# Approve cancer biosimilars on analytics and pharmacokinetics, no efficacy trials

Source: https://onco.cc/ideas/idea-reg-biosimilar-no-efficacy-trial/  
OnCo record `idea-reg-biosimilar-no-efficacy-trial` (Idea). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Copies of biological cancer drugs are still required to run large trials that rarely change the answer. Dropping them would cut years and tens of millions from each biosimilar.

## Summary

The MHRA has not required comparative efficacy trials for most biosimilars since 2021, the EMA published a 2025 reflection paper proposing to waive them where analytical and pharmacokinetic similarity are shown, and FDA has signalled the same direction. Comparative efficacy trials for oncology monoclonal antibodies (trastuzumab, bevacizumab, rituximab, and soon pembrolizumab and nivolumab as patents expire around 2028) cost tens of millions and have essentially never overturned a positive analytical package. The proposal is a coordinated ICH-level waiver with a shared analytical similarity standard, so a biosimilar programme runs once for all regions.

## Fields

- Kind: Idea
- Last checked: 2026-09-08
- Hypothesis: Waiving comparative efficacy trials reduces biosimilar development cost by at least $30 million and time by two years per product, doubling the number of biosimilar entrants for the first checkpoint inhibitor patent expiries, with no efficacy or immunogenicity signal in post-marketing surveillance.
- Rationale: Analytical methods now characterise antibodies far more sensitively than clinical endpoints can, and the accumulated experience of dozens of approved biosimilars shows clinical efficacy trials add cost without discriminating power.
- Proposed test: Track the number of entrants, development timelines and post-marketing safety for biosimilars approved under the MHRA and EMA waivers against those approved with efficacy trials; harmonise the standard through ICH.
- Maturity: being-tested-at-scale
- Actor: regulator

## Sources

- EMA reflection paper on biosimilar clinical requirements: https://www.ema.europa.eu/en/human-regulatory-overview/research-development/scientific-guidelines/multidisciplinary-guidelines/multidisciplinary-biosimilar

## Connected records

- technologies: [Monoclonal antibodies](https://onco.cc/technologies/monoclonal-antibody/)
- drugs: [Nivolumab](https://onco.cc/drugs/nivolumab/), [Pembrolizumab](https://onco.cc/drugs/pembrolizumab/), [Trastuzumab](https://onco.cc/drugs/trastuzumab/)
- bottlenecks: [Prices and value](https://onco.cc/bottlenecks/b-drug-pricing/), [Regulatory divergence between regions](https://onco.cc/bottlenecks/b-regulatory-fragmentation/)
- roadmaps: [Global access and affordability roadmap: essential medicines and generics → biosimilars and frugal trials → reliance, pooling and homegrown innovation](https://onco.cc/roadmaps/global-access-roadmap/)

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