How Japan Is Creating New Opportunities in the Field of Regenerative Medicine - SPONSOR CONTENT FROM THE GOVERNMENT OF JAPAN
As biotech companies race toward the opportunities in Japan, no life sciences sector has received more attention, government support, and regulatory reform than the important field of regenerative medicine. Regeneratives are pharmaceutical cell therapy products that replace or restore cells and tissues lost to disease or aging. Some regenerative medicines stimulate our bodies to regrow or repair, so the field can include the use of stem cells for tissue engineering. Japan’s rapidly aging population creates a perfect test kitchen for such regeneratives, especially as the aging trend expands to Europe and North America. Meanwhile, regenerative medicine is at the forefront of scores of research partnerships between private biotech companies and Japanese universities. “The Japanese want to be the regenerative medicine center of the world,” said Colin Lee Novick, managing director of the CJ Partners biotech consulting firm in Tokyo. “To be able to do that, they need to entice companies to come to Japan, and they need to entice their own pharmaceutical companies to license in and obtain the best.” The “regen” boom in Japan is based on a series of reforms and new laws for pharmaceuticals, but especially the landmark Pharmaceuticals and Medical Devices Act (PMD Act) and the Act on the Safety of Regenerative Medicine. The result is the ability to offer conditional marketing approval for a regen so it can move more quickly to commercialization – an approval process widely seen as the fastest in the world. Regens also benefit from what is known as the Sakigake Package of policy changes, which speed up the introduction of innovative medical products. The package focuses on everything from basic research to clinical research/trials, approval reviews, safety measures, insurance coverage, improvement of infrastructure and the environment for corporate activities, and global expansion. It includes a designation system that promotes R&D in Japan aimed at early practical application for innovative pharmaceutical products, medical devices, and regenerative medicines. It also includes a scheme for rapid authorization of unapproved drugs, which speeds the practical application of unapproved/off-label use of drugs for serious and life-threatening diseases. But the benefits don’t stop there, Novick points out. In addition to the conditional approval and Sakigake benefits, Japanese regens arrive in one of the world’s largest markets for pharmaceuticals. The result since 2014 has been a flood of partnerships, research studies, and licensing deals. Gene therapy is also a part of this trend. Agilis Biotherapeutics, a U.S. company, is partnered with Gene Therapy Research Institution of Japan to develop and obtain approval for medicines to treat Parkinson’s disease and another nerve disease. “R&D requires extremely high skills, but manufacturing requires even higher skills. There are very few places that can provide high-quality manufacturing capabilities in the U.S. and also in the world. After a thorough valuation, they came to believe the Japanese Gene Therapy Research Institution had the best capabilities (talent and skills),” said President Katsuhito Asai of Agilis GTRI Japan, a joint venture between Agilis and the Japanese Gene Therapy Research Institution. Foreign companies that have decided to conduct R&D in Japan for innovative medical products are entitled to a research grant from METI to help with their capital investment. In addition, local governments provide their own grants. Agilis GTRI Japan is located in a public-private research park in Kanagawa Prefecture, which has adopted the National Strategic Special Zone concept to cut regulation and offer other benefits for innovation companies. Among the larger partnerships are some iconic Japanese companies. Nikon continues to move into health care by partnering with Lonza of Switzerland – the world’s leading developer of regenerative medicine cells – to build a cell and gene therapy manufacturing plant. Hitachi has partnered with Caladrius Biosciences of the U.S., and Asahi Glass Co. is working with Regeneus of Australia. That these classic Japanese companies known for products in other fields are moving into life sciences underscores the lure of both regens and Japan’s lucrative home markets. Other partnerships or licensing deals involve Athersys of the U.S. and Healios of Tokyo, to develop and produce the MultiStem therapy for stroke victims; Celixir of the U.K., working with Daiichi Sankyo on the Heartcel heart failure medicine; the Belgian firm TiGenix partnering with Takeda Pharmaceuticals on a Crohn’s disease treatment; and Kolon Life Science of South Korea collaborating on the Invossa arthritis drug with Mitsubishi Tanabe Pharmaceutical Co. The mutual benefits of such partnerships were demonstrated when Takeda recently announced it was buying TiGenix. Among the companies directly crediting the conditional approval process in Japan for its interest is Israel’s Pluristem, which is partnering with Sosei CVC to develop placenta-derived cells to speed healing in patients with critical limb ischemia, or CLI. “The development of our CLI program through the accelerated regulatory pathway could allow a more rapid entrance into the sizeable Japanese market, as has been our strategy,” said Zami Aberman, Pluristem’s chairman and CEO. In regeneratives and the broader field of pharmaceuticals, a plethora of non-Japanese companies have formed research collaborations with Japanese universities, which have in recent years moved strongly into commercialization and outside relationships. From the U.S., Merck and Pfizer are working with the University of Tokyo, and the U.K’s AstraZeneca conducts collaborative research with Osaka University and National Cancer Center Japan. Pluristem works with Fukushima Medical University. Pointing out the many deals, collaborations, and new regen opportunities, the consultant Novick advises non-Japanese companies to “definitely jump in. … It’s a nascent and rapid-growth market. Because the cement is still drying on the rules and regulations and guidelines, if you want to be one of the parties that is going to be able to set the tone in this market, you must enter now.” Find more at Japan.go.jp/abenomics.
