This article discusses the advent of bespoke therapies, defined as the tailoring of medical treatment to the individual characteristics or symptoms and responses of a patient during all stages of care and as a new frontier beyond personalized medicine. The author covers the revolutionary genetic tools implementing such therapies and the clinical and nonclinical safety perspectives for bespoke therapies. The author concludes that with bespoke therapies we are entering a new era of highly individualized therapies where traditional paradigms of drug development are being significantly challenged.IntroductionBeginning in the mid-1980s and through the last decade, several scientific discoveries have paved the way for ultra-personalized therapeutics. Among these discoveries were the chemical synthesis of oligonucleotides (short bits of DNA and/or RNA), the mapping of the human genome, and the mechanisms of RNA interference (RNAi) and CRISPR (clustered regularly interspace short palindromic repeats) technologies.Having access to the map of the human genome and powerful genetic tools, such as RNAi and CRISPR, has allowed scientists and healthcare and regulatory professionals to forge a new therapeutic frontier. The first step toward this frontier came with the development of personalized medicines, more commonly referred to as gene therapies. In personalized medicines, a gene known to be the cause of a disease state for a significant population of individuals is targeted for therapeutic intervention. The second step pertained to the development of bespoke therapies, often referred to as the next chapter in personalized medicine.A bespoke therapy is a medical treatment that has been tailored to the specific individual characteristics, symptoms, and responses of a patient during all stages of care. The genetic tools developed in recent years have made these individualized therapies a reality. This approach translates into the treatment of a small patient population suffering from rare or ultra-rare genetic diseases for which the therapeutic agent is designed specifically for an individual.In March 2020, the FDA held a workshop on the development of individualized therapeutics to connect with the scientific and regulatory communities to better understand the needs, challenges, and opportunities in the bespoke therapies landscape.1 Based on its insights from the workshop, the agency plans to develop guidance to foster progress in the nascent field while recognizing and protecting the rights of the individual patients who will benefit from these therapies.Traditional models of regulation, clinical trial design, and business development are no longer valid for ultra-personalized therapies. Several questions arise:
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Bespoke therapies opportunities, challenges, and hope - Regulatory Focus
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