Regulatory Alert

The Court of Justice of the EU this week ruled that the European Medicines Agency (EMA) acted lawfully when it released certain toxicology reports and a clinical study report from two sponsors that wanted to keep that information confidential. The cases centered around PTC Therapeutics and MSD Animal Health, which claimed that EMA's disclosure of a clinical study report, in the case of PTC, and five toxicology reports from MSD Animal Health would undermine their commercial interests. But the Court of Justice concluded, siding with the General Court ruling from February 2018. Learn More

Later this month, the US FDA will formally launch its Drug Risk Management Board (DRMB) to coordinate safety activities among offices, Center for Drug Evaluation and Research (CDER) Director Janet Woodcock said in an email to staff. Woodcock says the DRMB is a center-level body that will also rapidly analyze and resolve drug safety issues by facilitating decision making across CDER on major clinical and quality-related safety issues with marketed products. The board is also expected to help foster better communications across the center. Learn More

As part of efforts to better align scientific advice across the EU, beginning February 1st, a dozen national competent authorities (NCAs) will participate in a pilot project to allow drug developers to obtain two NCA opinions simultaneously. The multi-national discussions aim to provide two NCA opinions within one application, in addition to earlier opinions, better explanations for possibly conflicting opinions, increased interactions between NCAs and the potential for further alignment or clarification among NCAs on different regulatory positions and/or requirements. Learn More

The US FDA last month offered a new Manual of Policies and Procedures (MAPP) to explain how the Center for Drug Evaluation and Research (CDER) reviews risk evaluation and mitigation strategy (REMS) assessment reports submitted to the agency. Although the statute does not specifically describe how companies should conduct their assessments, the agency has released two draft guidances (one on survey methodologies and another on planning and reporting REMS assessments, both from last January) and now, this new MAPP, effective December 2019, for further explanation. Learn More

Industry groups, the Mayo Clinic, Regeneron and others are seeking more clarity from the US FDA's revised draft guidance on clinical decision support (CDS) software. The 27-page draft from September builds on a previous draft from 2017, with which industry also raised concerns. The latest draft clarifies the categories of CDS software subject to FDA oversight, as well as the low-risk categories of CDS software for which FDA does not intend to enforce regulatory requirements and CDS categories that do not meet the definition of a device. Weighing in on the draft, industry group AdvaMed praised its discussions of what types of software are, or are not, subject to FDA's oversight. Learn More

As some insulin, human growth hormone and other products transition on March 23rd from new drug applications (NDAs) to biologics license applications (BLAs), the recently passed government spending bill included a further tweak to add new proteins to the transition. The NDA to BLA change effectively means that any follow-on products for these NDAs will need to win approval as biosimilars after March. The transition was created by the Biologics Price Competition and Innovation Act of 2009, which clarified the statutory authority under which certain protein products will be regulated by amending the definition of a "biological product" to include a "protein". Learn More

The FDA this week released two warning letters recently sent to China-based contract manufacturer Huaian Zongheng Bio-Tech and New Jersey-based drug manufacturer Health Pharma for GMP violations. For Huaian Zongheng FDA inspectors said the firm released over-the-counter drugs without conducting identity or strength testing for each active ingredient. The firm also failed to test incoming components used to manufacture its drug products to determine their identity. While for Health Pharma, FDA inspectors, following a visit to the Rahway, New Jersey site last May and June, said the firm's quality unit (QU) lacked the appropriate oversight of its drug manufacturing operations. Learn More

As API quality concerns linger, China's National Medical Products Administration (NMPA) has agreed to work with the European Commission's DG Sante, the European Medicines Agency (EMA) and others to identify similarities and differences between the Chinese and EU regulatory systems for APIs. The commission says it has created a budget to conduct a gap analysis, which would be used to identify Chinese training needs in the API space. EU experts will also conduct a fact-finding visit to China to assess the regulatory, control and enforcement system governing the implementation of GMP standards, according to the agreement forged at a bilateral EU/China meeting in October 2019. Learn More

The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended 30 new active substances (NAS) for marketing authorization in 2019, down from 42 the previous year. The agency also recommended just six new orphan medicines for authorization, the fewest in recent years and a drop from the 17 new orphan medicines recommended in 2018. In comparison, the US FDA (FDA) Center for Drug Evaluation and Research (CDER) approved 48 novel drugs in 2019, down from 59 in 2018, 21 of which were orphan products. While most of the products EMA recommended in 2019 have also been approved by FDA, four have not been approved in the US. Learn More

The US FDA this week said it plans to withdraw the approval of 249 abbreviated new drug applications (ANDAs) after their manufacturers repeatedly failed to submit annual reports for the generic drugs. The FDA says the manufacturers will have the opportunity to request a hearing to appeal the decision by filing a written request and providing the agency with a rationale for why the ANDA should not be withdrawn. Some of the companies impacted include major drugmakers and their subsidiaries, including Pfizer Laboratories, Novartis' Sandoz and Fresenius USA, although the vast majority of the companies are smaller manufacturers. Learn More

As the use of complex innovative design (CID) trials for new cancer treatments continues, a group of UK professors from the University of Oxford, Birmingham and others, as well as representatives from the UK's Medicines and Healthcare products Regulatory Agency and Pfizer have offered a series of recommendations on the flexible trials. Published this week in the British Journal of Cancer, the consensus statement explains how unlike conventional trials in which patients are recruited by tumor of origin, patients enrolled in one kind of CID trial, known as "master protocol" trials, incorporate molecular biomarkers. Learn More

The Pharmaceutical Inspection Co-operation Scheme (PIC/S) this week released a new guideline explaining its pre-accession procedure for competent authorities wishing to join the inspection scheme. Membership in PIC/S is open to any competent authority "having the arrangements necessary to apply an inspection system comparable to that enforced by" existing members. "The scheme is primarily based on mutual confidence between participating authorities (PA). Such confidence can only be achieved on the basis of a thorough knowledge of each other's inspection systems and inspection practice and standards," PIC/S writes. Learn More

The US FDA has issued a draft guidance on requesting feedback from the agency on scientific and regulatory questions during the development of a combination product. The 18-page draft guidance fulfills an obligation under the 21st Century Cures Act to publish guidance on best practices when seeking feedback on combination products and to explain how combination product agreement meetings (CPAMs) work. The guidance also details the submission process and what information should be included in CPAM requests and outlines how the agency will respond to such requests. Learn More

