Monday, September 16, 2013

DIA Content Currents: EDITION PUBLISHED: September 6, 2013

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

EDITION PUBLISHED: September 6, 2013
August 26, 2013, FDA released a draft guidance for industry entitled, “ANDAs: Stability Testing of Drug Substances and Products; Questions and Answers,” which provides answers to questions received during the public comment period for this draft guidance document originally published on September 25, 2012. The draft guidance addresses issues related to drug master files, drug product manufacturing and packaging, stability studies, amongst others in order to provide clarification on the recommendations for stability testing data for ANDAs. [Draft Guidance] (

August 27, 2013, FDA released a guidance on “Guidance for Institutional Review Boards, Clinical Investigators, and Sponsors: Institutional Review Board Responsibilities for Reviewing the Qualifications of Investigators, Adequacy of Research Sites, and the Determination of Whether an Investigational New Drug/Investigational Device Exemption is Needed”. This guidance is is intended to assist institutional review boards (IRBs), clinical investigators, and sponsors involved in clinical investigations of FDA-regulated products in fulfilling responsibilities related to reviewing the qualifications of investigators and adequacy of research sites, and determining whether an investigational new drug (IND) application or investigational device exemption (IDE) is required, to protect the rights and welfare of human subjects involved in biomedical research. [Federal Register]

August 28, 2013, FDA released a draft guidance on “The Applicability of Good Laboratory Practice in Premarket Device Submissions: Questions and Answers.” This draft guidance answers commonly asked questions about the applicability of good laboratory practice (GLP) to nonclinical laboratory studies conducted in support of research and marketing applications for medical devices. [Federal Register]

August 30, 2013, FDA released a draft guidance on “Select Updates for Non-Clinical engineering Tests and Recommended Labeling for intravascular Stents and Associated Delivery Systems.” This guidance is to inform the coronary and peripheral stent industry about selected updates to FDA's thinking regarding certain non-clinical testing for these devices. While FDA is considering more substantial updates, it is issuing this update on select sections in order to notify the industry in a timely manner of its revised recommendations. [Federal Register]

September 4, 2013, FDA announced the availability of a draft guidance document for industry entitled, “Specification of the Unique Facility Identifier (UFI) System for Drug Establishment Registration.” The draft guidance is intended to address provisions in sections 701 and 702 of FDASIA that direct the agency’s Secretary to specify the UFI system for registration of domestic and foreign drug establishments. [Draft Guidance] (

September 5, 2013, FDA announced the availability of a “Draft standard operating procedure (SOP) for Level 1, Immediately in Effect (IIE) Guidance Documents on Premarket Data Issues.” In the draft, CDRH outlines its process to clarify and more quickly inform stakeholders when it has changed its expectations relating to, or otherwise has new scientific information that could affect, data submitted as part of an Investigational Device Exemption (IDE), Premarket Notification (510(k)), Premarket Approval (PMA), or Humanitarian Device Exemption (HDE) submissions. [SOP] [FR Notice] ( (See related information in “FDA Notes” below)


FDA Establishes Program Alignment Group to Adapt to Rapid Changes in the Regulatory Environment
September 6, 2013, Janet Woodcock sent an email to CDER staff announcing the establishment of the Program Alignment Group (PAG). “In order for FDA to best adapt to the ongoing rapid changes in the regulatory environment, driven by scientific innovation, globalization, the increasing complexity of regulated products, new legal authorities and additional user fee programs, the Commissioner has formed a Program Alignment Group (PAG). Comprised of senior Agency leaders, the PAG is charged with identifying and developing plans to modify FDA’s functions, processes, and possibly its structure in order to address these matters and best achieve mission-critical Agency objectives. This group of senior leaders will achieve this goal by working together to promote the strategic, operational, and resource management alignment needed for FDA to continue to fulfill its mission.” More detail at link above. (

Draft SOP on IIE Guidance Explained
In a new Federal Register posting focusing on CDRH, FDA acknowledges that when it releases a guidance document, that document may well reflect formal agency policy years after it is first put into informal practice at the review level. Due to agency resource restrictions, it may be a long process to get a guidance document, regulation or compliance policy guide published. In the meantime, industry may still be operating under an assumption that the status quo framework is still in effect, when in fact a more current one is the de facto standard.
To bridge the gap between unstated official policy and the issuance of guidance to industry, rapid tools for communication, broader communication, and standards for communicating to specific subsets of manufacturers are desirable. Thus, FDA will begin to issue what it calls Level 1, IIE guidance documents "when prior public participation is not feasible or appropriate."
Revising the 2011 draft SOP on “Notice to Industry” letters in response to industry concerns, FDA has released the new “Draft Standard Operating Procedure (SOP): Level 1, Immediately in Effect Guidance Documents on Premarket Data Issues.” The documents will still be released through regular Federal Register postings but will not be subject to a comment process prior to implementation. Comments will be accepted for 60 days after the release and will be considered during development of the related guidance. (See link to draft guidance in Section 1 above.)

