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Content Currents


DIA's Content Currents provides you with new and important global regulatory developments and their impact on pharmaceutical, biotechnology, and medical product development.



Guidance Documents CBER is Planning to Publish in 2015
This is the list of guidance topics CBER is considering for development during Calendar Year 2015.  The list includes topics that currently have no guidance associated with them, topics where updated guidance may be helpful, and topics for which CBER has already issued Level 1 drafts that may be finalized following review of public comments.

CDER 2015 Plan for New & Revised Guidances
The link above is the new CDER list of guidances planned for new issue or revision in Calendar Year 2015.

Searchable Database of All Official FDA Guidance Documents and Other Regulatory Guidance
FDA has created a database that provides a convenient way y to search for all FDA guidance documents from a single location. You can search for documents using key words, and you can narrow or filter your results by product, date issued, FDA organizational unit, type of document, subject, draft or final status, and comment period. Access the database at the link above. (FDA.gov)

CDRH FY 2015 Proposed Guidance & Focused Retrospective Finalized Guidance
The lists below include guidance documents that CDRH intends to publish this fiscal year (FY2015) as well as previously-issued final guidances for which CDRH would appreciate external feedback on whether these final guidances should be revised or withdrawn. We have provided three lists: (1) a list of guidance documents that the Agency fully intends to publish (the “A-list”); (2) a list of guidance documents that the Agency intends to publish as resources permit (the “B-list”); and (3) a list of final guidance documents that issued in 2005, 1995, and 1985 subject to focused retrospective review. Although resource constraints and new issues that emerge over the course of the year may preclude CDRH from issuing every guidance document on the A-list and B-list and may require that CDRH issue guidance documents not on the lists, the A-list and B-list are intended to provide helpful information about CDRH’s current priorities for the upcoming fiscal year. CDRH plans to update all three lists every year.  (FDA.gov)

On April 13, 2015, FDA announced the availability of a guidance for industry and staff entitled “Balancing Premarket and Postmarket Data Collection for Devices Subject to Premarket Approval.” This guidance clarifies FDA’s current policy on balancing premarket and postmarket data collection during the Agency’s review of premarket approval applications (PMA). Specifically, this guidance outlines how FDA considers the role of postmarket information in determining the appropriate type and amount of data that should be collected in the premarket setting to support premarket approval, while still meeting the statutory standard of safety and effectiveness. FDA believes this guidance will improve patient access to safe and effective medical devices that are important to public health by improving the predictability, consistency, transparency, and efficiency of the premarket process.  [Federal Register]

On April 15, 2015, FDA issued a “Request for Nomination for Industry Representatives and Participation From Industry Organizations on Public Advisory Committees.” any industry organizations interested in participating in the selection of nonvoting industry representatives to serve on its public advisory committees for the Center for Drug Evaluation and Research (CDER) notify FDA in writing. FDA is also requesting nominations for nonvoting industry representatives to serve on CDER’s public advisory committees. A nominee may either be self-nominated or nominated by an organization to serve as a nonvoting industry representative. Nominations will be accepted for current vacancies effective with this notice. Request letters must be submitted by May 15, 2015. [Federal Register]

On April 20, 2015, FDA announced the availability of the “Pilot Program for Center for Devices and Radiological Health Electronic Submission for Home Use Device Labeling.” The CDRH electronic submissions Pilot Program database to house labeling for home use devices. Participation in the pilot is open to applicants who label their device(s) for home use. Participation in the pilot project is voluntary. Participants will be asked to navigate through the electronic submissions system and practice submitting labels and package inserts. The pilot project is intended to provide industry and CDRH staff the opportunity to evaluate the submissions process and system and to receive comments from industry participants. FDA will accept applications for participation in the voluntary electronic submissions CDRH Home Use Device Labeling Pilot Program from May 1, 2015, through May 31, 2015. The pilot project will occur July 1, 2015, through December 31, 2015. [Federal Register]

April 21, 2015, FDA issued a draft guidance for industry and staff entitled “Acceptance of Medical Device Clinical Data from Studies Conducted Outside the United States.” This draft guidance articulates FDA’s current policy of accepting scientifically valid clinical data obtained from foreign clinical studies in support of premarket submissions for devices. The guidance describes special considerations that apply when using such data, including applicability to populations within the United States and study design issues and provides recommendations to assist sponsors in ensuring their data are adequate under applicable FDA standards to support approval or clearance of the device in the United States. This guidance is not intended to announce new policy, but to describe FDA’s existing approach to this topic.  Comments should be submitted by July 20, 2015, to assure consideration. [Federal Register]

