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. 2011 Aug 30;3:117–186. doi: 10.2147/CEOR.S14407

Table 3.

Comparison of clinical evidence requirements

Country Centralized reimbursement review/decision-making body (role) Clinical evidence requirements
Topic Preferred clinical outcomes Type Source
Austria

  • Association of Austrian Social Security Institutions (decisions)55

  • Pharmaceutical Evaluation Board (recommendations)56

  • Target patient population and indications (therapeutic claim)

  • Pharmacology

  • Safety

  • Efficacy

  • Effectiveness (across population subgroups)

  • Frequency and duration of treatment55

  • Not specified

 Preference for:

  • Double-blind randomized controlled trials

  • Systematic reviews and meta-analyses of randomized controlled trials complying with internationally recognized guidelines55

  • Published, peer-reviewed studies

  • Unpublished reports and studies may be accepted in exceptional circumstances55

  • Commercial, in-confidence data55
Belgium

  • Minister of Social Affairs (decisions)

  • Commission on reimbursement of medicines/Drug Reimbursement Committee (recommendations)9,60

  • Target patient population and indications (therapeutic claim)

  • Safety

  • Efficacy

  • Effectiveness (across population subgroups)11

  • Morbidity

  • Adverse events/complications

  • Quality of life

  • Overall survival/mortality (life-years gained)

  • Quality-adjusted life years (QALYs) – measured in patients but valued by public/society

  • Other relevant disease-specific outcomes4

  • Final endpoints11

 Preference for:

  • Randomized controlled trials

  • Observational head-to-head comparative studies11,17

  • Effectiveness studies (over efficacy studies)17

  • Minimum of one positive superiority trial on primary endpoints against active control or placebo (if no alternative treatments exist)17

  • Published, peer-reviewed studies

  • Unpublished reports and studies17

  • Abstracts not accepted9
Czech Republic

  • State Institute for Drug Control (decisions)65

  • Safety

  • Efficacy

  • Effectiveness66

  • Not specified

  • All clinical trials138

  • Not specified
Denmark

  • Danish Medicines Agency (decisions)68,69,121

  • Reimbursement Committee (recommendations)68,121

  • Target patient population and indications (therapeutic claim)

  • Safety

  • Efficacy

  • Effectiveness (across population subgroups)38

  • Not specified

 Preference for:

  • Randomized controlled trials comparing pharmaceutical to standard care38

  • Not specified
Estonia

  • Ministry of Social Affairs (decisions)72

  • Pharmaceuticals Committee (recommendations)67

  • Safety

  • Efficacy

  • Effectiveness139

  • Adverse events/complications

  • Side effects

  • Overall survival/mortality139,140

 Preference for:

  • Randomized controlled trials84

  • Published, peer-reviewed studies84
Finland

  • Pharmaceuticals Pricing Board (decisions)73,74,76

  • Pharmaceuticals Pricing Board Expert Group (recommendations)75

  • Target patient population and indications (therapeutic claim)

  • Severity and burden of illness

  • Effectiveness (across population subgroups)76

  • Not specified

 Preference for:

  • Randomized controlled trials comparing pharmaceutical to standard care76

Evidence from other available direct comparative experimental and observational studies, as well as meta-analyses, should be included76

  • Published, peer-reviewed studies76
France

  • Ministry for Health and Social Security (decisions)20,78

  • French National Authority for Health (recommendations)20,78

  • Target patient population and indications (therapeutic claim)

  • Current management

  • Place of technology in care pathway

  • Safety

  • Efficacy

  • Effectiveness (across population subgroups)16

  • Morbidity

  • Overall survival/mortality

  • Quality of life16

 Preference for:

  • Head-to-head, double-blind randomized controlled trials

  • Other direct comparative studies

  • Post-market studies

  • Systematic reviews and meta-analyses of randomized controlled trials complying with internationally recognized guidelines16

  • Published, peer-reviewed studies

  • Current national and international clinical practice guidelines

  • Expert opinion

  • Surveys of practice

  • Commercial, in-confidence data16
Germany

  • Federal Joint Committee (decisions)19

  • Institute for Quality and Efficiency in Health Care (recommendations)19,141

  • Target patient population and indications (therapeutic claim)

  • Severity and burden of illness

  • Safety

  • Efficacy

  • Effectiveness (across population subgroups)8

  • Morbidity

  • Overall survival/mortality

  • Quality of life

  • Adverse events/complications

  • Side effects

  • Duration of illness

  • Health status8,83,142–144

  • Topic specific outcomes identified in consultation with patient organizations18

