Regulatory Alert

Peter Marks, head of the FDA's Center for Biologics Evaluation and Research, said his top concern for gene therapy's future is manufacturing, and how companies will scale up production to match scientific advances. "When the cost of goods are very high, they help justify when people charge astronomical prices," Marks said in a recent interview with BioPharma Dive. "If we can help see cost of goods and ability to manufacture reproducibly improve, I think that'll be a big thing. That's something we're working on but something that keeps me up at night." The FDA aims to finalize six draft guidance documents by the end of 2019 related to gene therapy, including some that will address specific manufacturing issues, Marks said. Learn More

Catalyst has filed a lawsuit against the U.S. FDA claiming the regulator's recent approval of a new drug is illegal and should be thrown out. The lawsuit is the latest chapter in a bizarre competition between Catalyst Pharmaceuticals and Jacobus Pharmaceutical, two small biotechs that both have FDA-approved drugs for a rare disease called Lambert-Eaton Myasthenic Syndrome, or LEMS. At the core of Catalysts lawsuit are two claims, the first being that the labeling of the drug from Jacobus is illegal. The second claim being that the the FDA violated the orphan drug exclusivity period by approving a competitor after just a few months had passed. Learn More

Genentech's blood cancer drug Polivy earned an accelerated approval from the U.S. FDA due to the complete response rate shown during clinical trials. This marks the fifth blood cancer drug approved for Genentech. Polivy, which was also granted Breakthrough Therapy designation, uses ADC technology developed by Seattle Genetics and licensed to Genentech. The FDA's Accelerated Approval Program allows conditional approval of a medicine that fills an unmet medical need for a serious condition. Polivy was approved two months ahead of its August PDUFA date. However, the FDA noted that further clinical trials are required. Learn More

The US FDA has warned Taipei, Taiwan-based drugmaker Vida International over GMP violations following an inspection of the company's Taoyuan City facility. As a result of the observations made during the inspection, the FDA placed Vida on import alert in March. In the warning letter, the FDA cites Vida for failing to conduct necessary batch testing for one of its products before release. The FDA also says the firm failed to test incoming raw materials, including active ingredients, to determine their identity and whether they conform to written specifications before use. According to the FDA, Vida also prepared an inaccurate batch record for a lot of one of its drugs. Learn More

The U.S. FDA will host a 2-day public workshop on leveraging randomized clinical trials to generate real-world evidence (RWE) for regulatory decisions. The Duke-Margolis Health Policy Center will convene the workshop under a cooperative agreement with FDA to explore how randomized clinical trial designs, including those that incorporate pragmatic design elements, can use real-world data (RWD) to generate RWE in clinical settings. Considerations noted in the discussions will inform the potential usefulness of RWE to support regulatory decision-making. The workshop is part of FDA's ongoing effort to implement the RWE framework unveiled by CDER. Learn More

A draft bill in the U.S. Congress that would overturn a landmark U.S. Supreme Court decision that barred the patenting of human genes and ease other restrictions on patenting software and biomedical inventions is drawing fierce criticism from some scientific societies and patient advocates. This week, the ACLU along with more than 100 other signatories from research institutes and advocacy groups, released a letter to lawmakers arguing the changes would stifle medical research and hinder patients' access to diagnostic tests. The bill aims to address the complaint that recent Supreme Court decisions have created confusing and overly stringent patent eligibility rules. Learn More

The U.S. FDA is "open for business" in considering real-world evidence to clear expanded uses of new cancer drugs, acting agency head Ned Sharpless said this week, but warned industry against relying too heavily on alternative sources of data. Speaking at the BIO conference in Philadelphia, the former National Cancer Institute director acknowledged the rising cost and complexity of clinical trials, and indicated the FDA is aware of how difficult enrolling studies of rare disease subtypes can be. The FDA's willingness to consider alternate sources of data isn't new. Learn More

The UK's Medicines and Health care products Regulatory Agency (MHRA) this week opened a three-month public consultation on its plans to apply analytical quality by design (AQbD) principles to its pharmacopoeial standards. The consultation follows the exploration of AQbD in the International Council for Harmonization's (ICH) recently released concept paper ICH Q14: Analytical Procedure Development and Revision of Q2(R1) Analytical Validation last November. Ahead of the consultation, MHRA conducted a case study that focused on how AQbD principles could be applied to the development of a pharmacopoeial assay procedure for atorvastatin tablets. Learn More

Drugmakers and industry groups are calling for changes to the U.S. FDA's recently released draft guidance, Quality Considerations for Continuous Manufacturing. The 27-page draft guidance was released for comment in February and is part of FDA's effort to encourage the adoption of continuous manufacturing, which the agency believes can improve consistency and reduce the risk of drug shortages. While there is great interest in continuous manufacturing at the FDA, drugmakers have been slow to transition to continuous manufacturing processes. To date only a handful of drugs are manufactured using continuous manufacturing. Learn More

Amarin will get a regulatory decision sooner than expected on an important label expansion for its fish oil-based pill, Vascepa. The company announced this week that the U.S. FDA will decide by September 28th on approving the drug for treating heart risk. The sped-up timeline comes courtesy of a priority review granted by the FDA, setting up a potential approval in late 2019 rather than early 2020. The biotech is still waiting, however, on further word from the agency on whether its application will require an advisory committee review, a critical step in the process that could bring heightened scrutiny on Vascepa.  Learn More

Many people don't believe in evolution. Only one-third of Americans say it occurs through processes like natural selection and no involvement by God. But besides religion, there are several reasons why some reject the fact life evolves, such as not seeing adaptation happening with your own eyes. Scientists have now provided further proof of evolution in action with a video of bacteria evolving resistance to an antibiotic drug. The short film shows the results of a study by microbiologists at the Université Claude Bernard Lyon 1 in France, which reveals how rapidly Escherichia coli can acquire genes that confer the ability to resist the effects of tetracycline. Learn More

The U.S. FDA has approved a new way for cancer drugs to be approved more quickly. The approval for the breast cancer treatment Piqray in combination with fulvestrant and alongside a companion diagnostic - came more than three months ahead of its PDUFA deadline and a little more than five months after it was submitted thanks to FDA's Real-Time Oncology Review (RTOR) pilot and Assessment Aid (AAid) programs. Up until now, the FDA had only approved supplemental new drug applications (sNDAs) and supplemental biologics license applications (sBLAS) under the RTOR pilot. The FDA had never even publicly said that it was expanding the RTOR pilot to NDAs until announcing that Piqray's NDA had been approved. Learn More

