The effective use of external control arm studies remains a challenging proposition which requires significant expertise and experience to successfully navigate the data selection, design, analyses, and reporting required.
Why use an ECA?
To contextualize single-arm trial data (efficacy comparison, benchmark), when:
You’ll find a collection of in-depth thought leadership resources on the topic of ECA studies below, including a publications list showing examples of our ECA-related work across therapeutic areas, and our recent contribution to ISPOR’s Value & Outcomes Spotlight publication.
Informed by published guidelines and frameworks, our teams offer a strong track record of scientific leadership in ECA study design and execution, deep methodological expertise, and a data-agnostic approach that ensures the data we leverage is fit-for-purpose.
Our ECA leadership team reflects the deep experience and expertise we have in this field
Director of
Pharmacoepidemiology & Safety
Allie is a pharmacoepidemiologist who has led numerous label expansion, external control arm, natural history, post-marketing, post-authorization safety, and meta-analytic studies, many of which have been submitted to regulatory agencies/HTA bodies and successfully supported authorization and reimbursement decision-making. She has 10+ years of experience, authored 20+ peer-reviewed publications, and received her ScM in Epidemiology and Maternal Child Health from the Harvard T.H. Chan School of Public Health.
Executive Director &
Head of Biostatistics & Analytics
Craig has 18 years of experience in research conduct and 13 years of experience in the field of statistics. He is an expert in causal inference methods with a focus on external control arms, having conducted or consulted on more than 15 external control arm studies. He has contributed to over 40 peer-reviewed journal articles across many fields and therapeutic areas. Prior to Genesis Research Group, he spent over 9 years as a statistician at Yale Center for Outcomes Research and Evaluation.
Vice President &
Head of Real-world Evidence
Deb has 30+ years of experience in the design and conduct of observational research studies, including 17 years partnering with pharmaceutical clients on the design, implementation, and strategic considerations of pharmacoepidemiology and drug safety studies and RWE for both pre- and post-approval regulatory decision-making (including external control arm studies). Deb holds an MPH in Epidemiology from the University of Washington and was trained at the Fred Hutchinson Cancer Research Center.
The Role of External Control Arms in Drug Development and Considerations for Success by Alexandra Z. Sosinsky, Craig S. Parzynski, and Deborah Casso
To contextualize effectiveness of tisagenlecleucel, an autologous anti-CD19 CAR-T cell therapy, against physician’s choice standard of care in relapsed/refractory follicular lymphoma, to support a submission to the European Medical Association (EMA) requesting a Type II variation to the marketing authorization.
Genesis Research Group developed and executed an external control arm study for submission to the EMA. It was accepted as supportive evidence and the EMA ultimately approved the Type II variation to the marketing authorization in question.
In spring 2024, we produced a webinar referencing external control arm studies and our approach to data selection titled Fit-for-purpose Matchmaking: a Data Selection and RWE Approach for both Regulatory and HTA Audiences. Speakers included Allie Sosinsky, Director, Pharmacoepidemiology and Safety at Genesis Research Group, and Carole Longson MBE, Professor and Independent Senior Advisor.
Our team members have co-authored over 1000 publications. View our ECA-related publications below.
| Title | GR Author(s) | Year | Therapeutic Area | Data Source | Journal/Conference | Link |
|---|---|---|---|---|---|---|
| An Externally Controlled Trial to Indirectly Compare Tazemetostat Plus Lenalidomide and Rituximab Versus Lenalidomide and Rituximab after at Least One Prior Systemic Therapy in Patients with Refractory and Relapsed Follicular Lymphoma. | Sosinsky A, Crowley A, Lieb C | 2023 | Oncology | Symphony/FHRD | Blood | View |
| External Control Analysis of Overall Survival in BRAF V600 Mutated Metastatic NSCLC: Comparing Single-Arm Dabrafenib + Trametinib Clinical Trial Outcomes with Real-World Standards of Care | Kanakamedala H, Wu WH | 2020 | NSCLC | Flatiron | Value in Health | View |
| Comparison of clinical outcomes of patients with METΔex14 NSCLC treated with first-line capmatinib in the GEOMETRY mono-1 study with those of a cohort of real-world patients | Kanakamedala H, Wu WH | 2020 | Oncology | Flatiron | Annals of Oncology | View |
| Utilization of External Control Arm to Contextualize Clinical Efficacy of Tisagenlecleucel Treated Among Patients with Relapsed/Refractory Follicular Lymphoma from the Single-Arm Elara Trial | Wei-Chun Hsu, Parzynski C, Rhodes R, Wen-Hsing Wu | 2021 | Oncology | n/a | Blood | View |
| Comparison of Clinical Outcomes Among Patients with Relapsed/Refractory Follicular Lymphoma Treated with Tisagenlecleucel in the Elara Trial Versus a Real-World External Control Arm of Patients Treated with Standard of Care | Wei-Chun Hsu, Parzynski C, Rhodes R, Wen-Hsing Wu | 2021 | Oncology | FHRD | Blood | View |
| Effectiveness of Alpelisib + Fulvestrant Compared with Real-World Standard Treatment Among Patients With HR+, HER2–, PIK3CA-Mutated Advanced Breast Cancer | Kanakamedala H, Wei-Chun Hsu | 2021 | Oncology | CGDB | The Oncologist | View |
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