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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: November 21, 2014

SECTION 1 FDA GUIDANCES & MAPPS

CDER List of Guidance Documents (Updated)

CDER Guidances New/Revised/Withdrawn 1/1/14 - 9/30/14

The links above lead to the List of Guidance Documents (CDER) updated on October 27, 2014, and to CDER Guidances that are new, revised, or withdrawn through the third calendar quarter of 2014.

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

CDRH FY 2014 Proposed Guidance Development

On November 17, 2014, FDA announced the availability of a draft guidance entitled ‘‘Rare Pediatric Disease Priority Review Vouchers.’’  FDA will award priority review vouchers to sponsors of certain rare pediatric disease product applications that meet the criteria specified in that section. These vouchers can be used when submitting future human drug marketing applications that would not otherwise qualify for priority review. These vouchers can be sold or transferred for use to another sponsor any number of times before the voucher is used, as long as the sponsor making the transfer has not yet submitted the application. Because there exists a need for products for rare pediatric diseases, this program is intended to encourage development of new drug and biological products for prevention and treatment of certain rare pediatric diseases.  Comments should be submitted by January 16, 2015.  [Federal Register]

On November 17, 2014, FDA announced a correction to the proposed rule entitled “Additions and Modifications to the List of Drug Products That Have Been Withdrawn or Removed From the Market for Reasons of Safety or Effectiveness.” The document proposed amending FDA’s regulations to revise the list of drug products that may not be compounded under the exemptions provided by the Federal Food, Drug, and Cosmetic Act because the drug products have been withdrawn or removed from the market after the drug products or components of such drug products were found to be unsafe or not effective. The document also withdrew the previous proposed rule regarding additions to this list (see the Federal Register of January 4, 2000). The document was published with an incorrect RIN number. This document corrects the error. [Federal Register]

On November 17, 2014, FDA announced the update of a CDER Manual of Policies and Procedures (MAPP) entitled “INDs: Exception from Informed Consent Requirements for Emergency Research.”  Trials designed with an exception from informed consent requirements, although uncommon, can raise complex scientific and ethical considerations as well as clinical trial design and conduct challenges. (CFR) Section 50.24 was developed to make important research possible for patients with a life-threatening condition that necessitates urgent intervention (for which available treatments are unproven or unsatisfactory) and who, because of their condition, cannot provide informed consent. This MAPP describes policies and procedures for consistent review and administrative oversight of an investigational new drug application (IND) for emergency research in which the clinical investigation includes a request, pursuant to 21 CFR 50.24, for an exception from the requirement to obtain informed consent from patients. [FDA.gov]

On November 18, 2014, FDA announced the update of a CDER Manual of Policies and Procedures (MAPP) entitled “Chemistry Review of Question-based Review (QbR) Submissions.”  QbR is a general framework, recommended as a submission format by the draft guidance for industry ANDA Submission - Content and Format of Abbreviated New Drug Applications, for a science and risk-based assessment of product quality. It contains important scientific and regulatory review questions related to product and process design and understanding, product performance, and control strategy. This MAPP clarifies how drug substance and drug product reviewers in the Office of Pharmaceutical Science (OPS) should assess new drug applications (NDAs), abbreviated new drug applications (ANDAs), or Type II DMF submissions that follow a Question-based Review (QbR) format in conjunction with the International Conference on Harmonisation (ICH) guidance M4Q: The CTD – Quality, (ICH M4Q) Module 2.  [FDA.gov]

On November 19, 2014, FDA announced the availability of a document entitled ‘‘Validation Rules for Study Data Tabulation Model (SDTM) Formatted Studies.’’  CDER is making this document available to improve the standardization and quality of clinical data submitted to CDER, as well as to improve the predictability of data quality and usefulness. [Federal Register]

On November 19, 2014, FDA announced the availability of a guidance entitled “Vaginal Microbicides: Development for the Prevention of Human Immunodeficiency Virus Infection; Guidance for Industry.” The purpose of this guidance is to assist sponsors in all phases of development of vaginal microbicides, defined as vaginal drug products that prevent human immunodeficiency virus (HIV) acquisition. The guidance outlines the types of nonclinical studies and clinical trials recommended throughout the drug development process to support approval of vaginal microbicides. This guidance finalizes the draft guidance issued on November 23, 2012. [Federal Register]

On November 19, 2014, FDA announced the opening of a public docket and request for comments proposed criteria for ‘‘First Generic’’ Submissions for Purposes of Abbreviated New Drug Application Review Prioritization Under the Generic Drug User Fee Amendments.” The purpose is to facilitate FDA’s establishment of review prioritization under the Generic Drug User Fee Amendments of 2012 (GDUFA). Establishing clear criteria for this review prioritization category will allow FDA to appropriately prioritize ANDA submissions and track them in a manner consistent with the review prioritization commitments FDA made under GDUFA. Clear criteria for this category will also lead to less industry confusion and more consistent identification of ‘‘first generic’’ submissions. Comments should be submitted by December 19, 2014. [Federal Register]

