[co-author: Elinor Pecsteen]

On 8 December 2016, the European Medicines Agency (“EMA”) organised a workshop in collaboration with the European Commission to gather the views and proposals from stakeholders on the adaptive pathways approach.

The aim of the workshop was to gather stakeholder feedback in light of the practical experience gained during the adaptive pathways pilot project EMA ran between March 2014 and August 2016. In addition, the EMA sought to plan the next steps in the exploration of the concept of adaptive pathways.

The workshop was attended by representatives from patients’ and healthcare professionals’ organisations, pharmaceutical companies, Health Technology Assessment (“HTA”) bodies, National Competent Authorities (“NCAs”), the European Ombudsman, the European Commission and staff from EMA.

Background

The adaptive pathways is an approach to development and data generation related to medicinal products which aims to facilitate access to medicinal products expected to have a significant clinical impact in patient populations with high unmet needs.

The adaptive pathways approach is a regulatory route that intends to make use of existing legislative and regulatory tools in a more efficient way. The concept involves a stepwise approach, permitting limited approval of medicinal products in small, tightly defined populations until more data are available. It also makes more use of real world data and randomized controlled clinical trials where these trials are not adequate in themselves.

In July 2016, the EMA published a report concerning the pilot project that was conducted between March 2014 and August 2016. EMA also issued guidance for companies that wish to use the adaptive pathways approach.

The EMA issued a report on 13 December 2016 concerning the EMA workshop on adaptive pathways (the “workshop report”). In this report, EMA recognizes that while stakeholders acknowledge the potential value of adaptive pathways for patients, there have also been concerns about the possible impact the adaptive pathways approach has on standards of evidence for approval of medicinal products in the European Union (“EU”). The report highlights the importance of early involvement of stakeholders and of development of a methodology for using real-world data.

The challenge of using real world data

A main point of discussion at the workshop held on 8 December 2016 concerned the use and reliability of real world data in evaluating treatment effects.

Real world data, including observational data, are data collected outside the controlled environments of randomized clinical trials. Real world data are often collected for purposes other than evaluating a particular product (e.g. electronic health records).

The workshop report provides that stakeholders and EMA discussed at the workshop how real world data can best support clinical trial data in cases when further information is necessary and cannot be obtained from clinical trials, when the trials themselves are difficult to run or when they cannot fully answer the questions raised. In its report, EMA commits itself to being transparent about the results to build further trust in these techniques.

The role of HTA bodies

HTAs bodies are regional or national bodies that provide post-authorisation recommendations concerning the financing or reimbursement of medicinal products and other health technologies by the healthcare system in an individual EU Member State or region.

EMA highlighted in its workshop report that the experience to date with conditional marketing authorisations granted on the basis of the adaptive pathways approach shows that HTA bodies need to be involved early in development. Early involvement would ensure that more medicinal products meet HTA criteria and are made available to patients in a timely fashion. EMA noted that all stakeholders involved in the adaptive pathways pilot project stressed the value of establishing a “safe harbor” dialogue in which participants can freely raise concerns at the start, in shaping the development process.

Managing uncertainty

In the adaptive pathways approach, the objective of finding treatments for patients with high unmet needs must be balanced against available relevant evidence and the possibility of obtaining further data after approval in cases where full data are not yet available.

In the workshop report, EMA set out its aim to reduce the inherent uncertainty over the product life-cycle in the adaptive pathways approach by planning better from the beginning. Better planning and appropriate study design will allow medicinal products to be provided faster and more efficiently to a suitably restricted patient group. EMA concludes in its report that early collaboration between companies, regulators and HTAs is key to achieve these objectives.

For further information visit: http://www.ema.europa.eu/docs/en_GB/document_library/Press_release/2016/12/WC500217951.pdf