Authors' objectives: The technical assessment described in this document was carried out to provide information to the Australian Government Department of Health as part of the Life Saving Drugs Programme (LSDP) Review. The objective of the Review was to examine important issues such as access and equity, value for money and the future administration of the LSDP. The technical assessment included consideration of: - the effectiveness and safety of drugs currently funded through the LSDP; - treatments and diseases for which funding through the LSDP may be sought in the future; - international approaches to defining rare diseases and funding drugs that treat those diseases; and - the value for money of the currently funded drugs. The technical assessment also aimed to establish a framework for collecting data on rare diseases in Australia and to assess how this could function internationally. What is the LSDP? The LSDP provides subsidised access for eligible individuals to expensive and potentially lifesaving drugs for rare life-threatening diseases. The issues considered during the assessment process involve: - the review of the evidence presented by the drug sponsor; and - whether the drug is suitable for listing on the Pharmaceutical Benefits Scheme (PBS) (drugs considered cost-effective are listed on the PBS).

Authors' recommendations: This technical assessment has shown that most of the drugs currently funded by the LSDP are clinically effective and with an acceptable safety profile. However, there are warning signs that the LSDP is unlikely to be sustainable in the future, given current patterns of drug development and marketing. Lessons learned from international experience in the public funding of orphan drugs and from economic theory show a range of approaches that might be adopted to work towards a sustainable LSDP. One of these approaches could include the development of a drug surveillance registry to help determine whether each drug performs as expected.

Project Status: Completed

Year Published: 2015

URL for published report: http://www.pbs.gov.au/reviews/lsdp-technical-assessment-report/lsdp-review-technical-assessment-april-2015.pdf

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: Australia

Organisation Name: Adelaide Health Technology Assessment

Contact Address: School of Public Health, Mail Drop 545, University of Adelaide, Adelaide SA 5005, AUSTRALIA, Tel: +61 8 8313 4617

Contact Name: ahta@adelaide.edu.au

Contact Email: ahta@adelaide.edu.au

Copyright: Adelaide Health Technology Assessment (AHTA)