Genetic Engineering & Biotechnology News

AUG 2017

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D community with critical information on the tools, technologies, and trends that drive the biotech industry.

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Genetic Engineering & Biotechnology News | | AUGUST 2017 | 27 Alex Philippidis Two recent approvals underscore what the FDA says is its commitment to precision medicine treatments—a commitment the agency recently said it will carry out by expanding master protocols, case studies, and biomarker qualification standards. In a post on FDA's blog, Janet Wood- cock, M.D., director of the agency's Center for Drug Evaluation and Research (CDER), cited the May approvals of new indications for Merck & Co.'s cancer-fighting anti- programmed cell death protein 1 monoclo- nal antibody Keytruda ® (pembrolizumab) and Vertex Pharmaceuticals' cystic fibrosis treatment Kalydeco ® (ivacaftor). Keytruda won accelerated approval for pediatric and adult patients with unresect- able or metastatic microsatellite instabili- ty-high (MSI-H) or mismatch repair solid tumors that have progressed following prior treatment and for which there is no suitable alternative therapy and colorectal cancer that has progressed following ther- apy with fluoropyrimidine, oxaliplatin, and irinotecan. More significant, as the FDA noted at the time, the Keytruda additional approv- al was granted based on tumor response rates and durability of response, with con- tinued approval to depend upon data from confirmatory trials. "[CDER] has approved more than 25 new drugs that benefit patients with specific genetic characteristics. And we have ap- proved many more new uses—also based on specific genetic characteristics—for drugs already on the market," Dr. Woodcock stat- ed. "Precision medicine holds great prom- ise, but to continue developing targeted therapies, we will need scientific advances in the use and development of 'biomarkers'." Focus on Biomarkers To that end, Dr. Woodcock and Lisa M. LaVange, Ph.D., director of CDER's Of- fice of Biostatistics Organization, wrote a review article published earlier this month in the New England Journal of Medicine ("Master Protocols to Study Multiple Therapies, Multiple Diseases, or Both") discussing master protocols for clinical tri- als, designed to "evaluate more than one or two treatments in more than one pa- tient type or disease within the same over- all trial structure." More master protocols have been devel- oped for studies of cancer therapy than oth- er therapeutic areas, Drs. Woodcock and LaVange noted, due to advances in iden- tifying tumor subtypes or mutations. One such protocol is I-SPY 2, an exploratory- phase platform trial designed to investigate new treatments for biomarker-identified subtypes of early-stage breast cancer in the context of neoadjuvant therapy. I-SPY 2 has been used for evaluating 12 therapies from nine sponsors as of March, of which five have advanced for further study, according to the authors—with dis- cussion under way on an I-SPY 3 master protocol designed to provide evidence of effectiveness for therapies that successfully complete I-SPY 2. Master protocols, Drs. Woodcock and LaVange wrote, are also distinguished by two types of innovation: One is the use of a trial network capable of streamlining trial logistics, improving data quality, and fa- cilitating data collection and sharing. The other is the use of a common protocol that incorporates statistical approaches to study design and data analysis, enabling a broader set of objectives to be met more effectively than would be possible in independent trials. Under the 21st Century Cures Act, en- acted last year, the FDA was required to establish a qualification process for "drug development tools," a category that in- cludes biomarkers as well as clinical out- come assessments. The agency has since created a Bio- marker Qualification Program, through which biomarkers can be qualified for use "in any drug development program under the context for which it obtained qualifica- tion." The qualification process consists of three stages: initiation, consultation, and advice, including preparation and submis- sion of an initial briefing package; and re- view of the full qualification package. As part of the program, CDER last month posted on FDA's website fictional case studies "intended to help patients, pa- tient advocacy groups, health professionals, small businesses, and pharmaceutical and clinical innovators learn more about the role of biomarkers and biomarker qualifi- cation in drug development." n BioLector ® Pro Microbioreactor Develop your BioProcess in a couple of days! EUROPE i n f o @ m 2 p - l a b s . c o m P h o n e + 4 9 - 2 4 0 1- 8 0 5 - 3 3 0 USA i n f o U S @ m 2 p - l a b s . c o m P h o n e + 1 - 6 3 1 - 5 0 1 - 1 8 7 8 2015.07 | vierviertel · Process development · Fed-batch optimization · Media screening · pH profiling · Induction profiling The Microbioreactor Company 32 Microbioreactors + pH Control + Feeding = Full Bioprocess Control MATH CAN BE SO EASY FDA Spotlights Commitments to Precision Medicine, Biomarkers Bioprocessing FDA Emphasizes the Role of Biomarkers in Drug Discovery

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