A review of the challenges and proposals for improving patient access to advanced therapeutic medicinal products from a European Level perspective

Executive Summary

VIDEO FROM RARE IMPACT LAUNCH EVENT OF 23 NOVEMBER 2020

RARE IMPACT is a multi-stakeholder initiative working to improve patient access to advanced therapy medicinal products (ATMPs). This patient-focused initiative aims to assess challenges and propose actionable solutions to concerns regarding access to these transformative rare disease treatments in Europe. Through engagement with health technology assessment (HTA) agencies, regulatory bodies, payers, patient groups, clinicians, manufacturers and other experts across Europe, RARE IMPACT partners identified challenges and have proposed solutions for better access to ATMPs in Europe.

This report aims to stimulate multi-level stakeholder discussions on patient access to ATMPs and is not intended to capture all challenges to patient access to ATMPs. The RARE IMPACT initiative was launched at the European Conference on Rare Diseases and Orphan Products in 2018.

There are two levels of European challenge. Firstly, there are challenges that are common across all countries in Europe that largely require country-level policy change to overcome
(e.g., acceptance of data from ATMP trials with existing HTA assessment criteria). Secondly, there are challenges that exist at the European level that can be addressed largely through collaboration and guidance from European institutions that govern many aspects of the ATMP patient access journey. This report primarily focuses on the later level of challenge, with many of the common country-level challenges addressed in individual country reports.

The report addresses challenges across four identified areas in the accessibility, assessment, availability and affordability of gene and cell therapies across the European Union. It highlights seven solutions including calls for:

Greater collaboration between EMA, HTA bodies and Heads of Medicines Agencies on guidance on HTA assessment of advanced therapies, as the complexity of these therapies as much as the new type of uncertainties require long term follow up;

A coordinated approach on the development and use of registries serving multiple purposes (e.g. the follow up of patients, assessment and reimbursement), as the limited set of data and today’s fragmented approach needs to be addressed;

Greater cooperation and clarity on use of the cross border healthcare provisions, as many advanced therapies are delivered in only a few highly specialised hospital centres across Europe and cannot be delivered in all countries;

More informative and technical discussions to grasp the specifics on advanced therapies’ cost and value; and

Payment based on outcomes and payment over time, though innovative payment mechanisms will be pre-empted by the removal of barriers.

The challenges and solutions contained within this document are those that have been proposed as priorities for discussion by members of the Working Group. The solutions proposed in this report are the views of the RARE IMPACT consortium and therefore not all solutions represent fully the views of each individual participant, EURORDIS or Dolon.

Please note, the feasibility assessment of proposed solutions considers all solutions implementable, depending on the level of stakeholder dialogue and collaboration. The assessment has marked these as N/A to avoid prioritisation as all solutions are deemed important for improving patient access across Europe.

Domain (Impact)^*	Challenge	Proposed Solution	Feasibility^**
Assessment
4	There is a gap between regulators and HTA bodies in their evidence requirements and addressing durability and long-term safety uncertainties	Collaboration between EMA, HMA and HTAs to issue guidance on ATMP assessment with focus on methodological standards to address remaining clinical uncertainty (incl. long-term evidence generation) building on existing initiatives to form a framework for assessment at the country level	N/A
4	Registry-type data required for post-approval regulatory requirements differs from that required by payers for managed entry agreements or other innovative payment models	Key stakeholders should collaborate to support the development and use of registries and real-world datasets	N/A
Affordability
4	There are barriers to annuity payments and innovative payment options across Member States	Work with stakeholders at the national level to remove barriers to annuity and innovative payment models	N/A
4	There is concern about the sustainability of prices for ATMPs and poor understanding of the role of price in promoting development of these therapies	Explain and discuss the ATMP development cycle, costs and incentives with all stakeholders	N/A
Availability
4	Variability in the use of cross-border healthcare legal provisions (Form S2) across Member States and the sustainability of this pathway is uncertain	Coordinate and issue formal guidance on cross-border pathways at the European level to facilitate access to different ATMPs	N/A
4	Hospital exemption legislation interpretation varies across Member States thereby creating inconsistent implementation	Seek clarification from EC on the exact intention of the hospital exemption, its implementation by Member States and introduce oversight by the Pharmacovigilance Risk Assessment Committee of the EMA	N/A
Accessibility
4	There is a need to treat patients in specialist centres which have variable certification requirements	Assess and educate around the existing system in place for treatment centre qualification. Encourage stakeholder dialogue to explore options to reduce administrative burden and ensure consistency across Member States	N/A

Notes
*The working group assessment of the relative impact of the challenge of each domain on patient access is represented by Harvey balls from highest (represented by a full blue Harvey ball) to lowest (represented by an empty, white Harvey ball); **Feasibility: Working Group assessment of feasibility of solutions to be implemented. + low feasibility, ++ medium feasibility, +++ high feasibility.

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