Certified product details (CPD)

Most sponsors would be familiar with the CPD document and its content. The requirement for the preparation and submission of CPDs was first introduced as a firm TGA policy in 1990 after consultation with industry. The primary aim of requiring a CPD is to ensure that there is a single document which provides details of the finished product specifications (ie test parameters, limits and test methods) agreed between the sponsor and the TGA for registered drug products. The document is particularly useful for the TGA Laboratories (TGAL) for sample testing purposes. The CPD also includes additional information on the product, including formulation, pack sizes and shelf-life/storage conditions.

The following guidance is provided to assist sponsors in deciding at which point in the evaluation process and/or under what circumstances a CPD should be prepared and submitted.

1. DO submit a CPD incorporating the final agreed finished product specifications, including test methods, following the conclusion of the evaluation of the chemical and pharmaceutical (Part II) data in a Category 1 application. It is preferred that submission of the CPD be upon request by the TGA. Alternatively, submission may be at the sponsor's initiative if it is confident that all Part II matters have been resolved. In either case, it is in the sponsor's interest to provide the document in a timely manner for checking by the evaluation section so that it is available for use by TGAL if sample testing is required.
2. A CPD will be requested of a sponsor upon approval of any Category 3 application which involved changes to certain elements of the finished product specifications, eg. changes to test parameters, limits or test methods. A one month timeframe is generally appropriate when such a request is made.
3. DO submit a CPD incorporating the relevant change(s) made to a product when notifying the TGA of a self-assessable change if it is a requirement in the self assessment guidelines (Appendix 8 of the AGRD Vol 1, 2nd edition). Such changes again would relate in some way to product specifications.
4. It is generally NOT advisable to submit a CPD with the Part II data supporting a Category 3 application because experience has shown that the contents of the document often require further revision during the evaluation process and new editions are then generally required.
   It is preferred that the document is submitted when requested by the TGA (see point 2).
5. DO NOT submit a CPD following changes to product details such as formulation, shelf- life/storage conditions, pack sizes or container type for a product even though such information is in the CPD document. This is because such information is generally not critical for laboratory testing purposes. Additionally, since these aspects of the Part II data of a product do undergo variations rather frequently, it is undesirable and an unnecessary use of resources to constantly update the CPD due to such variations. Details of any changes of these types are in any case clearly reflected either in a specific TGA approval letter or in the sponsor's notification form if the change is self-assessable.

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Orphan drug program

Orphan drug products are drugs, vaccines or in vivo diagnostic agents which physicians use to treat, prevent or diagnose rare diseases. Usually the drugs are not commercially viable. Pharmaceutical companies often do not develop and market such products because the financial return is small compared with the costs of development and marketing. When a drug has no sponsor for this reason, it is known as an "orphan" drug. The lack of such drugs may deprive patients with rare diseases of diagnosis and treatment but these patients have rights to drugs of the same quality, efficacy and safety as patients with common illnesses. The TGA intends the Orphan Drug Program to overcome this problem and has based its Program on that of the Food and Drug Administration of the USA.

The Australian Program encourages sponsors to market orphan drugs in Australia by reducing costs through waiving fees and by providing exclusive approval. The TGA will waive fees for the application for orphan drug designation, the application for registration and for the initial evaluation of data. A further commercial advantage is an expected shorter approval time than the statutory 255 working days for the usual Category 1 applications. Annual fees will still apply but may be reduced on the basis of low volume of usage and low value.

Designation of a drug product as an orphan drug: The first step in registering an orphan drug product is for the sponsor to apply to the Drug Safety and Evaluation Branch of the TGA for orphan drug designation. Orphan status is determined by the indication so that a drug is not designated an orphan except by reference to a specific indication.

Two requirements are to be met. The first is that the prevalence of the disease or disorder to be treated should be equal to or less than 2,000 affected individuals, or, if the drug is a vaccine or in vivo diagnostic agent, the persons to whom the drug will be administered in Australia are fewer than 2,000 per year at the time the request is made. The second requirement is when the prevalence of a disease or disorder is greater than 2,000 individuals but the sponsor can show the marketing of the drug would not be financially viable. The information needed to support this claim is the same as that required by the FDA.

If orphan status is given, the TGA will advise the sponsor and publish the drug's designated orphan status, with the dose form, indication and name of the sponsor in the Australian Government Gazette. The designation of an orphan drug is not an exclusive process and the TGA may designate more than one drug as orphan drugs for the same indication.

Registration of a designated orphan drug product: The second step is for the sponsor to apply to register the designated drug on the Australian Register of Therapeutic Goods (ARTG). The TGA will not approve registration of a drug product containing the same drug for the same indication as a product which is already registered for a rare disease or condition, unless it is shown to be clinically superior to the registered product. The definition of clinical superiority is that of the FDA for this purpose. Biologicals are a special case because criteria for the "sameness" of two biologicals are complex.

Under certain conditions, such as the inability of a sponsor to supply the orphan drug product in adequate quantity, the TGA may approve another product with the same active ingredient for registration for the same indication.

Processing and Evaluation Times: Initial applications to register a drug or to extend the indication for a registered drug will be Category 1 with a legal time frame for completion of 255 days. However the TGA expects the applications to be completed well before this date, especially when FDA designation and evaluation reports are available. It is not possible to estimate this time at present, but a review of the program, planned twelve months after its start, will indicate a reasonable time to completion. Category 3 application will incur full fees and be completed in the existing time frame of 45 days as for non-orphan Category 3 applications.

Referral to the ADEC: Applications for either designation or registration that are refused by the TGA, will be referred to the ADEC for advice and comment.

Special Cases: The TGA recommends that the following classes of products be discussed as soon as possible with the Orphan Drug Unit of the Drug Safety and Evaluation Branch because special circumstances may apply to their registration and use in Australia: products from blood and plasma; recombinant products; vaccines; products from human tissue regulated as drugs; products classed as those for gene therapy; and products for use in children.

Information: The TGA intends to publish its full policy in booklet form.