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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: September 26, 2014

SECTION 1 FDA GUIDANCES & MAPPS

CDER List of Guidance Documents

CDER Guidances: New/Revised/Withdrawn through 6/30/14

The links above lead to the List of Guidance Documents (CDER) updated on July 7, 2014, and to CDER Guidances that are new, revised, or withdrawn through the second calendar quarter of 2014.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 September 23, 2014, FDA announced the availability of a draft report entitled ‘‘Report on the Standardization of Risk Evaluation and Mitigation Strategies.”  This report describes the Agency’s findings concerning strategies to standardize risk evaluation and mitigation strategies (REMS), where appropriate, with the goal of reducing the burden of implementing REMS on practitioners, patients, and others in various health care settings. As part of the reauthorization of the Prescription Drug User Fee Act (PDUFA), FDA has committed to standardizing REMS to better integrate them into the existing and evolving health care system. FDA is publishing this report to allow the public to provide comment on the report as it relates to PDUFA.  Comments should be submitted by November 24, 2014. [Federal Register]

On September 24, 2014, FDA announced the availability of a guidance entitled ‘‘Custom Device Exemption.’’ The intent of this guidance is to define terms used in the custom device exemption, explain how to interpret the ‘‘five units per year of a particular device type’’ language contained in the FD&C Act, describe information that FDA proposes manufacturers should submit in the custom device annual report, and provide recommendations on how to submit an annual report for devices distributed under the custom device exemption. [Federal Register]

On September 25, 2014, FDA published a list of premarket approval applications (PMAs) that have been approved. Entitled, ‘‘Medical Devices; Availability of Safety and Effectiveness Summaries for Premarket Approval Applications,’’ this list is intended to inform the public of the availability of safety and effectiveness summaries of approved PMAs through the Internet and the Agency’s Division of Dockets Management. [Federal Register]

On September 26, 2014, FDA announced it is reopening the comment period for the draft guidance entitled “Informed Consent Information Sheet.” A notice of availability requesting comments on the draft guidance document appeared in the Federal Register of July 15, 2014. The Agency is reopening the comment period to update comments and to receive any new information. Comments should be submitted by October 27, 2014. [Federal Register]

SECTION 2 FDA NOTES & RELATED NEWS

Updated List of CDER Key Officials Posted

FDA Launches "Know Your Source" Program for Prescription Drugs
On September 23, FDA launched “Know Your Source,” an educational program advising health care professionals and practice administrators to only purchase prescription drugs from wholesale drug distributors licensed in your state to protect your patients from unsafe and ineffective drugs.

Drugs from rogue wholesale drug distributors may be harmful and expose patients to unknown risks or side effects. To reduce the chance of a potentially unsafe drug reaching patients, FDA offers tips for the health care professional prescribing and providing prescription drugs.  More at link above. (FDA.gov)

Three Encouraging Steps Towards New Antibiotics
In an FDA Voice blog on September 23, Dr. Janet Woodcock discussed three newly approved antibiotics that exemplify the results of ongoing FDA efforts to build the nation’s arsenal of effective antimicrobial drugs.  The three, approved in May, June, and August of this year, treat patients infected with methicillin-resistant Staphylococcus aureus (MRSA).  She writes:

“In these approvals, the drug’s manufacturer was able to take advantage of recently enacted incentives to help bring new antimicrobials to market. Each of these drugs was approved after being designated as a Qualified Infectious Disease Product (QIDP) under the GAIN Act. As part of this QIDP designation, FDA’s review of the drug application was expedited. The designation also qualified the drugs for five years of marketing exclusivity to be added to certain exclusivity already provided by the Food, Drug, and Cosmetic Act. To date, FDA has granted the QIDP designation to 39 antibiotics under development.