On April 21, 2015, FDA announced that it intends to add a non-voting industry representative to the “Allergenic Products Advisory Committee.” The Committee reviews and evaluates available data concerning the safety, effectiveness, and adequacy of labeling of marketed and investigational allergenic biological products or materials that are administered to humans for the diagnosis, prevention, or treatment of allergies and allergic disease, and makes appropriate recommendations to the Commissioner of Food and Drugs of its findings. The process for consideration for participation in the selection process and for nominations is described in the Federal Register notice.   [Federal Register]

On April 22, 2015, FDA announced a notice of public meeting and establishment of a docket to request comments on the interim assessment for the “Program for Enhanced Review Transparency and Communication for New Molecular Entity (NME) New Drug Applications (NDAs) and Original Biologics License Applications (BLAs) (the Program).” The Program is part of the FDA performance commitments under the fifth authorization of the Prescription Drug User Fee Act (PDUFA), which enables FDA to collect user fees for the review of human drug and biologics applications for fiscal years (FYs) 2013– 2017. The Program is described in detail in section II.B entitled ‘‘PDUFA Reauthorization Performance Goals and Procedures Fiscal Years 2013 through 2017.’’ The Program is being evaluated by an independent contractor with expertise in assessing the quality and efficiency of pharmaceutical and biopharmaceutical development and regulatory review programs. As part of FDA’s performance commitments, FDA is providing a period for public comment on the interim assessment of the Program.  The meeting will be held on May 20, 2015. [Federal Register]

On April 22, 2015, FDA announced the availability of a draft guidance for industry entitled “Providing Regulatory Submissions in Electronic and Non-Electronic Format—Promotional Labeling and Advertising Materials for Human Prescription Drugs.” This draft guidance explains how manufacturers, packers, and distributors (firms) that may either be the applicant or acting on behalf of the applicant, should make submissions pertaining to promotional materials for human prescription drugs and biologic products (‘‘drugs’’) to the Office of Prescription Drug Promotion (OPDP) in the Center for Drug  Evaluation and Research (CDER) and the Advertising and Promotional Labeling Branch (APLB) in the Center for Biologics Evaluation and Research (CBER). This draft guidance describes the various types of submissions of promotional materials and general considerations for submissions. In addition, this draft guidance discusses the specific aspects of submission of promotional materials using module 1 of the electronic Common Technical Document (eCTD) using version 3.3 or higher of the us-regional-backbone file. This guidance does not address the more general requirements for a valid electronic submission using eCTD or the specifications for module 1 of the eCTD.  Comments should be submitted by June 22, 2015, to assure consideration. [Federal Register]

On April 22, 2015, FDA announced the availability of a guidance for industry entitled “Clinical Trial Endpoints for the Approval of Non-Small Cell Lung Cancer Drugs and Biologics.” This guidance provides recommendations to applicants on endpoints for cancer clinical trials submitted to FDA to support effectiveness claims in new drug applications, biologics license applications, or supplemental applications for the treatment of non- small cell lung cancer. This guidance focuses on endpoints specifically for lung cancer trials to support drug approval or labeling claims. This guidance should speed the development and improve the quality of protocols submitted to FDA to support anticancer effectiveness claims. This guidance finalizes the draft guidance issued on June 17, 2011. [Federal Register]

On April 22, 2015, FDA issued a correction to the final rule entitled “Administrative Detention of Drugs Intended for Human or Animal Use,” which appeared in the Federal Register of May 29, 2014. The rule sets forth the procedures for detention of drugs believed to be adulterated or misbranded and amends the scope of FDA’s part 16 regulatory hearing procedures to include the administrative detention of drugs. The rule published with incorrect statements regarding the impact of the final rule on small entities. This document corrects those errors.  The corrections are effective April 22, 2015 and applicable beginning June 30, 2014. [Federal Register]

On April 24, 2015, FDA announced an extension of the comment period for the proposed rule entitled “Abbreviated New Drug Applications and 505(b)(2) Applications,” that appeared in the Federal Register of February 6, 2015. In the proposed rule, FDA requested comments on its proposal to implement portions of Title XI of the Medicare Prescription Drug, Improvement, and Modernization Act of 2003 (MMA), which amended provisions of the Federal Food, Drug, and Cosmetic Act (FD&C Act) that govern the approval of 505(b)(2) applications and abbreviated new drug applications (ANDAs). FDA also requested comment on its proposal to amend certain regulations regarding 505(b)(2) applications and ANDAs to facilitate compliance with and efficient enforcement of the FD&C Act. The Agency is taking this action in response to requests for an extension to allow interested persons additional time to submit comments. Comments should be submitted by June 8, 2015, to assure consideration. [Federal Register]

