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: November 15, 2013
SECTION 1 FDA GUIDANCES & MAPPS
On November 13, FDA proposed to amend its regulations on “Supplemental Applications Proposing Labeling Changes for Approved Drugs and Biological Products.” The proposed rule would revise and clarify procedures for application holders of an approved drug or biological product to change the product labeling to reflect certain types of newly acquired information in advance of FDA’s review of the change. The proposed rule would create parity among application holders with respect to such labeling changes by permitting holders of abbreviated new drug applications (ANDAs) to distribute revised product labeling that differs in certain respects, on a temporary basis, from the labeling of its reference listed drug (RLD) upon submission to FDA of a ‘‘changes being effected’’ (CBE–0) supplement. [Federal Register]
See Section 4 “Other Regulatory Authorities and Organizations” for a statement by the Generic Pharmaceutical Association (GPhA) regarding the proposed rule.
On November 14, FDA distributed a draft guidance entitled “Medical Device Development Tools. Draft Guidance for Industry, Tool Developers, and FDA Staff.”
The document provides draft guidance on a voluntary process for qualification of medical device development tools (MDDT) for use in device development and evaluation programs in the Center for Devices and Radiological Health (CDRH). CDRH believes that application of this policy will facilitate the development and timely evaluation of innovative medical devices, by providing a more efficient and predictable means for collecting the necessary information to make regulatory assessments. The purpose of this guidance is to describe the framework and process for voluntary CDRH qualification of MDDT. [Federal Register
SECTION 2 FDA NOTES & RELATED NEWS
Ensuring Safe Food and Medical Products: A Partnership with the Mekong Region
In the November 12 “FDA Voice,” Dr. Margaret Hamburg writes about her participation in a forum hosted by the Center for Strategic and International Studies entitled “U.S. Health Partnerships in the Mekong Region.” The day’s discussions highlighted the growing strategic importance of the region to the United States and the long-standing and ongoing partnerships between U.S. agencies and regional partners in health and development, including the central focus of the FDA, to ensure the safety of food and medical products in the United States.
“The U.S. is increasingly and inextricably linked to the Mekong Region through global supply chains,” she writes. “That’s why we work closely with our regional counterparts in these countries through such organizations as the Association of Southeast Asian Nations (ASEAN) and the Asia Pacific Economic Cooperation (APEC), sharing with them our own regulatory requirements, our knowledge of good manufacturing practices and our laboratory and inspection techniques. Through such information sharing we believe we can prevent tainted or otherwise unsafe foods from reaching our borders.” More at title link above. (FDA.gov)
FDA Was Hacked During Government Shutdown
US Food and Drug Administration (FDA) officials quietly announced to members of industry late Friday, November 8, that their biologics online submission system had been "compromised" by an unauthorized user—hacked, in common parlance—leading to the access of users' account information, email addresses and passwords.
FDA said the intrusion was detected on 15 October 2013, a date when much of the agency's staff were not at work due to a shutdown of all non-essential government operations. The affected databases included the Biologic Product Deviation Reporting System (eBPDR), the Electronic Blood Establishment Registration System eBER) and the Human Cell and Tissue Establishment Registration System (eHCTERS). While these databases are not as highly trafficked and do not contain as much commercially confidential information as some of FDA's other databases, FDA said it was nevertheless aware that user names, user information, phone numbers, email addresses and passwords had all been accessed. In all, 14,000 accounts—both past and current—were accessed, FDA said.
"The agency temporarily disabled the system, immediately implemented corrective security measures, and administered password resets, as was communicated by email on October 18 to active account holders," FDA wrote in an email to industry. "The agency has confirmed that no system data has been altered, and we are continuing our analysis to confirm that there have been no unauthorized logins to the system." (Regulatory Focus)
FDA Recognizes IEEE 11073 Standards for Medical Device Communication
IEEE, the world’s largest professional organization dedicated to advancing technology for humanity, today announced that it received a key recommendation from the U.S. Food and Drug Administration (FDA). The FDA recently recognized multiple standards that collectively help support medical-device interoperability and cyber security. Among the 25 standards listed, 12 originate from within the IEEE 11073™ family of standards for medical-device communication.
