Writing Clinical Research Protocols
Writing Clinical Research Protocols. Manoo Bhakta, MD Principal Investigator, COG Associate Professor, UT. Thinking about writing a Protocol?. Protocol- “ …a complete written description of, and scientific rationale for, a research activity involving human subjects.” (Protomechanics NIH)
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- significant risks
- good study design section
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Writing Clinical Research Protocols
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- Writing Clinical Research Protocols Manoo Bhakta, MD Principal Investigator, COG Associate Professor, UT
- Thinking about writing a Protocol? • Protocol- “ …a complete written description of, and scientific rationale for, a research activity involving human subjects.” (Protomechanics NIH) • 3 general types of protocols: • 1. Retrospective review- usually with data • 2. Natural History study- may get tissue samples, DNA • 3. Interventional – Phase I/II, Phase III, Phase IV
- If you plan to do clinical research you need a copy of the regulations www.clinicalresearchresources.com
- Writing a Protocol – First steps • As PI, you usually are the one who has an idea • Is it reasonable? Do we have the resources? • Significant risks? • Do you have the patient population? • Include those important to the conduct of the protocol as an Associate Investigator • IND request, outside investigators, COI • Initially most ICs will ask you to write an abbreviated conceptual sheet outlining what you wish to do- to the Pre-IRB committee
- Who Reads Protocols? • Keep the “audience” in mind: • Other physicians • Nurses/CRAs • IRB members • Scientific reviewers
- Using Templates • Many NIH programs encourage or require the use of protocol templates, such as the ones available here: • http://ctep.cancer.gov/guidelines/templates.html • Following template guidelines can help guide authors, but not every part of a template will necessarily apply in a given study.
- Introduction/Abstract Objectives (including study schema) Background/Rationale Eligibility criteria Study design/methods (including drug/device info) Safety/adverse events Regulatory guidance Statistical section (including analysis and monitoring) Human subjects protection/informed consent Parts of the Protocol Adapted from: Protomechanics: Chapter 1 (http://www.cc.nih.gov/ccc/protomechanics/), CTEP Investigators’ Handbook, 2002 (http://ctep.cancer.gov/forms/Hndbk.pdf )
- Objectives • Objectives should be stated clearly as hypotheses to be tested. • Each objective should have a corresponding discussion in the statistical section. CTEP Investigators’ Handbook, 2002 (http://ctep.cancer.gov/handbook/index.html)
- Background and Rationale • All protocols require a section detailing the scientific rationale for a protocol and the justification in medical and scientific literature for the hypothesis being proposed. • Introductory section should be as succinct as possible and should be organized in a logical, sequential flow.
- Background and Rationale • Double check all citations: • “Bibliographic inaccuracies harm the citing author and may cast doubt on the quality of the research being reported…” Wyles DF, Behavioral and Social Sciences Librarian, 2004 • “…[A]uthors should verify references against the original document.” Uniform Requirements for Manuscripts Submitted to Biomedical Journals, ICMJE, 2005 (http://www.icmje.org/)
- Writing Eligibility Criteria STOP BEFORE YOU WRITE! • Eligibility criteria are the largest barrier to accrual to clinical trials.1 • Poorly written or poorly conceived criteria may undermine a trial’s generalizability and scientific validity.2 1Fuks A, J Clin Epidemiol, 19982George SL, J Clin Oncol, 1996
- Writing Eligibility Criteria • Eligibility criteria—stated as either exclusion or inclusion criteria—define and limit the kinds of patients that can participate in a clinical trial. • Reasons for imposing eligibility criteria can include scientific rationales, safety concerns, regulatory issues, and practical considerations. George SL, J Clin Oncol, 1996
- Writing Eligibility Criteria • Problems with restrictive criteria: • Limitations of generalizability • Failure to mimic clinical practice • Increased study complexity • Increased costs • Decreased patient accrual George SL, J Clin Oncol, 1996
- Writing Eligibility Criteria • Recommendations: • The number of eligibility criteria should be kept to a minimum. • Criteria should include only those absolutely necessary to ensure scientific validity and patient safety. • Eligibility criteria should be clearly defined and verifiable by an external auditor. • Adapted from: • George SL, J Clin Oncol, 1996Fuks A, J Clin Epidemiol, 1998
- Writing Eligibility Criteria • Eligibility criteria should be straightforward and unambiguous. Which of these criteria would you choose? • Pregnant and/or nursing women are not eligible. • All women of childbearing age are required to have a serum pregnancy test. • Pregnant and/or nursing women are not eligible for this study. All women of childbearing potential (defined as…) must have a negative pregnancy test (serum or urine) within 2 weeks of study enrollment.
- Writing Eligibility Criteria • However, be aware of the consequences of highly specific criteria: • For example: consider the issues that will follow from mandating a particular serum concentration of some marker, rather than building the definition around institutional upper limits of normal.