The U.S. FDA has approved Zolgensma, the first gene therapy for a type of spinal muscular atrophy. The disease is caused by a defect in a gene that makes SMN, a protein necessary for the survival of motor neurons. Zolgensma uses a re-engineered virus to deliver a functional copy of the defective gene so that SMN protein can be produced. Launching Zolgensma, which at $2.1 million will be the world's most expensive drug, promises to be a big test for Novartis. Alert readers will recall that the biopharma giant spent $8.7 billion to acquire AveXis last spring based in large part on the potential of Zolgensma. Learn More

Scott Gottlieb, who recently stepped down as the head of the U.S. FDA, has rejoined venture capital firm New Enterprise Associates as a full-time investing partner. Gottlieb had been a venture partner with NEA for 10 years before President Trump tapped him to lead the FDA. He's expected to lead investments in life sciences startups and take board seats on behalf of the firm. Gottlieb also recently rejoined the American Enterprise Institute as a resident scholar, and he plans to maintain that position. Alert readers will recall that Mr. Gottlieb resigned abruptly from his leadership position at the FDA just a few months ago. Learn More

A week after finalizing guidance on developing interchangeable biosimilars, the U.S. FDA has drafted guidance for biosimilar developers on the use of comparative analytical studies that can be used to assess whether a proposed product is biosimilar to a reference product. The 28-page draft guidance revises a final guidance from 2015 on quality considerations for demonstrating biosimilarity and serves as a replacement for a 2017 draft guidance on statistical approaches to evaluating biosimilarity that was withdrawn last year after industry questions. Learn More

In line with efforts to assess the impact and sources of the sartan safety scandal, India-based Torrent Pharmaceuticals and Cadila Healthcare separately drew lengthy U.S. FDA Form 483s over, among other issues, failures to thoroughly review unexplained discrepancies. The manufacturing site inspection at Torrent Pharmaceuticals FDA investigators conducted over the course of about a week last month resulted in four inspectional citations, with the 483 largely focusing on that firm's investigations into out-of-specification (OOS) results. The FDA's visit to Cadila Healthcare's site from late April to early May revealed 14 observations of manufacturing deficiencies. Learn More

The U.S. FDA and biopharma industry experts gathered this week in Silver Spring, MD, to discuss how the FDA assesses the benefits and risks of new drugs from the preclinical to post market phases. The meeting was conducted as part of FDA preparations in drafting new guidance in FY 2020 on the benefit-risk assessment of new drugs and biologics. Theresa Mullin, associate director for strategic initiatives at the FDA's CDER, explained how the guidance will be an "articulation of our decision-making context" for benefit-risk assessments "throughout the lifecycle of a medical product." She said the guidance will provide information to help companies communicate and submit data to FDA. Learn More

The U.S. FDA this week released its latest batch of product-specific guidance documents to support the development of generic drugs, with 25 new and nine revised draft guidances. HIV antiretrovirals and antibiotics topped the list of new draft guidances with a total of five, followed by drug products containing active ingredients to treat ophthalmic ailments and cancers. The product-specific guidance is part of the agency's push to spur generic competition. Each guidance provides product-specific recommendations to aid manufacturers in the design of bioequivalence studies in support of abbreviated new drug applications. Learn More

Back in the 1970s and 1980s, the U.S. FDA made clear that at least two adequate and well-controlled studies were necessary to establish a new drug's effectiveness, except in only the rarest of circumstances. Then in 1997, the FDA Modernization Act was passed, and Congress clarified that FDA may consider "data from one adequate and well-controlled clinical investigation and confirmatory evidence" to approve a new drug. However, recently IQVIA released a report finding that 25 of 59 (42%) novel drugs approved in 2018 were approved on the basis of only one trial. And one out of eight approvals relied only on Phase 1 or 2 trials, with no Phase 3 trials. Learn More

In an exclusive interview with NBC News Massoud Motamed, a former inspector with the U.S. FDA, says the regulatory agency struggles to police overseas drug manufacturers who may hide problems in their production lines. Case in point, he cites the valsartan recall and the fact that more than a year before the notices went out, he had tried to sound the alarm on what he flagged as potential systemic problems at two facilities in China and India that produce the active ingredients in generic valsartan and other blood pressure medications. However, the FDA ultimately overruled his recommendation. Learn More

The U.S. FDA has finalized a long-awaited guidance spelling out how biosimilars can achieve an interchangeable status, which means they may be substituted for the reference biologic without a prescriber intervening. No interchangeable biosimilars have been approved in the US yet, and the number of companies seeking approval for an interchangeable has remained at just one, with Boehringer Ingelheim publicly disclosing that it's begun an interchangeability study for its adalimumab biosimilar. But former FDA Commissioner Scott Gottlieb said last month that the final interchangeability guidance will provide sponsors with more certainty on how to develop interchangeable products. Learn More

The U.S. House this week unanimously passed two bills that would increase the utility of the U.S. FDA Orange Book for generic drugmakers and Purple Book for biosimilar developers. The passage of HR 1503, the "Orange Book Transparency Act of 2019," and the passage of HR1520, the "Purple Book Continuity Act of 2019" enables the bills to now proceed to the U.S. Senate. The Orange Book is used as a resource by both brand name and generic drug companies to obtain timely information on medicines. While the more recently created Purple Book includes information on when biosimilars and interchangeable biosimilars were licensed and whether the FDA evaluated the product for reference product exclusivity. Learn More

Acacia Pharma has received a Complete Response Letter from the U.S. FDA regarding its New Drug Application (NDA) for Barhemsys. The letter marks the second for this particular submission and identified continuing deficiencies at the contract manufacturer of amisulpride, the active pharmaceutical ingredient used in Barhemsys. The same issue was raised initially in October 2018 when the company received their first Complete Response Letter from the FDA. Despite the rejection, no concerns were raised by the FDA on any of the clinical or non-clinical data in the NDA and no further studies or data analyses will be required for approval. Learn More

Synthetic CBD from Zynerba Pharma has earned fast track status from the U.S. FDA for its Zygel transdermal CBD lotion to treat symptoms associated with the genetic condition Fragile X. There currently are no drugs to treat the symptoms of Fragile X syndrome, which include developmental problems from learning disabilities to cognitive impairment. Zynerba is in the final stages of a clinical trial of Zygel and hopes to have it on the market by late 2020. The FDA already had granted the gel orphan-drug status, which gives Zynerba a seven-year period to market the drug exclusively in the U.S. as well as granting the company tax credits for clinical research expenses. Learn More

