Current regulations in drug development field assume complicated and lengthy multistage process, typically taking years if not decades. Often there are no effective treatment for orphan (rare) diseases, and many patients suffering from such conditions simply can’t last long enough to get effective treatment, especially if their lives depend on the drug under development. There are approximately 60 million people suffering from orphan diseases in the EU and the US, hence the total burden of orphan diseases is huge. Similarly, the approval process is suboptimal if a new drug is being developed for the treatment of serious diseases or for use in emergency situations. EMA grants conditional marketing authorization for such medications since 2006. In other words, the agency gives marketing authorization before all the required data is submitted by the manufacturer. Recently
EMA published a report on the 10-year experience of practicing such approach. At the beginning, this innovation seemed risky: 30 medicines received conditional marketing authorization since 2006 and for half of them phase III trials were not completed by the time of submission. It took an average of 4 years to collect and summarize the additional data needed and to convert a conditional marketing authorization into a full marketing authorization. Yet, no drug has been withdrawn from the market because of unsatisfactory safety profile in the course of the program. Thus, new approach accelerated access to absolutely indispensable, efficient and safe drugs for about 4 years. Conditional marketing authorization was granted to new drugs used in oncology, ophthalmology, neurology and infectious diseases.
US FDA practices a similar approach – administration grants accelerated approval for drugs used to treat serious and rare diseases since 1992. For accelerated registration, the efficacy of the drug should be proven at least through surrogate endpoints, i.e. any parameter that is a predictor of clinical efficacy but doesn’t represent clinical efficacy measure itself. This type of approval is given under the condition that clinical efficacy is proven later in phase IV trials.
Due to safety and benefits of conditional and accelerated registration for patients, EMA and FDA decided to collaborate on orphan products, harmonizing their efforts on the procedural level.