On November 20, 2014, FDA announced the submission to OMB of a proposed collection of information on a ‘‘Risk and Benefit Perception Scale Development.” FDA needs a pool of reliable and valid measurement items for assessing consumers’ drug risk and benefit perceptions—as well as other elements of prescription drug decision making—consistently across studies. The purpose of this project is to create that measurement pool, thus increasing the rigor and efficiency of FDA’s research. Comments should be submitted by December 22, 2014.  The study design is described in the Federal Register notice.  [Federal Register]

On November 21, 2014, FDA announced the availability of a guidance entitled “Changes to an Approved Application: Biological Products: Human Blood and Blood Components Intended for Transfusion or for Further Manufacture.” Frequently, a manufacturer of a licensed product determines that it is appropriate to make a change in its product, production process, quality controls, equipment, facilities, responsible personnel, or labeling as documented in its approved BLA(s). This guidance document provides manufacturers of licensed whole blood and blood components intended for transfusion or for further manufacture, including source plasma, with recommendations concerning submission of changes to an approved biologics license application (BLA). The guidance document also provides manufacturers of licensed whole blood and blood components recommendations in connection with the applicability and content of comparability protocols and labeling changes. [Federal Register]

On November 21, 2014, FDA announced the availability of three additional policy documents to assist entities that compound sterile human drugs with registering as outsourcing facilities. The policy documents will also assist entities with complying with provisions of the Drug Quality and Security Act (DQSA), which was enacted in November 2013:

Comments will be accepted on the draft guidance until January 20, 2015. [FDA.gov]


SECTION 2 FDA NOTES & RELATED NEWS

Updated List of CDER Key Officials Posted

NIH and FDA Toughen Rules for Reporting Clinical Trial Results
In a bid to make clinical trial results more widely available, the U.S. Department of Health and Human Services has proposed new rules that require researchers in industry and academia to post findings of all studies funded by the federal government on the publicly available Clinicaltrials.gov website. The proposal would strengthen existing requirements that findings must be submitted to the website.

Right now, only about 15,000 of the approximately 178,000 trials registered on clinicaltrials.gov include summaries of results. NIH officials say they expect that the proposed rules will at least double the number of summary results posted on the website per week to between 200 and 250.

To accomplish this, the HHS wants to expand the scope of information that must be submitted to include summary results for products that are not currently approved or licensed in the U.S., as well as main results and additional tables showing breakdowns of adverse events. The rules also spell out who is responsible for posting data and the timing for providing the information, along with needed updates.  More at link above. (Pharmalot)

Diabetes Endpoints, CV Requirements Get Special Handling At FDA-Patient Meeting
Summary: FDA solves citizen petition with first-of-its-kind virtual patient meeting; diabetes group now hopes trifecta of requests will make it from Commissioner’s Office to top CDER officials.

Diabetes patients and advocacy organizations are urging the drug side of FDA to catch up to its device counterpart and look beyond hemoglobin A1c as an endpoint for diabetes products.

Diabetes advocates are also asking FDA to relax requirements for cardiovascular outcomes studies of diabetes drugs pre-approval and for the agency to develop a guidance that outlines a drug development and approval pathway for pre-diabetes treatments.

The trio of requests were shepherded by the diaTribe Foundation at a Nov. 3 FDA-Patient dialogue on unmet needs in diabetes, co-organized by diaTribe, a patient education and advocacy organization and FDA’s Cardiovascular and Endocrine Liaison program, part of the Office of Health and Constituent Affairs in the Commissioner’s Office.

The America Diabetes Association, the Juvenile Diabetes Research Foundation (JDRF) and patients also participated in person, as well as about 1,000 online participants.

Prior to the meeting, diaTribe conducted an online survey on the unmet medical needs in diabetes from Oct. 15 through Oct. 23 to present FDA with a better idea of how diabetes patients are managing their disease. The survey had more than 6,000 respondents, including nearly 4,000 patients and 2,000 parents or partners/caregivers of people with diabetes.