As part of Novartis' new drug application for its recently approved multiple sclerosis drug Mayzent, the U.S. FDA questioned the drugmaker's data practices during a clinical inspection. "During the sponsor inspection, significant data reliability concerns were identified," the FDA said, citing the company with an Official Action Indicated (OAI). The FDA explained how in one study protocol, blinding "was not adequately maintained as specified in the protocol throughout the course of the trial at 62 (21%) of 294 sites. Study personnel were given inappropriate access to the first dose and main databases affecting 285 (17%) out of 1651 total study subjects." Learn More

Manufacturing issues have tripped up two experimental drugs, resulting in the U.S. FDA handing their respective makers Complete Response Letters this week. Heron Therapeutics disclosed this week that the regulator had rejected HTX-011, a non-opioid painkiller for postoperative pain. According to Heron, the FDA wants additional information on chemistry, manufacturing and controls to proceed, along with more non-clinical data. Nabriva Therapeutics, meanwhile, said this week that its drug for complicated urinary tract infections was turned down by the agency. Learn More

A modernization of the way the U.S. FDA reviews NDAs and BLAs will be instituted over the next two years, CDER Director Janet Woodcock told attendees of the Food and Drug Law Institute's annual conference this week. Six active initiatives related to this modernization are ongoing at CDER, including initiatives focused on integrated reviews for marketing applications, IND application review management, post-market safety management, assessing talent, reorganization management and administrative operations. The new integrated reviews, which are currently being phased in, will be used among all CDER divisions beginning in 2020. Learn More

The Seoul Central District Prosecutors' Office has arrested an executive and another employee at Samsung Bioepis, the biosimilar division of Samsung Group, on suspicion of destroying evidence to mask an alleged accounting fraud. The move comes after South Korea's Securities and Future Commission ruled in November that Samsung Biologic violated accounting rules by intentionally inflating the value of Samsung Bioepis and the two employees have said they destroyed evidence on their own but prosecutors are reviewing whether there was an order from management or whether the group's strategic office orchestrated the process. Learn More

The move comes just two months after Bayer, and darolutamide-partner Orion, revealed positive late-stage data for the non-steroidal androgen receptor inhibitor. Known as ARAMIS, the phase 3 program evaluated the safety and efficacy of darolutamide in patients with nmCRPC who are currently being treated with androgen deprivation therapy (ADT) and are at high risk of their cancer metastasising. The drug reduced the risk of this happening, along with risk of death, by 59%, achieving a median metastasis free survival of 40.4 months, versus 18.4 months in the placebo arm. There was also an increase in overall survival of 29% versus placebo. Learn More

Samsung Bioepis has confirmed that the company's biosimilar to Amgen's Enbrel, to be known as Eticovo, has been approved by the US FDA. This is the second US approval of an Enbrel biosimilar, with the first coming in August 2016 for Sandoz's Erelzi, although Erelzi has yet to launch. In the EU, however, both Sandoz and Biogen/Samsung have successfully launched their Enbrel biosimilars. Meanwhile, Amgen and Sandoz are continuing to battle in court over when Erelzi can launch in the US, with analysts predicting anywhere from 2021 to 2029. Learn More

Raising concerns over data collection and quality control, the US FDA has reportedly issued a 10-page Form 483 to Sun Pharma for its Dadra plant. As per published reports, the regulator found deficiencies relating to medicine quality, data collection, maintenance as well as quality control resulting in 11 observations. The observations include lack of written procedures for production and process controls designed to assure that drug products have identity, strength, quality, and purity they are represented to possess. Reportedly, FDA inspectors also made observations specific to shortcomings in data integrity specific to lab records. Learn More

Eli Lilly this week said it is working to pull its cancer medicine Lartruvo from global markets, about three months after the pharma disclosed the drug failed a Phase 3 study. The U.S. FDA conditionally approved Lartruvo in October 2016 as a frontline treatment for certain types of soft tissue sarcoma. However, Lilly's confirmatory trial failed, leading the biopharma to suspend promotion in January. Lilly has set up a program to allow patients already on the drug to carry on with treatment - even after withdrawal - if they've been informed of the study results and risks of continuing therapy. Learn More

AbbVie has announced that the U.S. FDA has approved its Skyrizi drug as a treatment for plaque psoriasis at a time when its blockbuster psoriasis drug, Humira, faces patent pressures. The approval of Skyrizi, an injectable drug, is based on results from AbbVie's global Phase 3 psoriasis program, which assessed the safety and efficacy of the drug in adults with moderate to severe plaque psoriasis. AbbVie said it expects Skyrizi to be available in the United States in early May. Skyrizi, which won regulatory approval in Canada and Japan earlier this year, is part of a collaboration between Boehringer Ingelheim and AbbVie, with AbbVie leading development and commercialization of the drug. Learn More

The US FDA this week announced it will hold a public meeting on May 16th, 2019 to evaluate ways to apply FDA's benefit-risk framework throughout the human drug lifecycle and best approaches to communicating FDA's benefit-risk assessment. After gathering industry, patient, researcher and other stakeholders' input, the agency said it will draft a guidance document in FY 2020 on benefit-risk assessment for new drugs and biologics. The meeting will be convened by Duke University's Robert J. Margolis Center for Health Policy (Duke-Margolis). The meeting will also discuss how relevant patient experience data may inform the benefit-risk assessments. Learn More

Novartis, which this week announced positive interim trial results for its experimental gene therapy Zolgensma for spinal muscular atrophy, has made public that an investigation is underway into whether a second trial death could be related to the treatment. Novartis has filed for U.S. FDA approval of the gene therapy and a decision is expected within weeks. The FDA submission was based on findings from a trial of 15 babies treated with Zolgensma. During which one patient died from respiratory failure, which was deemed by the investigator and an independent monitor to be unrelated to the gene therapy. Learn More

With more than two dozen companies developing bispecific antibodies, the next frontier of cancer therapy may well involve these genetically engineered, recombinant antibodies, which is why the US FDA this week released a new draft guidance to help developers of these products. Currently, only one bispecific antibody is marketed in the US: Amgen's Blincyto, which brought in $230 million in worldwide sales in 2018, up 31% from 2017. But others are coming, and recent safety concerns, such as a partial clinical hold initiated by FDA after Xencor reported two patient deaths from a Phase 1 study of a bispecific antibody in February, may have triggered the need for more FDA guidance. Learn More

In his first "all hands" speech at the US FDA's White Oak campus this week, Acting Commissioner Ned Sharpless explained how he will pick up where Scott Gottlieb left off, and how next week, the FDA will launch a new and improved FDA.gov website. "The work done over the past year to get us here will provide a more accessible, user-friendly site for the public to learn about recalls, get product safety alerts, and other important information about the work we do to protect the public. This will be important," Sharpless, the former director of the National Cancer Institute (NCI), said. Learn More

