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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: August 29, 2014

SECTION 1 FDA GUIDANCES & MAPPS

CDER List of Guidance Documents

CDER Guidances: New/Revised/Withdrawn through 6/30/14

The links above lead to the List of Guidance Documents (CDER) updated on July 7, 2014, and to CDER Guidances that are new, revised, or withdrawn through the second calendar quarter of 2014.

Guidance Agenda: Guidance Documents CBER is Planning to Publish During Calendar Year 2014 (PDF - 31KB)

CDRH FY 2014 Proposed Guidance Development

On August 25, 2014, FDA announced the “renewal of certain FDA advisory committees”   by the Commissioner of Food and Drugs.  The Commissioner has determined that it is in the public interest to renew the charters of the committees listed in the announcement for an additional 2 years beyond charter expiration date.  See Committees and expiration dates at the following link.  [Federal Register]

On August 25, 2014, FDA announced the amendment of certain “Medical Device Regulations” to correct minor errors in the Code of Federal Regulations (CFR).  This action is editorial in nature and is intended to correct outdated Web site addresses.  [Federal Register]

On August 25, 2014, FDA announced the “Classification of the Hemoglobin A1c Test System (Medical Devices; Clinical Chemistry and Clinical Toxicology Devices)” into Class II (special controls).  The special controls that will apply to this device are identified in this order and will be part of the codified language for the hemoglobin A1c test system classification. The Agency is classifying the device into class II (special controls) in order to provide a reasonable assurance of safety and effectiveness of the device.  [Federal Register]

On August 27, 2014, FDA announced the availability of the draft guidance entitled “Controlled Correspondence Related to Generic Drug Development.”   The guidance document provides information regarding the process by which human generic drug manufacturers and related industry can submit correspondence to FDA requesting information on generic drug development. This guidance also describes FDA’s process for providing communications related to such correspondence.  Comments should be submitted by October 27, 2014. [Federal Register]

On August 27, 2014, FDA announced the availability of the guidance entitled “Highly Multiplexed Microbiological/Medical Countermeasure In Vitro Nucleic Acid Based Diagnostic Devices.”   This guidance is to provide industry and Agency staff with recommendations for studies to establish the analytical and clinical performance of highly multiplexed microbiological/medical countermeasure in vitro nucleic acid- based diagnostic devices (HMMDs) intended to simultaneously detect and identify multiple pathogen nucleic acids extracted from a single appropriate human specimen or culture.  [Federal Register]

On August 27, 2014, FDA announced the “Fee for Using a Tropical Disease Priority Review Voucher in FY 2015.”  FDA is authorized to determine and collect priority review user fees for certain applications for approval of drug or biological products when those applications use a tropical disease priority review voucher awarded by the Secretary of Health and Human Services. These vouchers are awarded to the sponsors of certain tropical disease product applications, submitted after September 27, 2007, upon FDA approval of such applications. The amount of the fee submitted to FDA with applications using a tropical disease priority review voucher is determined each FY based on the average cost incurred by FDA in the review of a human drug application subject to priority review in the previous FY. This notice establishes the tropical disease priority review fee rate for FY 2015.  [Federal Register]

On August 27, 2014, FDA announced it is correcting a notice entitled “Biosimilar User Fee Rates for FY 2015.”  These appeared in the Federal Register of August 1, 2014.  The document announced the rates for biosimilar user fees for fiscal year 2015. The document was published with the incorrect docket number. This document corrects that error.  [Federal Register]

On August 27, 2014, FDA announced it is correcting a notice entitled “Outsourcing Facility Fee Rates for FY 2015.”  These appeared in the Federal Register of August 1, 2014.  The document announced the rates for fiscal year 2015 for the establishment and reinspection fees related to human drug compounding outsourcing facilities that elect to register under the Federal Food, Drug, and Cosmetic Act. The document was published with the incorrect docket number. This document corrects that error.  [Federal Register]

On August 28, 2014, FDA announced the availability of the draft guidance entitled “International Cooperation on Harmonisation of Technical Requirements for Registration of Veterinary Medicinal Products; Draft Guidance for Industry on Electronic Exchange of Documents: File Format Recommendations.” This draft VICH guidance is intended to provide recommendations to industry on electronic file format specifications for individual documents and collections of multiple related documents that need no subsequent editing and are utilized for electronic exchange between industry and regulators in the context of regulatory approval of veterinary medicinal products. Comments should be submitted by October 27, 2014. [Federal Register]

