Phase III

Rare & Genetic

pain 88#7volume 884#28

Pain rank 7 of 76, volume rank 28 of 76 by industry trials. Trials here amend about 1.5x and fail enrollment about 1.4x 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 61 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 $27M at stake per 100 trials. Across the roughly 884 industry trials here, the floor is $242M.

If amendments fell by10%$24M30%$73M50%$121M

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 A trial counts as concluded once it has finished, whether completed, terminated or withdrawn.

Industry sponsors88pain index
Eligibility amendments
61%vs map 40%proxyn = 46sampled
Enrollment failure
18%vs map 13%n = 634concluded
Startup lag
81 dvs map 45.5 dn = 609with dates
Median duration
28.4 mon = 859completed
All sponsors85pain index
Eligibility amendments
49%proxyn = 91sampled
Enrollment failure
19%n = 818concluded
Startup lag
83 dn = 751with dates
Median duration
29.7 mon = 1,113completed
Why these trials stoppedsponsor decision leads

The most common category here is sponsor decision (32 of 116 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.

Sponsor decision32 (28%)
Efficacy or futility24 (21%)
Low accrual22 (19%)
Safety or tolerability11 (9%)
Not reported8 (7%)
External or regulatory5 (4%)
Business or funding4 (3%)
No enrollment recorded (inferred)inferred3 (3%)
Feasibility or operational failure3 (3%)
Logistics or operations2 (2%)
Never initiated1 (1%)
Other stated reason1 (1%)
n = 116terminated or withdrawn
Evidence12 trials

A sample of terminated or withdrawn trials in this cell. Each links to its ClinicalTrials.gov record.

TrialSponsorStatusStated reason
NCT07197034Aardvark Therapeutics, Inc.terminatedExternal or regulatory
NCT06828861Aardvark Therapeutics, Inc.terminatedExternal or regulatory
NCT06664853Quince Therapeutics S.p.A.terminatedSponsor decision
NCT06422377TakedaterminatedSafety or tolerability
NCT06121375Intercept PharmaceuticalsterminatedExternal or regulatory
NCT05815004AVROBIOwithdrawnSponsor decision
NCT05764239SynlogicterminatedLow accrual
NCT05689164PfizerterminatedLow accrual
NCT05676853Aeglea BiotherapeuticsterminatedSponsor decision
NCT05655520Supernus Pharmaceuticals, Inc.terminatedSponsor decision
NCT05604170Marinus PharmaceuticalsterminatedSponsor decision
NCT05494619Hoffmann-La RochewithdrawnSafety or tolerability

Rare & Genetic at other phases