DIA's Content Currents provides you with new and important global regulatory developments and their impact on pharmaceutical, biotechnology, and medical product development.
EDITION PUBLISHED: December 6, 2013
SECTION 1 FDA GUIDANCES & MAPPS
On December 5, FDA released a draft guidance entitled “Draft Guidance for Industry on Bioequivalence Studies With Pharmacokinetic Endpoints for Drugs Submitted Under an Abbreviated New Drug Application” This guidance provides recommendations to applicants planning to include bioequivalence (BE) information in abbreviated new drug applications (ANDAs) and ANDA supplements. The guidance describes how to meet the BE requirements set forth in FDA regulations. [Federal Register]
On December 5, FDA announced that a proposed collection of information has been submitted to OMB on “Over the Counter Drugs; labeling Requirements.” In 1999, (the 1999 labeling final rule), FDA amended the regulations governing requirements for human drug products to establish standardized format and content requirements for the labeling of all marketed over-thecounter (OTC) drug products. The regulations require OTC drug product labeling to include uniform headings and subheadings, presented in a standardized order, with minimum standards for type size and other graphical features. Currently marketed OTC drug products are already required to be in compliance with these labeling requirements, and thus will incur no further burden to comply. The burden to comply with the labeling requirements is a one-time burden applicable only to new OTC drug products introduced to the marketplace under new drug applications, abbreviated new drug applications, or an OTC drug monograph, except for products in ‘‘convenience size’’ packages. [Federal Register]
On December 4, 2013, FDA distributed three draft guidances:
“Draft Guidance for Industry on Interim Product Reporting for Human Drug Compounding Outsourcing Facilities Under Section 503B of the Federal Food, Drug, and Cosmetic Act.” The draft guidance addresses new provisions in the Federal Food, Drug, and Cosmetic Act (the FD&C Act), as amended by the Drug Quality and Security Act (DQSA), and sets forth an interim electronic submission method for human drug compounders that choose to register as outsourcing facilities. [Federal Register]
“Draft Guidance for Industry on Registration for Human Drug Compounding Outsourcing Facilities Under Section 503B of the Federal Food, Drug, and Cosmetic Act.” The draft guidance addresses new provisions in the Federal Food, Drug, and Cosmetic Act (the FD&C Act), as amended by the Drug Quality and Security Act (DQSA). The draft guidance is intended to assist human drug compounders that choose to register as outsourcing facilities (outsourcing facilities) in registering with FDA. The draft guidance provides information on how an outsourcing facility should submit facility registration information electronically. [Federal Register]
“Draft Guidance; Pharmacy Compounding of Human Drug Products Under Section 503A of the Federal Food, Drug, and Cosmetic Act; Withdrawal of Guidances.” The draft guidance announces the Agency’s intention with regard to enforcement of the Federal Food, Drug, and Cosmetic Act (the FD&C Act) to regulate entities that compound drugs, now that the FD&C Act has been amended by the Drug Quality and Security Act. When final, the guidance will reflect the Agency’s current thinking on the issues addressed by the guidance.
The Agency is also announcing the withdrawal of a guidance entitled, ‘‘Enforcement Policy During Implementation of Section 503A of the Federal Food, Drug, and Cosmetic Act,’’ which was issued in November 1998, and the withdrawal of CPG Section 460.200 of the Compliance Program Guidance (CPG) Manual entitled, ‘‘Pharmacy Compounding,’’ which was issued in May 2002. These guidances are being withdrawn because they are no longer consistent with the Agency’s current thinking on the issues they address. [Federal Register]
Also on December 4, 2013, FDA issued proposed rules:
“Bulk Drug Substances That May Be Used To Compound Drug Products in Accordance With Section 503B of the Federal Food, Drug, and Cosmetic Act, Concerning Outsourcing Facilities; Request for Nominations.” FDA is preparing to develop a list of bulk drug substances (bulk drugs) that may be used to compound drug products in accordance with section 503B of the Federal Food, Drug, and Cosmetic Act, concerning outsourcing facilities. To identify candidates for this bulk drugs list, interested groups and individuals may nominate specific bulk drug substances, and FDA is describing the information that should be provided to the Agency in support of each nomination. Submit either electronic or written nominations for the bulk drug substances list by March 4, 2014. [Federal Register]
“Drug Products That Present Demonstrable Difficulties for Compounding Under Sections 503A and 503B of the Federal Food, Drug, and Cosmetic Act; Request for Nominations.” FDA is preparing to develop a list of drug products that present demonstrable difficulties for compounding (difficult to compound list). To identify candidates for this list, FDA is encouraging interested groups and individuals to nominate specific drug products or categories of drug products and is describing the information that should be provided to the Agency in support of each nomination. Submit written or electronic comments by March 4, 2014. [Federal Register]
