OnCo
ideasIdea

Approve cancer biosimilars on analytics and pharmacokinetics, no efficacy trials

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.

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.

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.
What would test it
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
Who has to act
regulator
Cost to try
Small (under $1M)
Years to first evidence
3
Bottlenecks it attacks
  • Prices and value · New cancer drugs routinely cost over $150,000 a year, often for months of benefit. Systems cannot afford them and patients go bankrupt.
  • Regulatory divergence between regions · Regulatory divergence means a drug approved in one country can take years to reach another, or never arrive.

Connected

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