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by Niv Sultan This week, all the attention’s on Judge Neil Gorsuch, President Donald Trump‘s nominee to become an associate justice of the U.S. Supreme Court. But there’s another position in play: Tomorrow (April 5), the Senate Committee on Health, Education, Labor and Pensions will hold a confirmation hearing for Scott Gottlieb, who’s in line to head the Food and Drug Administration. It would be a return trip to the agency for Gottlieb, who was a deputy commissioner under President George W. Bush. Gottlieb, whose stance is relatively deregulatory, wrote in 2012 that “a few key structural reforms” could improve the FDA’s “counterproductive” culture. He also wrote about the agency’s “cumbersome [drug] approval process,” which dovetails with Trump’s description of the FDA as “slow and burdensome” — though many note that the U.S. drug approval process is already the speediest in the world. But Gottlieb has held posts that could please people on both sides of the aisle. He’s worked at FDA in two other capacities, besides being deputy commissioner, and he’s been a clinical assistant professor at New York University’s School of Medicine since 2011. More controversial is Gottlieb’s history of working with pharmaceutical companies. According to his personal financial disclosure, Gottlieb has been on the boards of directors of eight pharmaceutical companies and one laboratory company, and is still on the board of four of them. He’s also a member of the product investment board at GlaxoSmithKline, the world’s sixth-largest public pharmaceutical company. Gottlieb’s non-board positions in health-related companies have also been numerous, from serving as acting CEO and director of Cell BioTherapy, Inc., an animal medicine company, to being a venture partner at New Enterprise Associates, a venture capital firm that invests in technology and health care companies. Critics believe that appointing Gottlieb, a fellow at the conservative American Enterprise Institute since 2007, would invite conflicts of interest and other ethics troubles. “The major problem is that he has deeply-rooted ties to big pharma that span more than a decade,” said Michael Carome, director of Public Citizen’s Health Research Group. “His ties include receiving hundreds of thousands of dollars in payments to be a consultant and a speaker; he’s a venture partner at a venture capitalist company that funds multiple large and small companies that do work on biopharmaceuticals and medical devices; and he is someone who in addition to being entangled with multiple industry ties, he’s advocated for a dangerous deregulatory approach to the review of medications.” The pharmaceutical industry has rewarded Gottlieb well. Over the past 12 months, GlaxoSmithKline paid him more than $87,000, and Daichii Sankyo U.S. sent over $52,000 his way. Tolero Pharmaceuticals paid him $35,000, and he exercised and sold another $193,111 in Tolero stock options. For more information, including sources of income exceeding $5,000 per year (lots of speaking gigs there) and employment agreements and arrangements that Gottlieb had in the past 12 months, check out his personal finance disclosure statement. For his part, Gottlieb, in his ethics agreement, says that for a period of one year, he’ll recuse himself from FDA decisions involving the companies where he currently holds positions. That year-long firewall would begin as soon as he resigns from the firms. A similar recusal would apply to his consulting clients, but the one-year period would be retroactive to the last date he did work for them. For companies in which he has financial interest, like Collective Health and Golden State Medical, the recusal would last until he divests his stock — but would apply only to issues that, to Gottlieb’s knowledge, have “direct and predictable effect[s] on the financial interests” of the organizations. Carome’s not convinced that those recusals will suffice. “The letter basically affirmed what we already knew: That he’s entangled in a wide web of financial conflicts of interests. No amount of recusal will disentangle himself from all those conflicts of interests,” he said. “He’s going to be involved in decisions that will impact the bottom line of these companies for years.” Alternatively, Paul S. Ryan, vice president of policy and litigation at Common Cause, acknowledged that Gottlieb’s plan of recusal and divestment fits the bill of what’s expected of federal employees. “It sounds like he’s doing the right thing, the required thing, when he divests from the pharmaceutical companies,” he said. But Ryan also raised Gottlieb’s potential to return to work in the pharmaceutical industry after having overseen it. Trump’s executive order on ethics restricts how soon a govenment official can take a job lobbying for an organization; Gottlieb’s case is different, Ryan said. “[W]e’re talking about going to work at a company and getting a big paycheck after having done things to benefit that company during your time as a federal employee.” That situation’s not unique to Gottlieb, of course. James Shehan, head of the FDA regulatory practice at Lowenstein Sandler, is more heartened by than worried about Gottlieb’s background. Regarding what some perceive as potential conflicts of interest, Shehan said, “To me, it’s indicative that he’s familiar with a lot of the issues that are relevant to FDA because he’s been continuously involved with them for many years. And I kind of expect him to be a good FDA commissioner.” “To me, the question is never does someone have a potential conflict of interest — it’s how do you deal with it,” he added. Here are the organizations listed in Gottlieb’s ethics letter, with the recusal terms for each of them. Gottlieb’s Senate confirmation hearing is scheduled to begin at 10 a.m. tomorrow, April 5. -- This feed and its contents are the property of The Huffington Post, and use is subject to our terms. It may be used for personal consumption, but may not be distributed on a website.
