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Pilot Studies Nuts and Bolts

Pilot Studies Nuts and Bolts. Deborah Grady, MD, MPH Professor of Medicine Associate Dean for Clinical and Translational Research UCSF. Pilot Studies. Small studies aimed at helping you: plan the best study possible determine that the study can be done get funding for your study

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Pilot Studies Nuts and Bolts

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  1. Pilot StudiesNuts and Bolts Deborah Grady, MD, MPH Professor of Medicine Associate Dean for Clinical and Translational Research UCSF

  2. Pilot Studies • Small studies aimed at helping you: • plan the best study possible • determine that the study can be done • get funding for your study • Distinguish from pre-testing of questionnaires or measurements • Phase I and phase II trials • phase I - determine toxicity • phase II - determine best dose

  3. Why Do a Pilot Study? • Feasibility • determine if you can do it • Logistics • determine the fastest, easiest, most cost-effective way to do it • Sample size ?? • estimate rate in placebo group, effect size, variance • To convince funders you can do it

  4. Feasibility • Feasibility of recruitment • obtain data on numbers eligible • survey centers, participants • measure time, expense of recruitment • Feasibility of interventions, measures • can overweight/obese persons perform the 8 yoga postures and practice at home? • will there be side effects? • can participants do 2-h GTT/other tests? • Feasibility of adherence and follow-up

  5. Logistics and Cost • What staff are needed? • How long do visits take? • Where will participants park? • Can participants answer questionnaires while completing GGT? • How much will the trial cost?

  6. Sample Size Estimates • To determine sample size for full-scale trial: • Rates of outcome in typical participants • Estimate of effect size • Variability of the main outcome • Some question validity of effect size estimates based on small studies

  7. How Big Should Pilots Be? • “It depends” • Sample size estimates rarely useful • Traditions and guesses • Cancer phase I trials start with 3 • Pilots for feasibility often 5-20 • Pilots to estimate effect size can be large • Often depends on resources and time

  8. Do Pilots Need Control Groups? • “It depends” • Phase I trials (maximum tolerable dose) • generally uncontrolled • Phase II trials (minimum effective dose) • generally controlled • Feasibility and logistics • controls generally not needed • Effect size/sample size estimates • controls helpful

  9. Funding for Pilots • Local institution • Research Allocation Program (RAP) • Residency Training Program • CTSI • NIH R03, R21, U34, K awards • Other • disease foundations • pharmaceutical companies • mentors • Can sometimes be done without funding

  10. IRB Approval for Pilots • Like any study, pilots involving people (or medical records) need IRB approval • Testing questionnaires, developing yoga postures, etc. that are devised and pre-tested with advisors and staff generally do not need IRB approval

  11. Nuts and Bolts • Resources • Contracts • Budgets • Study team • Space • Recruitment • Start-up • Forms • Adherence and follow-up • Money Matters • Closeout

  12. Funding Sources • Small trials • NIH - R03, R21, R01 • Specialty organizations/foundations • AHA, ACS, ACOG, ADA, etc • Foundations • Pharmaceutical, device companies • Large trials • NIH - R01, P01, U01 • VA, AHRQ, CDC, DOD • Pharmaceutical, device companies

  13. UCSF Resources • Senior colleagues in your discipline • Experienced trialists and trial staff • CTSI BREAD • design • ethics • statistical issues • database development and management

  14. Contracts • Required if trial supported by industry • Must be approved by UCSF Industry Contracts & company’s legal division • lots of puzzling legalese • you should understand and revise, esp. scope of work • best to start with UCSF template, revise and submit to company • Between UCSF Regents and company • signed by PI

  15. Elements of a Contract • Scope of work • Timetable for deliverables • Budget and timing of payments • get a bolus up front • don’t link payment to deliverables out of your control • Ownership of data and publishing rights • Rules for breaking contract • Confidentiality, indemnification • Patents and inventions

