For regulatory and clinical development teams, securing PRO language in approved prescribing information represents one of the most consequential—and contested—outcomes of the FDA review process. Whether a patient-reported endpoint reaches Section 14 labeling determines how a product's symptom benefit can be communicated to prescribers, payers, and health technology assessment bodies, making early endpoint selection and qualification strategy a critical cross-functional decision.
This analysis examines how CDER has incorporated patient-reported outcomes into approved prescribing information over the five-year window from mid-2021 through mid-2026, drawing on Drugs@FDA label texts, approval letters, and review documents. It covers the therapeutic areas and PRO instruments most frequently reflected in labeling, the sections of the PI where PRO data typically appear, and the distinction between primary efficacy endpoints and supportive symptom measures.
Want Rhizome's help on your own question? Try it for free.
Patient-reported outcomes in CDER-approved prescribing information (2021 to 2026)
Short answer
Yes. Patient-reported outcomes (PROs) appear in the prescribing information (PI) of multiple products approved or relabeled by CDER over the past five years. They show up in two ways: as formal efficacy endpoints described in Section 14 (Clinical Studies), and as supportive symptom measures. The most consistent uptake is in dermatology (itch scales), acute migraine (most bothersome symptom freedom), acute pain, and inflammatory bowel disease. Examples below span new molecular entities, new therapeutic biologics, and labeling supplements to older products.
PROs used as efficacy endpoints in Section 14
The clearest signal is products where a patient-reported instrument served as, or directly supported, the registrational efficacy endpoint.
Dermatology (itch as a co-primary or primary endpoint). Itch is inherently patient-reported, and CDER has accepted validated single-item itch scales as pivotal endpoints.
- DUPIXENT (dupilumab) for prurigo nodularis: efficacy was assessed by the proportion of subjects achieving a reduction of at least 4 points on the Worst Itch-Numeric Rating Scale (WI-NRS), a single-item 0 to 10 scale, in the PRIME and PRIME2 trials, described as a primary efficacy endpoint 2. For atopic dermatitis, Dupixent labeling reports the Peak Pruritus NRS 45678121320.
- NEMLUVIO (nemolizumab-ilto), a CDER-reviewed biologic approved December 13, 2024 101, uses the Peak Pruritus NRS (PP-NRS), a weekly average of daily 0 to 10 maximal-itch scores, in prurigo nodularis (OLYMPIA 1/2) and atopic dermatitis (ARCADIA 1/2). In ARCADIA, a PP-NRS improvement of more than 4 points at Week 16 was a key secondary endpoint alongside the IGA/EASI-75 co-primaries 91595.
- LYNAVOY (linerixibat) for pruritus in primary biliary cholangitis, approved March 17, 2026 136, is built around a Worst Itch NRS: patients rate worst itch twice daily on a 0 to 10 scale, aggregated into Worst Daily, Weekly, and Monthly Itch Scores 1.
Acute pain (numeric pain rating). JOURNAVX (suzetrigine), a non-opioid NDA approved January 30, 2025 98, measured pain intensity with a patient-reported 11-point Numeric Pain Rating Scale (NPRS) and evaluated efficacy by the time-weighted sum of pain intensity difference over 48 hours (SPID48) versus placebo in acute pain after abdominoplasty and bunionectomy 6264.
Acute migraine (most bothersome symptom freedom). "MBS freedom" at 2 hours is a patient-reported co-primary endpoint now standard across the gepant and ditan classes, and it is carried in current labeling for:
- NURTEC ODT (rimegepant), pain freedom and MBS freedom at 2 hours 4348515254 (original approval February 27, 2020 135);
- UBRELVY (ubrogepant), pain freedom, pain relief, and MBS freedom 475556 (December 23, 2019 124);
- REYVOW (lasmiditan), pain freedom and MBS freedom 5758 (October 11, 2019 148);
- VYEPTI (eptinezumab-jjmr), headache pain freedom and absence of MBS on infusion day 5961 (February 21, 2020 142);
- ELYXYB (celecoxib oral solution), pain freedom and MBS freedom 44 (May 5, 2020 149);
- SYMBRAVO (meloxicam/rizatriptan), pain freedom and MBS freedom at 2 hours, approved January 30, 2025 4546495096.
Several of these migraine products were originally approved just before the five-year window, but the MBS PRO content is present in their current, in-window labeling.
Inflammatory bowel disease. OMVOH (mirikizumab-mrkz), approved as a biologic on October 26, 2023 119, reports patient-reported abdominal pain (improvement as early as Week 6), stool frequency (by Week 12), and fatigue measured by the FACIT-Fatigue scale at Week 12 in its Crohn's disease program; the label also references a patient-reported clinical-response definition used to re-randomize placebo non-responders 757682.
PROs as supportive measures
A second group reports PROs descriptively rather than as the registrational endpoint.
- COSENTYX (secukinumab): in plaque psoriasis trials PsO1/PsO2, labeling reports Week 12 improvements in itching, pain, and scaling versus placebo using the Psoriasis Symptom Diary 656671.