On April 27, 2015, FDA announced the availability of a draft guidance for industry and staff entitled “‘M8 Electronic Common Technical Document (eCTD) v4.0 Draft Implementation Guide v2.0’’ (the M8 eCTD draft implementation guidance) and a related document entitled ‘‘eCTD v4.0 Implementation Package Draft Specification for Submission Formats v2.0’’ (the draft specifications document). The M8 eCTD draft implementation guidance and the draft specifications document were prepared under the auspices of the International Conference on Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use. The M8 eCTD draft implementation guidance provides instructions for creating the eCTD v4.0 Health Level 7 Regulated Product Submission (RPS) message for Modules 2 through 5 of the eCTD. The draft specifications document provides specifications for creating files for inclusion in the eCTD. These draft documents represent major updates to the eCTD specifications.  Comments should be submitted by May 27, 2015, to assure consideration. [Federal Register]


FDA Announces Availability of Grant Funding for IMEDS Project
On April 15, FDA announced the availability of grant funds for the support of Center for Drug Evaluation and Research (CDER) Innovations in Medical Evidence Development and Surveillance (IMEDS)-Methods project. IMEDS-Methods is a program within the Reagan-Udall Foundation that supports FDA’s scientific mission of serving public health needs by initiating and facilitating research into the methods of safety evaluation in large databases. IMEDS-Methods aims to improve the tools for conducting post-marketing safety surveillance using automated healthcare data and to foster their adoption.

IMEDS plans to conduct methods

research in five core areas: (1) Addressing bias in estimates from observational studies; (2) better understanding uses and limitations of the data; (3) applying lessons learned from earlier IMEDS projects to FDA surveillance activities; (4) expanding the surveillance question to continuous risk/benefit assessment; and (5) continuing to support qualified investigators in industry, government, and academic settings by providing access to de-identified electronic healthcare data and computing resources through the IMEDS Research Laboratory.

Eligibility is restricted to the Reagan-Udall Foundation.  The application is due June 15, 2015, and the program will run from July 15, 2015 – June 16, 2016.  (Federal Register)

FDA and CMS Form Task Force on LDT Quality Requirements
Jeff Shuren, Director of FDA’s CDRH, and Patrick Conway, Acting Principal Deputy Administrator CMS Chief Medical Officer, devoted the April 16 FDA Voice blog to a discussion of FDA and CMS efforts to coordinate quality oversight of laboratory developed tests (LDTs). They write:

“Health care providers and their patients expect that laboratory tests used in clinical management of patients should be consistent and of high quality.

“Under FDA’s proposed framework for the oversight of laboratory developed tests (LDTs), outlined in draft guidance documents issued in October 2014, FDA would oversee the quality of these laboratory tests, alongside the Centers for Medicare and Medicaid Services (CMS), which regulate the laboratories themselves through the Clinical Laboratory Improvement Amendments (CLIA). We have heard stakeholder confusion about the roles of the two agencies in ensuring quality and concerns about potentially duplicative efforts. To coordinate efforts across the Department, FDA and CMS are establishing an interagency task force that will continue and expand on our collaboration related to the oversight of LDTs, which are tests intended for clinical use and designed, manufactured, and used within a single lab. The task force, comprised of leaders and subject matter experts from each agency, will work to address a range of issues, including those involving quality requirements for LDTs.”

“The goals of the FDA/CMS Task Force on LDT Quality Requirements include:

  • identifying areas of similarity between the FDA quality system regulation and requirements under CLIA;
  • working together to clarify responsibilities for laboratories that fall under the purview of both agencies; and
  • leveraging joint resources to avoid duplication and maximize efficiency.
The task force is currently exploring areas where collaboration may realize greater oversight efficiency and produce the greatest benefit to patients, providers, and laboratories.” More at link above. (FDA.gov)

FDA Acting Commissioner Ostroff Plans to Hold Course

Acting FDA Commissioner Stephen Ostroff told BioCentury he plans to keep FDA on the course set by his predecessor, Margaret Hamburg. Ostroff, who is starting his third week as acting commissioner, presented a detailed agenda for the agency to reporters Monday, focusing on food safety and regulation of tobacco products. He said it is "hard to imagine substantial new priorities, at least in the short-term" beyond those Hamburg set over the last six years.

Ostroff reiterated concerns Hamburg had expressed publicly that a discussion draft of the 21st Century Cures Act could divert FDA resources from medical product reviews and approvals, and that provisions in the draft could lower safety and efficacy standards. He reported that FDA staff have been advising the House Energy & Commerce Committee about provisions that would be better deferred for consideration in PDUFA reauthorization negotiations rather than legislated in the 21st Century Cures Act.

