{"entity":{"id":"idea-bio2-n-of-1-framework","kind":"idea","name":"A standing rulebook for one-patient treatments","aka":[],"tldr":"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.","summary":"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.","asOf":"2026-09-08","links":[{"label":"FDA Oncology Center of Excellence (page moved; nearest live section)","url":"https://www.fda.gov/about-fda/"}],"tags":[],"related":["clinicaltrials-gov"],"cancers":[],"sections":[],"technologies":["antisense-sirna","neoantigen-mrna-vaccine","programmable-dna-targeting-therapeutics"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":["b-rare-cancers","b-regulatory-fragmentation","b-negative-results"],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"hypothesis":"A published n-of-1 oncology framework shortens the time from decision to treatment from over a year to under four months, and captures outcomes for over 80% of cases in a public registry.","rationale":"Rare disease neurology has already shown that bespoke therapy is technically feasible and that the binding constraint is regulatory and ethical process. Registry capture is what converts anecdotes into evidence, as it did for expanded access programmes that later informed approvals.","test":"One regulator publishes a draft framework and runs 20 cases through it, reporting time to treatment, safety events and completeness of registry capture.","maturity":"speculative","actor":"regulator","cost":"small","horizonYears":5},"route":"/ideas/idea-bio2-n-of-1-framework/","neighbours":{"collection":[{"id":"clinicaltrials-gov","kind":"collection","name":"ClinicalTrials.gov","route":"/collections/clinicaltrials-gov/"}],"technology":[{"id":"antisense-sirna","kind":"technology","name":"Oligonucleotide therapeutics","route":"/technologies/antisense-sirna/"},{"id":"neoantigen-mrna-vaccine","kind":"technology","name":"Personalised neoantigen (mRNA) vaccines","route":"/technologies/neoantigen-mrna-vaccine/"},{"id":"programmable-dna-targeting-therapeutics","kind":"technology","name":"Programmable DNA-targeting therapeutics","route":"/technologies/programmable-dna-targeting-therapeutics/"}],"bottleneck":[{"id":"b-negative-results","kind":"bottleneck","name":"Failures are hidden","route":"/bottlenecks/b-negative-results/"},{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"},{"id":"b-regulatory-fragmentation","kind":"bottleneck","name":"Regulatory divergence between regions","route":"/bottlenecks/b-regulatory-fragmentation/"}]}}