Following its Brexit-related departure from London, the European Medicines Agency's (EMA) staff has been reduced from 897 staffers to 775 as noted in an update from a board meeting in its new home in Amsterdam. At the same meeting, the EMA board also agreed to the mandatory use of the ISO standard for individual case safety reports for the reporting of suspected side effects of medicines. The board also signed off on the EMA's budget for 2020, which is $399 (€358) million. And for the new clinical trial regulation, the EMA is moving forward with a proposal to begin an audit of its Clinical Trial Information System in December 2020. Learn More

The US FDA this week issued a draft guidance explaining how drug-device and biologic-device combination product sponsors can bridge data from earlier stages or development or other development programs to support an application. In the 14-page draft guidance, the FDA describes how sponsors can develop an analytical framework for identifying information gaps that could be addressed with bridging. The draft provides three examples of bridging scenarios for different drug-device combination products. The draft guidance comes just after the deadline set in the FDA's Prescription Drug User Fee Act goal letter to issue guidance on the topic by the end of FY2019. Learn More

The European Medicines Agency (EMA) this week announced the launch of a two-year pilot that will allow regulators across the EU, US, UK, Australia and Canada to share information from good manufacturing practice (GMP) inspections of sterile medicines manufacturing sites. Specifically, the pilot will enable information sharing from GMP inspections of manufacturers located in countries that are not participating in the pilot and allow participants to organize joint inspections. The pilot, which EMA says will last for a minimum of two years, is modeled after an earlier joint inspection program for active pharmaceutical ingredients (APIs), which was piloted in 2008 and fully implemented in 2012. Learn More

After years of criticism from the FDA, Sun's Halol plant still can't get through an FDA inspection without the agency finding some deficiency. In June, it was just one observation, but a just completed inspection ended with a Form 483 for Halol with significantly more. The Indian drugmaker has recently reported that an inspection which took place from December 3rd through December 13th had resulted in the FDA citing the key facility with eight observations. It didn't say what issues were uncovered but insisted it was committed to working with the FDA to fix these problems as well as to improve its manufacturing systemwide. Learn More

The US FDA has issued a draft guidance explaining its qualification process for drug development tools (DDTs) in line with the 21st Century Cures Act. The 20-page draft guidance comes just ahead of the deadline set in the Cures Act and fulfills some of FDA's Prescription Drug User Fee Act (PDUFA VI) commitments to enhance its DDT qualification pathway for biomarkers. Once qualified, the FDA says a DDT can be used within its context of use (COU) to support regulatory submissions, including investigational new drug applications (INDs), new drug applications (NDAs) and biologics license applications (BLAs) for any drug or biologic. Learn More

The full US Senate voted this week 72-18 to confirm Stephen Hahn, chief medical executive of the MD Anderson Cancer Center in Houston, to be the next commissioner of the US FDA. The vote came just over a month since he was nominated, although in that time little information has come to light about how he plans to direct the FDA. Still, senators on both sides of the aisle seemed convinced that he'll be able to handle the job. Learn More

The US reliance on imported pharmaceuticals and ingredients is rising as foreign drug facility inspections decreased by about 10% from 2016 to 2018. Part of the reason for the decline: The US FDA said it's still struggling to hire new inspectors. Testifying under oath before the House Subcommittee on Oversight and Investigations of the Committee on Energy & Commerce this week, Janet Woodcock, director of FDA's Center for Drug Evaluation and Research, defended the FDA's approach and discussed some of the vulnerabilities of the agency's foreign inspection program. She said the FDA is looking to hire 50 new inspectors, but there are difficulties. Learn More

The US FDA recently warned two US-based drugmakers, Teligent Pharma in Buena, NJ, and OHM Pharma in Mineral Wells, TX, for good manufacturing practice (GMP) violations at their respective facilities. In its warning letter to Teligent Pharma, the FDA cites the company for failing to thoroughly investigate out-of-specification (OOS) test results. While the FDA's warning letter to OHM Pharma comes after a 10-day inspection in May that uncovered issues with the company's cleaning practices, laboratory controls and manufacturing practices. Learn More

Regulators including the US FDA, EMA, Health Canada and the UK's MHRA are looking into whether formulations of the diabetes drug metformin contain N-nitrosodimethylamine (NDMA) impurities. The announcements come after Singapore's Health Sciences Authority (HSA) recalled three out of 46 locally marketed metformin medicines after determining they contained NDMA "above the internationally acceptable level." The three drugs were marketed by Singapore-based Glorious Dexa Singapore and Pharmazen Medicals Pte Ltd. According to HSA, the risk posed by three medicines is low as they "have only been supplied locally for a short period of time since last year." Learn More

Ipsen has announced that the U.S. FDA has placed a partial clinical hold effective immediately for studies conducted under IND120181 and IND135403 evaluating the investigational drug candidate. The partial clinical hold was issued following recent safety reports submitted by the company to the FDA of cases of early growth plate closure in pediatric patients with FOP treated with palovarotene. The FDA has placed the studies on partial clinical hold pending review of additional details regarding these events and plans to issue additional requests for information within the next 30 days. Learn More

As manufacturers of popular heartburn drugs, like Zantac, continue to pull their products from shelves, the US FDA is now telling the companies to test their drugs for an impurity, known as N-nitrosodimethylamine (NDMA), before making them available. The new requirement comes as earlier last month the FDA noted that although many of these NDMA levels observed via FDA testing are much lower than the levels some third-party scientists first claimed, "some levels still exceed what the FDA considers acceptable for these medicines," said Janet Woodcock, director of FDA's Center for Drug Evaluation and Research. Learn More

The US FDA this week sought comments on whether it should run a voluntary pilot program to evaluate the toxicology and quality of novel excipients. While the agency does currently review new excipients as part of an investigational new drug application (IND) or a marketing application (NDA or BLA), FDA explains how certain novel excipients may provide public health benefits, such as improved drug delivery or utility in abuse-deterrent opioid formulations. The term "excipient" is defined by FDA as any ingredient intentionally added to a drug or biologic that is not intended to have a therapeutic effect but may improve product delivery. Learn More