Ostroff joined FDA in 2013 as CMO in the Center for Food Safety and Applied Nutrition and senior public health advisor to FDA's Office of Foods and Veterinary Medicine. He was appointed FDA Chief Scientist in January 2014. Prior to joining FDA, Ostroff was deputy director of the CDC's National Center for Infectious Diseases.  (BioCentury)


Patient-Focused Drug Development: Disease Area Meetings Planned for FY2013-2015

Public Workshop. 2015 Office of Regulatory Science and Innovation Science Symposium. April 27, 2015.  White Oak Campus, Silver Spring, MD.  The purpose of the public workshop is to increase scientific collaborations with government institutions, academia, industry and other stakeholders, working to improve science, training, and networking in accordance with the FDA mission of the advancement of regulatory science. This venue will also enhance knowledge and awareness of the FDA ORSI resources and provide guidance of its available services. [Federal Register]

Public Meeting. Chagas Disease Patient-Focused Drug Development. April 28, 2015.  White Oak Campus, Silver Spring, MD.  FDA is interested in obtaining patient input on the impact of Chagas disease on daily life and patients’ views on currently available therapies to treat the condition. [FDA.gov]

Public Advisory Committee Meeting. Ear, Nose, and Throat Devices Panel of the Medical Devices Advisory Committee. April 30 – May 1, 2015.  Gaithersburg, MD.  On April 30, 2015, the committee will discuss and make recommendations regarding the classification of Hearing Protectors, Circumaural Hearing Protectors, Middle Ear Inflation Devices, Tactile Hearing Aid Devices, and Vestibular Analysis Apparatuses.  On May 1, 2015 the committee will discuss key issues related to a potential pre- to post-market shift in clinical data requirements for modifications to cochlear implants in pediatric patients. [Federal Register]

New: Public Workshop. Methodological Considerations to Address Unmeasured Information About Important Health Factors in Pharmacoepidemiology Studies that Rely on Electronic Healthcare Databases to Evaluate the Safety of Regulated Pharmaceutical Products in the Postapproval Setting.  May 4, 2015.  White Oak Campus, Silver Spring, MD.  The purpose of the public workshop is to engage in constructive dialogue among regulators, academicians, pharmaceutical industry, clinicians, other stakeholders and the general public on potential strategies to improve availability of information on important health factors in pharmacoepidemiology studies that rely on electronic healthcare databases to evaluate the safety of pharmaceutical products in the postapproval setting. Co-sponsored by FDA and the University of Maryland Center for Excellence in Regulatory Science and Innovation.  Registration will be charged for some participants (see notice). [Federal Register]

Public Meeting. Patient-Focused Drug Development for Functional Gastrointestinal Disorders. May 11, 2015.  White Oak Campus, Silver Spring, MD.  FDA is holding this public meeting and an opportunity for public comment on Patient-Focused Drug Development for functional gastrointestinal (GI) disorders, including irritable bowel syndrome, gastroparesis, chronic persistent symptomatic gastroesophageal reflux despite standard therapeutic interventions, and chronic idiopathic constipation. [Federal Register]

Public Advisory Committee Meeting. Vaccines and Related Biological Products Advisory Committee.  May 12, 2015.  White Oak Campus, Silver Spring, MD. The committee will meet in open session to discuss the development and licensure of Ebola vaccines. [Federal Register]

Public Advisory Committee Meeting.  Blood Products Advisory Committee.  May 13, 2015.  White Oak Campus, Silver Spring, MD. The committee will meet in open session to discuss strategies for implementation of serological and nucleic acid testing for Babesia microti  in blood donors. The committee will also hear update presentations on the following topics: (1) FDA considerations for Hemoglobin S Testing in blood donors; and (2) FDA considerations for a revised blood donor deferral policy for men who have sex with men. [Federal Register]

Public Advisory Committee Meeting. Gastroenterology and Urology Devices Panel of the Medical Devices Advisory Committee. May 14-15, 2015.  White Oak Campus, Silver Spring, MD.  FDA is convening this committee to seek expert scientific and clinical opinion related to reprocessing of duodenoscopes and other endoscopes, as well as automated endoscope reprocessors, based on available scientific information. [Federal Register]

New: Public Meeting.  Preparation for ICH Steering Committee and Expert Working Group Meetings in Fukuoka, Japan.  May 15, 2015.  White Oak Campus, Silver Spring, MD.  FDA is holding this public meeting to provide information and receive comments on the International Conference on Harmonization (ICH) as well as the upcoming meetings in Fukuoka, Japan. [Federal Register]

New: Public Meeting. Interim Assessment of the Program for Enhanced Review Transparency and Communication. May 20, 2015.  White Oak Campus, Silver Spring, MD.  FDA is holding this public meeting to discuss the above referenced interim assessment and to provide an opportunity for public comment on the assessment and the Program to date. [Federal Register]

Public Workshop. FDA Science Forum 2015.  May 27 - 28, 2015.  White Oak Campus, Silver Spring, MD.  The public workshop will highlight science conducted at the FDA by showcasing how scientific research informs regulatory decision making and to provide a forum for developing collaborations within FDA and with external organizations. The focus of the forum will be the eight FDA Regulatory Science priority areas. [Federal Register]