Despite the lack of CDER attendance, the first-of-its-kind meeting was hailed as a successful pilot. It resulted from a citizen petition diaTribe submitted to FDA after diabetes was not selected as one of the possible disease areas for the 20 patient-focused drug development meetings being conducted under PDUFA V to help reviewers involve patients in the creation of risk-benefit criteria.  More at link above.  (The Pink Sheet, paid subscription required)

Public Workshop - Regulatory Science Considerations for Software Used in Diabetes Management, November 13, 2014
On November 13, 2014, FDA held the workshop entitled:  “Regulatory Science Considerations for Software Used in Diabetes Management.”  The purposes of this workshop were to foster greater stakeholder collaboration in the area of diabetes device interoperability and to seek input form the clinical community, academia, government, industry and other stakeholders regarding the makeup of the intended use population for insulin bolus calculators, and the technical considerations for their design and use.  FDA has now posted to its website the full agenda with webcast recordings and slides.  More at link above. (FDA.gov)

FDA to Add Staff in China
FDA Commissioner Margaret Hamburg will meet with Chinese officials this week to discuss the agency's staffing needs in China.
Agency spokesperson Christopher Kelly said increased staffing in China "will further strengthen the safety, quality and effectiveness of food and medical products produced in China for export to the United States." Chinese officials agreed last December to allow FDA to expand its presence in the country.

Opened in 2008, FDA's China office now employs 13 full-time staff, including eight U.S. civil servants. Kelly said four positions in China have been vacant for the past year because FDA has been unable to secure visas.

Kelly said FDA expects to obtain visas for additional staff "in the coming weeks and months," and that nine staff members are "preparing to deploy." The agency plans to add a total of 10 drug inspectors, seven food inspectors, and one administrative position, bringing U.S. staff to 26 in the office.
In the meantime, FDA has brought in temporary inspectors from the U.S. to compensate for staff shortages. (BioCentury)

China Journal: strengthening relationships to protect public health
Dr. Margaret Hamburg visit China last week, and in her FDA Voice blog writes: “..a key reason for my trip is the important and growing collaboration between FDA and our counterpart agencies in China to ensure the safety of the large volume of foods and medical products exchanged between our two nations.

“Of the 200 countries that export their products to the United States, China ranks first in exports (in dollar value) to our nation. It is the sixth largest provider of food and the sixth largest provider of drugs and biologics. Only the United States has more FDA-registered drug establishments than China. And these numbers are growing.

Ensuring the safety and quality of U.S.-destined FDA-regulated goods is a major challenge.  Among the many efforts in this area, an important component is the FDA’s establishment of a permanent outposts staffed by FDA experts in China; there are 13 FDA staff members currently with plans to triple that number. Their job is to help ensure that the food and medical products being exported from China meet our standards. FDA’s China Office does this by providing significant support for the Agency’s inspections in China, by strengthening our relationships with Chinese regulators, by working with industry and other stakeholders, by providing important information and technical assistance to all interested parties, and by analyzing trends and events that might affect the safety of FDA-regulated products exported from China to the United States.

Hamburg writes, “This week, we took an important step forward in strengthening our relationship with China when we signed an Implementing Arrangement with the China Food and Drug Administration (CFDA). We expect to sign a similar Implementing Arrangement with the General Administration of Quality Supervision, Inspection and Quarantine (AQSIQ) in the coming weeks. These documents, which build on 2007 agreements with the same two agencies, help to frame the work our inspectors will do in China and create mechanisms for collaboration on inspections.”  More at link above.  (FDA.gov)


FDA Delays Final Rule on Allowing Generic Drug Makers to Update Labels
A controversial new rule for updating generic drug labeling that the FDA was expected to finalize next month will not be published until the fall of 2015, an agency spokeswoman says.

The rule, which the FDA proposed last year, would allow generic drug makers to independently update safety warnings, something that only brand-name drug makers can currently do before receiving FDA permission.

The agency proposed the rule in response to a 2011 U.S. Supreme Court decision that federal law does not permit generic drug makers to make such changes independently and, therefore, they should not be held accountable for a failure to warn against a risk.

An FDA spokeswoman wrote, “FDA received a great deal of public input from various stakeholders during the comment period on the proposed rule. We are committed to reviewing and considering all of the comments received as we develop the final rule.” More at link above. (Pharmalot)


SECTION 3 AGENCY AND ADVISORY COMMITTEE MEETINGS

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

Public Advisory Committee Meeting. Anesthetic and Analgesic Drug Products Advisory Committee. November 24-25, 2014. White Oak Campus, Silver Spring, MD. The committee will discuss the risk of serious neurologic adverse reactions associated with epidural steroid injections (ESI) administered to reduce inflammation for pain management. The committee will also consider the efficacy of ESI and the overall risk benefit balance of injecting steroids in the epidural space to treat pain. These considerations will assist the FDA in its discussions of possible regulatory options, including but not limited to changes to the product labeling. [Federal Register]

Public Advisory Committee Meeting. Blood Products Advisory Committee. December 2-3, 2014. White Oak Campus, Silver Spring, MD. On December 2, 2014, the Committee will meet in open session to hear scientific data related to reconsideration of the current blood donor deferral policy for men who have had sex with another man (MSM) even one time since 1977. An informational presentation will be made regarding the emergence of chikungunya virus infections in the Western Hemisphere and potential implications for blood transfusion safety. On December 3, 2014, the Blood Products Advisory Committee will be seated as a device classification panel. In open session, the panel will discuss the appropriate device classification of blood establishment computer software (BECS) and accessories to BECS. [Federal Register]