FDA has announced the appointment of Melinda Plaisier as permanent director of the Office of Regulatory Affairs, effective September 3, 2013. She has been serving as Acting Associate Commissioner for ORA since October of last year, when the position was vacated by Dara Corrigan. Plaisier will report to the Deputy Commissioner for Global Regulatory Operations and Policy (now vacant since the departure of Deborah Autor). (

FDA Flexible with Accelerated Approval Evidence, Analysis Finds
Executive Summary: Attorneys Sasinowski and Varond write that in some cases weak clinical evidence on the surrogate endpoint was not a barrier, but strong findings on unmet need and rarity usually are necessary for accelerated approval.
An analysis of some drugs that used the accelerated approval pathway found FDA is flexible in determining the scientific evidence needed, but also determined a strong showing among some regulatory factors likely is required. The analysis seemed to point out that an accelerated approval decision does not seem to hinge on one specific factor, although a strong performance among some may offer a better chance at success.
Among the findings was that the agency increasingly has been flexible in the level of clinical evidence it has accepted showing an effect on the surrogate endpoint and clinical benefit. Full article at link above. (Pink Sheet)

What is Regulatory Science
The link above is for a short YouTube video featuring Margaret Hamburg and other FDA officials explaining what regulatory science is and why it’s important to the work of FDA and to the health and well-being of consumers. (

Considering Women's Needs in Developing Medical Devices: Here's "HoW"
Women differ from men in anatomy, physiology, risk factors and disease symptoms. They are also likely to use more medical devices over the course of their lives than do men.
This month we published a snapshot of how FDA is doing with such efforts. A congressionally-required report (Section 907 of FDASIA) looked at the inclusion and analysis of women and other demographic subgroups in clinical studies supporting the approval of medical devices and other FDA-regulated medical products.
One specific activity highlighted in the report was a workshop sponsored earlier this summer by CDRH to formally launch a new program designed to more closely look at medical device use and the health of women (HoW). The three main goals of HoW are to: Improve the availability, consistency and communication of information to patients and providers that is specific to women’s needs for the safe and effective use of medical devices. Address identified gaps and unmet needs through targeted resources. Foster the development of innovative strategies, technology and clinical study models.
With the issuance of the 907 report and an accompanying docket to receive comments from the public, we hope to gain an even more in-depth understanding about demographic subgroups. The input we receive will become the starting point for developing an Action Plan, to be released next year. (

FDA Takes Step to Encourage Pediatric Drug Studies

In a recent FDA Voice blog, Dr. Lynne Yao, Associate Director, Pediatric and Maternal Health Staff in CDER, wrote that under the reauthorized Pediatric Research Equity Act (PREA), FDA can waive, defer, or grant extensions for deferred pediatric studies in certain circumstances. In the past, deadlines for deferred studies have been missed. Now, in cases where sponsors fail to seek or obtain an extension or fail request approval for a required pediatric formulation, FDA can send a non-compliance letter and publish it on the web [Non-compliance Letters]. Last week, FDA published the first of the non-compliance letters and sponsors’ responses. (

Medical device manufacturers are claiming that FDA does not have the legal authority to release masked and de-identified clinical trial data to researchers. This follows a June, 2013 proposal by the agency’s Regulatory Science Initiative to improve the product development process. [Proposal] (The Gray Sheet via Becker)

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

Pediatric Ethics Subcommittee of the Pediatric Advisory Committee. September 9-10, 2013. Discuss ethical issues in pediatric product development, including medical counter measures, focusing on the concepts of minimal risk, disorder or condition, and exposure of pediatric subjects to risks under 21 CFR 50.54. [FR Notice]

Public Meeting: FDA Patient Network Annual Meeting - Demystifying FDA: An Exploration of Drug Development. September 10, 2013. This meeting will serve as a forum for FDA’s patient stakeholders and the general public, including health professionals, academia, and industry to learn about regulatory issues related to drug development, analyze where in the process patient input may be most practical and most valuable, and explore practicable approaches to incorporating meaningful patient input that will represent broad patient perspectives in medical product development and regulatory decision-making.
More information

NCTR global summit on regulatory science focusing on nanotechnology. September 11-12, held in Little Rock, AR.)

