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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: April 3, 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final guidances for which CDRH would appreciate external feedback on whether these final guidances should be revised or withdrawn.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 April 6, 2015, FDA issued a final rule regarding the “Anti-Infective Drugs Advisory Committee.” The Committee name has been changed to the following: Antimicrobial Drugs Advisory Committee. The Agency changed the name to better reflect the products and issues that will be brought to the committee. The change became effective March 4, 2015. The Committee reviews and evaluates available data concerning the safety and effectiveness of marketed and investigational human drug products for use in the treatment of infectious diseases and disorders. [Federal Register]

On April 6, 2015, FDA announced that a proposed collection of information on “Comparative Price Information in Direct-to-Consumer and Professional Prescription Drug Advertisements” has been submitted to OMB. The Office of Prescription Drug Promotion plans to investigate, through empirical research, the impact of price comparison information and additional contextual information on prescription drug product perceptions. This will be investigated in direct-to-consumer (DTC) and healthcare-directed professional advertising for prescription drugs.  The Federal Register posting describes the proposed study design overview and procedure.  Comments may be submitted until May 6, 2015. [Federal Register]

On April 6, 2015, FDA announced that a proposed collection of information on “Survey of Health Care Practitioners for Device Labeling Format and Content” has been submitted to OMB. The purpose of this study is to compare existing device labeling from approximately six different types of medical devices with a standard content and format of the same labeling that FDA researchers will develop using the existing labeling as their source of the information. Building upon the research methodology and success of the approach FDA used to evaluate drug labeling, we propose to measure the usability and usefulness of a draft standard content and format of device labeling against existing manufacturer labeling of the same device.  Findings will provide evidence to inform FDA's planned regulatory approach to standardizing medical device labeling across the United States.  Comments should be submitted by May 6, 2015.  [Federal Register]

On April 7, 2015, FDA announced the availability of a guidance for industry entitled “Risk Evaluation and Mitigation Strategies: Modifications and Revisions.” This guidance provides information on how FDA will define and process submissions for modifications and revisions to risk evaluation and mitigation strategies (REMS), as well as information on what types of changes to approved REMS will be considered modifications of the REMS and what types of changes will be considered revisions of the REMS.  There are different procedures for submission of REMS modifications and revisions to FDA as well as different timeframes for FDA review and action of such changes. In addition, this guidance provides information on how REMS modifications and revisions should be submitted to FDA and how FDA intends to review and act on these submissions. The definitions of REMS modifications and revisions apply to all types of REMS. FDA indicates that comments may be submitted on this guidance until June 8, 2015.  [Federal RegisterNote: This guidance, except for the portion setting forth the submission procedures for REMS revisions, is being implemented without prior public comment because the Agency has determined that prior public participation is not feasible or appropriate (21 CFR 10.115(g)(2)). The Agency made this determination because, consistent with the requirements of FDASIA, FDA is issuing this guidance to establish a less burdensome policy and process for submitting certain changes to REMS that is consistent with public health. Although the guidance document is immediately in effect, except for the submission procedures for REMS revisions, it remains subject to comment in accordance with the Agency’s good guidance practices.

On April 8, 2015, FDA announced the “Center for Devices and Radiological Health: Experiential Learning Program.” This training component is intended to provide CDRH staff with an opportunity to understand the policies, laboratory practices, and challenges faced in broader disciplines that impact the device development life cycle. The purpose of this document is to invite medical device industry, academia, and health care facilities to apply to participate in this formal training program for FDA’s medical device review staff, or to contact CDRH for more information regarding the ELP.  The ELP is intended to provide CDRH staff with an opportunity to understand the policies, laboratory practices, and challenges faced in broader disciplines that impact the device development life cycle. This is a collaborative effort to enhance communication and facilitate the premarket review process. Furthermore, CDRH is committed to understanding current industry practices, innovative technologies, regulatory impacts, and regulatory needs. A listing of the specific areas of interest for CDRH staff is provided in this posting.  Requests for participation should be submitted by May 8, 2015.  [Federal Register]

On April 9, 2015, FDA a guidance for industry and staff entitled “Balancing Premarket and Postmarket Data Collection for Devices Subject to Premarket Approval.” This guidance clarifies the Food and Drug Administration’s (FDA or the Agency) current policy on balancing premarket and postmarket data collection during FDA review of premarket approval applications (PMAs). More specifically, this guidance outlines how FDA considers the role of postmarket information in determining the extent of data that should be collected in the premarket setting to support premarket approval while still meeting the statutory standard of reasonable assurance of safety and effectiveness. [FDA.gov]

