For many patients in the US, the passing of the 21st Century Cures Act was a great success. This new law will allow Real World Data (RWD) and patient experiences to be to be included in the drug development and approval process, meaning patients will have their voices heard, now more than ever, and will be able to take advantage of new medications quicker.
However, the US is not the first region to consider taking advantage of data from the everyday patient experience. In fact, the European regulatory authority has been developing a similar process, known as Adaptive Pathways, for several years.
Adaptive Pathways is a prospectively planned, iterative approach to bringing new treatments to market. It is based on three principles:
- Iterative development i.e. approval in stages
- Collecting and using evidence from real-life patient experiences to supplement clinical trial data
- Ensuring early involvement of both patients and health and technology assessment bodies in the discussions on a new drug’s development
Under this program, investigational new drugs are made available, initially, to a well-defined group, made up of those patients who are likely to benefit the most from a given treatment. To gain meaningful data from such a small and tightly defined population, RWD is gathered and used to complement the data from randomized controlled clinical trials. In this way, the patients and their day-to-day experiences are considered and are used to develop a broader understanding of the effectiveness of new drugs.
This initial stage of data collection is then followed by several more iterative phases of data gathering. Each of these phases introduces a more diverse and varied subset of the patient population, so that as more data becomes available, a wider patient population can gain access to the drug.
This approach was developed to support drug development in areas where evidence generation is challenging, like the rare disease space. As such, the Adaptive Pathways concept is not, at this stage, intended to be applicable to all medicines. Rather, it is only for those drugs that are being developed to treat a patient population with an unmet medical need.
Adaptive Pathways is not yet an official drug approval procedure. However, the pilot project, which ran from March, 2014, until August, 2016, had positive outcomes, which have been summarized in the Final Report on Adaptive Pathways. The concept will now continue to be fine-tuned as more investigational new drugs are considered for this approach.