The European Medicines Agency (“EMA”) has published a new guidance document for companies that wish to participate in the Adaptive Pathways initiative (“New Guidance”).
Adaptive Pathways was established as part of the EMA’s objective to improve timely access for patients to new medicinal products. Adaptive Pathways applies to medicinal products with the potential to treat serious conditions with an unmet medical need. The concept of Adaptive Pathways is to grant applicants that develop such medicinal products either:
- an initial marketing authorisation with a therapeutic indication in a well-defined patient subgroup. Identification of the initial therapeutic indication could be based on surrogate endpoints. This initial therapeutic indication could be subsequently extended to include a larger patient population based on gradual phases of post-marketing authorisation evidence-gathering, including real world data; or
- an early conditional marketing authorisation.
In March 2014, the EMA invited companies developing new medicinal products to participate in a pilot project for the Adaptive Pathways approach (“Pilot Program”). The Pilot Program will be implemented in two stages:
- Phase I of the Pilot Program focused on receiving applications from interested companies that are at the early stage of developing investigational medicinal products.
- Phase II of the Pilot Project will include in-depth, face-to-face meetings with the selected applicants.
Phase I for receiving applications closed on 28 February 2015. The EMA will, however, still consider new applications to take part in Stage II of the Pilot Program after February 2015 if such applications are well-developed. The New Guidance provides information to applicants that wish to submit a new application to take part in Stage II of the Pilot Program I.
According to the New Guidance, applicants must demonstrate that their product development plan meets three criteria before being considered for the Adaptive Pathways Pilot Program.
- Applicants must plan on either pursuing a staggered marketing authorisation or conditional marketing authorization route
Staggered marketing authorisation involves extending the therapeutic indication of a marketing authorisation from the initial restricted patient population in which the benefit outweighs the risk to increasingly wider populations. According to the Guidance Document, most companies did not specify a proposed authorization route while a number of applications included both a staggered and conditional marketing authorization route.
- Applicants must gather evidence through real-life data to supplement clinical trial data
This data could include information gathered from disease registries, natural history studies, compassionate use data and/or post-authorisation studies.
- Applicants must involve patients and health technology assessment (“HTA”) bodies in the discussions concerning the product development design.
According to the Guidance Document, the successful applicants sought patient and HTA advice on specific issues before submission of the request to be considered for Phase II of the Pilot Program. For instance, some applicants sought advice on whether proposed endpoints and post-approval studies were acceptable.
Applicants are not required to submit an application form to participate to the Pilot Program. Interested applicants are invited to email the EMA at the following address: firstname.lastname@example.org to obtain advice concerning the content and suitability of their request to be considered for Phase II of the Pilot Program.
Once an applicant submits their application for inclusion as part of the Pilot Program, the application will be considered by the Adaptive Licensing Discussion Group (“ALDG”). If a product is selected for inclusion in the Pilot Program, the applicant and the ALDG will assess the possibility to adopt an Adaptive Pathway in relation to the proposed development of the product.
Following this assessment, the applicant will receive a list of comments similar to those received following a pre-submission scientific advice procedure. The applicant can chose to revise the clinical development plans for the product on the basis of the feedback received. If required, further confidential discussions between the applicant and the relevant stakeholders can take place before taking any further steps.