Your report, “Health Experts Question the Cost of Gene Therapy” (December 15), explores the future of medicine and the rapidly evolving science to treat and possibly cure devastating diseases. contributes to important conversations about social competence.

Cost and access should be discussed. But we also need to ask whether we can afford to sustain our current healthcare system, where too many patients face death and severe disability, with few or no treatment options. For those who are “lucky” enough to have options, the status quo is often a burdensome chronic treatment that may only partially relieve symptoms and suffering.

Cell and gene therapies seek to disrupt this broken model. Currently, there are innovative treatments for diseases that are resistant to conventional treatments such as chemotherapy, such as spinal muscular atrophy, hemophilia, beta-thalassemia, and blood cancers such as leukemia. Patients are benefiting. 17-year-old American Emily Whitehead, who just celebrated her 10th anniversary of cancer, has become the world’s first pediatric patient to receive chimeric antigen receptor T-cell therapy for aggressive leukemia. rice field. She responds to other treatments.

We know that cell and gene therapies are challenging the traditional medical system by design. That is why we are working with payers and stakeholders in Europe, the United States and beyond by creating new and innovative reimbursement models that share risk across multiple stakeholders and improve patient access. are modernizing their healthcare system. In contrast to the way the healthcare and pharmaceutical markets have historically worked, cell and gene therapy developers are often willing to reimburse payers if their treatments do not deliver the intended effect for patients. It happens often.

It is a critical time for Europe through changes to EU medicines law, and important reforms to the government-run insurance system in the United States are needed. The future of medicine is rapidly approaching. The healthcare system needs to catch up with science.

Timothy D. Hunt
Alliance for Regenerative Medicine Chief Executive, Washington DC, USA