On August 28, 2014, FDA announced the availability of the guidance entitled “Recommendations for Screening, Testing, and Management of Blood Donors and Blood and Blood Components Based on Screening Tests for Syphilis” dated September 2014. The guidance is intended for blood establishments that collect Whole Blood or blood components, including Source Plasma. The guidance announced in this notice finalizes the draft guidance of the same title, dated March 2013 (2013 draft guidance), and supersedes the memorandum of December 12, 1991, entitled ‘‘Clarification of FDA Recommendations for Donor Deferral and Product Distribution Based on the Results of Syphilis Testing.’’ [Federal Register]

On August 29, 2014, FDA announced the availability of the draft guidance entitled “Electronic Submission of Lot Distribution Reports.”  The draft guidance document provides information and recommendations pertaining to the electronic submission of lot distribution reports for applicants with approved biologics license applications (BLAs). FDA recently published in the Federal Register a final rule requiring that, among other things, lot distribution reports be submitted to FDA in an electronic format that the Agency can process, review, and archive.  Comments should be submitted by November 28, 2014.  [Federal Register]


SECTION 2 FDA NOTES & RELATED NEWS

Updated List of CDER Key Officials Posted

New FDA Generics Team to Improve Review Efficiency, Consistency
FDA enlisted two of its generic drug veterans to head a new group tasked with priming the review system as it prepares for many applications to be under a review clock.  Acting OGD Director Kathleen Uhl has created a Quality Oversight Team (QOT) to enhance the OGD review system in advance of the formal review goals, which are set to launch in fiscal year 2015.

OGD veterans Robert West and Peter Rickman are running the new group as associate director of review quality and associate director of submission quality, respectively.  The QOT will oversee all ANDA-related actions, develop an internal quality program for review actions and “refine the review process to best position OGD to meet its GDUFA goals,” according to an Aug. 7 memo to OGD staff.

According to the memo, the team will reside in the OGD Office of Regulatory Operations immediate office, above the Division of Quality Management Systems, because of the importance of its work.  More at link above.  (Pink Sheet, paid subscription required)

FDA Clears Stem Cell Trial for Spinal Cord Injuries
The San Francisco Business Times (8/28, Subscription Publication) reports in its “Biotech SF” blog that the FDA gave the green signal to Asterias Biotherapeutics Inc. to proceed with a 13-person safety study of “oligodendrocyte progenitor cells, or OPCs.” The cells, derived from embryonic stem cells, are believed “to stimulate the growth of new nerve cells around the spinal cord and could help paralyzed patients regain movement.” The company expects to begin enrolling patients in early 2015, the blog posting notes.  (Via the 8/28/14 DIA Daily, a DIA membership benefit)

US Needs to Reform Discovery, Development, Delivery of Drugs
U.S. regulators need to guarantee rewards for innovation, promote wider data sharing while protecting privacy and improve the existing drug-development model, write Nobel Prize winner Phillip Sharp, Tufts Center for the Study of Drug Development Director Kenneth Kaitin and former FDA Commissioner Dr. Andrew von Eschenbach. Because Massachusetts is a leader in life sciences innovation, it is the right place to examine greater ways to incentivize innovative drugmakers for bringing greater value to patients, they write. The state can only maintain and expand its leadership in the life-sciences sector if the U.S. changes its process of discovering, developing and delivering better drugs to patients, they write. (Boston Herald blog via FDLI Smartbrief 8/25/14)

Diagnostics Industry is Cautious, Wary, and Diplomatic on FDA Push to Regulate LDTs
The FDA's decision to pursue broad, risk-based regulatory oversight for genetic and other laboratory-developed tests (LDTs) is drawing cautious, diplomatic and hopeful responses from industry.

Reading between the lines of the range of statements issued after the FDA announcement of pending draft guidance suggests there will be plenty of pushback and industry lobbying before any final regulations are put in place.

The Association for Molecular Pathology (AMP) insisted that these tests already have sufficient oversight by way of the Centers for Medicare & Medicaid Services' Clinical Laboratory Improvement Amendments (CLIA) program, which doesn't require rigorous and lengthy FDA premarket review. Regulators want to begin using the PMA process for certain higher risk tests and expand that oversight even further over time.

