Q（Question）

When a physician or pharmacist requests clinical trial data on an unapproved drug, off-label drug, or dosage not approved domestically, what information may be provided?

A（MHLW answer）

Same as A1.

However, the portion relating to case reports is excluded.

Items requiring particular attention: (4), (5), (6)

So what (meaning): Clinical trial data for unapproved/off-label use may be provided under the same conditions as Q1, but the case-report provision of Q1 does not apply here. Scientific basis and trial methodology must be clearly presented.

So why (rationale): Clinical trial data has undergone controlled-study procedures unlike case reports, so the caveat about selecting case reports non-arbitrarily and declaring limited evidence does not need to be applied separately.

Commentary — background, application, practical notes

Clinical trial data is structurally different from case reports — it is collected through scientific procedures such as randomized controlled trials and dose-escalation studies. Accordingly, the Q1 provision that case reports must be accompanied by an explicit statement of limited evidence does not apply to clinical trial data, and Q4 explicitly excludes that clause. However, the prohibitions against arbitrary selection (provision (5)) and omission of safety information (provision (6)) apply equally to trial data.

Typical scenarios include a physician at a clinical trial site requesting information on interim results of an ongoing trial for a new indication, or an inquiry about investigator-initiated trial data relevant to a planned supplemental approval. In such cases, information registered and publicly accessible in JRCT or ClinicalTrials.gov may be provided; unpublished internal analyses are outside the scope of permissible provision.

The most common practical error is providing favorable interim analysis results while withholding final data in which the primary endpoint was not met. The Q1 principle that negative information must also be provided applies equally to trial data — selectively presenting only successful outcomes from a specific trial is not permissible. Similarly, supplementing publicly registered trial information with internal analysis not yet disclosed — even when framed as 'still under internal review' — is prohibited.