title: Assessment of biotechnology drugs: what are the issues?
abstract: Biotechnology is increasingly regarded as an important reservoir for the development of new and innovative, but generally expensive, pharmaceuticals. At the same time, concerns about cost containment have triggered a keen interest in evaluating and comparing the values of diverse health care interventions. In this paper we studied the process of assessment and diffusion of biotechnology drugs by studying three cases, i.e. nebacumab, colony stimulating factors and recombinant human growth hormone. These cases are evaluated in a standardised format, concerning safety, efficacy, cost-effectiveness and ethical, legal and social issues. Many factors that determine the fate of a biotechnology drug seemed to be similar to those of 'classical' drugs. The definition and measurement of clinically relevant outcomes has been identified as a key factor in the assessment process. Another important issue is the relatively small population for the primary indications of biotechnology drugs and the subsequent process of broadening of indications. Paradoxically, the current trend towards evidence-based medicine means that we will increasingly have to make decisions based on 'incomplete' knowledge'.
authors.affiliation: Department of Pharmaco-Epidemiology and Pharmacotherapy, Utrecht Institute for Pharmaceutical Sciences, The Netherlands. j.e.f.vanrijkom@pharm.uu.nl
authors.forename: J
authors.initials: J
authors.lastname: van Rijkom
authors.forename: H
authors.initials: H
authors.lastname: Leufkens
authors.forename: D
authors.initials: D
authors.lastname: Crommelin
authors.forename: F
authors.initials: F
authors.lastname: Rutten
authors.forename: A
authors.initials: A
authors.lastname: Broekmans
mesh_terms: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D001709:Biotechnology / Q000639:trends*; D003115:Colony-Stimulating Factors; D003362:Cost-Benefit Analysis; D003625:Data Collection; D004059:Diffusion of Innovation; D017277:Drug Approval; D004345:Drug Industry / Q000191:economics / Q000458:organization & administration*; D013006:Growth Hormone; D006801:Humans; D009426:Netherlands; D011994:Recombinant Proteins; D013673:Technology Assessment, Biomedical / Q000191:economics
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D003115:Colony-Stimulating Factors; D011994:Recombinant Proteins; D013006:Growth Hormone; C068069:nebacumab