Meeting. Second Annual Meeting of C-Path and the Innovative Medicines Initiative. December 3, 2014. Bethesda, MD.  Entitled “Accelerating the development of drugs, diagnostics, and devices through partnerships that expand the precompetitive space,” the event aims to identify and address the challenges and opportunities of Public Private Partnerships (PPPs), e.g. mechanisms for assessing productivity and enhancing effectiveness, how to coordinate efforts and avoid duplication, and how to adopt best practices on data sharing. [More information]

Public Advisory Committee Meeting. Anti-Infective Drugs Advisory Committee. December 4, 2014. University of Maryland University College, Hyattsville, MD.  The committee will discuss issues related to clinical development programs and clinical trial designs for antibacterial products for the treatment of patients with serious bacterial infections for which there are limited or no therapeutic options. [Federal Register]

Meeting. Realizing the Benefits of the Unique Device Identifier in Health Care.  Office of the National Coordinator and Pew Charitable Trusts.  December 9, 2014. Washington, DC. This meeting will present discussion on the benefits and challenges of integrating the unique device identifiers (UDI) into clinical care, registries, the supply chain, and other facets of health care delivery. [FDA.gov]

New: Public Workshop. PCORI Research Dissemination and Implementation Framework & Toolkit. December 10, 2014. Webinar. The purpose of the workshop/webinar is to obtain input on evidence-based strategies for effective research dissemination and implementation (D&I). Input is sought on the D&I Framework and Toolkit, which PCORI will use to develop and execute strategic plans for sharing new information with key stakeholders.  [PCORI.org]

New: Public Workshop. Developing and Using Precision Therapies in the ‘‘Omics’’ Era: Generating and Interpreting Evidence for Rare Subsets. December 12, 2014. White Oak Campus, Silver Spring, MD.  Cosponsored with the Center for Translational and Regulatory Sciences at the University of Virginia (UVA). The goals of this public workshop are to facilitate discussion on current scientific approaches using rare subsets during drug development programs and to further seek input from multiple stakeholders on approaches to obtain evidence that inform the regulatory evaluation of therapeutic products in rare subsets of patients identified through in-vitro diagnostic testing when specific, controlled trials are not feasible. [Federal Register]

New: Public Workshop. Immunology of Protection from Ebola Virus Infection. December 12, 2014. Rockville, MD and Webcast.  The Food and Drug Administration (FDA), the National Institutes of Allergy and Infectious Diseases (NIAID), the Department of Defense (DoD), the Centers for Disease Control and Prevention (CDC), and the Biomedical Advanced Research and Development Authority (BARDA) are co-sponsoring a workshop, entitled “Immunology of Protection from Ebola Virus Infection.” The purpose of this workshop is to discuss important aspects of Ebola virus and vaccine immunology in order to inform future clinical, scientific and regulatory decision-making related to vaccines against Ebola. [FDA.gov]

New: January 8-9, 2015.  Two-day public meeting on prioritization of Lab-Developed Tests (LDTs).  At this meeting, FDA will discuss its proposal for a risk-based framework to address regulatory oversight of laboratory developed tests (LDTs). The agency plans to discuss with stakeholders how to balance patient safety and access to LDTs..  (FDA.gov)

Public Policy Committee Meeting. Health Information Technology Policy Committee Advisory Meeting.  Monthly beginning January 13, 2015.  Locations to be published.  This notice announces forthcoming meetings of a public advisory committee of the Office of the National Coordinator for Health Information Technology (ONC). The function of the committee is to provide recommendations to the National Coordinator on a policy framework for the development and adoption of a nationwide health information technology infrastructure that permits the electronic exchange and use of health information as is consistent with the Federal Health IT Strategic Plan and that includes recommendations on the areas in which standards, implementation specifications, and certification criteria are needed. [Federal Register]

Public Standards Committee Meeting. Health Information Technology Standards Committee Advisory Meeting.  Monthly beginning January 28, 2015.  Locations to be published.  This notice announces forthcoming meetings of a public advisory committee of the Office of the National Coordinator for Health Information Technology (ONC). The function of the committee is to provide recommendations to the National Coordinator on standards, implementation specifications, and certification criteria for the electronic exchange and use of health information for purposes of adoption, consistent with the implementation of the Federal Health IT Strategic Plan, and in accordance with policies developed by the Health IT Policy Committee. [Federal Register]

Public Workshop. Seventh Annual Sentinel Initiative. February 5, 2015.  Washington, DC.  Convened by the Engelberg Center for Health Care Reform at the Brookings Institution and supported by a cooperative agreement with FDA, this 1-day workshop will bring the stakeholder community together to discuss a variety of topics on active medical product surveillance. There will also be a live webcast for those unable to attend the meeting in person. [Federal Register]

