Health Policy Impact of Economic, Regulatory, and Patent Policies on Innovation in Cancer Chemoprevention

Chemoprevention agents are an emerging new scientific area that holds out the promise of delaying or avoiding a number of common cancers. These new agents face significant scientific, regulatory, and economic barriers, however, which have limited investment in their research and development (R&D). These barriers include above-average clinical trial scales, lengthy time frames between discovery and Food and Drug Administration approval, liability risks (because they are given to healthy individuals), and a growing funding gap for early-stage candidates. The longer time frames and risks associated with chemoprevention also cause exclusivity time on core patents to be limited or subject to significant uncertainties. We conclude that chemoprevention uniquely challenges the structure of incentives embodied in the economic, regulatory, and patent policies for the biopharmaceutical industry. Many of these policy issues are illustrated by the recently Food and Drug Administration–approved preventive agents Gardasil® and raloxifene. Our recommendations to increase R&D investment in chemoprevention agents include (a) increased data exclusivity times on new biological and chemical drugs to compensate for longer gestation periods and increasing R&D costs; chemoprevention is at the far end of the distribution in this regard; (b) policies such as early-stage research grants and clinical development tax credits targeted specifically to chemoprevention agents (these are policies that have been very successful in increasing R&D investment for orphan drugs); and (c) a no-fault liability insurance program like that currently in place for children's vaccines. The significant progress over recent years in oncology has mainly been in developing new therapeutics to treat patients with established cancers (1). Although chemoprevention agents and cancer vaccines offer great promise, research and development (R&D) investment in these therapies has been relatively limited. Preventive agents face a number of scientific, regulatory, and economic barriers that have kept R&D investment low despite the promise of important medical benefits and outcomes (2). “Chemoprevention” entered the cancer research lexicon in 1976 through the work of Michael Sporn, M.D., (3) and has advanced into U.S. and global markets through new products such as Gardasil® [a quadrivalent human papillomavirus (types 6, 11, 16, and 18) recombinant vaccine]. Some existing agents for cancer therapy or other standard applications are adding new indications for use in cancer chemoprevention or “risk reduction” For example, tamoxifen was a standard breast cancer therapy drug before it was extensively tested in, and approved by the U.S. Food and Drug Administration (FDA) for, breast cancer risk reduction (4, 5); Evista® (raloxifene) was a standard osteoporosis prevention and treatment drug before it also was thoroughly tested and FDA approved for breast cancer risk reduction (5). Investment in basic research and clinical trials has increased for medicines specifically developed for cancer prevention and for re-purposing existing medicines for preventive purposes. However, these new preventive therapies are difficult to bring to commercialization when subjected to existing policies designed to build an armamentarium of chronic and acute treatments and diagnostics. This article will review and analyze how various policy actions would lessen or exacerbate the barriers to R&D investment in cancer chemoprevention. Given that underinvestment in chemopreventive agents is associated with the unrealized promise of sizeable social benefits, it is appropriate to consider policy options that would be applicable to pharmaceuticals in general and to class-specific drugs in particular. The former options would include changes in patent and market exclusivity policies; the latter could involve special R&D tax credits like those used for orphan drugs. These options are discussed below. Authors' Affiliation: Department of Economics and the Fuqua School of Business, Duke University, Durham, North Carolina Received 01/14/2008; accepted 03/06/2008. Grant support: C-Change, a nonprofit organization. Note: Supplementary data for this article are available at Cancer Prevention Research Online (http://cancerprevres.aacrjournals.org/). Requests for reprints: Henry G. Grabowski, Duke University, Box 90097, Durham, NC 27708. E-mail: [email protected]. ©2008 American Association for Cancer Research. doi:10.1158/1940-6207.CAPR-08-0048 84 Cancer Prev Res 2008;1(2) July 2008 www.aacrjournals.org Cancer Research. on June 23, 2017. © 2008 American Association for cancerpreventionresearch.aacrjournals.org Downloaded from Published OnlineFirst April 14, 2008; DOI: 10.1158/1940-6207.CAPR-08-0048