The Orphan-Medicinal-Product Regulation was introduced in the European Union (EU) to incentivise the development of medicines for rare diseases. Orphan designation can be granted at any stage of the development process to medicines that are intended for diagnosis, prevention or treatment of life-threatening or very serious conditions that affect not more than 5 in 10,000 people in the EU or for which development costs would not be covered by the marketing return without incentives. If methods of treatment, prevention or diagnosis for the rare disease exist, designation is possible only where there is significant benefit brought about to patients by the medicine. Sponsors of medicines designated as orphan medicines by the European Commission benefit from a range of incentives, including market exclusivity for 10 years, fee reductions for some of the Agency's services, protocol assistance and access to the centralised authorisation procedure.
The success of the orphan incentives is underlined by the steady increase in the number of designations for orphan designation: 107 designations were granted in 2011, 148 in 2012, and more than 150 are expected in 2013. In line with this trend, in 2012, 19 applications for marketing authorisation concerned designated orphan medicines, compared with 14 in 2011.
In 2012, a number of substances that received orphan designation were intended for the treatment of diseases for which no treatment existed and no orphan designation had previously been granted, such as Prader-Willi syndrome and hereditary inclusion body myopathy (HIBM).
Expanding international cooperation
The COMP has an established an active international cooperation, which will be expanded in 2013. In 2008, the Committee started collaboration with the United States Food and Drug Administration (FDA), allowing applications for orphan-drug designations to be submitted in parallel to the two agencies. The parallel submission process helps rationalise the development of orphan medicines by facilitating access to parallel scientific advice (protocol assistance) from the two regulatory authorities. Based on the success of this collaboration, which led to 62% of applications submitted in parallel in the EU and the FDA in 2012, last year the COMP began to collaborate with the Japanese regulatory authorities. An increase in the number of Japanese orphan-drug designations with prior European designations was observed in 2012. A dialogue with Health Canada has been established and a closer collaboration with this country is anticipated in 2013.
Collaboration with HTA bodies
The Agency has started working closely with EUnetHTA, an organisation that focuses on scientific cooperation in health technology assessment (HTA) in Europe. HTA bodies provide recommendations on the medicines that can be paid for or reimbursed by the healthcare system in a particular Member State. With respect to orphan medicines, the Agency is working with EUnetHTA towards a better understanding on orphan designation, marketing authorisation of orphan medicines and national competent authorities' initiatives on availability of designated orphan medicines. The dialogue takes the specific characteristics of orphan medicines and the rare conditions they are used for into consideration, and will explore ways of sharing information for the common benefit of patients affected by rare diseases and the financial sustainability of the healthcare systems.
Integrating views of patients
To better serve and address patients' needs, the COMP will proactively request contributions from expert patients in areas where their advice is key, such as contribution to patient care, improvement of quality of life, assessment of significant benefit and the development of medicines in specific indications. The Committee will work to increase the involvement of patient organisations and integrate their views in various areas, such as treatment modality and new formulations.
In particular, the COMP will strengthen its interactions with disease-specific patient organisations and the European Organisation for Rare Diseases (EURORDIS), and develop new interactions with international patient organisations.
"The work of the COMP is evolving and constantly adapting to better serve and address patients' needs, the growing scientific knowledge on rare diseases, and the regulatory framework," explains Bruno Sepodes.