New: 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]


SECTION 4 OTHER REGULATORY AUTHORITIES & ORGANIZATIONS

Community Engaged Network for All Announces the Addition of Two More Patient-Powered Research Networks in PCORnet
Community Engaged Network for All (CENA), a Patient Centered Outcomes Research Institute (PCORI) funded project announced the launch of registries for dyskeratosis congenita (DC) and related telomere biology disorders, and pseudoxanthoma elasticum (PXE) today. These registries join three others that have launched as part of this project and are built on CENA's Platform for Engaging Everyone Responsibly (PEER), developed in conjunction with technology partner Private Access.

PEER allows each individual to determine his or her own level of personal data sharing, privacy and access preferences. As individuals enter health information into the registry, they see real-time comparisons of their responses with the grouped anonymous answers of other participants.

The ultimate goal of the DC Outreach Registry is to improve the lives of those with Dyskeratosis Congenita and Telomere Biology Disorders by providing more appropriate means of information sharing and facilitating reciprocal medical research and care.  More at link above.  (Genetic Alliance release)

Tufts: Cost to Develop and Win Marketing Approval for a New Drug Is $2.6 Billion
Developing a new prescription medicine that gains marketing approval, a process often lasting longer than a decade, is estimated to cost $2,558 million, according to a new study by the Tufts Center for the Study of Drug Development.
The $2,558 million figure per approved compound is based on estimated:

  • Average out-of-pocket cost of $1,395 million
  • Time costs (expected returns that investors forego while a drug is in development) of $1,163 million
Estimated average cost of post-approval R&D—studies to test new indications, new formulations, new dosage strengths and regimens, and to monitor safety and long-term side effects in patients required by the U.S. Food and Drug Administration as a condition of approval—of $312 million boosts the full product lifecycle cost per approved drug to $2,870 million. All figures are expressed in 2013 dollars.

The new analysis, which updates similar Tufts CSDD analyses, was developed from information provided by 10 pharmaceutical companies on 106 randomly selected drugs that were first tested in human subjects anywhere in the world from 1995 to 2007. More at link above. (Tufts)

Gates to Fund Ebola Survivor Blood, Chimerix Drug Trials
The Bill & Melinda Gates Foundation committed $5.7 million to help produce and test potential blood-based therapies and antiviral treatments for Ebola.

The funds will support efforts in West Africa to evaluate plasma and other blood products from survivors of Ebola virus disease as a means of treating the infection in others, the Seattle-based foundation said in an e-mailed Nov. 18 statement. Various drug candidates will also be tested, including Chimerix Inc.’s antiviral brincidofovir.

The first human trials of experimental Ebola treatments are starting next month in Guinea, Liberia and Sierra Leone, the World Health Organization said this month, fast-tracking the development of pharmaceuticals to fight a disease that’s killed more than 5,000 people.

“The Gates Foundation is focusing its R&D investments on treatments, diagnostics and vaccines that we believe could be quickly produced and delivered to those who need them if they demonstrate efficacy in stopping the disease,” said Papa Salif Sow, a senior program officer and infectious diseases doctor with the foundation’s global health program, in the statement.  More at link above. (Bloomberg News)

2014 Access to Medicine Index Published
The annual report “The Access to Medicine Index” for 2014 has been released by the Access to Medicine Foundation, funded by the Bill & Melinda Gates Foundation, the UK Department for International Development, and the Netherlands Ministry of Foreign Affairs. The report opens with a passage acknowledging that significant milestones for improving access to medicine have been achieved, for example, important new drugs for TB and Hepatitis C, but goes on to say that “there is still no sustainable model for ensuring the poorest patients have access to the medicine they need.”

Through the Index, the Foundation aims to provide companies with clear guidance, by reporting on what they and their peers are already doing well, and by showing where solutions are still needed.  This year’s Index is “our clearest ever picture of the industry’s strengths, weaknesses, progress and struggles,” according Founder and CEO Wim Leereveld.

A company report card is provided for 20 large pharma companies, which are ranked based on a scored derived from their performance on such indicators as pricing, patents, capability, donations, and public policy. Full report at link above. (Press)

Biosimilars Add Urgency to Medicare Payment Reforms: MedPAC Commissioner
Summary: Developing alternatives to the ASP-based reimbursement system for Medicare Part B drugs may become increasingly important to ensure the program will benefit from lower-cost biosimilars, Medicare Payment Advisory Commission member points out.

The upcoming introduction of biosimilars into the U.S. market will make Medicare Part B payment reforms that address financial incentives for prescribers even more pressing, Medicare Payment Advisory Commission (MedPAC) member Jack Hoadley, a health policy researcher at Georgetown University, commented during a recent meeting of the panel.