  16. Budgets • NIH-style (NIH, VA, AHRQ, CDC) • prepared far in advance • governed by strict rules • funds restricted to categories • permission for carry-over required • certain expenses not allowed • subject to government audit • Pharmaceutical company budgets • prepared just before trial starts • negotiable • much more flexible

  17. Money Matters • Pre-award manager • help prepare budget and “face” pages • help with budget justification • make sure you follow rules/timelines • Post-award manager • pay salaries, buy equipment and supplies • monthly report of expenditures • projections over life of trial

  18. Who is the Study Team? • Principle investigator • Project director/clinic coordinator • Recruiter • Data manager • Programmer/analyst • Statistician • Quality control supervisor • Administrative assistant • Financial/personnel manager

  19. How to Hire the Study Team • Work with your department or unit personnel (HR) manager • write a job description • decide on job series, step, salary • post the job at UCSF and advertise • review resumes • interview • select

  20. Where to Find the Study Team • Other studies • UCSF employees • Recent graduates, students • Friends and colleagues • Chronicle, web

  21. Training the Study Team • Describe, emphasize and demonstrate the importance of: • following the protocol/operations manual • meeting recruitment goals • complete adherence and follow-up • full outcome ascertainment • maintaining participant safety and confidentiality • ethical conduct of research • Formal training and certification of staff

  22. Operations Manual and Training Meeting • Purpose and design of the trial • Each measurement and visit procedure • methods • qualified personnel • calibration and testing • Completing and altering study forms • Data entry or submission • Safety and ethical issues • Certification and training of new staff

  23. Team Leadership • Management training useful • You are responsible and the boss • Keep your staff happy • involvement in the science • salary, working conditions • level of responsibility • Set an example • Promote good staff interactions • Some staff should be fired

  24. Space • Considerations • accessibility, parking • design and décor • privacy • clinical needs, special tests • safety measures • cost • CTSI CRCs • On-campus or medical space • Off campus rental

  25. Recruitment • Hire an experienced recruiter • Provide adequate time and money • Monitor results • Make changes • different approaches • more centers • longer recruitment • change eligibility

  26. Recruitment • Mailing • Targeted (age, gender, disease, etc) • UCSF Integrated Data Repository • Kaiser • DMV, HCFA, registries • Mass • DMV, Voter Registration, HCFA • Private companies • Email • Follow HIPPA and confidentiality rules

  27. Recruiment • Referral from providers • Advertise • hospital, clinics, special sites, churches • radio, newspaper, TV, internet • celebrities, leaders follow HIPPA and confidentiality rules • CTSI planning a recruitment service

  28. Start-up • Protocol • Operations Manual • Forms • IRB approval • Hiring • Training • Database design • Database validation

  29. Forms and Database Development • Define variables, create data dictionary • Determine method of data entry • Humans • Machine readable forms • Design database (CTSI Consultation) • Use prior forms - forms libraries • Pretest, validate

  30. Adherence and Follow-up • Monitor rates • Obtain contact data at baseline • Pleasant, professional staff • Develop personal relationships • Encourage resumption of intervention and return to follow-up • Cards, small gifts, meetings, parties

  31. Quality Control • Standard Operating Procedures • protocol • operations manual • statistical analysis plan • data and safety monitoring plan • Training and certification of staff • ethical conduct of research • measurements and other visit procedures • data entry • Performance review

  32. Quality Control • Special procedures for quality control • study drug • laboratory procedures • data quality • missing, out of range, illogical • variation by site, investigator, staff member • Periodic checks of source data • Periodic reports (missing forms/data, queries, differences, drift)

  33. Closeout • Final visit and post-trial plans • make final measurements • say goodbye and thank you • inform of test results • ?inform of treatment status • ?inform of trial results • ?make clinical recommendation

  34. Summary • Trails are costly and complicated • get advice from experienced colleagues • get materials used in prior trials • use UCSF services • financial, legal, IRB/CHR • CTSI CRCs • CTSI Consultation Service • Design, ethics, statistics, database management

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