The Bioindustry Association’s (BIA) Rare Disease Industry Group has launched a report A Rare Chance for Reform in collaboration with PwC which calls for action to ensure that patients are able to access innovative medicines for rare diseases.
The report identifies several key areas where reform would accelerate patient access to rare disease medicines, including:
- Address systemic issues to build a strong environment for access to orphan and ultra-orphan medicines,
- Accelerate access through a conditional access period,
- Increase sustainability in funding arrangements for orphan and ultra-orphan medicines,
- Evaluate orphan medicines and ultra-orphan medicines through a single rare disease pathway, and
- Adopt a single process to ensure that all orphan and ultra-orphan medicines are assessed by a process that accounts for their unique challenges.
The report references MAP BioPharma’s 2019 report Access to Orphan Medicines: A Case for Change which finds that the assessment route for orphan medicines has a significant impact and that the NICE Single Technology Appraisal does not deliver equal access for patients, with routine availability often delayed due to inflexible, inappropriate assessment processes.