AUTHORS: Amy Boyers, Solen Monteil, Mariam Bibi
OBJECTIVES
Early Access Programmes (EAPs) provide a critical pathway for patients to access innovative medicines before full regulatory approval. However, manufacturers can struggle to identify EAPs and understand the opportunities that would best complement their pricing and reimbursement strategies. We have reviewed EAPs across four Nordic countries (Denmark, Sweden, Norway and Finland) to establish the eligibility criteria, initiation processes and funding opportunities and ultimately assess the opportunities for manufacturers of orphan drugs and implications for patients.
METHODS
Early access programmes available in Denmark, Sweden, Norway and Finland were identified through secondary research. Eligibility criteria, initiation processes, evidence-generation possibilities and funding opportunities were extracted for each EAP. The suitability of each EAP for manufacturers of orphan drugs and implications for patients was evaluated.
RESULTS
Eight EAPs were identified across the four countries; Finland had three, Sweden and Norway had two and Denmark had one. Six EAPs were physician-initiated and managed. Only the two compassionate use programmes in Sweden and Norway require therapies to treat chronic or seriously debilitating illnesses or life-threatening illnesses that cannot be satisfactorily treated with an authorised medicine. However, neither EAP offers paid early access to manufacturers. Of the remaining EAPs, two had no predefined inclusion criteria and four required no other authorised medicine to be available. Finland, Sweden, and Denmark have EAPs that offer paid early access for manufacturers, but Norway does not.
CONCLUSION
There are multiple opportunities for EAPs across the four Nordic countries. The majority are physician-led, highlighting the importance of strong relationships between manufacturers and healthcare providers to secure early access. The compassionate use programmes with the most relevant criteria for orphan drugs are not funded, potentially limiting their use. Furthermore, the lack of EAPs that offer paid early access in Norway could significantly limit the number of manufacturers able to provide patients with early access to potentially life-saving medicines.
Read our full research below (available post-conference).
Plus, read our other research abstracts for WODC Europe 2024 here.