The US FDA has finalized the guidance on adaptive clinical trial designs for drugs and biologics. The 33-page guidance, which finalizes a draft version released for comment in September 2018 and replaces an earlier guidance from 2010, sets out the FDA's recommendations on adaptive trial design principles and the information the FDA will review from adaptive studies submitted as part of investigational new drug applications (INDs), new drug applications (NDAs), biologics license applications (BLAs) and supplemental applications. While the final guidance is largely the same as the draft version, the FDA says it has revised the guidance to clarify its recommendations for Bayesian adaptive designs... Learn More

PIC/S last week published a draft recommendation offering guidance for GMP inspectors seeking to evaluate the effectiveness of a company's pharmaceutical quality system (PQS) in relation to risk-based change management. The 8-page draft recommendation, which is not open for comments, will be applied on a 6-month trial basis by PIC/S participating authorities, which include the US FDA, and other regulators across Europe, Australia, Canada, South Africa, Turkey, Iran, Argentina and more. The document was created in recognition of the fact that the PIC/S GMP Guide requires companies to demonstrate the effectiveness of their PQS and to apply quality risk management (QRM) principles to change control activities. Learn More

The U.S. FDA has approved an expanded indication for Sanofi's Toujeo for blood sugar control in adult and pediatric patients who are ages six years and older. Previously, Toujeo was approved only for adults aged 18 years and older. The company announced the results of the clinical trial earlier in the month. It was the first randomized, controlled trial to compare Toujeo to Gla-100 in this patient population. The trial met its primary endpoint, demonstrating comparable decreases in average blood sugar over six months with both treatments and similar risk of low blood sugar events. Based on the data, the EMA's Committee for Medicinal Products for Human Use recommended the expanded label in Europe. Learn More

AstraZeneca has announced that the U.S. FDA has given its go ahead for the company's Calquence drug to treat chronic lymphocytic leukemia (CLL), one of the most common types of leukemia in adults. The drug was approved roughly two months after being granted a Breakthrough Therapy Designation by the U.S. FDA in collaboration with drug watchdogs in Canada and Australia. The decision to also approve Calquence for the treatment of small lymphocytic lymphoma, a similar disease, will pit the AstraZeneca drug against AbbVie and Johnson & Johnson's established CLL treatment Imbruvica. Learn More

The UK's MHRA has announced the fifth recent recall of the heartburn medicine ranitidine due to possible contamination with the carcinogen NDMA. The recall, which affects 13 over-the-counter ranitidine medicines marketed by four different companies, follows four previous recalls in October and November and comes as international regulators including the US FDA and EMA investigate the source of the impurities. The FDA previously said the levels of the impurity in ranitidine drugs for heartburn and also in the chemically similar ulcer drug nizatidine "are similar to the levels you would expect to be exposed to if you ate common foods like grilled or smoked meats." Learn More

The US FDA this week released product-specific guidance documents for 81 active pharmaceutical ingredients to aid generic drug development, including 28 new draft guidances and 53 revised draft guidances. The guidances, when finalized, are intended to promote generic competition by clarifying the FDA's expectations for the studies required to demonstrate that a generic drug is equivalent to a reference listed drug. So far, the FDA has issued nearly 1,800 product-specific guidances. The FDA is committed to issuing product-specific guidances for 90% of non-complex new chemical entity new drug applications (NDAs). Learn More

The World Health Organization (WHO) recently drafted a new guideline on data integrity that explains the important elements to ensure the reliability of data and information in the production and control of biopharmaceuticals. The WHO echoed similar concerns expressed by the US FDA in noting an uptick in recent years of the number of observations regarding the integrity of data, documentation and record management practices during inspections of good manufacturing practice (GMP), good clinical practice (GCP) and good laboratory practice (GLP). While recommending companies use a written data integrity policy, the guideline outlines the principles of data integrity and good documentation practices before delving into quality risk management. Learn More

Earlier this month, the European Commission's (EC) Pharmaceutical Committee met to discuss how the use of duplicate marketing authorization applications (MAAs) - including the use of duplicate reference biologics, dubbed "autobiologicals" - can impact the availability and pricing of biological products and biosimilars. According to the EC, requests for duplicate MAAs have increased steadily, and this is a trend that is likely to continue. As such, some are calling for stricter scrutiny of autobiologicals especially as they can be considered generics and therefore cut biosimilars out of certain markets where pharmacy substitution for biosimilars is not allowed. Learn More

As the EMA announced that it's finally completing its move from London to Amsterdam, its Committee for Medicinal Products for Human Use (CHMP) has recommended five new medicines for approval, revealed the outcomes of its safety reviews of Pfizer's Xeljanz and Sanofi's Lemtrada and discussed the withdrawal of three applications. Following the initial move to a temporary building in Amsterdam last March, EMA has been waiting on a new building with new technical equipment that was under construction. Staff will likely move into their new offices and workspaces in January 2020, the agency said. Learn More

The FDA's Endocrinologic and Metabolic Drugs Advisory Committee this week voted 16-0 to recommend that the agency expand the label of Amarin's Vascepa, a derivative of fish oil, to add an indication on reducing the risk of cardiovascular events. The meeting was closely watched because the expanded label could push Vascepa, which six years ago received a negative opinion from this same committee on an expanded label, to blockbuster status. In 2018, Vascepa brought in about $229 million in net revenue. Learn More

The US FDA needs to reconsider its use of surrogate outcomes in some guidance documents on developing treatments for infectious diseases, researchers from Harvard and George Washington University School of Medicine wrote in a review published this week in JAMA Internal Medicine. The review evaluated 22 FDA guidance documents, which included recommendations for pivotal clinical trials in 27 disease indications. For six indications (22%), only direct clinical outcomes were specified as primary endpoints, while for the other 21 indications, guidance documents recommended surrogate outcomes as sole primary endpoints or as part of composite primary endpoints. Learn More

The US FDA this week released a warning letter sent last week to Mylan's active pharmaceutical ingredient (API) manufacturing site in Andhra Pradesh, India, highlighting "contamination at levels above the limit" for some API batches. The FDA's inspection of the site in May and June uncovered, "Multiple contract manufacturers supplied solvents that were contaminated with , but your firm lacked documentation of which tanks were used to store these solvents," the letter says. "Although you acknowledged that there was no record of usage for each of the recovered solvent tanks, your response did not provide sufficient information on attempts to retrospectively reconcile the number, identification, and usage of the tanks." Learn More

