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Medical technology is revolutionizing healthcare for patients worldwide and is now more than ever becoming crucial in the prevention, diagnosis, treatment, and management of numerous diseases. We are increasingly seeing the ability of medical technology to personalize treatments and target patients’ individual needs. However, to ensure we are enabling access to these treatments, regulatory agencies need to consider new approaches to the approval process.
Historically, product approvals have focused on the standardization of a drug or a therapy to treat a large population of people with the same disease, safely and effectively. But in this new age of personalized care, these standards are insufficient for products coming to market that are designed to meet unique patient needs.
Currently, regulatory agencies including FDA seek large clinical data sets to demonstrate that drug products are safe and effective for patients. While safety and efficacy need to be held to the highest standards for certain areas of healthcare, such as regenerative medicine, these data sets are both difficult and unnecessary to achieve. In cases where the patient population is small and varied, requiring large clinical trials creates needless challenges.
Large clinical trials are very costly to run, difficult to recruit patients for, and often do not align with clinical endpoints for products that are intended to work individually for patients as opposed to a large population pool. Companies may be unable to find the required number of patients to enroll in large trials, or the cost of the trial may be prohibitively expensive, so without more nuanced approaches to product approval, smaller companies cannot move their innovations forward.
Sports medicine, for example, is at an all-time high, and ACL reconstruction is a growing need that regenerative medicine can help meet. Regenerative medicine allows a patient’s own body to repair itself, providing longer-lasting solutions. The physical and psychological benefits to patients are significant compared with that of other treatment options. Yet the data required to provide a statistically relevant clinical trial are too onerous and expensive to allow commercialization of such products. To offer patients truly transformative and curative treatments, regulatory requirements need to balance approval of products that are designed to meet specific needs with ensuring the safety of the general public.
The solution to this large data burden could be found in real-world data (RWD). RWD provides real-time patient data that can be used to assess a product’s safety and efficacy. When supplemented with smaller clinical trials, RWD could allow the approval environment to be more agile and efficient while also reducing costs.
RWD would not only help to advance healthcare innovations, but it could also improve how data is collected and stored. Mandatory data registries have been successfully developed in countries like Norway and Sweden and could effectively and affordably bridge the gap in data requirements needed for product approvals, increasing the validity of the data in return.
Of course, ensuring that safety and efficacy are still held at the highest standards is of paramount importance, but in certain areas of healthcare, regulatory agencies could consider adapting regulatory pathways to enable new, life-changing treatments for patients without sacrificing quality. As the healthcare landscape transforms, so must our traditional processes in order to unlock potentially beneficial treatments and continue to advance scientific innovation for patients worldwide. Promisingly, FDA has already announced a new plan for modernizing its technology infrastructure to use data sets more efficiently so it can consider new data, like RWD, more easily across the agency. We look forward to seeing this plan develop and hope that our healthcare systems can evolve to meet patients’ personalized needs.