Month: February 2024

Elizabeth Currid-Halkett writing in the NYTimes discusses her son’s muscular dystrophy and his treatment with the controversial gene-therapy Elevidys. Currid-Halkett, like many parents whose children have been treated with Elevidys, reports much better results than appear in the statistics.

On Aug. 29, [my son] finally received the one-time infusion. Three weeks later, he was marching upstairs and able to jump over and over. After four weeks, he could hop on one foot. Six weeks after treatment, Eliot’s neurologist decided to re-administer the North Star Ambulatory Assessment, used to test boys with D.M.D. on skills like balance, jumping and getting up off the floor unassisted. In June, Eliot’s score was a 22 out of 34. In the second week of October, it was a perfect 34 — that of a typically developing, healthy 4-year-old boy. Head in my hands, I wept with joy. This was science at its very best, close to a miracle.

…a narrow focus on numbers ignores the real quality-of-life benefits doctors, patients and their families see from these treatments. During the advisory committee meeting for Elevidys in May 2023, I listened to F.D.A. analysts express skepticism about the drug after they watched videos of boys treated with Elevidys swimming and riding bikes. These experts — given the highest responsibility to evaluate treatments on behalf of others’ lives — seemed unable to see the forest for the trees as they focused on statistics versus real-life examples.

Frankly, I side with the statistics. We don’t hear from the parents in the placebo group whose children also spontaneously made improvements.

Even though I side the statistics, I side with approval. Innovation is a dynamic process. It’s not surprising that the first gene therapy for DMD offers only modest benefits; you don’t hit a home run the first time at bat. But if the therapy isn’t approved, the scientists don’t go back to the drawing board and keep going. If the therapy isn’t approved, it dies and you lose the money, experience and learning by doing that are needed to develop, refine and improve.

Approval is not the end of innovation but a stepping stone on the path of progress. Here’s an example I gave earlier of the same principle. When we banned supersonic aircraft, we lost the money, experience and learning by doing needed to develop quieter supersonic aircraft. A ban makes technological developments in the industry much slower and dependent upon exogeneous progress in other industries.

You must build to build better.

Addendum: Peter Marks is the best and perhaps the most important director CBER has ever had. CBER, the Center for Biologics Evaluation and Research, is responsible for biological products, including vaccines and gene therapies. Marks has repeatedly pushed and sometimes overruled his staff in approving products like Elevidys. Marks named and was the driving force at the FDA behind Operation Warp Speed, a tremendous FDA success and break with tradition. Marks has been challenging the FDA’s conservative culture. I hope his changes survive his tenure.

Works in Progress has a good review of the state of compensating organ donors, especially doing so with nudges or non-price factors to avoid backlash from those who find mixing money and organs to be repugnant. My own idea for this, first expressed in Entrepreneurial Economics, but many times since is a no-give, no-take rule. Under no-give, no-take, people who sign their organ donor cards get priority should they one day need an organ. The great virtue of no-give, no-take is that it provides an incentive to sign one’s organ donor card but one that strikes most people as fair and just and not repugnant. Israel introduced a no-give, no-take policy in 2008 and it appears to have worked well.

In March 2008, to increase donations, the Israeli government imple­mented a ‘priority allocation’ policy to encourage more people to sign up to donate organs after their deaths. Once someone has been registered as a donor for three years, they receive priority allocation if they themselves need a transplant. If a donor dies and their organs are usable, their close family members also get higher priority for transplants if they need them – ​which also means that families are more inclined to give their consent for their deceased relatives’ organs to be used.

In its first year, the scheme led to 70,000 additional sign-ups. The momentum continued, with 11.1 percent of all potential organ donors being registered in the five years after the scheme was introduced, compared to 7.7 percent before. According to a 2017 study, when presented with the decision to authorize the donation of their dead relative’s organs, 55 percent of families decided to donate after the priority scheme, compared to 45 percent before.