Relative efficacy and effectiveness assessment of new pharmaceuticals in three EU member states: current practices and outcome agreement between Belgium, the Netherlands and France

Wilder, Philippe B. Van; Bormans, V; Dupont, Alain

doi:10.1007/s00228-013-1577-6

Eur J Clin Pharmacol

2013

DOI: 10.1007/s00228-013-1577-6

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Relative efficacy and effectiveness assessment of new pharmaceuticals in three EU member states: current practices and outcome agreement between Belgium, the Netherlands and France

Philippe B. Van Wilder

V Bormans

Alain Dupont

Abstract: Our findings reveal that the three similar EU MS under study agree on the REA-outcome of only half of the new drugs. Differences in applied methodology (e.g. inferences from study results, handling of uncertainty) between MS may exist. We suggest that a joint REA approach would benefit from a common understanding and application of the scientific assessment process using shared guidelines.

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Cited by 2 publications

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Besonderheiten von Onkologischen Studien im Rahmen der frühen Nutzenbewertung von Arzneimitteln

Lange

2015

Zeitschrift für Evidenz, Fortbildung und Qualität im Gesundheit

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Until May 31, 2015 the German Institute for Quality and Efficiency in Health Care (IQWiG) conducted 108 assessments for various diseases on the basis of 103 dossiers within the scope of the early benefit assessment of drugs pursuant to the Act on the Reform of the Market for Medicinal Products (AMNOG). 29 of these assessments (28 dossiers) referred to advanced stages of oncologic (including neoplastic-hematologic) diseases. In 21 of these 29 assessments (72%), IQWiG found an added benefit for at least one subpopulation or subgroup, compared to 33% with non-oncologic diseases. For oncologic diseases, the extent of benefit was classified as "major" in six assessments (21%), compared to 5% for non-oncologic disorders. In contrast, the conclusions of the oncologic studies were less certain: only one assessment provided proof (of an added benefit); for non-oncologic diseases, this was the case in eight assessments. A distinctive methodological feature of the available oncologic studies is that, as a rule, treatment switching was planned in the event of progression (normally on the basis of imaging or laboratory findings) and that shortly afterwards the follow-up of important endpoints (adverse events and patient-reported outcomes) was normally discontinued. In particular, the pre-specified option in the study protocol allowing the control group to switch treatment to the experimental intervention after progression ("protocol-permitted treatment switches") makes it extremely difficult to interpret the results beyond the outcome "progression" (or progression-free survival). This treatment switching is mostly justified by reference to ethical necessity. This, however, alleges that the experimental intervention (i. e., the new drug) is superior to the control intervention, which means that circular reasoning is unavoidable. But despite this, oncologic studies are better than their reputation. Hence, so far the results of the early benefit assessment of new drugs (regarding the existence and extent of an added benefit) are clearly better than expected. In this context, the IQWiG methods have not been shown to be extremely conservative. On the contrary: in all cases where IQWiG rated the extent of benefit as "major" for the oncologic indications, the Federal Joint Committee (G-BA) has so far not shared this assessment, and instead rated the extent of benefit to be smaller.

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Besonderheiten von Onkologischen Studien im Rahmen der frühen Nutzenbewertung von Arzneimitteln

Lange

2015

Zeitschrift für Evidenz, Fortbildung und Qualität im Gesundheit

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Are new models needed to optimize the utilization of new medicines to sustain healthcare systems?

Godman

Malmström

Diogène³

et al. 2014

Expert Review of Clinical Pharmacology

116

132

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Are new models needed to optimise the utilisation of new medicines to sustain healthcare systems?Godman B 1,2,3 , Malmström RE 4 , Diogene E 5 , Gray A 6 , Jayathissa S 7 , Timoney A 8 , Acurcio FA 9,10 , Alkan A 11 , Brzezinska A 12 , Bucsics A 13 , Campbell S 14,15 AbstractIntroduction: Medicines have made an appreciable contribution to improving health. However, even high income countries are struggling to fund new premium-priced medicines. This will grow necessitating the development of new models to optimise their use. Objective: Review case histories among health authorities to improve the utilisation and expenditure on new medicines. Subsequently, use these to develop exemplar models and outline their implications. Challenges and proposed models: A number of issues and challenges have been identified including the limited innovation level of new medicines alongside increasing requested prices for their reimbursement especially for oncology, orphan diseases, diabetes and HCV. Models centre on the three pillars of pre-, peri, and post-launch including critical drug evaluation and multi-criteria models for valuing medicines for orphan diseases alongside potentially capping pharmaceutical expenditure Discussion: Proposed models which involve all key stakeholder groups are critical for the sustainability of healthcare systems or enhancing universal access. The models should help stimulate debate as well as restore trust between key stakeholder groups.

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Relative efficacy and effectiveness assessment of new pharmaceuticals in three EU member states: current practices and outcome agreement between Belgium, the Netherlands and France

Cited by 2 publications

References 8 publications

Besonderheiten von Onkologischen Studien im Rahmen der frühen Nutzenbewertung von Arzneimitteln

Besonderheiten von Onkologischen Studien im Rahmen der frühen Nutzenbewertung von Arzneimitteln

Are new models needed to optimize the utilization of new medicines to sustain healthcare systems?

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