Hospitals, industry forging new path toward individualized therapies | Pediatric Research
Personalized, customized, individualized, bespoke — all these words are synonyms as they are used in everyday language. But when it comes to highly sophisticated therapeutics, they can have unique meanings and actually represent a spectrum of specificity.
A couple of weeks ago, I heard a fantastic analogy for this spectrum. Peter Marks, M.D., Ph.D., director of the Center for Biologics Evaluation and Research at the Food and Drug Administration, was part way through his keynote address at the Nationwide Children’s Hospital Office of Technology Commercialization Showcase, when he flipped to a slide with three suits pictured.
The first suit, “off the rack,” represents personalized medicine. For most people, finding a therapy from the available treatments will work out pretty well.
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The second suit, which has been “made to measure,” represents customized medicine. An available therapy is customized or “tailored” to a patient or group of patients.
The third suit is “bespoke.” It’s made completely to the specifications of the individual customer, from the design and fabrics to the buttons and the label. This level of precision and uniqueness represents individualized therapy. Individualized, or bespoke, therapies are created for a specific individual or just a few people. Currently, most bespoke therapies are gene therapies.
Bespoke therapies are becoming increasingly possible, but getting them from the lab to the clinic is often another story.
Technology commercialization is the process of taking a discovery or invention and making it commercially viable and available. While a lot of research and innovation work is happening in research institutes and hospitals, those places don’t often have the resources to scale up to make the discovery available for everyone who needs it. This is especially true for those bespoke therapies.
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Through technology commercialization and working with commercial partners, however, researchers can expand the impact of their discoveries.
But before a patient can receive a new therapy, the FDA must approve it. Whether the use is investigational (in a clinical trial), compassionate use (potentially helpful to a patient who has no other options) or commercial (fully approved and clinically available), the FDA has processes in place to ensure the drugs and therapies they approve are safe and effective. But those processes were not developed for bespoke therapies.
That’s why the National Institutes of Health and the FDA have formed the Accelerating Medicines Partnership program, a private-public partnership designed to improve our understanding of and validation of information that could help move therapies to patients faster. Established in 2021, the AMP Bespoke Gene Therapy Consortium was created to develop platforms and standards that could make development, approval and delivery of bespoke gene therapies much faster.
For patients with rare diseases, advancements in bespoke therapeutics are a beacon of hope. But when every second counts, it will take innovation in our regulatory, commercialization and research collaborations to make their promise a reality.
Abbie (Roth) Miller is the managing editor for Pediatrics Nationwide and manager for science and medical content at Nationwide Children’s Hospital.
Abbie.Roth@nationwidechildrens.org
This article originally appeared on The Columbus Dispatch: Hospitals, industry forging new path toward bespoke therapeutics