The FDA recognized three categories of standards: risk-management standards for a connected and networked environment; interoperability standards that establish nomenclature, frameworks and medical-device-specific communication (including system and software lifecycle processes); and cyber-security standards from the industrial-control area most relevant to medical devices.
IEEE 11073 standards are designed to help healthcare product vendors and integrators create interoperable devices and systems for disease management, health and fitness and independent living that can help save lives and improve quality of life for people worldwide. More at link above. (Business Wire)
Lawmakers Affirm FDA Authority Over Biosimilar Naming
A bipartisan group of lawmakers said FDA has the authority to determine the naming policy for biosimilars, according to a letter sent to FDA Commissioner Margaret Hamburg. The lawmakers said the agency should "establish a science-based policy" for determining non-proprietary names; the agency has not yet announced a position on naming. Regulators worldwide are debating whether biologics and interchangeable biosimilars should have the same international non-proprietary names (INNs). (BioCentury)
GDUFA: Industry Says New Information Flow Lacking
Executive Summary: The “complete response” letter system and problems gaining information under new communication policy is making generic product launch planning difficult; FDA and GPhA are considering solutions.
As FDA has implemented its policy of issuing “complete response” letters for ANDA applications, sponsors are complaining the lack of information flow is complicating product launch planning and status updates are difficult to obtain. Requiring “complete response” letters for ANDAs was among the improvements mandated in the generic drug user fee program, which launched in 2012. FDA said it would no longer send deficiencies as each discipline completed its review, instead embracing the new drug application practice of bundling them together and sending them in one letter.
Ideally, “complete response” letters will allow FDA to streamline and gain more control over the review process and communications with sponsors. Pre-GDUFA, the separate discipline-based responses and sponsor communications were fragmented. More at link above. (Pink Sheet November 11, 2013. Paid subscription required.)
FDA Wants Label and Packaging Changes for Some Topical Antiseptics
FDA wants label and packaging changes for certain topical antiseptics products to reduce the risk of infection. The agency said on Wednesday that it is evaluating “infrequent but continuing reports of infections resulting from antiseptic products labeled for preoperative or preinjection skin preparation.” The risk of infection apparently arises most often from user contamination; however, contamination can also occur during the manufacturing process, as topical antiseptics are not required to be manufactured as sterile.
FDA has recommended that these products, available as single or multiple use, be sold only as single use preparations. FDA has also requested that manufacturers voluntarily revise the product labels for topical antiseptics to indicate whether the drug is manufactured as a sterile or nonsterile product. (MedPage Today)
FDA Draft National Adverse Event Plan
Adverse drug events (ADEs) have been defined as an injury resulting from medical intervention related to a drug. ADEs can occur in any health care setting, including inpatient (for example, acute care hospitals), outpatient, and long-term care settings (for example, nursing homes).
Given the U.S. population's large and ever-increasing magnitude of medication exposure, the potential for harms from ADEs constitutes a critical patient safety and public health challenge.
To reduce and prevent the dangers associated with adverse drug events, the U.S. Department of Health & Human Services (HHS), in partnership with the U.S. Department of Defense, U.S. Department of Veterans Affairs, and the U.S. Bureau of Prisons has developed the National Action Plan for Adverse Drug Event (ADE) Prevention.
The draft Action Plan identifies the federal government's highest priority strategies and opportunities for advancement that will have the greatest impact on reducing ADEs. The implementation of these strategies is expected to result in safer and higher quality health care services, reduced health care costs, informed and engaged consumers, and, ultimately, improved health outcomes. More at title link. (Policy and Medicine)
Expediting Drug Development: FDA's New Breakthrough Therapy Designation
The November 14, 2013, issue of the New England Journal of Medicine published this article discussing the intent, provisions, and expected benefits of the new “breakthrough therapy” designation enabled by the passage of FDASIA in 2012. Authors Janet Woodcock, Rachel Sherman, Stephanie Shapley, and Melissa Robb write, “The breakthrough-therapy designation program is of great interest to patients and patient advocates. Because designations are given to drugs in development, it will be some time before the program's effect on access to important therapies can be assessed. This program may represent the initiation of a new paradigm for investigational drugs undergoing development in a setting of extensive mechanistic understanding of disease pathogenesis. As the pace of scientific discovery continues to increase, drug-development pathways will need to evolve in parallel.” Full article at link above. (NEJM)
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. Meta-Randomized Controlled Clinical Trials for the Evaluation of Risk to Support Regulatory Decisions. November 25, 2013. This meeting will obtain input on scientific approaches for the conduct and assessment of meta-analyses of randomized controlled clinical trials (RCTs) to evaluate safety risks associated with the use of human drugs or biological products within the framework of regulatory decision making.
