NIH launches real-world data platform to advance autism research and broader health goals

The National Institutes of Health (NIH) is launching a real-world data (RWD) platform to support research into autism spectrum disorder (ASD) and chronic diseases, NIH Director Dr Jay Bhattacharya announced during the April 21, 2025 Council of Councils meeting. The platform will integrate disparate datasets across federal agencies and the private sector to support research aimed at understanding the causes, treatments, and progression of ASD and other complex conditions.

“What we’re going to do at the NIH is launch a real-world data platform,” Bhattacharya said. “The idea of the platform is that the existing data resources are often fragmented and difficult to obtain. The NIH itself will often pay multiple times for the same data resource.”

Integrated data for autism and chronic disease research

The initiative responds to a request from President Trump and US Department of Health and Human Services (HHS) Secretary Robert F Kennedy Jr to accelerate understanding of ASD’s etiology. Bhattacharya emphasized the need for comprehensive research to address the increasing prevalence of ASD, citing a CDC estimate that 1 in 31 eight-year-old children had an ASD diagnosis in 2022.

“Autism spectrum disorder is a highly heterogeneous condition with many potential causes,” he said. “Understanding its etiology is absolutely crucial to developing treatments for the range of conditions that this disorder represents.”

According to Bhattacharya, the NIH will fund, “between 10 to 20 groups of researchers from across the country,” through a competitive grant process. These projects will use a range of methods, including basic science, epidemiology, and applied approaches, to investigate causes, risk factors, and treatment options.

“I recognize, of course, that autism is a range of manifestations, ranging from highly functioning children to children that are quite severely disabled. And, of course, the research will account very carefully for that.”

The RWD Platform will integrate data from pharmacy chains, health organizations, electronic health records (EHRs), claims and billing systems, environmental monitoring, and wearable technologies. It will support longitudinal datasets, drug development, regulatory approvals, and real-time health monitoring.

Protecting privacy and enabling access

Bhattacharya emphasized that while researchers will have access to the data, strict privacy protections will be in place.

“Researchers will be able to do their work on the platforms, but not be able to download the data themselves,” he said. “There are state-of-the-art protections to make sure... that these linkages do not in any way threaten the confidentiality of patients.”

The NIH is currently negotiating data use agreements with the Centers for Medicare and Medicaid Services (CMS) and other federal entities to expand data access. The initiative will also build on existing NIH efforts, such as the HEALthy Brain and Child Development Study and the NIA Data LINKAGE Program with CMS data.

“This platform will feature the cutting-edge knowledge that the NIH already has,” Bhattacharya noted.

Clarifying registry controversy

Although Bhattacharya mentioned the development of a national autism registry during his remarks, the HHS later clarified that no centralized autism registry will be created. Instead, the RWD Platform will link existing datasets to support research. The clarification followed privacy concerns raised by advocacy groups.

The Autism Science Foundation stated that, “The use of registries and registry data in general can be a valuable tool in helping to understand the causes of diseases and disorders, but in this case, the lack of clarity around how data will be collected, shared, maintained and tested for accuracy raises red flags.” Privacy advocates also warned about the implications of aggregating sensitive health and disability data.

A vision for biomedical research

In addition to the autism initiative, Bhattacharya used the session to outline his broader vision for the NIH under the “Make America Healthy Again” strategy. He outlined five major priorities for NIH moving forward: addressing chronic disease, restoring reproducibility in science, supporting bold research ideas, ensuring research safety and ethics, and fostering academic freedom.

“We have to find better ways to prevent, treat and cure chronic diseases,” Bhattacharya said. “Our research is not simply focused on how to manage late-stage chronic disease, but also on how best to prevent the development of chronic disease.”

He emphasized the need to rebuild public trust in science by making replication a central part of the research process:

“The practice of doing replication will become something that’s normalized in the scientific community under my watch.”

He also reaffirmed the NIH’s commitment to scientific integrity, academic freedom, and open debate:

“The NIH absolutely must play a leading role in encouraging academic freedom,” he said. “Scientific progress depends on the right for researchers to dissent against scientific dogmas.”

Bhattacharya acknowledged HHS Secretary Robert F Kennedy Jr’s directive to find the cause of autism by September. NIH is investing $50 million to support research into the causes and treatments for autism. Speaking to reporters, Bhattacharya said:

"We're going to get hopefully grants out the door by the end of the summer… people will get to work. We'll have a major conference, with updates, within the next year."