“List of Bulk Drug Substances That May Be Used in Pharmacy Compounding; Bulk Drug Substances That May Be Used To Compound Drug Products in Accordance With Section 503A of the Federal Food, Drug, and Cosmetic Act.” FDA is withdrawing the proposed rule to list bulk drug substances used in pharmacy compounding and preparing to develop a list of bulk drug substances (bulk drugs) that may be used to compound drug products, although they are neither the subject of a United States Pharmacopeia (USP) or National Formulary (NF) monograph nor components of FDA-approved drugs. To identify candidates for this bulk drugs list, interested groups and individuals may nominate specific bulk drug substances, and FDA is describing the information that should be provided to the Agency in support of each nomination. FDA is withdrawing the proposed rule published January 7, 1999 (64 FR 996), as of December 4, 2013. Submit written or electronic nominations for the bulk drug substances list by March 4, 2014. [Federal Register]
On December 2, 2013, FDA announced that a proposed collection of information was submitted to OMB, for review and clearance, entitled “Focus Groups About Drug Products as Used by the Food and Drug Administration.” FDA’s CDER, Office of the Commissioner, and any other Centers or Offices conducting focus groups about regulated drug products may need to conduct focus groups on a variety of subjects related to consumer, patient, or healthcare professional perceptions and use of drug products and related materials. Annually, FDA projects about 20 focus group studies using 160 focus groups with an average of 9 persons per group, and lasting an average of 1.75 hours each. FDA is requesting this burden for unplanned focus groups so as not to restrict the Agency’s ability to gather information on public sentiment for its proposals in its regulatory and communications programs. [Federal Register]
Also on December 2, 2013, FDA announced a proposed collection of information that has been submitted to OMB entitled “Guidance for Industry on Planning for the Effects of High Absenteeism To Ensure Availability of Medically Necessary Drug Products.” The guidance recommends that manufacturers of drug and therapeutic biological products and manufacturers of raw materials and components used in those products develop a written Emergency Plan (Plan) for maintaining an adequate supply of medically necessary drug products (MNPs) during an emergency that results in high employee absenteeism. The guidance discusses the issues that should be covered by the Plan. [Federal Register]
On December 2, 2013, FDA’s Office of New Drugs issued a Manual of Policies and Procedures (MAPP) entitled, “Good Review Practice: Clinical Review of Investigational New Drug Applications.” This MAPP was prepared to assist FDA clinical review staff in reviewing INDs during drug development. It provides policies and procedures for the clinical review of investigational new drug applications (INDs) using good review practices (GRPs) within the Center for Drug Evaluation and Research. MAPP
On November 29, 2013, FDA announced an opportunity for public comment on the proposed collection of information in connection with the research entitled ‘‘Eye Tracking Study of Direct-to-Consumer Prescription Drug Advertisement Viewing.’’ This study is designed to use eye tracking technology to explore how consumers view direct to consumer (DTC) prescription drug advertisements (ads) that include text regarding risk information and reporting side effects and that vary in the amount of distracting audio and visual content during the presentation of the risk information. Submit either electronic or written comments on the collection of information by January 28, 2014. [Federal Register]
On November 27, 2013, FDA reopened the comment period for the “Draft Guidance for Industry on Generic Drug User Fee Amendments of 2012: Questions and Answers (Revision 1).” FDA) is reopening the comment period for the notice of availability entitled ‘‘Draft Guidance for Industry on Generic Drug User Fee Amendments of 2012: Questions and Answers (Revision 1)’’, published in the Federal Register of September 10, 2013 (78 FR 55261). In that notice, FDA requested public comment on the draft guidance. FDA is reopening the comment period due to the inability of some commenters to submit comments through the www.regulations.gov Web site from November 4, 2013, through November 13, 2013, due to technical difficulties. Submit either electronic or written comments to the docket by December 11, 2013. [Federal Register]
On November 27, 2013, FDA announced a pilot project, “Transport Format for the Submission of Regulatory Study Data.” CDER and CBER are announcing a pilot project to evaluate the Clinical Data Interchange Standard Consortium (CDISC) Submission Data Standards (SDS) Extensible Markup Language (XML) transport format for the submission of regulatory study data. The current study data transport format supported by FDA is the SAS Transport (XPORT) version 5 file format. Submit either electric or written requests for participation in the pilot project by January 27, 2014. [Federal Register]
