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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: May 22, 2015

SECTION 1 FDA GUIDANCES, RULES & 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.

April 28 Update: New & Revised Draft Guidances CDER is Planning to Publish during CY2015
The link above is the new CDER list of guidances planned for new issue or revision in Calendar Year 2015.  In this revision, FDA has added a new title to the Biosimilarity category: “Nonproprietary Naming for Biological Products.”

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final guidances for which CDRH would appreciate external feedback on whether these final guidances should be revised or withdrawn.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 May 18, 2015, FDA issued a draft guidance for industry and staff entitled ‘‘Adaptive Designs for Medical Device Clinical Studies.” This document addresses adaptive designs for medical device clinical trials and is applicable to premarket medical device submissions including Premarket Approval Applications (PMA), premarket notification (510(k)) submissions, de novo submissions (Evaluation of Automatic Class III Designation), Humanitarian Device Exemption (HDE) applications and Investigational Device Exemption (IDE) submissions. This guidance can be applied throughout the clinical development program of a medical device, from feasibility studies to pivotal clinical trials. This guidance does not apply to clinical studies of combination products or codevelopment of a pharmaceutical product with an unapproved diagnostic test. However, the underlying principles may be applicable to such studies.  Comments will be due 90 days from the date the draft document appears in the Federal Register.  [FDA.gov]

On May 18, 2015, FDA announced the availability of draft recommendations for preparing a Study Data Standardization Plan (“Electronic Study Data Submission; Data Standards; Study Data Standardization Plan.”) The Standardization Plan is referenced in the Study Data Technical Conformance Guide (Guide). The Guide supplements the guidance for industry ‘‘Providing Regulatory Submissions in Electronic Format—Standardized Study Data’’ and provides specifications, recommendations, and general considerations on submitting standardized study data using FDA-supported data standards. The Guide recommends that, for clinical and nonclinical studies, sponsors include a plan that describes the submission of standardized study data to FDA. The proposed recommendations describe the information that should be included in the Standardization Plan. The proposed recommendations for creating a Standardization Plan are posted on FDA’s Study Data Standards Resources Web page at http://www.fda.gov/ forindustry/datastandards/studydatastandards/default.htm. To assure consideration, comments should be submitted by July 2, 2015. [Federal Register]

On May 18, 2015, FDA announced the availability of grant funds for the “Cooperative Agreement to International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use.” The goal of the ICH is to bring together leading global drug regulatory agencies and pharmaceutical manufacturer associations to achieve greater harmonization of technical standards to ensure that safe, effective, and high-quality medicines are developed and registered in the most resource-efficient manner. The program’s grant funds will support the ICH to develop a series of international guidelines for implementation according to each region’s requirements aimed at achieving the following: (1) Develop and register safe, effective, and high quality medicines in the most efficient and cost effective manner; (2) prevent unnecessary duplication of clinical  trials and minimize the use of animal testing without compromising safety  and effectiveness, and (3) provide  public assurance that the rights, safety, and well-being of subjects are protected during clinical trials. [Federal Register]

On May 20, 2015, FDA CDER published a new policy and procedure in the Manual of Policies and Procedures (MAPP) entitled “Conducting Effective Meetings in CDER: Remote Access Considerations,” (MAPP 4512.2 Rev.1). This MAPP establishes best practices for conducting effective web-based meetings in CDER. The focus is on remote access considerations. This MAPP applies only to internal CDER meetings for which hosts and participants are remotely connected. This MAPP outlines responsibilities of meeting hosts and participants, as well as more detailed procedures to ensure CDER meetings are effective when participants are working at multiple locations. Available tools and technology should be tailored to match the meeting’s structure and objectives. The MAPP notes that CDER has selected Adobe Connect as its preferred tool, and Cisco WebEx as the alternative web conferencing tool. [FDA.gov]

On May 22, 2015, FDA announced its intent to renew the charter of the “Medical Imaging Drugs Advisory Committee.” The new charter will remain in effect until its expiration on May 18, 2017. [Federal Register]

