1 Background
The benefit assessment is divided into the following ten steps |
1. Defining the preliminary research question |
2. Tendering and awarding the commission |
3. Defining the scope |
4. Developing the protocol (in IQWiG terms: report plan) |
5. Assessment of the evidence: in this phase literature is searched, critically appraised, and analysed |
6. Publication of the preliminary report |
7. Review of the preliminary report |
8. Publication of the final report |
9. Submission to the commissioning entity |
10. Appeal and planning update of the report |
2 Legal framework
Section 35b § 1: “(1) 1. Pursuant to Sect. 139b § 1 and 2, the Institute for Quality and Efficiency in Health Care can be commissioned to assess the benefit or the cost-benefit ratio of pharmaceuticals. 2. Assessments according to clause 1 can be made for each pharmaceutical with patented active ingredients that has become eligible for prescription for the first time, as well as for other pharmaceuticals of significance. 3. The assessment is made based on a comparison with other pharmaceuticals and therapy forms in consideration of the additional therapeutic benefit for the patients in proportion to the costs.4 4. With regard to patient benefit, especially the improvement of the state of health, a reduction in the duration of illness, an extension of the duration of life, a reduction of side effects and an improvement in the quality of life should be taken into account appropriately, as should the suitability and reasonableness of cost absorption by the community of insured people5 when making an economic assessment. 5. The Institute makes commission-related decisions on the methods and criteria for the development of assessments pursuant to clause 1 based on the international standards of evidence-based medicine and health economics acknowledged by the respective expert circles. 6. During the commission-related development of methods and criteria and the generation of assessments, the Institute ensures high procedural transparency and appropriate participation of the parties mentioned in Sect. 35 § 2 and Sect. 139a § 5. 7. The Institute shall publish the respective methods and criteria on the Internet. 8. Clauses 3 through 7 shall also apply to benefit assessments that have already been started.”
Section 35b, § 2: “(2) 1. The assessments according to Sect. 1 are fed to the Federal Joint Committee as a recommendation for decision-making according to Sect. 92, clause 1, § 2, No. 6. 2. They are to be checked at suitable intervals and, if necessary, to be adapted. 3. If new scientific evidence is available, the assessment is to be reviewed at the request of the manufacturers.
Section 139a § 4: “(4) 1. The Institute must ensure that the assessment of the medical benefit is made based on internationally acknowledged standards of evidence-based medicine and that the economic assessment is made based on the relevant internationally recognised standards, especially of health economics. 2. At regular intervals, the Institute must publicly report on the work processes and results including its basis for decision-making. (5) 1. In all important segments of the assessment procedure, the Institute must provide an opportunity for comment to the experts of medical, pharmaceutical and health economic science and practice, to pharmaceutical companies and the relevant organisations representing the interests of patients and the self-help organisations for chronically ill and disabled people, as well as the Federal Government Commissioner for Patients’ Affairs. 2. The comments must be included in the decision. 6. To ensure the professional independence of the Institute, the employees must—prior to being hired—disclose all relationships to interest associations and commissioning institutes, especially those of the pharmaceutical and medical products industry, including the type and amount of financial allocations.”
Section 139b § 3: “1. In order to fulfil its tasks according to Sect. 139a § 3 clauses 1–5, the Institute must commission scientific projects to external experts. 2. These experts must disclose all relationships to associations and contract organisations, particularly in the pharmaceutical industry and the medical devices industries, including details on the type and amount of possible remuneration received.”
3 Requirements of the assessment process
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Relevant organisations representing the interests of patients
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Experts in medical, pharmaceutical and health economic science and practice
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The professional organisations of pharmacists and pharmaceutical companies and experts on alternative therapies
3.1 Transparency of procedure
3.1.1 Topic identification and prioritisation
3.1.2 Tendering and awarding the commission
3.1.3 Defining the research question
3.1.4 Developing the protocol
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Patient population—what patients the assessment refers to
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Intervention—what medicine is evaluated (including dosing instructions and methods of use)
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Comparison intervention—which is/are the current standard treatment(s), including the rationale for choosing this or these treatment(s) as standard
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Outcomes—what outcomes are important and patient-specific
3.1.5 Assessment of the evidence
3.1.6 Publication of the preliminary report
3.1.7 Review of the preliminary report
3.1.8 Recommendations and final report
“The assessment process consists of an objective analysis of the quality, findings and implications of the (mainly research) evidence available as it relates to the appraisal question and context. The appraisal process, in contrast, is a consideration of the outputs of the assessment process within the context of additional information supplied by relevant parties such as clinical specialists and patient experts. The appraisal decision is a judgment on the importance of a range of factors that differ from appraisal to appraisal.” |
3.1.9 Appeals and planning updates to the report
3.1.10 Conclusion
3.2 Active participation of the affected parties
3.2.1 Topic identification and prioritisation
3.2.2 Defining the research question(s)
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Patient group variations, in particular differential baseline risk of the condition and capacity for different subgroups of patients to benefit;
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The particular circumstances in which treatment is delivered, including the need for concomitant treatments, the setting in which treatment is delivered and the requirements for additional professional input;
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The treatments that are currently used as standard practice and whether these may differ from what is considered best practice.
