questionnaires in and how to map as per CT and aCRF
Download as PPTX, PDF0 likes144 views
basic understanding of questionnaires in SDTMIG and how to map as per CT and ACRF.
questionnaires in and how to map as per CT and aCRF
- 2. What are Questionnaries ? Questionnaire can be defined as a standard data collection tool consisting of a series of questions or items that are used to collect information related to health, symptoms, quality of life, and functional status. These responses are often considered Patient-Reported Outcomes (PROs) or Clinician-Reported Outcomes(ClinROs). purpose of collection By comparing questionnaire responses before and after treatment, researchers assess how well the drug or intervention worked. Responses are coded and standardized (QSSTRESC/QSSTRESN), making them usable for statistical models which Supports primary or secondary endpoints. Regulatory Evidence:use PRO data as evidence for : • Treatment effectiveness. • Drug labeling claims (e.g., "improves quality of life“) • Risk-benefit analysis
- 3. Where are Questionnaires Captured? In the CRF (Case Report Form), a specific section is designed for questionnaire data collection. These may include: Paper CRFs – Questionnaire form used during site visits . eCRFs – electronic platforms (EDC systems like Medidata Rave, Oracle InForm) where patients or site staff enter data.
- 8. PHQ-9
- 12. Types of Questions in a Questionnaire Here are some commonly used question types: 1. Open-ended questions: • Open-ended questions offer participants a space into which they can answer by writing text. eg :a) “What brings you in today?” b) “Can you describe your symptoms?” 2. Closed-ended questions : • Closed-ended questions contain include a clear instruction that participants may select more than one response option (for example, 'tick all that apply’). • Which of the following symptoms have you experienced in the past week? Headache ☐ Nausea ☐ Fatigue ☐ Dizziness ☐
- 13. Types of Close Ended Questions 1. Dichotomous Question : These close ended question are indicative questions that can be answered either in one of the two ways, “yes” or “no” or “true” or “false”. • Do you currently take any medication for high blood pressure? Yes ☐ No ☐ 2. Multiple choice question : • What types of pain have you experienced today? Headache ☐ Muscle pain ☐ Joint pain ☐ Abdominal pain ☐ 3. Likert Scale : The Likert scale is a commonly used in clinical trials to assess patient-reported outcomes, such as symptoms, treatment satisfaction, and quality of life. It provides a range of response options—typically from "Strongly Agree" to "Strongly Disagree“. How severe was your joint pain in the past 24 hours? None ☐ Mild ☐ Moderate ☐ Severe ☐ Very Severe ☐
- 14. 4. Rating Scales A rating scale asks participants to rate their experience or opinion on a numeric or descriptive scale, such as 0 to 10 or from “Poor” to “Excellent.” 5. Checklist : • Checklist-type questionnaires are a form of closed-ended questions commonly used in clinical trials to let participants select all applicable responses from a list. • These are widely used to collect consistent and analyzable data on multiple topics such as symptoms, medical history, concomitant medications, and adverse events. 6. Matrix : • A matrix questionnaire consists of a series of rows with items to be answered with a series of columns providing the same answer options. • The EORTC QLQ-C30 is an example of a matrix questionnaire. 7. pictorial questionnaires: (eg; Wong-Baker FACES, Pictorial Blood Assessment Chart) used in clinical trials, especially when working with: • Children • Elderly • Low-literacy populations • Cognitively impaired patients
- 15. Questionnaire development • If a questionnaire has previously been used in similar trials to the one planned, its use will bring the added advantage that the results will be comparable and may be combined in a meta-analysis. • CIOMS Working Group prepares an example of the format and content of a possible questionnaire. Once the researcher has decided to design a new questionnaire, several steps should be considered: • If new questions are to be developed, the reading ease of the questions can be assessed using the Flesch reading ease score. • This score assesses the number of words in sentences, and the number syllables in words. Higher Flesch reading scores indicate material that is easier to read .
- 16. Examples of poorly phrased questions in a research questionnaire
- 17. Regulatory guidelines The International Conference on Harmonisation (ICH) of technical requirements for registration of pharmaceuticals for human use states: • The collection of data and transfer of data from the investigator to the sponsor can take place through a variety of media, including paper case record forms, remote site monitoring systems, and electronic transfer. • Whatever data capture instrument is used, the form and content of the information collected should be in full accordance with the protocol and should be established in advance of the conduct of the clinical trial. • Only collect data necessary for planned analysis. Clear distinction must be made between: Missing values Zero values Absent characteristics
- 18. Council for International Organisations of Medical Sciences (CIOMS) advises that: CIOMS is advising that all safety-related data can be captured neatly using checkboxes, dropdowns, or predefined categories in a Case Report Form (CRF) or questionnaire. • When unexpected or unusual events occur — especially those that: Do not fit into predefined categories. need additional explanation. What Are Predefined Data Fields? These are structured fields like: Checkboxes: (e.g., "Yes / No" for headache) But not everything fits into these formats ..continued……
- 19. • Did you experience any skin-related symptoms (e.g., rash, itching, redness)?" • Answer choices: ☐ None Mild ☐ Moderate ☐ Severe ☐ Very Severe ☐ • Patient Response: Selects: “Moderate rash” Follow-up : • “Please describe the symptom if different from above or if more detail is needed” Patient writes: “Rash started as red spots but now turned into painful blisters across my chest and arms within a day.”
