Australian process for subsidised access to orphan drugs.
In an article published in Expert Opinion Orphan drugs, the author reviews the process for registering orphan drugs for inherited disorders of metabolism and providing access to subsidised therapy through a centralized national program in Australia. The federal government of Australia subsidises an extensive range of drugs under the Pharmaceutical Benefits Scheme (PBS). In addition, financial assistance under the Life Saving Drugs Program (LSDP) is made available for certain expensive drugs to treat, for instance rare inherited disorders of metabolism. The government provides funding each year for the LSDP, which especially subsidises prohibitively expensive drugs, beyond the reach of the patient and which significantly lengthens the life expectancy of the patient. In Australia, the Therapeutic Goods Administration (TGA) provides marketing authorisation for all drugs in Australia. Once a drug is registered by the TGA, an application can be made by the drug company to the Pharmaceutical Benefits Advisory Committee (PBAC) for the drug to be funded under LSDP instead of PBC. PBAC is an independent, expert advisory body that makes recommendations to the Federal Minister for Health and Ageing on which drugs should be listed for subsidies. PBAC is considered and expert body as it is “comprised of doctors, a health economist, other health professionals and a consumer representative” who collectively assess and provide advice on the efficacy of a drug to be funded under LSDP. The LSDP is also aided by the Disease Advisory Committee (DAC) for making disease specific assessment of the needs of the patients, for eg., patients suffering from rare metabolic disorders.
This streamlined procedure where expertise is centralised and “all Australian patients are managed and treated under the aegis of disease-specific specialist clinical advisory committee” is especially helpful in a large but population sparse country like Australia where rare disease patients are few and expert centers can be a great distance apart. However, the author emphasises that since only medications that "significantly lengthen" lives are funded under these schemes, rare diseases patients may not have the option to access available medications that may not necessarily augment life but may significantly improve their quality of life.
source: eurordis