Ensuring scientific rigor in external control arms

Randomized controlled trials are the gold standard for determining the efficacy of a new therapy or evaluating its comparative effectiveness against the standard of care. Yet there are situations, such as in rare or orphan diseases, with breakthrough therapies, or in areas of high unmet medical need, when RCTs are either unfeasible or unethical.

In such cases, using external, or synthetic, control arms to gather non-randomized data outside of the trial setting is acceptable to regulators. And when drugs are approved for marketing on the basis of data from external control arms, it follows that health technology assessment bodies must make their reimbursement decisions on the same data.

Read the whitepaper for insights on how HTA bodies would proceed, what their expectations could be for data packages, and how accepting they may be of data from external control arms. Get insights on:

• HTA guidelines for external control arms in selected European countries
• the processes applied in some sample submissions
• the statistical methods used to adjust for imbalances between treatment groups