Novartis has announced that the US FDA has accepted its BLA for ophthalmology candidate, brolucizumab, for the treatment of wet age-related macular degeneration (AMD). However, competition is stiff in the wet AMD space from the likes of Regeneron's Eylea and Roche's Lucentis, among others. With that in mind, Novartis has optioned a priority review voucher so as to expedite a review by the US FDA. According to the biopharma giant, if approved, brolucizumab could be brought to market in the U.S. by the end of this year. The filing was based on encouraging phase III data from HAWK and HARRIER trials. Learn More

Clovis Oncology has announced that it is halting a mid-stage trial testing its lead drug in bladder cancer patients, sending shares down 12 percent. The company says its decision to discontinue the trial was based on recommendations of an independent committee, which suggested that the treatment may not provide a meaningful benefit to patients. However, Clovis said it would continue to test the drug, Rubraca, in combination with other treatments for bladder cancer. Rubraca belongs to a new class of cancer drugs called PARP-inhibitors, which work by blocking enzymes involved in repairing damaged DNA of cancer cells, thereby helping to kill them. Learn More

In the U.S. and Europe, drug regulators approve new drugs based on whether the benefits of the new product outweigh the risks. But neither of the regulators consider if such new drugs are superior to drugs currently marketed. Now, three senior officials at the European Medicines Agency (EMA) are explaining how an added therapeutic benefit criterion or head-to-head comparisons could help to drive more innovative therapies. "The aim of these proposals is to contain the rising cost of what is often perceived as the nebulous concept of 'innovation,'" EMA's senior medical officer, Hans-Georg Eichler, chair of EMA's human medicines committee Harald Enzmann and EMA's Executive Director Guido Rasi write in a commentary published in Nature Reviews Drug Discovery this week. Learn More

U.S. FDA Commissioner Ned Sharpless "should assess the effectiveness of the foreign offices' contributions," a Government Accountability Office report has recommended. GAO's report called attention this week to priority recommendations on the FDA's parent agency, the U.S. Department of Health and Human Services. As the agency continues testing performance metrics and evaluating the strategic workplan of its Office of International Programs, the FDA's acting commissioner was directed to systematically track data and measure whether OIP's activities are specifically targeting drug safety-related issues, such as inspections, import alerts and warning letters. Learn More

The U.S. FDA this week refused to fully review the marketing application for Zogenix's treatment for seizures associated with Dravet syndrome, a rare form of childhood epilepsy. Zogenix's drug, to be branded as Fintepla, uses a low-dose, liquid solution of fenfluramine, which was used in an obesity drug combination that was withdrawn from the market due to evidence of heart valve damage. The FDA, after a preliminary review, cited a lack of certain non-clinical studies to assess the chronic administration of fenfluramine and due to an incorrect dataset. Learn More

The U.S. FDA this week finalized a guidance from 2016 detailing the factors that the agency uses to determine whether a risk evaluation and mitigation strategy (REMS) is necessary for a drug. As defined by the guidance, a REMS is a required risk management plan that can include one or more elements to ensure that the benefits of a drug outweigh its risks. Such elements can include a medication guide, a package insert or a communication plan. And though the use of a REMS is decided on a case-by-case basis, the 10-page guidance explains how the Amendments Act requires the FDA to consider six factors in deciding whether to require a REMS. Learn More

FDA Commissioner Scott Gottlieb and Center for Biologics Evaluation Research (CBER) Director Peter Marks this week warned that the agency will step up its enforcement efforts against companies illegally marketing stem cell therapies. Gottlieb and Marks also said the agency will look into new ways to "delineate an efficient development path" for low-risk stem cell therapies being developed by firms that have filed investigational new drug applications (INDs) and are engaging in the regulatory process " in a responsible manner." While Gottlieb and Marks said that FDA will continue inspecting stem cell suppliers and clinics to ensure compliance, they emphasized that FDA "will not shy away from taking further steps when sees bad actors taking advantage of patients." Learn More

With the overall goal of reducing the number of complete response letters (CRLs) between tentative approval and final approval for abbreviated new drug applications (ANDAs), Teva and Pfizer are calling on the U.S. FDA to elaborate further on some specifics in a draft guidance on tentatively approved ANDAs. The 12-page draft guidance from last January provides recommendations on the timing and content of amendments to tentatively approved ANDAs to help companies reach final approval on the earliest date by which the ANDA may lawfully be approved. Learn More

Due to significant violations of CGMP regulations, the U.S. FDA sent warning letters to four manufacturers of homeopathic medicines in Florida, North Carolina, Oregon and India. The FDA highlighted how one company-Ashville, NC-based King Bio-"continues to concern us because of the low quality of their operation and the threat their products pose to consumers," FDA Commissioner Scott Gottlieb said. Another company, Oakland Park, FL-based Red Mountain, manufactures finished homeopathic drug products "containing ingredients with potentially toxic effects, including snake venom," without an established Quality Unit, the FDA said. Learn More

Outgoing U.S. FDA Commissioner Scott Gottlieb continued his farewell tour of interviews with a detailed discussion with former FDA Commissioner Mark McClellan at the Pew Charitable Trusts. The wide-ranging conversation covered topics like opioids, the regulation of cell and gene therapies, leveraging electronic health record data and whether the FDA should allow drug importation to bring down costs. As for what Gottlieb thinks might be the most enduring change made during his tenure, he pointed to the Center for Drug Evaluation and Research's evolving application process, which is moving away from a paper-based system to a collaborative, cloud-based document. Learn More

U.S. regulators plan to revamp rules governing how medicines are manufactured, in an effort to ensure the safety of the nation's drug supply as recalls of contaminated imports from developing countries widen. A Bloomberg investigation this year found that manufacturers of generic drugs and their key ingredients in China, India and elsewhere, had in some cases destroyed or hidden testing data revealing quality failures from official records - and showed passing results instead. The FDA wants to force drugmakers to do more rigorous testing to make sure that pills used by millions of Americans meets basic safety and effectiveness standards. Learn More

The U.S. Federal Trade Commission has sought additional information from Bristol-Myers Squibb and Celgene and is focusing on the companies' psoriasis treatments as part of its review of their planned merger. Celgene's psoriasis drug Otezla brought $448 million in sales in the quarter ended Dec. 31st, 2018. However, Alert readers will recall that the FTC review is not the only challenge the companies face. Some Bristol-Myers investors are against the planned $74 billion takeover of Celgene, as they see potential risk associated with Celgene's pipeline, and expect a drop in sales of its flagship multiple myeloma drug, Revlimid, when it loses U.S. exclusivity in 2022. Learn More

