Commonwealth Numbered Regulations Commonwealth Numbered Regulations
Therapeutic Goods Regulations 1990
1 Regulation 2 (definition of designated orphan drug )
Omit "an orphan drug designated under subregulation 16J(2)", substitute "a medicine in relation to which a designation under regulation 16J is in force".
2 Regulation 2
Insert:
"new dosage form medicine " means a medicine that:
(a) has the same chemical, biological or radiopharmaceutical active ingredient (or fixed combination of such ingredients) as another medicine that is included in the Register; and
(b) has an indication in common with that other medicine; and
(c) does not have the same dosage form as that other medicine.
3 Regulation 2
Repeal the following definitions:
(a) definition of orphan drug ;
(b) definition of rare disease .
4 Part 3B
Repeal the Part, substitute:
Part 3B -- Designated orphan drugs
16H Application to designate medicine as orphan drug
(1) The sponsor of a medicine may apply to the Secretary to designate, in writing, the medicine as an orphan drug.
(2) An application under subregulation (1) must be in a form approved, in writing, by the Secretary.
16J Designation of medicine as orphan drug
(1) On receiving an application under subregulation 16H(1) to designate a medicine as an orphan drug, the Secretary must:
(a) consider the application; and
(b) decide either:
(i) to designate the medicine as an orphan drug; or
(ii) to refuse to designate the medicine as an orphan drug.
(2) The Secretary may decide to designate the medicine as an orphan drug if the Secretary is satisfied, having regard to any matter that the Secretary considers relevant, that:
(a) if the medicine is not a new dosage form medicine--all of the criteria specified in subregulation (3) are satisfied in relation to the medicine; or
(b) if the medicine is a new dosage form medicine--all of the criteria specified in subregulation (4) are satisfied in relation to the medicine.
General criteria
(3) The following criteria are specified in relation to a medicine that is not a new dosage form medicine:
(a) the application is for only one indication of the medicine;
(b) the indication is the treatment, prevention or diagnosis of a life-threatening or seriously debilitating condition in a particular class of patients (the relevant patient class );
(c) it is not medically plausible that the medicine could effectively treat, prevent or diagnose the condition in another class of patients that is not covered by the relevant patient class;
(d) at least one of the following applies:
(i) if the medicine is intended to treat the condition--the condition affects fewer than 5 in 10,000 individuals in Australia when the application is made;
(ii) if the medicine is intended to prevent or diagnose the condition--the medicine, if it were included in the Register, would not be likely to be supplied to more than 5 in 10,000 individuals in Australia during each year that it is included in the Register;
(iii) it is not likely to be financially viable for the sponsor to market the medicine in Australia unless each fee referred to in paragraph 45(12)(c) were waived in relation to the medicine;
(e) none of the following has refused to approve the medicine for the treatment, prevention or diagnosis of the condition for a reason relating to the medicine's safety:
(i) the Secretary;
(ii) the United States Food and Drug Administration;
(iii) the European Medicines Agency;
(iv) Health Canada;
(v) the Medicines and Healthcare products Regulatory Agency of the United Kingdom;
(f) either:
(i) no therapeutic goods that are intended to treat, prevent or diagnose the condition are included in the Register; or
(ii) if one or more therapeutic goods that are intended to treat, prevent or diagnose the condition are included in the Register--the medicine provides a significant benefit in relation to the efficacy or safety of the treatment, prevention or diagnosis of the condition, or a major contribution to patient care, compared to those goods.
New dosage form medicines
(4) The following criteria are specified in relation to a new dosage form medicine:
(a) the application is for only one indication of the medicine;
(b) the indication is the treatment, prevention or diagnosis of a life-threatening or seriously debilitating condition;
(c) it is not likely to be financially viable for the sponsor to market the medicine in Australia unless each fee referred to in paragraph 45(12)(c) were waived in relation to the medicine;
(d) none of the following has refused to approve the medicine for the treatment, prevention or diagnosis of the condition for a reason relating to the medicine's safety:
(i) the Secretary;
(ii) the United States Food and Drug Administration;
(iii) the European Medicines Agency;
(iv) Health Canada;
(v) the Medicines and Healthcare products Regulatory Agency of the United Kingdom;
(e) either:
(i) no therapeutic goods that are intended to treat, prevent or diagnose the condition are included in the Register; or
(ii) if one or more therapeutic goods that are intended to treat, prevent or diagnose the condition are included in the Register--the medicine provides a significant benefit in relation to the efficacy or safety of the treatment, prevention or diagnosis of the condition, or a major contribution to patient care, compared to those goods.
Publication of decision
(5) If the Secretary decides to designate the medicine as an orphan drug, the Secretary must, as soon as practicable after making the decision, publish a notice on the Department's website stating the following:
(a) the name of the sponsor of the medicine;
(b) the indication referred to in paragraph (3)(a) or (4)(a);
(c) the dosage form of the medicine;
(d) that the medicine is a designated orphan drug.
Notification of decision
(6) As soon as practicable after making the decision, the Secretary must notify the applicant, in writing, of the decision.
(7) If the Secretary decides to refuse to make the designation, the notification must include the reasons for the decision.
16K Period during which designation is in force
(1) A designation under regulation 16J of a medicine as an orphan drug:
(a) comes into force when it is made; and
(b) remains in force for a period of 6 months.
(2) Subregulation (1) has effect subject to regulations 16L and 16M.
(1) If a designation under regulation 16J of a medicine as an orphan drug is in force, the sponsor of the medicine may apply to the Secretary to extend the designation for a further 6 months.
(2) An application under subregulation (1) to extend a designation must:
(a) be in a form approved, in writing, by the Secretary; and
(b) be made at least 28 days before the designation would cease to be in force.
(3) On receiving an application under subregulation (1) to extend a designation, the Secretary must decide either:
(a) to extend the designation; or
(b) to refuse to extend the designation.
(4) The Secretary may extend the designation if:
(a) the Secretary has not previously extended the designation; and
(b) the Secretary is satisfied that:
(i) if the medicine is not a new dosage form medicine--the criteria specified in paragraphs 16J(3)(e) and (f) are satisfied in relation to the medicine; or
(ii) if the medicine is a new dosage form medicine--the criteria specified in paragraphs 16J(4)(d) and (e) are satisfied in relation to the medicine; and
(c) the Secretary is satisfied that, if the Secretary were to extend the designation, the sponsor would make an application in relation to the medicine under section 23 of the Act before the end of the extended designation.
Notification of decision
(5) As soon as practicable after making the decision, the Secretary must notify the applicant, in writing, of the decision.
(6) If the Secretary decides to refuse to extend the designation, the notification must include the reasons for the decision.
(1) The Secretary may, by written notice given to the sponsor of a designated orphan drug, revoke the designation:
(a) on application by the sponsor; or
(b) if the Secretary is satisfied that:
(i) if the designated orphan drug is a new dosage form medicine--the criteria specified in subregulation 16J(3) are no longer satisfied in relation to the designated orphan drug; or
(ii) if the designated orphan drug is not a new dosage form medicine--the criteria specified in subregulation 16J(4) are no longer satisfied in relation to the designated orphan drug.
(2) Subsection 33(3) of the Acts Interpretation Act 1901 does not apply in relation to a revocation of a designation under regulation 16J of a medicine as an orphan drug.
5 Paragraphs 45(12)(a) and (b)
Repeal the paragraphs.
6 Subregulation 48(1) (paragraph (e) of the definition of initial decision )
Repeal the paragraph, substitute:
(e) subparagraph 16J(1)(b)(ii);
(ea) paragraph 16L(3)(b);
(eb) paragraph 16M(1)(b);