Uncertainty to certainty: mito clinical trials panel

Clinical Trial Phases and Drug Development Funnel

• The conversation is about clinical trials, and the process of developing medicines can be thought of as a funnel, with many compounds being worked on in laboratories, but only a few resulting in effective therapies 01:16.
• Clinical trials have different phases, including phase one, which focuses on safety, phase two, which collects evidence on whether the therapy works, and phase three, which is about collecting information on whether the treatment is effective 02:29.

Global Disparities in Clinical Trial Availability

• Mito Foundation has launched a clinical trials action plan to increase clinical trials in Australia, as currently, many trials are only available in the US and Europe, and Australians have had access to very few of them 04:20.
• Clinical trials for mito treatments often begin in the US due to its clinical infrastructure, mito population, and regulator support 10:47

US Clinical Trial Infrastructure and Ecosystem

• The US has a well-established ecosystem with patient advocacy networks, registries, and clinics with specialized care, allowing for efficient clinical trials 11:33
• When selecting sites for clinical trials, factors such as expertise, collaboration, infrastructure, and patient population are considered, with the goal of finding sites that can support the study’s requirements 14:16

Key Stakeholders and Clinician Roles in Trials

• Various parties are involved in clinical trials, including regulators like the Therapeutic Goods Administration, ethics committees, sponsor companies, contract research organizations, and healthcare professionals, all working together to ensure the trial’s success 18:01
• Clinician researchers, like Dr Sebastian Lush, play a crucial role in delivering clinical trials and making decisions about which trials to get involved with, based on factors like resources, expertise, and the potential impact of the trial 21:54

Clinician Responsibilities and Investigator-Led Trials

• Clinicians need to evaluate the science behind a product or molecule and understand the safety and potential risks to patients, as well as explain these risks to patients with confidence 23:04.
• Investigator trials are led by clinicians or clinician scientists who have a question about a disease or intervention, and these trials can be different from clinical trials run by medicines companies, often focusing on academic questions 24:46.

Patient Participation in Clinical Trials
When considering participation in a clinical trial, patients should approach their regular doctor to discuss eligibility and understand the requirements and potential risks, as well as the differences between clinical trials and regular clinical care 27:55.

Drug Approval Process and Post-Trial Steps

• The medical director of Biogen has a clinical background in nursing and stroke medicine, and is responsible for a medical team that explains how their drugs work to doctors, while also ensuring conversations are based on science and obeying regulations like the TGA 31:38.
• Clinical trials measure whether a drug improves symptoms, increases lifespan, and has acceptable side effects, with researchers choosing important factors to measure and considering the time required for the trial, which can take one to two years 34:44.
• After a clinical trial is completed, the results are submitted to the TGA for approval, and if approved, the drug is then considered for reimbursement, with the government evaluating its cost-effectiveness and potential impact on the budget, a process that typically takes 12 months 39:10.

Australia’s Regulatory Delays and Patient Advocacy

• Access to therapies in Australia takes almost one and a half years compared to other countries, with a lot of back and forth between pharmaceutical companies and the PBAC 42:26
• Patient comments are welcomed by the PBAC three times a year, allowing individuals to contribute to the decision-making process as experts on their disease 42:44
• The mito community has previously successfully contributed to the approval of genomic testing for mitochondrial disease, led by Professor Carolyn Sue, and hopes to have similar conversations with PBAC in the future 43:30