On May 22, 2015, FDA amended the regulations applicable to blood and blood components, including Source Plasma, issuing the final rule entitled “Requirements for Blood and Blood Components Intended for Transfusion or for Further Manufacturing Use.” The new rule is intended to make the donor eligibility and testing requirements more consistent with current practices in the blood industry, to more closely align the regulations with current FDA recommendations, and to provide flexibility to accommodate advancing technology. In order to better assure the safety of the nation’s blood supply and to help protect donor health, FDA is revising the requirements for blood establishments to test donors for infectious disease, and to determine that donors are eligible to donate and that donations are suitable for transfusion or further manufacture. FDA is also requiring establishments to evaluate donors for factors that may adversely affect the safety, purity, and potency of blood and blood components or the health of a donor during the donation process. Accordingly, these regulations establish requirements for donor education, donor history, and donor testing. These regulations also implement a flexible framework to help both FDA and industry to more effectively respond to new or emerging infectious agents that may affect blood product safety. The rule is effective May 23, 2016. [Federal Register]


SECTION 2 EMA CONSULTATIONS, GUIDELINES, & NEWS

Facilitating the development of gene therapies: EMA Invites Feedback on New Draft Guideline
EMA) has released a draft guideline on quality, non-clinical and clinical aspects of gene therapies for a three-month public consultation. The document aims to support and facilitate the development of these innovative medicines by giving guidance to developers on the types of evidence they should generate to support a marketing-authorisation application with a regulatory authority in the European Union.

The draft guideline follows a new approach which aims to address the needs of typical developers of gene therapies, which tend to be very small companies or from academia, and less familiar with the regulatory environment.  It follows the structure of the common technical document (CTD), the document that needs to be completed at the time of submission of a marketing-authorisation application.

It also provides detailed guidance on both the scientific and development aspects of this type of medicines, and on the regulatory requirements that companies need to fulfil, including good manufacturing practice requirements. Comments should be submitted on this draft guideline by August 31, 2015. More at title link above. (EMA.eu)

EMA to host workshop on therapeutic use of bacteriophages as new way to fight antimicrobial resistance
The European Medicines Agency (EMA) will host a workshop on the use of bacteriophages to treat bacterial infections on 8 June 2015. The workshop will be broadcast live on EMA’s website and will provide a platform for experts from academia, industry, regulatory bodies to discuss key issues related to the potential therapeutic use of bacteriophages as anti-bacterial agents. For more information, please see the agenda of the workshop.

In light of emerging antibiotic resistance, there has been renewed interest in this alternative treatment option. The therapeutic potential of bacteriophages is being investigated by research groups and pharmaceutical companies in the European Union, including in a phase I-II clinical trial in patients with infected burn wounds (the Phagoburn study), which is funded by the European Commission under the seventh Framework Programme for Research and Development.

With this workshop, EMA supports these efforts by providing a platform to proactively discuss current issues and open questions and reflect on potential ways forward for this therapy with various stakeholders. More at title link above. (EMA.eu)


SECTION 3 FDA NOTES & RELATED NEWS

Reagan-Udall, Pfizer Testing Ways to Tap Sentinel Network without FDA
“…The Reagan-Udall Foundation and Pfizer Inc. are launching a pilot project to test approaches to conducting post-market drug safety research using FDA's Sentinel data network without the agency's involvement...The Reagan-Udall project involves the foundation's Innovation in Medical Evidence Development and Surveillance (IMEDS) Evaluation program, which is a public-private partnership created to build on the progress made in research methodology by Sentinel and by the industry-funded Observational Medical Outcomes Partnership (OMOP).” More at link above. (The Pink Sheet Daily, paid subscription required)

From our perspective: The U.S. drug supply chain and patient safety
In her May 15, 2015 FDA Voice blog, Ilisa Bernstein, Pharm.D., J.D., Deputy Director of FDA’s Center for Drug Evaluation and Research’s Office of Compliance, shares her thoughts on the important role drug supply chain integrity plays in patient safety and quality care:

“I’ve seen a growing trend of health care professionals and medical practice administrators who purchase prescription drugs from rogue wholesale drug distributors outside of the legitimate supply chain. While the U.S. drug supply chain is one of the most secure in the world, drugs purchased from rogue wholesale drug distributors put patients at risk of not getting the treatment they need or even worse, experiencing harmful effects.

“Drugs sold by rogue distributors may be counterfeit, adulterated or may not have been evaluated by FDA for safety and effectiveness. Because FDA has not evaluated these products, there is no way of knowing if these drugs were made using quality manufacturing practices, contain harmful ingredients, or if they were stored under proper conditions.  For these reasons and more, it is important that health care professionals know the source of their prescription drugs.

“First, doctors should ensure they are receiving FDA-approved prescription drugs. ..By purchasing from only state-licensed wholesale drug distributors, doctors can help prevent unsafe and ineffective drugs from reaching their patients.