On November 25, 2013, FDA announced the availability of “Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products.” The guidance document provides sponsors and individuals that design and implement preclinical studies with recommendations on the substance and scope of preclinical information needed to support clinical trials for investigational products reviewed by the Office of Cellular, Tissue and Gene Therapies (OCTGT). The product areas covered by this guidance are cellular therapy, gene therapy, therapeutic vaccination, xenotransplantation, and certain biologic-device combination products, which OCTGT reviews. The guidance clarifies current expectations regarding the preclinical information that would support an investigational new drug application (IND) and a biologics license application (BLA) for these products. [Federal Register]
On November 20, 2013, FDA announced that it has extended the comment period for the draft guidance “Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products.” The draft guidance document provides sponsors of Investigational New Drug Applications for cellular therapy (CT) and gene therapy (GT) products (referred to collectively as CGT products) with recommendations to assist in designing early-phase clinical trials of CGT products. In the notice, we requested comments on the draft guidance. We are taking this action to allow interested persons additional time to submit comments and to allow for public discussion at the February 25–26, 2014, Cellular, Tissue, and Gene Therapies Advisory Committee meeting, where FDA will present the draft guidance document for review. FDA is extending the comment period on the draft guidance. Submit either electronic or written comments by May 9, 2014. [Federal Register]
On November 20, 2013, FDA announced the availability of a draft guidance entitled, “Draft Guidance for Industry on Product Name Placement, Size, and Prominence in Advertising and Promotional Labeling.” When finalized, the draft guidance will replace the guidance of the same title issued January 25, 2012. The draft guidance clarifies the requirements for product name placement, size, prominence, and frequency in promotional labeling and advertising for prescription human drugs, including biological drug products, and prescription animal drugs and articulates the circumstances under which FDA intends to exercise enforcement discretion. Comments are due January 21, 2014. [Federal Register]
In the November 15, 2013 issue of the Federal Register, FDA announced its participation in the “International Medical Device Regulators Forum; Medical Device Single Audit Program International Coalition Pilot Program.” The Medical Device Single Audit Program (MDSAP) was designed and developed to ensure a single audit will provide efficient yet thorough coverage of the diverse international regulatory requirements of medical devices quality management systems and other specific regulatory requirements of the regulatory authorities participating in the pilot program. FDA will be participating in the MDSAP and will accept the resulting audit reports as a substitute for routine Agency inspections. [Federal Register]
SECTION 2 FDA NOTES & RELATED NEWS
FDA CDER Strategic Plan 2013-2017
The FDA Center for Drug Evaluation and Research (CDER) released its 2013-2017 Strategic Plan (access at title link above). “CDER intends to retain the same strategic goals and objectives that define the focus—the “what”—of our regulatory activities. This strategic plan is focused on “how” we will enhance operations to better accomplish that work. We have identified four strategies for accomplishing our strategic objectives over the next 5 years.” The strategies include: 1) Smarter Regulation 2) Scientific Innovation 3) Lean Management and 4) Business Modernization. (FDA.gov)
Update of FDA Unique Device Identification Submission System
The US Food and Drug Administration has published draft technical specifications related to its Unique Device Identification (UDI) rule finalized earlier this year.
Manufacturers with US medical device market authorization are required to submit device identification data into a Global Unique Device Identification Database (GUDID) managed by the FDA. Firms now have two options for submitting information to the GUDID: a GUDID Web Interface
that requires users to set up GUDID accounts, and an HL7 SPL
option for submitting device information via xml file one record at a time. (Mass Device)
New Structure for the Office of Compliance
This page was updated on the CDRH web pages on November 18, 2013.
Cox Departing FDA
FDA Commissioner Margaret Hamburg announced in an internal memo that Virginia Cox will depart as associate commissioner for external affairs for the agency in January. Cox joined FDA in September 2011. Steven Immergut, acting assistant commissioner for media affairs, will serve as acting associate commissioner for external affairs. Erica Jefferson, deputy director of strategy in FDA's Office of Media Affairs, will serve as acting assistant commissioner for media affairs. (BioCentury 12/6/13)
Importer Sues FDA for Blocking Ingredient Shipment
In an unusual development, an importer has filed a lawsuit against the FDA for detaining a shipment of bulk acetaminophen that was destined for a customer – a contract manufacturer, specifically. And the refusal by the agency to clear the shipment is raising questions about how an ‘end user’ is defined.
In its lawsuit, H&M charges that the FDA was “overreaching, arbitrary and capricious, unlawful and discriminatory” in refusing to allow the shipment to proceed. The importer wants a temporary restraining order that would allow shipments and a ruling that the acetaminophen would not be considered misbranded.
