A Dolon Institute Report
Autoimmune diseases affect an estimated 5–10% of the global population, with prevalence continuing to rise, yet significant gaps remain in diagnosis, treatment and disease management. While conditions such as rheumatoid arthritis, multiple sclerosis and lupus are relatively well recognised, many rare and lesser-known autoimmune diseases remain underserved, leaving patients facing misdiagnosis, limited treatment options and substantial impacts on quality of life.
Supporting patients with underserved autoimmune diseases: challenges and opportunities for innovative medicines examines why these gaps persist and considers how policy and pharmaceutical innovation can help address unmet patient and public health needs. The paper places autoimmune disease within the broader context of how policy environments can either stimulate or constrain innovation, drawing on lessons from rare diseases and antibiotics to illustrate the importance of getting incentives and healthcare systems right.
The burden of autoimmune disease extends well beyond clinical symptoms. Fatigue, pain, cognitive difficulties and unpredictable flare-ups can significantly disrupt daily life and affect mental wellbeing, while the complexity of diseases involving multiple organ systems can make diagnosis and treatment particularly challenging. Families and caregivers can also face substantial ongoing demands, contributing to a wider socioeconomic burden on healthcare systems and society.
For patients with rarer autoimmune conditions, these challenges are compounded by limited disease understanding, heterogeneous presentations and insufficient research and development. Existing treatments may primarily focus on symptom management or broad immunosuppression, which can be ineffective for some patients and may carry significant risks with prolonged use. This creates an important opportunity for innovative therapies capable of addressing the underlying disease mechanisms rather than simply managing symptoms.
A particular focus is the growing development of multi-indication medicines, enabled by shared molecular pathways across autoimmune diseases. These therapies have the potential to expand treatment options and generate development efficiencies because their mechanisms of action and safety profiles may already be better understood. However, the paper highlights significant barriers across the medicine lifecycle – from regulatory review and HTA through to pricing, reimbursement and clinical uptake.
Pricing and reimbursement presents a particularly difficult challenge. A single price may fail to reflect the differing value of a medicine across indications, while subsequent indications can trigger price reductions that potentially weaken incentives to pursue additional development. In rare autoimmune diseases, small patient populations may also make it difficult to offset lower prices through increased volume. Limited data infrastructure for tracking medicine use by indication further restricts the feasibility of innovative pricing approaches.
The paper ultimately argues that supportive policy must address the entire innovation ecosystem – from R&D incentives and regulatory pathways to HTA, reimbursement and effective uptake. Lessons from successful orphan drug policies demonstrate that appropriately designed incentives can stimulate innovation, while the experience of antibiotics illustrates how an unfavourable economic environment can lead to development becoming unsustainable.
Read the report to explore the barriers facing underserved autoimmune disease patients, understand the policy and market dynamics shaping innovation, and consider how a more supportive environment could help translate scientific advances into meaningful improvements in diagnosis, treatment and patient outcomes.