Anthem’s decision to not pay for Exondys 51 for Duchenne’s Muscular Dystrophy is not surprising, since there is no solid scientific evidence that the drug works. This is exactly the concern we had when the FDA approved Exondys 51, over-ruling FDA scientists who agreed that the drug did not meet the requirements of proof that it is effective. FDA knows that health insurance companies do not pay for experimental treatments, which is one of the reasons why FDA is not doing patients any favors when they approve a drug or device that is not proven to work. We continue to strongly urge Sarepta to do the studies needed to support their claims that patients benefit from Exondys 51, and in the meanwhile Sarepta should ensure that patients in those studies receive the treatment for free.
In order to keep health insurance affordable, insurance companies need to ensure that they are paying for safe and effective treatments. When FDA fails to ensure those standards, then “FDA approval” is no longer a gold standard that insurance companies can rely on. Had FDA delayed an approval decision until the company proved that the product is effective (or not), patients could have continued to get the drug for free in clinical trials. Now that the drug is approved and costs about $300,000/year, who will be able to afford it? This is a tragic situation for patients and their families, and Sarepta and the FDA share responsibility for it.
This is a major issue of importance to all patients and physicians, not because of this one drug, but because FDA approved a drug despite its own scientists saying the drug doesn’t meet FDA standards for approval. FDA should listen to patients, using that information to understand what patients need as well as what their positive and negative experiences when using a medical product. But the bottom line is that FDA should not approve a medical product unless it is proven to be safe and effective based on solid scientific evidence. Listening to objective, knowledgeable scientists and listening to patients should go together, not oppose each other.
To see Anthem ‘s statement on Exondys 51, see (https://www.anthem.com/medicalpolicies/policies/mp_pw_c192386.htm).