Therapeutic Goods Regulations 1990

On receiving an application under subregulation 16H(1) to designate a medicine as an orphan drug, the Secretary must:

consider the application; and

decide either:

to designate the medicine as an orphan drug; or

to refuse to designate the medicine as an orphan drug.

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:

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

if the medicine is a new dosage form medicine—all of the criteria specified in subregulation (4) are satisfied in relation to the medicine.

The following criteria are specified in relation to a medicine that is not a new dosage form medicine:

the application is for only one indication of the medicine;

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);

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;

at least one of the following applies:

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;

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;

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;

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:

the Secretary;

the United States Food and Drug Administration;

the European Medicines Agency;

Health Canada;

the Medicines and Healthcare products Regulatory Agency of the United Kingdom;

either:

no therapeutic goods that are intended to treat, prevent or diagnose the condition are included in the Register (except in the part of the Register for goods known as provisionally registered goods); or

if one or more therapeutic goods that are intended to treat, prevent or diagnose the condition are included in the Register (except in the part of the Register for goods known as provisionally registered goods)—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.

The following criteria are specified in relation to a new dosage form medicine:

the application is for only one indication of the medicine;

the indication is the treatment, prevention or diagnosis of a life‑threatening or seriously debilitating condition;

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;

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:

the Secretary;

the United States Food and Drug Administration;

the European Medicines Agency;

Health Canada;

the Medicines and Healthcare products Regulatory Agency of the United Kingdom;

either:

no therapeutic goods that are intended to treat, prevent or diagnose the condition are included in the Register (except in the part of the Register for goods known as provisionally registered goods); or

if one or more therapeutic goods that are intended to treat, prevent or diagnose the condition are included in the Register (except in the part of the Register for goods known as provisionally registered goods)—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.

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:

the name of the sponsor of the medicine;

the indication referred to in paragraph (3)(a) or (4)(a);

the dosage form of the medicine;

that the medicine is a designated orphan drug.

As soon as practicable after making the decision, the Secretary must notify the applicant, in writing, of the decision.

If the Secretary decides to refuse to make the designation, the notification must include the reasons for the decision.