“Once a drug is received, there are additional safety steps that can be taken. Doctors should check the packaging to determine if the packaging or the product differs in any way from the FDA-approved packaging and product. The label and other safety information and warnings should be in English. Also, check the dosing to ensure the dosing recommendations, form and route of administration are familiar.

“And finally, patient feedback can also be an indication that there may be a problem with a product. If several patients report that they are experiencing a new side effect or lack of therapeutic effect from the same product, consider that the drug may be substandard or counterfeit. Health care professionals and patients should report adverse events related to the use of a suspect product and drug quality issues to FDA’s MedWatch Safety Information and Adverse Event Reporting Program.” More at link above. (FDA.gov)

FDA Teams With National Forum to Reduce Deaths from Heart Disease: Program is first of its kind
To advance the cause of a heart-healthy and stroke-free society, FDA’s Office of Health and Constituent Affairs has signed a Memorandum of Understanding with the National Forum for Heart Disease and Stroke Prevention to promote and increase the use of health knowledge, skills and practices by the public in their daily lives. The National Forum’s members include more than 80 U.S. and international organizations representing public, private, health care, advocacy, academic, policy and community sectors. The five-year agreement is a first-of-its-kind cooperative public education program to reduce the burdens of heart disease and stroke.

Because medication is not readily adhered to – and neither are lifestyle treatments – millions of people suffer from preventable cardiac episodes. As a nation, lack of medication adherence (which can be as simple as not getting a prescription filled or refilled) costs more than $100 billion annually in excess hospitalizations. FDA is taking the lead in support of Million Hearts®, a national initiative of the Department of Health and Human Services to prevent 1 million heart attacks and strokes by 2017.

A clear goal of the initiative is to create recommendations to improve compliance with prescribed medical therapies and implement the recommendations to improve the lives of patients living with heart disease.

FDA’s Dr. Helene Clayton-Jeter and Dr. Fortunato “Fred” Senatore are leading a diverse team in identifying strategies to help patients take their medicines as directed and follow the advice of their doctors.  More at link above. (FDA.gov)

Detailed Drug Risk in TV Ads Waste of Time FDA Official Says
Long lists of scary prescription drug side effects at the end of television advertisements are a waste of time for patients, one of the U.S.’s top drug regulators said.

“When they give the adverse events, or adverse reactions, it’s this litany of complaints” -- death, or “‘Don’t use if you have MAE1,’” said Richard Pazdur, director of the office of hematology and oncology products at the Food and Drug Administration.

“What patient knows what MAE1 is?” Pazdur said. “It’s just a waste of time.”

Pazdur’s remarks come after the FDA last year began studying whether long lists of side effects in TV ads may cause viewers to overlook the most dangerous aspects of the medicine. An agency document about the study said it might be best to list only the most serious side effects and cover any lesser risks with a statement saying there are other potential harms. Currently, the ads are required to include disclosures about potential side effects and other technical information.

Pazdur didn’t endorse a specific change to drug ads on television but said he “would encourage the agency to look at this whole advertising issue in a much more innovative approach.”

The FDA is surveying 1,500 study participants about ads with varying ranges of side-effect disclosure, then plans to measure their understanding of risk.  More at link above. (Bloomberg News)


SECTION 4 AGENCY AND ADVISORY COMMITTEE MEETINGS

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

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] Podcast brief of Science Forum content by Dr. Luciana Borio, FDA’s Acting Chief Scientist: [FDA.gov]

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 Advisory Committee Meeting. Transmissible Spongiform Encephalopathies Advisory Committee. June 1, 2015.  White Oak Campus, Silver Spring, MD.  The Advisory Committee will meet to hear updates on the variant Creutzfeldt-Jakob Disease (vCJD) situation worldwide, followed by presentations by FDA on current measures to reduce vCJD from transfusion in the US and additional topics. [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 Advisory Committee Meeting. Risk Communications Advisory Committee. June 8-9, 2015.  White Oak Campus, Silver Spring, MD.  The Committee will discuss approaches to communicating information about fetal effects in product labeling for methadone or buprenorphine maintenance therapy for opioid addiction, and about the maternal benefits and risks of treatment, to best enable patients and health care providers to make informed decisions about the use of these drugs during pregnancy. [Federal Register]