Similarly, the American Clinical Laboratory Association (ACLA) issued remarks supporting the CLIA regulatory system, expressing concern that more regulation could "stifle diagnostic innovation and ultimately jeopardize patient access to timely and effective treatments."

Part of the FDA's push for broader regulatory reach over diagnostic testing is new guidance to govern the approval or clearance of companion diagnostic genetic tests, which help doctors choose a cancer treatment that will produce the best patient response. AdvaMed's diagnostics arm (AdvaMedDx), meanwhile, issued a statement that "welcomes" the FDA's draft guidance on LDTs and stated membership support for a "modernized and flexible" approach to diagnostics regulatory review.  More at link above.  (Fierce Diagnostics)


SECTION 3 AGENCY AND ADVISORY COMMITTEE MEETINGS

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

Public Meeting. Nonprescription Drugs Advisory Committee Meeting.  September 3, 2014. White Oak Campus, Silver Spring, MD.   The committee will discuss the standards used to demonstrate that over-the-counter (OTC) topical antiseptics used in healthcare settings are generally recognized as safe and effective.  [Federal Register]
 
Public Workshop. Hemostatic Medical Devices for Trauma Use. September 3-4, 2014.  White Oak Campus, Silver Spring, MD.  The purpose of this workshop is to discuss factors that contribute to hemostatic medical device performance and reliability and types of studies used to assess bleeding and validate methods to evaluate the severity of bleeding, and to define regulatory pathways for novel products. [Federal Register]

Public Workshop. Clinical Development of Drugs for the Prevention of Infections Caused by Staphylococcus aureus in the Health Care Setting. September 5, 2014.  White Oak Campus, Silver Spring, MD.  This public workshop is intended to provide information for and gain perspective from health care providers, patients and patient advocacy organizations, academia, and industry on various aspects of clinical development of drugs to prevent Staphylococcus aureus infections including the design of clinical trials. [Federal Register]

Public Meeting. Advancing the Use of Biomarkers and Pharmacogenomics. September 5, 2014. Washington, DC.  The purpose of the public meeting is to initiate constructive discussion and information sharing on the advancement of biomarker science in the context of therapeutic product development among relevant stakeholders. [Federal Register]

Public Meeting in Collaboration with the National Cancer Institute. Methodological Considerations in Evaluation of Cancer as an Adverse Outcome Associated with Use of non-Oncological Drugs and Biological Products in the Post-approval Setting.  September 10 - 11, 2014.  Silver Spring, MD.  The purpose of the public meeting is to engage in constructive dialogue and information sharing among regulators, researchers, the pharmaceutical industry, public health agencies, health care providers, and the general public concerning challenges in designing and implementing post-approval studies to evaluate the risk of cancer associated with use of non-oncological drugs and biological products. The input from this meeting and public docket will be used to inform the Agency on best study design and methodological options to consider when evaluating cancer risk in the post-approval setting.  [Federal Register]

Public Advisory Committee Meeting. Cardiovascular and Renal Drugs Advisory Committee. September 10, 2014. White Oak Campus, Silver Spring, MD.  The committee will be asked to discuss the potential clinical utility of fixed-combination prescription drugs composed of an anti-hypertensive drug, aspirin, and a statin administered to reduce the risk of cardiovascular death, nonfatal myocardial infarction, and nonfatal stroke in patients with a history of cardiovascular disease. The committee will be asked to discuss the patient population that could benefit from such a product, whether that population would be likely to take such a drug long term, and how this could be assured. [Federal Register]

Meeting. Third Annual Patient Network Meeting; Under the Microscope: Pediatric Drug Development. September 10, 2014. Washington, DC.  The meeting will serve as a forum for FDA’s stakeholders (patients, caregivers, patient advocates, healthcare professional groups, the general public, academia, and industry) to learn about regulations that encourage pediatric product development; to discuss ways to advance pediatric product development, how health disparities impact pediatric product development, the importance of transparency in pediatric clinical trials, and how analysis of information from failed pediatric clinical trials might improve future designs for pediatric trials; and to identify ways patient input can benefit clinical trial design for pediatric trials. [Federal Register]

Meeting. International Medical Device Regulators Forum (IMDRF), September 15-19, 2014.  Embassy Row Hilton, Washington, DC.  A week of global meetings to discuss worldwide medical device regulation and harmonization efforts.  [FDA.gov]

