Drugs known as “orphan” drugs are those used to treat rare disease. However, how “rare” is rare disease? In Europe, a rare disease is one affecting 5 or fewer people per 10,000 residents. In the U.S. this figure is less than one in every 1,500 residents. Although each rare disease is–by definition–rare, about 350 million people worldwide (about the size of the entire U.S. population) suffer form a rare disease. Because these diseases are rare, in most cases it would not be profitable for life sciences firms to invest R&D funds in the treatment of theses diseases. To incentivize innovation to treat these diseases, many countries have specific policies to encourage the development of these treatments.
A recent paper by Sarnola et al. (2018) reviews the policies of different European countries surrounding orphan drugs used in outpatient care. They interviewed government authorities within the and included the following countries: Albania, Austria, Belarus, Bulgaria, the Czech Republic, Estonia, Finland, Germany, Hungary, Iceland, Italy, Latvia, Lithuania, Malta, the Netherlands, Norway, Poland, Russia, Slovakia, Slovenia, Spain, Sweden, Turkey and the United Kingdom. Although the Europeans Medicines Agency determines whether a drug counts as an orphan status, access and reimbursement policies vary across countries and do not guarantee that the treatment will be fully covered.
Most of the countries had not implemented any special regulations or policies for assessing the reimbursement status (22/24) or pricing (20/24) of reimbursable orphan medicines used in outpatient care
Although a number of countries had special dispensations for orphan drugs, some countries also had additional restrictions to control costs. For instance:
Cost-containment measures for expensive medicines in general were implemented for orphan medicines, too. In countries such as Finland, Hungary, Lithuania and Spain, the reimbursement of orphan medicines could be limited to defined clinical conditions, and Hungary and Russia required that the medicine was prescribed in a certain health care setting. Orphan medicines could also be subjected to price-volume agreements and confidential discounts. In Italy, on the other hand, marketing authorisation holders of orphan medicines were excluded from the payback arrangement.
The authors also looked at whether national governments made 10 recent orphan medicines available to their residents. They found significant variability in orphan medicine availability across countries.
The authors also found that “patients generally pay some of the costs when products are dispensed from community pharmacies, and receive products free of charge when they are dispensed from health care units.”
The study is a nice review of orphan drug policy across Europe.
- Sarnola, Kati, Riitta Ahonen, Jaana E. Martikainen, and Johanna Timonen. “Policies and availability of orphan medicines in outpatient care in 24 European countries.” European journal of clinical pharmacology (2018): 1-8.