Public Meeting and Request for Comment. Generic Drug User Fee Amendments of 2012. June 15, 2015.  White Oak Campus, Silver Spring, MD.  Before FDA begins negotiations with the regulated industry on GDUFA reauthorization, it will hold a public meeting at which the public may present its views on the reauthorization, including specific suggestions for changes to the goals referred to in the Generic Drug User Fee Act Program Performance Goals and Procedures (i.e., the Commitment Letter). A docket is also established for public comments. [Federal Register]

New: Public Meeting.  Pharmacy Compounding Advisory Committee.  June 17-18, 2015.  White Oak Campus, Silver Spring, MD.  FDA will discuss with the committee drugs proposed for inclusion on the withdrawn or removed list pursuant to sections 503A and 503B and on the section 503A bulk drug substances list. FDA will also discuss with the committee the criteria FDA proposes to use to evaluate candidates to be identified as difficult to compound pursuant to sections 503A and 503B. [Federal Register]

New: Public Meeting and Request for Comment. Exploring Naloxone Uptake and Use. July 1-2, 2015.  White Oak Campus, Silver Spring, MD.  FDA CDER, in collaboration with the National Institutes on Drug Abuse, the Centers for Disease Control and Prevention, the Substance Abuse and Mental Health Services Administration, and the Health Resources and Services Administration, will hold a public meeting to discuss increasing the use of naloxone to reduce the incidence of opioid drug overdose fatalities. During the meeting, academic and government experts, industry representatives, and patient advocates will discuss which populations are at-risk for opioid drug overdose and how we can work together to encourage the use of naloxone to reduce the risk of overdose from opioid drugs.   A docket is also established for public comments. [Federal Register]

New: Public Meeting.  Medical Device User Fee Act Reauthorization.  July 13, 2015.  White Oak Campus, Silver Spring, MD.  FDA will hold a public meeting to gather initial input on reauthorization of the Medical Device User Fee program, as required by section 738A of the Federal Food, Drug, and Cosmetic Act. Additional information and Federal Register announcement coming soon. [FDA.gov]

Public Meeting.  Prescription Drug User Fee Act.  July 15, 2015.  White Oak Campus, Silver Spring, MD.  FDA will begin the PDUFA reauthorization process for fiscal years 2018 through 2022 by requesting public input and holding a public meeting where the public may present its views on the reauthorization. FDA invites public comment as the Agency begins the process to reauthorize the program in FYs 2018–2022. [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 5 OTHER ORGANIZATIONS & AUTHORITIES

Genomics-Enabled Learning Health Care Systems: Gathering and Using Genomic Information to Improve Patient Care and Research - Workshop Summary
New gene-disease associations are being discovered through a variety of mechanisms and these findings are opening up new possibilities for the identification of therapeutic targets. However, the majority of this information is not being used clinically to inform the treatment of patients due to a lack of evidence regarding the validity of the association and the relationship between specific variants in a particular gene and disease outcome, progression, or prognosis. The inclusion of genomic data in a knowledge-generating health care system infrastructure is one promising way to harness the full potential of genome information to improve health.

To examine pragmatic approaches to incorporating genomics in learning health care systems, the IOM’s Roundtable on Translating Genomic-Based Research for Health hosted a workshop in December 2014. The workshop examined how a variety of systems are capturing and making use of genomic data to generate knowledge for advancing health care in the 21st century. It also sought to evaluate the challenges, opportunities, and best practices for capturing or using genomic information in knowledge-generating health care systems.  A summary of the workshop report can be downloaded at the link above. (Institute of Medicine)

U.S. science leaders to tackle ethics of gene-editing technology
The leading U.S. scientific organization, responding to concerns expressed by scientists and ethicists, has launched an ambitious initiative to recommend guidelines for new genetic technology that has the potential to create "designer babies."

The technology, called CRISPR-Cas9, allows scientists to edit virtually any gene they target. The technique is akin to a biological word-processing program that finds and replaces genetic defects.

CRISPR has also brought ethical concerns. Last month, scientists in China reported carrying out the first experiment using CRISPR gene-editing to alter the DNA of human embryos. Although the embryos were not viable and could not have developed into babies, the announcement ignited an outcry from scientists warning that such a step, which could alter human genomes for generations, was just a matter of time.

In response, the National Academy of Sciences (NAS) and its Institute of Medicine will convene an international summit this fall where researchers and other experts will "explore the scientific, ethical, and policy issues associated with human gene-editing research," the academies said in a statement.