At its Nov. 7 meeting, MedPAC discussed alternatives to the current Part B formula for reimbursing physicians for drugs based on average sales price (ASP) plus 6%. Options for change that were discussed included reimbursing interchangeable drugs within the same Medicare payment code (at a weighted average rate) and replacing ASP plus 6% payments with a flat fee.
Both would address concerns that the current reimbursement system encourages physicians to prescribe more expensive drugs when a lower-cost alternative is appropriate. More at link above. (The Pink Sheet, paid subscription required)

ICER Posts Draft Report on Newest Treatments for Hepatitis C Genotype 1 for Public Comment
The Institute for Clinical and Economic Review (ICER) has posted the draft report, “The Comparative Clinical Effectiveness and Value of Novel Combination Therapies for the Treatment of Patients with Genotype 1 Chronic Hepatitis C Infection” for public comment. The report examines the comparative clinical effectiveness of multiple new, all-oral direct-acting antiviral agent (DAA) regimens for the treatment of hepatitis C developed by Gilead Sciences Inc., AbbVie Inc., and Bristol-Myers Squibb Co. The report also assesses the potential financial impact on the health care system of several FDA-approved combination therapies, which can cost approximately $65,000 to $190,000 for a course of therapy. The report will be the subject of deliberation and vote at the next public meeting of the California Technology Assessment Forum (CTAF) on Thursday, December 18, 2014 in Oakland, California. Public comments on the report will be accepted through Monday, December 8, 2014 at 5:00 PM PT. More at links above. (ICER Release via National Pharmaceutical Council)

Institute of Medicine Workshop Summary: Ensuring Patient Access to Affordable Cancer Drugs
In recent years, patients' out-of-pocket costs for cancer care have been rising rapidly, including health insurance deductibles, coinsurance, and copayments for covered services, as well as services that are not covered by insurance. Many cancer patients are especially vulnerable financially because their illness and/or treatment impedes their ability to work, with some patients losing employment altogether. Even with insurance, cancer patients often experience financial hardships, such as going into debt, depleting all assets to pay for cancer treatment, and personal bankruptcy.

Although many elements contribute to the cost of cancer care, one important component is the cost of new cancer drugs, which has been escalating rapidly in recent years. To explore the issue of cancer drug costs and patient access to affordable, appropriate drug therapies, the Institute of Medicine's National Cancer Policy Forum convened a workshop on ensuring patient access to affordable cancer drugs in June 2014. Affordability was considered from both individual and societal perspectives. The workshop featured discussion panels as well as invited presentations from clinicians, researchers, representatives from the health insurance and pharmaceutical industries, and patient advocates. Ensuring Patient Access to Affordable Cancer Drugs summarizes the presentation and discussion of the workshop. More at link above, including downloadable pre-publication report.  (IOM)

National Institute on Aging Publishes Resource: Recruiting Older Adults into Research Toolkit
The National Institute on Aging (NIA) at NIH, the Centers for Disease Control and Prevention (CDC), and the Administration for Community Living (ACL) are collaborating on the Recruiting Older Adults into Research (ROAR) project to encourage older adults and their family caregivers, including underrepresented populations, to consider participating in research. They are starting with a focus on Alzheimer’s and dementia research.

Organizations can join this project and collaborate with their local area aging services, public health, and research colleagues to spread a message about healthy aging and research participation. To support such efforts, NIA has created a toolkit of easy-to-use, customizable materials that feature information on why research is important to healthy aging, what volunteers need to know about research studies, how older adults can make a difference by participating, and easy steps to take.

The ROAR Toolkit as a zip file, and materials may be customized with local information and for the needs of local audiences. More at link above. (NIA)

National Research Council Releases "Furthering America's Research Enterprise"
Scientific research has enabled America to remain at the forefront of global competition for commercially viable technologies and other innovations. For more than 65 years, the United States has led the world in science and technology. Discoveries from scientific research have extended our understanding of the physical and natural world, the cosmos, society, and of humans - their minds, bodies, and economic and other social interactions. Through these discoveries, science has enabled longer and healthier lives, provided for a better-educated citizenry, enhanced the national economy, and strengthened America's position in the global economy. At a time of budget stringency, how can we foster scientific innovation to ensure America's unprecedented prosperity, security, and quality of life?

Furthering America's Research Enterprise brings to bear a fresh approach informed by a more holistic understanding of the research enterprise as a complex, dynamic system. This understanding illuminates why America's research enterprise has historically been so successful; where attention should be focused to increase the societal benefits of research investments; and how those who make decisions on the allocation of funds for scientific research can best carry out their task.

