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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: February 20, 2015

SECTION 1 FDA GUIDANCES & MAPPS

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

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

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

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final guidances for which CDRH would appreciate external feedback on whether these final guidances should be revised or withdrawn.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

On February 19, 2015, FDA announced the publication of five draft guidances related to drug compounding and repackaging that will help entities comply with important public health provisions. The draft documents are applicable to pharmacies, federal facilities, outsourcing facilities and physicians. “The draft guidance documents provide information to pharmacies, outsourcing facilities, health care entities, and others about these FDA-proposed policies, which are critical to protecting the public health,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research.  The draft guidances are:

        Draft Guidance: For Entities Considering Whether to Register As Outsourcing Facilities under Section 503B of the Federal Food, Drug, and Cosmetic Act
The draft guidance provides an entity considering whether to register with the FDA as an outsourcing facility under the law with information about the regulatory impact of registering. For example, it explains that a facility engaged in only certain activities, including repackaging human drugs and compounding non-sterile drugs, should not register as an outsourcing facility because its drug products will not qualify for the exemptions provided in section 503B, including the exemption from the new drug approval requirements. Comments should be received by May 20, 2015.  [Federal Register]
        Draft Guidance for Industry: Repackaging of Certain Human Drug Products by Pharmacies and Outsourcing Facilities
The draft guidance describes the conditions under which the FDA does not intend to take action for certain violations of the law when state-licensed pharmacies, federal facilities or outsourcing facilities repackage certain drug products. Repackaging generally involves taking a finished drug product from the container in which it was distributed by the original manufacturer and placing it into a different container. Repackaged drug products are generally not exempt from any of the provisions of the FD&C Act related to the production of drugs, and the compounding provisions of the FD&C Act do not address repackaging. Therefore, the FDA is issuing guidance to describe how it intends to address repackaging when done in a state-licensed pharmacy, federal facility, or outsourcing facility. Comments should be received by May 20, 2015.  [Federal Register]
        Draft Guidance for Industry: Mixing, Diluting, or Repackaging Biological Products Outside the Scope of an Approved Biologics License Application (BLA)
The draft guidance describes the conditions under which the FDA does not intend to take action for violations of certain sections of the Public Health Service Act (PHS Act) and the FD&C Act when state-licensed pharmacies, federal facilities or outsourcing facilities mix, dilute or repackage specific biological products without an approved BLA, or when such facilities or physicians prepare prescription sets of allergenic extracts (used to treat allergies) without an approved BLA. The draft guidance notes that a biological product that is mixed, diluted or repackaged outside the scope of an approved BLA is an unlicensed biological product under section 351 of the PHS Act and may not be legally marketed without an approved BLA. Additionally, the compounding provisions of the FD&C Act do not address biological products subject to licensure under section 351 of the PHS Act. Therefore, the FDA is issuing guidance to describe how it intends to address these practices. Comments should be received by May 20, 2015. [Federal Register]
        Draft Guidance for Industry: Adverse Event Reporting for Outsourcing Facilities under Section 503B of the Federal Food, Drug, and Cosmetic Act
Entities registered as outsourcing facilities are required to report adverse events to the FDA. The draft guidance explains adverse event reporting for outsourcing facilities. Comments should be received by May 20, 2015. [Federal Register]
        Draft Memorandum of Understanding Between A State and the U.S. Food and Drug Administration Addressing Certain Distributions of Compounded Human Drug ProductsThe draft MOU under section 503A of the FD&C Act describes the responsibilities of a state that chooses to sign the MOU in investigating and responding to complaints related to compounded human drug products distributed outside the state, and in addressing the interstate distribution of “inordinate amounts” of compounded human drug products. Comments should be received by June 19, 2015.  [Federal Register]

On February 20, 2015, FDA announced the availability of the draft guidance entitled ‘‘‘Investigating and Reporting Adverse Reactions Related to Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) Regulated Solely Under Section 361 of the Public Health Service Act and 21 CFR part 1271’.” The purpose of this guidance is to provide manufacturers of human cells, tissues, and cellular and tissue-based products (HCT/Ps) for which no premarket submissions are required because they are not also regulated as drugs, devices, and/or biological products, with recommendations for complying with the requirements for investigating and reporting adverse reactions involving communicable disease in recipients of these HCT/Ps. Comments should be received by April 21, 2015. [Federal Register]


SECTION 2 FDA NOTES & RELATED NEWS

Jarow Named Acting Director of CDER Office of Medical Policy
Janet Woodcock, director of FDA's Center for Drug Evaluation and Research, announced in an internal memo that Jonathan Jarow will serve a 120-day detail as acting director of the Office of Medical Policy. Jarow is associate director of CDER's Office of Hematology and Oncology Products (OHOP).

