Do we have a plan for affordable genetic medicine?

The short answer is 'no', but we could build out this path in the next decade.

Hello friends,

I’m heading to a cell and gene therapy conference in Phoenix, AZ on Oct 7-9, Meeting on the Mesa. If anyone is attending the conference and would like to meet up, please respond to this email and we can schedule that in. The conference looks great. I’m ready for the Phoenix heat!

Broadening access to Genetic Medicine

I recently presented a seminar to the Caring Cross community on the commercial issues with Casgvey. Casgevy is the first gene editing drug to reach commercial status (FDA approved 12/2023).

Long term clinical data continues to demonstrate that Casgevy boosts hemoglobin levels and prevents pain symptoms (VOCs) in Sickle Cell Disease patients. This is great news for patients. However, as it stands today, only a small percent of Sickle Cell patients have access to this medicine. Casgevy is only approved in high income nations and it remains unclear how Medicaid patients in the U.S. will gain access.

Part of the challenge lies in the cumbersome drug delivery process (a process comparable to a bone marrow transplant). Patients must also be pre-treated with a harsh conditioning regiment (busulfan) and may need to stay many months in a hospital recovering from the treatment. Additionally, only qualified hospitals (authorized treatment centers) will contain the necessary medical infrastructure to deliver this medicine.

On top of those issues, the manufacturers of Casgevy set the list price at 2.2 million dollars. Vertex and CRISPR therapeutics are co-marketing the drug. This list price is for the U.S. and insurers may be able to negotiate down with rebates and discounts. Other high income nations (U.K., Bahrain, Saudi Arabia…) have made confidential negotiations for what I assume is a much smaller number.

As it stands now, Casgvey is not commercially available in any nation in Sub-Saharan Africa. This is a glaring omission since 80% of the 7.74 million Sickle Cell patients live in Sub-Saharan Africa.

For those familiar with the pharmaceutical industry, you will know that this is business as usual. New medicine (freshly branded and patented) first distributes to high income nations, most importantly to the U.S., since we will pay a premium price. Then after a few years, we may see some middle income nations negotiate a price with the pharma company (i.e. Brazil, Mexico). Finally, towards the end of market exclusivity (patents expiring) we may see the drug distribute into low income nations.

What can be done differently?

In the video below, I do my best to succinctly describe one solution to this inequitable distribution. I focus on a ‘bottom-up’ approach.

When new technology (i.e. Crispr/Cas9 gene editing tools) is developed in academic labs, the tech transfer office at the academic institutions could build in licensing provisions that demand biotech companies who license the IP must allocate a small percent of commercial revenue towards broadening out access to low income patients.

Most new technology coming out of academic or federally funded labs will never go on to contribute to an approved medicine. However, if we build in these licensing provisions, then the few big winners (i.e. Casgevy) will already contain a self-generating fund that can be directed towards enhancing patient access.

If this is of interest, please watch the video below. Tell me what’s wrong or right about this plan in the comments.

Kevin