The status of national strategies and a common Nordic roadmap.

Rare disease companies struggle with a range of challenges, one of which is how to balance funding R&D with a small market size. Healthcare systems and societies also face a balancing act when it comes to rare diseases – between innovation and accessibility. Having a national strategy for rare diseases in place could help set ambitions and unite stakeholders, but implementation, including financial support and political willingness, varies within the Nordic countries.

To ensure a truly impactful national strategy people living with rare diseases should be included in all processes of strategy development so their expertise is harnessed, both face-to-face and with the support of data.”

“As it stands, Sweden and Iceland are working on developing their first national strategy while Finland launched an updated strategy in January. Norway and Denmark have their strategies in place, but that doesn’t mean that the work stops there. A national strategy should continuously be updated to reflect reality. And to ensure a truly impactful national strategy people living with rare diseases should be included in all processes of strategy development so their expertise is harnessed, both face-to-face and with the support of data,” says Morten Frank Pedersen, Nordic Head of Public Policy, Takeda, one of the partnering companies around the 2nd Nordic Rare Disease Summit that took place in Stockholm in April 2023.

The importance of involving patient representatives in policy making became evident when the national strategy for rare diseases was developed, where a key factor was collaboration with the Danish national patient organization. This collaboration resulted in strategic points highlighted by the organization forming part of the final national strategy.

“Ever since 2014, when the first national strategy was developed, Rare Disease Denmark has been a recognized collaboration partner for the health authorities. Early in 2023, the Danish strategy was evaluated and as stated in the strategy, the patient organization was represented in the working group. This enabled a true, cross-stakeholder understanding of the strategy’s effects and relevance for patients,” says Frank Pedersen.

 

Morten Frank Pedersen, Nordic Head of Public Policy, Takeda.

 

An updated Nordic roadmap

Strengthened Nordic collaboration within the rare diseases area has led to the development of an updated common Nordic roadmap on rare diseases, which was requested for during the 2nd Nordic Rare Disease Summit. The updated roadmap will be published in the first half of 2024 and will provide measurable recommendations in the key action areas Early Diagnosis, Patient Empowerment, Access to Innovation, and Impactful Nordic strategies. This will be a forward-looking roadmap, taking into account all dialogues, and agreed upon by all ten of the participating partners of the Nordic Rare Disease Summit.

“Denmark, Finland, Iceland, Norway, and Sweden all have an ecosystem in place, which should be used to take their place as front-runners in rare disease care. We have public registers, biobanks, genome centers, vast amounts of health data, and comparable healthcare systems that can develop and support healthcare, research, innovation, and policy making,” says Helena Bladh, Public Policy Lead, Takeda Sweden.

The roadmap is a shared call to action containing four critical aspects: empowering people living with rare diseases, early diagnosis, patients’ access to innovative medicines, and the development of impactful Nordic rare disease strategies.”

“Nordic policy makers share similar aspirations, visions, and an understanding that strong commitments are needed to drive change. The roadmap is a shared call to action containing four critical aspects: empowering people living with rare diseases, early diagnosis, patients’ access to innovative medicines, and the development of impactful Nordic rare disease strategies. This set of recommendations and proposed measurable outcomes will hopefully enhance accountability and enable monitoring and tracking of progress with the purpose of improving treatment, diagnoses, and the lives of people living with rare diseases,” she says.

The roadmap also recommends that national strategies include a transparent governance model including realistic timelines marked by clear, measurable, and time-bound goals secured through adequate funding and resources.

“With national strategies and cross-country cooperation supported by the necessary political and economic ambition, there can be lasting progress on rare diseases. This would empower people living with rare diseases as well as enable a systematic shift in how to better support these individuals,” says Bladh.

 

The 10 partners behind the Nordic Rare Disease Summit 2023 and the new Nordic Roadmap for Rare Diseases are Takeda, Novo Nordisk, Sobi, Alexion AstraZeneca Rare Diseases, Rare Diseases Sweden, SBONN (The Nordic Network of Patient Associations in Rare Diseases), EURORDIS, EFPIA, EUCOPE and Lif Sweden. Photo: Teodor Axlund

 

Nordic roadmap – 4 key action areas

Early diagnosis: On average it takes 6-8 years before a person with a rare disease receives the correct diagnosis, and approximately 40% of all patients with a rare disease are initially misdiagnosed.

Patient empowerment: Patients with rare diseases often have more expertise about their diagnosis than the healthcare representatives they interact with.

Access to innovation: The access deadlock needs to be broken by recognizing the value of treatment in a holistic way and paying for innovation that adds true value for patients while securing sustainable healthcare systems.

Impactful Nordic strategies: A national rare disease strategy is important to raise awareness of rare diseases, ensure better funding for national patient associations and rare disease treatments, as well as secure a higher medical, political, and financial ambition.

Featured photo of Helena Bladh, Public Policy Lead, Takeda: Lukas Lennse