Annual Workshop. European Network of Pediatric Research at the EMA.  May 28, 2015. Canary Wharf, London, UK.  The aim of the annual workshop is to promote the conduct of high quality paediatric clinical studies.  Enpr-EMA is a network of research networks, investigators and centres with recognised expertise in performing clinical studies in children. Its main objective is to facilitate studies in order to increase the availability of medicines authorised for use in children.  [EMA.eu]

Public Hearing. Regulatory Science Initiatives under the Generic Drug User Fee Amendments of 2012.  June 5, 2015.  White Oak Campus, Silver Spring, MD.  This public hearing will provide an overview of the current status of regulatory science initiatives for generic drugs and an opportunity for public input on research priorities in this area. FDA is seeking this input from a variety of stakeholders—industry, academia, patient advocates, professional societies, and other interested parties—as it fulfills its commitment under the Generic Drug User Fee Amendments of 2012 (GDUFA) to develop an annual list of regulatory science initiatives specific to generic drugs. FDA will take the information it obtains from the public hearing into account in developing the fiscal year (FY) 2016 Regulatory Science Plan.  [Federal Register]

New: Public Meeting and Request for Comment. Generic Drug User Fee Amendments of 2012. June 15, 2015.  White Oak Campus, Silver Spring, MD.  Before FDA begins negotiations with the regulated industry on GDUFA reauthorization, it will hold a public meeting at which the public may present its views on the reauthorization, including specific suggestions for changes to the goals referred to in the Generic Drug User Fee Act Program Performance Goals and Procedures (i.e., the Commitment Letter). A docket is also established for public comments. [Federal Register]

Public Workshop. Robotically-Assisted Surgical Devices: Challenges and Opportunities. July 27-28, 2015.  White Oak Campus, Silver Spring, MD.  FDA is holding this public workshop to obtain information on the current challenges and opportunities related to robotically- assisted surgical medical devices, which are classified as Class II medical devices. The purpose of this workshop is to obtain public feedback on scientific, clinical, and regulatory considerations associated with RAS devices.  [Federal Register]


GPhA Launches Biosimilars Council
The Generic Pharmaceutical Association (GPhA) announced the launch of the Biosimilars Council, a division of GPhA.

The Biosimilars Council will comprise manufacturers and stakeholders working to ensure a positive regulatory, reimbursement, political, and policy environment that supports patient access to these more affordable new medicines. The Biosimilars Council also is the industry’s first educational resource for the general public and patient groups seeking information about the safety and effectiveness of biosimilars.

Among the first offerings of the Biosimilars Council is a new educational handbook, The Next Frontier for Improved Access to Medicines: Biosimilars and Interchangeable Biologic Products. This publication explains the benefits and science behind biosimilar medicines— safe, effective alternatives to costly biologic therapies. It explains who will benefit from access to these medicines, outlines the legal and regulatory framework, and illuminates the quality manufacturing and development process in approachable language.

The GPhA Biosimilars Council will begin operations immediately. More at link above. (GPhA release)

C-Path Institute Secures Regulatory Support for Skeletal Muscle Safety Biomarkers
The Critical Path Institute (C-Path) announced that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have issued Biomarker Letters of Support for four skeletal muscle safety biomarkers identified and evaluated by C-Path’s Predictive Safety Testing Consortium (PSTC)’s Skeletal Myopathy Working Group. This is the second time the two agencies have provided a Biomarker Letter of Support to PSTC in the past six months, following FDA and EMA support for the kidney safety biomarkers osteopontin (OPN) and neutrophil gelatinase-associated lipocalin (NGAL).

The skeletal safety biomarkers – myosin light chain (Myl3), skeletal muscle troponin I (sTnI), fatty acid binding protein 3 (FABP3), and creatine kinase muscle type (CK-M, the homodimer CK-MM) – are proteins that can be measured in plasma or serum. Higher levels of these biomarkers could indicate that skeletal muscle injury is occurring.

Each agency’s Letter of Support for Myl3, sTnI, FABP3, and CK-M is intended to encourage the biomarkers’ use in both nonclinical and exploratory clinical studies as markers of skeletal muscle injury. Combined FDA and EMA support indicates that these biomarkers have strong potential for use in humans and warrants additional exploration and gathering of data. With this milestone, work will continue in earnest on the qualification of Myl3, sTnI, FABP3, and CK-M for use in clinical trials. More at link above. (C-Path Institute)

PCORI Board Approves $120 Million for New Patient-Centered Research Projects
The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors today approved more than $120 million to fund 34 patient-centered clinical comparative clinical effectiveness research (CER) studies on a range of conditions and patient populations.

The new awards include nearly $58.5 million to fund five pragmatic clinical studies that seek to answer important questions about radiation therapy for breast cancer, fractures in older adults, and treatments for children with bipolar disorder and Crohn's disease.