Circulatory System Devices Panel of the Medical Devices Advisory Committee. September 11-12, 2013. Discuss and make recommendations regarding the proposed classification of external cardiac compressor (ECC) devices, one of the remaining preamendments class III devices regulated under the 510(k) pathway. [FR Notice]

Public Workshop. The Patient Preference Initiative: Incorporating Patient Preference Information Into the Medical Device Regulatory Processes. September 18-19, 2013. Engage and solicit information from stakeholders on (1) approaches for capturing, collecting, and validating patient preference information; and (2) the incorporation of patient preference information into regulatory review process. [FR Notice]

Pediatric Advisory Committee. September 19-20, 2013. Discuss an array of pharmaceutical and medical device pediatric-focused safety reviews as mandated by the Best Pharmaceuticals for Children Act. [FR Notice]

Public Meeting. Narcolepsy Public Meeting on Patient Focused Drug Development. September 24, 2013. Obtain patients’ perspectives on the impact of narcolepsy on daily life as well as the available therapies for narcolepsy. [FR Notice] This is the 4th in the series of public meetings through which FDA plans to gather input to help assess benefit-risk paradigms that specific patient groups are willing to accept in return for treatment.

Advisory Committee for Pharmaceutical Science and Clinical Pharmacology. September 25, 2013. Discuss optimal strategies for the evaluation, interpretation, and communication of drug-drug interaction (DDI) information through the drug label and labeling materials. [FR Notice]

Public Workshop. The Food and Drug Administration/European Medicines Agency Orphan Product Designation and Grant Workshop. October 4, 2013. Discuss information about the FDA and European Medicines Agency (EMA) Orphan Drug Designation programs, the FDA Humanitarian Use Device (HUD) Designation program, and the FDA Orphan Products Grant program to participants representing pharmaceutical, biotechnology, and device companies, as well as academics. [FR Notice]

Synergizing Efforts in Standards Development for Cellular Therapies and Regenerative Medicine Products. Public Workshop. October 7, 2013. Bring together a broad range of stakeholders to discuss current and future standards development activities involving cellular therapies and regenerative medicine products. [Federal Register] Location will be White Oak Campus in Silver Springs, MD; the workshop will also be webcast due to limited availability of seating at the live facility.

Anti-Infective Drugs Advisory Committee. October 17, 2013. Discuss susceptibility interpretive criteria for systemic antibacterial drugs and for dosing recommendations in product labeling. [FR Notice]

Public Workshop. Gastroenterology Regulatory Endpoints and the Advancement of Therapeutics. October 21-22, 2013. Consider issues related to endpoints that can support drug development for the treatment of pediatric & adult inflammatory bowel disease. [FR Notice]

Cellular, Tissue and Gene Therapies Advisory Committee. October 22-23, 2013. On October 22, discuss oocyte modification in assisted reproduction for the prevention of transmission of mitochondrial disease or treatment of infertility. On October 23, hear updates on the guidance documents issued from the Office of Cellular, Tissue and Gene Therapies, Center for Biologics Evaluation and Research, and discuss considerations for the design of early-phase clinical trials of cellular and gene therapy products. [FR Notice]

Third Annual Food and Drug Administration Health Professional Organizations Conference. October 24, 2013. Agenda includes FDA updates, an overview of FDA’s Network of Experts (public/private partnerships), and an FDA Town Hall. [FR Notice]

OPDP Enforcement Actions Webinar – October 28, 2013 at 10:30 AM
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 October 28, 2013, from 10:30 AM to 11:00 AM (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:

September 4, 2013, the Department of Health and Human Services’ (HHS) Office of Disease Prevention and Health Promotion (ODPHP) solicited public comments on the draft National Action Plan for Adverse Drug Event Prevention. The draft Action Plan focuses on federal engagement in the surveillance, prevention, incentives, oversight, and research of entities manufacturing or distributing products associated with high levels of harm and adverse events. [Action Plan] [FR Notice] (

