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Content Currents

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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: March 27, 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final guidances for which CDRH would appreciate external feedback on whether these final guidances should be revised or withdrawn.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 March 23, 2015, FDA announced the availability of a guidance entitled “Electronic Submission of Lot Distribution Reports” The 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. The guidance announced in this notice finalizes the draft guidance entitled ‘‘Guidance for Industry: Electronic Submission of Lot Distribution Reports’’ dated August 2014, and is intended to help licensed manufacturers of products distributed under an approved BLA (henceforth referred to as applicants) comply with the final rule.  [Federal Register]

On March 24, 2015, FDA announced the availability of a guidance for industry entitled “Determining the Need for and Content of Environmental Assessments for Gene Therapies, Vectored Vaccines, and Related Recombinant Viral or Microbial Products.” The guidance document provides investigational new drug application (IND) sponsors and applicants for a biologics license application (BLA) or a supplement to a BLA (BLA supplement), with recommendations on considerations when assessing whether to submit an Environmental Assessment (EA) for gene therapies, vectored vaccines, and related recombinant viral or microbial products (GTVVs). The guidance also contains recommendations as to what information should be included in an EA and what you can expect once an EA is filed. The guidance announced in this notice finalizes the draft guidance of the same title dated June 2014. [Federal Register]

On March 27, 2015, FDA announced an opportunity for public comment on the proposed collection of information concerning guidance for industry on “Establishment and Operation of Clinical Trial Data Monitoring Committees.” The DMC advises the sponsor regarding the continuing safety of current trial subjects and those yet to be recruited to the trial, as well as the continuing validity and scientific merit of the trial. The guidance document referenced in this document is intended to assist sponsors of clinical trials in determining when a DMC is needed for monitoring a study, and how such committees should operate. The guidance addresses the roles, responsibilities, and operating procedures of DMCs, describes certain reporting and recordkeeping responsibilities.  Comments should be submitted by May 26, 2015.  [Federal Register]


SECTION 2 FDA NOTES & RELATED NEWS

FDA Continues to Lead in Precision Medicine
In her FDA Voice blog, Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research, discusses FDA’s involvement in encouraging the development of personalized or precision medicines, or targeted drug therapies, since the 1990’s.  Beginning the with approval of Herceptin in 1998, the development of targeted therapies has grown rapidly, she says.  “…CDER approved 30 targeted therapies since 2012, including Kalydeco (ivacaftor), a targeted drug for cystic fibrosis. In 2014 alone, eight of the 41 novel drugs approved were targeted…”  More at link above. (FDA.gov)

Report:  Assessing CDER's Drug Safety-Related Regulatory Science Needs and Identifying Priorities
FDA announced the availability of this report on March 19, 2015. The report identifies drug safety-related regulatory science needs and priorities related to the mission of FDA's Center for Drug Evaluation and Research (CDER) that would benefit from external collaborations and resources. FDA hopes to foster collaborations with external partners and stakeholders to help address these needs and priorities. This notice asks stakeholders conducting research related to these needs to describe that research and indicate their interest in collaborating with FDA to address safety-related research priorities.  

There are seven areas highlighted in the report that FDA believes would benefit from internal and/or external collaboration:

  1. Improve access to postmarket data sources and explore the feasibility of their use in safety signal analyses
  2. Improve risk assessment and management strategies to reinforce the safe use of drugs
  3. Evaluate the effectiveness of risk communications of drug safety information to health care providers and the public
  4. Improve product quality and design, manufacturing processes, and product performance relating to safety 
  5. Develop and improve predictive models of safety in humans, including nonclinical biomarkers
  6. Improve clinical trial statistical analyses for safety, including benefit-risk assessment
  7. Investigate clinical biomarkers of safety, including standards for qualification
More at link above. (FDA.gov)

From New Jersey to New Delhi, a global focus on quality

It’s no secret there have been challenges associated with the quality of generic drugs coming out of some facilities in India. Some people have asked if the FDA is “singling out” India for increased inspections. FDA simply replies that increased exports to the U.S. result in increased inspection, no matter where you are in the world. FDA inspections ensure that when a firm wants to export drugs to the United States, the drugs meet FDA standards and will be of the quality patients and consumers want and deserve.

In the March 24, 2015, FDA Voice blog, Howard Sklamberg, FDA Deputy Commissioner for Global Regulatory Operations and Policy, and Cynthia Schnedar, J.D., Director of the Office of Compliance at FDA CDER, talk about their recent visit to India to meet with their regulatory counterparts in the Indian government and with drug manufacturers with facilities in India.  