This is the second round of awards to be made through PCORI's initiative to support pragmatic clinical studies. Such studies are generally conducted in typical clinical settings during routine care and aim to produce results applicable to a broad range of patients and care situations. More at link above. (PCORI.org)

PCORI Names 21 Inaugural Members of New Advisory Panel on Communication and Dissemination Research
The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors has approved 21 people to serve as the first members of PCORI’s new Advisory Panel on Communication and Dissemination Research (CDR). In addition, the Board named 26 other individuals to fill seats coming open on five of PCORI’s existing multi-stakeholder advisory panels.

The members of the new CDR advisory panel will lend their expertise and experience to help PCORI’s CDR program identify and prioritize key topics in the field for future research. The program concentrates on comparing approaches for communicating and using comparative effectiveness research information, including different approaches for shared decision making between patients and their care providers.

The CDR panel's 21 members represent a range of healthcare stakeholders, including patients, clinicians, researchers, payers, and industry, among others. They bring a variety of relevant experience, including expertise in health literacy, numeracy, and risk communication, as well as dissemination and implementation research.

“Research on how best to communicate and disseminate research results is vital because as the number of healthcare options grows, so does the need for patients and their clinicians to have useful information about what works best to help them make fully informed decisions,” said Jean Slutsky, PA, MSPH, PCORI’s Chief Engagement and Dissemination Officer and Director of the CDR Program. “We’ll benefit from the insights of this knowledgeable and diverse group of advisory panelists.” More at title link above. (PCORI.org)

Preventing Medication Errors in the EU: EMA invites comments on draft good practice guides
The European Medicines Agency (EMA), on behalf of the European Union (EU) Regulatory Network, has released two draft good practice guides that aim to improve the reporting, evaluation and prevention of medication errors by regulatory authorities and pharmaceutical industry throughout the EU. The deadline for stakeholders to send their comments to EMA is 14 June 2015.

One of the two guides focuses on the prevention of medication errors. It describes the main sources and types of these errors and proposes measures to minimise the risk of medication errors throughout the life cycle of a medicine.

The other guide provides guidance on how suspected adverse reactions that are caused by medication errors should be recorded, coded, reported and assessed. It also gives recommendations for marketing-authorisation holders on how to report information on medication errors that are brought to their attention but have not caused adverse reactions. This information must be provided in periodic safety update reports and in the risk management plans that are compulsory for all medicines. This allows a continuous evaluation of the benefits and risks of a medicine based on real life data by regulators.

The guidance released today is one of the key deliverables of the EMA/Heads of Medicines Agencies (HMA) joint action plan on medication errors agreed in 2013. It was developed in consultation with the European Commission’s Patient Safety Quality of Care Working Group and takes into account recommendations from stakeholders that were gathered during a workshop held at EMA in February 2013. More at link above. (EMA.eu)

Regulatory information – reflection on chemical structure and properties to be considered for the evaluation of new active substance status
EMA releases draft reflection paper for public consultation

The European Medicines Agency (EMA) has released a draft reflection paper that outlines the chemical structure and properties criteria to be considered by EMA’s Committee for Medicinal Products for Human Use (CHMP) for the evaluation of a new active substance status. The reflection paper also outlines the elements that applicants are required to include in their marketing authorisation applications in support of their new active substance status claim.

Stakeholders have until 24 July 2015 to provide their comments to qwp@ema.europa.eu using the form provided.

The draft reflection paper focuses on active substances that are structurally related to an already approved active substance (i.e., salt, ester, ether, isomer, mixture of isomers, complex or derivative of a chemical structure).

It does not cover biological and biotechnological active substances and active substances to be included in radiopharmaceutical medicines. These products will be addressed in a separate paper at a later stage. More at link above. (EMA.eu)

Scientific advice leads to stronger applications from industry
The majority of clinical development plans submitted for scientific advice to the European Medicines Agency (EMA) prior to a marketing authorisation application were found not suitable for future benefit-risk assessment. Companies that changed their clinical development plans in accordance with the recommendation from EMA were more likely to be granted a marketing authorisation.

These are the main findings of an analysis of marketing authorisation application outcomes between 2008 and 2012 conducted by staff members of EMA and its Scientific Advice Working Party (SAWP) and published in Nature Reviews Drug Discovery.

EMA, through its SAWP, provides scientific advice to companies during the development of their medicines to help them design trials that are scientifically sound and generate adequate data for the benefit-risk assessment by EMA’s Committee for Medicinal Products for Human Use (CHMP). More at link above. (EMA.eu)

Open Payments Dispute Resolution Call Reveals Tension Between Manufacturers and Covered Recipients
The Centers for Medicare and Medicaid Services (CMS) recently held a teleconference for physicians and teaching hospitals to help them through the process of reviewing their Open Payments data and potentially disputing their information. Notably, CMS has streamlined their teaching tools for physicians to a manageable 38-page PowerPoint. CMS re-iterated their position that they will not be involved in mediating disputes. However, “CMS will monitor disputes and resolutions to inform the program auditing process,” they stated. “How many disputes are initiated as well as the volume of unresolved disputes,” will be of particular interest to the government.

