# A DRUP-style protocol for off-label generic targeted drugs in rare tumours

Source: https://onco.cc/ideas/idea-reg-generic-targeted-therapy-drup/  
OnCo record `idea-reg-generic-targeted-therapy-drup` (Idea). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Cheap generic versions of targeted cancer drugs like imatinib exist, but patients with rare tumours carrying the matching mutation often cannot get them. A structured programme would treat them and collect the evidence.

## Summary

The Netherlands' Drug Rediscovery Protocol (DRUP) gives patients with actionable mutations access to approved targeted drugs outside their label, with cohorts expanding on response and payers covering successful cohorts; it has been replicated in several countries. Now that imatinib, erlotinib, gefitinib, sunitinib, abiraterone and others are generic, an analogous protocol for generic targeted drugs would be extremely cheap. The proposal is a DRUP-generic arm run across countries including middle-income ones, with genomic profiling, cohort-based Simon two-stage designs, and pre-agreed payer coverage for cohorts meeting response thresholds.

## Fields

- Kind: Idea
- Last checked: 2026-09-08
- Hypothesis: At least 20% of generic drug-mutation cohorts reach the pre-specified response threshold and gain routine coverage, treating patients with rare tumours at a drug cost under $2,000 per year.
- Rationale: DRUP has shown that a third of off-label cohorts show clinical benefit; with generic drugs the cost-effectiveness is almost guaranteed for any cohort that responds, and the evidence gap is the only barrier.
- Proposed test: Open ten generic drug cohorts across five countries with a common protocol; report response rates, coverage decisions and cost per responder at three years.
- Maturity: early-clinical
- Actor: clinic

## Sources

- Bottleneck evidence (No incentive to repurpose cheap drugs): Pantziarka et al., The Repurposing Drugs in Oncology (ReDO) Project (ecancer 2014): https://doi.org/10.3332/ecancer.2014.442

## Connected records

- technologies: [Comprehensive genomic profiling](https://onco.cc/technologies/cgp/), [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- drugs: [Imatinib](https://onco.cc/drugs/imatinib/)
- institutions: [Netherlands Cancer Institute (NKI-AvL)](https://onco.cc/institutions/nki/)
- terms: [Basket, umbrella, and platform trials](https://onco.cc/terms/basket-umbrella-platform/)
- bottlenecks: [No incentive to repurpose cheap drugs](https://onco.cc/bottlenecks/b-generic-repurposing/), [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/)
- key papers: [The Repurposing Drugs in Oncology (ReDO) Project](https://onco.cc/key-papers/paper-pantziarka-ecancermedicalscience/)
- roadmaps: [Trial modernisation roadmap: the randomised trial → platforms and adaptive designs → decentralised, pragmatic and always-on](https://onco.cc/roadmaps/trial-modernisation-roadmap/)

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