Duke University will pay $112.5 million to the federal government to settle allegations that researchers submitted applications and reports containing falsified data to win more than two dozen grants from the National Institutes of Health and the Environmental Protection Agency. The allegations were initially made in a whistle-blower lawsuit brought by Joseph Thomas, a research analyst who worked in Duke's pulmonary division. He claimed that another researcher, Erin Potts-Kant, had fabricated data linked to as much as $200 million in federal research grants. University officials said this week that they discovered the possible research misconduct in 2013, after Ms. Potts-Kant was fired for embezzling funds. Learn More

Congressional Democrats in the U.S. have seized on measures in the United States-Mexico-Canada Agreement that establish protections for drug companies, saying they are a boon to the pharmaceutical industry and could undermine efforts to make American health care more affordable. The new trade pact gives biologic drugmakers 10 years of protections against other products that would rely on the data they used to win approval. But Democrats are objecting to the provision, saying that enshrining the time period in a trade agreement could thwart their future efforts to lower health care costs through legislative changes. Lawmakers also argue that other provisions of the trade pact could hinder the development of generic drugs. Learn More

Beginning March 31st, the U.S. FDA Office of the Commissioner will reorganize and make several changes to various offices within FDA, including the Office of New Drugs (OND). With an eye toward efficiency and better connecting the Office of the Commissioner with center directors and other office leadership. A major change is the creation of the Office of Therapeutic Biologics and Biosimilars (OTBB) within OND. According to an email from Center for Drug Evaluation and Research (CDER) Director Janet Woodcock to staff, the OTBB will house policy and scientific review staff and realign staff from the OND Immediate Office to OTBB. Learn More

Novo Nordisk has submitted its oral semaglutide drug, a pill it hopes will transform the diabetes market, for approval in the United States. The world's biggest maker of diabetes drugs said this week that it had used a so-called priority review voucher to speed up the anticipated review time of the drug to six months. The tablet belongs to a blockbuster class of treatments known as GLP-1s that stimulate insulin production. So far, all have been via injection and a pill would make it quicker and easier for diabetics to take their medication. The semaglutide molecule is the backbone of Novo's future growth hopes amid intensifying pricing pressure in its traditional insulin business. Learn More

AbbVie has announced that the U.S. FDA has placed a partial clinical hold on all trials of its cancer drug Venclexta for multiple myeloma, after a review of data found a higher proportion of deaths in the Venclexta arm of the late-stage study compared to those given a placebo alongside other treatment. Patients who are currently enrolled in studies and receiving the therapy may continue with the treatment, but no new patients should be enrolled in trials testing Venclexta for multiple myeloma until further analysis of the data is completed, the drugmaker said. Venclexta is jointly sold by AbbVie and Roche AG in the United States, while AbbVie sells it in other markets. Learn More

Further modernizing generic drug and new drug reviews and development, improving medical device safety and additional efforts to support the opioid crisis will all see additional funding next year, according to the FY 2020 budget justification from the U.S FDA. The 384-page justification breaks down where all the newly requested funding is expected to go, with $27 million for generic drug reviews and includes a proposal to expand current efforts to streamline and automate the review of the drug quality section of abbreviated new drug applications to include other disciplines engaged in such application assessments. Learn More

Modernizing clinical trials is an agency-wide priority. As more diseases are being redefined based on genomic subtype, researchers have more novel targets and more opportunities to precisely modulate or even repair the basic biological drivers of illness. But these opportunities can be delayed or stymied by a clinical research enterprise that is often extraordinarily complex and expensive. Efforts to streamline medical product development based on advancing science can be frustrated by legacy business models that discourage collaboration and data sharing, and the adoption of disruptive technologies that make clinical research more effective. Learn More

The U.S. Department of Health and Human Services (HHS) announced this week that Dr. Ned Sharpless, current director of the National Cancer Institute (NCI) at the National Institutes of Health, will serve as acting commissioner of the U.S. FDA when Scott Gottlieb steps down next month. Sharpless, who has served as NCI director since October 2017, previously served as director of the University of North Carolina (UNC) Lineberger Comprehensive Cancer Center, a position he held since January 2014. No word yet on if he will be nominated by the White House to fill the position permanently and as such be subject to the confirmation process. Learn More

The U.S. FDA, in a warning letter to Pfizer's subsidiary Hospira, says that products made at the company's facility in Tamil Nadu, India are adulterated as a result of data integrity breaches and laboratory testing shortcomings observed during a week-long inspection last spring. According to the FDA, Hospira's microbiology lab did not accurately report microbial growth test results. During the inspection, the FDA requested that Hospira retest multiple batches of its products that were all reported as passing by the company. Each batch was found to be out-of-specification during retesting. Learn More

The U.S. FDA this week approved the 18th biosimilar, Pfizer's Trazimera, which is the fourth approved in the U.S. for Roche's Herceptin. Accoridng to published reports Pfizer is working to make Trazimera available to patients as soon as possible but has not provided a specific launch date. None of the other three approved Herceptin biosimilars have launched in the U.S. Competitors include Merck and Samsung Bioepis' Ontruzant, approved in January; Celltrion's Herzuma, approved in December 2018; and Mylan and Biocon's Ogivri, approved in December 2017. Learn More

In a rare move, the U.S. FDA this month has rescinded two companies' breakthrough therapy designations (BTDs) because of clinical trial data. For Tonix Pharmaceuticals, the FDA informed the company recently that the BTD granted in December 2016 for Tonmya has been rescinded because interim analysis data did not support the continuation of the BTD. Similarly, this week, based on the FDA's review of data from Trevena's Phase 3 studies of oliceridine, the FDA informed Trevena that under the conditions studied, "these data were not sufficient to support the continuation of FDA's previously granted Breakthrough Therapy designation." Learn More

The FDA says it no longer intends to retrospectively modify the names of biologics approved without an agency-designated suffix, according to updated draft guidance on the naming of biologics released this week. Under the naming convention, FDA adds a unique four-letter suffix that is devoid of meaning to non-proprietary names of all biologics, including originators and biosimilars. The draft guidance will revise 2017 final guidance on the topic, in which FDA said the suffix would apply to newly and previously approved biologics. In the draft guidance, the agency says it has decided to assign a unique suffix that distinguishes an interchangeable product from other biologics that share the same core name. Learn More

