Abstract
This survey study assesses understanding of and responses to the proposed US Food and Drug Administration conditional drug approval pathway among individuals who have received prior treatment for cancer or are currently undergoing treatment.
Introduction
US Food and Drug Administration (FDA) accelerated drug approval allows use of surrogate or intermediate clinical end points reasonably likely to demonstrate clinical benefit, with regular approval contingent on a required confirmatory trial.1 A proposed new pathway for diseases with unmet needs would require less evidence for market entry, with time-limited approval to generate additional evidence.2 This pathway would require safety evidence from a phase 1 study and indication of potential efficacy in ongoing phase 2 studies and for pediatric populations with terminal conditions, only demonstrated safety in phase 1 human trials and efficacy in animal models.2 We assessed understanding and responses to this pathway among adult patients and survivors (aged ≥18 years) treated for cancer.
Methods
This survey study assessed responses to the Survivor Views3 survey fielded between July 26 and August 30, 2024, to patients and survivors treated for cancer in the past 7 years. The study was determined exempt by Morehouse School of Medicine’s Institutional Review Board; survey completion indicated informed consent. The study follow the AAPOR reporting guideline.
Patient demographics (eg, age, gender [man, woman, nonbinary], self-reported race and ethnicity [Black, Hispanic/Latino, White, multiracial, or other (American Indian or Alaska Native, Asian, Middle Eastern or North African, Native Hawaiian or Pacific Islander)] were measured using descriptive statistics to assess cohort representativeness. Likelihood to enroll in a clinical trial studying a novel drug was measured on a scale of 1 (not likely) to 7 (highly likely) under 2 hypothetical situations: no effective treatments available and a conditionally approved drug available. To assess understanding, the survey provided conditional approval disclaimer language proposed in legislation to create such a pathway.2
Differences between groups were considered significant at P < .05 by Wilcoxon signed rank test. The data were analyzed using SPSS, version 30 (IBM Corp). Methodological details and the survey instrument are provided in eAppendixes 1 and 2 in Supplement 1.
Results
The study included 1597 respondents (848 aged <65 years [53.1%] and 749 aged ≥65 years [46.9%]; 284 men [17.8%], 1312 women [82.2%], 1 gender nonbinary [0.1%]; 191 of Black [12.0%] 156 of Hispanic/Latino [9.8%], 1157 of White [72.5%], 92 of multiracial or other [5.8%] race and ethnicity); 876 (54.9%) had breast cancer. Respondents were significantly less willing to enroll in a clinical trial in the presence of a conditionally approved drug (z = −11.29; P < .001). The corresponding mean (SD) willingness was 4.8 (2.0) and 4.2 (1.7), with 891 respondents (55.8%) net likely (5-7) to enroll when no other treatment was available and 593 (37.1%) when a conditionally approved drug was available (Table 1). In a separate discrete-choice question, 573 respondents (35.9%) would choose a conditionally approved drug, 518 (32.4%) would enter a trial, and 57 (3.6%) would forgo treatment.
Table 1. Patient Willingness to Enroll in Clinical Trials With or Without a Conditionally Approved Drug (N = 1597).
| Willingness to enroll in clinical trial | Patients, No. (%) | |
|---|---|---|
| No drug available | Conditionally approved drug available | |
| 1 (Not likely to join a trial at all) | 201 (12.6) | 159 (10.0) |
| 2 | 55 (3.4) | 124 (7.8) |
| 3 | 39 (2.4) | 132 (8.3) |
| Net unlikely (1-3) | 295 (18.5) | 415 (26.0) |
| 4 (Neutral) | 411 (25.7) | 589 (36.9) |
| 5 | 215 (13.5) | 233 (14.6) |
| 6 | 211 (13.2) | 141 (8.8) |
| 7 (Very likely to join a trial) | 465 (29.1) | 219 (13.7) |
| Net likely (5-7) | 891 (55.8) | 593 (37.1) |
Respondents were more likely to believe that safety and efficacy were extensively tested (824 [51.6%]) and less likely to believe that testing was minimal (202 [12.6%]) (Table 2). Similarly, more respondents believed that the drug would likely control their cancer (931 [58.3%]) vs unlikely (143 [9%]).