- XYWAV (mixed oxybate) for idiopathic hypersomnia reports the Epworth Sleepiness Scale and Patient Global Impression of change, alongside the Idiopathic Hypersomnia Severity Scale 232631 (original approval July 21, 2020 115).
- CYMBALTA / DRIZALMA SPRINKLE (duloxetine) fibromyalgia labeling reports Fibromyalgia Impact Questionnaire results and Patient Global Impression of Change 3639.
- XALKORI (crizotinib) reports patient-reported lung-cancer symptoms (dyspnea, cough, chest pain) as exploratory measures in ALK-positive metastatic NSCLC 252728.
- ZOLOFT (sertraline) social anxiety disorder labeling includes patient-rated instruments such as the Marks Fear Questionnaire Social Phobia Subscale 3041.
Summary table
| Product (active ingredient) | Therapeutic area | PRO instrument in PI | Role in labeling | Approval / relabeling context |
|---|---|---|---|---|
| DUPIXENT (dupilumab) | Prurigo nodularis; atopic dermatitis | WI-NRS; Peak Pruritus NRS | Primary/efficacy endpoint 24 | sBLA labeling within window |
| NEMLUVIO (nemolizumab-ilto) | Prurigo nodularis; atopic dermatitis | Peak Pruritus NRS | Key secondary (AD) 95; efficacy (PN) 915 | BLA approved 2024-12-13 101 |
| LYNAVOY (linerixibat) | PBC-associated pruritus | Worst Itch NRS | Core efficacy measure 1 | NDA approved 2026-03-17 136 |
| JOURNAVX (suzetrigine) | Acute pain | NPRS / SPID48 | Efficacy endpoint 6264 | NDA approved 2025-01-30 98 |
| SYMBRAVO (meloxicam/rizatriptan) | Acute migraine | MBS freedom at 2h | Co-primary 45464950 | NDA approved 2025-01-30 96 |
| NURTEC ODT (rimegepant) | Acute migraine | MBS freedom at 2h | Co-primary 4348515254 | Orig. 2020-02-27 135 |
| UBRELVY (ubrogepant) | Acute migraine | MBS freedom at 2h | Co-primary 475556 | Orig. 2019-12-23 124 |
| REYVOW (lasmiditan) | Acute migraine | MBS freedom at 2h | Co-primary 5758 | Orig. 2019-10-11 148 |
| VYEPTI (eptinezumab-jjmr) | Migraine prevention | Absence of MBS | Reported endpoint 5961 | Orig. 2020-02-21 142 |
| ELYXYB (celecoxib solution) | Acute migraine | MBS freedom at 2h | Co-primary 44 | Orig. 2020-05-05 149 |
| OMVOH (mirikizumab-mrkz) | Crohn's disease | FACIT-Fatigue; abdominal pain; stool frequency | Reported PRO endpoints 757682 | BLA approved 2023-10-26 119 |
| COSENTYX (secukinumab) | Plaque psoriasis | Psoriasis Symptom Diary | Supportive 656671 | In-window labeling |
| XYWAV (mixed oxybate) | Idiopathic hypersomnia | ESS; Patient Global Impression | Supportive 232631 | Orig. 2020-07-21 115 |
| CYMBALTA / DRIZALMA (duloxetine) | Fibromyalgia | FIQ; Patient Global Impression of Change | Supportive 3639 | In-window labeling |
| XALKORI (crizotinib) | ALK+ NSCLC | Patient-reported lung-cancer symptoms | Exploratory 252728 | In-window labeling |
| ZOLOFT (sertraline) | Social anxiety disorder | Marks Fear Questionnaire (SP subscale) | Supportive 3041 | In-window labeling |
Reading the pattern
Two things stand out for a labeling strategist. First, where the disease is defined by a symptom the patient alone can report (itch, pain, migraine bother), CDER has accepted a validated single-item PRO as a primary or co-primary endpoint, and that endpoint reaches Section 14 verbatim: WI-NRS and PP-NRS in dermatology, MBS freedom in acute migraine, and NPRS/SPID in acute pain are now effectively expected. Second, in areas where PROs supplement objective endpoints (IBD, psoriasis, oncology, sleep, psychiatry), the instruments still appear in labeling but as descriptive support rather than the basis of the indication. The distinction between an original approval within the window and a labeling supplement matters here: several migraine and sleep products carry in-window PRO labeling despite original approvals in 2019 to 2020.
Caveats and suggested follow-ups
This survey is illustrative, not exhaustive. PRO terminology varies across reviewers, and some instruments (for example generic quality-of-life questionnaires) are more likely to sit in review documents than in the final PI. If you need a defensible, complete list, useful next questions to put to Rhizome directly include: a full enumeration of Section 14 PRO endpoints for a specific therapeutic area; whether a given PRO was labeled as primary, key secondary, or supportive for a named product; and how a specific instrument (say, WI-NRS or FACIT-Fatigue) was validated and described across the reviews that supported approval.