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
  1. 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
  2. Availability of alternative treatments
    • There is no alternative method of diagnosis, prevention or treatment of the indicated conditions
  3. Marketing of the product does not generate sufficient return to justify the investment
How to apply for orphan designation
  1. Application for orphan designation may be made at ANY stage of product lifecycle
    • During development, marketing authorization or post-approval
  2. 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
  3. Orphan designation is attained when a medicine is accepted as an orphan medicine
  4. The Authority shall review the documents and inform the Applicant if the orphan designation is approved within 30 days
  5. Evaluation shall be completed 2 months after the applicant has submitted a complete dossier
Incentives for registering orphan medicines
  1. Exemption from some provisions of the Fees and Chargers
    • No application fees, no retention fees
  2. Expedited review within two months of orphan designation
  3. 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
  1. Only therapeutic indications related to orphan disease shall be approved
    • Applicants may apply for separate marketing authorization for other non-orphan indications
  2. Registration is valid for 2 years after which renewal is required