Public Advisory Committee Meeting. Cellular, Tissue and Gene Therapies Advisory Committee. September 17, 2014. Hyattsville, MD.  In open session, the committee will hear updates of research programs in the Laboratory of Biochemistry, Division of Therapeutic Proteins, the Laboratory of Molecular Oncology and the Laboratory of Molecular and Developmental Immunology, Division of Monoclonal Antibodies, Office of Biotechnology Products, Office of Pharmaceutical Sciences, Center for Drug Evaluation and Research, FDA. Another portion of the meeting will be closed to the public.  [Federal Register]

Public Hearing.  Generic Drug User Fee Amendments of 2012: Public Hearing on Policy Development.   September 17, 2014.  Hyattsville, MD.  FDA seeks input on the five draft guidance documents FDA has issued or will issue shortly to facilitate implementation of GDUFA. FDA also seeks input on additional policy priorities under GDUFA, such as the Agency’s consideration of generic drug exclusivity and the category of first generics. FDA will take the information from the public meeting into account in developing the fiscal year 2015 GDUFA priorities. [FDA.gov]

Public Advisory Committee Meeting. Joint Meeting of the Bone, Reproductive and Urologic Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee. September 18, 2014. White Oak Campus, Silver Spring, MD.  The committees will discuss the appropriate indicated population for testosterone replacement therapy and the potential for adverse cardiovascular outcomes associated with this use. [Federal Register]

Public Workshop. Pediatric Clinical Investigator Training Workshop. September 22, 2014.  Bethesda, MD.  The purpose of this workshop is to provide investigators with training and expertise in designing and conducting clinical trials in pediatric patients that will lead to appropriate labeling. [Federal Register]

Public Meeting. Patient-Focused Drug Development for Hemophilia A, Hemophilia B, von Willebrand Disease, and Other Heritable Bleeding Disorders. September 22, 2014. White Oak Campus, Silver Spring, MD.  The public meeting is intended to allow FDA to obtain patient perspectives on the impact of Hemophilia A, Hemophilia B, von Willebrand Disease, and other heritable bleeding disorders on daily life as well as patient perspectives on the available therapies for these disorders. [Federal Register]

Public Workshop. Next-Generation Sequencing Technology, Data Formats Standardization and Promotion of Interoperability Protocols.  September 24-25, 2014. National Institute of Health Campus, Bethesda, MD. The purpose of the workshop is to engage NGS stakeholders in a forum to discuss the current use of the technology and the development of data standards of NGS-related information. [Federal Register]

Public Meeting. Patient-Focused Drug Development for Idiopathic Pulmonary Fibrosis. September 26, 2014. White Oak Campus, Silver Spring, MD.  The public meeting is intended to allow FDA to obtain patient perspectives on the impact of idiopathic pulmonary fibrosis on daily life as well as patient views on treatment approaches for idiopathic pulmonary fibrosis. [Federal Register]

Public Workshop. Additive Manufacturing of Medical Devices: An Interactive Discussion on the Technical Considerations of 3–D Printing. October 8-9, 2014. White Oak Campus, Silver Spring, MD.  The purpose of this workshop is to provide a forum for FDA, medical device manufactures, additive manufacturing companies, and academia to discuss technical challenges and solutions of 3–D printing. The Agency would like input regarding technical assessments that should be considered for additively manufactured devices to provide a transparent evaluation process for future submissions. [Federal Register]

Public Meeting.  Patient-Focused Drug Development and Scientific Workshop on Female Sexual Dysfunction.   October 27-28, 2014.  White Oak Campus, Silver Spring, MD.  FDA is conducting a Patient-Focused Drug Development public meeting and scientific workshop on Female Sexual Dysfunction (FSD).  [Federal Register]

New:  Public Advisory Committee Meeting. Risk Communications Advisory Committee. November 3-4, 2014. White Oak Campus, Silver Spring, MD.  The Risk Communication Advisory Committee will discuss methods for effective risk communication with a focus on messages about the importance of eating adequate amounts of fish, while avoiding certain fish with higher amounts of methyl-mercury. These messages are especially important for women who are pregnant or nursing, or for anyone who prepares food for young children. [Federal Register]

Clinical Investigator Training Course. November 4-6, 2014. College Park, MD.  This training course, a co-sponsored event of FDA CDER and Duke University Office of Continuing Medical Education, is intended to provide clinical investigators with expertise in the design, conduct, and analysis of clinical trials; improve the quality of clinical trials; and enhance the safety of trial participants.  [Federal Register]