The purpose of the workshop is to initiate constructive discussion and information sharing among regulators, researchers, health-care providers, representatives from the pharmaceutical industry and health care organizations, and others from the general public, about the use of meta-analyses of randomized trials as a tool for safety assessment in the regulation of pharmaceutical products. Meeting Information
Public Hearing. Over-the-Counter Ophthalmic Drug Products – Emergency Use Eyewash Products. December 4, 2013.
Obtain information on the formulation, manufacturing, and labeling of currently marketed over-the-counter (OTC) emergency first aid eyewash drug products, including the components of these products, and the conditions under which such products are safe and effective for their intended use. [FR Notice
Public Meeting. Medical Gas Regulation Review. December 6, 2013.
FDA is announcing a public meeting on whether any changes to Federal drug regulations are necessary for medical gases. The topic to be discussed is whether any changes to the Federal drug regulations are necessary for medical gases as part of the implementation of the Food and Drug Administration Safety and Innovation Act (FDASIA). [Federal Register
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 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 Registe
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
Physician Payment Sunshine Act: CMS to Host Webinars for Company Registration and Data Submission
The Centers for Medicare & Medicaid Services (CMS), in response to stakeholder requests, on November 19, from 1:00 p.m. to 3:00 p.m. EST, CMS will present the first two sessions in a series of technology-focused webinars designed to introduce features of the Open Payments system currently in development. The webinars focus on two major Open Payments components: registration and data submission. November 19 Webinar Registration
On December 3, 2013, CMS will host a follow-up Q&A session after participants have had the opportunity to fully review the data submission resources. More information on that webinar will be available following the November 19 webinars. (Policy and Medicine)
GPhA Statement (Excerpt) Regarding the FDA Proposed Update to Generic Labeling Regulations
“Patient safety is the foremost concern for manufacturers of generic medicines, which is why both brand and generic companies comply with federal law and strict FDA labeling rules and regulations. Generic drug companies proactively participate with the FDA in ensuring the timeliness, accuracy, and completeness of drug safety labeling in accordance with current regulatory requirements, to ensure doctors and all healthcare professionals and patients have access to the most up-to-date information.
GPhA has received the FDAs proposed rule and is in the process of careful review with our member companies. With this proposed rule comes the public response period, during which we will fully evaluate the implications of any rule change. We will evaluate this proposed rule both for any elements that could impact patient safety, and for our member company business practices that currently ensure access to affordable, life-saving medicines for millions of Americans. More at link above. (GphA, November 8, 2013)
GAO is asked to assess federal efforts on regenerative medicine
The Government Accountability Office has been urged by a group of Democratic and Republican senators to study the government's efforts involving regenerative medicine. The senators want to know, among other things, how government research funds are being spent and what federal programs and policies have been crafted to foster the development of regenerative medicine products. Clinica (subscription required)
(11/13) (via FDLI SmartBrief 11/14/13)
New US Guidelines Recommend Statins for More Groups
Statins should be prescribed to adults 40-75 years of age who have a 10-year risk of a heart attack or stroke that is 7.5% or higher, according to a series of guidelines released on Tuesday by the American Heart Association and American College of Cardiology. Previous 2004 statin guidelines recommended the drugs for people with a 10-year risk of coronary heart disease that was 20% or higher. Those guidelines did not account for risk of stroke.