They write: “The inspections associated with drug production have been a central discussion point on this trip, and we’ve brought news that has been well-received, especially by the drug industry. We shared our proposed plan to create a new approach to facility inspections, one that will not only note problems, but will also allow our inspectors to document where a firm’s quality management system exceeds what would be required to meet regulatory compliance. To put it simply: the inspections can yield also carrots, and not just sticks.”  More at link above. (FDA.gov)

FDA Responds to Alexander Inquiry About Draft Guidances
Responding to the concerns expressed by four US Senators that the FDA takes too long to issue a final guidance, prompting concerns that a draft guidance becomes “default policy,” FDA responded earlier this month with a 12-page explanation of how the Agency views guidance documents and the guidance development process. The FDA response, which attached a list showing how many draft guidances are still pending, noted the agency is “continually” seeking to improve the process for developing guidances, no specific estimates were offered for cutting the actual amount of time that may elapse between draft and final guidances.  More at links above.  (WSJ Pharmalot)

The FDA and the Bonn Call for Action: Update on the Initiative to Reduce Unnecessary Radiation Exposure from Medical Imaging
In December 2012, the International Atomic Energy Agency sponsored, and the World Health Organization co-sponsored an International Conference on Radiation Protection in Medicine: Setting the Scene for the Next Decade” in Bonn, Germany. The meeting was attended by 536 participants from 77 countries and 16 organizations, including FDA. The conference resulted in a list of priorities for radiation protection in medicine for the next decade, called the Bonn Call for Action.

The Bonn Call for Action is divided into ten principal actions, each of which is considered essential for strengthening radiation protection over the next decade. As a regulatory agency, FDA shares in the responsibility for strengthening radiation protection of patients and health workers with other national and international agencies, researchers, educators, medical institutions, professional societies and individual practitioners.

Some of the actions proposed by the Bonn conference are outside of FDA’s role and authority. Many of the actions, however, are areas where FDA has made efforts and contributions. The update lists selected actions and sub-actions where FDA is participating actively to implement the Bonn Call for Action. Among these are:

  • Enhance the implementation of the principle of optimization of protection and safety (through development and application of technological solutions for patient exposure records, harmonization of the dose data formats provided by imaging equipment, and increased utilization of electronic health records)
  • Strengthen manufacturers’ role in contributing to the overall safety regime (through ensuring improved safety of medical devices by enhancing the radiation protection features in the design of both physical equipment and software and to make these available as default features rather than optional extra features) and (strengthening cooperation and communication between manufacturers and other stakeholders, such as health professionals and professional societies)
  • Foster an improved radiation benefit-risk-dialogue.  

More at link above.  (FDA.gov)


SECTION 3 AGENCY AND ADVISORY COMMITTEE MEETINGS

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

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 Workshop. FCC/FDA Joint Workshop on Wireless Medical Device Test Beds.  March 31, 2015.  FCC Commission Meeting Room, Washington, DC.  Federal Communications Commission (FCC) and the FDA will host a public workshop on the role of wireless medical test beds and their influence on the development of converged medical technology for clinical and non-clinical settings. A wireless test bed is an environment where devices can be evaluated across a range of interference scenarios. The FCC and FDA encourage the participation of a broad range of stakeholders, including device manufacturers, health care facilities and clinicians, test labs, standard-setting bodies, innovators, patient safety groups, researchers, and entrepreneurs, among others. [FDA.gov]

Public Workshop. Clinical Outcomes Assessment Development and Implementation: Opportunities and Challenges. April 1, 2015.  White Oak Campus, Silver Spring, MD.  The purpose of the public workshop is to provide updates on accomplishments, challenges, and ongoing efforts in the use of clinical outcome assessments (COAs), and plan for the future of COA development and utilization in drug development programs, including how to incorporate the patient voice in drug development using well-defined and reliable patient-centered outcome measures. [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. More information and materials as they become available at  Breast Cancer Patient-Focused Drug Development    [Federal Register]

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 Advisory Committee Meeting. Neurological Devices Panel of the Medical Devices Advisory Committee. April 17, 2015.  White Oak Campus, Silver Spring, MD.  The committee will discuss the current knowledge regarding the conduct of clinical studies and evaluation of clinical study data for flow diverter technology. FDA is convening this committee to seek expert opinion on scientific and clinical considerations relating to the study design and existing clinical studies, for flow diverter technology indicated for the neurovasculature. [Federal Register]

New.  Public Workshop. Ninth Annual DIA/FDA Statistics Forum. April 20-22, 2015.  Bethesda, MD.  The purpose of the conference is to discuss relevant statistical issues associated with the development and review of therapeutic drugs and biologics. A primary focus for this meeting will be to establish an ongoing dialogue regarding FDA’s ‘‘Critical Path’’ initiative— emphasizing the regulatory and statistical challenges associated with innovative approaches to the design and analysis of clinical trial data and measuring the progress being made in designing and implementing innovative solutions.  [Federal Register]

