Category: Law

ProPublica: In January, the Biden administration pledged to increase public access to a wide array of Medicare information to improve health care for America’s most sick and vulnerable.

…So researchers across the country were flummoxed this week when the Centers for Medicare and Medicaid Services announced a proposal that will increase fees and diminish access to claims data that has informed thousands of health care studies and influenced major public health reforms.

Using big Medicare databases has never been cheap or easy. Under the current system, researchers could have the data transferred to secure university computers for about $20,000–that’s a lot but once the data was on the university computers it could be accessed by multiple researchers, cutting costs. A professor could buy the data and their PhD students, for example, wouldn’t have to pay again. Under the new system it will cost $35,000 for one researcher to access the data which will be held on government (CMS) computers. Moreover, it’s unclear how complex statistical analysis will be performed or how congested the CMS systems may become.

Research teams on complex projects can include dozens of people and take years to complete. “The costs will grow exponentially and make access infeasible except for the very best resourced organizations,” said Joshua Gottlieb, a professor at the University of Chicago’s Harris School of Public Policy.

Public data should be open access to researchers, with appropriate anonymization. We know from IP law that barriers to access reduce research and innovation; and in the medical sphere research and innovation saves lives. Open access is also a check on how governments spends taxpayer money and the effectiveness of such spending. I also worry that raising the dollar cost of access is a prelude to other restrictions. The NIH, for example, is restricting access to genetic data if it thinks the researcher will be asking forbidden questions. Even without such explicit restrictions, there is a chilling effect when researchers are beholden for access to the government and indeed to the very agencies they may be researching.

I place a high value on privacy but I get suspicious when governments invoke privacy to block citizen access to government data but not to block government access to citizen data. Medicare databases have always been appropriately anonymized and care is taken so the data are secured but the dangers of these databases in anyone’s hand, let alone researchers, is far less than anti-money laundering, KYC laws and suspicious transaction reports in banking, automated license plate readers that the police us to scan billions of license plates or mass surveillance of the communications of US citizens under FISA. Sadly, this list could easily be extended. Liberty thrives on the people’s privacy and the government’s transparency.

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.