The new guidelines also recommend statins for three other groups of patients: those with cardiovascular disease, an LDL-C level of 190 mg/dL or higher or with Type II diabetes between 40-75 years of age. The new guidelines -- which also provide updated risk-assessment formulas to better calculate a patient's risk of heart attack or stroke, including formulas that are race- and sex-specific -- also cover lifestyle management to reduce cardiovascular risk and the management of obesity in adults. (BioCentury)
Groups Seek Safety Reg Scope Limitation On Health IT Systems
(11/12, Conn, Subscription Publication) reports that two associated health IT groups are seeking “to limit the scope of proposal to impose safety regulations on health IT systems.” In a letter to Health and Human Services Secretary Kathleen Sebelius, the Healthcare Information and Management Systems Society (HIMSS) “said ‘most electronic health records and clinical decision support systems, according to current capabilities, neither fit the definition of, nor would be appropriately regulated as medical devices.’” Separately, in its letter to Secretary Sebelius, the Electronic Health Record Association (EHRA), which is affiliated with HIMSS, specifically criticized the output of a workgroup dealing with the matter under the Food and Drug Administration Safety Innovation Act, saying that the group’s “‘risk grid...does not provide ‘a valid and reliable basis’ to assign risk to health IT applications and ‘lacks any formal model and associated weights’ to make those determinations.” Link to EHRA letter: Letter
(DIA Daily 11/12/13)
HHS Releases Exchange Enrollment Figures
According to an HHS report
, 106,185 people selected a qualified health plan in the healthcare exchanges in the first month of open enrollment, including 79,391 (75%) in state-run exchanges and 26,794 (25%) in federally-run exchanges. The figure -- which includes enrollees for Oct. 1 through Nov. 2 -- includes those who have not yet paid the first month's premium. HHS said an additional 975,407 people have completed the application process but have not selected a plan. Thirteen states and the District of Columbia are running their own exchanges, while the federal government is facilitating or running the exchanges in the remaining states. HHS also said 396,261 people have been ruled eligible for Medicaid or the Children's Health Insurance Program (CHIP). More at link above. (BioCentury)
First Director Named for National Human Genome Research Institute Divison of Genomics and Society
Lawrence C. Brody, Ph.D., a pioneering genetics and genomics researcher, has been selected to be the first director of the newly established Division of Genomics and Society at the National Human Genome Research Institute (NHGRI). NHGRI is one of the 27 institutes and centers that make up the National Institutes of Health.
Dr. Brody is currently chief of the Genome Technology Branch within NHGRI’s intramural research program, and the chief scientific officer of the trans-NIH Center for Inherited Disease Research. His expertise and interests are wide-ranging, from human genetics and genomics to the public understanding of science. As a bench scientist, he played an instrumental role in early and important discoveries about the BRCA1 gene, which is responsible for a hereditary form of breast cancer.
The Division of Genomics and Society was established in 2012 as part of an institute-wide reorganization. It is one of four divisions that make up the institute’s extramural research program. This new division is now responsible for an expanded program related to the many societal issues relevant to genomics research and genomic advances, incorporating and extending the activities of NHGRI’s Ethical, Legal and Social Implications (ELSI) research program. The latter was established in 1990 as part of the Human Genome Project and aims to pursue multidisciplinary research and training designed to explore the impact of genomics on society. (NIH)
Final Coverage with Evidence Development Guidance Delayed by CMS Staff Cuts
The CMS coverage and analysis group has had to divert its attention from finishing a CED guidance to finalizing proposed changes to the process for awarding Medicare coverage for IDE trials, primarily due to staff cuts, says Louis Jacques, who heads the group. (The Gray Sheet – Paid Subscription Required)
PCORI Building Non-Traditional Research Capacity With "Pipeline To Proposal" Awards
(11/14, Gregory Twachtman, The Pink Sheet Daily
) reports “...PCORI Director of Patient Engagement Susan Sheridan added that the institute is ‘really focusing on how patients and other stakeholders really partner in the research that we fund’...‘We've developed this to really create partnerships, develop community, develop infrastructure and to facilitate those interested in getting what we called "PCOR-ready" or "research ready" to eventually submit a proposal to PCORI or other funding agencies that fund PCOR or comparative effectiveness research’ Sheridan continued.” Paid Subscription Required
(via National Pharmaceutical Council)
New Center to Deliver Research-Based Solutions to Rising Health Care Costs
(11/13, Indiana University School of Medicine Press Release) “...The newly designated Center for Innovation and Implementation Science, or CIIS...will oversee four specialized research and discovery units managed by IU School of Medicine researchers at Indiana University Health, Riley Hospital for Children at IU Health, Eskenazi Health and the Richard L. Roudebush VA Medical Center in Indianapolis...The center will also provide a platform to attract federal funds to Indiana through special government programs focused on health care innovation, including the Center for Medicare and Medicaid Innovation and the Patient-Centered Outcomes Research Initiatives...” “The center is also a direct response to an influential IOM report that called for US health care systems to transform into highly adaptive, learning health care systems.” Full
(via National Pharmaceutical Council)
FTC to Examine State Legislation, Naming for Biosimilars
The U.S. Federal Trade Commission is examining how state regulations and international naming conventions could affect the development of and competition for biosimilars. A Dec. 10 workshop will cover the topics and will also focus on how state regulations could be structured to facilitate competition. The FTC is accepting comments on the topics until March 1, 2014.
