Access to orphan drugs in Sweden has become a contentious issue as political pressure intensifies.

Anders W. Jonsson, a pediatrician and the health spokesperson for the Centre Party (Renew), has publicly challenged Acko Ankarberg Johansson, the Swedish health minister and a member of the European People's Party (EPP), to address the accessibility of orphan drugs in Parliament.

Jonsson argues that while Sweden's pricing and reimbursement model for medicines functions adequately for common conditions, it fails to support patients requiring medications for rare diseases, mainly because these conditions affect small patient groups which leads to higher drug costs.

Jonsson's concerns echo sentiments among various opposition parties, including the Social Democrats, the Left Party, and the Green Party, with even the Liberal Party, part of the ruling coalition, expressing support for special funding to aid regional drug access.

The debate has gained urgency, particularly following decisions made by the Swedish Dental and Pharmaceutical Benefits Agency (TLV).

In 2023, the TLV opted not to reimburse Voxzogo (vosoritide), the first EU-approved treatment for children with achondroplasia, citing its cost relative to the modest increases in height it provides.

Achondroplasia, a genetic condition resulting from a mutation in the FGFR3 gene, affects bone growth and results in short stature.

Approximately five children are born with this condition annually in Sweden, translating to an estimated fifty children who could potentially benefit from this medication.

Responses to the TLV's decision from health advocates underline the psychological and physical importance of every centimeter of growth for individuals with short stature.

Jens Händel Hall, representing the Association for people with short stature, has noted that Voxzogo is standard treatment in several EU countries, contrasting with its unavailability in Sweden.

Recent studies have suggested significant benefits from the drug, showing an increase in height and better body proportions in treated children compared to untreated groups.

The TLV has acknowledged that while availability of orphan drugs has improved, the existing pricing model requires further refinement.

A new model was introduced in early 2023 to allow for higher pricing for orphan drugs prescribed to fewer than 100 patients.

Nevertheless, patient associations argue that the adjustments are insufficient for extending necessary treatments to those in need.

Health Minister Acko Ankarberg Johansson indicated that she would consider the impact of the revised pricing model before committing to additional funding.

She also announced a forthcoming inquiry aimed at developing a new pharmaceutical pricing and reimbursement strategy to enhance access for patients with rare diseases.

Critics, including Jonsson, express concern that such inquiries could prolong access to necessary medications for patients while negotiations continue.

The performance of Sweden in the provision of orphan drugs has drawn scrutiny.

A recent report from the European Federation of Pharmaceutical Industries and Associations (EFPIA) highlighted that of the 66 new orphan drugs approved by the European Medicines Agency from 2020 to 2023, only 19 were accessible to patients in Sweden, comparatively lower than the EU average of 28. Regional health authorities contest these findings, citing discrepancies such as underreporting and regional discretion in reimbursement decisions as contributing factors to Sweden's performance.

In a relatively positive assessment from a TLV survey covering the years 2015 to 2022, around two-thirds of orphan drugs reviewed received favorable reimbursement decisions.

However, the lack of formal data regarding the uptake of new orphan drugs remains a significant gap in assessing overall accessibility.

As Sweden is set to introduce its first national strategy for rare diseases, it remains unclear how this initiative will address the pharmacological necessities of patients suffering from these conditions.