A rare disease advocacy network has applauded the European Medicines Agency’s (EMA) efforts to incentivize the development of treatments for rare diseases, but says the variance in treatment access across the EU is “unacceptable.”
In an interview with EurActiv, Terkel Andersen, president of the advocacy group EURODIS, said that EMA and the European Commission (EC) are “doing their utmost to try to make rare diseases ‘attractive’ for the pharmaceutical industry.”
Andersen finds dialogue between patient groups, regulatory authorities and members of industry to be highly beneficial, and says that the EC has helped foster patient participation in the drug development process.
The EU's Regulatory Framework for Rare Disease Treatments
In recent years, EMA has worked to promote the development of treatments for rare diseases.
One of the ways the agency has done so is through the creation of its orphan product designation, which applies to treatments for rare diseases. A product with orphan designation is eligible to receive free protocol and scientific advice from EMA, and if approved, can receive 10 years of market exclusivity.
Orphan product designation was first introduced in the EU in 2000 under Regulation (EC) No 141/2000.
To qualify for orphan product designation, a treatment must fit several criteria:
- The medicine must treat, prevent or diagnose a life-threatening or chronically debilitating condition.
- The condition must affect fewer than 5 in 10,000 people in the EU. Alternatively, if a condition affects more than 5 in 10,000 people in the EU it may still be considered for orphan designation if it is “unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development.”
- There must be no existing approved treatments for the indicated condition, or if there are, the product in question must offer significant improvements over the other options.
EMA recommended 17 drugs with orphan product designation for approval in 2014, the highest number by the agency in a single year.
Access to Treatment Still a Struggle
Despite improvements made at EMA and EC, Andersen finds the differences in level of care in different European nations to be problematic.
According to Andersen, patients continue to face two major challenges to their treatment and care.
First, patients may face obstacles to obtaining an accurate or timely diagnosis due to a lack of awareness or expertise of a rare disease. Second, for many rare diseases, there may be no approved treatments available. Even in cases where a treatment is available, accessing the treatment could require travelling to a specialized facility, which is difficult or impossible for some patients.
Andersen also thinks the EC could do more to increase awareness of rare diseases at the country level, and says that having varying levels of awareness and care from country to country is unacceptable from the point of view of EU citizens.
While incentives—and the potential for high prices—have piqued the pharmaceutical industry’s interest in rare diseases, Andersen says that developing treatments for rare diseases should eventually benefit other patients as well.
Andersen says that industry is beginning to recognize opportunities to translate “a lot of what is being learned from rare diseases into the development of personalized medicines” in the future.
"This experience will be also valuable in the future for a broader patient community."