Biosimilars developers, manufacturers of originator biologics, payers and consumer groups are battling in state capitals, at FDA and at the World Health Organization over rules that will shape perceptions and prescribing practices for biosimilars. (BioCentury)
Alleged US Proposal Seen Putting Limits On Generic Drugs In Other Countries
(11/14, Wingfield, Decker) reports people in other countries could find it difficult to get hold of generic drugs due to a US proposal in a Pacific trade agreement, citing patent specialists who have seen papers leaked by the WikiLeaks group. Bloomberg says the proposal would boost “patent protections for brand-name medicines in some participating countries” and put curbs on “access to low-cost generic drugs.” WikiLeaks has said the document is a draft of “the intellectual-property chapter of the Trans-Pacific Partnership being negotiated by 12 nations, including the U.S. and Japan.” Consumer-activist group Public Citizen’s Global Access to Medicines program criticized the US move as limiting Internet freedom and “access to life-saving medicines throughout the Asia-Pacific region.” (DIA Daily 11/15/13)
EMA Updates on Development of Its Policy on Publication and Access to Clinical Trial Data
In-depth analysis of more than 1000 stakeholders’ comments received on draft policy currently underway
The European Medicines Agency is currently reviewing and analysing more than 1,000 comments received during the public consultation on its draft policy on publication and access to clinical-trial data, which ran from June to end of September 2013.
The public consultation on the policy has generated input from an unprecedented range of stakeholders. Patients, healthcare professionals, pharmaceutical industry representatives, researchers, transparency campaigners, academic and public institutions, health technology assessment bodies and a range of others sent their comments to the Agency.
In order to conduct the appropriate in-depth analysis required, the Agency will spend additional time in this reviewing phase which may therefore delay the finalisation of the policy initially planned for the end of 2013. An update on timelines will be provided at the latest following the EMA Management Board meeting on 11-12 December 2013. More at link above. (EMA)
EMA Posts Presentations from "Workshop on Biosimilars"
The workshop, held October 31, 2013, was intended to discuss the draft guideline on similar biological medical products, the draft guideline on similar biological medical products containing biotechnology-derived proteins as active substance: non-clinical and clinical issues, and the draft guideline on similar biological medical products containing biotechnology-derived proteins as active substance: quality issues. Presentations have been posted at the link above. (EMA)
EMA Posts Presentations from Workshop "Best Use of Medicines Legislation to Bring New Antibiotics to Patients & Combat the Resistance Problem"
This workshop, held November 8, 2013, was organised with the European Commission and discussed regulatory options for approval of new antibacterials and actions that could be taken to increase the appropriate use of the current armamentarium. Research activities for antibacterials and the impact on regulatory aspects were discussed. Participation was by invitation only, but the presentations have been posted at the link above. (EMA)
World's Biggest Cancer Database Launched
A major new database, developed by researchers at The Institute of Cancer Research in London and funded by Cancer Research UK, will use artificial intelligence to help scientists discover new cancer treatments.
