Jaap Jan Boelens

Jaap Jan Boelens, MD, PhD, is Chief Medical Officer of the International Society for Cell and Gene Therapy (ISCT). A clinician who came up through bone marrow transplant, he was pulled into advanced therapies more than two decades ago and now treats the patients living inside the access gap he describes.

Boelens sees cell and gene therapy from the chair across from the patient. He treats sickle cell patients weighing a gene therapy they qualify for against a standard transplant, and he has watched single-case agreements with payers drag on so long that patients back out of the newer therapy entirely. He is a proponent of the long-term, real-world monitoring these products demand, noting that while the field has five decades of data on bone marrow transplant, it has only a few years on many ex vivo gene therapies, for patients who may need the result to hold for decades.

Boelens appeared on Open Door Salon alongside Bambi Grilley, President-Elect of ISCT, for a conversation with host Lori Ellis on why patients walk away from cures they qualify for. He explained why the contracting and single-case-agreement phase is its own barrier to access, why in vivo gene therapy for genetic disease is likely more than a decade away, and what worries him most about what we do not know regarding cell and gene therapy activity in China.