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...FDA Commissioner Scott Gottlieb, MD, announced that the Center for Drug Evaluation and Research would be taking steps to modernize the organization and functions of the Office of New Drugs in order to address scientific and medical advances within the industry. The goal is to make the review process more integrated across science and regulatory expertise. Janet Woodcock, director of CDER, plans on elevating the role of FDA scientists and medical officers and providing these officers with more tools and support “to advance the clinical and regulatory principles that the FDA uses to evaluate new drugs for safety and efficacy.”...Other changes will include the development of guidance documents, giving review staff more time with sponsors, and getting sponsors involved earlier in the development process. Engaging disease specialists, academic researchers, regulatory partners at other agencies, and patient groups is also a goal of CDER.

Cut-price copies of an expensive Roche biotech drug for blood cancer have taken 80 percent of the British market since launching last year, saving the healthcare system $113 million a year...The rapid adoption of two so-called biosimilar forms of rituximab from Celltrion and Novartis has been accompanied by discounts of 50 to 60 percent as the National Health Service has used tenders to bring down costs...The situation contrasts sharply with the United States, where regulators have lagged Europe in approving biosimilars while a complex system of rebates offered to insurers by original-brand drugmakers has created barriers to use...The U.S. logjam prompted Food and Drug Administration Commissioner Scott Gottlieb to complain of "rebating mischief" and a "rigged payment scheme"...

A risk-based approach to inspection regulations could result in increased audits of more complex manufacturing sites, the FDA says...The final rule...revises time of inspection requirements and removes the duties of inspector requirements, both contained in section 600​ of the Code of Federal Regulations (entitled ‘Biological Products: General’)...It will affect how often the Food and Drug Administration is inspecting certain facilities and implement a risk-based schedule replacing...the biennial inspection requirement for drug and biological product establishments required in the Federal Food Drug and Cosmetic Act ​ and aligning the requirements with 2012’s Food and Drug Administration Safety and Innovation Act ...“This change clarifies the FDA’s flexibility under FDASIA to inspect facilities either more or less frequently, according to the potential risk the establishment’s operations present, without diminishing public health protections,”...According to Commissioner Scott Gottlieb, the ruling is part of efforts to modernise the FDA’s regulations, remove inefficient policies and reduce costs.

The FDA has been helping dozens of U.S. drugmakers in Puerto Rico get power and supplies to their plants in the wake of the infrastructure disaster left by hurricanes Irma and Maria. But with a shortage of saline getting worse in the U.S., the FDA is moving three Baxter plants on the island to the front of the line...FDA Commissioner Scott Gottlieb today said that state and federal authorities have been responsive to its request that a “subset of critical production facilities,” including those plants that manufacture IV saline bags, get priority in getting fuel for their generators and hooked back to the grid ahead of others...“Unfortunately, most manufacturers are still relying on generator power, and even those that have returned to the electrical grid continue to face interruptions as the grid is rebuilt," Gottlieb said. "We’re hopeful that these companies manufacturing medically important products will see their power needs addressed on an accelerated basis.”...A shortage of some presentations of the medically essential saline solution has fettered hospital care since at least 2014. But the issue immediately got worse when hurricanes knocked down power lines and tore up roads in Puerto Rico, interrupting production and distribution from more than 40 U.S. plants on the island, including Baxter’s three producing saline...

The House is set to take up a controversial “right-to-try” bill next week — and if it passes, the Food and Drug Administration will have to work harder to protect patients than it would if a different version of the legislation were advancing, Commissioner Scott Gottlieb told STAT...right-to-try legislation aims to give terminally ill patients a different pathway to access experimental treatments that are not FDA approved...“In terms of making sure that it balances [access to experimental drugs] against appropriate patient protections, I think the Walden bill gives us less work to do,” Gottlieb said. “With the Johnson bill, we’d have to do a little bit more … in guidance and perhaps in regulation to achieve some of those goals, and I think those are the goals that Congress wants us to achieve.”