New.  Public Hearing. Homeopathic Product Regulation: Evaluating FDA’s Regulatory Framework After a Quarter-Century.  April 20-21, 2015.  White Oak Campus, Silver Spring, MD.  The purpose of the meeting is to obtain information and comments from stakeholders about the current use of human drug and biological products labeled as homeopathic, as well as the Agency’s regulatory framework for such products. These products include prescription drugs and biological products labeled as homeopathic and over-the-counter (OTC) drugs labeled as homeopathic. FDA is seeking participants for the public hearing and written comments from all interested parties. [FDA.gov]

Public Workshop. An Interactive Discussion on the Clinical Considerations of Risk in the Postmarket Environment.  April 21, 2015.  White Oak Campus, Silver Spring, MD. The purpose of this workshop is to provide a forum for an interactive discussion on assessing changes in medical device risk as quality and safety situations arise in the postmarket setting when a patient, operator, or member of the public uses the device. FDA has also opened a docket for comments on this topic (due May 19, 2015). [Federal Register]

Public Workshop. 2015 Office of Regulatory Science and Innovation Science Symposium. April 27, 2015.  White Oak Campus, Silver Spring, MD.  The purpose of the public workshop is to increase scientific collaborations with government institutions, academia, industry and other stakeholders, working to improve science, training, and networking in accordance with the FDA mission of the advancement of regulatory science. This venue will also enhance knowledge and awareness of the FDA ORSI resources and provide guidance of its available services. [Federal Register]

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 Advisory Committee Meeting. Ear, Nose, and Throat Devices Panel of the Medical Devices Advisory Committee. April 30 – May 1, 2015.  Gaithersburg, MD.  On April 30, 2015, the committee will discuss and make recommendations regarding the classification of Hearing Protectors, Circumaural Hearing Protectors, Middle Ear Inflation Devices, Tactile Hearing Aid Devices, and Vestibular Analysis Apparatuses.  On May 1, 2015 the committee will discuss key issues related to a potential pre- to post-market shift in clinical data requirements for modifications to cochlear implants in pediatric patients. [Federal Register]

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]

Public Advisory Committee Meeting. Vaccines and Related Biological Products Advisory Committee.  May 12, 2015.  White Oak Campus, Silver Spring, MD. The committee will meet in open session to discuss the development and licensure of Ebola vaccines. [Federal Register]

Public Advisory Committee Meeting. Gastroenterology and Urology Devices Panel of the Medical Devices Advisory Committee. May 14-15, 2015.  White Oak Campus, Silver Spring, MD.  FDA is convening this committee to seek expert scientific and clinical opinion related to reprocessing of duodenoscopes and other endoscopes, as well as automated endoscope reprocessors, based on available scientific information. [Federal Register]

Public Hearing. Regulatory Science Initiatives under the Generic Drug User Fee Amendments of 2012.  June 5, 2015.  White Oak Campus, Silver Spring, MD.  This public hearing will provide an overview of the current status of regulatory science initiatives for generic drugs and an opportunity for public input on research priorities in this area. FDA is seeking this input from a variety of stakeholders—industry, academia, patient advocates, professional societies, and other interested parties—as it fulfills its commitment under the Generic Drug User Fee Amendments of 2012 (GDUFA) to develop an annual list of regulatory science initiatives specific to generic drugs. FDA will take the information it obtains from the public hearing into account in developing the fiscal year (FY) 2016 Regulatory Science Plan.  [Federal Register]

Public Workshop. Robotically-Assisted Surgical Devices: Challenges and Opportunities. July 27-28, 2015.  White Oak Campus, Silver Spring, MD.  FDA is holding this public workshop to obtain information on the current challenges and opportunities related to robotically- assisted surgical medical devices, which are classified as Class II medical devices. The purpose of this workshop is to obtain public feedback on scientific, clinical, and regulatory considerations associated with RAS devices.  [Federal Register]


SECTION 4 OTHER REGULATORY AUTHORITIES & ORGANIZATIONS

Obama Administration Releases National Action Plan to Combat Antibiotic-Resistant Bacteria
Today, the White House released a comprehensive plan that identifies critical actions to be taken by key Federal departments and agencies to combat the rise of antibiotic-resistant bacteria.  The National Action Plan for Combating Antibiotic-Resistant Bacteria, which was developed by the interagency Task Force for Combating Antibiotic-Resistant Bacteria in response to Executive Order 13676: Combating Antibiotic-Resistant Bacteria, outlines steps for implementing the National Strategy on Combating Antibiotic-Resistant Bacteria and addressing the policy recommendations of the President’s Council of Advisors on Science and Technology (PCAST) report on Combating Antibiotic Resistance.

