News About The Industry

‘It’s not working': An FDA insider’s view of where medical innovation falls short

September 7, 2019 | Los Angeles Times

Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, says there is a disconnect between discoveries in the laboratory and medicines that reach the clinic. At first, it sounded like a breakup. “It’s not working, and it won’t work in the future,” said Dr. Janet Woodcock, the U.S. Food and Drug Administration’s top drug regulator. She was speaking to an audience of about 150 researchers who are toiling to discover new medicines that will save lives. The room was silent. “I don’t want to bum everyone out,” she said. “The science is fabulous, but that’s not enough.” To be sure, Woodcock wasn’t looking to sever ties with the researchers, who work at universities, research institutes and pharmaceutical companies. But she did call for major reforms in the scientists’ relationships with regulators, doctors and one another. Without a wide-ranging overhaul, she said, their successes in the lab won’t make it to the patients who need them most. Also see Q&A with Dr. Janet Woodcock at https://www.latimes.com/science/story/2019-09-05/future-of-medicine-fda-janet-woodcock

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A molecule designed by AI exhibits 'druglike' qualities

September 2, 2019 | WIRED

Alex Zhavoronkov, CEO of Insilico Medicine, a startup that generates potential drugs using artificial intelligence, was recently given a challenge by one of his pharma company partners. His team would see how quickly Insilico’s AI could identify new molecules that bind with a protein associated with tissue scarring. Then they’d put the molecules to the test, synthesizing a few of them in the lab to see if the AI was onto something, or only dreaming. Why the rush? It now costs $2.6 billion, by one estimate, to get a new drug to market, and pipelines are only getting slower and more expensive. There’s hope—and hype—that AI could help chip away at that figure by reducing the time and labor before a drug starts clinical trials. The idea is that the same techniques used to generate realistic deepfakes and deftly play Go might be able to decipher the complex rules of drug design and generate molecules from scratch. There are signs AI has potential. In December, Alphabet’s DeepMind debuted AlphaFold, an algorithm designed to predict protein folding—an important step for identifying potential disease targets. It beat the longstanding competition in the pharmaceutical industry, handily. Still, some experts remain skeptical of whether AI can dream up molecules that are both effective and truly practical.

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Elanco to become No.2 in animal health with $7.6 billion Bayer deal

August 20, 2019 | Reuters

Elanco Animal Health agreed to buy Bayer’s veterinary drugs unit on Tuesday in a cash and stock deal valued at $7.6 billion, creating the second largest maker of medicines for pets and livestock and expanding Elanco’s reach online. The deal is the latest in the fast-growing animal health market, which has recently seen Elanco floated by Eli Lilly and Co and rival U.S. drugmaker Pfizer also spinning off its veterinary medicine business. It also adds to the list of assets sold by Bayer, as the German company looks to slash debt from its $63 billion takeover of seed maker Monsanto last year and as it braces for a potential settlement of lawsuits over an alleged cancer-causing effect of weedkiller Roundup.

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FDA updates draft guidance on rare pediatric disease priority review voucher program

July 29, 2019 | FDA News Release

The U.S. Food and Drug Administration today issued a revised draft guidance, Rare Pediatric Disease Priority Review Vouchers – Guidance for Industry. The revised draft guidance incorporates public comments received on the initial draft and provides FDA’s thinking regarding the provisions of the Advancing Hope Act of 2016, which updated the definition of a rare pediatrics disease as one that is a rare disease and one where the disease is serious or life-threatening with the serious or life-threatening manifestations primarily affecting individuals from age zero to 18.

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Epidermolysis Bullosa: FDA seeks to help development of new treatments

July 8, 2019 | Regulatory Affairs Professionals Society

The US Food and Drug Administration (FDA) recently released guidance to help companies develop new treatments for epidermolysis bullosa (EB), which is a debilitating and often fatal rare disease. The six-page draft guidance offers details on considerations for clinical trial design, including trial population (estimates suggest 25,000-50,000 people in the US have EB), discussion of efficacy endpoints (i.e. effects on patients’ signs or symptoms such as itching, pain, blister prevention and wound healing) and special considerations to maximize patient comfort (i.e. allowing photo or video documentation of wounds during routine dressing changes in the home).

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