Regenexbio has filed a lawsuit against the US FDA for failing to provide an explanation for placing a hold on a clinical trial for an experimental gene therapy to treat wet age-related macular degeneration. The FDA's decision effectively halted the development of the potential one-time subretinal treatment without a clear basis, the complaint said. "By failing to provide advance notice of, or any reasoned basis for, the clinical hold, FDA violated the FDCA and its own regulations. FDA's clinical hold on RGX-314 is therefore contrary to law and arbitrary and capricious under the Administrative Procedure Act," the company said. Learn More

The US FDA last week heard from more than two dozen stakeholders on opportunities for the agency to promote more effective drug development programs at a public workshop at its headquarters in Silver Spring, Maryland. During the meeting, speakers representing drugmakers, patient groups, research institutions and consulting firms suggested policies and approaches the FDA could pursue to promote more effective drug development. Jennifer Hamilton, head of precision medicine at Regeneron Pharmaceuticals, suggested that FDA and industry could make drug development "smarter" by collaborating on regulatory standards and guidance for using genomic data to support submissions and labeling changes. Learn More

Following an inspection that uncovered GMP deficiencies, the Danish Medicines Agency said this week that it has revoked Danish pharmaceutical company Scanpharm A/S's authorization to manufacture medicinal products and intermediate products. A three-day inspection in September showed the company lacked the necessary knowledge regarding its responsibilities related to qualified persons, as well as other issues with reporting of out-of-specification (OOS) results, OOS and missing data for stability studies and inconsistencies between registration files and specifications. The Danish Medicines Agency also found the company used non-validated analytical methods and did not perform its self-inspection sufficiently. Learn More

As quality issues have led to drug shortages, Donald Ashley, director of the FDA's Office of Compliance, raised several major concerns this week with the active pharmaceutical ingredient (API) industry, noting three trends related to the obfuscation of supply chain information, an increasing number of data integrity question marks and impurity concerns that have led to recalls. On the obfuscation front, Ashley noted that API companies sometimes fail to obtain and retain documents with the identity of the original manufacturer and certificate of analysis. While on data integrity, Ashley noted that of the warning letters issued to API manufacturers over the past four years, 73% have included data integrity charges. Learn More

Aurobindo Pharma is under the scrutiny of the US FDA once again. According to published reports, 3-4 inspectors from the U.S. FDA recently completed their inspection of the company's Unit IV in Telangana, India and this week are at the biopharma's Units V and VIII sites in Hyderabad. Spread over 33 acres, Aurobindo's Unit-IV is a dedicated mid-size manufacturing facility for generic sterile injectables, opthalmics and low volume parenterals. By contrast the facilities under the FDA lens this week, Units V and VIII, are smaller facilities that manufacture antibiotics, ARVs, CVSs and CNSs. Learn More

While noting that full regulatory harmonization may be out of reach, Suzette Kox, secretary general of the IGBA this week called for further convergence among stringent regulators in the biosimilar space, particularly on the acceptance of foreign-sourced reference products. Kox, speaking at the Association for Accessible Medicines' GRx+Biosim conference in Bethesda, MD, explained how a global framework for "truly global biosimilar development" is necessary to avoid repetitive, and therefore unethical, clinical studies. She also called for more regulators to share unredacted biosimilar assessment reports, although not publicly, especially as FDA often shares such reports with the WHO. Learn More

President Donald Trump has nominated Stephen Hahn, chief medical executive of the MD Anderson Cancer Center in Houston, to be the next US FDA commissioner. If confirmed by the Senate, a process that could take several months, Hahn will take the reins of FDA from Acting Commissioner Ned Sharpless, who moved over to FDA following a stint as head of the National Cancer Institute (NCI). Sharpless will return to direct NCI, while Brett Giroir, assistant secretary for health at the US Department of Health and Human Services (HHS), will serve as acting FDA commissioner while Hahn's confirmation process occurs. Learn More

Novartis this week said that the US FDA had placed a partial hold on intrathecal clinical trials of its gene therapy Zolgensma based on findings in a small preclinical animal study. This comes after a draft report of the preclinical safety findings was presented to the AveXis safety management team last March and the company "determined at that time that the safety finding should be included in the annual update of the investigator brochure planned for September 2019. Unfortunately, a mistake was made, and this update was not implemented." The omission was later identified as part of a Novartis review related to the Form 483 response from August. Learn More

As drug and API manufacturing moves overseas to cut costs, quality and reliability concerns have been raised and the House Energy & Commerce Committee's Subcommittee on Health met this week to discuss the supply chain with one of the US FDA's top officials and other experts. Janet Woodcock, director of the FDA's CDER, explained how part of the problem is that FDA does not know the volume of APIs coming from China or other countries to the US, and only knows the number of manufacturing facilities approved. According to Woodcock's written testimony, China has 230 (13%) of the API manufacturing facilities serving the US, while the US has 510 (28%) and the rest of the world has 1048 (59%). Learn More

Mylan is voluntarily recalling one lot of the anti-anxiety prescription drug Alprazolam, a generic form of Xanax, nationwide because of "the potential presence of a foreign substance," according to a recall notice posted on the website of the U.S. FDA. "Clinical impact from the foreign material, if present, is expected to be rare, but the remote risk of infection to a patient cannot be ruled out," the notice states. "To date, Mylan has not received any adverse events related to this batch." The recall is for Alprazolam 0.5 mg tablets that were distributed in July and August 2019 in 500-count bottles. The affected drug's lot No. is 8082708 and it has a September 2020 expiration date. Learn More

The US FDA Task Force on Drug Shortages supports the idea of creating a new rating system to help drug purchasers, including consumers, better understand the quality management of drug manufacturing facilities. "This idea envisions that pharmaceutical companies could, at their discretion, disclose the rating of the facilities where their drugs are manufactured," according to Janet Woodcock, director of FDA's CDER. The idea of a drug manufacturing ratings program follows the revelation that many drug shortages occur in the US because of quality issues. A team of FDA economists examined a sample of 163 drugs that first went into shortage between 2013 and 2017 and found that 62% were associated with manufacturing or product quality problems. Learn More

As another delay may push back Brexit to the end of January, the UK's Medicines and Healthcare products Regulatory Agency (MHRA) this week published new guidance on comparator products (CPs) used in bioequivalence (BE), pharmacokinetic (PK) and therapeutic equivalence (TE) studies to support generic drug and other abridged marketing authorization applications following a no-deal Brexit. The guidance explains how it may be possible for an applicant to compare a proposed medicinal product with a non-UK-sourced CP, although the application would still be required to refer to an eligible UK reference medicinal product (RMP).  Learn More