The National Action Plan provides a roadmap to guide the Nation in rising to the challenge of antibiotic resistance and potentially saving thousands of lives.  The Action Plan outlines Federal activities over the next five years to enhance domestic and international capacity to prevent and contain outbreaks of antibiotic-resistant infections; maintain the efficacy of current and new antibiotics; and develop and deploy next-generation diagnostics, antibiotics, vaccines, and other therapeutics.  These activities are consistent with investments in the President’s FY 2016 Budget, which nearly doubles the amount of Federal funding for combating and preventing antibiotic resistance to more than $1.2 billion. More at title link above. (WhiteHouse.gov)

Health Information Management Systems Society to Focus on Transforming Health
Chicago welcomes the 2015 HIMSS Annual Conference & Exhibition, April 12-16, 2015, at McCormick Place. More than 38,000 healthcare industry professionals are expected to attend to discuss health IT issues and view innovative solutions designed to transform healthcare.

DIA is proud to support this annual event that helps health IT professionals find the right solutions for their organizations.
Conference education sessions include preconference symposia on clinical and business analytics, HIEs, innovation, mobile health, nursing informatics, physicians’ IT and more. More than 300 peer-reviewed sessions, including workshops and roundtables, round out education offerings at HIMSS15.

George W. Bush, 43rd President of the United States, leads a strong roster of speakers that also includes Alex Gourlay, President, Walgreens; Bruce  D. Broussard, President and CEO, Humana; and Jeremy Gutsche, Founder of Trendhunter.com and Author of “Exploiting Chaos.”

The HIMSS15 Exhibit Hall will feature the Connected Patient Gallery, Federal Health IT Solutions Pavilion, HIMSS First-Time Exhibitors Area, HIMSS Health IT Value Suite, HIMSS Interoperability Showcase™, Intelligent Health™ Pavilion (formerly known as Intelligent Hospital Pavilion), and three Knowledge Centers focused on clinical and business intelligence, disaster preparedness and mobile health.

As a HIMSS15 Endorser, DIA members are eligible to receive the member discount to attend the conference. To receive the discount, go to the online registration at www.himssconference.org/registration, and select DIA from the “Conference Endorsing Organizations” drop-down in the registration process. Enter Endorser Number “2015”.  More information at title link above. (HIMSS.org)

Alzheimer's Association Releases "2015 Alzheimer's Disease Facts and Figures"
On March 24, the Alzheimer’s Association released the report, “2015 Alzheimer’s Disease Facts and Figures.”  The report states that today, an estimated 1 in 9 older Americans has Alzheimer’s disease, about 5.3 million Americans in total.  Cases among those 65 and older number about 5.1 million, while 200,000 individuals under age 65 have a younger onset form of the disease. Alzheimer’s is now the 6th leading cause of death in the US and the only cause in the top 10 in the US that cannot be prevented, cured, or slowed.  The current and projected costs of care are detailed in the report, as are areas of active research in detection, diagnosis, and treatment.  More at link above.  (Alzheimer’s Association)

Academia, the NHS and industry collaborate on 100,000 genomes project
AstraZeneca, GlaxoSmithKline and Roche are among the first 10 biopharma companies that will collaborate with academia and NHS Genomic Medicine Centres on the 100,000 Genomes Project, which aims to map 100,000 genomes from patients with rare diseases or cancer by 2017.

The companies* have formed the GENE Consortium (Genomics Network for Enterprises Consortium), which will oversee work on a year-long industry trial aiming to identify the most effective and secure way of bringing industry expertise into the Project “in order to realise the potential benefits for patients”, providing a platform for future work.

Under the collaboration, industry partners will each contribute seed funding and scientists to work on the project, who will have access to information from 5,000 whole genomes collected from the first stage of the Project “in full compliance with data protection laws,” AZ noted. Ultimately, it is hoped that the unique partnership will transform treatment for patients with rare diseases and cancer, with faster access to the right treatment and personalised care.

* Members of the GENE Consortium are: AbbVie, Alexion, AstraZeneca, Biogen, Dimension Therapeutics, GSK, Helomics, Roche, Takeda and UCB.  More at link above. (PharmaTimes)

NIH and Columbia University Establish Study Results Tracking Site
A new tool has been developed by researchers at NIH and Columbia University to help people track published studies related to specific clinical trials.  Called “ArticlesAboutMe,” this service sends an email to patients who participated in a clinical study when an article with results of the trial has been published.  More at link above.  (ArticlesAboutMe.org)

New NIMH strategic plan aims to focus, accelerate mental health research
The National Institute of Mental Health (NIMH) has issued a new Strategic Plan for Research. Informed by the successes and challenges of recent years, the new plan updates the strategic objectives of its 2008 predecessor with the aim of balancing the need for long-term investments in basic research with urgent mental health needs.  According to recent estimates mental illnesses account for 21.3 percent of all years lived with disability in the United States.

