New Drugs at FDA: CDER’s New Molecular Entities and New Therapeutic Biological Products

Innovation drives progress. When it comes to innovation in the development of new drugs and therapeutic biological products, FDA’s Center for Drug Evaluation and Research (CDER) supports the pharmaceutical industry at every step of the process. With its understanding of the science used to create new products, testing and manufacturing procedures, and the diseases and conditions that new products are designed to treat, FDA provides scientific and regulatory advice needed to bring new therapies to market.

Novel New Drugs
The availability of new drugs and biological products often means new treatment options for patients and advances in health care for the American public. For this reason, CDER supports innovation and plays a key role in helping to advance new drug development.
Each year, CDER approves a wide range of new drugs and biological products. Some of these products are innovative new products that never before have been used in clinical practice. Others are the same as, or related to, previously approved products, and they will compete with those products in the marketplace.
Certain drugs are classified as new molecular entities (“NMEs”) for purposes of FDA review. Many of these products contain active moieties that have not been approved by FDA previously, either as a single ingredient drug or as part of a combination product; these products frequently provide important new therapies for patients. Some drugs are characterized as NMEs for administrative purposes, but nonetheless contain active moieties that are closely related to active moieties in products that have previously been approved by FDA. For example, CDER classifies biological products submitted in an application under section 351(a) of the Public Health Service Act as NMEs for purposes of FDA review, regardless of whether the Agency previously has approved a related active moiety in a different product. FDA’s classification of a drug as an “NME” for review purposes is distinct from FDA’s determination of whether a drug product is a “new chemical entity” or “NCE” within the meaning of the Federal Food, Drug, and Cosmetic Act.

Monitoring EGFR mutational status in lung cancer patients

SAN DIEGO—Trovagene Inc. announced recently that it has entered into a clinical collaboration with California-based City of Hope to conduct studies to determine the clinical utility of detecting and monitoring EGFR mutations in lung cancer patients using Trovagene’s Precision Cancer Monitoring platform.

Trovagene“Tracking various alterations in the EGFR oncogene, particularly emergence of the T790M mutation, has potential to improve therapeutic strategies for treating patients with non-small cell lung cancer,” said Dr. Mihaela Cristea, lead investigator and associate professor for the City of Hope Lung Cancer and Thoracic Oncology Program. “We look forward to evaluating Trovagene’s molecular diagnostics for the monitoring of circulating tumor DNA found in both urine and blood, with the goal of delivering highly personalized cancer treatment to improve patient outcomes.”

The clinical study is expected to enroll 75 patients with lung cancer. Primary objectives of the study include evaluating concordance between urinary circulating tumor DNA (ctDNA), blood ctDNA and tumor tissue for determining EGFR mutational status. Additionally, the study investigators will evaluate the quantitative and qualitative performance of longitudinal EGFR mutation monitoring using both urine and blood specimens, as they relate to response to therapy over time. Exploratory objectives include evaluating the feasibility of identifying the TKI-resistant mutation, T790M, in urinary and blood ctDNA at the time of progression.

“Enabling physicians to detect the emergence of problematic mutations in real-time is a key benefit of our Precision Cancer Monitoring platform, and one such mutation is EGFR T790M, which drives treatment resistance in non-small cell lung cancer patients,” said Dr. Mark Erlander, chief scientific officer of Trovagene. “This is an important part of our strategy to realize the full potential of our platform, as we partner with major cancer treatment centers in the U.S. to obtain clinical utility data and to integrate our technology into clinical practice.”

FDA Regulations

The U.S. life sciences industry is a business sector which faces the significant task of developing, FDAtesting and manufacturing pharmaceuticals. Due to the complex environment in which pharmaceuticals are developed, there is a strong need for IS and other quality systems to monitor, manage and control the production processes. The quality systems can drive each of the steps with the product life cycle and is a critical factor for guaranteeing that the optimum quality standards are met.

The FDA plays an important role in this systematic testing process by applying rigorous oversight to the pharmaceutical industry, to ensure high quality standards, efficacy, and the safety that the general public demands. Throughout the research, development, and the manufacturing life cycle of drugs, vaccines, and other biopharmaceutical products, the FDA’s role is that of a principal supervisory agency. Their job is to assure that optimum quality is being achieved, and the best practices are being followed.

So how can you prepare your bio-pharmaceutical company for one of these surprise inspections? Well, one thing you can do is to hire a life sciences consulting firm that specializes in the development of quality systems, in which they can design, or redesign your entire system, to meet FDA regulations. The FDA uses it’s oversight to guarantee that such issues and problems with contaminants and failed processes are quickly sniffed out and identified. Without the help of an experienced consulting firm, your company may fail to take the preventive actions necessary to successfully pass an FDA audit. Because of the severity of the consequences in developing possible faulty pharmaceutical products, the FDA has very little tolerance for variability or deviation from its quality inspection regulations. This means if your products do not pass this FDA quality inspection, you will be shut down, and shut down fast.

The pharmaceutical industry, like any traditional manufacturing industry, uses traditional, older production processes and many companies have outdated information systems. In the past, the work flow in the pharmaceutical industry was optimized for straightforward production including cost reductions, rather than focusing on transparency and quality production. Furthermore, even though many pharmaceutical companies are starting to catch up to the rest of the manufacturing industry, and have begun to develop more sophisticated systems, many companies wouldn’t be able to pass a simple FDA regulatory inspection if they had to. If you want your company to be properly prepared, talk to a pharmaceutical consulting firm that specializes in quality control, and they will be able to prepare your company properly in case of a surprise FDA inspection in your company’s future.

Article Source: Nigel Smart, Author

The CDO Fast Track Advantage

The CDO Fast Track Advantage

A CDO is a Contract Diagnostic Organization that specializes on the development of diagnostics, companion diagnostics (CDx) and in vitro diagnostics (IVD) for the pharmaceutical, biotechnology, research, and diagnostic industries. Unlike other providers with internal biases toward their own products and platforms, a CDO is solely focused on the customer’s goals and objectives.

A CDO can offer a comprehensive array of services including:
• Assay development
• Platform evaluation and validation
• Validation of clinical samples in CLIA-licensed CAP-accredited clinical laboratory
• Project management of all levels of regulatory compliance, processes, applications and approvals
• FDA submissions for 510(k) or Premarket Approval (PMA) clearances
• GMP manufacturing

A CDO provides a complete outsourcing partner to seamlessly initiate and manage the parallel development of companion diagnostic tests in synergy with drug development through FDA approval. A CDO provides integrated services, including diagnostics research, an accredited clinical laboratory, project management, regulatory expertise, manufacturing, and consulting. By combining all of these services under a single organization, a CDO can ensure the fastest and most cost effective path to market.

For more information on what a CDO can do for your company, contact ResearchDx at (Louis Sintasath) or call 858-344-8398.