The fact that the recently enacted 21st Century Cures Act includes flexibility for inclusion of real-world evidence into the drug development process has stirred up controversy, but it might allay the concerns of some patient safety groups to know that the European regulatory authority is implementing a similar process.
Adaptive pathways, as the European Medicines Agency describes it, “is a scientific concept for medicine development and data generation which allows for early and progressive patient access to a medicine.” Unlike Cures, which raises the possibility that patient experience data will be included into assessment of a new drug, the EMA stresses that adaptive pathways approach is not a new regulatory route.
Similarly, it is intended only for medicines that promise to make a significant impact on meeting urgent public health needs. To generate evidence to confirm the benefit-risk balance of a tentative approval in small and tightly defined populations, real-world data is allowed to complement those from randomized controlled trials until more data are presented.
The same concerns against the Cures have also been voiced. Some question the reliability of real-world data and worry that EMA’s approach would impact standards of evidence for medicines approval in the EU in unexpected ways. At a recent workshop organized by the agency and the European Commission with stakeholders, it was widely agreed upon that the use of adaptive pathways be strictly limited, and that there be appropriate abort strategies when required data continue to be absent.
The agency launched in March 2014 a pilot project exploring the feasibility of the medicines development concept and finished with a final report at the end of this July. After examining 18 selected cases, the agency recognized that adaptive pathways can support drug development in areas where evidence generation is challenging, such as infectious diseases, Alzheimer’s and rare cancers, while also identified that it needs methodologically-sound strategies of real-world evidence in supporting assessment of new drugs.
During the workshop, according to a release by the EMA, the agency emphasized collaboration among pharma companies, regulators, health technology assessment bodies and healthcare professionals in managing the uncertainty in the evaluation of the benefits and risks of medicines. “Collaboration is key,” said Andrzej Rys of the EC’s Directorate-General for Health and Food Safety. “We all want to make sure that patients in the EU get access to safe, effective and affordable medicines.”
The workshop also discussed other important questions arising from the pilot program, including how to generate appropriate data, and how to maintain high standards for approval. The European regulatory authorities pledge to provide updates regularly and continue consultation with all stakeholders.