New:  Public Advisory Committee Meeting. Cellular, Tissue and Gene Therapies Advisory Committee. November 6, 2014. White Oak Campus, Silver Spring, MD. The committee will discuss the draft guidance for industry entitled ‘‘Design and Analysis of Shedding Studies for Virus or Bacteria-Based Gene Therapy and Oncolytic Products’’ and the Dear Gene Therapy IND or Master File Sponsor Letter.  [Federal Register]

Public Workshop. Brain-Computer Interface Devices for Patients With Paralysis and Amputation. November 21, 2014.  White Oak Campus, Silver Spring, MD.  The purpose of this workshop is to obtain public feedback on scientific, clinical, and regulatory considerations associated with BCI (Brain-Computer Interface) devices. Ideas and suggestions generated during this workshop may facilitate development of draft guidance to provide our initial thoughts regarding the content of premarket submissions for emerging BCI technologies to help speed development and approval of future submissions.  [Federal Register]


SECTION 4 OTHER REGULATORY AUTHORITIES & ORGANIZATIONS

NIH Issues Finalized Policy on Genomic Data Sharing
The National Institutes of Health has issued a final NIH Genomic Data Sharing (GDS) policy to promote data sharing as a way to speed the translation of data into knowledge, products and procedures that improve health while protecting the privacy of research participants. The final policy was published in the NIH Guide for Grants and Contracts Aug. 27, 2014.

The GDS policy is an extension of and replaces the Genome-Wide Association Studies (GWAS) data sharing policy. Since 2007, the GWAS policy has governed biomedical researchers’ submission and access to human data through the NIH database for Genotypes and Phenotypes (dbGaP). Its two-tiered data distribution system has made some information and data available to the public without restrictions. Access to other data has been controlled and made available only for research purposes consistent with the consent provided by participants in the original study.

A key tenet of the GDS policy is the expectation that researchers obtain the informed consent of study participants for the potential future use of their de-identified data for research and for broad sharing. NIH also has similar expectations for studies that involve the use of de-identified cell lines or clinical specimens.

The two-tiered system for providing access to human data is based on data sensitivity and privacy concerns developed under the GWAS policy will continue. For controlled-access data, investigators will be expected to use data only for the approved research, protect data confidentiality (including not sharing the data with unauthorized people), and acknowledge data-submitting investigators in presentations and publications.  More at link above. (NIH.gov)

NIH Announces National Biosafety Stewardship Month to Promote Lab Safety
The National Institute of Health (NIH) is taking remedial action and precautionary steps to improve its lab safety protocols and procedures, minimize the risk of recurrence, and increase timely reporting of potential problems. To raise awareness of these critical issues, NIH and other agencies within the Department of Health and Human Services (HHS) are initiating National Biosafety Stewardship Month during the month of September—a time for federal labs engaged in biomedical research to focus special attention on safe practices.

NIH also issued a Guide Notice to reinforce the message that its grantees, universities, hospitals, and other research organizations across the nation that receive NIH funding must meet all applicable federal, state, and local health and safety standards for research conduct. More at title link above.  (NIH.gov)

White House calls for labs to check inventories
In a memorandum, the White House said all federally funded labs working with infectious agents must take "immediate and longer-term steps" to enhance biosafety and biosecurity, including taking an inventory of infectious samples and reviewing safety and security protocols. The memo was sent to federal agencies earlier this month but made publicly available on Thursday.

The request follows "three recent U.S. biosafety and biosecurity incidents," including the potential, unintentional exposure of laboratory personnel at a U.S. Centers for Disease Control and Prevention lab to viable anthrax.  More at link above. (BioCentury)

NIH to Launch Human Safety Study of Ebola Vaccine Candidate
Initial human testing of an investigational vaccine to prevent Ebola virus disease will begin next week by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health.

The early-stage trial will begin initial human testing of a vaccine co-developed by NIAID and GlaxoSmithKline (GSK) and will evaluate the experimental vaccine’s safety and ability to generate an immune system response in healthy adults. Testing will take place at the NIH Clinical Center in Bethesda, Maryland.

