Policy experts call on Medicare to ensure data from Alzheimer’s drug registries is shared publicly

More than two dozen prominent health policy experts are urging the Biden administration to ensure that patient data collected about a new Alzheimer’s treatment is accessible to the public, which they say is critical to understanding the risks and benefits of the medicine.

The drug, called Leqembi and made by Eisai, is the first to have been found to slow progression of the disease. Before providing coverage, Medicare officials are requiring physicians to submit data to a patient registry for the medication. The agency runs such a registry and has committed to making the data available to researchers.

For now, the only option for doctors is to submit to this registry. But the Centers for Medicare & Medicaid Services has indicated it will allow physicians to submit data to registries run by other organizations. But in a letter sent to federal health officials Thursday, health policy experts expressed concern that registries developed by private companies may limit access to their respective patient data.

If that were to happen, they warned, Medicare and independent researchers would have a hard time gaining a complete picture of patient outcomes. And for this reason, they argued that Medicare coverage should not be provided for any registry that fails to appropriately share population-level patient data.

“We are very concerned that Leqembi registries developed by medical societies, medical centers, nonprofit organizations, or other non-governmental entities may limit access to their respective data potentially making it difficult for (Medicare) and other researchers to conduct independent analysis of the data,” wrote the experts, who are medical professors and bioethicists at leading universities.

Patient registries are especially valuable for population health researchers who want to understand treatments as they are used in the real world — not just in the subset of patients who qualified for a clinical trial or lived close to a study site. Ideally, a patient being treated in a small specialist’s office in Missouri could be included in the same research as one who visits a large academic medical center in Boston.

Collection of standardized patient data in a registry, along with careful anonymization of health records, makes it possible for registries to be used as powerful epidemiological tools, not just post-approval safety and outcomes data. But that real-world evidence loses much of its value when it’s not collected consistently and shared transparently.

Already, CMS has approved one patient registry that will be run by Beth Israel Deaconess Medical Center in Boston, which is starting to enroll patients. A hospital spokeswoman wrote to say that “patient privacy regulations prevent us from sharing data from any registry publicly.”

She added, using registry data, the hospital has a study approved by CMS to evaluate outcomes of such drugs. The data will be submitted for peer-reviewed publication in the future and the hospital  will share de-identified population data through collaborative studies that have been approved by its institutional review board.

Meanwhile, the Alzheimer’s Association, a patient advocacy organization, is creating its own registry to collect information on patient outcomes. Although its nascent registry, which is called ALZ-NET, does not yet qualify for Medicare coverage, it is the only one mentioned in the Leqembi prescribing label that is targeted at physicians.

The organization is still waiting for CMS to approve its registry, but plans to provide access to “aggregated data… for a variety of stakeholders,” including CMS and the Food and Drug Administration, a spokesperson wrote us. “This is why we are collecting regulatory-grade data, so they can be useful to a variety of investigators and government agencies.”

Nonetheless, the Alzheimer’s Association, which plans to pay study sites to participate in its registry, continues to argue that registries should not be a requirement for Medicare coverage and urged CMS to reconsider its policy.

“To be clear, the Alzheimer’s Association believes that appropriately designed and implemented registries are valuable research and quality improvement tools to collect and apply real-world evidence. However, they should not be used as a condition of coverage,” the advocacy group said in a statement. “Doing so is unnecessary, and poses a risk to access particularly for underserved communities.”

However, one of the experts pushing for data sharing argued that making only aggregated data available is insufficient.

“Aggregated data means it is looking at a group of people combined and already analyzed and the data are presented in summary form. They aren’t data that can be analyzed by anyone else.” explained Diana Zuckerman, who heads the National Center for Health Research, a nonprofit think tank. “What we are seeking is a dataset that can be analyzed to answer new questions or re-analyze the answers to previously answered questions.”

The experts also went on to note that different registries can pose another problem — a lack of consistent data. “If different measures are used by different registries, and those registries also vary in terms of the diversity of patients or other variables studied, it will be difficult if not impossible to draw conclusions” about the impact Leqembi is having on cognitive impairment or safety, they wrote.

Katie Palmer contributed to this story.

The story was updated with comment from Beth Israel Deaconess Medical Center.