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Donald Trump will nominate Scott Gottlieb, a conservative drug industry insider and former FDA and CMS official, to serve as FDA commissioner, industry sources close to the White House tell POLITICO.Gottlieb, a physician, is a far more traditional pick to lead the Food and Drug Administration than other candidates Trump had considered. One, Silicon Valley investor Jim O’Neill, had shocked public health advocates with his comments that the FDA should approve drugs without proof they worked. However, Gottlieb’s close ties with industry are likely to draw Senate scrutiny. A classic Washington insider who has occupied conservative turf in the health care debate for two decades, Gottlieb is a board member or adviser to various drug companies and plays a role in funding the industry through a venture capital firm, New Enterprise Associates.If confirmed, Gottlieb would play a major role in the Trump’s administration’s efforts to speed up the regulatory approval of drugs. Trump, who’s been critical of high drug prices, has often argued that shortening the FDA approval process and cutting regulations would bring prices down.The White House could announce Gottlieb's nomination as soon as Friday.The FDA, which oversees the review and approval of drugs, vaccines and medical devices, and the regulation of tobacco, food and cosmetics, has an annual budget of $5 billion, close to 15,000 employees, and plays a regulatory role in about a fifth of the economy. A slew of FDA user fee programs, in which industry essentially funds half the FDA budget, are up for reauthorization in the fall; it would fall upon Gottlieb, if approved, to shepherd the agreements — negotiated under his predecessor — through Congress.Gottlieb has broad experience in regulating health care and working for the industry. He received nearly $200,000 in payments in 2015 from eight pharmaceutical companies, according to federal database tracking drug industry payments. All were classified as “general” payments, meaning they were for things like travel and lodging, consulting or speaking fees. In 2014, he received more than $160,000 in general payments from companies.Gottlieb has been on GlaxoSmithKline’s research and development board since 2010, according to his résumé, and previously served on its oncology board. He advises Bristol Meyers Squibb on its cancer drugs and Cell Biotherapy, an oncology start-up. Gottlieb was a senior adviser to Vertex Pharmaceuticals, maker of expensive cystic fibrosis drugs, from 2009 through 2016. He holds seats on the boards of drug companies Daiichi Sankyo and Tolero Pharmaceuticals; medical lab company American Pathology Partners; MedAvante, a contract research organization, and Glytech, which makes an FDA-approved insulin dosing support system. He also has served on the board of insurance and medical diagnostic companies.Gottlieb was the FDA’s deputy commissioner for medical and scientific affairs from 2005 to 2007, and chief policy adviser to the CMS administrator in 2004, during implementation of Medicare Part D. Earlier, he was a senior adviser and director of medical policy development at the FDA, where he worked on issues like orphan drugs and combination products. Gottlieb has long been a resident fellow at the right-leaning American Enterprise Institute. He serves on the Federal Health IT Policy Committee, which makes recommendations to the Department of Health and Human Services Office for Healthcare Information Technology. He practices medicine at New York University where he is a clinical assistant professor. Gottlieb's free-market health policy positions are well known, thanks to regular op-eds in The Wall Street Journal and Forbes, and FDA-related testimony to Congress. This past fall, he gave testimony criticizing the FDA’s regulation of generic drugs, during a hearing on pharmaceutical industry competition occasioned by the rising prices of the EpiPen.