The study is the first of several Phase 1 clinical trials that will examine the investigational NIAID/GSK Ebola vaccine and an experimental Ebola vaccine developed by the Public Health Agency of Canada and licensed to NewLink Genetics Corp. The others are to launch in the fall. These trials are conducted in healthy adults who are not infected with Ebola virus to determine if the vaccine is safe and induces an adequate immune response.  More at link above.  (NIH.gov)

Alzheimer's Forum Showcases a Look at Life with Early Alzheimers Disease
What is it like to care for a person with dementia? For most people, it can be hard to imagine the daily struggle, the exhaustion, the emotional pain. And in truth, recent portrayals of Alzheimer’s in books, newsprint, and advocacy pleas have shied away from showing the progression of the disease as it truly is; some give the impression that the worst of Alzheimer’s is a patient sitting in a wheelchair or lying in bed with a vacant stare. It is not—and Meryl Comer’s upcoming book makes this utterly clear. In Slow Dancing With a Stranger (HarperCollins Publishers, 2014), she offers a glimpse behind closed doors at the raw, difficult reality of day-to-day life with an Alzheimer’s patient. The book will hit stores September 2, but is available online for pre-order.  More at link above.  (AlzForum)

Apple Tells Developers They May Not Sell Personal Health Data to Advertisers
Apple has tightened its privacy rules relating to health apps ahead of next month's product launch, which is expected to see the unveiling of an updated iPhone and could include new wearable technology.

The technology firm has told developers that their apps, which would use Apple's "HealthKit" platform on the forthcoming products, must not sell any personal data they gather to advertisers. The rules add that they could share their data with "third parties for medical research purposes" as long as they get users' consent. The move could stave off concerns users might have around privacy as Apple seeks to move into the health data business.  More at link above.  (The Guardian)

EHR Update: As Adoption Increases, So Do Privacy and Data Security Concerns
Physicians in all specialties have steadily adopted electronic health records (EHRs). However, as doctors have embraced technology for streamlining patient records, we have seen a marked uptick in data breaches by hackers who want access to the reams of information stored in these systems.

Ironically, the very technology that allows health records to be exchanged between physicians and hospitals also makes them susceptible to data breaches. Furthermore, because EHRs bundle a lot of important information in one place, hackers have access to personal identification information, insurance information, lab results, and a host of other private, potentially lucrative data.

Last week, the FBI issued a flash alert warning to healthcare organizations that they are being directly targeted by hackers (Reuters).  An important takeaway from these latest breaches is that the threat to patients is more than financial. In addition to the potential for false billings and data to insurance companies, alterations to medical records may follow a patient forever, providing subsequent health care providers with incorrect medical information that could alter a diagnosis.  More at link above.  (Policy and Medicine)

Todd Park to Step Down from US Chief Technology Officer Post
After five years in the District of Columbia, Todd Park--who initially served as the first chief technology officer for the U.S. Department of Health & Human Services before being named U.S. CTO by President Obama in 2012--is heading to the other side of the country.

Park will step down as U.S. CTO by the end of 2014, according to an article in Forbes. He will take a new role with the White House, working in California to recruit tech talent for government roles, according to Forbes.

Park, co-founder of health IT companies Athena health and Castlight, became CTO of HHS in 2009.  More at link above.  (Fierce Health IT)

Cochrane Announces Opening of US Cochrane West
Cochrane announced this week (8/25/14) the official opening of the West Coast branch of the US Cochrane Center (USCC).  Oregon Health and Science University (OHSU) in Portland, Oregon has become the West Coast branch of the US Center of The Cochrane Collaboration.

US Cochrane West will promote evidence-based decision making in health care in the US. It will work in conjunction with its primary center at the John Hopkins Bloomberg School of Public Health by facilitating accurate, up-to-date Cochrane Reviews and providing support and training to new authors of Cochrane Reviews.

US Cochrane West will be led by Mark Helfand, a professor of medicine, medical informatics, and clinical epidemiology at OHSU, and a staff physician at the Portland VA Medical Center. He is a vastly experienced US leader in systematic healthcare reviews and evidence-based medicine.  More at link above.  (Cochrane.org)

IBM Watson Speeds Drug Research
IBM's Watson cognitive computing technology has helped doctors, loan officers, corporations, and even military veterans find answers to complex questions. The next big challenge for Watson is helping researchers explore the unknown.

