Sample questions

a) In microdose Approach 1, the total dose is not more than 100 µg and is also ≤ 1/100th of the NOAEL.

b) In microdose Approach 2, a subject may receive up to 10 administrations of 100 µg each.

c) Approach 2 requires a washout of 6 or more actual or predicted half-lives between doses.

d) Approach 1 is supported by an extended single-dose toxicity study in one species, usually rodent.

e) Microdose trials are useful for PET receptor-binding and tissue-distribution studies and for PK assessment.

a) Critical to quality factors are study attributes whose integrity is fundamental to participant protection and to the reliability of results and decisions.

b) Identifying critical to quality factors is mainly the role of the investigator site once the study has started.

c) Risks to critical to quality factors are accepted or mitigated according to their probability, detectability and impact.

d) Collecting as much data as possible is encouraged, to protect study quality.

e) Consulting patients early in study design can help identify critical to quality factors.

a) Cardiovascular system.

b) Central nervous system.

c) Respiratory system.

d) Renal system.

e) Gastrointestinal system.

a) Long-term testing may be at 25°C ± 2°C / 60% RH ± 5% RH or 30°C ± 2°C / 65% RH ± 5% RH.

b) Accelerated testing is at 40°C ± 2°C / 75% RH ± 5% RH.

c) At least 6 months of accelerated data are needed at submission.

d) At submission, long-term data must cover at least 24 months on one primary batch.

e) In the first year, long-term testing should normally be every 3 months.

a) The threshold level of regulatory concern is around 5 ms.

b) This threshold is evidenced by an upper bound of the 95% confidence interval around the mean effect on QTc of 10 ms.

c) It is typically conducted in patients at increased risk of arrhythmia.

d) It is intended to identify drugs as pro-arrhythmic.

e) It should include randomisation, blinding and a concurrent placebo control.

a) About 1500 individuals in total are typically expected to be exposed.

b) 300-600 patients should have been treated for 6 months.

c) 100 patients should have been exposed for at least one year.

d) These numbers apply equally to life-threatening indications.

e) If no serious ADE is seen in 100 patients exposed for one year, the true cumulative one-year incidence is reasonably assured to be no greater than 3%.

a) Assay sensitivity is a trial's ability to distinguish an effective treatment from a less effective or ineffective one.

b) A successful non-inferiority trial directly shows that the trial had assay sensitivity.

c) A non-inferiority trial without assay sensitivity may find an ineffective treatment non-inferior.

d) In a non-inferiority trial, assay sensitivity is inferred from historical evidence of sensitivity to drug effects plus appropriate trial conduct.

e) A baseline-controlled study, comparing patients' status on therapy with their status before it, has a valid internal control.

a) It should have at least five members.

b) At least one member's primary area of interest should be non-scientific.

c) At least one member should be independent of the institution/trial site.

d) All members must be physicians.

e) The investigator should notify the sponsor of all SAEs within 30 days, unless the protocol exempts them.

a) The sponsor should determine the extent and nature of monitoring based on identified risks.

b) Monitoring should be performed by persons not involved in the clinical conduct of the trial at the site being monitored.

c) Centralised monitoring may be used on its own, without site monitoring.

d) 100% source data verification at every site is required.

e) Quality tolerance limits are pre-specified ranges whose exceedance should prompt evaluation for a possible systemic issue.

a) Placebo is acceptable where no proven intervention exists.

b) Placebo may be used for compelling and sound methodological reasons even if participants risk serious or irreversible harm.

c) Post-trial provisions must be arranged before the trial begins.

d) Research must be registered in a publicly accessible database before recruitment of the first participant.

e) Only positive results need be published.

a) Consent may be obtained remotely where appropriate, but the investigator must verify the participant's identity.

b) When consent is collected electronically, participants may be offered a paper-based alternative.

c) Revised consent materials with new safety information may be used straight away and sent to the IRB/IEC afterwards.

d) Consent information may include a clause releasing the sponsor from liability for negligence, if the participant agrees to it.

e) By signing the consent form, the person who held the consent discussion attests that consent was freely given and the information was apparently understood.

a) The intention-to-treat principle implies that the primary analysis should include all randomised subjects.

b) Failure to take at least one dose of trial medication may justify exclusion from the full analysis set.

c) In an active-control equivalence trial, the full analysis set may be biased towards demonstrating equivalence.