CDER spokesperson Kristofer Baumgartner said the detail is part of Woodcock's selection process for a permanent director. He said it allows Jarow to "try out" the position before the agency makes a final decision.

OMP has been without a director since Rachel Sherman retired in January 2014. Denise Hinton, who has served as OMP's acting director for the past 13 months, will resume her role as deputy director.  (BioCentury)

Recent Progress on Demographic Information and Clinical Trials
In her FDA Voice blog on February 19, Dr. Barbara Buch, Associate Director for Medicine in FDA’s Center for Biologics Evaluation and Research (CBER), wrote about FDA’s progress toward the FDASIA mandate to look at questions on clinical trial data availability based on sex, age, race and ethnicity. Specifically, FDA was asked to look at these questions on a broader scale: to investigate how well demographic subgroups (sex, age, race and ethnicity) are included in clinical trials; whether they are analyzed for safety and effectiveness by these subgroups; and to improve on making the resulting information available to the public.

In August 2013 the Agency reported that it was doing well, but it created an improvement plan that include three priorities to: improve the completeness and quality of demographic subgroup data collection, reporting and analysis; identify barriers to subgroup enrollment in clinical trials and employ strategies to encourage greater participation; and improve the public availability of demographic subgroup data.

Dr. Buch discussed the “Drug Snapshots” web page, which extract demographic subgroup data for FDA approved products and the leveraging of existing IT platforms to enhance FDA’s systems for collecting, analyzing, and communicating standardized data collection categories by age, racial and ethnic groups in submitted applications.  More at link above. (FDA.gov)

FDA permits marketing of first direct-to-consumer genetic carrier test for Bloom syndrome
FDA today (February 19) authorized for marketing 23andMe’s Bloom Syndrome carrier test, a direct-to-consumer (DTC) genetic test to determine whether a healthy person has a variant in a gene that could lead to their offspring inheriting the serious disorder.

Along with this authorization, the FDA is also classifying carrier screening tests as class II. In addition, the FDA intends to exempt these devices from FDA premarket review. The agency plans to issue a notice that announces the intent to exempt these tests and that provides a 30-day period for public comment. This action creates the least burdensome regulatory path for autosomal recessive carrier screening tests with similar uses to enter the market.

“The FDA believes that in many circumstances it is not necessary for consumers to go through a licensed practitioner to have direct access to their personal genetic information. Today’s authorization and accompanying classification, along with FDA’s intent to exempt these devices from FDA premarket review, supports innovation and will ultimately benefit consumers,” said Alberto Gutierrez, Ph.D., director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health. “These tests have the potential to provide people with information about possible mutations in their genes that could be passed on to their children.” More at link above.  (FDA.gov)

FDA to Study Quality of Long-Acting Generic Drug Products
The US Food and Drug Administration (FDA) is prepared to spend nearly a million dollars over the next two years to study the quality and effectiveness of long-acting generic drug products, including levonorgestrel-based birth control products, the agency announced last week.  The two other awards announced by FDA relate to two specific types of products: birth control implants and periodontal drugs.

The FDA focus is on generic long acting injectables. The active pharmaceutical ingredient (API) in long acting injectable (LAI) products is usually encapsulated in microspheres (i.e., glycolide/lactide matrix) that extends the release of API into the systemic circulation, giving a relatively long apparent half-life and a unique PK profile.  The current bioequivalence (BE) guidance in regards to LAIs is that the generic formulations are required to be qualitatively (Q1) and quantitatively (Q2) the same as the reference-listed drug (RLD).