“OTC drug products are often manufactured by a single manufacturer for many private label distributors,” the law blog writes. “As long as the finished product is ultimately compliant, FDA’s refusal to permit the import of otherwise lawful bulk drug product simply because a customer for the finished product had not yet been identified at the time of import is difficult to understand, and does not appear to be consistent with the agency’s regulations on drugs intended to be further manufactured.” More at link above. (Pharmalot)
SECTION 3 AGENCY AND ADVISORY COMMITTEE MEETINGS
Patient-Focused Drug Development: Disease Area Meetings Planned for FY2013-2015
NOTE: Rescheduled Date: OPDP Enforcement Actions Webinar – January 30, 2014 at 11:00 AM ET
The Office of Prescription Drug Promotion (OPDP) of the U.S. Food and Drug Administration (FDA) invites you to participate in the next Enforcement Webinar on January 30, 2014, from 11:00 AM to 12:00 Noon (ET). Viewers can begin submitting questions 30 minutes prior to the webinar start time. During the webinar, OPDP will give stakeholders a chance to directly communicate with the Agency about clarifications or questions concerning recent Warning Letters and Untitled Letters issued by OPDP. This particular webinar will cover Warning Letters and Untitled Letters issued from July 2013 through September 2013.
These focused webinars support OPDP's mission to protect the public health by assuring that prescription drug information is truthful, balanced, and accurately communicated.
To join the meeting: https://collaboration.fda.gov/opdp1028
Public Meeting. FDA Patient Focused Drug Development: Fibromyalgia. December 10, 2013.
The public meeting is intended to allow FDA to obtain patients’ perspectives on the impact of fibromyalgia on daily life as well as the available therapies for fibromyalgia. [Federal Register
Public Advisory Committee Meeting. FDA Risk Communications Advisory Committee. December 17, 2013.
This meeting will be open to the public. The Committee will meet to identify and discuss new methods for communicating risk information as part of Risk Evaluation and Mitigation Strategies (REMS) to health care providers. The discussion will also address how sponsors and FDA can evaluate whether REMS communications are reaching the targeted population, are increasing awareness and understanding of the key risk messages, as well as whether the communications are having the intended impact on knowledge, behaviors, and/or outcomes. [Federal Register
Public Workshop. Complex Issues in Developing Drug and Biological Products for Rare Diseases. January 6-7, 2014.
The workshop will discuss complex issues in clinical trials for developing drug and biological products for rare diseases, including endpoint development and selection, use of surrogate endpoints, and other topics. FDA seeks input on these topics from multiple stakeholders to develop a strategic plan to encourage and accelerate the development of new therapies for rare diseases. (Federal Register
Public Workshop. Complex Issues in Developing Medical Devices for Pediatric Patients Affected by Rare Diseases. January 8, 2014.
The purpose of the public workshop is to discuss issues related to a series of broad topics associated with medical devices for the diagnosis and treatment of pediatric patients affected by rare diseases. The input from this public workshop will help in developing a strategic plan to encourage and accelerate the development of new medical devices and therapies for pediatric patients affected by rare diseases. (Federal Register
Public Workshop. Sixth Annual Sentinel Initiative. January 14, 2014.
Convened by the Engelberg Center for Health Care Reform at the Brookings Institution and supported by a cooperative agreement with FDA, this 1-day workshop will bring the stakeholder community together to discuss a variety of topics on active medical product surveillance. (Federal Register
Public Meeting. FDA Patient Focused Drug Development: Sickle Cell Disease. February 7, 2014.
The public meeting is intended to allow FDA to obtain patients’ perspectives on the impact of sickle cell disease on daily life and on available therapies for sickle cell disease. (Federal Register
SECTION 4 OTHER REGULATORY AUTHORITIES & ORGANIZATIONS
Obama announces $100M HIV cure initiative
The Obama administration will shift $100 million from existing programs to a newly announced initiative to find a cure for HIV. President Barack Obama also signed an extension of the President's Emergency Program for AIDS Relief and said the U.S. would donate $5 billion over the next three years to the Global Fund to Fight AIDS, TB and malaria if other countries contribute $10 billion. National Public Radio/Shots blog
(12/2) (Via DIA Global Smartbrief 12/5/13)
Pamela McInnes Named NCATS Deputy Director
Pamela M. McInnes, D.D.S., has been named deputy director of the National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health. McInnes currently serves as director of the Division of Extramural Research at the National Institute of Dental and Craniofacial Research (NIDCR). She will join NCATS in January 2014.
At NIDCR, McInnes was responsible for all of the institute’s extramural research, which ranges from basic through clinical research, including large and complex clinical and population-based trials. She is committed to the rigorous and robust conduct of clinical trials that adhere to the highest standards for human subject protection and data integrity. Her work in translational sciences has led to numerous product development and clinical evaluation programs, and she is involved actively with the broader extramural research community in efforts to reduce morbidity and mortality attributable to a large spectrum of diseases and disorders. (NIH News)
FTC Announces Agenda for Workshop Examining Competition Issues Surrounding Biologic and Follow-on Biologic Medicines: Biosimilars and Interchangeables
The Federal Trade Commission has announced the agenda for the upcoming public roundtable on “Follow-on Biologics: Impact of Recent Legislative and Regulatory Naming Proposals on Competition.” The workshop will be held in the FTC Conference Center at 601 New Jersey Ave., N.W., in Washington, DC, on December 10, 2013.
