CLINICAL CHECKLIST

Pharmacist
Health Services Researcher
Economist
Lead Assessor
Date of NDC Meeting
Full submission or resubmission
1. REGISTRATION DETAILS
1.1 Medicine (generic name, strength, form[proprietary name])
1.2 Submitting company
1.3 Licensed indication under review
1.4 Other licensed indications (if relevant to place indication under review in context)
1.5 Any proposed positioning
1.6 Patient and Clinician Engagement (PACE) criteria / Company submission / SMC validated
End of life medicine (a medicine used to treat a condition at a stage that usually leads to death within 3 years with currently available treatments)? / Yes/No / Yes/No/N/A
EMA designated orphan or a medicine to treat an equivalent size of population (5 per 10,000) (full licensed indication) / Yes/No / Yes/No/N/A
A medicine used to treat a condition with prevalence of <1 in 50,000 people, i.e. ultra-orphan status (full licensed indication) / Yes/No / Yes/No/N/A
1.7 Is it an EMA designated orphan medicine? Provide date of designation and number.
1.8 Was this medicine included in the Early Access to Medicines Scheme (EAMS)? Provide date and full indication if different to licensed indication.
1.9.1 Date of licensing / 1.9.2 Date of availability
1.10Dose
1.11Disease context and potential comparator(s)
1.12Relevant comparator(s). Whatmight it replace in Scottish practice?
1.13Indirect comparison: If an indirect comparison was presented was it appropriate and was it pivotal to the economic case? (Confirm with economist if not clear)
1.14Type of economic case (e.g. cost minimisation analysis, cost utility analysis)
2. SUMMARY
2.1 Summary of submitting company’s clinical case
2.2 Strengths of clinical evidence
2.3 Weakness of clinical evidence
2.4 Key issues and uncertainties in the evidence with respect to the submitting company’s clinical case
3. CLINICAL EVIDENCE

Overview of Clinical Evidence

Methodology / Patient nos. / Treatment Allocations / Source of funding

*Key studies are highlighted in bold and will be appraised in detail

Detailed critical appraisal checklists for studies.

DESCRIPTION of study A

EFFICACY

3.1 DESCRIPTION OF THE STUDY DESIGN

3.2 DESCRIPTION OF THE STUDY POPULATION

3.2.1 Does the study population (or a subgroup) represent the indication under review or proposed positioning (if applicable)?
3.2.2 Key inclusion and exclusion criteria
3.2.3 Were the inclusion criteria appropriate to the indication under consideration?
3.2.4 Did any exclusion criteria affect the generalisability of the study to the Scottish population?
3.3 DESCRIBE THE STUDY TREATMENTS
3.4 DESCRIPTION OF THE STATISTICAL METHODOLOGY/PLAN
3.4.1 What was the primary outcome(s) and was it clearly defined?
3.4.2 Was the primary outcome measure valid? Was there a clear description of the techniques/scoring system used to identify or measure events?
3.4.3 Were any methods used to enhance the accuracy of the measurements e.g. central review, multiple measurements, training of assessors?
3.4.4 What population was used in the analysis? If not appropriate, please explain.
3.4.5 Was the study appropriately powered? If not, explain.
3.4.6 Were there any concerns about the statistical analysis? If yes, explain.
3.4.7 Did the methodology described account for the possibility of missing data?
3.4.8 Was the study and follow up of an appropriate duration for the disease state and intervention under review?
3.5 RESULTS
3.5.1 Was there a CONSORT diagram and were all patients accounted for?
3.5.2 Did the study maintain comparable groups throughout or was there crossover or high drop-out rate in any one group?
3.5.3 Were the baseline characteristics of patients in different groups well matched for demographics, prognostic factors, co-morbidities, concomitant treatment or other relevant factors?
3.5.4 Provide details of the primary outcome giving all relevant information to how the results were presented.
3.5.5 Provide details of relevant subgroup analyses that support the licensed indication or proposed positioning.
3.5.6 Describe briefly any relevant secondary outcomes?
3.5.7 Provide details of patient reported outcomes.
3.6 EXTENSION STUDY
3.6.1 Was there a relevant extension study? Provide brief details/results.
3.7 ANY ADDITIONAL IMPORTANT DETAILS

SAFETY

3.8 DESCRIPTION OF ADVERSE EVENTS
3.8.1 Describe the overall adverse event profile.
3.8.2 Describe any relevant comparative overall adverse event information including incidence of any important treatment related adverse events.
3.8.3 Give details of any treatment related deaths during the study or at follow up.
3.8.4 Detail any important safety aspects that may be specific to this medicine including rare and/or life-threatening treatment related adverse events.
4. ADDITIONAL RELEVANT INFORMATION
4.1 Give brief details of any relevant additional information including supportive studies that have augmented the data available for the indication under review in this submission.
5. SUMMARY OF CLINICAL EFFECTIVENESS
5.1 Provide brief description of pharmacology for the medicine under review and any background details that may have a bearing on the use of this medicine e.g.is this medicine first in class for this indicationor are there any other special circumstances?
5.2 Provide a description of the disease and currently available treatments. If this is an end of life medicine, include a brief outline of overall survival estimates with currently available treatment for the relevant patient population or proposed positioning.
5.3 Provide a brief summary of the key study(or studies) outcome(s).
5.4 Are there any study data relative to a relevant active comparator?
5.5 Did the primary outcome measure a direct health outcome and was it statistically significant and/or clinically relevant?
5.6 Are there any limitations of the study methodology, any subgroup or post-hoc analyses, high drop-out rates or significant crossover of patients or other factor(s) affecting the quality of the evidence and which may affect the application of the study results?
5.7 Was there anything in the way the results were reported that should be taken into consideration e.g. wide confidence intervals, small relative differences presented as significant, insufficient treatment duration, relevant (long-term) outcomes not available, relative not absolute risk or other factor(s)?
5.8 Are there any aspects of the study population, comparator or concomitant treatment (including interventions over and above that in normal clinical practice), subgroup or post-hoc analysis or other factor(s) that might affect the generalisability of the study results to the Scottish population?
5.9 Would the introduction of this intervention have advantages or disadvantages for the service or patient? Are there any practical issues for the patient or carer? Are there any service implications?
5.10 Were there any significant safety concerns?
5.11 Give details of any other important information specific to this medicine.

APPENDIX 1

Published guidelines relevant to the indication under review.

APPENDIX 2

Detail previous SMC advice relevant to the indication under review.

APPENDIX 3

List of references used in critical appraisal.

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