In general, the FDA recommends in vivo single-dose, randomized, parallel BE study in healthy volunteers. However, parallel BE studies with LAI products are very challenging due to high inter-subject variability, complex PKPD profiles, study length and expenditure. FDA said it hopes its research will be able to "assist development of generic LAI products.  More at link above. (Regulatory Focus)

CBER Announce Regulatory Site Visit Training Program
CBER is announcing an invitation for participation in its Regulatory Site Visit Training Program (RSVP). This training program is intended to give CBER regulatory review, compliance, and other relevant staff an opportunity to visit biologics facilities. These visits are intended to allow CBER staff to directly observe routine manufacturing practices and to give CBER staff a better understanding of the biologics industry, including its challenges and operations. The purpose of the announcement is to invite biologics facilities to contact CBER for more information if they are interested in participating in this program. Submit either an electronic or written request for participation in this program by March 19, 2015.  (Federal Register)


SECTION 3 AGENCY AND ADVISORY COMMITTEE MEETINGS

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

Pharmacy Compounding Advisory Committee Meeting. February 23-24, 2015.  White Oak Campus, Silver Spring, MD.  The purpose of this meeting is to discuss proposed revisions to the agency's lists of bulk substances that may or may not be compounded under sections 503A and 503B of the Federal Food, Drug, and Cosmetic Act. [FDA.gov]

18th US – Japan Cellular and Gene Therapy Conference. February 26, 2015.  Molecular Imaging in Cellular Therapy.  White Oak Campus, Silver Spring, MD.  The goal of the conference is to exchange ideas on cutting edge areas of biomedical research and enhance opportunities for collaborations among scientists from the US and Japan. Molecular Imaging in Cellular Therapy is the broad theme of this year’s meeting. Six speakers each from Japan and the US will discuss the relevance of imaging in biology and medicine. [FDA.gov]

Public Advisory Committee Meeting. Science Board to the Food and Drug Administration. March 4, 2015.  White Oak Campus, Silver Spring, MD.  Among other agenda items, the Science Board will be provided with a progress or final draft report the Commissioner’s Fellowship Program Evaluation subcommittee and will hear a progress report from Science Moving Forward subcommittee. The Science Board will be asked to provide feedback on FDA’s public access policy. FDA will seek the Science Board’s input regarding approaches to regulatory science training coordination. [Federal Register]

Public Webinar. Overview of Medical Device Data Systems, General Wellness Devices, and Medical Device Accessories. February 24, 2015.  Webinar.  The webinar is intended to give an overview of FDA’s approach to regulating MDDS and to help stakeholders understand the draft policies described in the general wellness and medical device accessories guidance documents and solicit comments.  Following a brief presentation, the FDA will respond to questions regarding all four guidance documents. [FDA.gov]

Public Advisory Committee Meeting. Vaccines and Related Biological Products. March 4, 2015.  Silver Spring, MD.  The committee will meet in open session to discuss and make recommendations on the selection of strains to be included in the influenza virus vaccines for the 2015–2016 influenza season.  [Federal Register]

Public Conference. Serious Drug-Induced Liver Injury: The Importance of Getting It Right: How To Measure and Interpret Drug-Induced Liver Injury Information and Make Correct Diagnoses.  March 18-19, 2015.  Hyattsville, MD.  The purpose of the public conference is to discuss, debate, and share views among stakeholders in the pharmaceutical industry, academia, health care providers, patient groups, and regulatory bodies on how best to detect and assess the severity, extent, and likelihood of drug causation of liver injury and dysfunction in people using drugs for any medical purpose. [Federal Register]

New. Public Meeting. Supplemental Applications Proposing Labeling Changes for Approved Drugs and Biological Products. March 27, 2015.  White Oak Campus, Silver Spring, MD.  The purpose of the meeting is to provide a public forum for FDA to listen to comments on the proposed rule on ‘‘changes being effected’’ supplements that was published in the Federal Register of November 13, 2013, and alternatives offered to this proposed rule. [Federal Register]

Public Workshop. Gastroenterology Regulatory Endpoints and the Advancement of Therapeutics.  March 30-31, 2015.  White Oak Campus, Silver Spring, MD.  The purpose of this workshop is to provide a forum to consider issues related to selection of endpoints and clinical outcome measures appropriate for drug development in the following disease areas: Inflammatory bowel diseases and celiac disease. [Federal Register]