September 4, 2013, the Office of the National Coordinator’s (ONC) Health Information Technology (HIT) Committee released a set of recommendations for mobile health technology regulations. FDASIA 2012 calls for the HHS Secretary to “post a report—within 18 months (or by January 2014)—that contains a proposed strategy and recommendations on a risk-based regulatory framework pertaining to health IT, including mobile applications, that promotes innovation, protects patient safety, and avoids regulatory duplication”. The committee called on FDA to work within its current regulatory frameworks to allow for the innovation of mobile health technologies, rather than undertake a complete revamp of the current mobile health technology regulations. [Meeting Materials] [Recommendations Draft] (

The International Conference on Harmonization (ICH) has issued a statement and published a manual entitled, “ICH Procedures,” which is intended to create greater transparency on the activities, processes, and procedures conducted by the regulatory entity. The manual summarizes ICH’s “5-Step” regulatory process, and also includes a glossary of terms, definitions and explanations of ICH activities, member roles and responsibilities, and its document templates. [ICH Statement] [ICH Procedures] (Regulatory Focus)

The United Kingdom’s National Institute for Health and Care Excellence and India’s Department of Health Research, Ministry of Health and Family Welfare signed a Memorandum of Understanding effective June 14, 2013. The memorandum confirmed the nations’ goal to strategically and technically collaborate in the development and use of evidence to inform clinical policy and practice in the context of universal healthcare coverage. (BioSpectrum via Becker)

August 27, 2013, Germany’s Federal Ministry of Education and Research (BMBF) announced that it will provide up to EUR 27 million through 2018 to fund national and European research for rare disease diagnostics, therapeutic procedures, as well as disease mechanisms and genetic causes. The effort for this funding was led by Germany’s National Action League for People with Rare Diseases (NAMSE), and the group will now be responsible for implementation of these measures. (BioCentury)

Last week, the European Public Health Alliance (EPHA), a collaboration of nonprofits and non-government organizations, released a position paper entitled, “EPHA Position on Proposals for Regulations on Medical Devices & IVD.” The network maintains in the paper that, following member discussion and European Union (EU) developments, it supports more stringent and aligned procedures, more transparency in the authorization process, and stricter controls and requirements for manufacturers. [Position Paper] (FierceMedicalDevices via Becker)

Applications for new human medicines under evaluation by the Committee for Medicinal Products for Human Use

This document lists information on applications for centralised marketing authorisation for human medicines that the European Medicines Agency has received for evaluation. It includes the international non-proprietary names (INN) and therapeutic areas for all new innovative medicines under evaluation by the Committee for Medicinal Products for Human Use (CHMP). For generic and biosimilar medicines, it includes the INN (active moiety only, with no information on salt, ester or derivative) and therapeutic area. (EMA) Released September 5, 2013. Document at link above.

Russia Developing New Guidance for IVDs

Russia’s Federal Service on Healthcare Surveillance (Roszdravnadzor) has launched a public consultation to develop new guidelines for the evaluation of the quality, effectiveness and safety of in-vitro diagnostics products. According to the agency, the guidance will be prepared under the direction of the Ministry of Health’s Head Specialist for Clinical Laboratory Diagnostics, Anatolia Kochetova, with the assistance of expert organizations and leading experts in the field of in-vitro diagnostics. Representatives of the business community, academia, and professional associations have also offered to take part in the guidelines’ development. (RegLink News)

China-ASEAN Drug Safety Summit Opened

At the second session of the China-ASEAN Drug Safety Summit, which opened on September 3 in Nanning, Deputy Commissioner of the China FDA (CFDA) Yin Li noted that the trade volume in the China-ASEAN Free Trade Area increased by 13% to $ 5.114 billion in 2012 from $4.5 billion in 2011. Moreover, Li said that China’s pharmaceutical industry had undergone rapid growth rate of more than 20% annually. The session was co-sponsored by the CFDA and the Guangxi Zhuang Autonomous Region People's Government, with attendance by the Lao People's Democratic Republic Deputy Minister of Health, the Myanmar Deputy Minister of Health, the Socialist Republic of Vietnam Deputy Health Minister and other ASEAN countries as well as the Hong Kong and Macao health and drug regulatory agency representatives. (RegLink News)

