How drug research actually reaches patients
From the first laboratory grant to the day your GP can prescribe it. In plain English.
Only 9% of Americans have ever been invited to take part in a clinical trial.
Treatments are usually further along than patients realise. The science is often years ahead of the pharmacy shelf. Plenty is being done; the trouble is that the pipeline sits in government databases written for specialists. Voxsanity reads that data for you, for your condition.
Source: Williams CP et al., Demographic and Health Behavior Factors Associated With Clinical Trial Invitation and Participation in the United States, JAMA Network Open, 2021 (doi:10.1001/jamanetworkopen.2021.27792): 439 of 3,689 US adults surveyed (9%) had been invited to a clinical trial. Also reported by the U.S. Department of Health & Human Services, Office of the Assistant Secretary for Planning and Evaluation (ASPE), Empowering Patients through Clinical Trials (PDF), 2025.
The five stages, and what each one means for you
Before anyone tests a drug in a human, governments fund basic science. The US National Institutes of Health invests billions each year into conditions like yours. When that funding grows, it is an early signal that more treatments are coming, though it takes years to show up as trials.
For Spinal Muscular Atrophy, tracked US NIH funding totals US$177 million. This reflects the whole condition, not Risdiplam specifically. It is early money into the field that treatments like Risdiplam were eventually built on.
Researchers publish findings in peer-reviewed journals. When multiple groups publish promising results on the same target, drug companies take notice and invest in trials. This is where scientific consensus begins to form around a potential treatment.
Years of published research across many groups built the scientific case for treating Spinal Muscular Atrophy, the groundwork any specific therapy, including Risdiplam, is built on. That history is well documented in the literature; Voxsanity simply does not currently count it.
Trials run in three phases. Phase 1 tests safety in a small group. Phase 2 tests whether it works. Phase 3 compares the new treatment to the current standard of care at scale. This is the last hurdle before a regulator will consider approval. About 65% of drugs that reach Phase 3 eventually get approved.
Spinal Muscular Atrophy currently has 42 Phase 3 trials tracked (a condition-level count). Risdiplam itself worked through Phase 1 to Phase 3 before any regulator approved it.
After a successful Phase 3 trial, the company submits all data to regulators. In Australia the TGA reviews the evidence and decides whether to approve the drug. The FDA in the US and EMA in Europe often approve first, and those decisions usually predict what is coming here within months.
Risdiplam (brand Evrysdi) received US FDA approval in August 2020, a real, dated milestone. Australian TGA registration followed; we don't display a drug-specific TGA date where our source data doesn't carry one.
TGA approval means a drug is safe and effective. PBS listing means Australians can actually afford it. The Pharmaceutical Benefits Scheme subsidises approved drugs, but listing is a separate process that can take an additional 1 to 3 years after TGA approval. Without it, a drug can cost tens of thousands of dollars a year out of pocket.
Risdiplam was listed on the PBS in August 2021, 1 year after its FDA approval. Both dates are real. That gap is exactly the distance Voxsanity helps you see and plan around.
A real, live example · rare disease
Risdiplam for Spinal Muscular Atrophy
Approved by the US FDA in August 2020, but not listed on Australia's PBS until August 2021: a wait of 1 year for affordable Australian access after the first major approval.
This example is chosen automatically from current data (a recently PBS-listed medicine with a traceable approval history) and refreshes as the data updates. FDA and PBS dates are drug-specific; funding and research figures shown in the timeline above are condition-level context.
Voxsanity currently covers 103 conditions. We are expanding coverage regularly.
Not medical advice. Information on this site does not constitute medical advice. Data is sourced from publicly available government registries including ClinicalTrials.gov, the FDA, the NIH and the PBS. Trial and listing status can change — always verify current status directly with the source before making any decision, and confirm Australian availability with your doctor.