ORPHAN DRUGS REGULATION IN
USA AND CANADA

D e partment O f Re g u l ator y A f fa i rs

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USA CANADA

Orphan drug act (January 4, 1983) No orphan drug policy

i. Designation granted based on prevalence of i.Emergency drug release
disease in the population of less than 200,000 program/investigational new drugs (EDRP/IND)
people (approximately 0.1%) or no reasonable provides access to unapproved drugs.
expectation of profitability.

ii. SR and ED [scientific research (SR) and
ii. Protocol assistance to design research experimental development (ED)] tax incentive
protocols. program would support R and D in the area of

orphan drugs.
iii. Tax credits for clinical research.

iii. Provision for reduction of fees for small
iv. Market exclusivity. market drugs under cost recovery.

v. Funding grants for clinical research to iv. Process patents granted for biotechnology.
support development.

vi. Penalty for intentionally false statement of
orphan status.

vii. Parallel track program and treatment INDs
provide access to unapproved drugs.

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