On April 9, 2015, FDA announced the availability of a guidance for industry and staff entitled “Expedited Access for Premarket Approval and De Novo Medical Devices Intended for Unmet Medical Need for Life Threatening or Irreversibly Debilitating Diseases or Conditions.” This guidance explains FDA’s new, voluntary program for certain medical devices that demonstrate the potential to address unmet medical needs for life threatening or irreversibly debilitating diseases or conditions and are subject to premarket approval applications (PMAs) or de novo requests. FDA believes that the “Expedited Access Pathway for Unmet Medical Needs for Life Threatening or Irreversibly Debilitating Diseases or Conditions” (“Expedited Access Pathway” or “EAP”) program will help patients have more timely access to these medical devices by expediting their development, assessment, and review, while preserving the statutory standard of reasonable assurance of safety and effectiveness for premarket approval1 and the statutory standards for granting a de novo request consistent with the Agency’s statutory mission to protect and promote public health. The Expedited Access Pathway Program (EAP) will start accepting requests for EAP designation on April 15.  [FDA.gov]


SECTION 2 FDA NOTES & RELATED NEWS

FDA Gives a Nod for Alzheimer's and Parkinson's Biomarkers

In three letters signed last week by Janet Woodcock, who directs the agency’s Center for Drug Evaluation and Research, the FDA backed commonly used markers: hippocampal volume and CSF measures of Aβ42, tau, and phospho-tau for prodromal AD; and dopamine transporter imaging for early PD. The public letters were issued to the Tucson, Arizona-based Critical Path Institute’s Coalition Against Major Diseases (CAMD), in response to the coalition’s recent submission of data supporting the use of the markers. While the letters signify the FDA’s support of the biomarkers, the agency will require more clinical trial data before officially qualifying any of them. Qualification would mean that the agency agrees the markers predict disease progression reliably enough for the purpose of trial enrichment. “Six years later, thanks to an extraordinary leadership team and dedicated employees, we’ve seen enormous and important progress. We have renewed, expanded, and refined our mission and activities in important and powerful ways.”

“Today, FDA approves drugs faster on average than all other advanced nations, and the vast majority of the time, the U.S. is the first country in the world to approve important and novel medicines. Substantial improvements in the efficiency of medical device reviews have also been made.

These biomarkers are already widely used in drug development, so who needs FDA qualification? According to Diane Stephenson of CAMD, FDA qualification would relieve trial sponsors of the burden of having to convince the agency that the biomarkers are reliable and reproducible every time they run a trial. “Clearly, trial sponsors have been using biomarkers for a long time, and continue to do it without formal regulatory qualification,” Stephenson said. “But qualification could save both the FDA and sponsors a tremendous amount of time and money.”  More at link above.  (Alzheimer’s Research Forum)

Reagan-Udall Foundation for the FDA is releasing two Request for Proposals
The Reagan-Udall Foundation for the FDA is releasing two Request for Proposals. These two requests are for work relating to two of the projects outlined in the 2015 IMEDS-Methods Research Agenda:

PROMPT Assessment RFP (RUF-IMEDS-FP_0004): The Prospective Routine Observational Monitoring Program Tools (PROMPT) provides semi-automated routine surveillance by applying general tools with minor adaptations to address a specific product. This request is for investigators to propose and carry out evaluations of PROMPT.

Proposal Release Date: April 10, 2015
Proposal Deadline: Friday, June 10, 2015 at 4:30 p.m. ET

Center of Excellence for Monitoring Research on Bias in Research using Electronic Health Data RFP (RUF-IMEDS-FP_0003): IMEDS proposes to enter into collaboration with an academic center that could serve as a Center of Excellence for annually collating and summarizing research at the intersection of data analysis and medical data generation, particularly as these pertain to the evaluation of safety and effectiveness of medical products.

Proposal Release Date: April 10, 2015
Proposal Deadline: Friday, June 10, 2015 at 4:30 p.m. ET

Providing Timely Patient Access to High-Quality, Safe and Effective Medical Devices

In his April 8 FDA Voice bog, Dr. Jeffrey Shuren, Director of the FDA Center for Devices and Radiological Health (CDRH) writes: “We know that patients with life-threatening or irreversibly debilitating conditions lack treatment and diagnostic options. For these patients, earlier access to promising new devices is critically important. At the same time, delayed access may mean the difference between life and death, or may result in irreversible disability.

