HOW IS A CANCER MEDICINE APPROVED FOR USE IN AUSTRALIA?
- Australian law requires all medicines marketed for human use to be registered by the TGA. Medicines approved for registration are listed on the Australian Register of Therapeutic Goods ARTG):https://www.tga.gov.au/australianregister-therapeutic-goods
- The TGA applies scientific and clinical expertise to assess evidence of the benefits and risks of therapeutic products.
- For a medicine to be registered by the TGA, it must have a sponsor which is usually the patent holder. The sponsor’s application includes information about a product’s safety, efficacy and quality.
- The process for registration and feedback from the TGA, through to the announcement of a registration for successful applications, can take up to 12 months and is managed through a number of milestones. You can view the schedule on the TGA website: http://www.tga.gov.au/ prescription-medicines-registration-process
- More information about the TGA and its processes can be found at http://www.tga.gov.au
WHAT IS THE PBS?
- The PBS subsidises the cost of medicines for the community. It is an Australian Government program administered by the Department of Health: http://www.pbs.gov.au/pbs/home
How do Medicines get approved for the PBS?
- PBAC is an independent, expert statutory body which reviews submissions from the sponsors of new medicines (generally pharmaceutical companies) for PBS subsidy:http://www.pbs.gov.au/info/industry/listing/participants/pbac
- PBAC makes recommendations to the Government about whether to subsidise new medicines. The PBAC’s recommendations inform the Government’s decision about which medicines to list on the PBS.
- The submission process to PBAC takes 17 weeks from the time of submission to the consideration of the submission by PBAC
- Following this, the post PBAC process can include price negotiations, cabinet approval or resubmission.
- The PBAC may reject an application or choose to make specific recommendations regarding medicine’s uses and any conditions or restrictions on those uses.
- The Health Minister cannot add a medicine to the PBS without the PBAC’s recommendation. A medicine must be approved by the TGA for a specific use (indication) before it can be added to the PBS
WHAT IS THE APPLICATION PROCESS FOR GETTING A NEW CANCER MEDICINE ON THE PBS?
- The PBAC meets three times a year in March, July and November to review submissions to list new medicines on the PBS.
- To lodge a submission, pharmaceutical companies must follow strict guidelines. The PBAC evaluates submissions according to the clinical benefit and cost-effectiveness of a new medicine when compared to existing medicines or products for the same condition.
- The PBAC process is well defined and can be found on the PBAC website: http://www.pbs.gov.au/info/industry/useful-resources/pbscalendar
The key steps are:
- The company (sponsor) makes a submission for the PBAC’s review four months prior to the PBAC meeting.
- The PBAC agenda is published 10 weeks prior to the meeting.
- Members of the public and healthcare professionals can submit comments about a submission to the PBAC from the date the PBAC agenda is published until one month prior to the meeting.
- PBAC recommendations are published six weeks after the meeting: http://www.pbs.gov.au/pbs/home
- If the PBAC rejects a submission, it will explain why and the sponsor may resubmit, if and when it believes it can address the PBAC’s concerns.
Under special circumstances, the Government may agree to subsidise the cost of a medicine on a provisional basis. This process is known as the “managed access program”. PBAC can agree to a managed access program for medicines:
- Where patients have a high and urgent clinical need.
- There is enough evidence to show the benefit of the medicine, but not enough to demonstrate cost-effectiveness.
- Where the sponsor will be able to provide more evidence in a reasonable time period so the PBAC can undertake a full assessment of its value.
- The managed access program does not guarantee that a medicine will be listed on the PBS indefinitely. The PBAC may recommend “de‑listing” based on its assessment of further evidence about the efficacy and safety of the medicine.
DO PATIENTS HAVE A SAY IN THE PBAC REVIEW PROCESS?
- Anyone interested in a particular medicine and the decision on whether it should be subsidised (listed on the PBS) can have their voice heard. This includes those living with a disease, a loved one who has been impacted by the disease, members of the public, carers, family members, healthcare professionals and patient or consumer groups.
- Consumer involvement allows the submission to demonstrate how the availability or unavailability of a drug can impact an individual’s quality of life.
What is it like to have lymphoma?
How does it impact on everyday life?
What are the effects of the new drug? Has this changed your life?
Are you able to access this treatment now? Why is it important to have access?
WHAT HAPPENS AFTER THE PBAC MAKES IT RECOMMENDATIONS?
- The Health Minister (or delegate) considers the PBAC’s recommendations and the Health Department’s advice on the cost to the PBS which is developed following negotiations with the sponsor.
- The decision to subsidise a medicine goes to Cabinet if the cost is expected to add more than $20 million a year to the PBS. If Cabinet approves the subsidy, the decision on when to add the medicine to the PBS is made by the Health Minister.
- The timing for the Cabinet or Ministerial review can vary. Typically, the Cabinet or Minister makes a decision within six months of the PBAC recommendation.
HOW CAN A PATIENT GET ACCESS TO A MEDICINE PRIOR TO REGISTRATION OR SUBSIDY?
- Prior to approval by the TGA, most medicines are not available to patients.
- In certain cases, there is a regulatory process– known as a Special Access Scheme – which enables doctors to request an unapproved medicine on behalf of their patients once the patient has exhausted all other approved treatment options. More information can be found here: https://www.tga.gov.au/form/special-access-scheme
- Some pharmaceutical companies also offer Patient Access Programs for medicines that are approved by the TGA before they are listed on the PBS. In these programs, patient eligibility is determined by the company and there may be accost to patients.
- Only doctors can request medicines on behalf of their patients. Pharmaceutical companies in Australia are guided by a Code of Conduct and are limited in what information they can share directly with patients about these programs.
If you think there is a treatment that could help you, don’t be afraid to discuss this with your doctor.
4 PBAC Minutes for the Managed Access Program Framework - March 2015, Accessed on 7 August 2015,
Available at: http://www.pbs.gov.au/info/news/2015/07/pbac-minutes-for-the-managed-access-program-framework/