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Published on: October 3, 2018
Low participation rates and disparities in participation in interventional clinical trials for myelodysplastic
Charlotte K Brierley1, Emily C Zabor2,3, Rami S Komrokji4
1Weatherall Institute of Molecular Medicine, Oxford, United Kingdom.
Background:
The development of novel therapies for the myelodysplastic syndromes (MDS) is hampered by inadequate trial recruitment. Factors contributing to low trial accrual are incompletely understood.
Methods:
This study analyzed a pooled patient database from institutions of the US MDS Clinical Research Consortium to compare the characteristics of participants in interventional trials with those of patients who did not enroll in a trial.
Results:
Data were identified for 1919 patients with MDS, and 449 of these patients (23%) participated in an interventional clinical trial. The median age of all patients was 68 years, and 64% were male. Patients who participated in trials were significantly younger than nonparticipants (P = .014), and men were more likely to participate in a trial (71% of trial participants were male, whereas 61% of nonparticipants were; P < .001). Race and ethnicity were not associated with trial enrollment. Patients in more affluent ZIP codes had a higher participation rate (P < .001). Patients with intermediate- and high-risk disease according to the revised International Prognostic Scoring System were overrepresented (P = .004), and trial participants less frequently had treatment-related disease (P < .001). In multivariable analyses, participation in a clinical trial was associated with a reduced hazard of death (P = .004). Even at large referral centers, only a minority of patients with MDS enrolled in interventional trials.
Conclusions:
Restrictive trial eligibility criteria that exclude patients with MDS on account of age, comorbidities, or a history of another cancer are limit enrollment of MDS patients to clinical trials. Gaining insight into the barriers to trial accrual may help investigators and study sponsors to design trials that will accrue more rapidly and augment treatment options for patients with MDS.
Insights
Recruiting for myelodysplastic syndromes (MDS) clinical trials is challenging. Younger, male patients from wealthier areas with higher-risk disease were more likely to enroll, suggesting barriers to trial access.
Area of Science:
- Hematology
- Clinical Trials
- Oncology
Background:
- Developing new therapies for myelodysplastic syndromes (MDS) is hindered by low patient recruitment in clinical trials.
- Understanding the factors contributing to poor trial enrollment is crucial for advancing MDS treatment options.
Purpose of the Study:
- To compare the characteristics of patients with myelodysplastic syndromes (MDS) who enrolled in interventional trials versus those who did not.
- To identify barriers and facilitators of clinical trial participation in MDS patients.
Main Methods:
- Analysis of a pooled patient database from the US MDS Clinical Research Consortium.
- Comparison of demographic, clinical, and socioeconomic factors between trial participants and non-participants.
Main Results:
- Of 1919 MDS patients, 23% (449) participated in interventional trials.
- Trial participants were younger, more likely male, and from more affluent ZIP codes compared to non-participants.
- Patients with intermediate/high-risk disease and those without treatment-related MDS were overrepresented in trials; trial participation was associated with a reduced hazard of death.
Conclusions:
- Restrictive eligibility criteria (age, comorbidities, prior cancer) limit enrollment of MDS patients in clinical trials.
- Identifying and addressing barriers to trial accrual is essential for designing inclusive trials and expanding treatment options for MDS patients.
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