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

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.

Access to ATMPs in Sweden has been challenging to date

Agreement on Yescarta required a rebate to regional budget holders, Kymriah was not recommended for one of its two indications, while Luxturna and Alofisel are not recommended by the New Therapy (NT) Council based on the health economic assessment of the products. In order to secure sustainable access, challenges in the assessment process for ATMPs should be addressed as a priority.

Flexibility in the assessment process for ATMPs is needed without compromising the integrity and rigour of the current assessment process

As with other HTA/cost-effectiveness analysis (CEA) markets, the major challenge for ATMPs in Sweden is the quality of evidence, the ability to extrapolate from short-term, surrogate endpoints to outcomes, indirect comparison, and the cost of comparator treatments. The Tandvårds-läkemedelsverket (TLV) conducts value-based pricing assessments, which in theory could be helpful to potentially curative treatments for rare diseases. There is some flexibility in the TLV ICER threshold, which reflects the three principles of the assessment process; human value, needs & solidarity and cost-effectiveness. The willingness to pay can therefore differ on a case-by-case basis based on the first two principles, and higher ICER thresholds can be provided for treatments for rare diseases with high unmet need. Nonetheless, even with this additional flexibility, TLV’s ICER thresholds are often insufficient for orphan medicines and such drugs have been rejected by the TLV in the past. After the Cerezyme & Vpriv assessments, the TLV concluded that under certain circumstances they can find it appropriate to allow the rarity of a disease to motivate a higher cost in comparison to the benefit the treatment gives. Taking other factors into account, the TLV concluded it might then be reasonable to accept a cost of up to SEK2M (€180,000) per QALY.

The assessment route is also evolving in Sweden. TLV does not always assess inpatient-only drugs; for these treatments, funding decisions may be taken by individual regions or on recommendation from the NT Council. To date, reimbursement decisions for ATMPs have been taken for individual regions by the NT Council. If an orphan product is not recommended by the NT council, the regions cannot subsequently fund it.

As funding decisions in Sweden can be influenced by medical need there may be a willingness to pay for treatments for conditions that are very rare and/or have severe unmet need. Budgets are held by regions and the high-cost ATMPs’ patient populations could be unequally distributed across the country, representing a funding challenge for individual regions (particularly smaller ones). However, exemptions are made between regions whereby if some conditions are more prevalent in some areas, costs can be shared, and this could potentially be a model that could be applied for ATMPs for rare conditions in the future.

Tripartite negotiations are in some cases possible between the TLV, NT Council and manufacturers, which provides an opportunity for dialogue on more innovative contractual arrangements. In general, there has been little experience with outcomes-based contracting in Sweden nor with staggered payments. A new programme to manage the introduction of new therapies has been developed which will enhance monitoring of new medicines and collect data that can impact cost-effectiveness and possibly form part of outcomes-based contracting. Further, a recent government funded report reviewing the current financing, subsidy and pricing systems for pharmaceuticals has made recommendations for funding ATMPs. The report proposes a special state contribution to regions to support the use of certain new pharmaceutical areas, such as ATMPs. The government are currently reviewing stakeholder comments before any processes are put in place for this funding route.

When products are not funded nationally or in a home region in Sweden, patients have previously received access to treatment via cross-border initiatives (E112) and in other local regions. While positive for these patients, the sustainability of making products available on case-by-case basis is challenging. A national level initiative would facilitate patients’ access to treatments in accredited centres in other regions, eliminate economic burden of unequally distributed patient populations across the country and remove potential disincentives of referrals outside of the region.

Accessibility may present greater difficulties, particularly in the northern parts of Sweden, as ensuring proximity to a treatment centre could be challenging, given the size of the country. The Swelife-ATMP initiative aims to strengthen Sweden’s preparedness for ATMPs, which could be leveraged to provide a solution to cross-regional patient transfer when expertise is concentrated in few treatment centres.

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