Biotechnology has contributed to important advances in the healthcare field. Products include various hormones, enzymes, cytokines, vaccines, and monoclonal antibodies, with use in diverse therapeutic areas. The majority of approved biotechnology-derived therapeutic products are recombinant proteins. Many have orphan drug status and, therefore, are used in relatively small patient populations. Newer generation biotechnology products are likely to include small molecules, gene therapy products, and increased numbers of vaccines and monoclonal antibody products. Biotechnology provides the means to develop diverse, innovative, and effective approaches to the prevention, treatment, and cure of human disease.
PNS185 Solutions to the Comparator Question in the Absence of Licensed Comparators? a Retrospective Analysis of Orphan Drug and GENE Therapy Reimbursement in EU5 Markets