In addition, NAS - an honorary body that was chartered by Congress in 1863 and performs studies for the federal government and others - will appoint a multidisciplinary, international committee to study the scientific basis and the ethical, legal, and social implications of human gene editing. The NAS committee will recommend guidelines for gene-editing technologies. More at link above. (Reuters)

How the Trans-Pacific Partnership Will Impact Prescription Drugs
For the last several years, the US government has been negotiating a free-trade agreement known as the Trans-Pacific Partnership (TPP) with 11 other countries across the Asia-Pacific and Latin American regions, which could have major impact on the pharmaceutical market.  When finalized it will be the largest free-trade agreement in history, impacting up to one-third of world trade and roughly 40 percent of the global gross domestic product. The deal has attracted a fair share of criticism from a wide range of groups, including concerns over proposed regulations for biologic drugs in participating countries. Specifically, critics are concerned about the length of data exclusivity granted to the companies that hold the patents on these drugs. Below is a primer on biologics and how they are being addressed in the TPP.

Under current FDA regulations, biologic drugs are granted 12 years of data exclusivity following approval. During this period of exclusivity, the FDA may not approve a biosimilar application that relies on the data submitted as part of the original biologic application. This form of temporary monopoly is distinct from patent protection, which is granted well before approval and is not related to clinical data.

For the 11 countries besides the U.S. that are involved in the TPP, current data exclusivity protections range from zero (Brunei) to eight years (Japan). Under the Obama Administration’s current proposal, participating countries would increase those periods to match the US standard of 12 years. Imposing US standards on the 11 member countries would inevitably restrict competition at the global level, and many patient advocacy and international humanitarian organizations have argued that doing so would undermine the efforts of US global health initiatives like the Vaccine Alliance and the Global Fund to Fight AIDS, Tuberculosis and Malaria, which rely on price competition to manage program costs.

It is unclear whether the US will be successful in its efforts. There have been reports that the issue of data exclusivity has become a significant point of contention, and the US delegation may seek to compromise on its demands. However, the details of the negotiations are largely confidential, which makes it challenging to assess the possibilities, their relative advantages, or how the US Trade Representative (which is leading the US negotiations) is balancing the need to ensure adequate incentives for innovation with the need to control drug costs and facilitate patient access to potentially life-saving therapies. More at link above. (Brookings)

Critical Path Institute Launches New Neonatal Consortium
Today (May 19), the Critical Path Institute (C-Path), a pioneering nonprofit organization dedicated to accelerating the pace and reducing the costs of medical product development by facilitating unique partnerships among a wide range of stakeholders, formed its ninth consortium, the International Neonatal Consortium (INC). The launch took place at the European Medicines Agency (EMA) during a widely attended workshop focused on the needs of the neonate. Dr. Jordi Llinares Garcia (Interim Head, Human Medicines Research & Development Support Division, EMA) welcomed the collaborative efforts to create novel and improved methods to evaluate treatments that will one day benefit a vulnerable and underserved population.

Dr. Janet Woodcock, Director of the U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER), added that “By uniting stakeholders from research institutions, drug developers, regulatory agencies, patient advocacy and other organizations, INC can develop practical tools that can be incorporated into clinical trials for neonates, which will then lead to more successful, efficient trials and provide this population with better treatments.”

The C-Path-appointed Executive Director of the new consortium is Dr. Alan Bedrick, who will continue to serve as Chief of Neonatology and Developmental Biology at the University of Arizona College of Medicine – Tucson.

INC will concentrate on conditions commonly observed in neonatal intensive care units (NICUs). Therapeutic areas include neonatal brain, lung, and gastrointestinal injury; neonatal sepsis; retinopathy of prematurity; and neonatal abstinence syndrome. Workshop participants prioritized an array of high-impact initiatives, such as developing clinical trial endpoints to assess efficacy of treatments. More at link above. (C-Path)

"Reconnecting the Dots — Reinterpreting Industry–Physician Relations" Provides A Balanced Look At Physician-Industry Collaboration and Conflicts of Interest
Lisa Rosenbaum, MD recently published an article entitled “Reconnecting the Dots—Reinterpreting Industry-Physician Relations,” in The New England Journal of Medicine. Rosenbaum’s article provides a refreshingly balanced analysis of financial conflicts of interest in medicine. “Although most observers agree that we must mitigate the risk of bias introduced by these relationships, the benefits wrought by interactions between physician-scientists and industry at the basic or translational research level are equally clear,” she states. “The question, then, is how to best manage conflicts of interest while preserving the collaborations on which medical advances depend.”

