The pace of new technology and novel medical treatments has reached a point at which regulators and assessors require additional expert evaluation in reviewing and assessing marketing authorizations. A recent Nature editorial has called for increased support from research scientists into translational medicine, specifically in regards to cutting edge and novel technologies now seeking marketing approval in both the EU and USA. Citing gene therapy approaches to retinal disorders, the Nature opinion seeks to encourage specialist researchers in fields such as genetics, stem cell biology and others, to assist regulators assess and evaluate new therapies that rapidly progress from academic research to clinical application. Recent examples include Spark Therapeutics’ (NASDAQ:ONCE), announcement on the submission of a marketing application to the European Medicines Agency for Investigational “LUXTURNA” (voretigene neparvovec). The application arises from several years research involving academic, regulatory and industry experts to devise a therapy to treat RPE-65 related retinopathies.
In citing some of the challenges faced by regulators reviewing new drug applications, the UK journal’s editorial asks, “When a treatment is based on a person’s own cells, how can the manufacturer ensure that each dose is as safe and potent as the last? Or when a treatment involves a virus, how can a company ensure that it will be safe over the long term, will not develop the ability to replicate and will not, itself, cause cancer?” As the speed of research and translational medicine increases, the journal observes that regulatory agencies may not always have the expertise to answer such complex questions. Recent evaluation of a novel oncology gene therapeutic targeting leukemia is understood to have spent considerable time on not just the statistical and trial design aspects of evaluation, but also on the manufacturing and safety issues pertinent to entirely new mechanisms of delivery and quality control. Regulatory approval for such novel therapeutics is likely to increase in the coming decades as academic, clinical and industrial experts advance ever more sophisticated treatments to market.
In acknowledgment of the challenges, the head of the FDA in the US, Scott Gottlieb, has supported an agency initiative to more actively compete for the limited pool of talent with expertise in new and specialized technologies. In contrast, in the EU’s EMA, it is likely that similar challenges exist however, priority at present will need to be focused on the challenges of Brexit. A number of media reports suggest the EU agency is currently working diligently on the migration of the regulator to the new EU location in Amsterdam. The agency has commented that it has developed and initiated a business continuity plan to deal with the uncertainty and workload implications of Brexit, and on minimizing the impact of the agency’s departure of the UK on both industry and patients. It will be equally critical for the EMA to maintain and recruit specialized expert researchers in order to ensure EU patients benefit from access to next generation therapeutics.