FTC Chairwoman Edith Ramirez will provide welcoming remarks at 8:30 a.m. to open the workshop. Her remarks will be followed by presentations from a wide range of experts in the research, development, commercialization and sale of biosimilar and interchangeable follow-on biologic medicines. Biologic medicines comprise the fastest-growing sector of pharmaceuticals and target such difficult-to-treat diseases as cancer, diabetes, and multiple sclerosis. More at title link above. (FTC.gov)
GPhA Requests Extension of Comment Period on Proposed Labeling Rule
In a letter dated November 27, 2013, the Generic Pharmaceutical Association (GPhA) requested a 60-day extension to provide comments on the FDA’s Proposed Rule - Supplemental Applications Proposing Labeling Changes for Approved Drugs and Biological Products.
GPhA noted in their request that: “The proposed rule includes revisions that would dramatically alter the current regulations governing generic drugs with respect to both when and how a labeling change would be required. The changes as proposed will have far reaching consequences affecting patient safety as well as health care costs. They also create an additional burden not anticipated in the business and compliance models of generic and biosimilar manufacturers and marketers.” GPhA requests this extension “to afford all interested stakeholders an opportunity to provide commentary based on a robust analysis of the various legal and commercial implications of the proposal.” More at link above. (Lachman Consultants)
Innovation in Medical Evidence Development and Surveillance (IMEDS) Methods Research Agenda Report Released
The FDA introduced the Sentinel Initiative as a national electronic system intended to transform FDA’s ability to track the safety of marketed drugs, biologics, and medical devices. Launched in May 2008 by FDA, the Sentinel Initiative aims to develop and implement a proactive system that will complement existing systems that the Agency has in place to track reports of adverse events linked to the use of its regulated products.1
IMEDS-Methods is a program within the Reagan-Udall Foundation that supports FDA’s mission 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 the adoption of its findings as appropriate. In meeting this mission, IMEDS-Methods will also be able to add to the general body of knowledge for using automated health data for broader post-market evidence generation on regulated products.2
This is a report of the IMEDS methods research agenda with proposed projects that would be funded by IMEDS. Research projects will be released publicly in the form of RFPs which will be open to qualified researchers. Full report at title link above. (Reagan Udall Foundation)
Blog: PCORI's Strategic Plan: Setting the Framework for Success
(11/29, Joe Selby, MD, MPH, The PCORI Blog) comments “...Now, we have a focused roadmap to further guide our efforts – PCORI's strategic plan, approved by our Board at its Board's recent meeting in Atlanta...Our strategic plan identifies three primary goals that we must achieve to realize our mission and vision: Substantially increase the quantity, quality, and timeliness of useful, trustworthy information available to support health decisions; Speed the implementation and use of patient-centered outcomes research (PCOR) evidence; and Influence clinical and healthcare research funded by others to be more patient-centered.” Full (CER Daily Newsfeed, National Pharmaceutical Council, 12/2/13)
AHRQ Director Kronick Puts Stamp On Agency With Refocus On Evidence
(11/27, Scott Steinke, The Pink Sheet Daily) reports “...[AHRQ Director Richard Kronick] outlined four main agency priorities that parallel the mission statement. The first is to improve health care quality by accelerating implementation of patient-centered outcomes research (PCOR) in health care practice...Kronick said that while some large physician groups have made progress in creating information sharing and team-based support needed to incorporate PCOR into their practices, ‘these kinds of efforts have been particularly hard for small- and medium-sized practices, and we'll be working on an intervention to try to provide the support for these practices to really make progress.’” Paid Subscription Required (CER Daily Newsfeed, National Pharmaceutical Council, 12/2/13)
IOM Report: Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee
In response to concerns about gene transfer, NIH established in 1974 the Recombinant DNA Advisory Committee (RAC) to provide oversight and a public forum for discussion. Today, NIH-supported researchers are still required to submit all research protocols to the RAC. The RAC then determines which of these require further review and public discussion by the RAC.
With the accumulation of safety data and increased experience with its applications, gene transfer research’s associated risks are now better understood. Therefore many have argued that today RAC review is redundant and unnecessary in its current form. NIH asked the IOM to form a committee to determine whether gene transfer research continues to raise concerns that warrant extra oversight by the RAC of individual clinical trial protocols involving gene transfer. In most cases, human gene transfer research is no longer novel or controversial enough to require additional review from the RAC, says the report. Access report at title link above. (IOM News)
IOM Report: Improving and Accelerating Therapeutic Development for Nervous System Disorders - Workshop Summary
Although there is a high burden associated with nervous system disorders, development of new therapeutics remains stagnant. Over the last decade, fewer new drugs for nervous system disorders have garnered approval in comparison to other therapeutic areas. There are several challenges to the current drug development pipeline for nervous system disorders.
