Table 3.
FDA Oncology Drug Approvals with a PRO Claim in the Label
| Generic (brand name) | Manufacturer | Therapeutic indications | PRO end points | PRO instruments | Approval date |
|---|---|---|---|---|---|
| Pre–FDA guidance | |||||
| Gemcitabine hydrochloride (Gemzar) | Eli Lilly | Carcinoma NSCLC Pancreatic neoplasms | HRQOL Clinical benefit response, a measure of clinical improvement based on analgesic consumption, pain intensity, performance status, and weight change | HRQOL was assessed using FACT-L, EORTC QLQ-C30, and EORTC QLQ-LC13 Pain intensity was assessed using Memorial Pain Assessment Card | May 1996 |
| Imatinib mesylate (Gleevec) | Novartis | Chronic myeloid leukemia, acute lymphoblastic leukemia | Improvement in symptoms of interferon toxicity HRQOL | FACT-BRM questionnaire | May 2001 |
| Irinotecan hydrochloride (Camptosar) | Pfizer | Colorectal neoplasms | HRQOL | EORTC QLQ-C30 | June 1996 |
| Leuprolide acetate (Eligard) | Atrix Laboratories | Prostatic neoplasms | Improvement in bone pain, urinary pain, and urinary signs and symptoms | Both bone pain and urinary pain were assessed by patients using a VAS ranging from 1 (no pain) to 10 (worst pain possible) Urination symptoms were assessed on a VAS ranging from 1 (no difficulty) to 10 (very difficult) | January 2002 |
| Mitoxantrone (Novantrone) | Immunex Corporation | Prostatic neoplasms, acute nonlymphocytic leukemia | Improvement in pain and analgesic use | Pain intensity was measured using the Symptom Distress Scale pain item 2, a 5-point scale Analgesic use was measured using a 5-point scale where 0 = no analgesics, 1 = nonnarcotics taken occasionally, 2 = nonnarcotic analgesics taken regularly, 3 = narcotic analgesics taken occasionally, and 4 = narcotic analgesics taken regularly The pain scale was derived from the present pain intensity of the McGill Pain Questionnaire | December 1987 |
| Paclitaxel (Taxol) | Mead Johnson, a Bristol-Myers Squibb Company | Carcinoma NSCLC | HRQOL | Quality of life was evaluated using the FACT-L questionnaire | December 1992 |
| Pamidronate disodium (Aredia) | Novartis | Osteolytic bone metastases of breast cancer Osteolytic lesions of multiple myeloma | Pain narcotic use | Pain score was calculated as the product of pain severity times pain frequency Both were assessed on a 4-point scale, where 0 = none to 3 = severe for pain severity and from 0 = none to 3 = constant (most of the time) for pain frequency Narcotic score was also assessed using a 4-point scale, from 0 = none to 3 = strong narcotic | October 1991 |
| Thyrotropin alfa (Thyrogen) | Genzyme | Thyroid neoplasm | HRQOL | HRQOL was measured using the SF-36 health survey | November 1998 |
| Topotecan hydrochloride (Hycamtin) | GlaxoSmithKline | Small-cell lung cancer Metastatic ovarian carcinoma | Symptom improvement | Data were collected on patients' self-assessed scores for 9 symptoms of disease: shortness of breath, interference with daily activity, fatigue, hoarseness, cough, insomnia, anorexia, chest pain, and hemoptysis. Each symptom was rated on a 4-category scale | May 1996 |
| Alitretinoin (Panretin) | Ligand Pharmaceuticals | Kaposi's sarcoma | Treatment satisfaction The subjective assessment of lesions | Patients were asked about their overall satisfaction with the treatment, which was 1 item of a 9-item QOL questionnaire The subjective assessment of all treated lesions was scored by patients using a 7-point ordinal scale | February 1999 |
| Bicalutamide (Casodex) | AstraZeneca | Prostatic neoplasms | HRQOL | Self-administered patient questionnaires on pain, social functioning, emotional well-being, vitality, activity limitation, bed disability, overall health, physical capacity, general symptoms, and treatment-related symptoms No additional detail was provided | October 1995 |
| Post–FDA guidance | |||||
| Ruxolitinib (Jakafi) | Incyte | Myelofibrosis | Reduction in total symptom score | Myelofibrosis Symptom Assessment Form | November 2011 |
EORTC QLQ-C30 indicates European Organization for Research and Treatment of Cancer Quality of Life Questionnaire; EORTC QLQ-LC13, European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Lung; FACT-BRM, Functional Assessment of Cancer Therapy-Biologic Response Modifiers; FACT-L, Functional Assessment of Cancer Therapy-Lung; FDA, US Food and Drug Administration; HRQOL, health-related quality of life; NSCLC, non–small-cell lung cancer; PRO, patient-reported outcome; QOL, quality of life; SF-36, Short Form 36-Item; VAS, visual analog scale.