d) A non-significant test of no difference is adequate evidence of non-inferiority.

e) Equivalence trials should be analysed with two-sided confidence intervals.

a) Monitoring trial quality (protocol adherence, accrual, retention) needs no access to comparative treatment effects and has no impact on type I error.

b) An interim analysis needs unblinded access to treatment group assignment and comparative summaries.

c) The statistical plan for an interim analysis may be added after the first unblinded look if it is fully documented.

d) Changes to entry criteria during recruitment should be made without breaking the blind and described by a protocol amendment.

e) If accrual falls well below the projected level, the reasons should be found and remedial action taken to protect the trial's power.

a) The estimand attributes are treatment, population, variable (endpoint), handling of intercurrent events and a population-level summary.

b) Under the treatment policy strategy, the variable of interest is used whether or not the intercurrent event occurs.

c) The treatment policy strategy works well when the intercurrent event is death.

d) The same strategy must be used for every intercurrent event in a trial.

e) Progression-free survival is an example of a composite variable strategy.

a) Under Reg. (EC) 1901/2006 Art. 36, submitting results that comply with an agreed PIP can earn a 6-month extension of the supplementary protection certificate.

b) For orphan medicinal products, the reward is instead an extension of orphan market exclusivity from 10 to 12 years (Art. 37).

c) Orphan designation under Reg. (EC) 141/2000 Art. 3 requires a prevalence of no more than 5 in 10,000 in the EU (or the alternative return-on-investment criterion).

d) Orphan designation is granted by the national competent authority of each member state.

e) The Paediatric Committee (PDCO) adopts an opinion on a valid proposed PIP within 60 days; the decision agreeing the PIP is the Agency's.

a) A generic application can rely on the reference product's data once the reference has been authorised for at least 8 years.

b) A generic may be placed on the market as soon as it is authorised, at the 8-year point.

c) The 10-year period can extend to at most 11 years if, within the first 8 years, a new indication with significant clinical benefit is authorised.

d) A marketing authorisation is first valid for 5 years; after renewal it is normally valid for an unlimited period.

e) The renewal dossier must be submitted at least 3 months before the authorisation expires.

a) A fatal, unexpected ADR should be notified no later than 7 calendar days after first knowledge by the sponsor.

b) That notification should be followed by as complete a report as possible within 8 additional calendar days.

c) Serious, unexpected ADRs that are not fatal or life-threatening must be filed within 15 calendar days.

d) Serious but expected reactions ordinarily require expedited reporting.

e) An identifiable reporting source is one of the minimum criteria for an initial report.

a) The DSUR annual period starts from the Development International Birth Date (DIBD): the sponsor's first authorisation to conduct a clinical trial in any country worldwide.

b) A separate DSUR should normally be prepared for each indication of the investigational drug.

c) The DSUR should be submitted no later than 60 calendar days after the data lock point.

d) When a trial starts in a second country, a new DIBD is set for that country.

e) Once a product is marketed and clinical development continues, the DSUR is replaced by the PSUR.

a) The Safety Specification summarises important identified risks, important potential risks and important missing information.

b) Every new product needs additional pharmacovigilance actions, such as post-approval safety studies.

c) Children, the elderly, and pregnant or lactating women are among the populations the Specification should consider as not studied or studied only to a limited degree.

d) A Safety Specification and Pharmacovigilance Plan can be prepared for a product already on the market, for example for a new indication.

e) The guideline recommends that pharmacovigilance experts get involved only once the marketing application has been submitted.

a) Promotional material must be certified in its final form by one UK-registered medical practitioner or pharmacist (or dentist, for dental-only products).

b) The person who drew up the material may also certify it, if they are a registered medical practitioner.

c) Material still in use must be recertified at intervals of no more than two years.

d) In printed promotional material, prescribing information may be given through a clear and prominent QR code.

e) Putting a separate SmPC in the same envelope as a promotional letter is enough to meet the prescribing-information requirement.

a) Since April 2026, NICE technology appraisals use a standard threshold of £25,000–£35,000 per QALY gained.

b) The previous standard range was £20,000–£30,000 per QALY.

c) The new standard threshold also applies to highly specialised technologies (HST).

d) Under the new threshold, NICE routinely re-evaluates medicines it rejected earlier on cost grounds.

e) The increase came from a government (DHSC) direction to NICE, not a NICE methods review.