Biotechnology: a special case for health technology assessment?

Many substances produced naturally in a wide range of living organisms have been identified to be of benefit in the treatment of human disease. Current health biotechnologies recreate DNA-recombinant cellular processes in laboratory settings to produce 'natural' therapeutics: these are potentially a step forward from traditional pharmacology which has developed synthetic analogues or sought to extract products from donor material. However, with increasing financial pressures, decision makers require evidence that the benefits of biotechnologies justify their costs. The challenges experienced when evaluating the cost-effectiveness of biotechnologies are explored with reference to three examples: HA-1A human monoclonal antibody, erythropoietin and DNase. Difficulties in economic evaluation are similar to those experienced with conventional pharmaceuticals: use of short-term clinical endpoints rather than meaningful health outcomes, the artificial nature of clinical trial protocols, and uncertainty about the applicability of economic data. However, early clinical and economic assessments are required by decision-makers, particularly where biotechnology products fill major gaps in therapy. The financial structure of biotechnology companies may limit movement towards adequate clinical and economic research for health technology assessment. Governments should negotiate with the industry to promote more relevant studies, and develop policies for the managed introduction of products as evidence on effectiveness and cost expands. New technologies often present additional costs requiring reallocation of existing resources. Careful resource planning is required so that cost-effective innovation are not denied to patients.