“Physician-industry interactions have been critical to the development of a large percentage of the medical products that allow physicians to prevent heart attacks, cure cancers, and restore mobility to the elderly,” Thomas Stossel, M.D., a distinguished Harvard hematologist and research recently noted. “Despite such progress and the role of physician-industry interactions in fomenting it, physicians are reducing or severing their relationships with biopharmaceutical and medical device companies out of fear that their patients will mistakenly view such interactions as a sign of corruption, rather than expertise.”

To this end, Rosenbaum sets out to find a “reasoned approach” to physician-industry interactions that acknowledges both the benefits of the collaboration, while recognizing the risks of bias involved.  More at title link above. (Policy and Medicine)

Personalized Medicine Is Gaining Traction, but Faces Multiple Challenges
While development of personalized medicines has grown since the human genome was first sequenced in 2001, biopharmaceutical sponsors face a number of hurdles that are impeding more rapid market uptake, according to a recently completed study by the Tufts Center for the Study of Drug Development (CSDD).

Fourteen years after the human genome was initially sequenced, paving the way for development of personalized medicine, 13% of drugs marketed in the United States today post pharmacogenomic information on the label, but developers continue to encounter challenges relating to basic science, regulatory and reimbursement policies, and, equally critical, clinical adoption, according to Tufts CSDD.

“In particular, the continued development of personalized medicine depends on identifying biomarkers and developing clinically useful diagnostic tests,” says Joshua Cohen, associate professor at Tuft CSDD. He noted, however, that higher R&D success rates alone may not lead translate into commercial success without physicians increasing the rate at which they prescribe personalized medicines, supported by payer willingness to reimburse users.

These assessments, based on a survey of major drug manufacturers and interviews with leading drug and diagnostics companies, were reported in the May/June Tufts CSDD Impact Report, released today. Other highlights include the following:

  • Biopharmaceutical firms said they expect investment in personalized medicine to increase 33%, and medicines in development to increase 69%, over the next five years.
  • Biomarker identification and diagnostic test development rank highest in terms of scientific challenges, followed by regulatory and reimbursement issues.

More at link above. (Tufts)

Canadians lagging well behind similar countries on access to new medicines: report
A new report commissioned by Rx&D, the association of Canada’s research-based pharmaceutical companies, finds that Canadians face wait times of over 460 days in order to get access to new, potentially lifesaving medicines in public drug plans.  The 2015 Access to New Medicines in Public Drug Plans: Canada and Comparable Countries report finds Canada seriously lagging compared to other similar OECD countries in terms of public drug plan reimbursement.

“It’s unimaginable to know that, in 2014, only 23% of 141 Health Canada-approved new medicines were included in public plans, ranking Canada 17 out of 18 on this front,” said Brett Skinner, Rx&D Executive Director, Health and Economic Policy.

The report notes that:

  • In Canada, 29% of cancer medicines were covered in public drug plans across provinces comprising at least 80% of the eligible national public drug plan population, ranking Canada in 16th place of 18 countries.
  • Canadian public drug plans placed reimbursement conditions on 90% of new medicines when measured across provinces comprising 80% of the eligible national public drug plan population. 
  • In Canada, 20% of new biologic medicines were reimbursed in public drug plans across provinces comprising at least 80% of the eligible national public drug plan population, putting Canada in 17th place of 18 countries.

More at title link above. (Rx&D release)

NICE to advise firms on how to speed up NHS adoption of their drugs
The UK National Institute for Health and Care Excellence (NICE) is to set up a new Office of Market Access to advise companies producing new drugs on how to speed up their adoption by the NHS.

The decision, discussed at a NICE board meeting on 20 May, was outlined on 21 May at a Westminster Forum seminar on healthcare research by Carole Longson, NICE’s director of health technology evaluation. Research was the key to generating new drugs, she said, and moves to accelerate the adoption of new products were being encouraged by regulators in the form of early access schemes, where drugs may be approved on much less evidence than in the past.

In this new environment, she asked, “Is NICE sending the right signals to the research environment, and are we doing it in a way that can be picked up?” She answered her own question by admitting, “Well, I think we can do (better)..” More at link above. (The BMJ)

EFPIA: Clinical Trial Data Sharing in Best Interest of All
On May 20, EFPIA issued a statement indicating that the organization “and its member companies believe that sharing clinical trial data sharing (is) in the best interests of patients, clinicians, medical research and the pharmaceutical industry.”