CFDA Expands Bilateral Agreements
China’s FDA recently expanded bilateral cooperation agreements with two European countries and the World Health Organization (WHO) within a one-week time frame. On August 22, Commissioner Zhang Yong met with WHO Director-General Margaret Chan on the ongoing bilateral cooperation on food safety and drug regulatory issues. On August 20, Commissioner Yong met with the Czech Minister of Health and reached agreement on further cooperation and exchanges on drug and medical device regulatory matters. On August 16, Deputy Commissioner Yin Li met with Swiss Federal Councilor and Minister of the Interior, Mr. Seth Alan Bell, on a bilateral regulatory cooperation memorandum. (RegLink News)

Bill creating Indian Central Drugs Authority introduced
Legislation has been introduced in India that would establish a new Central Drugs Authority (CDA) with overarching power to regulated drugs, devices and cosmetics. The Drugs and Cosmetics (Amendment) Bill, 2013, introduced today in the Rajya Sabha (Upper House of Indian Parliament) by Health and Family Welfare Minister Ghulam Nabi Azad would replace the Drugs and Cosmetics Act, 1940.

The CDA would be composed of 19 members, headed by the Secretary of Health and Family Welfare. It would establish regulations and norms for effective functioning of central and state drug licensing authorities and periodically assess their functioning. The new agency would also have the authority to review, suspend or cancel any permission or license for drugs and cosmetics manufacturing.

The bill establishes centralized licensing for 17 categories of critical drugs, and includes separate chapters with regulatory frameworks governing medical devices, product exports and clinical trials, including provision for compensation for deaths or injury of a trial subjects. Penalties for violations are also set forth in the bill. (RegLink News)

August 30, 2013, India’s Central Drugs Standard Control Organization (CDSCO) issued a notice explaining the requirement that payment and legal agreements between sponsors and trial investigators be disclosed in clinical trial applications. India’s Good Clinical Practices (GCP) regulations now require contracts between trial investigators and sponsors be completed prior to the start of the study, with financial support information and the nature of their relationship clearly defined. [CDSCO Notice]

Pharma Loses Lawsuit Over Take-Back Law

In a rebuke to the pharmaceutical industry, a federal judge has dismissed a lawsuit that charged a California county acted inappropriately last year by passing an ordinance that requires drugmakers and biotechs to cover the cost of a prescription drug ‘take back,’ or disposal program.
County officials pointed out that drugmakers make money from the sale of medicines and, therefore, should also have an obligation to help with appropriate disposal. But in its lawsuit, the PhRMA trade group maintained that safe disposal of unwanted medicines is a shared responsibility and the ordinance unfairly requires drugmakers to develop, manage and fund disposal operations. Failure to comply carries up to a $1,000 per day fine. Full article at link above. (Pharmalot)

As FDA Considers First Wave of Biosimilar Drugs, Are States Limiting Their Eventual Impact?

This is an interesting article about the California bill to prevent substitution of a biosimilar for a reference biologic licensed drug unless FDA has specifically found the two products to be interchangeable. Both FDA and GPhA have criticized the bill, indicating that it would create unnecessary barriers between patients and new, lower-cost versions of biologic therapies.

The bill (SB 598) has been approved in both the Assembly and the Senate and was sent to the Governor’s desk on September 4 for signature. Full article at link above. (Regulatory Focus)

Biosimilars State Legislation Scorecard

With the news last week that the Assembly in the bellwether state of California passed a bill – S.B. 598 – by a 58-4 vote concerning the substitution of biosimilar and interchangeable biosimilar products for their brand-name reference product counterparts, and with bills currently passed, failed or pending in other states (and the likelihood that legislation will continue to be pushed in several states for some time to come), we thought it was high time to put together a new scorecard – the Biosimilars State Legislation Scorecard – to keep tabs on such legislation.

See full article at link above. (FDA Law Blog)

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; Eye on FDA; Federal Register Table of Contents; Fierce Medical Devices; Fierce Pharma; Fierce Vaccines; FDLI Smartbrief; Health Industry Washington Watch; Institute of Medicine Report; Medical Device Daily; Medical Device & Diagnostic Industry; NPC Bulletin; Pharmabiz; Pharmalot; PharmaTimes; PhRMA website; PM Live; Policy and Medicine (newsletter); Regulatory Focus; RegLink News; US FDA Daily Digest Bulletin.
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