobby for funding for ALS research. According to a feature in STAT:

[They] have galvanized what is likely the most successful patient advocacy campaign of the 21st century. Since starting from scratch in 2019, the couple has built a movement that culminated, last month, in President Biden signing legislation to fund $600 million of ALS research and patient-focused programs in the next six years.

While their fortitude and fierce energy are inspiring, is it fair that ALS receive gigantic funding while other rare diseases languish without celebrity backing? ALS is a terrible disease. However, more common diseases affect far more people in the developing world and receive relatively little funding. How should funding decisions be made? – by lobbying? by severity? by how common they are?

One facet of this issue is discussed in a recent issue of the Journal of Medical Ethics. Monica Magalhaes, of Rutgers University, attempts to clarify the ethical issues. She contends that “the goal of orphan drug policy should be to make prevalence irrelevant to funding decisions”. Her conclusion is:

Current orphan drug policies aim to address the lack of access to treatments by patients with severe rare diseases, out of a concern that meeting their claims is as important as meeting claims of patients with common diseases. The prevalence of a disease is irrelevant to the strength of these claims. Accordingly, policies aiming to treat equal claims equally should make prevalence irrelevant to funding decisions. It is severity, not prevalence, that drives our judgments that important claims are being overlooked when treatments for severe rare diseases are not funded. Unlike prevalence, severity is an appropriate consideration for priority setting. Therefore, we have reasons to raise the cost-effectiveness threshold for costly severe diseases, or otherwise give these diseases some form of special treatment, regardless of how rare they are.