Piloting international production of rapid relative effectiveness assessments of pharmaceuticals.
BACKGROUND
This article describes the lessons learned from an international pilot assessment using the first version of the HTA Core Model® and Guidelines for rapid Relative Effectiveness Assessment (REA) of pharmaceuticals based on input from three different perspectives: the assessors, the users (health technology assessment organisations) and the marketing authorisation holder.


METHODS
A pilot assessment was performed of pazopanib for the treatment of advanced or metastatic renal cell carcinoma for which 54 individuals from 22 EUnetHTA member organisations from 16 European countries gave their contribution. The work was divided in eight domain teams. Subsequently, results of these domain teams were synthesised in one pilot report. Feedback on the outcomes of the pilot was gathered throughout the project and through structured surveys.


RESULTS
The first version of the assessment was produced in six months and consisted of 55 question and answer pairs, 8 domain reports and a synthesis section that combined the results from the different domains. The organisation of the pilot required intense coordination. Main points of criticism on the assessment were the lengthiness of the document and overlap of information throughout the assessment.


CONCLUSIONS
A reduction in the number of authoring organisations and individuals participating is necessary to avoid information overlap and increase efficiency in undertaking the assessment. Involving several organisations (e.g. five) in an in-depth review could still ensure the benefit of broad participation from various countries. The focus of a rapid REA should be on the first four domains of the Model.
Zorginstituut Nederland & Division of Pharmacoepidemiology and Clinical Pharmacology,Faculty of Science,Utrecht Institute for Pharmaceutical Sciencesskleijnen@zinl.nl.
Sarah
S
Kleijnen
Zorginstituut Nederland & Summaryx Ltd.
Iris
I
Pasternack
Finnish Medicines Agency.
Piia
P
Rannanheimo
GlaxoSmithKline.
Jenni M
JM
Vuola
Rijksinstituut voor ziekte- en invaliditeitsverzekering,Directie Farmaceutisch Beleid & KU Leuven.
Marc
M
Van de Casteele
Hauptverband der Österreichischen Sozialversicherungsträger and University of Vienna.
Anna
A
Bucsics
Directorate for Pharmaceutical Affairs,Ministry for Energy and Health.
Isabelle Zahra
IZ
Pulis
HTA Unit,"A.Gemelli" Teaching Hospital.
Rossella
R
Di Bidino
Institute of Bioethics,"Agostino Gemelli" School of Medicine" School of Medicine.
Dario
D
Sacchini
Italian Medicines Agency.
Simona
S
Montilla
Zorginstituut Nederland.
Payam
P
Abrishami
Directorate for Pharmaceutical Affairs,Ministry for Energy and Health.
Sylvana Magrin
SM
Sammut
Italian Medicines Agency.
Luisa Anna Adele
LA
Muscolo
Finnish Medicines Agency.
Pertti
P
Happonen
Zorginstituut Nederland & Division of Pharmacoepidemiology and Clinical Pharmacology,Faculty of Science,Utrecht Institute for Pharmaceutical Sciences.
Wim G
WG
Goettsch
D020533:Angiogenesis Inhibitors / Q000494:pharmacology*; D002292:Carcinoma, Renal Cell / Q000188:drug therapy; D057186:Comparative Effectiveness Research; D005060:Europe; D006801:Humans; D007191:Indazoles; D007391:International Cooperation; D007680:Kidney Neoplasms / Q000188:drug therapy; D010865:Pilot Projects; D015397:Program Evaluation; D000091402:Prohibitins; D011743:Pyrimidines / Q000494:pharmacology*; D013449:Sulfonamides / Q000494:pharmacology*; D011795:Surveys and Questionnaires; D013673:Technology Assessment, Biomedical / Q000458:organization & administration*
D020533:Angiogenesis Inhibitors; D007191:Indazoles; C000717331:PHB2 protein, human; D000091402:Prohibitins; D011743:Pyrimidines; D013449:Sulfonamides; C516667:pazopanib
International cooperation