PART 2: How patients can help advance treatment discovery and have their say in the medicine approval processes
Patients are critical to the treatment discovery process from the outset, starting from pre-clinical research through to each stage of clinical trials, all the way through to influencing the approvals processes for subsidised access to new medicines.
Treatment discovery
Pre-clinical research stage
Pre-clinical research, usually conducted on animal models, is a crucial first step before clinical trial testing. However, due to fundamental differences between animals and humans, many promising treatments fail to translate to human use. This has driven growing interest in patient-derived models, which enable drug testing on human tissues in a laboratory setting, offering a more relevant alternative for pre-clinical evaluation.
The development of patient-derived models, such as retinal organoids, requires the donation of blood or skin samples. These models carry the same genetic mutation as the donor and can be used safely and efficiently to study disease mechanisms and test potential treatments for IRDs before proceeding to human trials.
Natural history studies and other research
Natural history studies track the progression of a disease over time without intervention, providing critical data on how a condition develops, varies, and impacts patients. They are essential for understanding disease mechanisms, identifying biomarkers, and designing effective clinical trials, especially for rare diseases such as IRDs. These studies help define baseline progression, select trial participants, and determine meaningful treatment outcomes.
We also need research beyond biology, like studies on economic costs, social and emotional impacts, and the burden on health systems. This broader evidence helps demonstrate the true impact of a condition, and supports advocacy, policy change, and future research funding. Together, natural history and impact studies lay the foundation for smarter trials, more targeted funding, and better treatments.
Participants can include diagnosed individuals, those at risk, caregivers, and sometimes healthy controls.
Clinical trials
New treatment discovery for IRDs is dependent on patient participation in clinical trials also. Clinical trials are used to evaluate the safety and effectiveness of a new procedure, medication or device to prevent, diagnose or treat a disease or disorder.
Taking part in a clinical trial is a meaningful way to contribute to science, so that new treatments can be developed. It is also a way to access potential new treatments at an earlier stage than you otherwise would. However, it is important to remember that clinical trials are experimental, and so the treatment may not necessarily help you. There are also risks associated in being involved in clinical trials. Retina Australia recommends discussion about the pros and cons of participation with your ophthalmologist and the clinical trial research team before you make a decision.
If you are interested in participating in research, you can sign up to an IRD registry (listed on the last page), a research centre, or check for projects on the Australian Clinical Trials website, or Retina Australia’s IRD Research and Clinical Trial Register (summary provided in this newsletter).
Approvals process
The consumer view is incredibly important in the medicine approval process because it ensures that regulatory decisions reflect the real-world experiences and the needs of those who will be using the medicine. Including the patient perspective in decision-making helps ensure that medicines not only meet clinical standards but also genuinely improve patients’ lives in terms of effectiveness, quality of life, and accessibility. Consumers include patients, care givers, family members, potential patients, patient advocacy groups, and the general public. You can have your say!
1. Participate on a TGA Advisory Committee
The TGA does not seek public comment on individual medicine approvals, as its role focuses on assessing safety, quality, and efficacy. Consumer input instead occurs through representation on advisory committees like the Advisory Committee on Medicines (ACM) or TGA Consumer Consultative Committee (CCC).
2. Submit personal evidence to PBAC
Consumers can however, have their say when a medicine is being considered by PBAC for listing on the PBS for medicine cost subsidisation. These comments help decision-makers understand the real-life impact of a condition and the potential benefit of a new treatment, and these are considered during the HTA review process.
PBAC has 3 main cycles per year where it considers applications for funding of new medicines. This is usually March, July and November. It publishes its main meeting agenda 14 weeks before each meeting, which includes the list of applications for new medicines that it will consider at the upcoming meeting.
Consultation is open for 8 weeks. During this period, the current process is for consumers to submit their views via an online survey. Where consumers are unable to access the website, they can write a letter and post it to the Office of Health Technology Assessment, Department of Health Disability and Ageing.
Providing specific details about the reality of living with your condition and how the medicine will positively impact you personally, especially in terms of quality of life, symptom relief, or daily functioning, will provide valuable information for consideration by PBAC.
3. Serve on a PBAC Advisory Committee
Consumers can be involved in the PBAC process as representatives on the PBAC itself, through working groups, or as part of subcommittees. This participation ensures that the patient voice is integrated into decisions regarding medicine approvals and PBS listings. If you are interested in becoming a consumer representative, keep in touch with organisations like the Consumers Health Forum of Australia, which often manages these nominations and advocacy efforts.
4. Collaborate with or join a Consumer Advocacy Group
The Consumers Health Forum of Australia, Rare Voices Australia, and the Patient Voice Initiative actively represent consumers by facilitating, informing, and strengthening patient involvement in HTA and related decision-making processes. These organisations collaborate with consumers and contribute detailed submissions and reports that reflect the patient experience. They also provide guidance and resources to support individuals in contributing directly to the medicine approval and reimbursement process.