Teva subsidiary Actavis has received a warning letter from the U.S. FDA regarding quality unit concerns and inconsistencies with its manufacturing processes, among other violations at the company's Davie, Florida site. Of particular concern to FDA inspectors was the sites history of similar cGMP violations in three previous inspections in December 2013, January 2016 and November 2017. "These repeated failures demonstrate that executive management oversight and control over the manufacture of drugs is inadequate," the FDA said. Learn More

Scott Gottlieb, commissioner of the U.S. FDA has resigned, according to reports. Gottlieb will leave the regulatory agency in about a month. Gottlieb intends to spend more time with his family, according to published reports. At 46, he is the father of three children, nine-year-old twins and a five-year-old. He commutes weekly from Connecticut. Since taking over the role of commissioner in May 2017, Gottlieb has aggressively supported the approval of new branded drugs, as well as generics. Additionally, he has taken on multiple public health problems including the opioid epidemic sweeping the country and vaping. His decision to resign is said to have caught many by surprise, including the White House. Learn More

BioPharma companies and clinical trial sponsors have been directed to follow new guidelines for regulatory submissions that would have to be directly submitted to the UK's Medicines and Health products Agency (MHRA) if Britain leaves the EU without reaching an agreement by the end of this month. The guidance, posted this week, describes the steps applicants and sponsors need to follow for access to the new MHRA gateway for UK submissions from day one post-Brexit in a no-deal scenario. It also applies to all current Eudravigilance Gateway users seeking access to the MHRA submissions portal. Learn More

After 24 years in London, the European Medicines Agency (EMA) has officially departed and is on its way to Amsterdam following a Brexit relocation vote in November 2017. The EMA's permanent Amsterdam headquarters, a tailor-made building in the Zuidas business district, are planned for completion next November. On a temporary basis, the Dutch government has granted EMA the Spark building in the Sloterdijk area of Amsterdam until the permanent building is completed. The move to Amsterdam also comes with a time zone switch, which could have an impact on submissions and deadlines. Learn More

The U.S. FDA is backing early adopters of continuous manufacturing and other advanced manufacturing technologies through its Emerging Technology Team, and by requesting funding as part of the president's 2019 budget. The FDA has also issued draft guidance on the development and implementation of continuous manufacturing for brand, generic and over-the-counter drugs, which furthers a global effort among regulatory authorities to encourage adoption and implementation of the practice. Less than four years ago, only one drug made with continuous manufacturing had been approved. Now there are five products from four drugmakers, and more than 20 companies engaging with the FDA. Learn More

The Association for Accessible Medicines and its Biosimilars Council, as well as the Pharmaceutical Care Management Association, are backing a Pfizer petition that calls on the U.S. FDA to do more to counteract untruthful or misleading information on biosimilars. The AAM comment, related to Pfizer's petition, specifically targets the term "non-medical switching" and the concept of interchangeability as areas where originator biologic companies and others have been misleading. Learn More

The U.S. FDA this week issued a draft guidance laying out quality considerations for continuous manufacturing (CM) for new and generic drugs. The 27-page draft guidance is part of the FDA's effort to encourage the adoption of continuous manufacturing and covers quality considerations for solid oral small molecule drugs and builds on feedback from a public consultation launched in 2017. According to FDA Commissioner Scott Gottlieb and CDER Director Janet Woodcock, continuous manufacturing "transforms the traditional, step-wise manufacturing process into a single system that's based on modern process monitoring and controls. Learn More

Miniaturized stirred bioreactors (MSBRs) are gaining popularity as a cost-effective approach to scale-down experimentation. However, realizing conditions that reflect the large-scale process accurately can be challenging. This article highlights common challenges of using MSBRs for scale-down. The fundamental difference between oxygen mass transfer coefficient (kLa) and oxygen transfer rate scaling is addressed and the difficulty of achieving turbulent flow and industrially relevant tip speeds is described. By highlighting these challenges, the article aims to create more awareness of these difficulties and to contribute to improved design of scale-down experiments. Learn More

As part of its push to increase generic competition, the U.S. FDA has published 74 product-specific guidances, including 22 new and 52 revised guidances. Four of the new draft guidances and 45 of the revised guidances are for complex drug products, including 16 products for which there are currently no approved abbreviated new drug applications (ANDAs). Among the new product-specific guidances is help for companies looking to develop generic versions of AstraZeneca's mantle cell lymphoma treatment Calquence, Vertex's cystic fibrosis treatment Symdeko and the antidepressant isocarboxazid, among others. Learn More

A great blog written by Aoife Brennan, CEO of Synlogic, has been posted on the Life Sci VC website this week. An excerpt from which reads "It was apparently random, although I secretly suspect it was karmic punishment for my lack of imagination in writing the 2019 quality goal. It has been 'Maintain the quality management system and documentation in inspection-ready form' for the past 3 years. While we take quality and regulation seriously, our assumption, like that of many early-phase companies, had been that an actual FDA inspection was several years off..." Be sure to read the rest of this posting, it is at once both informative and entertaining. Learn More

Canada's Competition Bureau this week discontinued its investigation into allegations that Johnson & Johnson subsidiary Janssen inhibited the entry of biosimilars that compete with its blockbuster Remicade (infliximab). The Bureau said that although it confirmed that Janssen has engaged in, and continues to engage in, conduct that could raise concerns, there was not adequate evidence "that this conduct was likely to substantially lessen or prevent competition." The Bureau said it will continue to monitor the biologic and biosimilar industries to identify other types of conduct by companies that sell reference biologics that could raise concerns. Learn More

Roche may soon secure U.S. FDA approval for two of its experimental cancer medicines, announcing this week that the regulator had granted priority review to both entrectinib and polatuzumab vedotin. A decision on approval for entrectinib is set to occur by Aug. 18, 2019. If all goes well, entrectinib could be joined on market in the U.S. one day later by polatuzumab vedotin. The FDA has set a target action date of Aug. 19, 2019 for that drug. This comes amid the reality that Roche's three main cancer drugs; Herceptin, Rituxan and Avastin, are nearing the end of their patent protection. Learn More

The U.S. FDA recently warned two drugmakers, Vasco Rx and Samson Pharmaceuticals, for GMP violations at their facilities. The FDA's warning letter to Vasco Rx comes after the agency inspected the company's manufacturing site in March and April 2018. At the end of the inspection in April, Vasco Rx stopped production and later voluntarily recalled all sterile drugs produced in its laminar airflow hood over sterility concerns. In December, the FDA sent a warning letter to Samson Pharmaceuticals over violations stemming from an inspection in January and February 2018 detailing issues with the company's aseptic processing and product testing practices. Learn More

