Orphan drugs are medicines that have been developed to treat a rare medical condition.
To stimulate the research and development into such medicines for rare diseases, many jurisdictions have put in place incentive programs to support orphan drug development. Otherwise, there may not be sufficient interest because of a lack of profitability.
For example, the United States of America provided an incentive program through its Orphan Drug Act as early as 1983. The European Union adopted similar regulations in 2000 (Regulation (EC) No 141/2000 on Orphan Medicinal Products).
Incentives usually include scientific advice from regulators, expedited approval process, reduced fees to obtain marketing approval, tax breaks and market exclusivity.
Initially, orphan drugs were mostly developed by smaller pharmaceutical companies for rare and genetic diseases. In recent years, however, there has been growing interest amongst big pharma and financial investors. The reasons include:
- With the patent cliff and less new drug blockbusters, investors are more keen on smaller markets with medical needs that are often still unme
- Orphan drugs command premium pricing due to a lack of competition.
- Precision or personalised medicine is becoming more popular. Medical treatment is becoming more targeted in smaller patient populations. As such, more drugs become eligible for orphan drug status, e.g. in oncology.
In Singapore, the Medicines (Orphan Drugs) (Exemption) Order (the “Orphan Drugs Order”) defines an “orphan drug” as a medicinal product which:
- has been identified by any doctor or dentist as an appropriate and essential remedy with no effective substitute available for the treatment of any rare disease (which means a life-threatening and severely debilitating illness affecting less than 20,000 persons);
- has not been granted a product licence under the Medicines Act (Cap. 176); and
- has been approved by the competent health authorities either of the country of the origin or of any other country where the orphan drug has been used.
The Health Sciences Authority (the “HSA") may permit any person to import or supply any orphan drug without a product licence if the drug is to be used by a doctor or dentist who has prescribed the drug for the treatment of a patient under his care (the “Permitted Person”).
Any imported orphan drug must be kept in a hospital and be under the charge and control of a doctor, dentist or pharmacist appointed by that hospital (the “Custodian”).
Where an orphan drug is urgently required for patient treatment, the drug may be supplied by the Permitted Person or the Custodian directly to the prescribing doctor or dentist.
The Permitted Person also has to (amongst others):
- maintain written records pertaining to the quantity imported or supplied; the date received or supplied; and the name and address of the person to whom the drug is supplied; and
- notify the HSA in writing if he intends to import any orphan drug, and provide information relating to the name, quantity and other relevant particulars of the drug so imported.
Failure to comply with these obligations may constitute an offence and is punishable with a fine of SGD1,000 (about USD720).
The Orphan Drugs Order prohibits any person from publishing or causing the publication of any advertisement which is likely to lead to the use of any orphan drug. Contravention of this prohibition may be punishable with a fine of SGD1,000 (about USD720).
This prohibition does not apply to any advertisements contained in a publication intended for circulation to any person in charge of a hospital or other institution for use by persons engaged in the hospital or institution for education or research purposes.