Novel immunotherapeutic drugs for rare rheumatic diseases
In 2016 ReumaNederland and health care professionals launched the Arthritis Research and Collaboration Hub (www.arch.nl), a national expertplatform to improve outcomes of patients with rare systemic autoimmune diseases (also referred to as IMID, for immune-mediated inflammatory disease), by 1. streamlining referral pathways, 2. sharing medical expertise via multidisciplinary digital consultations, 3. upgrading and aligning national disease registries and 4. facilitating patient access to new novel immunotherapeutics.
Whilst huge progress has been made with achieving the first three aims, helping patients and doctors give access to novel drugs constitutes a challenge. Clinical trials in rare diseases are notoriously difficult and expensive to conduct, hence only very few novel immunotherapeutic drugs are being developed for rare systemic autoimmune diseases. Disease mechanisms in rare systemic autoimmune diseases do not differ fundamentally from those operative in common IMIDs such as rheumatoid arthritis, psoriasis and inflammatory bowel disease, and hence novel immunosuppressive drugs can be expected to be effective in rare IMIDs as well. Unfortunately, most novel drugs are not licensed for use outside their indication and can only be prescribed to individual patients off-label if approved by a patient’s insurer. Drug options for patients with rare IMIDs therefore remain limited, which has a negative impact on outcomes in those patients not responding to traditional immunosuppressants.
The DRIMID consortium (Drug Rediscovery in IMIDs) aims to conduct a 5-year prospective pilot basket trial for patients with selected rare IMIDs who have exhausted standard treatment options and are offered off-label treatment with filgotinib, a new JAK-inhibitor licensed for use in arthritis. Data collected from this project may pave the way for reimbursement of filgotinib and development of other potentially effective drugs in selected rare IMIDs.