CanSAR is the biggest disease database of its kind in the world, and is capable of holding more information than the Hubble space telescope would gather in a million years of use. It is more than double the size of a previous version, and will cope with a huge expansion of cancer data following advances in technologies such as DNA sequencing, says Cancer Research UK.
The CanSAR system will make 1.7 billion experimental results available to researchers, pooling knowledge and data in one freely-accessible resource. Information stored in the database will be analysed by technology similar to that used to predict weather and will allow researchers to predict potential targets for anticancer drugs in the future, says the charity. Full article at link above. (PharmaTimes)
UK Alzheimer's Charity to Fund Drug Discovery Institute
Alzheimer’s Research U.K., the country’s leading dementia research charity, will fund a new drug discovery institute to address a gap in treatments it says the pharmaceutical industry has failed to fill.
The charity will ask universities in the U.K. to apply to host the institute, which will aim to develop new treatments for Alzheimer’s, it said in a statement today. Alzheimer’s Research U.K. will fund the institute with a 2 million-pound ($3.2 million) grant per year for at least five years, spokeswoman Kirsty Marais said by phone.
This innovative idea will bring together the best of both academic and industry expertise to drug discovery, an approach which has already shown benefits in tackling cancer,” said Alison Goate, a professor of genetics in psychiatry at Washington University in St. Louis. “Now is the ideal time for a similar approach to be made in neurogenerative diseases.” Full article at title link. (Pharmalot)
MHRA Issues New AED Guidance Warning Against Switching
The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has issued new guidance that establishes three categories of anti-epileptic drugs (AEDs) based on the potential effects of switching from one drug to another, which may result in a loss of seizure control or the occurrence of side effects, or both. The guidance follows a review by the UK Commission on Human Medicines (CHM) which evaluated the evidence on patients switching between different AED products. Due to risks that can be associated with switching between a branded originator and a generic product, and between different generic products, CHM advised that AEDs could be classified into three categories:
- Category 1, in which doctors are advised to ensure that their patient is maintained on a specific manufacturer’s product. The AEDs in this category are: phenytoin, carbamazepine, phenobarbital, primidone.
- Category 2, in which the need for continued use of a particular product should be based on clinical judgment and consultation with patient and/or caregiver taking into account factors such as seizure frequency and treatment history. The AEDs in this category are: valproate, lamotrigine, perampanel, retigabine, rufinamide, clobazam, clonazepam, oxcarbazepine, eslicarbazepine, zonisamide, topiramate.
- Category 3, in which it is not usually necessary to ensure that patients are maintained on a specific product unless there are particular concerns such as patient anxiety, and risk of confusion or dosing errors. The AEDs in this category are: levetiracetam, lacosamide, tiagabine, gabapentin, pregabalin, ethosuximide, vigabatrin. (RegLink News 11/13/13)
Big Pharma Takes on UK Drug Watchdog
In a joint letter to The Telegraph yesterday, nine of the world’s biggest pharm companies accused the UK’s drug pricing body, the National Institute for Health and Care Excellence (NICE), of blocking too many new and innovative medicines.
The open letter — signed by representatives of Novartis, Pfizer, Sanofi, Eisai, Novo Nordisk, Amgen, Celgene, Merck, and UCB — conceded that while there are cost pressures on the National Health Service (NHS), the notion that medicines are expensive is a “prevailing myth”. Drug spend, the letter explained, was less than 10% of total NHS expenditure in 2011, and the UK pays less for its medicines than most places in Europe. In light of this, NICE’s approval of “fewer than one-in-three medicines” since 2005 — a rejection rate that is increasing — is damaging the “health of the British public and wealth of the British economy”.
“Medicines should not just be seen as a cost,” the letter went on. “Action to manage costs should be matched with action to drive better health outcomes.”
