As more pharmaceutical companies develop and launch drugs to treat rare diseases, reimbursement decisions indicate that payers are looking at a range of issues, including disease prevalence, with new levels of scrutiny and that their willingness to accept high prices may be reaching its limits. There are signs that they are less willing to overlook the limitations on clinical data often associated with these drugs, negotiating rebates and setting budget limit thresholds. Payers across the globe continue to face budget challenges that new rare disease therapies might present while assessing cost against the overall goal in healthcare: to provide access to optimal treatment options to as many patients as possible. For rare disease drugs, payers must consider a range of factors including clinical benefit, high prices, disease prevalence, unmet need and often limited efficacy and safety data in their reimbursement decision-making.
In European Pharmaceutical Review, we assess the relationship between drug pricing for rare disease therapies and disease prevalence in Europe, taking a closer look at payer assessments of orphan drugs in Germany.