Presentation on theme: "Phase 0 & Patients From the Patient Side of the Desk September 5, 2007 NCI Phase O Workshop Deborah Collyar PAIR: Patient Advocates In Research."— Presentation transcript:
Phase 0 & Patients From the Patient Side of the Desk September 5, 2007 NCI Phase O Workshop Deborah Collyar PAIR: Patient Advocates In Research
Patient Advocate Comments HOPES Promising idea, could eliminate guesswork New approaches are good ‘Same old ways’ don’t cut it Changing status quo Tied to specific targets PK/PD = good for patients Like possibility to screen multiple agents, doses Holy grail if drug + imaging agent = same thing How likely? CONCERNS Multiple biopsies not good Imaging helps, but not a panacea Need known/validated markers Limits to short list of drugs/agents Biomarkers: must correlate with disease outcome Getting to tumor isn’t enough No benefit to participant Not like phase I, II, III, or IV trials Will patients really understand? Opportunity cost Stage IV may choose phase I for slight benefit potential over none Quick poll of PAIR, SPORE, and CALGB patient advocates Overall: too new for many opinions (N=11)
Phase 0 = Preclinical Testing in People Language is very important This is truly experimental… we need to say so Exploratory research v. ‘phase 0 clinical trial’ “Biomarker “: what kind? Validated? How to tie to outcome? There is still time to differentiate terms! Be a broken record: NO treatment benefit possible What ramification/price for patient to wait? Mental, physical, social, logistical, performance status? Which patients are actually available in a 7-day window? Does the body react the same way to microdose v. regular dose? What collateral damage is possible (e.g. ‘nuclear’ fallout)? ‘Cool’ science is only possible if people agree to it Could save $ + animals, but what does Aunt Sally get? Consider assuming costs for participant’s future treatment
Not ready for ‘prime time’ yet NCI is the right place to pilot exploratory studies New findings that may help everyone (e.g. WBCs) How to analyze, what technology needed, who should be on team, etc. How to screen multiple agents, doses, etc. Careful procedures + dedicated team of specialists e.g. terrific imaging resources, careful assay development Motivated patients, shorter wait time for new trial Full financial coverage for studies VERY important: clear standards for exploratory research The Assay is the KEY to success before launching an exploratory study Use only with a KNOWN target Not for ‘me too’ drugs Not for non-biologics What happens if it doesn’t work? How is a go/no go decision made? Was it the imaging probe, technology, assay, sample, pharmacogenetics, agent, or what?
Wary of ‘for benefit of science’ Is it another band aid or systemic change? We now have phases 0, I, I/II, II, II/III, III, IV… How can we truly improve this system? Efforts to date = disappointing Will a whole phase 0 dept be next? Where is Office of Human Research Protections (OHRP)? People tire of promises Is this about drugs or patients? Does this reduce or substitute steps? Need solid progress vs. policies based on enthusiasm How to we measure success? Does ‘success’ involve people? All for improving the process/speeding up clinical trials, but… Are we re-stacking blocks or designing a new structure?
In Summary… Exciting new method: approach cautiously Share knowledge gained at NCI, develop standards Suggestions Conduct these types of exploratory studies ONLY at NCI until utility is validated Especially for assays and validation Consider training teams at NIH; certification? Need stringent adherence to (yet to be established) firm standards/guidelines Improve methods for entire clinical trial system Don’t create more Tuskegees, Gelsingers, etc. Call it ‘exploratory research’, not a clinical trial Stress no benefit; study impact to patients Focus on benefit for PATIENTS, not just drugs To garner enduring support… Explain how this gets to end goal of better answers more quickly for PEOPLE