The plan has revised the original four high-level strategic objectives as follows:

  1. Define the mechanisms of complex behaviors.
  2. Chart mental illness trajectories to determine when, where, and how to intervene.
  3. Strive for prevention and cures.
  4. Strengthen the public health impact of NIMH-supported research.

These four objectives form a broad roadmap for the institute’s research priorities over the next five years.  More at title link above. (NIH.gov)

NIH selects awardees to help speed development of health technologies
The National Institutes of Health has selected three new proof-of-concept hubs to help speed the translation of basic biomedical discoveries into commercial products, such as new drugs, devices, and diagnostics, to improve patient care and enhance health. The hubs are part of the NIH-supported Research Evaluation and Commercialization Hubs (REACH) program and will be funded at $9 million over three years.

REACH is based on an initiative created by the NIH’s National Heart, Lung, and Blood Institute (NHLBI) called the NIH Centers for Accelerated Innovations (NCAI). These centers are a unique public-private partnership whose objective is to change how to identify and develop innovations with scientific and commercial potential. The effort utilizes industry-style project management to arrive at technologies that are poised to launch. With the addition of the REACH, NIH has created a nationwide network of six centers and hubs to develop best practices in translating academic innovations into products to improve health.

The hubs will foster the development of therapeutics, preventatives, diagnostics, devices, and tools that address diseases within the NIH’s mission in a manner consistent with business case development. Each hub will provide funding for feasibility studies and coordinated access to expertise in areas required for early stage technology development, including scientific, regulatory, reimbursement, business, legal, and project management. In addition, the hubs will provide skills development and hands-on experience in entrepreneurism.

The new REACH awardees are:

  • The Long Island Bioscience Hub: Stony Brook University, Cold Spring Harbor Laboratory, Brookhaven National Laboratory, New York
  • The University of Louisville, Kentucky
  • The University of Minnesota, Minneapolis

More at title link above.  (NIH.gov)

Study Demonstrates Success of NCATS’ Rare Diseases Therapeutic Development Programs
Researchers at MIT and NIH conducted at financial analysis of rare disease projects developed through the NCATS (National Center for Advancing Translational Sciences) Therapeutics for Rare and Neglected Diseases (TRND) and Bridging Interventional Development Gaps (BrIDGs) programs.  The study, published Feb. 25, 2015, in Science Translational Medicine, found that the scientific and operational processes utilized in TRND and BrIDGs projects led to reduced cost of developing new drugs, reduced financial risks, and effectively provided a way to develop promising therapeutics to the point where they could be handed off to the private sector for final testing and marketing.

TRND and BrIDGs projects’ lower cost and greater success rates were accompanied by longer pre-clinical development times than the industry average. This result was not unexpected, because the programs take a methodical, step-by-step ‘sequential approach’ that reduces drug development costs.

The sequential approach differs from the industry standard, which undertakes multiple investigations into a new drug simultaneously. This method moves a successful therapeutic more quickly through the pipeline to market, but it can also cost more if a drug fails, because there is more up-front investment.  More at link above.  (NIH.gov)

CMS to emphasize clinical utility of prognostic cancer tests
At Tuesday's meeting of the Medicare Evidence Development and Coverage Advisory Committee (MEDCAC) to discuss prognostic tests for cancer, CMS's James Rollins said the agency will rely more heavily on clinical utility, in addition to analytical and clinical validity, when evaluating coverage of molecular pathology tests. Rollins is director of CMS's division of items and devices.

The committee expressed "intermediate confidence" that existing evidence is sufficient to support clinical utility of the Oncotype DX breast cancer assay from Genomic Health Inc.. The average vote of the 8-member panel was 2.9 on a scale of 1-5, where 1 is "low confidence" and 5 is "high confidence."

The panel found insufficient evidence for the other nine tests to support clinical utility. The committee reviewed Genomic Health's Oncotype DX colon cancer assay and tests for BRAF and K-Ras (KRAS) mutations, microsatellite instability and DNA mismatch repair protein Mlh1 (MLH1) promoter methylation in patients with colorectal cancer. The committee also considered breast cancer tests including MammaPrint from Agendia N.V. (Amsterdam, the Netherlands) and NSCLC tests including those for anaplastic lymphoma kinase (ALK), EGFR and KRAS mutations.

Panelists generally sought additional data on the effect of molecular pathology tests on treatment decisions and health outcomes.

The committee also discussed the reliability of evidence of laboratory-developed tests (LDTs) compared to FDA-approved tests. Two panelists expressed higher confidence in FDA-approved tests because they go through a standard review process and have a system in place for adverse event reporting. More at link above. (BioCentury)

Deloitte: Life science companies preparing for next wave
Professional services firm Deloitte recently released its '2015 Global Life Sciences Outlook' report, titled ‘Adapting in an era of transformation’, in which it examines the current state of the life sciences sector; describes trends impacting markets and organisations; and suggests considerations for stakeholders as they seek to grow revenue and market share.

