Understanding Research
Researchers around the world are currently working on macular diseases to find reasons for these diseases and the answers needed. There are many ongoing clinical trials and the Foundation is proud of the many brilliant and dedicated Australian researchers who form part of this international team.
Since 2011 Macular Disease Foundation Australia has committed $2.8 million to its Research Grants Program which directly supports the work of world-leading Australian researchers who aim to reduce the impact and incidence of macular degeneration and ultimately, find a cure.
You can support this research by making a donation to Macular Disease Foundation Australia today.
Clinical trials
Clinical trials are studies in humans, which aim to find a better way to manage a particular disease. They aim to establish: correct dosage, safety, efficacy (how well it works), interactions with other drugs, comparisons to other treatments, cost effectiveness and use in specific medical situations. Trials are designed in a way that minimise the possibility of bias or incorrect conclusions.
Medical Research Phases
There are several medical research phases that must be completed before a new drug can be registered by Australia’s Therapeutic Goods Administration. Note that the duration of each phase is an approximate one and can be much longer than stated below.
|
Research Phase |
Patients Studied |
What Studied |
Duration |
|
Discovery and development |
Usually laboratory work |
What causes a disease; identify targets (eg identify a ‘key’ that turns off an unwanted process) |
Many years |
|
Pre-clinical |
Animals or cell cultures |
Proof of principle, safety in animals, safe starting dose, toxicity |
4 years |
|
Phase 1 |
20 to 80 volunteers |
Safety and dosing |
1 – 2 years |
|
Phase 2 |
100 to 300 volunteers with disease |
Initial efficacy, dosing, larger scale safety |
2 years |
|
Phase 3 |
500 to 3000 volunteers with disease |
Detailed efficacy, safety, comparison to other treatments |
3 years |
|
Registration and reimbursement |
|
Regulators review studies and detailed manufacturing dossier to decide if treatment should be registered and subsidised |
18-24 months |
|
Phase 4 |
Consenting patients using the test treatment once launched |
Long term safety and efficacy |
Ongoing |
Drug approval for use in Australia for safety and efficacy
Once a manufacturer has completed the pre-clinical and phase 1 to 3 clinical studies for a new treatment, the Therapeutic Goods Administration (TGA) reviews vast amounts of data on how the research was conducted, and its findings. The TGA also reviews information about the manufacturing process to ensure that drugs are manufactured to specification. Only after the TGA is satisfied that the treatment has an acceptable safety profile and is effective, can it be registered for use in Australia.
Making drugs affordable
Following registration, the Pharmaceutical Benefits Advisory Committee (PBAC) reviews additional data regarding the safety, efficacy (effectiveness) and cost-effectiveness of a new treatment to decide whether it should receive a government subsidy and be placed onto the Pharmaceutical Benefits Scheme (PBS).
Once a treatment is placed on the PBS, the patient will only pay a part of the actual cost of the drug, with the rest being subsidised by the taxpayer.