Public Meeting. Patient-Focused Drug Development for Breast Cancer. April 2, 2015.  White Oak Campus, Silver Spring, MD.  The public meeting is intended to allow FDA to obtain patient perspectives on the impact of breast cancer on daily life and patient views on treatment approaches. [Federal Register]

New.  Public Workshop. Assessment of Neurocognitive Outcomes in Inborn Errors of Metabolism and Advancing the Development of Pediatric Therapeutics: Assessment of Neurocognitive Outcomes. April 16-17, 2015.  White Oak Campus, Silver Spring, MD.  Day 1 of the workshop will focus on approaches for assessing the efficacy of therapeutic products based on neurocognitive outcomes in patients diagnosed with inborn errors of metabolism disorders. On Day 2 of the workshop, participants will discuss identification of signals in animal studies and clinical trials that warrant further clinical investigation and testing that may be predictive of neurocognitive outcome in children. [FDA.gov]

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

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


SECTION 4 OTHER REGULATORY AUTHORITIES & ORGANIZATIONS

Two New Studies Indicate the Venture Capital Growth in Orphan Drugs is Aided by Regulatory Certainty
Rare disease drug development is proving increasingly attractive to big pharma and private investors alike, with two new analyses highlighting the growing amount of venture capital being funneled into this space.

Venture capital funds have gravitated to companies focused on rare disease drug development due to regulatory certainty and development incentives for such products, according to separate studies by the Biotechnology Industry Organization and Jonathan Fleming, president and treasurer of the Network for Excellence in Health Innovation.

Investors in this space need only look at FDA’s record number of orphan novel drug approvals in 2014 – 17, or 41% of the novel drug and therapeutic biologic total – as reason to feel good about the regulatory prospects for such treatments. In addition, reimbursement prospects for orphan drugs carry less uncertainty, which is a critical factor in venture capital funds’ investment decisions, and there is less price-sensitivity for products intended to treat rare diseases with high unmet need.

The BIO study analyzed four databases encompassing $38 billion of venture capital into more than 1,200 U.S. drug companies over 10 years (2004-2013). It looked at funding by phase of development, disease and indication, modality, and level of novelty.

The choices made by venture capital firms about what compounds to invest in, which patient populations to study first, and how much money to invest are “profoundly influenced by uncertainty,” Network for Excellence in Health Innovation’s Fleming said at a Feb. 5 briefing. The briefing was sponsored by Health Affairs to highlight papers, including one by Fleming, in its February issue focused on biomedical innovation. Fleming, who also is managing partner of the venture capital firm Oxford Bioscience Partners, pointed to the orphan/rare disease area as one in which “the regulatory process is extremely straightforward and fixed. You pretty much know what you have to do to get approval there. That’s an attractive area for people because it’s not moving around a lot.”

For his study, Fleming used data from the PricewaterhouseCoopers/Money Tree database on venture capital investment to compare funding between 2009 and 2014 in life sciences companies, including biotechnology firms and medical device firms. During this period, investments in life sciences companies fell as a percentage of total VC investment, from 35.7% to 19.9%. “Clearly, there has been a shift away from life sciences to other industry sectors as well as a shift within the life sciences from early-stage to later-stage investments,” Fleming concluded. These later stages of development carry less risk and uncertainty around regulatory approval and reimbursement. “For people who are starting at the very beginning, all that uncertainty is really scary,” he said at the briefing.

In his paper, Fleming suggests some policy changes aimed at reducing uncertainty around regulatory approval and reimbursement decisions and which, in turn, would encourage investment in startup and life science projects, particularly in areas of great public health importance. More about both studies at title link above. (The Pink Sheet, paid subscription required)

PhRMA Foundation Launches New Web Platform to Support Researchers in Developing Life-Saving, Cost-Effective Medicines
On February 18, the Pharmaceutical Research and Manufacturers of America (PhRMA) Foundation announced the launch of its new platform, www.phrmafoundation.org, which features new resources on its competitive research grants and fellowships for young scientists in the most challenging areas of drug discovery.

The Foundation awards more than $3.3 million per year in annual grants to emerging researchers with support from PhRMA's member companies. Recent grants have focused on topics such as comparative effectiveness, adherence improvement and health outcomes. More than 170 research articles are housed on the new website, demonstrating the substantial research efforts of past award recipients.

