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From ICER’s President and CEO Sarah K. Emond, MPP:

On Tuesday, I had the pleasure of participating in a fascinating panel titled “The Importance of Ethics in Cell and Gene Therapy” at the Alliance for Regenerative Medicine’s (ARM) Meeting on the Mesa. I shared the stage with four talented thinkers, including: ARM’S CEO, a Jesuit priest who has PhDs in molecular genetics and bioethics, the head of the International Society for Cell & Gene Therapy, and a thought-leader pharmacist whose career has spanned the life sciences, payer, and PBM industries. I was honored to offer ICER’s perspective.  

When scientists started sequencing the human genome twenty years ago, we could only dream of the astounding treatment landscape emerging today. Now that many of these treatments have arrived, we must meet the promise of this moment and reflect on the serious pricing and access considerations for cell and gene therapies.  

What companies charge society for their medicines, and what payers ultimately craft for access policies reflect a set of values about profits, fairness, and ultimately, patient and population health. Discussions about these values and the decisions that flow from them are especially urgent in the gene therapy space. Given the tremendous benefit that some therapies provide, a value-based pricing approach—like the one we use at ICER—awards effective treatments with price tags in the millions of dollars. This is good news for incentivizing truly innovative treatments, but tougher news for a health system desperately trying to afford them.  

During Tuesday’s panel, we discussed the need for innovative payment solutions to ensure fair price and fair access; we talked about what incentives are needed in the system to ensure future innovation; and we talked about the importance of prices being tied to the benefit the drugs can deliver for patients so that we can have sustainable access for all patients.  I learned a lot. And I applaud ARM for dedicating a panel to such important ethical questions!