The U.S. FDA has made public the Form 483 that was issued to Immunomedics documenting serious breaches of data integrity at the company's Morris Plains, New Jersey manufacturing facility. Among the 13 observations noted in the document are manipulation of bioburden samples, misrepresentation of a test procedure in the batch records and backdating of batch records, including the dates of analytical results. The FDA notes that it was unable to determine whether data integrity issues may have affected earlier batches because Immunomedics "questioned plant personnel under "attorney/client privilege and no additional documentation is available." Learn More

Biopharma community leaders, drug manufacturers and trade associations alike, have raised questions regarding two U.S. FDA guidance documents on biosimilars, including one explaining how the FDA plans to transfer drugs previously approved under new drug applications (NDAs) to be biologic license applications (BLAs). On this so-called "Deemed to be a License" guidance, which explains the NDA to BLA transition, all but one of the biopharma companies and industry groups took issue with the agency's approach to exclusivity. Learn More

In an email to staff this week, Janet Woodcock, director of the U.S. FDA's Center for Drug Evaluation and Research (CDER) announced that Sally Choe will take over for Kathleen Uhl as Director of the Office of Generic Drugs (OGD) upon Uhl's retirement at the end of the month after serving more than 20 years at the agency. Choe, who currently serves as the deputy director of the Office of Study Integrity and Surveillance (OSIS) within the Office of Translational Sciences (OTS), returned to the agency in 2017 after a six-year stint as a senior director at Parexel overseeing the firm's Asia-Pacific and Japan offices. Learn More

The U.S. FDA regulatory framework for the use of real-world evidence (RWE) drew positive reactions from biopharma companies in comments submitted to the docket, although certain recommendations and proposals were also made. The agency released the framework that describes its RWE program last December and has received 28 comments since then. The comment period remains open, but the submitted comments already offer a look into industry's take on the framework. Regeneron, Sanofi, Pfizer and Novartis are among those that have provided feedback alongside industry groups PhRMA and BIO. Learn More

Daiichi Sankyo has been granted a priority review from the U.S. FDA for its drug pexidartinib. The Japanese drugmaker filed for approval of pexidartinib based on a phase III study (ENLIVEN) in 120 people with TGCT, a non-malignant tumour of the joint or tendon sheath that can cause damage to the joints and surrounding tissues, and is now hoping for FDA approval by early August. The Japanese company acquired the drug when it bought Taiwanese biotech Plexxikon for a little under $1 billion back in 2011. Learn More

The U.S. FDA this week issued a call for nominations for nonvoting industry representatives to serve on 17 of the Center for Drug Evaluation and Research's (CDER) public advisory committees. The agency is also calling for industry groups to participate in the selection process. FDA advisory committees include four types of members, scientific and medical experts in the area of medicine or the scientific and technical field the committee covers, as well as a consumer, patient and industry representative. Industry representatives are charged with acting on the behalf of the regulated industry, but do not represent specific companies and do not vote on matters before the committees. Learn More

In a complaint filed against the U.S. FDA, Vanda Pharmaceuticals called on a federal district court to lift a partial clinical hold on its experimental drug tradipitant. The complaint, filed with the US District Court for the District of Columbia this week, alleged that the FDA acted in violation of the Administrative Procedure Act of 1946 (APA) in imposing the partial clinical hold last December. The hold was imposed on a protocol for a new clinical study and a proposed 52-week open-label extension study for patients who completed Phase II of the tradipitant clinical study originally initiated in 2016, according to court filings. Learn More

The Communist Party of China has expelled a former deputy director of the China Food and Drug Administration. Wu Zhen was ousted from the party following an anti-corruption investigation that began in the wake of the vaccine scandal that roiled China last year. According to the Chinese government, Wu took bribes, engaged in nepotism, accepted offers of trips and invitations to banquets and otherwise abused his position at the Chinese drug regulator. China's Central Commission for Discipline Inspection and the National Supervisory Commission made the allegations following an investigation that began in August. In addition to expelling Wu from the CPC, China has canceled his benefits and confiscated illicit gains. Learn More

The U.S. FDA has announced that Leah Christl, director of the Therapeutic Biologics and Biosimilars Staff (TBBS) in the Office of New Drugs (OND), has decided to leave the FDA and will depart February 22nd, 2019. Christl is moving to Amgen to become the executive director of global regulatory and R&D policy beginning March 11th, 2019. A frequent speaker at industry and other conferences, Christl "was instrumental in policy development and the interpretation and implementation of the Biologics Price Competition and Innovation (BPCI) Act for CDER and FDA, which has led to 17 FDA approved biosimilars to date," according to CDER Director Janet Woodcock and OND Director Peter Stein. Learn More

The U.S. FDA has rejected a Sunovion Pharmaceuticals drug for Parkinson's disease, delaying the company in its bid to compete with Acorda Therapeutics, which has a similar product that was recently approved for the market. Sunovion said this week that the FDA asked for more information to support the application for its drug, APL-130277. But the Marlborough, MA-based company, a subsidiary of Japanese pharmaceutical firm Sumitomo Dainippon Pharma, said that the regulator did not ask for new clinical trials. Learn More

Roche Holding and partner AC Immune SA called a halt to two late-stage clinical trials of their crenezumab drug for early Alzheimer's, the latest in a string of failures to find a treatment for the progressive brain disease. The recent announcement comes after an interim analysis indicated it was unlikely to be effective. Shares of AC Immune fell 65% on the news. Drugmakers such as Eli Lilly, AstraZeneca Plc and Johnson and Johnson have all abandoned trials testing their associated experimental drugs. Learn More

The European Medicines Agency, one of the biggest EU regulators and one of the first casualties of Brexit, has closed its doors in the UK for the last time with the loss of 900 jobs. Staff lowered and folded up the 28 national flags that adorned the lobby in the company's Canary Wharf headquarters in London on Friday night, bidding it farewell before moving to their new offices in Amsterdam. Its departure from London was lamented widely as it marks not just the loss of highly skilled jobs but the UK's central place in pharmaceutical evaluation and monitoring. Learn More

Stressing the need for speed and efficiency, U.S. FDA Commissioner Scott Gottlieb this week offered a look at the FDA's plans around real-world data (RWD) and real-world evidence (RWE) in 2019. The speech at the Bipartisan Policy Center built off a framework, released in December, that sought to begin the conversation on how RWD and RWE can support changes to labeling, including adding or modifying an indication, changes in dose, dose regimen or route of administration; changing or adding new populations, or the addition of comparative effectiveness or safety information. Learn More