"By enhancing the accessibility of our grants and fellowships, we hope to foster the careers of young scientists who are exploring a variety of challenging disciplines, such as cardiovascular disease, Alzheimer's and cancer," said Eileen Cannon, Executive Director of the Foundation. "The PhRMA Foundation is committed to investing in people, programs and partnerships to advance scientific research that will ultimately improve patient care and treatment options." More at link above. (PhRMA.org)

Gene Therapy Companies Experiment with New Payment Model
As U.S. drugmakers face growing resistance to the high price of cutting-edge treatments, a handful of companies are working on a new payment model that rewards them for the long-term performance of their medicines.  The effort, industry executives told Reuters, is being led by firms developing so-called gene therapies, which aim to cure inherited diseases like hemophilia by “fixing” the single faulty gene responsible for the disorder.

If these new hemophilia drugs and others like them succeed, a one-time infusion could replace the need for frequent, life-long injections of blood clotting proteins that can cost up to $300,000 a year for a single patient. These existing treatments are expected to command annual sales of more than $11 billion by 2016.

"The place that we are moving toward is more of a pay-for-performance type of strategy," said the CEO of one of the participating firms. Under this model, the price would be amortized over a period of time and contingent on proof that the treatment is effective and safe. The annuity-like payments would be stopped if medical testing, such as the level of clotting protein measured in a patient's blood sample, showed that the therapy was not working.

Many barriers remain to implementing such a model, and the drugs for which it is being considered may not reach the market for several more years, if at all. Since Americans often switch health insurers, contracts - or even legislation - would be needed to require payers to pick up the ongoing tab for patients who change their coverage.

But the interest in new payment models reflects the healthcare industry's intention to find new ways to bolster profits as insurers push back against drug prices. Some backers of the new model say the payment streams could eventually be packaged and sold to investors, as happens now with securities backed by financial assets like credit card receivables. More at link above.  (Reuters Press)

CVS Girds for Battle over New Cholesterol Drugs
Drug retail giant CVS Health Corp. is girding to do battle with pharmaceutical companies over a coming class of cholesterol drugs that CVS says could cost the nation’s health-care system $150 billion a year.

CVS, the nation’s biggest health-related company with nearly $140 billion in annual sales , says this new strata of cholesterol drugs, believed to be a cut above previous medications, would severely strain the health-care system if prescribed to all the 15 million Americans who could benefit from the drugs.

With annual costs running between $7,000 and $12,000 for the medication, CVS officials say the drugs -- known in the industry as PCSK9 inhibitors -- could weigh on the system more than a class of hepatitis C drugs put on the market in the past year and a half.  

“We believe that resiliency is about to be challenged in a manner unlike we have seen in the past, at least in the area of pharmaceuticals,” a group of CVS officials led by William Shrank, the company’s chief medical officer, wrote in a blog post for the trade magazine Health Affairs early Tuesday.

Shrank warns that while the hepatitis C drugs could also cost $150 billion, that would be spread out over a decade. But this class of cholesterol medicines could cost that much in a single year. With potential users numbering 15 million, that is five times the 3 million believed eligible for hepatitis C drugs.  

CVS’s concerns are overwrought, says Holly Campbell, spokeswoman for PhRMA, the drug industry’s trade group. She says the company is incorporating all potential users of the drug into the mix, with all paying list prices, with its $150 billion figure.

“That’s not actually what happens,” she said. Campbell says competition and market forces are bringing down the impact the price on these new cholesterol drugs. She says that with these cholesterol drugs, the competition will be at least as vigorous as with hepatitis C, if not more so. More at link above. (MarketWatch)

NIH Expands Key Tuberculosis Research Program
The National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, is expanding its Tuberculosis Research Units (TBRU) program in an effort to drive innovation in tuberculosis (TB) research. NIAID is awarding up to $15.2 million in fiscal year 2015 and as much as $105.3 million over seven years to fund four institutions that will act as a collaborative TBRU network.

