Orphan medicines
Key definitions
- Orphan medicine is a medicine intended to diagnose, treat and prevent rare conditions, referred to as orphan diseases
- Orphan disease is a rare disease affecting less that 25,000 patients
- Orphan designation is attained when a medicine is accepted as an orphan medicine
Rationale for Regulations of Orphan Medicines
- To ensure that patients suffering from orphan diseases receive the same quality of healthcare as other patients
- To provide commercial incentives in order to encourage developing and marketing of orphan medicines
- Moral responsibility of the Government to facilitate access and availability of orphan medicines
Criteria for Orphan Designation
- Therapeutic indications of the medicine
- The medicine must be intended for a life threatening seriously debilitating or serious and chronic disease affecting less than 25,000 persons
- Availability of alternative treatments
- There is no alternative method of diagnosis, prevention or treatment of the indicated conditions
- Marketing of the product does not generate sufficient return to justify the investment
How to apply for orphan designation
- Application for orphan designation may be made at ANY stage of product lifecycle
- During development, marketing authorization or post-approval
- Submit a cover letter expressing interest for orphan designation including the following:
- Details of the proposed indication & rare disease
- Demonstration that criteria for orphan medicine have been met
- Orphan designation is attained when a medicine is accepted as an orphan medicine
- The Authority shall review the documents and inform the Applicant if the orphan designation is approved within 30 days
- Evaluation shall be completed 2 months after the applicant has submitted a complete dossier
Incentives for registering orphan medicines
- Exemption from some provisions of the Fees and Chargers
- No application fees, no retention fees
- Expedited review within two months of orphan designation
- Technical assistance in meeting regulatory requirements
- Collaborative approach & real time communications regarding additional information and pending issues
- Pre-submission meetings
Limitations of marketing Orphan Medicines
- Only therapeutic indications related to orphan disease shall be approved
- Applicants may apply for separate marketing authorization for other non-orphan indications
- Registration is valid for 2 years after which renewal is required