In weighing the benefits and risks of new technologies for these patients, we understand the need to place greater weight on the benefit of earlier access, and to also account for the risks of delayed access. That’s why  we’ve developed the Expedited Access Program (EAP): to speed qualifying devices to patients with life-threatening or irreversibly debilitating conditions without compromising FDA’s high standards for safety and effectiveness.

Under this voluntary program, sponsors of devices for life-threatening or irreversibly debilitating conditions that meet an unmet need can request an EAP designation. Also under this program, CDRH staff- including senior management – work collaboratively with developers of such devices earlier and more often. These efforts include the creation of a Data Development Plan that provides predictability and leverages postmarket data collection. The Data Development Plan will shift premarket data collection to the postmarket setting, to the extent appropriate, taking into account the public health benefit of these devices, while still meeting the U.S. approval standard of reasonable assurance of safety and effectiveness. Starting April 15th, this program will be up and running and we will begin to accept requests for EAP designation. 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. 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]

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]

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.  Blood Products Advisory Committee.  May 13, 2015.  White Oak Campus, Silver Spring, MD. The committee will meet in open session to discuss strategies for implementation of serological and nucleic acid testing for Babesia microti in blood donors. The committee will also hear update presentations on the following topics: (1) FDA considerations for Hemoglobin S Testing in blood donors; and (2) FDA considerations for a revised blood donor deferral policy for men who have sex with men. [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]

New: Public Workshop. FDA Science Forum 2015.  May 27 - 28, 2015.  White Oak Campus, Silver Spring, MD.  The public workshop will highlight science conducted at the FDA by showcasing how scientific research informs regulatory decision making and to provide a forum for developing collaborations within FDA and with external organizations. The focus of the forum will be the eight FDA Regulatory Science priority areas. [Federal Register]

New: Annual Workshop. European Network of Pediatric Research at the EMA.  May 28, 2015. Canary Wharf, London, UK.  The aim of the annual workshop is to promote the conduct of high quality paediatric clinical studies.  Enpr-EMA is a network of research networks, investigators and centres with recognised expertise in performing clinical studies in children. Its main objective is to facilitate studies in order to increase the availability of medicines authorised for use in children.  [EMA.eu]

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

Margaret A. Hamburg Appointed as Institute of Medicine Foreign Secretary
Margaret A. (Peggy) Hamburg, former commissioner of the U.S. Food and Drug Administration, has been appointed as the next foreign secretary of the Institute of Medicine, the health arm of the National Academy of Sciences.  In this part-time position, Hamburg will serve as a senior adviser on international matters to the IOM president and council and as liaison to foreign academies of medicine and science.  Her term, which begins on April 6 and is effective through June 30, 2019, follows the outstanding service over the past eight and a half years in this role of Jo Ivey Boufford, president of the New York Academy of Medicine.  As IOM engagement in matters of health that extend beyond the United States continues to expand, so does the importance of the foreign secretary’s role.  Thanks to Boufford’s leadership, the IOM is highly sought after as a partner in international efforts to improve health and advance the role of academies in advising governments on policy issues that impact health.

"Global engagement and impact are strategically important for the IOM.  I am delighted that Peggy has agreed to take on this important responsibility," said IOM president Victor J. Dzau.  "Her experience and interests equip her superbly for the role of foreign secretary, and I look forward to working with her to expand IOM’s global leadership."

Established in 1970 under the charter of the National Academy of Sciences, the Institute of Medicine provides independent, objective, evidence-based advice to policymakers, health professionals, the private sector, and the public. More at link above. (National Academies Release)

Policy Issues in the Development and Adoption of Biomarkers for Molecularly Targeted Cancer Therapies
A long-held goal in oncology has been to develop therapies that target the specific abnormalities in each patient's cancer rather than simply treating cancers based on the tissue of origin. In the past decade, advances in technology have enabled researchers to relatively quickly and inexpensively determine, in minute detail, the genetic makeup of tumors. Although relatively few targeted cancer therapies are currently available in the clinic and it is not yet clear whether all cancers are driven by genetic changes that can be targeted, there is widespread optimism in the cancer community that this new ability to assess the genetic abnormalities in tumors will ultimately lead to better cancer treatments and improved patient outcomes.