A troublesome point for physicians and teaching hospitals that consistently came up during the call is the lack of context or consistent reporting of the “Nature of Payments” categories by manufacturers. For example, one caller articulated the process of reviewing payments for physicians who received a device or instrument loan. “One manufacturer reported it as a gift, some as space rental, and some as consulting payments,” the caller noted. Indeed, it is up to the manufacturer to determine the nature of payment of certain transfers of value. For compliance personnel working in hospitals attempting to verify whether or not these payments are correct, it can be very difficult to determine the accuracy of such varying payment titles. This is especially true when manufacturers report payments to physicians as aggregate totals throughout a given year.

CMS agreed that the nature of payment categories may be expanded or clarified in the future, but noted that a public forum—with input from all parties—would be necessary to change the system. More at link above.  (Policy and Medicine)

Chinese Scientists Edit Genes of Human Embryos, Raising Concerns
The experiment with human embryos was dreaded, yet widely anticipated. Scientists somewhere, researchers said, were trying to edit genes with a technique that would permanently alter the DNA of every cell so any changes would be passed on from generation to generation.

Those concerns drove leading researchers to issue urgent calls in major scientific journals last month to halt such work on human embryos, at least until it could be proved safe and until society decided if it was ethical.

Now, scientists in China report that they tried it. The Chinese researchers did not plan to produce a baby — they used defective human embryos — but did hope to end up with an embryo with a precisely altered gene in every cell but no other inadvertent DNA damage. None of the 85 human embryos they injected fulfilled those criteria. In almost every case, either the embryo died or the gene was not altered. Even the four embryos in which the targeted gene was edited had problems. Some of the embryo cells overrode the editing, resulting in embryos that were genetic mosaics. And speckled over their DNA was a sort of collateral damage — DNA mutations caused by the editing attempt.

“Their study should give pause to any practitioner who thinks the technology is ready for testing to eradicate disease genes during I.V.F.,” said Dr. George Q. Daley, a stem cell researcher at Harvard, referring to in vitro fertilization. “This is an unsafe procedure and should not be practiced at this time, and perhaps never.” More at link above. (NY Times)

Big Medical Device Makers Form Their First Industry Trade Group in Asia
The Asia Pacific Medical Technology Association, or APACMed, which is expected to be announced on Friday, hopes that by working together in the historically fragmented region, governments and regulators will take the group’s interests more seriously.

The broad constituent base, which the group hopes will include local companies in addition to multinational device makers, “will make the government and regulators and policy makers look at us a little bit differently,” APACMed ceo Fredrik Nyberg tells us. All of the founding APACMed members are western companies but the leadership is talking with a number of companies in Japan, Korea, China and Singapore, as well as an Indian industry association, he adds.

Among the key issues to address, he explains, are new regulations in China for medical device clinical trials that were issued last year and could stall the time it takes to get products to market by 12 to 24 months.

Though there have been medical device industry groups in the U.S. and Europe for decades, Asia is a newer and more fragmented market. And so, Nyberg says that senior industry leaders have been talking for the last year or two about the importance of working together and forming an industry group to more effectively affect change in regulations, treatment guidelines, laws and ethics.  More at link above. (Pharmalot)

Indian Parliamentary committee recommends price caps for all drugs
A parliamentary committee has recommended that price caps should be extended to all drugs in the country and that the government should expedite the capping process.

The law allows the National Pharmaceutical Pricing Authority (NPPA) regulator to fix prices of drugs on a list of essential medicines, thereby keeping prices in check in a country where a majority of people live on less than $2 a day and health insurance is scarce.

But the standing committee on chemicals and fertilisers, a panel of 31 lawmakers, noted in a report tabled in parliament that all medicines should be available in the market at an affordable price.

"The committee recommend that the scope of price control needs to be enlarged to make all the drugs available, especially lifesaving drugs in all parts of the country," the committee's report said.  More at link above. (Reuters)


21st Century Cures Legislative Phase Continues
After nearly a year of listening to patients, innovators, researchers, providers, and regulators, 21st Century Cures next week will take the next step in the legislative phase with a hearing, “Legislative Hearing on 21st Century Cures,” on Thursday, April 30, 2015, at 10:00 a.m. in room 2123 of the Rayburn House Office Building. Subcommittee members will discuss a forthcoming bipartisan discussion draft of this highly anticipated legislation, the product of a yearlong listening session and bipartisan negotiations.

The committee has held eight hearings and issued several white papers in this initiative since its launch last year. Committee members, both in Washington, D.C., and across the country, have also hosted more than two-dozen roundtables to generate ideas for this initiative. Staff continues to work together around the clock as they have for the past several months and legislative text will be made available in the coming days.