The US FDA this week finalized a question and answer guidance on the type of manufacturing facility information that should be included in applications submitted to the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research. The FDA explains that the guidance is meant to help companies understand how not to submit extraneous information or misplaced or missing information that could result in delays, Refusal to File or Refuse to Receive actions. Learn More

The revised draft guidance from the US FDA on drug master files (DMFs) deals with submissions on confidential information about facilities, manufacturing, processing, packaging and storing drugs. The FDA says that the update includes new procedures for DMFs referenced in abbreviated new drug applications (ANDAs), more detailed instructions on submitting original DMFs versus amendments, reference to the electronic submission requirements under section 745A of the Federal Food, Drug, and Cosmetic Act (FD&C Act) that apply to certain DMFs, clarification and reorganization of material associated with Type III and Type IV DMFs and a change in FDA's contact person for the guidance. Learn More

AstraZeneca announced this week that the U.S. drug regulator will give a speedy review to its experimental breast cancer treatment, which could put the British drugmaker in direct competition with bigger rival Roche. The U.S. FDA accepted AstraZeneca's application and granted the treatment, trastuzumab deruxtecan, a priority review for the treatment of HER2-positive metastatic breast cancer, the drugmaker said. This comes after the biopharma giant agreed to pay up to $6.9 billion to work with Japan's Daiichi Sankyo on the treatment, in a direct challenge to the world's biggest cancer drug maker Roche. Learn More

The US FDA has sent warning letters to Indian drugmakers Glenmark Pharmaceuticals and Torrent Pharmaceuticals and Bahamian drugmaker Coral Pharmaceuticals related to violations of good manufacturing practices at their manufacturing facilities. In the case of Glenmark, the FDA warning comes after inspection of the Himachal Pradesh facility. While the warning letter to Torrent comes after an inspection of the company's Gujarat facility. In its warning letter to Coral, the FDA inspection which resulted in the warning letter occurred at its Freeport City facility on Grand Bahama island. Learn More

Following inspections in late September, India-based generic drugmakers Lupin, Aurobindo and Cipla received Form 483s from the US FDA for observations related to cleanliness, investigations into out-of-specification results and other deficiencies. The Lupin site in Tarapur received three observations while the Aurobindo site in Polepally had seven observations and the Cipla site Goa has twelve observations.  While the findings noted in the Form 483's were partially redacted the viewable data illustrates concerns about contamination, procedures for handling deviations and approach to documentation management. Learn More

The Pharmaceutical Inspection Co-operation Scheme (PIC/S) is seeking comments on revisions to two parts of its GMP Guide (Annex 2A and Annex 2B) that deal with the manufacture of advanced therapy medicinal products (ATMPs) and biological medicinal substances and products for human use. PIC/S explains that draft Annex 2A accounts for international developments in the regulation of ATMPs, with particular attention to the European Commission guideline on GMP for ATMPs, while addressing concerns related to patient safety and the proportionate regulation of ATMPs. The document is divided into two parts related to the manufacture of ATMPs. Learn More

As the wrangling over the US-Mexico-Canada trade agreement continues, a provision to require Mexico and Canada to accept 10 years of biologic exclusivity stirred up controversy because it would be two more years than what Canada currently enjoys and add five more years for Mexico. In the US, biologic exclusivity is set at 12 years. But in reality, a 30-year run for a biologic without competition is possible, following a court win for Amgen last August. And 15-plus years without competition has become the norm rather than the exception. Meanwhile, Democrats have introduced legislation to reduce the 12 years of exclusivity. Learn More

A study published this week in JAMA Network Open finds that currently available real-world data sources can only be used to feasibly replicate 15% of clinical trials. The aim of the study was to determine whether RWD could be used to power observational studies that answer the same clinical questions as traditional clinical trials. Randomized controlled trials (RCTs) are considered the gold standard for clinical evidence to support the safety and efficacy medical products due to high levels of internal consistency and reduced bias. However, as the authors of the study write, "Compared with RCTs, RWE better reflects the actual clinical environments in which medical interventions are used... Learn More

Commenters have recently argued that the US FDA's drug approval standards are becoming inappropriately low and that the required post approval evaluations are either inadequate or left undone. But three senior FDA officials offered several counterpoints this week at the fifth annual Biopharma Congress in Washington, DC. Janet Woodcock, director of the FDA's CDER explained that the agency is working on its own analyses to provide "a more robust response" to these critiques. She also explained how the high number of approvals in recent years for rare diseases may be influencing this perception of a lower bar, especially as more treatments are approved. Learn More

Glenmark Pharma has received a warning letter from the U.S. FDA related to its Baddi facility in India. The warning letter follows an inspection of the facility by the FDA April 15 and April 20, after which the regulator issued a Form 483 with eight observations. The company said that it is in the process of preparing a detailed response to the FDA within 15 working days. For its part Glenmark notes in a statement that they do not expect the warning letter to adversely impact the existing manufacturing of product s at the facility and note that there are no major approvals pending at this facility for the next 12 months. Learn More

The US FDA recently released tables that can be included in submissions for new drug and biologic applications to provide information regarding the bioanalytical methods for pharmacokinetic assessments. "The templates in this guidance are applicable to bioanalytical procedures such as chromatographic assays (CCs) and ligand-binding assays (LBAs) that quantitatively determine the levels of drugs and their metabolites and therapeutic proteins in biological matrices such as blood, serum, plasma, urine, and tissue such as skin," the FDA explains. Learn More

A ban on exporting up to 30 critical drugs will be imposed by the UK in an attempt to prevent shortages before a no-deal Brexit. The government will announce the ban this week after being warned by industry that speculators are poised to raid stockpiles of medicines to profit from a collapse in the pound. They include common treatments for conditions such as epilepsy as well as hormone replacement therapy. The ban, which is expected to take effect immediately, will cover all drugs the UK believes could be affected by supply shortages in the coming weeks. Learn More

