Rare Disease: patient access summary
A printable summary of what is publicly known about access to treatment for Rare Disease in Australia: PBS subsidy, Australian trial recruitment, and where the gaps are. Nothing here is new information; it is the same reviewed data as the Rare Disease page, laid out to print. Not medical advice.
Access in Australia
Can you access treatment for Rare Disease in Australia right now? Here's what's publicly known.
- No PBS-subsidised medicines are currently mapped for Rare Disease on Voxsanity. Your doctor or pharmacist can advise on what may be available.
- 11 clinical trials currently recruiting at Australian sites for Rare Disease.
Information on this site does not constitute medical advice. Data is sourced from publicly available government registries. Always consult your doctor before making any health decisions. Trial status should be verified directly with the trial site.
Clinical trial activity
| Trials on record | 997 | This is every trial tagged to this condition at any stage whose registry record agrees it is for this condition, except those withdrawn before starting or terminated early. Most are completed rather than ongoing, so this is a measure of research history, not of current activity. It is also smaller than the sitewide "trials tracked" figure, which does include withdrawn and terminated trials. |
|---|---|---|
| Recruiting now | 242 | This counts only trials whose registry status is "Recruiting" and whose registry record agrees they are for this condition. Trials listed as not yet recruiting or enrolling by invitation are excluded, so this understates how many may open to new participants soon. |
| Recruiting with an AU site | 11 | This counts trials counted under "Recruiting now" that list at least one Australian site. A site being listed does not mean it is currently enrolling, and Australian trials registered only on ANZCTR are not counted yet, so this understates local availability. |
| Phase 3 trials | 13 | This counts trials counted under "Recruiting now" that are at Phase 3. |
The trial catalogue is checked nightly, but individual trial records are refreshed on a rolling basis rather than all at once, so a record may be up to several weeks old. Each trial page and trial card shows the date its own record was last synced. Where a record has lagged, its status, sites and enrolment can differ from the current registry state in either direction, so check the registry link on the trial page before acting on them.
Trials recruiting at Australian sites
Why this list is shorter than "Trials on record". Of the 997 trials on record for Rare Disease, 314 are listed on Voxsanity. The list carries trials we can show a plain English summary for: every trial open or about to open to new participants, plus concluded trials that have published their results. The great majority of the difference is research that finished without publishing a results summary, so there is nothing to read and nothing to join. A smaller part is trials that are open now but whose plain English summary has not been written yet. Nothing is being withheld: the larger figure counts research history, this list counts what is useful to read today.
11 of them are recruiting and list at least one Australian site. A listed site is not proof it is enrolling today; confirm with the trial's own contacts before relying on it.
| Registry ID | Phase | Sponsor | Status |
|---|---|---|---|
| NCT01793168 | Not Applicable | Sanford Health | Recruiting |
| NCT02560883 | Not Applicable | Ohio State University Comprehensive Cancer Cente... | Recruiting |
| NCT05589714 | Not Applicable | Jaeb Center for Health Research | Recruiting |
| NCT05607498 | Phase 1 | EpimAb Biotherapeutics (Suzhou)Co., Ltd. | Recruiting |
| NCT05967689 | Phase 2 | Taiho Oncology, Inc. | Recruiting |
| NCT06016946 | Not Applicable | Friedreich's Ataxia Research Alliance | Recruiting |
| NCT06706076 | Phase 2 | BlossomHill Therapeutics | Recruiting |
| NCT07039084 | Not Applicable | Murdoch Childrens Research Institute | Recruiting |
| NCT07070232 | Phase 2 | BioNTech SE | Recruiting |
| NCT07185997 | Phase 3 | ArriVent BioPharma, Inc. | Recruiting |
| NCT07746895 | Phase 4 | Swedish Orphan Biovitrum | Recruiting |
Full eligibility for each trial is at voxsanity.com.au/trials/<registry ID>/, and on the registry itself: clinicaltrials.gov, or isrctn.com for an ISRCTN ID.
What this summary does not cover
- Australian registration dates. Voxsanity holds no TGA registration record for any medicine, so no Australian registration date appears here or anywhere on this site. The TGA's own Australian Register of Therapeutic Goods (tga.gov.au/resources/artg) is the authoritative place to check. Where a medicine is on the PBS it must already be registered for supply in Australia, because a PBS listing cannot happen before that: that is read from the PBS, not from the TGA.
- Access outside the PBS and outside a trial. Where a medicine is not registered or not available through a trial, the TGA Special Access Scheme may allow a doctor to apply for access for an individual patient. A plain English explainer is at voxsanity.com.au/sas-navigator/.
- Whether any of it is appropriate. Nothing on this sheet is a recommendation. A medicine being subsidised, or a trial recruiting nearby, says nothing about whether either suits a particular person. That is a clinical judgement this sheet cannot make.
Not medical advice. Voxsanity republishes public government health data in plain English and does not provide medical advice. Always consult a qualified health professional before making any health decision. Trial status should be verified directly with the trial site. Sources: ClinicalTrials.gov and other public trial registries.