This report will be of special interest to policy makers who support or manage the research enterprise, to others in public and private institutions who fund research, to scholars of the research enterprise, and to scientists and engineers who seek to better understand the many pathways through which their research benefits society.  More at link above.  (National Academies Press)

National Academies Press Tool: Academy Scope
National Academies Press has announced the launch of AcademyScope, an interactive visualization that allows you to explore relationships between thousands of reports on NAP.edu.

You'll find links to AcademyScope in the Browse by Topics menu, as well as on every topic page. AcademyScope doesn't support mobile devices at this time but will in the near future.  See the tool at either of the links above. (National Academies Press)

Conflicts of interests: revised EMA policy reflects more balanced approach
The European Medicines Agency (EMA) has published its revised policy on handling declarations of interests for scientific committee members and experts. The revisions reflect a more balanced approach to handling conflicts of interests that aims to effectively restrict the involvement of experts with possible conflicts of interests in the Agency’s work while maintaining EMA’s ability to access the best available expertise.

The revised policy, which was endorsed by the EMA Management Board in March 2014, takes into account input from stakeholders at the Agency’s September 2013 public workshop 'Best expertise vs conflicts of interests: striking the right balance'.

'The priority of EMA is to ensure that the integrity of our scientific assessments of medicines is not compromised by private interests in the pharmaceutical industry,' said Noël Wathion, EMA’s Chief Policy Adviser. 'But over recent years we have also recognised that an overly-rigid approach may limit the availability of expertise needed to ensure the robust scientific assessment of medicines. The updated policy should now allow a level of involvement better tailored to the interest profile of each expert.'  (EMA.europa.eu)

NICE Joins Global Group to Accelerate Access to New Drugs
NICE has joined a global group of organisations whose goal is to speed up the development and delivery of new, effective and affordable drugs to patients.

The New Drug Development Paradigms (NEWDIGS) program, led by the Center for Biomedical Innovation (CBI) at the Massachusetts Institute of Technology (MIT) in Cambridge, Massachusetts brings together pharmaceutical companies, academic researchers, patient advocacy groups, government agencies involved in healthcare and other experts to collaborate on ways to reliably and sustainably deliver new, better, affordable therapeutics to the right patients faster.

“Through NEWDIGS we’ll work with others to explore how our guidance development processes can contribute to faster and better drug development and delivery,” said Professor Sarah Garner, NICE’s associate director for R&D. “The initiative uses the principles of systems engineering – something MIT is famous for – to design and test scenarios for the development of new drugs.” More at links above. (NICE Release)

Britain begins review to speed access to new drugs
The British government has launched a review to speed the path to market for new drugs and medical devices in an attempt to improve patient care and make the country a more attractive place for investment in life sciences.

British manufacturing relies heavily on the pharmaceuticals sector, including domestic giants GlaxoSmithKline and AstraZeneca, but companies complain that the state-run National Health Service (NHS) is too slow to adopt new treatments.

The review, announced at a meeting between industry leaders and ministers at Prime Minister David Cameron's Downing Street office on Thursday, aims to tackle the issue by studying innovative models for drug development.

This will include an examination of the scope for more collaborative work between companies and regulators to ensure that new medicines and devices are assessed more quickly, with greater input from patient groups and charities such as Cancer Research UK.

An independent organization will be appointed to carry out the Innovative Medicines and MedTech Review, starting work early next year and reporting later in 2015. (Reuters)

Japan Pharmaceutical Companies Release their Own Sunshine Data
The Japanese pharmaceutical industry has been tracking and reporting transfers of value to healthcare professionals and academic centers based in Japan. This Policy and Medicine article walks through the provisions of the Japanese transparency guideline before looking at the payment data and corresponding news stories surrounding the recent release of that data.

The Japanese Pharmaceutical Manufacturers (JPMA) approved the “Transparency Guideline for the Relation between Corporate Activities and Medical Institutions,” in 2011. The voluntary code states that JPMA member companies and member companies’ affiliates based in Japan will track their payments to medical institutions and healthcare professionals whose primary place of practice is in Japan. The code requires tracking of five categories of payments: R&D expenses, Academic research support expenses, Honoraria, Medical Seminars and Education, Hospitality and “social courtesy” expenses. Japanese manufacturers do not have to track meal payments, a category that dominates the total transactions in the U.S., but corresponds to very low payment amounts.  

The Japan News, an English-language source published by Japan’s largest newspaper, Yomiuri Shimbun, wrote that the top ten pharmaceutical manufacturers paid doctors and hospitals ¥190 billion last year (about $1.63 billion). More at link above.  (Policy and Medicine)


SECTION 5 LEGAL AND COMPLIANCE

21St Century Cures Upton and DeGette at Partnering for Cures
Chairman Fred Upton (R-MI) and Rep. Diana DeGette (D-CO) joined medical thought leaders in New York City this week at the 2014 Partnering for Cures event to discuss how they are working together to speed up the discovery, development, and delivery cycle of cures in America. Upton and DeGette launched the bipartisan 21st Century Cures initiative earlier this year to do just that, speeding up the cures process by bringing our laws up to speed with medical technology.