3.2.3 Participation in other phases of the assessment
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Written comments and oral discussion for the protocol
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Written comments and oral discussion for the preliminary report
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Appeal for the final decision of the FJC
3.3 Conclusion
4 Requirements of assessment methods
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The institute must ensure that the assessment of the medical benefit is made in accordance with internationally recognised standards of evidence-based medicine (EBM).
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The benefit assessment is conducted in comparison with other medicines and/or treatment forms under consideration of the additional therapeutic benefit for the patients. This requires the definition of current treatment standard(s) with which a (new) intervention is compared. Co-interventions which are widely used should be allowed.
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The minimum catalogue of criteria for assessing patient benefit, as given by law, are
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Improvement of the state of health
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Shortened duration of illness
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Extension of the duration of life
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Reduction of side effects
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Improvement in the quality of life
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4.1 Evidence-based medicine
4.1.1 Best available evidence
4.1.2 Internal versus external validity
4.1.3 Role of systematic reviews in EBM
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Level 1: RCTs
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Level 2: controlled observational studies, e.g. cohort studies, case-control studies
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Level 3: observational studies without a control group, e.g. case series
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Level 4: expert opinion [12]
4.1.4 Assessment of the evidence for each outcome
4.1.5 Evidence-based methods to prevent bias in the assessment
4.2 Choice of comparators
4.3 Benefit
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Improvement of the state of health
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Reduction in the duration of illness
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Extension of the duration of life
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Reduction of side effects
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Improvement in the quality of life
4.4 Conclusion
5 International standards/methods used by NICE and IQWiG
5.1 Methods of NICE and IQWiG
5.2 Assessment process used by NICE and IQWiG
IQWiG | NICE | |
---|---|---|
Topic identification and prioritisation | ||
The criteria for selecting topics are publicaa
| ±a
| +o
|
The criteria for prioritizing topics are public | −b
| NAp
|
Tender and commission | ||
The criteria experts should fulfil are public | +c
| ±q
|
The criteria for selecting experts are public | −d
| −r
|
The procedure for the commission is public | −e
| − |
Research question | ||
Relevant parties are involved in this phase | −f
| +s
|
An oral hearing is implemented in this phase | ±g
| + |
All comments from relevant parties are public and evaluated | NAh
| +t
|
Protocol | ||
The protocol is published | +i
| +u
|
Relevant parties are involved in this phase | ±j
| −v
|
An oral hearing is implemented in this phase | ±g
| − |
All comments of relevant parties are public and evaluated | ±k
| NA |
Preliminary report | ||
The report as written by experts is published | − | +w
|
Relevant parties are involved in this phase | + | + |
An oral hearing is implemented in this phase | ±g/l
| − |
All comments of relevant parties are public and evaluated | ±m
| + |
Review procedure | ||
The criteria for inviting reviewers are public | − | −x
|
All comments of the reviewers are public | − | − |
Final report | ||
Results appraisal is published separately from results assessment | − | +y
|
The underlying evidence for each recommendation is made public | + | + |
Appeal | ||
Appeal possible? | NAn
| +z
|
5.3 Conclusion
6 How should a benefit assessment be implemented?
6.1 Topic identification and prioritisation
6.2 Tendering and awarding the commission
6.3 Defining the research question
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To evaluate and, if required, propose a revision of the problem
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To suggest clinically relevant comparative therapies
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To propose patient-relevant outcomes, including for each relevant party a definition and operationalisation of the term ‘benefit’
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To propose relevant subgroups that could benefit more or less from the intervention
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To suggest a commission-related methodology, including inclusion and exclusion criteria for the selection of literature
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To highlight relevant issues to the external experts in order to inform the development of the protocol and the appraisal