The letter concluded with a call for the UK government to set a new mandate for NICE to ensure that “NHS patients are among the first in the world to benefit from new treatments, and innovation is recognized, valued and rewarded.” More at link above. (PharmExec)
Australia and NZ Launch Next Phase of Regulatory Harmonization
Australia’s Therapeutic Goods Administration (TGA) and New Zealand’s Medicines and Medical Devices Safety Authority (Medsafe) announced
on November 13 the next stage of their joint efforts toward regulatory harmonization and the launch of a joint regulatory agency, ANZTPA. This new phase of joint work will cover the next two and a half years, and targets 14 activities across the following 6 regulatory areas: medicines (prescription and non-prescription), medicines ingredients, safety, medical devices, and biological and blood products. The Medsafe/TGA ANZTPA Implementation Steering Committee will oversee the multiple harmonization activities to link with the ongoing Australian Blueprint reforms program and the needed timing for development of ANZTPA Rules and Orders. (RegLink News 11/13/13)
TGA Adopts E2B Reporting System
Australia’s Therapeutic Goods Administration (TGA) has adopted a streamlined way for sponsors to submit adverse event reports regarding medicines and vaccines, which aligns the TGA's system with the European Union, the US, Japan and Canada. Sponsors are now able to submit adverse event reports via email (firstname.lastname@example.org
) using the international E2B standard. Data supplied in this format can be entered directly into the TGA's adverse event database with minimal user interaction. The change offers sponsors the potential to avoid double-handling and the need to copy information into CIOMS
forms that have been widely used to date. (RegLink News 11/13/13)
CFDA Announces Administrative Review Process
China’s FDA (CFDA) issued a notice
on November 6 establishing an administrative review process to take effect as of January 1, 2014. A requested review must allege that the specific administrative acts infringe upon the legitimate rights and interests of citizens, legal persons or other entities. The burden will be on the applicant to establish the factual and legal basis for the reconsideration of a specific administrative act. The administrative review process is to be lawful, fair, open, and timely and the cost of the process will be borne by the CFDA. (RegLink News 11/13/13)
AHWP Releases Six Documents on Regulation of Devices and Diagnostics for Consultation
On November 13, the Asian Harmonization Working Party (AHWP) released six proposed documents for consultation (see all at link above). The are: Adverse Event Reporting Guidance for the Medical Device Manufacturer or Its Authorized Representative; Quality Management System – Medical Devices – Nonconformity Grading System for Regulatory Purposes and Information Exchange; AWHP Regulatory Framework for IVD Medical Devices; Essential Principles of Safety and Performance of IVD Medical Devices; Summary Technical Documentation (STED) for Demonstrating Conformity to the Essential Principles of Safety and Performance of IVD Medical Devices; and Comparison between the GHTF Summary Technical Docementation formats for Medical Devices and IVD Medical Devices and the Common Submission Dossier Template format.
Asian Harmonization Working Party (AHWP) is established as a non-profit organization. Its goals are to study and recommend ways to harmonize medical device regulations in the Asian and other regions and to work in coordination with the Global Harmonization Task Force, APEC and other related international organizations aiming at establishing harmonized requirements, procedures and standards. The Working Party is a group of experts from the medical device regulatory authorities and the medical device industry. Membership is open to those representatives from the Asian and other regions that support the above stated goals.
Twenty-three Economies make up the members and include among others: People’s Republic of China, Chinese Taipei, Hong Kong SAR, Singapore, Republic of Korea, Myanmar, Indonesia, Philippines, India, Pakistan, South Africa, Kuwait, Saudi Arabia, Jordan, and Chile. (AHWP)
Indian Health Ministry Plans to Mandate 6-year Post-Marketing Surveillance Requirement
The Indian Health Ministry is planning to make the post-marketing surveillance of drugs mandatory for six years, instead of the current practice of four years, under the Schedule Y provisions of the Drugs and Cosmetic (D&C) Rules. This would apply to all drugs permitted to be marketed in India. The PMS should include spontaneous/voluntary reporting including reporting from scientific literature, publications or meetings, voluntary or required reporting from observational studies and randomized clinical trials, drug-induced injury, detection of events not seen in clinical trials such as new, previously unknown adverse events or new drug interactions, and any observed increase either in quantity or severity of a known adverse event, according to the panel. More at link above. (PharmaBiz)
SECTION 5 LEGAL, POLICY, AND COMPLIANCE NEWS
Senate Votes To Advance Compounding Pharmacy Bill
The AP (11/13) reports that in a 97 to 1 vote, the Senate on Tuesday voted to advance a bill to impose tighter oversight on “pharmacies that custom-mix prescription drugs.” The AP notes that the vote indicates the measure’s “overwhelming support in the Senate.” Final Senate passage sending the bill to the President “could come as early as Wednesday.”