The IOM Forum on Neuroscience and Nervous System Disorders held a workshop to examine opportunities to accelerate early phases of drug development for nervous system drug discovery. Participants at the workshop discussed challenges in neuroscience research for enabling faster entry of potential treatments into first-in-human trials, explored how new and emerging tools, technologies, and techniques may improve the efficiency of research, and considered mechanisms to facilitate a more effective and efficient development pipeline. This document summarizes the workshop. Access report at link above. (IOM News)
Building the Science for a Population Health Movement
In this discussion paper, Adler and colleagues argue that research and action must go hand in hand in the movement for population health improvement. Their paper traces the integrative and rapidly evolving nature of recent developments in the field, with increasing collaboration across disciplines to build a "multilevel understanding of health, from cells to society." The authors call for building better bridges among researchers, policy makers, and practitioners to create an innovative, continuously updated learning environment. They also identify several areas where more research is needed, including: understanding how social and economic disadvantage "gets under the skin" to shape health outcomes, and learning how to "isolate the effects of a single aspect of the environment from the multiple confounding effects of the social system in which it is embedded.” Access report at link above. (IOM News)
Recommendations for Next Generation Health Information Exchange
Twenty years after the Workgroup for Electronic Data Interchange issued the WEDI Report that ushered in the HIPAA standards to automate health care administrative and financial transactions, the group has released a roadmap to guide the next generation of health information exchange.
More than 200 volunteer stakeholders worked throughout 2013 to identify opportunities, challenges and action plans for four “megatrend” areas: Patient Engagement, Innovative Encounter Models, Data Harmonization and Exchange, and Payment Models. The 122-page 2013 WEDI Report, “The Right Information to the Right Place at the Right Time,” includes 10 recommendations supported with action plans for the four “megatrend” areas. More at title link. (Health Data Management)
Pfizer Expands Clinical Trial Data Access Policy and Launches Data Access Portal
On December 4, 2013, Pfizer Inc. announced an update of its clinical trial data access policy that will simplify and broaden access to information gathered in Pfizer-sponsored clinical trials. The updated policy builds upon and expands the company’s established methods of clinical trial information sharing, including Pfizer’s long track record of submitting for publication results from all interventional clinical trials in patients and its pioneering efforts to provide clinical trial results and data to study participants.
Pfizer’s updated policy meets or exceeds the “Principles for Responsible Data Sharing”
issued by the Pharmaceutical Research and Manufacturers of America (PhRMA) and the European Federation of Pharmaceutical Industries and Associations (EFPIA) in July 2013. More at title link. (Pfizer)
More Drugmakers to Join GSK Effort to Disclose Trial Data
In yet another bid to respond to calls for greater disclosure of clinical trial data, several other drugmakers next month are expected to join to a web site that was recently launched by GlaxoSmithKline in order to satisfy the clamor for wider access to detailed study data.
“Our aim is to progress to becoming an independent (portal) that accommodates multiple partners,” said Perry Nisen, who is a senior vp for science and innovation at Glaxo. “This is a start – a step in what we hope will be widespread movement to provide research data for clinical trials as well as other sources of clinical and health-related data.”
Among those that have agreed to participate is Roche, although its involvement had actually been disclosed several months ago (this is the site
). The other companies were not named, but he indicated a half dozen are expected to become involved. More at title link. (Pharmalot)
Informatics Research Team Awarded Grant for Clinical Effectiveness Research
(12/3, Indiana University Press Release) “The Patient-Centered Outcomes Research Institute...has awarded a $2 million grant to a research team from the IU School of Informatics and Computing at Indiana University-Purdue University Indianapolis to conduct comparative clinical effectiveness research. Headed by Brad Doebbeling, Department of BioHealth Informatics chair and a professor of informatics, medicine and biomedical engineering, the team is the first in Indiana to receive an institute grant designed to include patients in the discussion of how to improve and expedite medical care.” Full (CER Daily Newsfeed, National Pharmaceutical Council, 12/4/13)
Physician Payment Sunshine Act: CMS Offers Supplemental Data Submission Resources
December 4, 2013, the Centers for Medicare & Medicaid Services (CMS) released a number of new materials to assist reporting entities with the data submission process, available here
. These new tools include: Submission Data Mapping Document, Bulk Data Upload Instructions, XML Schema and XML Samples Files, and CSV Sample Files. More at title link. (Policy and Medicine)
ICH November 2013 Steering Committee Meeting Outcomes
The International Conference on Harmonisation (ICH) Steering Committee (SC) and its Expert Working Groups (EWGs) met in Osaka, Japan on 9 - 14 November 2013. The ICH SC furthered discussions on governance and increased engagement of regulators globally that had taken place in La Hulpe (Brussels) in June 2013. The SC agreed on organisational reform measures to foster international cooperation. In addition, the SC agreed to develop a 5-year plan for future ICH work and discussed potential new ICH topics to be further explored before the next meeting.