A critical component of NIAID’s tuberculosis research, the TBRU program was established in 1994 with the aim of integrating basic and clinical research to study TB in countries where the disease is endemic. Although the number of individuals becoming sick or dying from TB worldwide has slowly declined in recent years, the global burden of the disease remains substantial. About one-third of the world’s population carries latent TB bacteria, according to the World Health Organization. In 2013, an estimated 9 million people became ill with TB and 1.5 million died from the disease.

During the past 15 years, a sustained effort in TB research has translated to a new pipeline of drug, vaccine and diagnostic candidates. In 2012, FDA approved the first new tuberculosis drug in more than 40 years. However, a limited understanding of the stages of TB disease has hampered transformation in optimal management of the disease.

By integrating epidemiology, immunology, and microbiology the four awardee institutions will work together to examine how Mycobacterium tuberculosis interacts with the human host and the immune system to cause disease. Through animal studies and clinical research, the TBRU program will focus on TB latency and persistence and their relation to active TB disease in humans.

New TBRU awards were made to Boston Medical Center, Boston (Biomarkers and Mechanisms of Paucibacillary and Latent Tuberculosis); Brigham and Women’s Hospital, Boston (Metabolic Factors that Control the Spectrum of Human Tuberculosis); Emory University, Atlanta (Role of Antigen-Specific T Cell Responses in the Control of TB); and Weill Cornell Medical College, New York City (Persistence and Latency).  More at link above. (NIH.gov)

NIH-supported researchers map epigenome of more than 100 tissue and cell types
Much like mapping the human genome laid the foundations for understanding the genetic basis of human health, new maps of the human epigenome may further unravel the complex links between DNA and disease. The epigenome is part of the machinery that helps direct how genes are turned off and on in different types of cells.

Researchers supported by the National Institutes of Health Common Fund’s Roadmap Epigenomics Program have mapped the epigenomes of more than 100 types of cells and tissues, providing new insight into which parts of the genome are used to make a particular type of cell. The data, available to the biomedical research community, can be found at the National Center for Biotechnology Information website.

Researchers from the NIH Common Fund’s Roadmap Epigenomics Program published a description of the epigenome maps in the journal Nature. More than 20 additional papers, published in Nature and Nature-associated journals, show how these maps can be used to study human biology. More at link above. (NIH.gov)

First US Chief Data Scientist Named by White House
The White House today (2/18/15) announced the appointment of Silicon Valley veteran DJ Patil to the position of chief data scientist and deputy chief technology officer for data policy, a new position that underscores the mainstream importance of big data with a strong focus on healthcare.

As Chief Data Scientist, Patil will help shape policies and practices to help the U.S. remain a leader in technology and innovation, foster partnerships to help responsibly maximize the nation’s return on its investment in data, and help to recruit and retain the best minds in data science to join us in serving the public.

Patil will also work on the Administration’s Precision Medicine Initiative, which focuses on utilizing advances in data and health care to provide clinicians with new tools, knowledge, and therapies to select which treatments will work best for which patients, while protecting patient privacy.

Patil joins the White House following an incredible career as a data scientist — a term he helped coin — in the public and private sectors, and in academia. Most recently, he served as the Vice President of Product at RelateIQ, which was acquired by Salesforce. He also previously held positions at LinkedIn, Greylock Partners, Skype, PayPal, and eBay. Prior to his work in the private sector, Patil worked at the Department of Defense, where he directed new efforts to bridge computational and social sciences in fields like social network analysis to help anticipate emerging threats to the United States.  (White House Press Release)

National Pharmaceutical Council to Discuss Results of 5th Annual Survey of CER in Healthcare Decision Making
The National Pharmaceutical Council (NPC) reports that every year since 2011, it has been keeping its finger on the pulse of health care stakeholders to gauge their opinions about comparative effectiveness research (CER) and its impact on health care decision-making. Through this annual survey, NPC says it has learned a lot about which organizations are perceived as influential in areas such as prioritizing, funding, conducting and monitoring CER, and about the status of other issues affecting the current health care environment.

To discuss the most recent survey results, NPC will host a webinar on March 26 featuring Kimberly Westrich, NPC vice president of health services research.  She will be joined by Joe Selby, MD, MPH, executive director, Patient-Centered Outcomes Research Institute; Lisa Simpson, MB, BCh, MPH, FAAP, president and CEO, AcademyHealth; and Dan Leonard, MA, president, NPC.  Registration information at link above.  (NPC)

Identification of medicines: EU task force to implement new international standards
EMA is establishing a task force for the implementation of international standards for the identification of medicinal products (IDMP) for human use in the European Union (EU). The Agency is inviting interested parties to express their interest in being part of the task force.