“We are committed to working with stakeholders to ensure that clinical trial information is shared responsibly, while ensuring patient anonymity and continuing to support the development of innovative new treatments with appropriate arrangements for commercial-in-confidence information.

“The EFPIA/PhRMA Joint Principles for Responsible Clinical Trial Data Sharing, implemented on 1 January 2014, have underscored the commitment of the pharmaceutical industry to disclosing clinical trial data.  EFPIA members have made significant progress in developing processes for clinical trial data access schemes, translating principles into practice.

“In addition to the industry led initiatives, EFPIA welcomes the adoption of the EU clinical trial regulation (536/2014) and publication of the EMA’s Transparency Policy (Policy 0070).  We are continuing to engage with stakeholders to ensure the successful implementation, with the aim of striking a balance between responsible reporting for public health benefit and safeguarding patient confidentiality, respecting the integrity of the regulatory systems worldwide, and maintaining incentives for investments in biomedical research.” More at link above.  (EFPIA)

India Health Ministry to roll out materio-vigilance programme for safety of medical devices
The Union health ministry has decided to come out with Materio-vigilance Programme of India (MvPI) in three months time to ensure safety of medical devices among the patients. This is being initiated in the backdrop of cases of malfunctioning of medical devices including the one of hip replacement implant of a leading MNC a couple of years ago.

The Ministry has constituted a national level committee to formulate a system of reporting of adverse events of medical devices under MvPI. The MvPI is meant to enable safety data collection in a systematic manner so that regulatory decisions and recommendations on safe use of medical devices for India could be based on data generated in India.

The programme is meant to monitor medical device associated adverse events (MDAE), create awareness among healthcare professionals about the importance of MDAE reporting in India and to monitor the benefit-risk profile of medical devices. It is also meant to generate independent, evidence-based recommendations on the safety of medical devices and to communicate the findings to all key stakeholders.

Envisaged to start with setting up of MvPI cells in 10 medical colleges of the country, the programme will be along the lines of the existing pharmacovigilance programme and haemovigilance programme in the country.  More at link above. (Pharmabiz.com)


SECTION 6 LEGAL AND COMPLIANCE

The 21st Centuries Act: UNANIMOUSLY APPROVED
The House Energy and Commerce Committee today (May 21, 2015) unanimously approved the nonpartisan 21st Century Cures Act 51-0. The nonpartisan legislation will help to modernize and personalize health care, encourage greater innovation, support research, and streamline the system to deliver better, faster cures to more patients. The bill has seen continued support throughout the process. H.R. 6, the 21st Century Cures Act, was authored by full committee Chairman Fred Upton (R-MI), Oversight and Investigations Subcommittee Ranking Member DeGette, full committee Ranking Member Frank Pallone, Jr. (D-NJ), Health Subcommittee Chairman Joe Pitts (R-PA), and Health Subcommittee Ranking Member Gene Green (D-TX).

"This historic day marks a big bipartisan step forward on our path to cures,” said Upton. “We have all said too many early good-byes to people we love and treasure. Every single person has a common goal: we want more time with those we love. In this, the greatest country in the world, Americans deserve a system second to none. We can and must do better. The time for 21st Century Cures is now.”

"In the last century, American medicine leapt from medicine shows to the mapping of the human genome,” said DeGette. “With the 21st Century Cures Act, we seek to support the biomedical community in making a similar leap forward in this next century. With billions in support for our premier research and development institutions and comprehensive reform of our systems, 21st Century Cures will make a real difference in the lives of patients and their families." More at link above. (Energy & Commerce)

FDA funding added to 21st Century Cures Act
The U.S. House Energy & Commerce Committee passed the 21st Century Cures Act (H.R. 6) on Thursday by a 51-0 vote following the last minute addition of FDA funding provisions. The bill includes a "Cures Innovation Fund" that provides FDA $110 million per year for five years. The $550 million total falls short of the $960 million FDA estimates it will cost to implement the bill, but is nonetheless a major achievement for FDA because prior versions did not appropriate additional money for the agency.

The bill also includes requests to the House Appropriations Committee to appropriate additional funding to FDA, and a provision exempting user fees from sequestration.