President Trump is set to nominate Dr. Stephen Hahn to lead the FDA, pending completion of the vetting process, according to two people familiar with the selection process. Hahn, an oncologist, is the chief medical executive at MD Anderson Cancer Center in Houston. Trump interviewed him for the job a month ago. A third person familiar with the White House's thinking confirmed that Hahn remained the frontrunner and that the administration was completing paperwork and final background checks. By law, the Trump administration must formally nominate a long-term FDA commissioner by November 1st. Learn More

The US FDA this week released a warning letter sent in August to China-based drug testing facility Shanghai Institute of Pharmaceutical Industry for refusing an inspection. The FDA had planned a surveillance and pre-approval inspection of the facility from November 29th to December 4th, 2018, but the company told the agency's China office in a written response that it was refusing the inspection. The firm is listed as a contract testing laboratory that provides API characterization and/or identification testing to support multiple ANDAs, according to the FDA. Learn More

The US FDA has announced the fees for using a priority review voucher (PRV) to speed the review of a new drug, biologic or medical countermeasure for vouchers redeemed in FY2020. The agency's three PRV programs are meant to incentivize drug development in underserved areas, such as tropical diseases, rare pediatric diseases and medical countermeasures used in public emergencies. By using a PRV, a sponsor can receive a six-month priority review for a new drug application (NDA) or biologics license application (BLA) that otherwise would not qualify for one. So far, the FDA has issued more than 30 PRVs to drugmakers. Learn More

The US FDA said this week that the planned reorganization of its Office of New Drugs (OND) will move forward thanks to Congressional approval, including changes to its Office of Translational Sciences (OTS) and the Office of Pharmaceutical Quality (OPQ). The changes will create offices for interrelated disease areas and divisions with clearer and more focused expertise, the FDA said, noting the number of OND offices that oversee its review divisions will increase from six to eight, and there will be increases in the number of OND clinical divisions from 19 divisions to 27, plus six non-clinical review divisions. Learn More

The US FDA this week released a preliminary list of the new drug applications (NDAs) that will be converted to biologics license applications (BLAs) on March 23rd, 2020. The shift is caused by the Biologics Price Competition and Innovation Act of 2009, which clarified the statutory authority under which certain protein products will be regulated by amending the definition of a "biological product" to include a "protein (except any chemically synthesized polypeptide)." The change effectively means that any follow-on products for these NDAs will need to win approval as biosimilars. Learn More

The U.S. FDA is again expanding its recall of widely prescribed blood pressure drugs due to contamination with a cancer-linked chemical. The announcement targets an additional five lots of the generic drug losartan made by Torrent Pharmaceuticals. The lots include losartan potassium tablets and losartan potassium hydrochlorothiazide tablets. The recalled lots contain trace amounts of N-methylnitrosobutyric acid, or NMBA, which has been linked to cancer. This is the fifth time in 2019 that Torrent Pharmaceuticals has expanded its voluntary recall of losartan. The recall was first announced in January. Learn More

Researchers at the US FDA have developed a new computational tool to predict whether the body will mount an immune response that blocks the activity of biological drugs. Specifically, the tool, dubbed TCPro, simulates how the body's CD4+ T cells will respond to biotherapeutics, and predicts whether the body will produce anti-drug antibodies. The FDA says its researchers validated the test by comparing its predictions for 15 protein therapies against reported clinical data for those same products. Learn More

As part of efforts to prepare for the continued rise in data-rich applications and to help fill the gap separating scientific advances and new therapies, the US FDA has unveiled its Technology Modernization Action Plan (TMAP). Near-term modernization in computer hardware and software technologies are the focus of FDA's TMAP, which offers a broad overview of how the agency needs to adapt. For instance, the TMAP report notes that FDA will need to have a technical infrastructure that can accept, evaluate and analyze novel sources of data (e.g., real-world data) and apply that data to regulatory decision making over the next several years. Learn More

The US FDA this week released 53 product-specific guidance documents to aid generic drug development, including 34 new guidance documents, 26 guidances for treatments that lack generic competition and 16 for complex products. When finalized, the guidance documents will represent the current thinking of FDA on, among other things, the product-specific design of bioequivalence studies to support abbreviated new drug applications. This is the third time in 2019 that FDA has released new and revised guidance documents, with the previous releases occurring in May and February. Learn More

As part of its work to improve and modernize the quality assessment of drug applications, the US FDA is developing a new, more standardized system, to be known as the Knowledge-aided Assessment & Structured Application (KASA), according to an article authored by officials from FDA's Center for Drug Evaluation and Research and published in the latest issue of the International Journal of Pharmaceutics. The authors describe KASA as a new system that captures and manages information in a structured format about the inherent risk and control approaches for product design, manufacturing and facilities. Learn More

Following its investigation into an impurity called N-nitrosodimethylamine (NDMA) in blood pressure medicines, the EMA, at the request of the European Commission, is beginning a review of ranitidine medicines after tests showed that some contained the impurity. NDMA is classified as a substance that could cause cancer on the basis of animal studies, although the EMA explains how it is present in some foods and in water supplies, but it is not expected to cause harm when ingested in very low levels. The FDA says it's also examining levels of NDMA in ranitidine and evaluating any possible risk to patients, saying it will provide more information as it becomes available. Learn More

As concerns persist over the safety of medicines made overseas, the U.S. FDA recently took the opportunity to tout data showing the number of facilities inspected in foreign countries had increased 20% over the most recent five-year period. Specifically, the agency inspected 993 foreign plants in fiscal year 2014 compared with 1,245 in fiscal year 2018. At the same time, though, the data indicates that the U.S. regulatory agency inspected fewer domestic facilities during that same period - 1,869 versus 1,662, which amounts to a 12.5% drop. Learn More

"It's not working, and it won't work in the future," said Dr. Janet Woodcock, the U.S. FDA'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 biopharma 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. Learn More

The White House's Office of Science Technology and Policy (OSTP) this week sought input from any and all biotech stakeholders on the innovation, products, technology and data derived from biologically-related processes and science that drive economic growth, promote health and increase public benefit. Referred to as the "Bioeconomy," OSTP said it's seeking comments before October 23rd from the public, including those with capital investments, those performing research or developing enabling platforms and applications in the field of biological sciences, including healthcare, medicine, pharmaceuticals, biotechnology and manufacturing. Submitted comments should be limited to 10 pages or less. Learn More

