A review of the challenges and proposals for improving patient access to advanced therapeutic medicinal products in Denmark

Executive Summary

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.

In recognition of the pipeline of ATMPs and the difficulty in providing patient access to these innovative therapies in a sustainable way with existing pathways, Denmark has begun to implement changes in the assessment system for ATMPs

Within the existing assessment frameworks there are evidence criteria challenges for ATMPs that are very similar to the challenges faced by orphan drugs; the evidence itself often contains many uncertainties, and the long-term clinical benefit is regularly uncertain. Although there are clearly outlined assessment requirements that ATMPs must meet, it may be difficult for them to fulfil these requirements.

Recognition that the standard assessment process does not fit well for ATMPs, along with a willingness to accept different methods/evidence levels that can more realistically be fulfilled, may contribute to making the process more conducive to the nature of these innovative products. It appears that this willingness is present in Denmark, as changes to the assessment process are planned for 2020

It is expected that these changes will likely address many of the existing assessment challenges which have resulted in challenging patient access for existing ATMPs.

While affordability is not an issue in Denmark, an emphasis on a need for socioeconomic responsibility places high scrutiny on high-cost medicines. Affordability issues specific to ATMPs arise when individual hospitals are responsible for delivering specialist treatment; specialist prescribed products are funded by hospitals, and the use of such products is likely to be restricted to a single hospital. The potential availability of specialist funds for ATMPs is uncertain, which leaves hospitals and health insurers to cover treatment costs. In addition to product costs, the logistical demands associated with ATMP delivery add further costs and budget impact. Solutions such as innovative payment plans, early access programmes and better security for specialist funds may help alleviate some of these challenges.

Denmark has a network of specialist centres that are capable of treating patients with ATMPs. There is, however, a burden of reconfiguring services for these treatments that will be felt by a small number of centres. Denmark is well-placed to configure its services to allow access to ATMPs. Having built-in service preparation requirements in a joint horizon scanning process at the Nordic level and identifying infrastructure and services that can be reconfigured for ATMPs, are two approaches to help ensure success and access for patients across regions.

Domain (Impact)^*	Challenge	Proposed Solution	Feasibility^**
Assessment
4	The Danish Medicines Council (DMC) is willing to accept surrogate outcomes, but evidence is graded lower than conventional endpoints	Key changes to assessment in Denmark are planned for the near future to address ATMP assessment	+++
4	DMC evidence requirements are clearly outlined, but a product achieves added clinical value only if a minimal clinically important difference is demonstrated	Adjust evidence requirements and include patient perspectives when defining minimal clinically important differences for ATMPs - this may be taken into account in the upcoming changes	++
4	Clarity is still needed on the assessment changes in terms of how they will be implemented and what will actually change	Clarity should be requested on anticipated amendment and the implications of not changing should be communicated, to ensure changes are successfully made	+++
Affordability
4	Affordability issues arise when individual hospitals are responsible for delivering specialist treatment	Innovative payment models could alleviate the burden of up-front payments A secure specialist fund for ATMPs to reduce burden on individual hospitals	+
4	Higher logistical demands associated with ATMP administration: patients require additional outpatient appointments which adds costs and affects budget impact	Collect data from early access programme to determine what should be included in the model for assessment	++
Availability
1	The outlook of physicians in Denmark emphasises a need for socioeconomic responsibility beyond advice from AMGROS	Early communication with physicians around benefit of ATMPs	+++
1	Patients have received access to treatment via cross-border initiatives – it is uncertain if patients will receive the same level of cross-border access to ATMPs	Denmark is involved in cross-border agreements which are not a challenge; but rationale for cross-border treatment will be required	+++
Accessibility
2	Denmark has a network of specialist centres that are capable of treating patients with ATMPs; however, there will be a burden of reconfiguring services for these treatments	Configure services for ATMPs by identifying infrastructure/services that can be reconfigured, and having built-in service preparation requirements in a joint horizon scanning process	++

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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