Published by PeptideFactSheets. Sources and methods · Research reporting, not clinical review.
Read five real evidence examples
1. Completed does not mean results posted
TRIUMPH-1 is marked completed in its registry, but the checked record has no posted results. That says where results are available, not whether results exist in a paper or company announcement. Recruitment, phase, publication and approval need separate checks. TRIUMPH-1 registry record (checked 2026-09-18).
2. A biomarker can change while function does not
In the 20-person NR pilot, blood NAD+ rose but cognition did not change during 10 weeks. This result supports target engagement; it does not show dementia prevention. Exploratory findings also face false-positive risk when many measures are tested. Nicotinamide riboside pilot in mild cognitive impairment.
- 1. Biological mechanism
Can a pathway be affected? - 2. Measured biomarker
Did the measured signal change? - 3. Patient outcome
Did people feel, function or survive better?
A result in one box does not establish the next. This is a conceptual diagram, not measured trial data.
3. Absolute difference and hazard ratio answer different questions
SELECT reported primary events in 6.5% versus 8.0% of participants. Subtracting those rounded figures gives 1.5 percentage points. The reported hazard ratio was 0.80 (95% confidence interval 0.72–0.90); it incorporates event timing and is not the same calculation as dividing those percentages. These are trial averages over a mean 39.8-month follow-up, not an individual forecast. SELECT: cardiovascular outcomes without diabetes.
4. Approval may still require confirmation of patient benefit
Forzinity received accelerated approval for a narrow Barth syndrome population using knee extensor strength as a measure reasonably likely to predict benefit. FDA requires a confirmatory trial of patient benefit. Neither this decision nor a trial phase establishes benefit for unrelated conditions. FDA accelerated approval: Forzinity for Barth syndrome.
5. Small, short studies leave large safety gaps
The MIB-626 NMN study enrolled 30 adults for 28 days. Similar adverse-event reporting between groups over that period cannot rule out rare events or establish long-term safety. Its measured physical-performance outcomes did not differ significantly. MIB-626: NAD augmentation in adults.
Try it yourself
Five questions to sharpen your research reading
Choose the interpretation the evidence supports. You can change an answer, read the explanation, or start again. Nothing is saved or sent anywhere.
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Check your interpretation
Ask: Is this a registered plan or a result? Which outcome was primary? What population was enrolled? How long were people observed? Is the comparison direct? Does the conclusion go beyond what was measured? A missing field should remain unknown.
Preclinical research
Before broad human testing, researchers may study a compound in cells, tissues, or animals. This can reveal mechanisms and obvious hazards, but it cannot prove a treatment works safely in people.
Phase 1: first questions in people
Phase 1 generally focuses on safety, tolerability, and what the body does with a drug. These studies are often small. They may include healthy volunteers or people with a condition, depending on the research.
Phase 2: does the signal hold up?
Phase 2 studies explore effectiveness, dose selection, and continued safety in people with the target condition. Encouraging Phase 2 results still need confirmation.
Phase 3: larger confirmation
Phase 3 trials are usually larger and designed to confirm benefits and characterize harms in the population intended for a potential label. A Phase 3 trial can fail, be delayed, or answer only a narrow question.
FDA review
A sponsor submits a formal application with clinical, manufacturing, and labeling information. FDA review is separate from trial registration and from a company announcing topline results.
Approval versus off-label use
Approval covers the product and uses described in its label. Licensed clinicians may sometimes prescribe an approved medicine off-label, but that does not make the off-label use FDA approved. This site does not recommend treatment choices.
Why early results can be misleading
- Small studies can exaggerate effects by chance.
- A press release may arrive before peer review or complete data.
- Dropouts and missing data can change the story.
- A surrogate endpoint may not predict how people feel or function.
- Rare harms often require larger or longer studies to detect.
How to read a trial update
- Check whether the result is registered, complete, and peer reviewed.
- Look for the primary endpoint—not only favorable secondary findings.
- Compare absolute results, adverse events, and discontinuations.
- Ask whether the comparison group and population fit the claim.
- Treat estimated completion dates as estimates, not promises.
Sources for this guide
These official and educational sources support the general regulatory and research concepts explained on this page. For a specific medicine, use the fact sheet's product-level sources.
- 1fdaFDA: the drug development process
Official overview of preclinical research, clinical research, FDA review, and post-market monitoring.
- 2clinical trialsClinicalTrials.gov: how to read a study record
Explains how to interpret recruitment status, endpoints, enrollment, results, and record history.
- 3nihNIH: clinical trials and you — the basics
Plain-English NIH overview of trial phases, participant protections, and what each phase can answer.
- 4nihNIH: understanding clinical studies
Explains why study design, comparators, and endpoints matter when interpreting findings.