Advaxis this week announced receipt of notification from the U.S. FDA that the Company's ongoing Phase 3, randomized, double-blinded, placebo-controlled, pivotal study of axalimogene filolisbac (AXAL) in high-risk, locally advanced cervical cancer (AIM2CERV) has been placed on partial clinical hold. The FDA's recent communication, received late last week, states that the partial hold is related to their requests for additional information pertaining to certain AXAL chemistry, manufacturing and controls (CMC) matters. The Agency did not cite any safety issues related to the trial. However, no new patients can enroll in AIM2CERV until resolution of this partial hold. Learn More

Translate Bio this week announced that the Company has received verbal notification from the U.S. FDA that the Agency has completed its review of the Company's Investigational New Drug Application (IND) submission for MRT5201 and has additional clinical and nonclinical questions. The Company submitted the IND in December 2018 to support the initiation of a Phase 1/2 clinical trial in patients with OTC deficiency. The FDA is placing the IND on clinical hold until these questions are resolved. Learn More

Samsung Bioepis this week announced that the U.S. FDA has approved ONTRUZANT®, a biosimilar referencing HERCEPTIN® approved by the European Commission in November 2017, across all eligible indications, namely adjuvant treatment of HER2-overexpressing breast cancer, metastatic breast cancer and metastatic gastric cancer. ONTRUZANT® is Samsung Bioepis' first oncology biosimilar to receive FDA approval, and will be marketed and distributed in the U.S. by Merck, which is known as MSD outside of the U.S. and Canada. Learn More

As the government shutdown enters its second month, U.S. FDA Commissioner Scott Gottlieb and fellow leaders at the agency will be tasked with difficult decisions on who to furlough. The agency is expected to halt its review of prescription drug applications around the first week of February, and other medical products reviewed under user fee laws will follow suit in the coming months. According to new statistics, of the FDA's 17,397 staffers, 37% are exempt, 31% furloughed, 23% partially exempt/excepted/furloughed and 9% excepted. If the shutdown lasts until PDUFA funds run short, more employees in the exempt bucket will be shifted to the furloughed or excepted. Learn More

The U.S. FDA has approved Merck and Samsung Bioepis' Ontruzant, the third biosimilar approved in the US to Roche's Herceptin. In the US, where no Herceptin biosimilar has launched, there are two other competitors: Celltrion's Herzuma, approved in December 2018, and Mylan and Biocon's Ogivri, approved in December 2017. In Europe, Ontruzant launched in March. There are now four competitors for Herceptin in Europe: Celltrion's Herzuma, which launched in May 2018, Amgen's Kanjinti, which started showing up in June 2018 in Germany; and Pfizer's Trazimera, which was approved in July of 2018. Learn More

An experimental Amgen osteoporosis drug that had sparked concern about cardiovascular side effects has won the backing of an FDA advisory panel. A total of 15 panelists this week voted that the benefits of the drug, romosozumab (Evenity), outweigh its risks, and are enough to support its approval. But many of those panelists also said the drug should carry a warning on its label, and that the FDA should require Amgen to conduct more studies to better understand the heart risks. Learn More

Unimed Pharma, a Slovakia-based pharmaceutical manufacturer, drew a statement of noncompliance after an inspection revealed two critical and 21 major good manufacturing practices (GMP) deficiencies. The statement of noncompliance took issue with the firm's manufacturing operations for sterile products, including aseptically prepared small volume liquids and batch certification. The statement was issued by the Slovakian State Institute for Drug Control (SIDC), following an inspection conducted last December. Learn More

The past year proved to be a big one for the U.S. FDA and the approval of novel drugs. Over the course of 2018, the FDA approved 59 different novel drugs that range for the treatment of various cancers, chronic obstructive pulmonary disease (COPD), traveler's diarrhea, migraine headaches and more. Over the past 190 years, the FDA has averaged an approval of 33 novel drugs each year, with 2018 having the highest number. In 2017, the FDA approved 46 novel drugs, but only 22 in 2016. Learn More

As UK Prime Minister Theresa May's Brexit deal is expected to be rejected today in a House of Commons vote, the EMA has said that after closely monitoring staffers' intentions to relocate to Amsterdam, it currently expects a staff loss of about 25%. The 25% figure might seem like a lot of staffers to lose, but that figure is on the low end of predictions from a survey in September 2017 that found EMA expected to lose at least 19% of its staffers no matter which city was selected. In addition to the staffing losses, the EMA says it will further temporarily reduce or suspend activities in the first half of 2019 as it moves into the final phase of its physical relocation. Learn More

Akorn has announced the receipt of a warning letter from the U.S. FDA following an inspection of the company's Decatur, Illinois manufacturing plant. The warning letter lists violations related to the agency's current good manufacturing practice regulations, which include poor aseptic behavior, as well as those related to environmental and personnel monitoring. The Lake Forest, Illinois-based company also failed to follow "appropriate written procedures" to prevent microbiological contamination of the drugs being produced at the plant, the letter said. The regulator also noted that foreign fibers and other contaminants were found on gloves that were supposed to be sterile. Learn More

Nearly every person who's run the U.S. FDA in recent history agrees the agency should break free from its political supervisors - a rare consensus from commissioners who served under Republican and Democratic administrations alike. In two papers published this week, all seven of the FDA's most recent commissioners wrote that the current setup - in which the agency is a mere subdivision of the Department of Health and Human Services - interferes with the ability of its scientists to protect the health of the public. They described a situation in which a tangled web of responsibilities, along with political overseers who aren't necessarily motivated by science, all make it harder for the FDA to keep people safe. Learn More

The U.S. FDA plans to create a new office to improve the review of new medicines - one that will develop a standardized approach to using personalized medicine, digital data, and patients' own reports, according to Commissioner Scott Gottlieb. Gottlieb will outline the plan for the new 52-person group, called the Office of Drug Evaluation Science (ODES), as part of a talk at the annual J.P. Morgan Healthcare Conference this week. Learn More

Novartis, Regeneron, Pfizer and industry group BIO called on the US Food and Drug Administration (FDA) to expand a recently released draft guidance on master protocols to make it applicable beyond oncology. The 21-page draft guidance on master protocols, also known as "umbrella," "basket" or "platform" trial designs, is meant to help sponsors of cancer drugs or biologics regarding the design and conduct of clinical trials intended to simultaneously evaluate more than one investigational drug and/or more than one cancer type within the same overall trial structure in adult and pediatric cancers. Learn More