- 20. History and origin • The first documented patient-reported outcome dates back to 1914, marking early recognition of patient feedback in healthcare. • In 1970, the World Health Organization (WHO) led efforts to standardize outcomes in cancer trials. • In the early 1980s, especially in rheumatology, clinical trials began systematically incorporating self-report tools • EQ 5D instrument emerged in 1987 and was finalized in 1995, becoming a global standard for measuring health-related quality of life. • Starting in the 1990s, the shift from paper to electronic patient-reported outcomes (ePRO) occurred—using tablets, smartphones, and IVR systems to collect data in real time, with better compliance and reduced errors
- 21. Before QS Domain: Where Were Questionnaires Stored? • Before CDISC introduced the QS (Questionnaires) domain in SDTMIG v1.2 (2008), questionnaire data did not have a dedicated domain. • Instead, it was stored inconsistently in other domains, based on the type of question or sponsor preference. Old Domain When It Was Used for Questionnaires Example VS (Vital Signs) If the response was numeric (e.g., pain scale 0–10) Pain VAS score stored as VSTESTCD = "PAIN" MH (Medical History) If the question asked about prior illness/symptoms "Have you ever had depression?" → MHTERM = Depression CM (Concomitant Medications) If the question related to drug use "Are you taking aspirin?" → CMTRT = Aspirin FA (Findings About) If the data was structured but had no specific domain A cognitive test result stored as a finding about a condition
- 22. Development Flow R&D / Clinical Design Stage Choose validated instruments (e.g., SF-36, EQ-5D) Define questionnaire items in the CRF eCRF Creation • Map questions and responses to standard formats Data Collection • Subject self-completes or interviewer-administered via edc or crf Raw Dataset • Collected in raw SAS datasets • SDTM Mapping • Mapped to the QS domain using CDISC SDTM rules
- 23. List of commonly used questionnaries …….continued………
- 25. List of Questionnaries used by sponsor without Licenses(free version) • Alcohol Use Disorders Identification Test - Consumption Questions • Clinical Global Impression • Combat Exposure Scale • Craig Handicap Assessment and Reporting Technique - Short Form Interview Version • Craig Handicap Assessment and Reporting Technique - Short Form Paper Version • Deployment Risk and Resilience Inventory-2 • Expanded Disability Rating Sale - Postacute Interview Caregiver Version • Expanded Disability Rating Sale - Postacute Interview Survivor Version • Extended Glasgow Outcome Scale • Visual Functioning Questionnaire - 25 Self Administered • Satisfaction With Life Scale
- 26. List of Questionnaries used by sponsor with Licenses • Airway Questionnaire • Alzheimer's Disease Assessment Scale – Cognitive • Arizona Sexual Experiences Scale – Male • Asthma Daytime Symptom Diary • Bladder Control Scale • Children's Depression Rating Scale, Revised • Clinical COPD Questionnaire (CCQ) 1 Week Version • Columbia Suicide Severity Rating Scale – Baseline • Diabetes Distress Scale for Adults with Type 1 Diabetes • Disability Assessment for Dementia
- 27. Data Cleaning Steps for Questionnaires in CRFs Missing Data Checks : Check if any question in the questionnaire is left blank. Action: Raise a query to site. Invalid/Mismatched Responses : Check that responses match allowed values. Invalid Example: A response like “5” or “N/A”. Out-of-Range Values : For numerical scales (e.g., VAS score: 0–100), ensure data falls within range. Date Validations : Check that the date of questionnaire Duplicate CRF Entries : Remove or resolve duplicates.
- 28. SDTMIG What is QS Domain? • QS (Questionnaires) is a Findings domain that contains data for named, stand- alone instruments designed to assess a psychological, physical, or health-related trait or condition. • Label of dataset- Questionnaires. • Submission format- qs.xpt. • structure -One record per questionnaire per question per time point per visit per subject. • Purpose –Tabulation. • Topic variable- QSTESTCD Who Maintains Questionnaires in CDISC? • The CDISC QRS (Questionnaires, Ratings, and Scales) team develops supplements for individual instruments,while the ADQRS team develops ADaM supplements
- 30. Therapeutic-Specific QS Examples Therapeutic Area Sample Instruments CNS / Psychiatry PHQ-9, MADRS, HAM-A, C-SSRS Neurology ADAS-COG, MMSE, UPDRS Oncology EORTC QLQ-C30, FACT-G Respiratory SGRQ, CAT (COPD) Pain Brief Pain Inventory (BPI), McGill Dermatology DLQI, Skindex-16 Rheumatology HAQ-DI, BASFI
- 31. Mapping to sdtm
- 33. • CT for PHQ
- 35. How efficacy is measured? • Womac Purpose: Measures pain, stiffness, and physical function in patients with osteoarthritis (OA). • Format: 24 items across 3 domains: Pain – 5 items Stiffness – 2 items Physical Function – 17 items. WOMAC is primarily used to assess efficacy of investigational treatments in OA trials by measuring change from baseline over time.
- 36. Time Point WOMAC Pain Score Interpretation Baseline 60 Moderate pain Week 12 35 41% improvement in pain Week 24 20 66% improvement — effective
- 37. How Is Safety Measured Using Questionnaires? • Safety questionnaires capture: Symptoms (e.g., fatigue, nausea, dizziness). Severity of side effects. Frequency or duration of adverse experiences. Patient's perceived tolerability of treatment.