Policy Issues in the Development and Adoption of Biomarkers for Molecularly Targeted Cancer Therapies is the summary of a workshop convened in November 2014 by the Institute of Medicine's National Cancer Policy Forum to discuss recent trends in the development and implementation of molecularly targeted cancer therapies and explore potential policy actions to address specific challenges. This report highlights the presentations and discussions at the workshop. More at link above. (National Academy of Sciences)

NCI researchers say cancer drugs prices are not related to efficacy
A study by researchers at NIH's National Cancer Institute found that the annual treatment price for new cancer drugs is not correlated with the benefits seen in clinical trials.

The study, published in the April 2 Journal of the American Medical Association, analyzed 51 cancer drugs that were approved in the U.S. from 2009-13, including line extensions; and the efficacy data used to support the drug applications, including response rate, progression-free survival (PFS) and overall survival (OS). The group then compared the efficacy data to the annual treatment costs of the drugs and found no significant relationship between percentage improvement in PFS or OS and treatment costs (PFS, p=0.10; OS, p=0.09).

The researchers concluded that current pricing models are "not rational" but instead "reflect what the market will bear."  More at title and study links above. (BioCentury)

PCORI Offers Up to $90 Million in Funding in Fourth Call for Pragmatic Clinical Study Proposals
The Patient-Centered Outcomes Research Institute (PCORI) today issued the latest funding announcement under its Pragmatic Clinical Studies initiative, offering up to $90 million in support for projects evaluating the effectiveness of different care options in “real-world” settings.

PCORI will provide up to $10 million in direct costs for studies lasting up to five years through this funding opportunity, the fourth offered through this initiative.

The initiative’s aim is to support large patient-centered studies that can address information gaps on health problems that place heavy burdens on patients, families, and the healthcare system. These projects are designed to produce evidence generally applicable to a wide range of patients’ needs and clinical settings and to answer critical comparative clinical effectiveness questions faced by patients and clinicians.

More information and a listing of the focus areas for the funding announcement is available at the link above. (PCORI)

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)

NIST seeks comments on path forward for cybersecurity of consumer data
The National Institute of Standards and Technology (NIST) is seeking comments on suggested future cybersecurity efforts to protect consumer data, including healthcare data. Comments will be accepted through May 17, 2015.

NIST published a report from its Executive Technical Workshop on Improving Cybersecurity and Consumer Privacy, which brought together IT and security leaders from industries such as retail, hospitality and healthcare to discuss the challenges they face in securing consumer data.

Workshop participants represented a wide variety of sectors and organizations in a range of sizes. Despite their organizational differences, they agreed on many broad points, including:

  • Both organizations and consumers are responsible for safeguarding digital assets, and organizations can help consumers realize stronger cybersecurity protections through education, training and privacy policies that are more transparent and clear—and security measures that are easier for consumers to use.
  • People who develop software and applications need security tools that are easier to include in their products.
  • Cybersecurity products and services must be easier for security technologists to use.

The entire cybersecurity community—including government, industry and academia—needs to work together to address large issues. Participants asked NIST to act as a convener for these efforts.  More at link above. (Fierce Health IT)

TransCelerate BioPharma Launches Two New Global Initiatives
TransCelerate BioPharma Inc. today announced that it has established two new global initiatives which will accelerate and enhance clinical trials. Both initiatives - Placebo / Standard of Care Data Sharing and Electronic Labels for Clinical Trials (e-Labels), are made possible through the collaboration of participating Member Companies. They aim to create solutions that will have a direct impact on patients, sponsors and investigative sites.

The Placebo / Standard of Care Data Sharing Initiative has the potential to create a framework for data sharing that offers the potential to reduce the patient population needed to be enrolled in a clinical trial, as it intends to allow for the leverage of data from previous studies respecting boundaries of informed consent. It would thereby decrease the time spent on cumulative trial execution, and assists in acceleration of new therapies to patients. It also offers the potential for more rapid understanding of safety signals to better manage patient safety during clinical trials, as well as identify statistical techniques that may deliver more accurate study design and statistical power calculations.

The e-Labels Initiative will support TransCelerate Member Companies in establishing an innovative information channel: Electronic Labels (e-Labels). This initiative will work to enhance label utility for patients, provide more consistent labeling approaches for sites and offers the potential to reduce clinical labeling timelines and provides cost efficiencies for sponsors. The Initiative, in collaboration with Regulatory Agencies, will develop guidance to facilitate implementation of e-Labeling.  More at link above. (TransCelerate)

