Orphan Drugs and Rare Diseases
An insight into orphan drugs and rare diseases in Denmark. Prepared in association with Gorrissen Federspiel, a leading law firm in Denmark, this is an extract from The Pharma Legal Handbook: Denmark, available to purchase here for GBP 75.
1. What is the definition of Rare Diseases in your country?
Pursuant to Regulation (EC) no. 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products (“Orphan Drug Regulation”), a medicinal product may be designated as an orphan medicinal product if its sponsor can establish that
- it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not more than five in 10 thousand persons in the community (the prevalence criterion), or
- it is intended for the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic condition in the Community and that without incentives it is unlikely that the marketing of the medicinal product in the Community would generate sufficient return to justify the necessary investment (the insufficient return on investment criterion), and
- there exists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorised in the community or, if such method exists, that the medicinal product will be of significant benefit to those affected by that condition.
Applications for orphan medicinal product designations are assessed by the Committee for Orphan Medicinal Products.
Denmark has adopted the European Commission definition of rare disease, but the National Strategy for Rare Diseases (“National Strategy”, as published by the Danish Health Authority in 2018), uses a different definition of the term rare diseases (Danish: sjældne sygdomme). The definition used in the National Strategy is:
- A number of rare usually congenital, hereditary complex and serious diseases and conditions that require special knowledge and skills, and who needs a highly specialized especially well-planned coordinated efforts in the form of highly specialized diagnosis, treatment, monitoring and control 1- 2 places in the hospital service,
- Rare diseases occur with a prevalence of no more than 1-2 in 10,000 persons, i.e., up to approximately 500-1,000 people in Denmark. The diseases often cannot be cured, but with appropriate effort, the consequences of the diseases might be prevented, restricted or treated and patients thus ensured better quality of life and survival.
2. Does the designation of ‘Orphan Drug’ exist in your country? (Does it correspond with the definition of Rare Diseases?)
Denmark has adopted the Orphan Drug Regulation.
3. What is the regulatory framework for the authorization of an Orphan Drug? (Is this regulatory framework based on Rare Disease status or can it alternatively be based on Orphan Drug foreign status?)
A designation as an orphan medicinal product under the Orphan Drug Regulation does not mean that the product can be marketed. As for other medicinal products, a marketing authorization must be obtained prior to placing the orphan drug on the market.
As mentioned above, the centralized procedure is mandatory for orphan drugs. The application for a centralized marketing authorization must submitted to the EMA who is responsible for review of the application, and the European Commission oversees final authorization.
In its publication “Rare diseases, orphan medicines” dated 28 February 2018, the EMA has stipulated that an orphan drug designation granted outside the EU does not count within the EU.
4. Does your country have provisions for relaxed clinical trial/scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?
No, there are no relaxed clinical trial requirements special to orphan drugs. However, the Orphan Drug Regulation contains several incentive schemes intended to support research, development, and availability of orphan medicinal products. For example, the EMA offers “protocol assistance” which is a specific type of scientific advice for the sponsors once a medicinal product has received the designation as an orphan medicinal product.
Sponsors of orphan medicinal products may also apply for reduced fees for regulatory activities.
Furthermore, as a starting point, authorised orphan medicinal products benefit from ten years of protection from market competition with similar medicines.
According to the National Strategy, there are no special incentives provided in Denmark, and the Danish Health Authority is not aware of any plans for future incentives. However, with regard to measures to support the availability of orphan medicinal products, a compassionate use permit may also be applied for with respect to orphan medicinal products.
5. Is there an expedited pathway for Orphan Drugs?
The maximum timeframe for evaluation of marketing authorizations under the centralized procedure is 210 days.
The EMA has stated that orphan drugs will not automatically qualify for accelerated assessment. However, in exceptional cases, the Committee for Medicinal Products for Human Use may initiate an accelerated evaluation.
6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?
As a general rule, foreign marketing authorizations are not recognized in Denmark. However, Denmark recognizes authorizations granted by the European Commission under the centralized procedure.
7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan drugs?
There is no specific reimbursement procedure for orphan drugs. However, orphan drugs may be determined eligible for reimbursement under the general reimbursement system. Please see Question 2 on Regulatory, Pricing and Reimbursement Overview.
8. How are the prices of Orphan Drugs regulated?
Please see Question 2 on Regulatory, Pricing and Reimbursement Overview.
9. In case of reference price based on a basket of countries, what countries are included?
10. Have there been any significant legal/judicial developments in relation to Orphan Drugs in your country?
11. Are there proposals for reform or significant change to the regulation of Orphan Drugs? If yes, when are they likely to come into force?
The Danish Health Authority’s National Strategy of 2018 contains a large number of initiatives to support rare diseases in Denmark. With respect to orphan medicinal products, the Danish Health Authority makes references to the Danish Medicines Council (which was established in 2017) as having a significant role in ensuring access to medicinal products for rare diseases and patient groups.
The Danish Medicines Council has established a number of committees within specific disease areas, e.g. haemophilia. The committees will assess and evaluate new medicinal products and indications. Furthermore, the Danish Medicines Council is are wording on a model for ensuring that the Danish Regions make expedient decisions on taking new medicinal product for rare diseases into service.
The Danish Health Authority conducts continuous evaluation of the strategy for rare diseases and orphan medicinal products in Denmark to ensure that the strategies are implemented. The next National Strategy is expected in 2021.