now calls for healthcare delivery systems to catch up and develop

ways to equitably and justly deliver high quality care

It is an important moment to honor the hundreds of scientists, including two inspiring women in science (Emmanuelle Charpentier and Jennifer Daoudna, who won the 2020 Nobel prize for CRISPR gene-editting technology underpinning one of the two therapies), researchers, clinicians, and most importantly clinical trial volunteers and patient advocates who have worked hard, sacrificed, and in the case of the clinical trial volunteers, literally risked their lives, over decades to get our society to this moment. Collaborative, open science in thoughtful and engaged dialogue with patient advocates has won the day.

As Medical Director of the Adult Sickle Cell Center at Yale and professor of medicine, Dr. Cecilia Calhoun, so clearly stated on the Health and Veritas podcast, “because of the transatlantic slave trade, we are here.” (4)

The access and social justice problem associated with sickle cell care is as much a part of the issue as the genetic problem.

While the new gene therapies address the core genetic problem, they do not address the core social problem. We must ensure this historical scientific moment is leveraged to address both.

Sickle cell gene therapy is not cheap.

Over the lifetime of a person with sickle cell disease, the total cost of care for their condition to government health insurance programs is many fold higher than the $3 million one time gene therapy cost. From the perspective of the insurer and the tax-paying public, the $3 million is actually a really good investment.

For the other 93%, there are increasingly effective non-gene therapy treatments available, including good old fashion hydroxyurea and use of outpatient day hospitals and infusion centers instead of ED in acute setting. The major barrier to people accessing these therapies is our fragmented health system. Advanced primary care, care coordination, telehealth, and complex care models are the key to just sickle cell care. They become even more important in post-gene therapy era.