Rachel Sherman, MD, Associate Director for Medical Policy, within FDA’s Center for Drug Evaluation and Research, gave a press briefing today. Below is a summary. Read more »

Quality-by-design or “enhanced” approaches to drug manufacturing offer several benefits to industry, including better process understanding and better understanding of the interrelationship of material attributes and process parameters. This knowledge can lead to fewer nonconformances and less rejection and rework, according to several International Conference on Harmonization (ICH) Q11 expert working group members. (Q11 is the pending guideline on drug substance and manufacture.) Such an approach can also bring flexibility, whether it be regulatory flexibility or manufacturing flexibility. PharmTech asked the FDA members of the ICH Q11 working group about this issue and what industry should focus on and expect when applying a QbD approach. Read more »

written by Janet Woodcock, MD, Director for FDA’s Center for Drug Evaluation and Research

Targeting a drug for small subgroups of patients is a new way to find effective therapies. This is often called personalized medicine, and it’s one of today’s most promising areas of new drug development. Last year, FDA approved two important targeted medicines: Xalkori (crizotinib), a lung cancer drug that targets tumors with the abnormal ALK gene, and Zelboraf (vemurafenib), a drug to treat malignant melanomas that have a certain gene mutation. Both drugs were approved with companion diagnostic tests to identify if patients have a susceptible tumor.

Today, the FDA approved Kalydeco (ivacaftor) to treat a specific subgroup of patients with cystic fibrosis (CF). Cystic fibrosis is an inherited genetic disease that affects a person’s lungs and other organs and may lead to an early death. What makes the availability of Kalydeco even more unique is that the drug’s developer, Vertex Pharmaceuticals, teamed up with the Cystic Fibrosis Foundation to develop and study the drug.

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Last night, President Obama issued a call to action for manufacturers that involves reducing outsourcing. His 2012 State of the Union address provided a blueprint for rebuilding America’s economy—and a large component is of that plan is to bring manufacturing back to the US. “Tonight, my message to business leaders is simple: Ask yourselves what you can do to bring jobs back to your country, and your country will do everything we can to help you succeed,” he said. Read more »

Ever wish you were a fly on the wall in another laboratory? Well, a new journal website offers the industry opportunities to do just that—to see through video how other scientists carry out particular experiments and processes. Read more »

Guest blog by William Looney, editorial director of Pharmaceutical Executive

2012 will be a transition year for pharma, one of the most important in the industry’s history of product cycles that spin from plenty to penury. On the positive side, the fires of drug discovery are finally being stoked by a growing understanding of how genomics shape the biology of disease. This is leading to promising new treatments that target critical areas of unmet medical need while also increasing the efficacy of interventions geared to the individual patient. Evidence that these next-generation innovations can advance the science while improving outcomes will hopefully lead to ready acceptance in the market, despite the growing leverage of a much more skeptical and discerning customer base. Read more »

This post was written by Eric Langer, Managing Partner, BioPlan Associates, Inc.

Yes, doing surveys is a pain.  So there’d better be a darn good reason.  Here’s a survey with 7 good reasons that I consider worth the effort.  The industry and participants benefit from the 9th Annual Report and Survey of Biopharmaceutical Manufacturing, and here’s why. Read more »

FDA Commissioner Margaret Hamburg held a special press briefing this morning on Genentech’s Avastin drug and its indications for metastatic breast cancer. She announced that the agency is revoking that indication based on FDA follow-up studies which did not show promising results. Read more »

Biopharmaceutical characterization—especially of unknowns—is becoming far more complex and far higher on the regulators’ radar.  Of particular interest for industry is compliance with harmonized guidelines that address FDA’s concept of a “well-characterized biological product,” which involves being able to measure and control the product’s identity, purity, impurities, potency, and concentration. Read more »

Consortiums are nothing new, but they do seem to be popping up more than usual throughout the pharmaceutical sector lately. Rx-360 and its supply-chain efforts come to mind, as does the IQ consortium on advancing innovation in the quality of pharmaceutical development. There’s also an international pharmaceutical privacy consortium, a consultants consortium, and more.

The fact is, industry seems to be tired of waiting for standard-setting bodies and regulators to take action on solving key challenges such as counterfeiting, economically motivated adulteration, the implementation of quality by design, and so forth. It’s not that the agencies and organizations aren’t working on these issues, but they have to do deal with more legal and governmental bureaucracy than do representatives from various companies sitting around a table over coffee or drinks. Read more »