Upton and DeGette indicated that they plan to move their bill through Congress in 2015. More at link above. (Energy and Commerce Release)

Patent Trolls Eye Biotech Firms: New PTO Proceedings are Game Changer
Biotech companies are accustomed to the hazards of research failure, but now may need to get used to another risk that could undermine their investments: Patent trolls that threaten to drag their key patents into inter parte review proceedings unless the firms pay them millions of dollars.

The proceedings, established under the Leahy-Smith America Invents Act, the landmark patent reform bill enacted in 2011, enable parties to challenge the validity of patents nine months after they issue. Challengers petition the Patent and Trademark Office’s newly created Patent Trial and Appeal Board (PTAB) to review patent claims and, if the board agrees to do so, it issues a final decision within 12 months.

Companies are concerned that the rules of the proceedings are biased against patent owners, and the majority of decisions by the PTAB have been in favor of the challengers. Teresa Stanek Rea, former PTO acting director and now a partner at Crowell & Moring, noted at a conference in June that about 95% of claims that go into the inter parties review proceeding do not survive the ordeal.

As a result, the number of cases being brought before the appeal board has surged since it was established in September 2012. While only a small percentage of cases before the board involve biopharma companies, the number is growing as the PTAB provides faster resolution of disputes, has a lower burden of proof and seems to favor the petitioners. The PTAB proceedings will thus have an enormous impact on the biopharma industry and the ability of innovator firms to protect their intellectual property.

The unfavorable rulings for patent owners may have put biotech companies in the crosshairs of so-called patent trolls, patent assertion entities that buy up patents and enforce them against others. Patent trolls have largely ignored the biopharmaceutical industry, focusing instead on the technology industry. The outcome of PTAB proceedings could change, however, as the patent office is evaluating the way they are conducted. In June it issued a call for stakeholder input on whether certain rules should be changed.  More at link above.  (The Pink Sheet, paid subscription required)

U of Maryland Health Law Regulatory Competition Scheduled for February 2015
The 4th Annual Health Law Regulatory & Compliance Competition will be held on Saturday, February 21, 2015, at the University of Maryland Francis King Carey School of Law.

The Health Law Regulatory & Compliance Competition requires teams of two or three students to analyze a particular fact pattern using federal health regulations, rules, and agency documents, and to present a legal and policy solution and/or recommendations to a panel of regulatory and compliance attorneys.  Participating students will be evaluated on an oral presentation of issues identified in the fact pattern and proposed actions and solutions to such issues.

The Competition will focus on several interactions between various health care stakeholders, including hospitals, physicians, drug and device manufacturers, and related third parties. Specifically, this year's problem will challenge students to address several areas, including but not limited to: FDA life-cycle management, bioethical research issues, FDA post-marketing requirements, and health care fraud & abuse issues, including the Anti-Kickback Statute and applicable safe harbors and exceptions, Stark Law, exclusion, and the False Claims Act and applicable whistleblower or qui tam provisions.  Applications are due December 12, 2014. More at link above, including link to application forms.  (Policy and Medicine)

OIG Report: 2014 Top Management and Performance Challenges for HHS
Annually, the Office of Inspector General (OIG) prepares a summary of the most significant management and performance challenges facing the Department of Health and Human Services (HHS). We refer to this summary as the Top Management Challenges (TMC). These challenges reflect continuing vulnerabilities that OIG has identified for HHS over recent years as well as new and emerging issues that HHS will face in the coming year.

Challenge 10 on the list is “Ensuring the Safety of Food, Drugs, and Medical Devices.” OIG cites this as a challenge because “OIG work has revealed weaknesses in FDA's ability to adequately oversee the safety of drugs, biologics, medical devices, and food. It has also revealed failures by industry participants to follow processes designed to ensure the safety and efficacy of food, drugs, biologics, and medical devices.”

These high risk areas include:  drug compounding, imported drugs, and marketing requirements (food safety is also on the list).  Relating to marketing requirements, the report indicates “..manufacturers are prohibited from promoting products for uses for which FDA has not specifically approved them (known as off-label uses). OIG, in conjunction with its law enforcement partners, including FDA's OCI, has investigated many instances in which manufacturers illegally promoted products for off-label uses. Off-label promotion can undermine the system intended to ensure that drugs are safe and effective and can put patients at risk. Additionally, this illegal off-label promotion may lead to fraudulent claims for payment submitted to federal health care programs, including Medicare and Medicaid.”

Among OIG’s recommendations for improving in these areas: “The Department (FDA) also needs to continue its efforts to eliminate off-label promotion and reduce the importation of unapproved drugs from foreign sources to protect patients and HHS health care programs.” More at link above. (OIG release)


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.