New FDA Approved Sickle Cell Disease Treatments cost $2 - $3 Million.

How much should a drug cost?

Sickle Cell Disease impacts about 100,000 people in the US every year. It’s an incredibly painful disease where the cells become misshapen like a crescent moon or a sickle. This shape causes the cells to get trapped and restrict the blood flood which causes chronic pain, organ damage, strokes, and shortened life expectancy.

Bone marrow transplant has been the best treatment for patients, but it was very challenging to find a potential donor. Only 15% of siblings are a suitable match to be a donor and the chances of finding a match in the general population is about 10%. This means that only about 25% of patients suffering with Sickle Cell Disease had an option for treatment.

The FDA has now approved two new treatments, Casgevy and Lyfgenia.

“Both products are made from the patients’ own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant. Prior to treatment, a patients’ own stem cells are collected, and then the patient must undergo myeloablative conditioning (high-dose chemotherapy), a process that removes cells from the bone marrow so they can be replaced with the modified cells in Casgevy and Lyfgenia. Patients who received Casgevy or Lyfgenia will be followed in a long-term study to evaluate each product’s safety and effectiveness.” (Link to FDA release)

“bone marrow transplant was the first potential cure for sickle cell disease, but trying to find a good match for a transplant is a big barrier. This new technology uses gene therapy to allow patients to be their own match.” - Cece Calhoun, MD, MBA, a Yale Medicine hematologist-oncologist.

This is a huge advancement and this same type of gene therapy could be offering hope for other diseases in the near future.

But what about the cost?

Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2 million.

Lyfgenia the competing genetic medicine is priced at $3.1 million.

Those price points are hard to imagine. But let’s put some context around them..

Lets compare the cost of the new treatments versus the current standard of care. Insurance companies and the industry estimates the cost of managing sickle cell over a lifetime for someone with recurrent pain is between $4 - $6 million.

So $4-6 million for the current treatment vs $2-$3million for the new treatment.

How much did it cost to develop Casgevy? That has been a bit hard for me to track down exactly. Vertex (the maker of Casgevy) and CRISPR Therapeutics have an interesting development agreement where they split the cost and profits 60:40. Vertex made a $200,000,000 payment to CRISPR Therapeutics to cover some of the R&D expense as they crossed the milestone of getting FDA approval.

However, this doesn’t really represent the entire cost of creating Casgevy, because it relies upon the technology created with CRISPR itself. CRISPR alone has had more than $1billion in R&D expense in the past 3-4 years. So how much did it cost to develop Casgevy? It’s hard to know exactly, but well into the billions of dollars for sure.

So should Casgevy cost $2.2million per treatment? I don’t know. But it’s something we are going to have to think about as a society. On one hand, we want to be able to fund research and development that invents new technologies that could help hundreds of thousands of people every year. But we also don’t want a health care system that is so large that the budget eats into the entire US economy.

It’s going to be interesting to see how this plays out over the coming years. This isn’t the last drug that is going to cost millions of dollars to get.