- Study Design • The study design section of the protocol should contain a stepwise description of all procedures required by the study. • A good study design section includes sufficient information for the participating site to develop a comprehensive clinical pathway for study patients.
- Study Design • Parts of the study design section may include: • Initial evaluations • Screening tests • Required lab tests • Details of treatment and ancillary procedures • Agent information or device specifications • Dose scheduling and modification • Calendars Adapted from: Protomechanics: Chapter 1 (http://www.cc.nih.gov/ccc/protomechanics/)
- Safety • The Safety (or Adverse Events) section should include: • Detailed information for reporting adverse events, including reporting to the FDA and/or the sponsor • Unblinding processes (if applicable) • Lists of expected adverse events
- Human Subjects Protection • This section includes discussion of: • Subject selection and exclusion • Proposed methods of patient recruitment • Minority representation • Recruitment (or exclusion) of special subjects, including vulnerable subjects • Lists of potential risks and benefits, including justification for risks Adapted from: OHSR Information Sheet 5: Guidelines for Writing Research Protocols (ohsr.od.nih.gov/info/sheet5.html )
- Model Informed Consent • The Office of Human Subjects Research recognizes 3 fundamental conditions for a valid informed consent: • Disclosure of relevant information to prospective research subjects • Comprehension of the information provided • Voluntary agreement of the subject, free from coercion Adapted from: OHSR Information Sheet 6: Guidelines for Writing Informed Consents (ohsr.od.nih.gov/info/sheet6.html )
- Model Informed Consent • To these ends, the protocol model informed consent document must: • Be thorough and complete • Be written in simple, nontechnical language • Be carefully worded to avoid potentially coercive phrasing
- Model Informed Consent • OHSR recommends the following be included: • Statement that the study involves research • Purpose of the research and the length of the study • Description of risks and benefits • Discussion of alternative therapies • Confidentiality policy • Compensation for injury • Contact for further questions/information • Statement of voluntary participation Adapted from: OHSR Information Sheet 6: Guidelines for Writing Informed Consents (ohsr.od.nih.gov/info/sheet6.html )
- Model Informed Consent • Writing a good MIC is a balancing act between being thorough, being accurate, and being as concise and simple as possible. • Patient advocates may offer invaluable experience and insight during the drafting and review phase of an MIC.
- The Statistical Section • Make sure that study objectives and study design elements in the statistical section mirror those in described in the Objectives section! • If the study involves stopping rules, make sure that descriptions and definitions of toxicities in the statistical section match those in the Safety/AE section.
- Good Writing Matters “…Many existing clinical trials contain problems such as incompleteness, ambiguity, and inconsistency. Most of the errors are introduced during the protocol writing process…” Weng C, Medinfo, 2004
- Good Writing Matters • Costs of a badly written protocol? • Poorly written inclusion criteria have resulted in a number of ineligible and inevaluable patients being enrolled to a study. NOW WHAT?
- Good Writing Matters • To fix this problem, the protocol has to be amended. Remember: “Any change to the protocol document or Informed Consent that affects the scientific intent, study design, patient safety, or human subject protection is considered an amendment, and therefore mustbe approved by your IRB…” NCI CTEP Amendment Request Submission Policy, Version Date May 14, 2004 (http://ctep.cancer.gov/guidelines/templates.html)
- Tools for Better Writing: Proofreading • Fresh eyes • Working too long on a protocol may habituate eyes and brains to mistakes, simply because they’ve been there all along. Get an “outside” reviewer! • Spell-checkers, etc. • A document that has been “checked” by automatic software has NOT been proofread.
- NIH Guidance on Protocol Writing • Protomechanics: http://www.cc.nih.gov/ccc/protomechanics/ • The Office of Human Subjects Research: http://ohsr.od.nih.gov/info/info.html • The NCI Investigators’ Handbook: http://ctep.cancer.gov/handbook/index.html
- Templates • The NIH phase III template: http://www.ninds.nih.gov/funding/research/clinical_research/ProtocolTemplate.htm • The Cancer Therapy Evaluation Program (CTEP) Templates (phases I–III; based on NIH model): http://ctep.cancer.gov/guidelines/templates.html
- Informed Consents • Office of Human Subjects Researchhttp://ohsr.od.nih.gov/info/info.html • The Office for Human Research Protections (OHRP): http://www.hhs.gov/ohrp/policy/index.html#informed
- Writing Resources • The International Committee of Medical Journal Editors (ICJME) Uniform Requirements for Manuscripts Submitted to Biomedical Journals http://www.icmje.org/
- Writing Resources • The American Medical Association style manual may provide a useful “default” reference for protocol writing: Cheryl Iverson, Chair. The American MedicalAssociation Manual of Style. 9th ed. Lippincott Williams and Wilkins; 1998. • The Duke University Medical Center Library maintains a select bibliography of style guides at: http://www.mclibrary.duke.edu/subject/style
- Questions?
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