Over the past few decades, there has been growing demand for early access to medicines that treat serious or life threatening diseases, for which there are minimal alternative treatment options.
Four key drivers for early access:
- regulatory and reimbursement delays resulting in wide variation in access timelines between different countries
- increased collaboration with academic groups
- patient pressures, with advocacy groups gaining increased influence within regulatory and Health Technology Assessment (HTA) processes
- emerging health threats.
Based on these pressures, some countries have introduced new options and/or processes to speed up access to medicines.
- This includes access before marketing authorization (MA), speeding up the MA, or less often, the HTA processes
- The approach of each country differs, leaving patients, clinicians, and companies navigating a myriad set of rules and processes
- All approaches are focused on access for serious and/or life threatening diseases where significant unmet clinical need remains
- Approaches differ in whether companies are required to provide therapies free of charge, or if the government provides reimbursement during the early access period
- Many newer initiatives emphasize early dialogue with multiple stakeholders and an expanded toolbox for evidence generation, including real world studies