This year’s edition of the life sciences outlook report shows that four trends are expected to influence the healthcare industry in 2015: searching for innovation and growth; changing regulatory and risk environment; preserving and building shareholder value; and preparing for the ‘next wave.’

Life sciences companies are adapting their business models to take advantage of the ‘next wave’ of opportunities in the industry, with the ability to rapidly adopt and commercialise new technological and clinical discoveries becoming increasingly essential to gain a competitive advantage in a transforming marketplace. The researchers see opportunities for life sciences companies that embrace the ‘digital agenda’ and use technology, big data, and analytics to advance product development, such as personalised medicine, and care delivery.

As part of this ‘new wave’, healthcare is transitioning from volume-based to value-based care. As a result of this transition, comparative effectiveness research* (CER) is becoming a major factor in a treatment’s market uptake, with products not proved to be comparatively effective expected to struggle to generate demand or attain reimbursement.

Deloitte concludes by saying that life science companies will need to develop CER capabilities to avoid exclusions and sales losses, and to gain the advantages of the ‘new wave’ opportunities.

PCORnet Health Systems Demonstration Project PCORI Funding Announcement Development
The Patient-Centered Outcomes Research Institute (PCORI) at its March 24, 2015, Board of Governors meeting, announced it is developing a Funding Announcement for $5 million in funding for a project to demonstrate the ability of its "network of networks," known as PCORnet, to engage health system leaders and clinicians to conduct relevant research.

In the first step, it will provide $1 million to enable sites within PCORnet partner networks to work with health system leaders to identify and prioritize a set of data-driven research activities of high interest to those organizations and their clinicians. It will then provide up to $4 million to fund up to five one-year research studies on those priority topics.

PCORnet aims to harness the power of health data collected from sources such as electronic health records and patients' routine interactions with their healthcare providers to inform research. That requires the cooperation of the health systems where that care takes place.

The institute plans to release requirements for a limited competition in May, with applications due in June. More at link above. (PCORI.org)

PCORI Executive Director Joe Selby on the National Pharmaceutical Council's Fifth Annual Comparative Effectiveness Research Survey
PCORI Executive Director Joe Selby, MD, MPH, issued the following statement about today's release of the National Pharmaceutical Council’s fifth annual comparative effectiveness research survey.

“With more than 90 percent of respondents agreeing that comparative effectiveness research (CER) is important for our nation’s healthcare, the National Pharmaceutical Council’s fifth annual CER survey reaffirms the need for evidence that helps patients and those who care for them make better informed decisions.

“We’re pleased that those surveyed identified PCORI as the clear leader in setting CER priorities, establishing research standards, and funding studies.  All of our initiatives – including our pragmatic clinical studies initiative and our efforts to build PCORnet, a national patient-centered clinical data research network – aim to generate real-world evidence about what works best for which patients. We’ll begin to see the first results of the CER studies we’ve funded later this year. We look forward to working with our many partners to disseminate and implement the results.  More at link above.  (PCORI.org)

National Pharmaceutical Council Releases 5th Annual Survey on 2015 Comparative Effectiveness Research and the Environment for Health Care Decision-Making
The National Pharmaceutical Council’s (NPC) fifth annual survey, "Comparative Effectiveness Research (CER) and the Environment for Health Care Decision-Making," provides a snapshot of stakeholders’ perceptions of the key players in the main aspects of the CER process, from setting priorities to translating and disseminating the research findings.

Among the findings: CER’s importance in the health care landscape was clearly recognized by stakeholders, with 92% of respondents saying it is “very important” or “somewhat important,” which is consistent with their responses in previous years. Although these respondents view CER as important, its impact has not yet been felt. “Moderate” or “substantial improvement” in health care decision-making because of CER won’t likely be felt for another three (83%) to five years (93%). More at link above. (National Pharmaceutical Council)

Informed Consent and Health Literacy: Workshop Summary
The Institute of Medicine Roundtable on Health Literacy held a one-day workshop to explore what actions can be taken to help close the gap between what is required in the informed consent process and communicating it in a health-literate and meaningful manner to individuals.  

Informed consent - the process of communication between a patient or research subject and a physician or researcher that results in the explicit agreement to undergo a specific medical intervention - is an ethical concept based on the principle that all patients and research subjects should understand and agree to the potential consequences of the clinical care they receive. Regulations that govern the attainment of informed consent for treatment and research are crucial to ensuring that medical care and research are conducted in an ethical manner and with the utmost respect for individual preferences and dignity. These regulations, however, often require - or are perceived to require - that informed consent documents and related materials contain language that is beyond the comprehension level of most patients and study participants.