The ICH SC reviewed proposals for new topics provided by all parties and agreed on the development of concept papers for potential items such as providing further harmonisation on requirements for clinical trials. The SC will further discuss and prioritise potential new quality topics before the next face-to-face meeting. In order to improve the strategic oversight on ICH work, future topics will be integrated in a 5-year plan. More at link above. (ICH News Release)
European Commission Staff Working Document: Use of "-omics" technolgoies in the development of personalised medicine
The European Commission has just published the long-awaited “-Omics report.” This report provides an overview of the progress made in personalised medicine, and the opportunities and challenges it presents for healthcare systems. (European Society for Translational Medicine LinkedIn posting)
Early dialogue between regulators and health technology assessment bodies key to medicines development
EMA and multiple stakeholders to develop tools for industry
“A strong interaction between regulators and health technology assessment bodies (HTAs) is critical to enable innovation to reach patients, and ultimately for the benefit of public health,” said Guido Rasi, Executive Director of the European Medicines Agency (EMA) at the EMA-HTA workshop on parallel scientific advice which took place on Tuesday, 26 November. “This is the first workshop where we have tried to bridge these two worlds together to share views,” Rasi added.
There is a clear need to initiate early dialogue between medicines developers, the EMA and HTA bodies to discuss and agree on a development plan that generates data that both parties can use to determine a medicine's benefit-risk balance and value.
The workshop brought together over 280 representatives from the European Commission, European regulators, HTA bodies from 12 European Union countries, EUnetHTA, the pharmaceutical industry, payers, patients, healthcare professionals and academics, as well as representatives from the CHMP, the Pharmacovigilance Risk Assessment Committee, the Paediatric Committee, the Committee for Advanced Therapies, the Committee for Orphan Medicinal Products and the Agency’s Scientific Advice Working Party.
A report and video from the workshop will be published in early 2014. More at link above. (EMA)
Analysis: Biosimilars Could Save EU Billions
(12/3, Hirschler) reports in an analytical piece that biotech treatment copies could save most European Union nations billions of euros, but recent budget reductions leave them reluctant to try this new class of medicines. Although these biosimilars, which are less expensive versions of biotech medicines, could treat diseases like cancer and rheumatoid arthritis for a substantially lower cost, industry executives are disappointed the market has not developed as quickly as expected. Norway has proposed funding clinical studies in which patients switch from original medicines to biosimilars to convince physicians that these biosimilars are still of high-quality, but Norway is a relatively small market. (DIA Daily 12/3/13)
UK DoH Launches L10 Million Genomics Competition
The U.K. Department of Health launched a competition to award up to L10 million ($16.1 million) to companies developing high-tech products -- including new computer code or hardware and analysis algorithms -- to help identify and treat inherited diseases and cancer. The competition is focused on technologies that address the requirements of the U.K.'s 100K Genome Project, a project to sequence and analyze the genomes of up to 100,000 NHS patients over the next five years. The U.K. allocated L100 million ($161.4 million) for the project. Applications for the competition are due Feb. 5. (BioCentury)
The Asian Harmonization Working Party (AHWP) has posted proposed final documents for endorsement at the 18th AHWP Meeting in Malaysia. These include:
Summary Technical Documentation (STED) for Demonstrating Conformity to the Essential Principles of Safety and Performance of In Vitro Diagnostic Medical Devices
Summary Technical Documentation (STED) for Demonstrating Conformity to the Essential Principles of Safety and Performance of In Vitro Diagnostic Medical Devices
Comparison between the GHTF Summary Technical Documentation (STED) formats for Medical Devices and In Vitro Diagnostic Medical Devices and the Common Submission Dossier Template (CSDT) format
AHWP Regulatory Framework for IVD Medical Devices
(Regulatory Focus 12/3/13)
India declines to limit foreign investment by drug manufacturers
The Indian government has rejected a proposal to limit foreign drugmakers from purchasing more than a 49% share of domestic companies making "rare and critical" drugs, responding to concerns that the law would discourage foreign investment and harm the country's slowing economy. The proposal also would have required foreign investors to set aside a quarter of their investment to build new plants or research centers, now a lost opportunity, according to the Indian Pharmaceutical Alliance. The Wall Street Journal (tiered subscription model) (11/29) (DIA Global Smartbrief 12/3/13)
Committee on patented drug pricing in India shelved
India has dissolved a committee it formed to negotiate and reduce prices of patented drugs. Official sources said a new interministerial panel was formed by the Department of Pharmaceuticals to consider the issue. "The earlier committee and its report have been shelved because the department is of the view that market dynamics have changed over the years," an official said. Business Standard (India)
(12/2) (DIA Global Smartbrief 12/3/13)
SECTION 5 LEGAL, POLICY, AND COMPLIANCE NEWS
Obama Signs Drug Quality and Security Act To Oversee Compounding Pharmacies
Reuters (11/27) reported that last week, President Obama signed into law the Drug Quality and Security Act, which grants Federal officials greater regulatory authority over compounding pharmacies.
