Orphan Drug Legislation
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Some diseases are so rare that it is unlikely that expected sales of the medicinal product would cover the investment in its development (for example, myasthenia gravis). Products to treat some diseases which may be very common are still not a commercially viable proposition because the patients cannot afford to pay for treatment (tuberculosis). To encourage the development of treatments for such conditions many regulatory authorities have introduced systems of incentives for "orphan medicinal products".
European Union
A disease or disorder is defined as rare in Europe when it affects fewer than 5 in 10,000 citizens (Orphan Drug Regulation 141/2000). Although this may seem a small number, by this definition, rare diseases may affect as many as 30 million European Union citizens. According to EURORDIS (European Organisation for Rare Diseases) there are about 6000 to 8000 rare diseases, most of which have identified genetic conditions, and approximately five new rare conditions are described in the medical literature every week.
The small numbers of patients with any given rare disease (5:10,000 translates to 246,000 people in the EU) mean that it is unlikely that expected sales of the medicinal product would cover the investment in its development. In the EU, companies are offered a number of incentives for developing drugs for orphan indications, including:
- 100% reduction for protocol assistance and follow-up
- 100% reduction for pre-authorisation inspections
- 50% reduction for new applications for marketing authorisation
- 50% reduction for post-authorisation activities, including annual fees (applies only to small and medium-sized enterprises), in the first year after granting of a marketing authorisation
The United States
In the US, orphan drug designation can be obtained if the disease or condition for which the drug is intended affects fewer than 200,000 people in the United States.
Incentives for development of orphan drugs include:
- seven years of marketing exclusivity after approval of its orphan drug product
- tax incentives for clinical research undertaken on the orphan product
- coordination of research study design assistance for sponsors of drugs for rare diseases
- grant funding to defray costs of qualified clinical testing expenses incurred in connection with the development of orphan products
The Office of Orphan Products Development also encourages sponsors to conduct open protocols, allowing patients to be added to ongoing studies.
Japan
In Japan, a rare disease is defined as one with fewer than 50,000 prevalent cases. The orphan drug plan allows for market exclusivity following approval.
Australia
If a product is to be accorded orphan drug status it must be intended to treat, prevent or diagnose a rare disease; alternatively the sponsor must demonstrate that it is not commercially viable to supply the product to treat, prevent or diagnose another disease or condition. The Therapeutic Substances Regulations do not give a definition of a rare disease or orphan indication in terms of numbers of patients, other than to say that if the product is a vaccine or in vivo diagnostic it must not be intended for use in more than 2000 patients a year. To obtain orphan designation, "the application must show why the medicine is an orphan drug."
Incentives offered by the Therapeutic Goods Administration include assessment fee waiver and priority assessment. Annual fees are still payable but the authorisation holder can apply for a reduction on the basis of low volume/value sales..
How to obtain 'orphan drug designation'
Each of the above jurisdictions operates a system whereby an application is made to an expert committee for the purposes of demonstrating orphan status. The guidelines are more helpful in some jurisdictions than others! However they all set the same basic objective: either demonstrate that the indication is rare, by using government statistics on incidence or prevalence, or demonstrate using data on R & D and manufacturing costs compared to projected sales that the cost of developing the product would be prohibitive without the incentives on offer.
A simpler way would be to call Akos. With wide experience in obtaining orphan drug designations we can collate all the guidelines, apply them to your product and compile a dossier for submission to the appropriate agency. We can follow-up and support the application through the assessment process, including oral explanations or hearings as necessary.
To discuss your regulatory questions with Akos please contact us:
Tel: +44 (0)1582 766 339
Email: regulatory@akos.co.uk