  • Validated surrogate outcomes83

 Preference for:

  • Randomized controlled trials comparing pharmaceutical to placebo, standard care, or no active treatment8


Evidence from other available direct comparative experimental and observational studies, as well as systematic reviews and meta-analyses complying with internationally recognized guidelines, should also be included8

  • If no treatment alternative exists, well-documented case series acceptable8

  • Published, peer-reviewed studies

  • Commercial, in-confidence data not accepted unless it can be published18
Greece

  • Transparency Committee in the Reimbursement and Medicinal Products (makes decisions)85

  • Safety

  • Efficacy

  • Effectiveness145

  • Not specified

  • Not specified

  • Not specified
Hungary

  • Ministers of Health and Finance (decisions)

  • National Health Insurance Fund Administration (recommendations)88

  • Severity and burden of illness

  • Current management

  • Safety

  • Efficacy

  • Effectiveness (across population subgroups)90

  • Not specified

 Preference for:

  • Randomized controlled trials84

  • Published, peer-reviewed studies90
Ireland

  • Health Service Executive (decisions)91,92

  • Safety

  • Efficacy

  • Effectiveness (across population subgroups)146

  • Morbidity

  • Overall survival/mortality

  • Quality of life

  • QALYs – measured in patients but valued by public/society146

  • All other health benefits accrued by individuals147

 Preference for:

  • Randomized controlled trials147

Evidence from other available direct comparative experimental and observational studies, as well as systematic reviews and meta-analyses complying with Irish Health Technology Assessment Guidelines, should also be included146

  • Not specified
Italy

  • Italian Medicines Agency Technical Scientific Committee (decisions)94

  • Italian Medicines Agency Pricing and Reimbursement Committee (recommendations)95

  • Target patient population and indications (therapeutic claim)

  • Severity and burden of illness

  • Current management

  • Safety

  • Efficacy

  • Effectiveness (across population subgroups)148

  • Morbidity

  • Overall survival/mortality

  • Quality of life149

 Preference for:

  • Randomized controlled trials comparing pharmaceutical to standard care97

  • Evidence from other available direct experimental and observational studies comparing pharmaceutical with standard care148

  • Not specified
Norway

  • Norwegian Medicines Agency (decisions)98

  • Department of Pharmacoeconomics (recommendations)98

  • Target patient population and indications (therapeutic claim)

  • Severity and burden of illness

  • Current management

  • Place of technology in care pathway

  • Safety

  • Efficacy

  • Effectiveness (across population subgroups)34,149

  • Morbidity

  • Overall survival/mortality

  • Quality of life144

 Preference for:

  • Head-to-head, double-blind randomized controlled trials

  • Other direct comparative studies

  • Systematic reviews and meta-analyses complying with internationally recognized guidelines149,150

  • Published, peer-reviewed studies

  • Unpublished reports and studies149,150
Poland

  • Ministry of Health (decisions)99,100

  • Severity and burden of illness

  • Current management

  • Safety

  • Efficacy

  • Effectiveness (across population subgroups)151

  • Morbidity

  • Overall survival/mortality

  • Quality of life151

 Preference for:

  • Randomized controlled trials151

  • Published, peer-reviewed studies

  • Unpublished reports and studies151
Portugal

  • Ministry of Health (decisions)

  • INFARMED (recommendations)44

  • Safety

  • Efficacy

  • Effectiveness (across population subgroups)101

  • Morbidity

  • Overall survival/mortality

  • Quality of life101

 Preference for:

  • Effectiveness studies of target population (over efficacy studies)101

  • Comparative clinical trials

  • Other study designs accepted, but rationale must be provided101

  • Not specified
Scotland

  • National Health Service Scotland (decisions)50

  • Scottish Medicines Consortium (recommendations)

  • Target patient population and indications (therapeutic claim)

  • Severity and burden of illness

  • Current management

  • Place of technology in care pathway

  • “Comparative” safety

  • Efficacy

  • Effectiveness (across population subgroups)128

  • Morbidity

  • Overall survival/mortality

  • Quality of life

  • QALYs (strongly preferred)50

  • Randomized controlled trials required

  • Comparative observational studies accepted50

  • If no head-to head studies available, indirect comparison required50

  • Published, peer-reviewed studies

  • Unpublished reports and studies

  • Expert opinion

  • Submissions from patient and carer organizations128
Slovakia

  • Ministry of Health (decisions)