When Celgene Corp. revealed receipt of a Refusal-to-File letter for its blockbuster hopeful multiple sclerosis drug ozanimod, shares in the big biotech dropped as much as 10%...The decline is no surprise — such a major disclosure was bound to hit Celgene's stock price because the Food and Drug Administration's decision not to accept the New Drug Application will significantly impact the time when (and if) ozanimod ever gets to market, potentially costing Celgene hundreds of millions in future sales...But not all pharmas seem to be so upfront with shareholders about the letters from the FDA, according to a BioPharma Dive analysis...Public disclosure therefore falls to the company when it receives one of these rejections. Company executives can disclose as much or as little information about the letters as they deem appropriate...There has been controversy over the issue for years related to CRLs (Complete Response Letters) — and public and investor outcry for the FDA to publish the letters...it comes down to what is "material" to investors, something defined by the Securities and Exchange Commission but which also gives companies some latitude...

U.S. drug approvals hit a 21-year high in 2017, with 46 novel medicines winning a green light -- more than double the previous year -- while the figure also rose...in the European Union...recommended 92 new drugs including generics, up from 81...Yet the world’s biggest drugmakers saw average returns on their research and development spending fall, reflecting more competitive pressures and the growing share of new products now coming from younger biotech companies...projected returns at 12 of the world’s top drugmakers were at an eight-year low of only 3.2 percent...

Drugmakers may not be the only ones keeping less-expensive drugs off the market...The Food and Drug Administration has approved nine biosimilars, generic versions of biologic medicines, but only three are available...Manufacturers are using several schemes to "hamstring biosimilar competition," FDA Commissioner Scott Gottlieb said... he worries pharmacy benefit managers have been "complacent participants" in the schemes...PBMs and insurers may stick with branded biologics because they receive discounts from manufacturers on these treatments. That can leave consumers paying for costly treatments when less-expensive ones are available while PBMs make more money on these discounts, known as rebates...Many of these practices persist because high list prices enable lucrative returns across the drug supply chain as the spread between list and net price is carved up and shared among participants...he (Gottlieb) applauded insurers who have recently pledged to pass manufacturer's drug rebates directly on to some of their members...This is a bold action that will help create a fairer, more transparent market...I hope that other insurers, employers, and manufacturers follow their lead. I also hope that your industry will continue to innovate to make it more transparent to pass along these rebates...

The federal government...approved a device made by a private company...that will allow the first domestic production of a medical imaging isotope...a move the government said would enhance national security by reducing the need to transport weapons-grade uranium...The Food and Drug Administration granted the approval to NorthStar Medical Radioisotopes, which said it would begin delivering systems to make technetium-99...the most common isotope in medicine and is used in 40,000 procedures a day in the United States...consumers have long had to depend on a complicated and risky supply chain for the materials...The current process involves shipping weapons-grade, or highly enriched, uranium from the United States to research reactors in Australia, South Africa and Europe where it is irradiated to make molybdenum-99, which decays into technetium-99..."This is a win for our national security," said Peter Hanlon, an official with the National Nuclear Security Administration office of material management and minimization...

The Food and Drug Administration has issued guidance on 3D printing and the role it plays in manufacturing healthcare products...While the guidance focuses largely on medical devices, Commissioner Scott Gottlieb acknowledged in a Monday statement that the innovative technology also holds the potential to disrupt drug development. Already, the agency has approved one medicine crafted with 3D printers, Aprecia Pharmaceutical Co.’s Spritam..."This is likely just the tip of the iceberg given the exponential growth of innovative research in this field," Gottlieb said, referring to the 3D-printed products on the market. "We envision that burn patients in the near future will be treated with their own new skin cells that are 3D printed directly onto their burn wounds. Further down the road, there is the potential for this same technology to eventually be used to develop replacement organs."...The promise of 3D printing, however, could spark new innovation and invention in how drugs, and the devices that deliver them, are formulated and made. In response, the FDA is moving to stay ahead of the technology's advances....