FDA officials: Transforming evidence generation to tackle chronic diseases

Regulatory NewsRegulatory News | 05 February 2024 | Mary Ellen Schneider

FDA Commissioner Robert Califf at the 2022 MedTech Conference in Boston, MA. Photo credit: Ferdous Al-Faruque

Leaders at the US Food and Drug Administration (FDA) are putting forward strategies to address the epidemic of chronic disease, with a focus on improving the quality of evidence generation for medical interventions through innovative trial design, use of reliable biomarkers, and leveraging artificial intelligence.
 
The perspective, published in the New England Journal of Medicine on 3 February 2024, is authored by FDA Commissioner Robert Califf, as well as Hilary Marston, the agency’s chief medical officer, and Haider Warraich, senior advisor for chronic disease.
 
“The FDA’s remit spans drugs, biologic products, medical devices, food, and tobacco products. With collaboration among stakeholders, we can improve the way in which evidence is generated and interventions are developed and implemented to help address common chronic diseases,” Califf and colleagues wrote.
 
They proposed a strategy of “transforming evidence-generation methods,” specifically changing how the safety, efficacy and clinical usefulness of a chronic disease treatment is studied.
 
“These diseases often require lifelong treatment, which means that benefits may accrue or adverse events may occur over periods that exceed the length of traditional randomized, controlled trials,” they wrote.
 
Moving forward, trials may need to be longer and rely more on biomarkers and surrogate endpoints to evaluate efficacy. Additionally, randomized trials may need to have more relaxed inclusion criteria and employ other innovative approaches to recruit a representative participant pool. These approaches may include pragmatic trials and decentralized trial elements, FDA officials wrote.
 
The authors stressed the importance of developing and evaluating reliable biomarkers and surrogate endpoints for certain outcomes. The need is “urgent” as many investigational therapies are showing positive results in phase 2 trials but are not deemed to be effective in phase 3 trials. “The FDA also encourages assessment of functional status and quality of life using validated tools for measuring clinical outcomes, including patient-reported outcomes,” Califf and colleagues wrote.
 
Patient-centered approaches are especially important in chronic diseases, the FDA officials wrote, because these diseases disproportionately impact underserved populations. Additionally, chronic diseases often involve lifelong treatments and lifestyle changes, making patient input critical.
 
Technology also has a role to play in the postmarket setting. For instance, data from electronic health records could assist with the postmarket evaluation of health technologies or in routine clinical monitoring, while artificial intelligence could be used to reduce administrative burdens of postmarket surveillance and in evaluating safety reports. However, Califf and colleagues cautioned that it is important to understand the limitations of these technologies.
 
FDA is already moving forward in these areas. The perspective article details initiatives to support pragmatic clinical trials, investments in regulatory science, requirements for confirmatory trials, and guidance aimed at increasing trial enrollment among older adults.
 
“The pervasiveness of common chronic diseases requires a broad coalition, one that should include the biomedical and digital health industries, health systems, payers, patients, patient advocates, policymakers, and other government agencies. The reward for such a united front could be the realization of a thriving and more equitable society,” Califf and colleagues wrote.
 
New England Journal of Medicine perspective

 

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