The IDMP standards developed by International Organization for Standardization (ISO) establish data elements, formats and terminologies for the unique identification of medicines and the exchange of information on medicines, including pharmaceutical dose forms, routes of administration, packaging and active substances.

These standards are expected to simplify the exchange of information between regulatory authorities across the world and to support healthcare authorities in the development of electronic health records. They should also improve the safety monitoring of medicines by facilitating the assessment of data across classes of medicines and therapeutic areas.

With the establishment of an ISO IDMP task force, EMA and the European medicines regulatory network intend to open a dialogue with all interested parties to discuss the implementation of the ISO IDMP standards in Europe. Interested parties need to submit their expressions of interest to the EMA by 6 March 2015. More at title link above. (EMA.eu)

Major European research project on monitoring and evaluation of medicines concludes
The European Medicines Agency (EMA) hosted and broadcasted a symposium on 19 and 20 February to mark the conclusion of the five-year Pharmacoepidemiological Research on Outcomes of Therapeutics by a European Consortium (PROTECT) project.

PROTECT’s goal is to strengthen the monitoring of the benefit-risk of medicines marketed in Europe by developing innovative methods. It is a public-private partnership of 34 European partners coordinated by EMA and GlaxoSmithKline and funded by the Innovative Medicines Initiative Joint Undertaking. Its partners include national medicines authorities, academic institutions and pharmaceutical companies.

The symposium brought together the results and key recommendations of PROTECT and discussed how the findings can be applied to improve the monitoring and evaluation of the benefits and risks of medicines in Europe.

PROTECT results may significantly influence practices in pharmacovigilance and benefit-risk evaluation. Therefore, during 2015, EMA will systematically scrutinise PROTECT’s research outputs in order to identify priority results that are robust and which if implemented, have the greatest potential to positively impact public health.  More at link above. (EMA.eu)

Safety signals: recommendations now available in all EU languages
The European Medicines Agency (EMA) has started to translate its recommended changes to product information based on the assessment of safety signals into all official languages of the European Union (EU). The translations should be used by pharmaceutical companies to update the product information of their medicines.

This initiative is expected to accelerate the implementation of changes to product information and to ensure consistency across EU countries, thus leading to better information for patients on their medicines. More at link above. (EMA.eu)

Pharma and NICE Reject View that NHS Pays too Much for Meds
The NHS is paying too high a price for new medicines and the Cancer Drugs Fund represents particularly poor value.

These are the key claims to come out of research from the University of York which claims that the threshold used by the National Institute for Health and Care Excellence when gauging the cost-effectiveness of new drugs is too high. This means that their approval at this level “is doing more harm than good to NHS patients overall”.

The analysis notes that currently NICE uses a threshold of £30,000 per Quality Adjusted Life Year (QALY) but claims this should be £13,000 to provide most benefit across the NHS. The present level is leading to increased mortality in cancer, circulatory, respiratory or gastro-intestinal diseases and reduced quality of life in neurological diseases and mental health, the researchers argue.

Co-author Karl Claxton said that “the increasing pressure to approve new drugs more quickly at prices that are too high will only increase the harm done to NHS patients overall”. He added that “the political pressure to support a multinational pharmaceutical sector cannot justify the real harm that has and will continue to be done to NHS patients”.

Sir Andrew Dillon, NICE’s chief executive, said that Prof Claxton’s work is important “because it brings to life the dry concepts of cost effectiveness thresholds and opportunity costs. It shows that when you decide to move with the cutting edge of medicine, that there’s a price to pay, and with his work, we have one researcher’s views on who’s paying it”.

He added that “over the last 16 years, we think we’ve found a balance that reflects what the public expect the NHS to do”. Sir Andrew suggested that the £20,000-£30,000 per QALY “represents a reasonable compromise between ensuring everyone has fair and equitable access to the NHS and enabling access to new and innovative treatments”.  More at link above. (PharmaTimes)

WHO Urges Developing Countries to Fund Tropical Diseases Fight
The World Health Organization called on developing countries on Thursday to invest $1 per person per year until 2030 to tackle 17 neglected tropical diseases (NTDs) and improve the health and well-being of more than 1.5 billion people.