As the US FDA grapples with how to use real-world data for regulatory decisions, two FDA officials wrote in a perspective published last Friday in the Clinical Journal of the American Society of Nephrology that the agency is developing guidance on data quality issues unique to the real-world data setting and related study design considerations. New data standards will need to be developed and FDA is "currently exploring," they write, when the use of observational data "may be appropriate for evaluating effectiveness from a broad policy perspective and also, as individual drug manufacturers submit proposals for using real world data to support marketing applications." Learn More

The US FDA has announced its support for the latest set of Unified Code for Units of Measure (UCUM) codes for use in electronic submissions for drugs and biologics. With the notice, the FDA says it wants drugmakers to use UCUM coding for drug establishment registration, drug listing and in the content of product labeling in regulatory submissions to the Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research. While the FDA says it already supports the UCUM standard for structures product labeling (SPL) submissions, the agency says it will be updating the FDA Data Standards Catalog to reflect the standard's immediate implementation. Learn More

The US FDA has warned Chinese API distributor Yino and Costa Rican OTC drugmaker Polimeros y Servicios over GMP violations at their respective facilities. In its warning letter to Yino, the FDA took the company to task after a five-day inspection of the company's Chongqing facility in March identified issues with its handling of re-labeled API distributed to the US. While the FDA's warning letter to Polimeros y Servicios comes after a five-day inspection of the firm's San Jose, Costa Rica facility cited four issues related to the company's product testing and equipment cleaning and maintenance procedures for its OTC products, which the FDA says include repeat violations from a 2017 inspection. Learn More

Biocon has disclosed that a second Complete Response Letter (CRL) for its follow-on insulin glargine product referencing Lantus. This new regulatory challenge comes just weeks after Biocon announced that it had gained a certificate of good manufacturing practice from the European Medicines Agency's Irish inspection authority for the Malaysian manufacturing site. According to company officials, "The CRL has been issued pending completion of the Corrective And Preventive Actions...submitted to the US FDA in response to the observations made at the conclusion of the pre-approval inspection of our insulin manufacturing facility in Malaysia in June 2019."  Learn More

Time may be running out on Ned Sharpless' term as acting FDA commissioner. President Donald Trump must make a decision about the agency's top job by Nov. 1. If the White House decides to seek Senate confirmation for Sharpless, he would have to step down as acting commissioner during the confirmation process. He could be appointed to a placeholder position at FDA for this period. Alternatively, the administration could hand off the acting commissioner title to someone else, sending Sharpless back to his previous position as director of the National Cancer Institute. At least one candidate is vying for the position. Learn More

The US FDA has issued a warning letter to Lantech Pharmaceuticals for violation of cGMP, including failure to investigate the presence of a carcinogen in a solvent, at its manufacturing facility in Andhra Pradesh. This comes after inspectors from the regulatory agency visited the site from March 6th-15th, 2019. In its letter the FDA has asked Lantech to initiate a comprehensive, independent assessment of its overall system for investigations of deviations, atypical events, complaints, out-of-specification results, and failures. The health regulator said it strongly recommends the company to engage a consultant to assist the firm in meeting CGMP requirements. Learn More

Although regulators rarely offer public comments on another regulator's guidance, the EMA has offered line-by-line comments and edits on a recently released draft guidance from the US FDA on comparative analytical assessments for biosimilars. While noting the close collaboration between the EMA and FDA on the statistical aspects of comparative analytical assessments, the EMA comments feature questions for clarification, recommendations to omit and areas of commonality. EMA pledges its support for the sections of the guidance related to the preparation of a "(prospective) comparative analytical assessment plan" and "accounting for lots/batches to be used for comparison." Learn More

More than 50 academics and researchers from Harvard, Yale, Johns Hopkins and other universities around the world are calling on the US FDA to not replace its original reviews of medical products with an "integrated review" because of the valuable information that would be lost. The researchers claimed that such a shift would deprive them of information and data on the clinical studies and trials submitted to FDA, information on the post marketing requirements, and reviewer concerns with an application that might not be included in a summary document like the integrated review, among other important details. Learn More

AstraZeneca has announced that the U.S. FDA has granted fast track status for the development of its diabetes drug Farxiga to prevent heart and kidney failure in patients with chronic kidney disease (CKD). Farxiga, one of AstraZeneca's top 10 drugs by sales, is part of the SGLT2-inhibitor class of antidiabetics that cause the kidneys to expel blood sugar from the body through urine. In July, U.S. regulators declined to approve Farxiga as a supplement to insulin in adults with type-1 diabetes where insulin alone was not able to control blood sugar levels. The treatment is already approved in the United States to treat type-2 diabetes, the more common form of the condition. Learn More

Following a five-day inspection last month, the US FDA has sent a Form 483 to API manufacturer Shilpa Medicare Limited after the agency uncovered long-lasting power failures at the site. "During the walkthrough on July 24th2019, three power failures occurred within the facility during a 15-minute interval and a back-up generator did not resume power," FDA investigator Yvins Dezan wrote. And while reviewing audit trails for two stability chambers, Dezan said it was noted that the facility "frequently loses power, sometimes for over 8 hours." Shilpa manufactures generic versions of cancer treatments for the US market. Learn More

An action plan will be rolled out in the coming months to define the US FDA approach to modernizing its technology infrastructure and framework, FDA Principal Deputy Commissioner Amy Abernethy said during a keynote at the Office of the National Coordinator for Health Information Technology's (ONC) 3rd interoperability forum. Abernethy said that FDA's plan contains an agency-wide objective-advancing interoperability to inform regulatory decision-making in real-time. This, in turn, is intended to expedite determinations on submissions and improve regulatory oversight to increase patient access to products deemed to be safe and effective at a faster pace. Learn More

In an unexpected move, Gilead is challenging patents held by the federal government for using the Truvada pill to prevent HIV, a drug that has sparked controversy due to its cost and the extent to which taxpayer dollars funded crucial research. The drug maker said it asked the U.S. Patent and Trademark Office to examine the patents, which AIDS activists have argued would entitle the government to collect royalties from Gilead and use the funds to combat the virus. However, a Gilead official said the company refused to agree to a license in talks with the Department of Health and Human Services over the past three years. Learn More

The question and answer (Q&A) document released by the European Medicines Agency (EMA) this week describes when an exemption from EU batch re-testing can be granted for imported advanced therapy medicinal products (ATMPs). The three-page Q&A begins by noting that a Qualified Person (QP) has to ensure each batch of the imported ATMP is re-tested upon importation. But the QP can certify that the imported batch relies on controls that are conducted in a third country if the product has been manufactured and tested in a country with a relevant mutual recognition agreement or equivalent arrangements with the EU. Learn More