(11/27) reported that the law was passed after “a meningitis outbreak from contaminated steroid pain injections killed 64 people and sickened more than 750 across the country” in 2012. The outbreak was traced back to a compounding pharmacy in Massachusetts, which had been regulated under a hodgepodge of Federal and state laws that the Post wrote was a “legal gray area” until the law was signed. (DIA Daily 12/2/13)
Provider Demand to Drive Compounder Registration with FDA
FDA is moving fast to implement the drug compounding provisions of new Drug Quality and Security Act (DQSA), issuing new guidance to spur registration by outsourcing facilities just days after President Obama signed the new bill into law. But because FDA cannot compel compounders to opt for agency regulation, implementation will rely largely on market pressures to encourage health care providers to purchase compounded products only from registered facilities, explained FDA commissioner Margaret Hamburg at a press briefing Dec. 2, 2013.
The law stops short of setting specific criteria for which compounders should register and pay fees to FDA, and it’s unclear how many operators will choose to do so. FDA will post on its website the names and locations of registered outsourcers and the drugs they produce, which will promote the fact that they meet manufacturing quality standards and are regularly inspected by the agency. The hope is that hospitals and clinics will purchase more high-risk injectible medicines from such organizations. More at link above. (PharmaExec)
House Passes Another Patent Reform Act
The U.S. House of Representatives voted 325-91 to pass a bill that would make a number of changes to patent-related litigation in an effort to deter patent trolls. The Innovation Act (H.R. 3309) would amend language in the Leahy-Smith America Invents Act (AIA) -- which switched patent priority in the U.S. to first-to-file from first-to-invent -- covering the arguments a party can use to challenge a patent in court. Currently, a party cannot file suit based on earlier legal arguments used with the PTO or on any arguments that it "reasonably could have raised" with PTO but didn't. The bill also would allow judges to require the losing party in a patent suit to pay the legal fees for the other party, and, if the losing party cannot pay, to order other parties with a financial stake in the lawsuit to pay the fees.
Interest groups including the Biotechnology Industry Organization (BIO) have expressed opposition to the bill. The Innovation Act now goes to a Senate committee. More at link above. (BioCentury)
Lawmakers Urge OMB to Revisit FDA User Fee Sequestration
A group of 74 legislators from the U.S. House of Representatives sent a letter
to the U.S. Office of Management and Budget (OMB) asking the agency to "revisit" its decision that FDA user fees are subject to sequestration. The letter notes that the sequestration of the agency's user fees does not decrease the federal budget deficit and is "detrimental to patients, regulatory science, and public health." The letter also notes that including user fees in sequestration "compromises the good faith" of industry. Under sequestration, industry paid about $85 million in user fees in FY13 that will not be made available to FDA. (BioCentury)
Physician Payment Sunshine Act: Bipartisan Congressional Letter Calls for Exemption of Textbooks and Reprints
Twenty-three members of the United States House of Representatives sent a letter to the Centers for Medicare & Medicaid Services (CMS) to voice their disagreement with the Department of Health and Human Services (HHS) decision to include textbooks and scientifically peer-reviewed medical journals as "transfers of value" reportable under the Physician Payment Sunshine Act. As reported by the Coalition for Healthcare Communication, the letter demonstrates that there is widespread disagreement with the current HHS policy. The signatories state: "The importance of up-to-date, peer-reviewed scientific medical information as the foundation for good medical care is well documented." More at title link above. (Policy and Medicine)
SECTION 6 SOURCES REVIEWED FOR THIS NEWSLETTER
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); Daily Dose (Becker); DIA Daily; Drug Daily Bulletin; EMA website; Evaluate Pharma; Eye on FDA; FDA.gov; Federal Register Table of Contents; Fierce Medical Devices; Fierce Pharma; Fierce Vaccines; FDLI Smartbrief; Health Industry Washington Watch; Government Health IT; Health IT Security; Institute of Medicine News; MedCityNews; Medical Device Daily; Medical Device & Diagnostic Industry; NPC Bulletin; Pharmabiz; Pharmafile; Pharma IQ; Pharmalot; PharmaTimes; PhRMA website; PM Live; Policy and Medicine (newsletter); Regulatory Focus; RegLink News; US FDA Daily Digest Bulletin.