Topics covered in this workshop included an overview of the ethical imperative to gain informed consent from patients and research participants, a review of the current state and best practices for informed consent in research and treatment, the connection between poor informed consent processes and minority underrepresentation in research, new approaches to informed consent that reflect principles of health literacy, and the future of informed consent in the treatment and research settings. Informed Consent and Health Literacy is the summary of the presentations and discussion of the workshop.  Report available at link above. (IOM)

European trade groups seek lower barriers for IPOs
The European IPO Task Force, a group assembled by three financial trade organizations, issued a report proposing ways to make European public markets more accessible to smaller companies. The proposals echo those enacted in the U.S. via the Jumpstart Our Business Startups (JOBS) Act in 2012.

The report recommends reforming the regulatory environment for small and medium "emerging growth companies," including revising EU financial legislation to cut administrative costs of listing by 30-50%. The report also calls for measures to increase venture capital and retail investors' access to markets, to improve communication between European companies and their investors pre- and post-IPO, and to encourage investments into IPOs and equity markets via tax incentives.

The task force was established last year by EuropeanIssuers, the European Private Equity and Venture Capital Association and the Federation of European Securities Exchanges. (BioCentury)


SECTION 5 LEGAL AND COMPLIANCE

SEC updates JOBS Act
The SEC adopted final changes to Title IV of the Jumpstart Our Business Startups (JOBS) Act of 2012 that it says will make it easier for smaller companies to raise capital in public offerings.

Smaller companies can now sell up to $50 million of securities within a 12-month period through Regulation A offerings, which are exempt from some registration requirements. The previous cap was $5 million.

The rules provide for two tiers of offerings: Tier 1 offerings are limited to $20 million within a 12-month period, with no more than $6 million sold by affiliates of the issuer, while Tier 2 offerings can be up to $50 million, with no more than $15 million sold by affiliates. Tier 2 offerings are exempt from certain state registration requirements; Tier 1 offerings are not, although Tier 2 offerings are subject to stricter disclosure and reporting requirements.

The new rules will take effect 60 days after a notice of the changes is published in the Federal Register. (BioCentury)

Indiana Enacts "Right to Try" Bill - But Will it Matter?
Gov. Michael Pence this week signed a bill allowing people who are battling life-threatening conditions to gain access to experimental medicines. Known as a “right to try” law, the idea is to leapfrog a drug-development process that takes years before new treatments become available.

The move reflects rising frustration with an FDA program called expanded access, in which people who are seriously ill can obtain a drug under development, even though they aren’t enrolled in a clinical trial. The requests must be approved by both the agency and drug makers.

Critics complain the program is cumbersome and arbitrary, sparking desperate social media campaigns by families. But whether the “right to try” rallying cry, which is now law in 11 states, will make a difference is uncertain. Why?

“No one is obligated to do anything. It gives you the right to ask, but people have been asking drug companies in droves long before these laws existed,” says Art Caplan, who heads bioethics in the Division of Medical Ethics at the NYU Langone Medical Center. “It’s more like right to beg. And it’s easy for politicians to pass.”

The FDA has avoided taking a position on the laws, but maintains the existing drug approval process remains the best way to provide expanded access. However, the agency recently floated a document suggesting ways to hasten application procedures.

Yet, the FDA has a strong track record for approving requests, which are rising each year. During the most recent fiscal year, for instance, the agency signed off on 99.5% of nearly 1,900 requests received. More than half were considered emergencies.

“The FDA is not the obstacle to access,” says Patti Zettler, a former FDA associate chief counsel who is now a fellow at the Stanford Law School Center for Law and the Biosciences. “These laws aren’t doing anything to address understandable obstacles that companies face when a drug is requested.” More at link above. (WSJ Pharmalot blog)

Examining the Growing Problems of Prescription Drug and Heroin Abuse: State and Local Perspectives
On March 26, the House Energy and Commerce Committee (Oversight and Investigations Subcommittee) conducted a hearing to examine the growing problem of prescription drug and heroin abuse in the US. Members heard from a number of experts who confront this growing crisis in communities all across the country. The subcommittee last year held a hearing to discuss this issue with witnesses from federal agencies.

Witnesses from across the country shared their experiences and discussed efforts to confront the growing cycle of prescription drug and heroin abuse.  The background document, videocast, and statement documents can be found at the link above. (Energy & Commerce)

To Congress, From Pharma: Love, Hate And Fresh Ideas For 21st Century Cures
Summary: Industry and key stakeholders weigh in on House Commerce Committee’s first take at biomedical reform; brand companies offer lots of positive feedback but want many modifications; generic companies criticize multitude of new exclusivity provisions for brands.

A review of drug industry comments on the first version House Energy and Commerce Committee’s 21st Century Cures Initiative legislation reveals a bill that is being largely embraced by branded product sponsors, and criticized by generic companies.

