HTA bodies increasingly are accepting evidence generated from single arm studies complemented by external control arms.

Randomized controlled trials (RCTs) are considered the gold standard to determine the efficacy of a new therapy or to evaluate its comparative effectiveness against standard of care. However, regulators acknowledge situations when a RCT may not be operationally or ethically feasible, such as in rare disease patient populations. In those cases, data from single-arm studies complemented by external control arms are becoming more commonly accepted as evidence by both regulatory and HTA bodies.

Due to the lack of randomization, submissions based on external control arm studies should apply rigorous analytical methods to adjust for potential confounding caused by imbalances between treatment groups. While regulatory bodies have published guidance on the use of non-randomized evidence, recommendations from HTA agencies have long been limited.

Join us to learn about the challenges and opportunities when using external control arm studies in submissions to HTA agencies. In this webinar, we will:

  • Describe current guidance regarding the use of external control arms from selected HTA agencies in Europe, including, for example, Germany and France
  • Analyze and compare critical assessments made by HTA agencies on a number of recent submissions using external control arm trials
  • Illustrate and compare statistical methods to adjust for imbalances in key prognostic variables between treatment groups
  • Summarize recommendations and critical assessments from selected HTA agencies on statistical methods used in external control arm submissions


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Céline Taveau, PharmD, MSc

Principal, Global Health Economics and Outcomes Research, ICON

Céline has over 13 years of market access experience. Céline provides senior leadership for large projects on strategic market access and Health Technology Assessment (HTA)’s for pharmaceutical, vaccine and medical device companies. She has extensive experience with orphan drugs, and oncology indications, immunology, cardiovascular, respiratory and infectiology. Her experience includes market access strategies, systematic literature reviews, value proposition development, French HTA submissions, payer advisory boards and early scientific advice engagements.

Matthias Hunger, MSc, Dr. rer. biol. hum

Lead Epidemiologist, Real World Evidence Strategy and Analytics, ICON

Matthias is involved in all statistical components of research projects, from study design, protocol development, and data analysis to writing study reports and research manuscripts. He has extensive knowledge in the analysis of patient level data in SAS or R, including statistical analyses of health-related quality of life, utility, health care cost or survival data. Matthias has experience in various disease areas including breast cancer, skin cancer, diabetes and psoriatic arthritis.

Ankit Pahwa, BE, MS

Lead Epidemiologist, Real World Evidence Strategy and Analytics, ICON

Ankit has 14 years of experience in statistical modelling, analytics and programming. At ICON his work includes chart review studies, cross-sectional survey studies and indirect treatment comparison using external control arm.  Ankit has experience in various disease areas including a variety of cancers, diabetes, COPD, multiple sclerosis, ALS, ulcerative colitis, and others. He has extensive knowledge in the analysis of patient level data as well as aggregate data in SAS and R, including survival analysis, multivariate regression, and HCRU.


This program will be beneficial for pharmaceutical, medical device, and biotechnology professionals working in the following areas:

  • Rare disease
  • Epidemiology
  • Health technology assessment
  • Health economics and outcomes research
  • Marketing
  • Market access, pricing and reimbursement
  • Statistics
  • Real world evidence