The Gourd

Vol 2, Issue 4 May 17, 2022

Sickle Cell Disease and The Cost of Care

This month’s issue of The Gourd is devoted to sickle cell disease; but rather than review the genetics of the disease or the structure of hemoglobin, we will be discussing recent advances from a Public Health perspective.

When Harvoni® was released in 2014 to treat Hepatitis C, many were shocked by the $95,000 price tag and insurers set out to limit access. In many cases, only those with the most advanced liver damage could get coverage of this potential cure. Today, that initial price tag sounds like a bargain compared to some of the newer genetic treatments that are giving hope to patients and families dealing with devastating diseases, such as spinal muscular atrophy (SMA). Zolgensma® received much media attention for its initial price of 2.125 million dollars, but its use is limited to children under the age of two with SMA but without end-stage weakness. One could argue that the relatively small number of eligible patients (700 each year by some estimates) warrants the high cost in order to cover expenses for research and development (R&D). In fact, an MIT analysis found a positive correlation between the size of the eligible population and the price tag of new genetic therapy.

Sickle cell disease, however, affects around 100,000 Americans. Worldwide, it is estimated that about 300,000 babies are born with this condition every year resulting in millions of people suffering from its effects. Numbers like these make the proposed one-million-dollar cost for a new genetic treatment from Bluebird Bio a bit hard to comprehend. Here in the United States, it would not be surprising if insurers again try to limit access to the treatments, perhaps by requiring documentation of more advanced disease (e.g., splenic infarcts or hip replacements).

Dr. Dhruv Khullar, writing for the New Yorker, gives an excellent overview of the history of sickle cell disease and the development of recent genetic approaches to treatment. He notes that the disease gets a tenth of the research funding that flows into cystic fibrosis despite affecting many more people. Readers of this e-zine will not be puzzled over the reason for this funding disparity. In fact, the reason we now have several candidates for a genetic treatment (really a cure for the individual) is that sickle cell disease arises from a single mutation, making it ideal for such research. But the gap between the promise of these new therapies and the reality of a cure for many is vast. As Dr. Khullar writes, “a treatment that exists is not the same as a treatment that helps people.” What good is a cure that is inaccessible to so many?

Obviously, sickle cell disease is of unique interest to the Black community but the growing problem of paying for emerging genetic therapies affects everyone. At least for now, healthcare in this country is driven by the market and it can be argued that market forces drive important innovation in therapy. This also makes price-capping a difficult proposition. However, healthcare is unlike other market-driven goods in that it can often be a life-or-death necessity; such high prices serve to exacerbate an already tragic lack of equity in healthcare. So, if we can not cap the price charged by companies, what do we do? An excellent blog post from Alison Irvine discusses several payment reform options that deserve consideration. If the speed of development of amazing new therapies continues to outpace payment reform, we will spend more and more money to treat fewer and fewer people.

The Cork Board

May 30 - Wellness Fair and Costume Contest hosted by NM African American Tobacco Prevention Network. Info here.

June 18-19 - Juneteenth celebration in Albuquerque. More info here.
Aug 5-6 - 22nd Annual Santa Fe Bone Symposium presented by the Osteoporosis Foundation of New Mexico. Register here.

Still showing at the Albuquerque Museum - Facing The Rising Sun: The Journey of African American Homesteaders in New Mexico

If you have information you would like to share with this provider community, send it to DRoss@NMBLC.org.

Share the link for this publication with other Black health providers in the state and encourage them to send their contact information (see email address at the bottom of the page) so they can be added to the distribution list.

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