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Promotion of Progressive Patient Access bears fruit

June 2014

Children starting a race

EURORDIS is promoting Progressive Patient Access to speed up access to more safe, effective and affordable innovative therapies with real therapeutic added-value for unmet medical needs of rare disease patients in the European Union. Progressive Patient Access, also known as Adaptive Licensing or Adaptive Authorisation, has the potential to allow faster access via a staggered approval scheme that balances access with safety evidence. EURORDIS has been promoting the concept of Progressive Patient Access since 2012.

How does it work?

By working with the regulatory processes within the current legal framework in Europe, Progressive Patient Access would foster faster access to innovative therapies by allowing an earlier authorisation date under certain predefined conditions while continuing the process of gathering safety & efficacy data – in particular when combining the scheme of Conditional Marketing Approval with new tools within the new EU Pharmacovigilance Regulation and stringent Post-Approval Safety Studies and Post-Approval Efficacy Studies. Through progressive enlargement of the target population being treated, which would be determined by specific inclusion criteria, Adaptive Licensing would allow treatments to reach patients faster – particularly for unmet medical needs, i.e., patients with severe diseases who have no satisfactory alternative therapies available. Progressive Patient Access thus allows patients quicker access to life-saving treatments while simultaneously improving the collection of evidence in real life situations across the very heterogeneous targeted patient population of each rare disease.

The role of EURORDIS

Since 2012, EURORDIS has steadily advocated for Progressive Patient Access that could be applied within the context of the current EU regulatory framework. EURORDIS supported and participated in the different reflection processes and communications of the European Medicines Agency (EMA). The EMA evoked the possibility of staggered approval schemes in their Roadmap to 2015 publication. As the concept became mature, a EURORDIS Round Table of Companies (ERTC) was devoted exclusively to the topic in October 2013, gathering some 80 high-level stakeholders to discuss the advantages and challenges of Progressive Patient Access. Sustained interaction between EURORDIS and the EMA, the European Commission, and Health Technology Assessment (HTA) bodies, culminated in a letter that was co-signed by the European Patient Forum, the European Federation of Pharmaceutical Industries and Associations (EFPIA) and the European Association for Bioindustries (EuropaBio) and sent to the European Commission in December 2013 calling for Adaptive Licensing pilots. The European Commission was supportive and, in response, the EMA announced in March 2014 the launch of a pilot project on Adaptive Licensing and issued an invitation to clinical trial sponsors to submit on-going medicines development programmes for consideration.

Dialogue is crucial

To ensure the success of new models that improve access to orphan medicines while allowing more complete and thorough data generation, dialogue is essential between regulators, payers, industry, medical experts, and patient representatives. This dialogue needs to take place very early on to ensure that clinical trials are designed to facilitate progressive patient access by including early demonstrations of therapeutic value as well as anticipating data generation plans for post-marketing health technology assessment. The EMA pilot project calls for a “safe harbour environment” to facilitate “free exploration of the strengths and weaknesses of all options for development, assessment, licensing, reimbursement, monitoring, and utilisation pathways in a confidential manner and without commitment from either side“.

In early April 2014, a multi-stakeholder workshop, involving the EMA, FDA, the Massachusetts Institute of Technology (MIT) NEWDIGS (New Drug Development Paradigms) programme, the EFPIA, and EURORDIS, was organised at the EMA in London, to explore in a safe harbour environment, with all stakeholders, whether some concrete cases presented by industry would qualify for an adaptive licensing approach and what would be the benefits of such a progressive access pathway.

The recent European Conference on Rare Diseases and Orphan Products (ECRD 2014 Berlin) had a dedicated session on Progressive Patient Access Schemes and Patient Involvement in Benefit-Risk Assessment that allowed patients, regulators, industry and other stakeholders to share and compare perspectives. The presentations from this session are available online.

EURORDIS is committed to promoting the pilots and taking part in their implementation, in particular through the engagement of patient representatives in the risk/benefit assessment in order to bring to the fore of the regulatory assessors evaluation the perception of risks and of benefits from patients in need, while searching for optimal post-licensing evidence generation to reduce uncertainties.

EURORDIS will continue to create further opportunities for multi-stakeholder dialogue and to play an active role in shaping a favourable environment and defining innovative approaches that will improve treatment access for people living with a rare disease.

Louise Taylor, Communications and Development Writer, EURORDIS