Science Journalist Discusses Why Some Health Technologies Fail To Make An Impact In Developing World
In the New York Times (4/4, Maxmen) “Opinionator,” science journalist Amy Maxmen writes that about five years ago, “a device capable of isolating bacterial DNA and diagnosing TB within hours became available.” The CDC “said it was ‘revolutionizing tuberculosis control.’” However, according to Maxmen, the technology, called GeneXpert, has yet to make a major impact. Maxmen writes that “the situation illustrates our impetuous worship of technology: Innovations make a splash, but little consideration is given to the mundane systems required for their success.” She writes that in “the developing world, potentially lifesaving devices like GeneXpert gather dust for lack of affordable transportation that might connect patients to the technology, and for lack of trained staff, electricity and relatively expensive materials necessary for their operation.” (Via DIA Daily, available as a DIA member benefit)

Kaiser Enrolling 15,000 Participants in Autism Family Research Bank to Study Gene, Environment Links
Kaiser Permanente has unveiled an ambitious plan to corral 15,000 individuals into a database over the next three years that researchers can use to investigate the genetic and environmental factors involved in autism spectrum disorders.

Last week Kaiser announced that its Autism Research Program received a $4.6 million grant from the Simons Foundation to create a database of 5,000 family trios — an autistic individual under age 26 and his or her biological parents — over the next 36 months. The so-called Autism Family Research Bank will contain de-identified genetic, medical, and environmental information on these trios that researchers can use to advance their understanding of the complex condition and possibly apply to the development of treatments.

"The resource will facilitate research aimed at identifying genetic and non-genetic underpinnings of autism, which should facilitate the development of pharmacologic treatments for various aspects of the disorder," said Lisa Croen, director of the autism program within Kaiser's research division.

After the research bank is enrolled, Croen expects Kaiser will need additional resources to perform genetic analysis of the biospecimens, run other queries on the collected data, and pursue the different hypotheses born out of the research bank. "With additional funding, whole-genome or exome sequencing are likely possibilities," she noted.

This resource is unique in that it will be comprised of members of one large healthcare system, with extensive clinical information on the participants that can be harnessed from the electronic medical record," Croen said. She further highlighted that Kaiser has the ability to follow participants longitudinally and invite them to partake in future studies where researchers might collect more detailed data on them.

Croen noted that after the development of the Autism Family Research Bank, Kaiser is planning to collaborate with other scientists in academia and industry and make the resource available to qualified researchers.  More at link above.  (GenomeWeb, free subscription required to access.)

Deloitte Releases "State of the Industry Report: Leveraging benefit-risk information to achieve better outcomes"

There is increasing awareness about the need for all health care stakeholders to focus on patient outcomes, with the goal of improving health, addressing disparities, and lowering the cost of health care. For many health care interventions, there is a substantial body of evidence about benefits and risks, and increasing evidence about comparative effectiveness. However, there is   a significant variation in how different stakeholders, such as life science organizations, regulatory agencies, providers, health plans, and patients understand and measure benefit and risk. The different values and perspectives of stakeholders likely mean that we may not arrive at a single benefit-risk approach. That said, there is ample opportunity for better communication and understanding of benefit-risk information across stakeholder groups.

This report discusses drivers that emphasize focus on outcomes, the variety of stakeholder perspectives on benefit-risk, and ideas to gain greater collaboration, communication, and innovation across the health care industry. Findings in this report are meant to encourage greater stakeholder dialogue between life science organizations, regulatory agencies, and health plans to identify methods for greater collaboration and better use of benefit-risk information to enhance health and patient outcomes.

From the Executive Summary of the report.  Full report at link above. (Deloitte)

More Alzheimer’s Journals Starting Up This Spring
Two open-access companion publications of Alzheimer’s & Dementia, The Journal of the Alzheimer’s Association, make their debuts this spring. DADM, which stands for Diagnosis, Assessment & Disease Monitoring, hit the virtual newsstands last month, while TRCI, short for Translational Research & Clinical Interventions, will publish its first issue in April. DADM will detail research about biomarkers for progressive dementia, that is, markers that detect disease, track its severity, and reflect treatment response. TRCI will focus on clinical therapeutics for dementia and other aging processes that impair cognition, memory, or behavior.

These online-only publications take their names from two of the seven areas of AD research outlined in the “common Alzheimer’s disease research ontology” (CADRO) classification system proposed by the National Institute on Aging, the National Institutes of Health, and the Alzheimer's Association to help coordinate funding efforts around the world. Ara Khachaturian, who heads Alzheimer’s & Dementia, said that his journal has seen an explosion of important papers in these two areas, but many were of interest to only a subset of readers.