Branded companies do have their fair share of tweaks and modifications though, and a few provisions they’d rather not see move forward – expanded access provisions top that list. However, branded company suggested revisions generally come on top of initial support for the starting draft measures.

Generic makers who commented on the discussion document largely spent their time criticizing the many new exclusivity provisions offered in the bill, which they fear will upset the balance between incentivizing innovation and availability of affordable generics created by the Hatch-Waxman Act.

One area of agreement for both brands and generics was in modernizing and reforming clinical trials.

Nearly 140 organizations submitted comments to the Energy and Commerce Committee. Notably, the bulk were from disease advocacy organizations and physician groups, not industry. This review focused on industry and key stakeholders. It also includes some comments from organizations who spoke directly to “The Pink Sheet.” More at link above. (The Pink Sheet, paid subscription required).

FDA’s New Criminal Investigations Chief Wants Global Reach
George Karavetsos is applying experience gained prosecuting drug dealers in Florida to FDA’s Office of Criminal Investigations. The new OCI director, and former Florida-based U.S. Attorney, wants his agents and staff on equal footing with the counterfeiters and other criminals – to not be impaired by country lines.

Karavetsos is further integrating FDA’s globalization mantra into the agency’s chief enforcement division. Rather than identify and trace suspect products after they reach the U.S., he wants OCI, with the help of other regulators, fighting the criminals around the world.

Extending OCI’s ex-U.S. presence is potentially a shift in focus after the office has gained attention among industry investigating off-label promotion and other largely domestic cases, although Karavetsos indicated the transformation already is ongoing.

“My goal is to continue the work that I see OCI doing, and that is expanding its global presence by strengthening our existing relationships with our foreign counterparts … developing new partnerships with our foreign regulatory and law enforcement counterparts and really coming together as one to attack this, what I would deem to be a significant threat, and that is the complex global supply chain that we operate in today,” he said.

Inspections, including Good Manufacturing Practice violations, will remain a priority for his office, Karavetsos said, but he would not draw a bright line where compliance problems become criminal issues. He said each case will be judged on its own merits. More at link above. (The Pink Sheet, paid subscription required)

SGR Repeal: Bipartisan Leaders Announce Repeal To Be Considered Before March 31 Deadline
An SGR (Sustainable Growth Rate) agreement negotiated by Speaker of the House, John Boehner and the House Democratic leader, Nancy Pelosi, would repeal the Medicare formula that threatens to cut doctors’ fees each year. In its place, Congress would establish an “incentive payment system” to reward doctors who receive high performance scores from the government. Scores would be based on factors like the ability to keep patients healthy while controlling costs, notes the New York Times.

The bill, H.R. 1470 and S. 810, is an updated version of the policies set forth in last year’s bipartisan, bicameral Medicare payment reform bill. The House is expected to vote on the entire proposal before the March 31 expiration date of the current patch.

Just yesterday, the House Energy and Commerce and House Ways and Means Committees introduced H.R. 2, the Medicare Access and CHIP Reauthorization Act, to permanently replace Medicare’s SGR. The agreement builds upon H.R. 1470. Notably, the top Republicans and Democrats from the key committees of jurisdiction are all cited as co-sponsors, indicating strong bipartisan support. More at link above. (Policy and Medicine)

Securities Fraud Class Actions Doubled Against Life Sciences Companies in 2014
Life sciences companies are an “increasingly popular target of securities fraud class action lawsuits,” a recent survey by Dechert LLP has found. Securities class actions against pharmaceutical, biotechnology, and medical companies more than doubled last year, to 39 cases compared with 19 during 2013. This represented 23 percent of the 170 total securities fraud class action lawsuits brought against all companies during 2014. Interestingly the majority of the life science defendants were smaller companies—57% had market capitalization of less than $500 million dollars according to the survey.

View the full report: Dechert Survey of Securities Fraud Class Actions Brought Against U.S. Life Sciences Companies

The Dechert report notes that most securities fraud complaints made “industry-specific allegations,” including “alleged misrepresentations or omissions regarding marketing practices, prospects/timing of FDA approval, product efficacy, product safety, manufacturing and other healthcare-related allegations.” Fifty-six percent of 2014 claims “alleged misrepresentations or non-disclosures regarding product efficacy or prospects/timing of FDA approval.”  More at link above. (Policy and Medicine)


SECTION 6 SOURCES REVIEWED FOR THIS NEWSLETTER

A partial listing of sources reviewed for this newsletter:  AdvaMed Smartbrief; AHRQ Newsletter; Alzheimers Association; Alzheimers Research Forum Newsletter; BioCentury; Biopharma Reporter; BIOtechNow; CDISC Monthly Newsletter; CER Daily Newsfeed (NPC); Daily Dose (NSF); 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; Wall Street Journal Pharmalot.