Guidance for Sponsors
A guidance document has been developed to provide a clearly defined and transparent process and associated timelines in the consideration of medicines seeking funding through the LSDP. This guidance is intended to assist sponsors in preparing an application to the make a rare disease medicine available on the LSDP; ensuring access to treatment for Australians with rare diseases is not unnecessarily delayed.
LSDP guidance document (Version 1.0) (PDF 1139 KB)
LSDP guidance document (Version 1.0) (Word 1511 KB)
The Department can provide pre-submission advice on the preparation of an application for LSDP funding. This advice is not binding on the Expert Panel’s consideration of a subsequent application.
A sponsor can email a request for a pre-submission meeting to the LSDP Expert Panel secretariat
LSDP Funding Criteria for a medicine
The sponsor of a medicine must satisfy the following criteria for that medicine to be considered suitable for inclusion on the LSDP. The Expert Panel assesses the sponsor’s application against these criteria and provides their recommendation to the CMO. Sponsors should refer to the LSDP Guidance document for further information and guidance on how to apply for funding.
The drug must be found to meet each of the following criteria:
- There is a rare but clinically definable disease for which the drug is regarded as a proven therapeutic modality, i.e. approved for that indication by the Therapeutic Goods Administration.
- The disease is identifiable with reasonable diagnostic precision.
- Epidemiological and other studies provide evidence that the disease causes a significant reduction in age-specific life expectancy for those suffering from the disease.
- There is evidence to predict that a patient’s lifespan will be substantially extended as a direct consequence of the use of the drug.
- The drug must be accepted as clinically effective, but rejected for Pharmaceutical Benefits Scheme (PBS) listing because it fails to meet the required cost effectiveness criteria.
- There is no alternative drug listed on the PBS or available for public hospital in-patients, which can be used as lifesaving treatment for the disease. However, the availability of an alternative drug under the LSDP does not disqualify the proposed drug from consideration for the LSDP.
- There is no alternative non-drug therapeutic modality (eg. surgery, radiotherapy) which is recognised by medical authorities as a suitable and cost effective treatment for this condition.
- The cost of the drug, defined as the cost per dose multiplied by the expected number of doses in a one year period for the patient, would constitute an unreasonable financial burden on the patient or his/her guardian.
Consideration and advice will also be sought, if applicable, on:
- The proposed price of the drug compared with the effective price of the drug in comparable overseas markets.
- The proposed cost of the drug compared with the cost of comparable drugs, if any, that are already funded through the LSDP.
Only the cost of the drug will be funded through the LSDP. This may include a factor for importation and transportation of the drug by the manufacturer direct to the place of administration to the patient. No other transport, storage, administration, or any other hospital or medical expenses associated with the use of the drug, or management of the disease or condition, will be funded through the LSDP.
The chart below provides an overview of the new-medicine assessment process.
Your comments are welcome whether you are a patient, carer, member of the public, health professional or member of a consumer interest group. Once the agenda for the upcoming Expert Panel meeting is published, any interested parties are welcome to provide their input directly to the LSDP Expert Panel Secretariat
via email. Comments usually close two weeks before the meeting.
All stakeholder comments received throughout the PBAC process will be made available to the LSDP Expert Panel; therefore stakeholders do not need to duplicate responses. A summary of the stakeholder input will be provided to the sponsor along with the advice from the Expert Panel.
Role of the Expert Panel in new medicine applications
All applications for new medicines seeking funding through the LSDP are considered by the Expert Panel. The role of the Panel is to provide advice and assistance to the Commonwealth Chief Medical Officer (CMO) on a range of matters relating to the new medicines seeking funding, including assessment of how the medicine addresses the LSDP Criteria, the guidelines for medicine use and testing requirements, suitable pricing arrangements, and data collection required to inform any future review.
Materials considered by the Expert Panel include the sponsor’s application, assessment (overview) of the submission prepared by the LSDP secretariat, relevant materials from the Pharmaceutical Benefits Advisory Committee (PBAC) consideration (including ratified minutes/advice from PBAC and its sub-committees and responses from sponsors, and stakeholder comments), additional stakeholder input and presentations made to the Expert Panel at the meeting.
Following a meeting of the Expert Panel, sponsors receive the Panel’s advice within two weeks. Sponsors will then have one week to prepare a response.
The Expert Panel
page contains more information on the Expert Panel, including membership, meeting dates and agendas.
The CMO will make a recommendation on the suitability of a medicine for subsidisation through the LSDP within two to six weeks of receiving Expert Panel advice and the Sponsor’s response to this advice. Sponsors will receive verbal confirmation of the CMO’s recommendation, noting that the final decision is at the remit of the Minister for Health. Outcomes will be posted on the LSDP website for each application.
Following a positive recommendation by the CMO and agreement by the Minister for Health, the LSDP Secretariat will work with the sponsor to enter into a Deed of Agreement, and to develop application and treatment guidelines based on the advice of the Expert Panel.
How to apply for patient access to a new medicine
Treating Physicians can commence the application process to obtain access for patients once these documents are finalised. The patient, prescriber and pharmacist
webpage contains more information on patient access to LSDP medicines.
Status of new LSDP medicine applications
Cerliponase alfaOutcome Statement (PDF 672 KB)
Outcome Statement (Word 33 KB)
Application was considered by Expert Panel on 17 October 2018. On 1 May 2019, cerliponase alfa was added to the LSDP.
Status of previous medicine applications
This medicine was considered by the Chief Medical Officer prior to July 2018 when the Expert Panel and other changes were introduced.
Asfotase alfaOutcome Statement (PDF 812 KB)
Outcome Statement (Word 144 KB)