A standing rulebook for one-patient treatments
Sometimes a treatment must be designed for a single patient. Agreeing in advance what evidence and safety checks are needed would make that fast, fair and learnable.
Bespoke antisense oligonucleotides and personalised gene therapies have been given to individual patients with ultra-rare disease under case-by-case regulatory arrangements, and regulators have begun issuing platform-oriented guidance. Oncology has the same need for bespoke neoantigen products, personalised oligonucleotides and unusual repurposing. A published framework — pre-agreed manufacturing standards, ethics route, mandatory registry submission and shared outcome reporting — would turn one-off heroics into cumulative knowledge.
- Rare and paediatric cancers without markets · Taken together rare cancers are a fifth of all cancers, but each one alone is too small for a company to invest in.
- Regulatory divergence between regions · Regulatory divergence means a drug approved in one country can take years to reach another, or never arrive.
- Failures are hidden · Negative trials, failed drugs and abandoned programmes are rarely published, so the same mistakes are repeated.
Pages like this
not linked directly; found by shared links- IdeaAutomatic reciprocity of orphan and rare-paediatric designations between regulators
Shares Regulatory divergence between regions, Rare and paediatric cancers without markets.
- IdeaStop failing trials earlier with pre-registered aggressive futility rules
Shares ClinicalTrials.gov, Failures are hidden.
- IdeaPublish every complete response letter and negative opinion across all regulators
Shares Failures are hidden, Regulatory divergence between regions.
- IdeaNo new trial approval until the sponsor has reported its old ones
Shares ClinicalTrials.gov, Failures are hidden.
- IdeaLive tracker of the lag from first approval to real availability in every country
Shares ClinicalTrials.gov, Regulatory divergence between regions.
- IdeaOne clinical trial application accepted by regulators in every major region
Shares ClinicalTrials.gov, Regulatory divergence between regions.
- TechnologyEngineered exosomes as drug carriers
Shares Oligonucleotide therapeutics, Personalised neoantigen (mRNA) vaccines.
- IdeaOne global paediatric cancer development plan instead of separate FDA and EMA plans
Shares Regulatory divergence between regions, Rare and paediatric cancers without markets.