Neither of these new journals will charge readers. Instead, they will charge author fees. Authors pay $2,000 to publish a research article, though members of the Alzheimer's Association professional society, ISTAART, pay half that. Shorter articles cost less, and letters to the editor or brief perspectives are free. These fees are similar to those charged by other open-access journals. In addition, DADM and TRCI promise fast peer review, and publication within a month of acceptance.  More at title link above. (Alzheimer’s Research Forum)

New EU rules on human tissues and cells increase patient safety
The Commission has adopted two sets of rules for human tissues and cells to protect patients in the EU through ensuring high quality and safety standards. The first sets out technical requirements that facilitate tracing of all tissues and cells from donor to recipient, and vice versa. This will happen through a so-called 'Single European Code' and Commission hosted IT-platform ensuring uniform labelling of all tissues and cells distributed in the EU. In the case of a safety alert, this label will ensure that all recipients who received material from the same donor can be traced and treated as needed. It will also allow for unused tissues or cells to be discarded. The second directive covers imports and sets out procedures for ensuring that tissues and cells imported from third countries meet the same safety and quality standards as those procured, processed and distributed in the EU. These implementing rules will ensure safety for EU recipients in need of these tissues and cells, no matter their origin. (European Commission release)


SECTION 5 LEGAL AND COMPLIANCE

Patent and Trademark Office Revising Patent Challenge Proceedings
Heeding criticism from the biopharma industry and other stakeholders, the Patent and Trademark Office is changing the practices of its Patent Trial and Appeal Board. The revisions, which address motions to amend patent claims and the scope of discovery, may make it easier for patent owners to fight those seeking to invalidate their patents.

PTO Director Michelle Lee described the agency’s plans to revise PTAB’s trial proceedings in a in a three-part roll-out plan:

  • “Quick fixes” to be issued in the spring;
  • Second proposed rule package to come out in the summer; and
  • Update to the agency’s trial practice guide.

The PTAB oversees administrative trial proceedings established under the America Invents Act in which parties petition the board to review the validity of patent claims. If the board decides to institute a review, it must issue a final written decision within one year. The patent office went on a listening tour last year, holding meetings in cities around the country to get input on the board’s rules and trial proceedings.

The Pharmaceutical Research and Manufacturers of America criticized the rules and practices governing inter partes review and post-grant review of patents, saying they did not appear to “provide sufficient fairness and due process to patent holders.”  More at link above. (The Pink Sheet, paid subscription required.)

Proposed Incentivized Wellness Bill Raises Concerns about Employee Privacy Under GINA
Recently introduced legislation that would allow employers to ask people about their family medical history as part of financially incentivized wellness programs has patient health advocates concerned that the bill would put at risk privacy protections under the Genetic Information Nondiscrimination Act of 2008 (GINA).

The so called ''Preserving Employee Wellness Programs Act" (H.R. 1189/ S. 620) was introduced in March by Rep. John Kline (R-Minn.) in the House of Representatives and by Lamar Alexander (R-Tenn.) in the Senate. Employer groups and providers of wellness programs believe the legislation protects them from legal liability and enables them to more effectively provide incentivized employer-sponsored wellness programs.

However, some health advocacy organizations believe the bill could open the door for employers offering financial rewards for wellness programs to coerce their employees in to revealing their family medical history and genetic information that is currently protected under GINA.

Under the Affordable Care Act (ACA), employers can provide financial incentives – up to 50 percent off insurance premiums and other rebates – to employees for participating in programs that encourage healthy living. The latest bill from Alexander and Kline aim to "clarify" the rules guiding such programs, and would allow employers to collect information about employees' preexisting conditions or a disease that runs their families. Additionally, the bill clarifies that by offering a reward to wellness program participants, employers would not be violating the Americans with Disabilities Act of 1990 or GINA, as long as the program complies with the Public Health Service Act requirements.  More at link above.  (Genome Web, free subscription required.)

Arizona Law Allows Consumers to Order Lab Tests Directly
Arizona Gov. Doug Ducey signed legislation that allows state residents to order any lab test directly from a licensed clinical laboratory without authorization from a healthcare provider. Ducey signed the bill, H.B. 2645, in the presence of Elizabeth Holmes, the CEO of laboratory services provider Theranos Inc. (Palo Alto, Calif.), which had campaigned for its passage.

Under the law, patients will be responsible for consulting with a health care provider on the interpretation of lab test results. Providers will not be subject to liability or disciplinary actions for failing to review or act on test results they did not order. The law also prohibits labs from billing insurers for tests ordered directly by patients.  (BioCentury)


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.