Beginning September 20th, June Raine will be the new interim Chief Executive of the MHRA. Raine will replace Ian Hudson, who will step down after 18 years with MHRA, including six years as chief executive. In addition to Hudson, John Wilkinson, director of devices at MHRA, will step down at the end of October. In June, Wilkinson discussed several initiatives that MHRA is working on, including how to deal with Brexit. While working at MHRA's predecessor Medicines Division since 1985 and at MHRA since 2003, Raine has served as MHRA's director of the Vigilance and Risk Management of Medicines (VRMM) division since 2006. Learn More

The US FDA's Center for Drug Evaluation and Research this week announced it will conduct a small pilot project to test the processing and analysis of nonclinical study data provided electronically using an updated standard, known as the SEND 3.1, from the Clinical Data Interchange Standards Consortium (CDISC). CDER is seeking a maximum of five participants for the pilot, which will evaluate the compliance of sample SEND 3.1 datasets submitted to CDER. The FDA notes that studies initiated after December 17th 2016 must be submitted with data according to data standards listed in the FDA Data Standards Catalog for NDA's, BLA's and ANDA's.  Learn More

The EMA and US FDA concur more than 90% of the time in their decisions to approve new drugs, according to a new study from EMA and FDA officials that looked at 107 applications from 2014 to 2016. In just eight of the 107 applications, FDA initially declined to approve a new drug or biologic while EMA approved it, although in all eight of those cases, FDA ended up approving that drug or biologic. And in one case the FDA approved a treatment and the EMA initially did not, but later did. However, the EMA adopted negative opinions for two drugs in 2018 that were approved by FDA in 2017, and one sickle cell drug in 2019 that was also previously approved by FDA. Learn More

The MHRA has confirmed it will accept results from batch testing performed in the United States in the event of a no-deal Brexit. MHRA published the original list of countries authorized to perform batch testing weeks before the UK was due to leave the European Union in March. At that time, MHRA said no batch testing done in the US would be accepted after Brexit because such work was outside the scope of the mutual recognition agreement between America and EU until the assessment of member state equivalence was finished. Under the new guidance, MHRA will allow most medicines that undergo batch testing in the US into the UK if there is a no-deal Brexit. Learn More

While noting that tens of thousands of drug listing records have not been updated in the past year, the US FDA this week announced that it will begin inactivating drug and manufacturing establishment listings that are not current in one month. The announcement is part of FDA's push to encourage drugmakers to ensure that their listed human drugs and any information in their drug listings, including drug establishments, are accurate. FDA Commissioner Ned Sharpless noted in a statement that although there have not been any public health consequences as a result of the outdated listings, "inaccurate information has the potential to compromise the integrity of the FDA's database..." Learn More

As the US FDA explores plans to lower the cost of pharmaceuticals with imports from Canada and elsewhere, Sen. Chuck Grassley is calling on the agency to perform more unannounced inspections of foreign facilities. Moving forward, should the administration's plans to allow certain imports be put into effect, the administration "must require more foreign inspections generally and unannounced inspections specifically, particularly compared to previous administrations," Grassley writes.  Learn More

Sarepta Therapeutics says it has been informed that an adverse event report was erroneously submitted to the U.S. health regulator regarding an ongoing study of the company's gene therapy for Duchenne muscular dystrophy (DMD). Sarepta said its investigation indicated the report was not submitted by its employee or the study's principal investigator. The drug safety monitoring board has recommended the study to continue after a review, the company said in a statement. For its part, the US FDA says that it is looking into the matter but does not have any new information at the time of this writing. Learn More

An FDA advisory panel this week voted in favor of Gilead Sciences combination drug to reduce the risk of sexually acquired HIV infection in men and transgender women who have sex with men. The treatment, Descovy, is a combination of emtricitabine and tenofovir alafenamide, and is already approved to treat chronic HIV. The recommendation is a shot in the arm for Gilead as its third best-selling HIV drug, Truvada, faces exclusivity loss in the United States, beginning 2020. Learn More

In a statement issued this week, Peter Marks, director of the US FDA's CBER, said the agency is investigating data manipulation submitted in the BLA for Novartis' gene therapy Zolgensma, a drug developed by AveXis. Alert readers will recall that AveXis was acquired by Novartis in an $8.7 billion deal in April 2018 and was approved last May. The gene therapy is considered to be the world's most expensive drug at $2.1 million. According to Marks, the FDA was informed by AveXis on June 28th that some of the nonclinical data included in the BLA for Zolgensma was manipulated. Marks also said that AveXis was aware of the data manipulation and warned that the company could be subject to civil or criminal penalties. Learn More

The EMA this week clarified the documentation and declaration requirements for peptones used in the manufacture of active substances in an update to its quality of medicines questions and answers (Q&A) guide. The Q&A covers issues brought to EMA's Joint Committee for Medicinal Products for Human Use/Committee for Medicinal Products for Veterinary Use Quality Working Party and express the harmonized position of European Economic Area competent authorities. Specifically, EMA answered two questions pertaining to peptone use during manufacturing, explaining how manufactures should document its use or notify regulators of any change related to the peptone being used. Learn More

Sunovion has announced that the US FDA has accepted its New Drug Application for dasotraline, a novel dopamine and norepinephrine reuptake inhibitor (DNRI), for the treatment of patients with moderate-to-severe binge eating disorder (BED). The application is based on results from the SEP360-321 study, announced in the latter part of 2018, which showed a statistically-significant decrease in number of binge days per week, at the higher dose. The US Food and Drug Administration earlier rejected an application from the firm to market the drug for the treatment of ADHD. The action date by the FDA under the Prescription Drug User Fee Act is May 14, 2020. Learn More

Last November, officials from the EMA and US FDA met with industry representatives in London to discuss the various quality challenges that arise when the development of investigational products is accelerated. EMA's launch of its priority medicines (PRIME) scheme and FDA's breakthrough therapy designation have enabled companies to speed certain treatments to market, although there have been difficulties in identifying possible scientific and regulatory approaches to facilitate the type of robust quality data packages necessary to ensure that patient safety and product quality and efficacy are not compromised, according to a report released Wednesday on the workshop. Learn More