  • Reimbursement Committee for Medicinal Products (recommendations)105,107,130

  • Target patient population and indications (therapeutic claim)

  • Severity and burden of illness

  • Current management

  • Patient acceptance

  • Safety

  • Efficacy

  • Effectiveness

  • Frequency and duration of treatment106

  • Morbidity

  • Overall survival/mortality

  • Adverse events/complications

  • Quality of life106

 Preference for:

  • Comparative studies84

  • Published, peer-reviewed studies

  • Unpublished reports and studies with negative findings152
Spain

  • Ministry of Health Directorate General of Pharmacy and Health Products; Inter-Ministerial Pricing Commission (decisions)21,108

  • Target patient population and indications (therapeutic claim)

  • Severity and burden of illness

  • Current management

  • Safety

  • Efficacy

  • Effectiveness132

  • Morbidity

  • Overall survival/mortality

  • Quality of life

  • QALYs132

 Preference for:

  • Randomized controlled trials

Evidence from other available direct comparative experimental and observational studies should also be included132

  • Not specified
Sweden

  • Dental and Pharmaceutical Benefits Board (decisions)15,109

  • Target patient population and indications (therapeutic claim)

  • Severity and burden of illness

  • Current management

  • Safety

  • Efficacy

  • Effectiveness153

  • Morbidity

  • Overall survival/mortality

  • Quality of life

  • QALYs (preferred)109

 Preference for:

  • Randomized controlled trials comparing pharmaceutical to standard care109

Evidence from other available direct comparative experimental and observational studies should also be included15

  • Commercial, in-confidence data109

  • Published, peer-reviewed studies

  • Unpublished reports and studies

  • Ongoing studies

  • Submissions from patient and carer organizations51
Switzerland

  • Swiss Federal Office of Public Health (decisions)

  • Federal Drug Commission (recommendations)113–115

  • Safety

  • Efficacy

  • Effectiveness113

  • Not specified

  • Not specified

  • Not specified
The Netherlands

  • Ministry of Health, Welfare and Sport (decisions)

  • Medicinal Products Reimbursement Committee of the Dutch Healthcare Insurance Board (recommendations)116

  • Target patient population and indications (therapeutic claim)

  • Severity and burden of illness

  • Safety

  • Efficacy

  • Effectiveness154

  • Morbidity

  • Overall survival/mortality

  • Quality of life36,155

 Preference for:

  • Head-to-head randomized controlled trials

  • Systematic reviews or meta-analyses complying with internationally recognized guidelines35,156

  • Published, peer-reviewed studies

  • Unpublished reports and studies

  • Commercial, in-confidence data

  • Expert opinion154
United Kingdom

  • National Institute for Health and Clinical Excellence (decisions)

  • Technology Appraisals Committee (recommendations)7

  • Target patient population and indications (therapeutic claim)

  • Severity and burden of illness

  • Current management

  • Place of technology in care pathway

  • “Comparative” safety

  • Efficacy

  • Effectiveness (across population subgroups)29

  • Morbidity

  • Overall survival/mortality (life years)

  • Quality of life157

  • QALYs – measured in patients but valued by public/society29,158

 Preference for:

  • Head-to-head randomized controlled trials conducted in “naturalistic” settings

  • Effectiveness studies of target population (over efficacy studies)

  • Systematic reviews or meta-analyses complying with internationally recognized guidelines159

Evidence from other available direct comparative experimental and observational studies should also be included159Registries, case series, and follow-up studies also accepted159

  • Published, peer-reviewed studies

  • Unpublished reports and studies

  • Commercial, in-confidence data159

  • Submissions from patient and carer organizations159

  • Information from health care professional associations, administrators, government, and manufacturers157
Wales

  • Ministry for Health and Social Services (decisions)

  • All Wales Medicines Strategy Group (recommendations)120

  • Target patient population and indications (therapeutic claim)

  • Severity and burden of illness

  • Current management

  • Place of technology in care pathway

  • “Comparative” safety

  • Efficacy

  • Effectiveness (across population subgroups)28

  • Morbidity

  • Overall survival/mortality

  • Quality of life

  • QALYs (preferred)28

  • All types of clinical studies accepted, but greater importance given to high quality ones28

  • Published, peer-reviewed studies

  • Unpublished reports and studies

  • Expert opinion

  • Submissions from patient and carer organizations describing experiences of those who have taken the pharmaceutical28