Table 2. Patient Interpretations of Conditionally Approved Drug Safety and Efficacya,b.
| What would you think about the drug in terms of | Patients, No. (%) | |||||
|---|---|---|---|---|---|---|
| 1 | 2 | 3 | 4 | 5 | Don’t know | |
| 1. How extensively the drug’s safety has been tested in clinical trials? | 115 (7.2) | 87 (5.4) | 320 (20.0) | 384 (24.0) | 440 (27.6) | 251 (15.7) |
| 2. How extensively the drug’s efficacy has been proven in clinical trials? | 97 (6.1) | 89 (5.6) | 294 (18.4) | 397 (24.9) | 494 (30.9) | 226 (14.2) |
| 3. How likely the drug would be to control your cancer? | 67 (4.2) | 76 (4.8) | 272 (17.0) | 284 (17.8) | 647 (40.5) | 251 (15.7) |
To assess understanding, the survey provided conditional approval disclaimer language proposed in legislation to create such a pathway: “This drug is conditionally approved for use in a limited and specific population. This drug has not received full approval by the Food and Drug Administration. Conditional approval of this drug may be withdrawn at short notice.”2
Each question was scored on a scale of 1 to 5. For questions 1 and 2, 1 indicated minimally and 5 indicated extensively. For question 3, 1 indicated unlikely and 5 indicated likely. The analysis combined 1 and 2 for unlikely and 4 and 5 for likely.
Regarding whether patients and insurance companies should pay for conditionally approved drugs still being studied, 732 respondents (45.8%) disagreed and 544 (34.1%) agreed. For the same question regarding pediatric drugs conditionally approved with efficacy tested only in animals, 865 respondents (54.2%) disagreed and 249 (15.6%) agreed.
Discussion
This survey study found that a sample disclaimer may not convey lower evidence requirements and reduced certainty of efficacy of conditionally approved drugs, suggesting the need for robust education before consent for treatment with these drugs. After FDA accelerated approval of a drug, subsequent required confirmatory trials face challenges in accruing participants.4 Similarly, we found a significantly reduced likelihood of enrolling in a trial when a conditionally approved drug is available. Our findings suggest that patients with cancer do not broadly embrace payments for drugs that have not met traditional approval standards. Limitations included that survey respondents were a nonprobabilistic panel, the survey involved a hypothetical situation with frequent don’t know responses, and views reflected patients and survivors treated for cancer only.
eAppendix 1. Survey Questions and Methodology
eAppendix 2. Survey Instrument
Data Sharing Statement
References
- 1.Expedited programs for serious conditions: drugs and biologics. US Food and Drug Administration. May 2014. Accessed January 20, 2026. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics
- 2.S.4426 - Promising Pathway Act 2.0. Congress.gov. 2024. Accessed January 20, 2026. https://www.congress.gov/bill/118th-congress/senate-bill/4426/text
- 3.Survivor views. American Cancer Society Cancer Action Network. Accessed January 20, 2026. https://www.fightcancer.org/survivor-views
- 4.Deshmukh AD, Kesselheim AS, Rome BN. Timing of confirmatory trials for drugs granted accelerated approval based on surrogate measures from 2012 to 2021. JAMA Health Forum. 2023;4(3):e230217. doi: 10.1001/jamahealthforum.2023.0217 [DOI] [PMC free article] [PubMed] [Google Scholar]
Associated Data
This section collects any data citations, data availability statements, or supplementary materials included in this article.
Supplementary Materials
eAppendix 1. Survey Questions and Methodology
eAppendix 2. Survey Instrument
Data Sharing Statement