Dr. Kathy Hudson, Deputy Director for Science, Outreach, and Policy at the National Institutes of Health, Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research at the Food and Drug Administration, and Dr. Jeff Shuren, Director of the Center for Devices and Radiological Health at the FDA will testify. (Energy & Commerce Committee)

What’s Next for #Cures2015
Chairman Fred Upton (R-MI) and Rep. Diana DeGette (D-CO) this week traveled to Southwest Michigan to talk about their nonpartisan 21st Century Cures initiative in Upton’s home district. The two launched 21st Century Cures last spring and have spent the past year gathering ideas from patients and experts across the country for how to improve the complete health care innovation infrastructure in the United States.

“There is great excitement and anticipation building over the next phase of 21st Century Cures. Our teams have spent countless hours negotiating over the last few months on policy language and they continue to work by the hour in this landmark effort to provide folks more cures and treatments,” said Upton. “I am hopeful that we’ll have an agreement by the end of the month and the legislative phase can swiftly progress. This is an exciting time.” Upton said he plans to introduce the 21st Century Cures bill in the next few weeks and have it on President Obama's desk by the end of the year.

DeGette said that while the initiative has already received early bipartisan support, it's also gained "tremendous consensus" from a number of groups, including researchers, students and patient advocacy organizations. The only people currently against the plan are people who don't support the government conducting biomedical research, according to DeGette.

Upton said he plans to have the bill on the House floor by June so the legislation can stay far ahead of the politics surrounding the lead-up to the 2016 presidential election. More at link above. (Energy & Commerce)

House Approves Key Bill to Combat Prescription Drug Abuse
The House of Representatives approved on April 21 H.R. 471, the Ensuring Patient Access and Effective Drug Enforcement Act of 2015, as part of the committee’s ongoing effort to address the growing problem of prescription drug abuse.

H.R. 471, authored by full committee Vice Chairman Marsha Blackburn (R-TN) and Reps. Tom Marino (R-PA), Peter Welch (D-VT), and Judy Chu (D-CA), clarifies key phrases in the Controlled Substances Act to establish clear and consistent enforcement standards and promotes collaboration among government agencies, patients, and industry stakeholders to ensure patients have access to medications.

The bipartisan bill requires the Secretary of Health and Human Services, in coordination with the head of the Drug Enforcement Administration, to submit a report to Congress identifying obstacles to legitimate patient access to controlled substances, issues with diversion of controlled substances, and how collaboration between agencies and stakeholders can benefit patients and prevent prescription drug abuse. More at link above.  (Energy & Commerce)

Massachusetts Introduces Drug Cost Transparency Bill That Would Cap Prices For Certain Products; North Carolina and Pennsylvania Also Introduce Bills Requiring Drug Cost Reports
Massachusetts last week introduced a bill that calls for more transparency in the prices drug manufacturers charge for certain “critical prescription drugs.” Like California and Oregon before it, Massachusetts S.1048 would require that companies report a wide range of cost and pricing information. However, Massachusetts ups the ante further than other states because they do not stop at transparency. As outlined in the bill, if the state determines the price of the prescription drug is “significantly high,” they may set the maximum allowable price that the manufacturer can charge for that prescription drug sold for use in Massachusetts.

North Carolina and most recently Pennsylvania have also released cost transparency legislation. Pennsylvania’s bill would restrict insurance coverage for any company that did not produce a cost report. As an update on other state activity, California’s Assembly Health Committee is debating their Pharmaceutical Cost Transparency Act (Bill 463). The Committee will reportedly dwell on the various suggestions and come back to the bill next week. More at link above. (Policy and Medicine)


A partial listing of sources reviewed for this newsletter:  AdvaMed Smartbrief; AHRQ Newsletter; Alzheimers Association; Alzheimers Research Forum Newsletter; BioCentury; Biopharma Reporter; BIOtechNow; CDISC Monthly Newsletter; CER Daily Newsfeed (NPC); DIA Daily; Drug Daily Bulletin;  EMA website; EP Vantage; Evaluate Pharma; Eye on FDA; FDA.gov; FDA Law Blog; Federal Register Table of Contents; Fierce Medical Devices; Fierce Pharma; Fierce Vaccines; FDLI Smartbrief; Genomeweb; Health Industry Washington Watch; Government Health IT; Health IT Security; Institute of Medicine News; MedCityNews; Medical Device Daily; Medical Device & Diagnostic Industry; MedPage Today; NPC Bulletin; Nutra Ingredients USA; Pharmabiz; Pharmafile; Pharma IQ; PharmaTimes; PhRMA website; PM Live; Policy and Medicine (newsletter); Regulatory Focus; RegLink News; US FDA Daily Digest Bulletin.