Phase I
Rare & Genetic
Pain rank 54 of 76, volume rank 55 of 76 by industry trials. Trials here amend about 0.8x and fail enrollment about 1.0x the map median.
What this is worth
The amendment proxy, priced. Order of magnitude, anchor cited, assumptions on the table.
Per 100 industry trials in this cell, about 32 rewrite their eligibility criteria at least once. At the industry average of $450k direct cost per amendment, with the 6+ weeks of delay each not even counted, that is at least $14M at stake per 100 trials. Across the roughly 397 industry trials here, the floor is $57M.
Assumes $450k direct cost per amendment (industry average; Phase II median runs lower, Phase III higher, and delay cost is excluded), applied to the proxy rate. The proxy counts only trials with at least one eligibility change, so this understates the true exposure. How this is derived.
The signals
Industry sponsors first, since that is the commercial buyer, each figure shown against the map median. All-sponsor figures follow. A trial counts as concluded once it has finished, whether completed, terminated or withdrawn.
- Eligibility amendments
- 32%vs map 40%proxyn = 41sampled
- Enrollment failure
- 14%vs map 13%n = 333concluded
- Startup lag
- 37 dvs map 45.5 dn = 237with dates
- Median duration
- 10.0 mon = 392completed
- Eligibility amendments
- 30%proxyn = 86sampled
- Enrollment failure
- 16%n = 597concluded
- Startup lag
- 57 dn = 420with dates
- Median duration
- 18.7 mon = 745completed
Why these trials stoppedsponsor decision leads
The most common category here is sponsor decision (17 of 45 stopped). A keyword pass runs first; free text it could not place was sorted once by a frozen AI classification into these same categories, auditable rather than a live model. "No enrollment recorded (inferred)" is not a stated reason: it is read from a zero enrollment count, meaning the trial never enrolled anyone. "Not reported" means the sponsor left the field blank, shown rather than hidden.
Evidence12 trials
A sample of terminated or withdrawn trials in this cell. Each links to its ClinicalTrials.gov record.
| Trial | Sponsor | Status | Stated reason |
|---|---|---|---|
| NCT07255846 | Terremoto Biosciences Inc. | withdrawn | Low accrual |
| NCT06747273 | Sarepta Therapeutics, Inc. | terminated | Sponsor decision |
| NCT06681766 | Larimar Therapeutics, Inc. | terminated | Low accrual |
| NCT06597656 | Sarepta Therapeutics, Inc. | terminated | Sponsor decision |
| NCT06241950 | Sarepta Therapeutics, Inc. | terminated | Sponsor decision |
| NCT05906251 | Sarepta Therapeutics, Inc. | terminated | Sponsor decision |
| NCT05813314 | BioMarin Pharmaceutical | terminated | Safety or tolerability |
| NCT05692908 | Sorrento Therapeutics, Inc. | withdrawn | Business or funding |
| NCT05238324 | Homology Medicines, Inc | withdrawn | Sponsor decision |
| NCT05222178 | Homology Medicines, Inc | terminated | Sponsor decision |
| NCT05160558 | Biogen | terminated | Sponsor decision |
| NCT05095246 | Krystal Biotech, Inc. | withdrawn | Low accrual |