Expert Q&A: Gene-editing treatment for sickle cell disease may be out of reach for many

Federal approval of a breakthrough gene-editing technology that treats the pain and debilitating effects of sickle cell disease is cause for celebration among a community with few options for relief. 

The FDA's approval of CRISPR-based Exa-cel means patients with the inherited disease—more than 100,000 Americans—now have a treatment option other than a bone marrow transplant.

As welcome and significant as the medical breakthrough appears to be, it also comes with concerns that too few people can afford to pay for the therapy. There is also the issue of limited access and demands that come with the high-tech, intense 3- to 6-month treatment, which will, at least initially, be offered by only a handful of health care providers.

Melissa Creary, Ph.D., an IHPI member and assistant professor in health management policy and global public health in U-M's School of Public Health, is a social scientist who has worked with the sickle cell community as a scientist, policymaker and public health researcher for 20-plus years. 

As someone living with sickle cell, she brings an especially personal perspective to her expertise on sickle cell.

What are your concerns around access to this treatment, which is estimated to cost $1 million to $2 million?

In theory, this is going to be available to anyone with sickle cell who satisfies the eligibility criteria, but in reality the various levels of social and financial support required are likely to be challenging. Your doctor may not alert you to the availability of the treatment. If they do, you may not be able to afford it. If you can, you and your family might have to travel to receive the care. There are a number of barriers in place for the people who stand to benefit. 

How can that be addressed?

Hopefully, different stakeholders throughout the sickle cell community will make sure that information about this technology is widespread. Then it's about knowing all your options. There is information from the NIH and the Sickle Cell Disease Association of America available to those who are thinking about accessing this technology. This information can help you understand the kind of support you might need before, during and after. There's a lot to consider not just for the affected patient but for those who need to take care of you. Can they take off work? Can they afford to go with you? In practice it's going to be life-changing for some and not attainable to others, and this unfortunately is not unusual when it comes to high-tech medically advanced health care.

What is known about insurance coverage or Medicaid and Medicare benefits to pay for the treatment? 

We know that the federal government and places like CMS (Centers for Medicare and Medicaid Services) are working to figure out what payment models could look like. I don't think any of us know what that is as of now. What we do know is that a very large percentage of people living with sickle cell are on Medicaid, so extending Medicaid benefits to cover the treatment will be extremely important in guaranteeing access.  

Why is cost and accessibility not an integral part of the drug, medical development process?

I understand why cost and accessibility aren't part of the process, but I would love for that paradigm to shift. I would love for scientists to understand it's one thing to develop a transformative advancement, and it's another to make it accessible.

While there may be equity issues with the treatment, it's been said that Rodger Novak, the CRISPR technology developer, advocated for marginalized or ignored communities by choosing sickle cell as one of its diseases to target with CRISPR. Your thoughts?

CRISPR stands to transform the lives of those who are able to receive the technology. Many stakeholders have understood that by choosing sickle cell. It signals a recognition of the long history of inequities associated with the disease and the people living with the disease. As the technology comes to market and out of the trial phase, it will be interesting to see how the tensions between social justice and profits work themselves out. The technology as a tool for addressing the genetic mutation and providing life opportunities is promising, but it cannot fully address the deep inequities embodied in the experiences of the majority Black and brown people even once cured.