Pharmacoeconomic Considerations in CNS Drug Development
Recent advances in understanding of the blood–brain barrier (BBB) suggest that we are entering an era in which basic discoveries of BBB structure and function may be parlayed into meaningful disease applications. The development of impactful human therapi
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Pharmacoeconomic Considerations in CNS Drug Development David Gray
Abstract Recent advances in understanding of the blood–brain barrier (BBB) suggest that we are entering an era in which basic discoveries of BBB structure and function may be parlayed into meaningful disease applications. The development of impactful human therapies is a difficult and lengthy process; nonetheless, many researchers desire to see their work applied to the treatment of human disease. Significant financial reward will follow functional solutions to BBB delivery challenges; however, validating a trans-BBB delivery strategy to support and sustain a clinical program is costly and risky and requires multidisciplinary expertise. Investigators must necessarily garner funding and expertise for such campaigns, mainly through government grants, private investment firms, and industrial partnerships. Among the many considerations for researchers looking to advance brain delivery technology are the commercial aspects of the technology and the requirements attached to the funding mechanisms for clinical development. Early choices about delivery modality and target therapy (indication) have important consequences for the development process. An understanding of the forces that currently drive the pricing of therapeutics and the factors considered by potential investors who can fund expensive clinical development programs is helpful for framing a discussion about the pharmacoeconomics of BBB delivery. Complex multimodal delivery technologies may have added challenges for demonstrating safety and significantly higher drug manufacturing costs that can influence the risk–benefit analysis made by potential investors. Both the therapeutic application and the delivery system influence the path to generating commercial or government interest in advancing a particular brain delivery approach toward the clinic.
D. Gray (*) Pfizer Pharmatherapeutics Research, 700 Main Street, Cambridge, MA 02139, USA e-mail: [email protected]
M. Hammarlund-Udenaes et al. (eds.), Drug Delivery to the Brain, AAPS Advances in the Pharmaceutical Sciences Series 10, DOI 10.1007/978-1-4614-9105-7_13, © American Association of Pharmaceutical Scientists 2014
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Abbreviations BBB CNS GMP NIH QALY
13.1
Blood–brain barrier Central nervous system Good manufacturing practice National Institute of Health Auality-adjusted life year
Introduction
As described in Chaps. 1 and 2, blood–brain barrier (BBB) functionally limits the access of many reagents to the brain. Clearly, strategies that overcome the druglimiting aspects of the BBB could be important components to future treatment of CNS-based disease (Bisht 2011; Frank et al. 2011; Jain and Jain 2011; Malakoutikhah et al. 2011; Rajadhyaksha et al. 2011; Shinde et al. 2011; Tian et al. 2011; Tucker 2011). Significant progress in the development of protein, peptide, antibody, protein fusion, stem cell, viral, gene, and other therapeutic modalities (biotherapeutics) has redefined the landscape of investigational cl
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