Forecasting a total of $34 billion needed to fight NTDs for the next 16 years, the WHO said governments whose people are blinded, disfigured and killed by such diseases should recognize the great potential human and economic return on tackling them.

The investment would represent as little as 0.1 percent of current national health spending of the low and middle-income countries affected by NTDs, the WHO said, and could also encourage international donors to increase aid.

NTDs such as river blindness, rabies, guinea worm and elephantiasis cause disfigurement, disability and death among millions of poor people in developing countries. Also among the 17 being targeted by the WHO are Human African trypanosomiasis, or sleeping sickness -- a parasitic infection spread by tsetse flies that is almost 100 percent fatal without prompt diagnosis and treatment -- and dengue, sometimes known as "breakbone fever" -- a mosquito-borne disease that in its severe form can cause lethal complications.  More at link above. (Reuters Press)


SECTION 5 LEGAL AND COMPLIANCE

HHS Secretary Burwell to Testify February 26
The US House Energy & Commerce Committee, Subcommittee on Health, chaired by Rep. Joe Pitts (R-PA), has scheduled a hearing for Thursday, February 26, entitled, “Examining the FY 2016 HHS Budget.” Health and Human Services Secretary Sylvia Burwell will be the sole witness. Subcommittee members will question Secretary Burwell regarding the Health and Human Services budget, the second open enrollment period for the president’s health care law, the status of the backend of the exchanges, and the administration’s planning for the upcoming Supreme Court decision in the case of King v. Burwell. More at link above. (Energy & Commerce)

PLOS Published Study: Drug Makers Violate Voluntary Marketing Codes Regularly
Despite voluntary codes on marketing practices, drug makers in the U.K. and Sweden regularly violate the codes at least once a week, on average, according to a new study. And the study authors suggest that the findings indicate the pharmaceutical industry is unable to sufficiently police itself and that penalties may be inadequate.

The study, which was published in PLOS Medicine, analyzed voluntary codes along with information taken from oversight entities – such as complaints and rulings – between 2004 and 2012. In total, the Swedish and U.K. oversight groups found 536 and 597 cases, respectively, breached the codes, which worked out to more than one violation per week.

In both countries, a majority of the violations stemmed from misleading promotions about drug effectiveness. In some instances, violations involved marketing medicines to consumers, which is prohibited in the European Union. Nearly 20% of the cases were deemed to be serious breaches of voluntary codes and 46 drug makers were found to have committed a serious breach at least once.

“The prevalence and severity of breaches testifies to a discrepancy between the ethical standard approved by companies and codified in industry codes of conduct and the actual conduct of the industry,” the study authors write. But they argue that simply improving regulatory oversight and increasing penalties is not likely to improve practices and reduce the number of violations.

Instead, the authors suggest various regulatory reforms, such as reviewing promotional materials prior to use; intensify active monitoring of promotions; increase fines; and introduce other sanctions, such as greater publicity following adverse rulings. “These help to improve the quality of medicines information for health care professionals and the public,” they argue (you can read the complete study here).  More at link above. (WSJ Pharmalot)

Minnesota doctors seek to cut insurer red tape over prescriptions
Unable to make peace on their own with Minnesota’s health insurers, the state’s doctors are pressing legislation that would make it easier for them to overcome “prior authorization” insurance hurdles when prescribing drugs to patients.

While acknowledging that insurance restrictions play a role in preventing waste and drug misuse, leaders of the Minnesota Medical Association said Thursday that many patients are denied their medications or preferred brands with no discernible reasons.

And state Sen. Melisa Franzen, DFL-Edina, said doctors waste thousands of hours and millions of dollars each year haggling with insurers over prescriptions held up by prior authorization. “While intended as a way to ensure high-quality, cost-effective medicine, it is often getting in the way,” she said.

The bipartisan bill, introduced by Franzen and Rep. Tony Albright, R-Prior Lake, has long been expected. The medical association made prior authorization its top legislative target this year. More at link above. (Press)


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.