The Hill (11/13, Cox) “Floor Action” blog reports the Senate voted end debate on the Drug Quality and Security Act, already approved by the House, to “make it easier to trace drugs throughout the supply chain” and to “require manufacturers of compounded drugs to better report outsourcing facilities and incentivize direct supervision” of pharmacists. The no vote was by Sen. David Vitter (R-LA), who is proposing to amend the bill to “force members of Congress to disclose which of their staff they have exempted from enrolling in the healthcare exchange.”
Bloomberg News (11/13, Rowley) reports the measure would “broaden federal regulation of compounding pharmacies.” The Senate is expected to approve the bill “as early as today.” It would prohibit “large compounding pharmacies from copying drugs approved by the FDA and marketed by other pharmaceutical companies.” It would also “put larger companies, known as ‘outsourcing facilities,’ under FDA oversight if they agree to be inspected.”
The Boston Globe (11/13, Bierman) reports the act would grant the FDA “clearer power to regulate” large compounding pharmacies.
(11/12, Wilemon) reports the measure “defines the authority of the U.S. Food and Drug Administration to regulate compounding pharmacies that make drugs in large quantities,” and would also put into place “a tracking system using serial numbers to protect against counterfeit, stolen and other compromised medicines.” (DIA Daily 11/13/13)
Senate May Finish Work On Compounding Pharmacy Bill Monday
(11/15, Kim, Subscription Publication) reports that the Senate may be finished with its “compounding pharmacy legislation as early as Monday,” given that Majority Leader Harry Reid yesterday “filed a motion to end debate” on the bill, “and took procedural steps to block further amendments.” The motion will be voted on Monday afternoon. The bill “would clarify the Food and Drug Administration’s oversight of compounding pharmacies and establish a national system for tracing drugs through the distribution chain.” (DIA Daily 11/15/13)
Court Rules Biosimilar Sponsors Can't Preemptively Sue
judge in the U.S. District Court for the Northern District of California ruled that companies developing biosimilars can't file suit seeking declaratory judgment of non-infringement of patents covering the reference biologic until they have submitted an application for approval of the biosimilar to FDA. In June, the Sandoz Inc. generics unit of Novartis AG filed suit against Amgen Inc. asking the court to declare that Sandoz's proposed biosimilar of Enbrel etanercept does not infringe two U.S. patents covering the autoimmune drug. The court dismissed the suit, stating that under federal regulations covering biosimilars, Sandoz must first submit an application to FDA. (BioCentury)
HIV Organ Policy Equity Act Passes Both Chambers and Awaits Obama Signature
The HIV Organ Policy Equity (HOPE) Act, which passed the Senate in June 2013, was passed by the House of Representatives this week and now awaits President Obama’s signature. The bill will allow patients with HIV to donate organs to other HIV-positive individuals, but only if they are participating in clinical research approved by an IRB. Regulators may waive this requirement if they feel it is no longer warranted. Regulators will have two years to promulgate regulations to establish processes for transplants and safeguards to assure that HIV positive organs do not enter the non-infected organ supply system. (Regulatory Focus)
Automatic Spending Cuts Would Bite More in 2014
The first year of the automatic cuts didn't live up to the dire predictions from the Obama administration and others who warned of sweeping furloughs and big disruptions of government services.
But the second round is going to be a lot worse, lawmakers and budget experts say. One reason is that federal agencies that have emptied the change jar and searched beneath the sofa cushions for money to ease the pain of sequestration have been so far able to make it through the automatic cuts relatively unscathed. Employee furloughs haven't been as extensive as feared and agencies were able to maintain most services. Most of that money, however, has been spent in the 2013 budget year that ended on Sept. 30.
For the time being, Congress has frozen 2014 spending at 2013 sequestration levels while negotiators seek a budget deal that would ease some of the automatic cuts. Absent a deal, the spending "caps" on agency operating budgets will shrink by another $20 billion or so. (AP)
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 Report; 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.