Access to orphan drugs in Norway

Introduction and theory: The US and EU developed special legislation to promote the development of drugs for patients with rare diseases (orphan drugs). Rare diseases are defined as <5 patients out of 10,000 inhabitants. The priority criteria consist of; health-benefit, resource, and health loss. With the System for Managed Introduction of New Methods in Norway operationalising the priority criteria, there has been increasing debate on orphan drugs and New Methods extensive focus on price. With new pharmaceuticals required to undergo Health Technology Assessment, decision-makers expect orphan drugs to satisfy their willingness-to-pay threshold. However, there are no policies for the rapid implementation of orphan drugs in Norway. Methods: Quantitative methods were applied; we developed descriptive statistics and used the Mann-Whitney U-test, a two-sample test of proportion and logistic regression analysis. Results: 67 of 132 orphan drugs are accessible in Norway. Adoption of orphan drugs takes more than two years on average from Market Authorisation is granted by EMA. There is a significant disease burden for rare diseases in Norway; the burden is more significant for non-cancer patients than cancer patients. Patients with rare diseases could benefit significantly from the pharmaceuticals considered by New Methods, with a mean gain of 2 QALYs. The odds of reimbursement increase with the increasing disease burden, while increasing costs decrease the odds. The number of positive decisions on orphan drugs has decreased following the White paper on priority setting. NoMA and manufacturers evaluate the effect of new orphan drugs significantly different. Discussion: Patients with rare diseases in Norway are heavily burdened by their condition. The long period between a medication receiving marketing authorisation and a decision adds to the patient's burden and counteracts EU Regulations designed to promote rapid access to orphan drugs. Many orphan drugs are for cancer conditions; the current legislation might facilitate a lucrative drug area such as cancer. Several orphan drugs would qualify for New Methods' higher willingness to pay, except that they are too many patients. And New Methods might put too much weight on the priority criteria, neglecting other important factors. Conclusion: We recommend looking to other countries to improve the reimbursement process in Norway to rapidly adopt new pharmaceuticals and increase the legitimacy of the process. Observing the debate on rare cancer drugs within orphan drugs, we recommend that EU design two separate Marketing Authorisation tracks to provide them with appropriate incentives, respectively