Matthias Hunger, Céline Taveau, and Ankit Pahwa present the challenges and opportunities when using external control arm studies in submissions to HTA agencies.
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 trials complemented by external control arms are becoming more commonly accepted as evidence by both regulatory and Health Technology Assessment (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.
Who Should Attend?
This program will be beneficial for pharmaceutical, biotech, and medical device professionals involved in:
- Rare diseases
- Epidemiology
- Health technology assessment
- Health economics & outcomes research
- Marketing
- Market access, pricing and reimbursement
- Statistics
- Real world evidence
Presenters
Matthias Hunger
Matthias Hunger joined ICON in 2014. In his current role he is involved in all statistical components of research projects, from study design, protocol development, and data analysis to writing study reports or research manuscripts.
Céline Taveau
Céline Taveau provides senior leadership for large projects on strategic market access and Health Technology Assessment (HTA)’s for pharmaceutical, vaccine and medical device companies.
Ankit Pahwa
Ankit Pahwa has 14 years of experience in statistical modelling, analytics, and programming. At ICON, he is involved in chart review studies, cross-sectional survey studies, and indirect treatment comparison using external control arm.
Sponsor
ICON
Content Partners
HealthEconomics.com
HealthEconomics.com is the world’s largest Connected Community™ for Value, Evidence and Access in the life sciences industry.
HealthEconomics.com provides a single global link to the health economic and outcomes research (HEOR), real-world evidence (RWE), and Market Access (MA) stakeholder communities. Since 1993, we have provided unparalleled access to drive business, improve knowledge, and connect researchers and payer decision-makers world-wide.
Our 27,000 engaged readers rely on HealthEconomics.com as the most reliable, credible and curated digital hub for the latest news, insights, educational opportunities, and industry benchmarks in HEOR, RWE and MA. Our advertisers, numbering more than 500 with a 91% retention rate, depend upon HealthEconomics.com for best-in-class global brand awareness and business development solutions. Our aim is to connect people with expert-driven content and resources — all in one place — with the corporate mission to advance communication about healthcare value.