Baylor College of Medicine and IBM published this week a peer-reviewed study that came up with six promising paths for cancer research with the aid of Watson Discovery Advisor.  As part of Baylor's research, Watson analyzed more than 70,000 scientific articles related to p53, a protein that has been linked to many cancers. Automated analysis carried out by Watson helped Baylor biologists and data scientists identify six proteins that modify p53 and that should be targeted for new research. Most important, the discovery was made in a matter of weeks, according to IBM.  More at link above.  (Information Week)

Computer Vision Algorithm Could Enable Faster Diagnosis of Malaria
Researchers from the Institute for Molecular Medicine Finland, the University of Helsinki and Karolinska Institutet in Stockholm have designed a "man and machine" decision-support system that could diagnose malaria faster. The method, described in the journal PLOS One, involves the use of a computer vision algorithm to analyze blood cells, which are ranked according to probability of infection. A program creates a panel of images of the cells that are likely to be infected, which is used by an expert to make the final diagnosis.  (DIA Global Smartbrief 8/26/14)

EMA Publishes Booklet on European Regulatory System for Medicines
The European Medicines Agency (EMA) today published an illustrated leaflet explaining how the European regulatory system for medicines operates. It describes how medicines are authorised and monitored in the European Union (EU) and how the European medicines regulatory network – a partnership between the European Commission, the 50 medicines regulatory authorities in the EU and the European Economic Area (EEA), and the EMA – works to ensure that patients in the EU have access to safe and effective medicines. More at link above. (EMA.europa.eu)

EMA Proposes Some Advisory Committee Meetings Go Public
The European Medicines Agency is proposing to hold some of its advisory committee meetings in public.  The move is a further step towards increasing the transparency of EMA’s work that earlier this year included the release of edited minutes of CHMP meetings for the first time.

The agency issued a public consultation at the end of July on draft procedural rules for public involvement in monthly meetings of its Pharmacovigilance Risk Assessment Committee, the panel which discusses the safety of medicines. The public will be allowed to contribute to meetings, expressing their views on the safety of medicines and the management or risks. Currently, patient advocacy groups and patient charities can be invited to attend EMA meetings, or to provide written submissions, but wider participation has not been encouraged.  More at link above.  (Pink Sheet, paid subscription required)

Generic Drug-makers Tussle with International Standards Group
The International Generic Pharmaceutical Alliance, an umbrella group for trade associations from the U.S., Europe, Canada and other countries, is miffed that the International Conference on Harmonization has refused to give generic drug makers a position on the all-important steering committee that oversees decisions on drug safety, efficacy, clinical trials and manufacturing practices.

To date, the generic pharmaceutical industry has participated in ICH meetings, but only in an advisory role, according to David Gaugh, a senior vice president for regulatory sciences at the U.S. Generic Pharmaceutical Association and an IGPA spokesman for this particular issue.

In an Aug 8 letter to ICH, the umbrella group wrote that a decision last June to rebuff generic drug makers means the “generic industry will remain completely barred from the formal ICH decision-making process, even though the vast majority of ICH guidelines become regulatory requirements and guidances that are directly applicable to generic pharmaceutical manufacturers.” More at link above.  (WSJ Pharmalot)

NHS Boosts UK Cancer Fund
NHS England said it will provide the Cancer Drugs Fund (CDF) with an additional L160 million ($265.1 million) over two years. The fund is intended to give patients access to drugs that would not otherwise be available on the NHS.

CDF will also now re-evaluate and remove coverage of the least effective cancer drugs. CDF said drugs it deems excessively expensive could be removed from the national list of covered treatments unless their prices are adjusted. The list includes drugs appraised by the U.K.'s NICE but not recommended based on cost-effectiveness, as well as drugs not yet appraised by NICE.

CDF will not re-evaluate drugs that are the only proven treatments for particular cancers. CDF also plans to conduct a confidential analysis of drug costs and patient benefits.

NHS's bump will expand the fund's annual budget by 40% to L280 million ($464 million). Launched in 2010, CDF is funded until 2016.  (BioCentury)

India Outlines Plans for Upgrading Clinical Trial Procedures
In its latest bid to upgrade regulatory oversight of clinical trials and restore confidence among global drug makers, the Indian government recently issued a proposal to enhance procedures for gathering and monitoring key information, particularly concerning patients.

The proposal is designed to accomplish a few things: help streamline procedures for approving clinical trials; maintain a comprehensive database of clinical trial details; authenticate data that is generated by studies and monitor information to ensure patient rights and safety are protected.