In remarks to rare disease advocates a few hours after the committee vote, E&C Chairman Rep. Fred Upton (R-Mich.) predicted that the full House will pass H.R. 6 this summer with 300-350 votes. Upton said he expects the Senate to pass a much more modest medical innovation bill. Upton said he hopes the two bills would be reconciled into a single bill that can be sent with bipartisan support to President Obama.  More at link above. (BioCentury)

New York introduces cost transparency bill
New York became the latest state to introduce a drug cost transparency bill as state Sen. Ruben Diaz (D) introduced Bill S5338-2015, which would require drug companies to disclose to legislators and the public R&D costs, SG&A expenses and profits for any drug with a wholesale acquisition cost exceeding $10,000 annually or per treatment course. The Pharmaceutical Cost Transparency Act of 2015 does not include any cost-containment mechanisms. The bill was referred to the state's Committee on Health.  

Similar state transparency bills in California and Oregon have failed. Legislators from both states previously told BioCentury the main reason those bills could not get out of committee was that they did not include mechanisms to lower drug prices. Committee members in California and Oregon also noted the bills' requirements to disclose data publicly might violate federal confidentiality requirements. A transparency bill in North Carolina, HB 839, was not heard by the House Health Committee before an April 30 voting deadline.

Transparency bills are pending in Massachusetts and Pennsylvania. Neither bill would require cost information to be made public. The Massachusetts bill, S1048, would allow the state's Health Policy Commission to impose drug price caps. Pennsylvania's bill, HB 1042, does not include cost-containment measures but would allow insurance plan sponsors in the state to stop covering drugs in cases where companies do not submit the required cost data. (BioCentury)

#SubOversight Continues Efforts to Combat Prescription Drug & Opioid Abuse
Also on May 21, the Energy and Commerce Oversight and Investigations Subcommittee, chaired by Rep. Tim Murphy (R-PA), continued its review of the ongoing prescription drug and opioid abuse epidemic with a look at what is being done at the state level to combat these crises. The hearing continued the subcommittee’s review of this epidemic, previously focusing on, state and local perspectives, professional and academic perspectives, and the federal government’s response. More at link above. (Energy & commerce)

House passes R&D tax credit

The U.S. House of Representatives voted 274-145 to pass legislation that would increase and make permanent the R&D tax credit. H.R. 880, the American Research and Competitiveness Act, would increase the tax credit to 20% from 14%. The bill will now go to the U.S. Senate.

On Tuesday, the Obama Administration said it "strongly opposes" the bill because it does not include offsets and would add $180 billion to the deficit over the next 10 years. The administration said that if President Obama were presented with the bill, his senior advisors would recommend that he veto it.  (BioCentury)

California Assembly passes right-to-try bill
The California Assembly voted 74-2 to pass a bill that would allow drug manufacturers to provide investigational therapies that have completed a Phase I trial but have not been approved by FDA to eligible terminally ill patients. The legislation includes liability protection for health care providers, and authorizes but does not require health plans to cover investigational therapies.

Assemblyman Ian Calderon (D) sponsored the bill, AB-159, known as the Right to Try Act. Four Assembly members did not vote on the bill. A companion bill, SB-149, has passed the California Senate's Committee on Health.

The Goldwater Institute said 17 states have passed right-to-try bills. The institute drafted model legislation for the state bills.  (BioCentury)

Woman sues Anthem Blue Cross for refusing to cover hepatitis C drug
A West Hollywood woman sued insurer Anthem Blue Cross for refusing to cover the cost of an expensive drug that she says would cure her hepatitis C infection.

Shima Andre said in the lawsuit that Anthem has refused to pay the estimated $99,000 it would cost to be treated with the controversial drug Harvoni, which has been shown to destroy the deadly virus in most patients.

In a denial letter, Anthem explained that the drug was “not medically necessary” because Andre does not have advanced liver damage, the lawsuit said. “We may approve Harvoni when the liver has a certain amount of scarring on a liver biopsy,” the insurer explained. “Records we received do not show that your liver has this amount of scarring.” Andre, 42, who was diagnosed with hepatitis C in 2011.

“How can it not be medically necessary when your treating doctor recommends it, it's the standard of care and it will cure the disease?” said Ricardo Echeverria, Andre’s lawyer.

The lawsuit, filed Friday in Los Angeles County Superior Court, accuses Anthem of breach of contract, infliction of emotional distress and unfair business practices. It seeks certification as a class-action lawsuit, which would add others denied the drug to the suit.  More at link above. (LA Times)

SECTION 7 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.