Earlier this month, the regulator sent state authorities a list of practices that must be followed – or improved – in order to successfully inspect manufacturing facilities. The move came as the U.S. FDA cracks down on violators in the wake of alleged quality-control violations by Indian drug makers.  More at link above.  (WSJ Pharmalot)


SECTION 5 LEGAL AND COMPLIANCE

5 Takeaways from the 21st Century Cures Initiative Thus Far
The U.S. House of Representatives Energy and Commerce Committee has engaged in a flurry of activity since the May launch of the 21st Century Cures initiative that aims to accelerate the pace of cures and medical breakthroughs in the United States. The committee has held three roundtables and six congressional hearings on various topics affecting the full arc of the drug development process -- from discovery to development to delivery -- as well as published four white papers.  Five themes have emerged form testimony and public submissions thus far:

  • Encourage and incentivize public-private partnerships that accelerate medical research
  • Encourage new models for clinical trials that take advantage of today’s science
  • Leverage data collected to advance medical knowledge and improve patient care (EHR’s and patient registries)
  • Find the right incentives for encouraging the most innovative research
  • System needs a steady, predictable trajectory of support for medical research.
More at link above.  (FasterCures Blog)

Court upholds HRSA's 340B Orphan interpretive rule
The U.S. District Court for the District of Columbia upheld an interpretive rule allowing discounts for Orphan drugs under Medicare's 340B program when used for non-Orphan indications. The Pharmaceutical Research and Manufacturers of America (PhRMA) had asked the court to vacate the rule, which was issued in July by HHS's Health Resources and Services Administration (HRSA).


The 340B program requires manufacturers to deeply discount outpatient drugs to hospitals and clinics that provide healthcare for low income and other special populations. In 2010, Congress amended 340B to exclude Orphan drugs from discounting, but the law was unclear on whether the exclusion applied when the drugs were used for non-Orphan indications.  (BioCentury)

Black Box Warning Labels and Patent Cliff Have Greatly Reduced Lawsuits over Antidepressants
The number of lawsuits over violent acts committed under the influence of antidepressants, which “caused such angst for companies such as Eli Lilly and Co.,” have “pretty much subsided,” reports the Indianapolis Star (8/25, Swiatek). While use of the drugs “is as widespread as ever,” the number of suits has fallen off because of the FDA’s decision 10 years ago to require a “black box” warning about the link between antidepressants and suicidal behavior. Another reasons for the decline, reports the Star, is that the most popular antidepressants have lost patent protection. Lilly was “once the focus” of this type of litigation, but is “largely removed from it” now that Prozac and Cymbalta have gone off-patent and have “faded in significance for the company.”  (Via the 8/25/14 DIA Daily, a DIA membership benefit)

PhRMA Urges Court to Reject Off-Label Promotion Case
The Pharmaceutical Research and Manufacturers of America (PhRMA) says a California federal court should dismiss a whistleblower's False Claims Act suit against three drug companies on First Amendment grounds. The association filed a friend-of-the-court brief in a US Justice Department-backed case involving claims that Millennium, Schering-Plough and Merck illegally promoted Integrilin for off-label uses.

The brief asserts that the whistleblower says a drug manufacturer's truthful speech about an unapproved use of an FDA-approved drug violates the False Claims Act (FCA) solely because a different law, the Federal Food, Drug, and Cosmetic Act (FFDCA), makes it illegal for drug manufacturers to promote drugs for unapproved uses.

“The prevalence of unapproved but fully legal uses of many FDA-approved prescription medicines to treat patients makes it critical that healthcare professionals have access to accurate, comprehensive, and current information about such uses,” PhRMA's brief contends. More at link above. (Medical Marketing and Media)


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; EP Vantage; Evaluate Pharma; Eye on FDA; FDA.gov; FDA Law Blog; Federal Register Table of Contents; Fierce Medical Devices; Fierce Pharma; Fierce Vaccines; FDLI Smartbrief; Genomeweb; Health Industry Washington Watch; Government Health IT; Health IT Security; Institute of Medicine News; MedCityNews; Medical Device Daily; Medical Device & Diagnostic Industry; MedPage Today; NPC Bulletin; Nutra Ingredients USA; Pharmabiz; Pharmafile; Pharma IQ; PharmaTimes; PhRMA website; PM Live; Policy and Medicine (newsletter); Regulatory Focus; RegLink News; US FDA Daily Digest Bulletin.