The Biomarkers Consortium of the Foundation for the National Institutes of Health, academic and industry experts, and other contributors made valuable recommendations to the FDA regarding designing scientifically sound studies to show the effectiveness of these drugs in clinical trials. More at link above. (FDA.gov)

The FDA Drug Shortage Assistance Award… Recognizing manufacturers who help prevent or alleviate drug shortages
As part of its continuing commitment to prevent drug shortages and minimize their impact on public health, FDA has launched the FDA Drug Shortage Assistance Award.  In his September 24, 2014, FDA Voice blog, Dr. Douglas Throckmorton writes:

“This award recognizes efforts of drug manufacturers who have cooperated with FDA and implemented strategies to help provide medically necessary drugs in short supply for patients, while maintaining federally mandated quality standards.  

“The FDA Drug Shortage Assistance Award is given to drug manufacturers who, among other factors, have demonstrated a strong commitment to preventing or alleviating a shortage of a medically necessary drug, by: Taking one or more actions to alleviate or prevent a drug shortage, such as: increasing production or submitting an application for approval of a drug in shortage; making a significant impact on public health; and using a facility that was substantially compliant with current good manufacturing practice (CGMP) for at least one inspection prior to the intervention and during the time the candidate used it to manufacture the shortage drug.”

The first recipients of the award: Guerbet Group for helping to alleviate the shortage of ethiodized oil injection, an imaging agent for certain forms of liver cancer, and Clinigen Group plc, for helping to ensure supplies of a medication needed for patients with AIDS who also have a serious eye condition called CMV (cytomegalovirus) retinitis.

FDA plans to continue to recognize manufacturers with this award based on their ability to meet the criteria. More at link above. (FDA.gov)

FDA Celebrates 30 Years of Easier Access to Cost-Saving Generic Drugs
In her September 24, 2014, FDA Voice blog, Dr. Margaret Hamburg saluted the vision of Senator Hatch and Representative Waxman in creating their landmark legislation, the Drug Price Competition and Patent Term Restoration Act of 1984, better known today as the Hatch-Waxman Amendments. She writes, “This law, championed by Senator Orrin Hatch and Representative Henry A. Waxman, made it easier for generic drugs to enter the market, and has greatly expanded access to important—often life-saving—drugs. Over the 10-year period 2003 through 2012, generic drug use is estimated to have generated more than $1.2 trillion in savings to the health care system and to have benefitted the health and well-being of innumerable lives.”

“One of the strengths of this law is the fact that it provided financial incentives for pharmaceutical companies that develop and manufacture new and innovative trade name products. Under the law, sponsors of qualifying trade name drugs are provided an opportunity to extend a patent to make up for patent life lost during the process of testing and approval of the product. The law also, however, provided a clear pathway to market for generic drugs.”

“Despite the enormous success of Hatch-Waxman, FDA faces challenges as we continue efforts to ensure access to affordable and quality generic drugs.”  Dr. Hamburg credits GDUFA (the Generic Drug User Fee Amendments of 2012, with providing the resources to reduce the backlog of generic drug applications and to help FDA monitor production of ingredients around the globe to assure drug safety. More at link above.  (FDA.gov)

NIH and FDA win top award for intellectual property licensing of meningitis vaccine
The National Institutes of Health and the U.S. Food and Drug Administration will receive a top national award for the year’s most outstanding intellectual property licensing deal, for technology transfer of a pioneering, low-cost meningitis vaccine launched in sub-Saharan Africa. The 2014 Deals of Distinction Award will be presented to the two federal agencies and their collaborators by the Licensing Executives Society at the society’s 50th annual meeting, Oct. 5-8 in San Francisco.

NIH and the FDA teamed with PATH, a Seattle-based non-profit leader in global health innovation, and the Serum Institute of India (SII) to develop MenAfriVac. The vaccine has a low production cost and does not require constant refrigeration, making it ideal for use in remote locations. A critical part of the manufacturing process for the vaccine is based upon a patent license granted from the NIH Office of Technology Transfer (NIH OTT) to PATH. The technology was invented by FDA scientists and subsequently sublicensed by PATH to SII under the Meningitis Vaccine Project, a partnership of PATH and the World Health Organization (WHO). The vaccine targets the most common form of bacterial meningitis, known as serogroup A, found in sub-Saharan Africa. According to WHO, 80 to 85 percent of all meningitis infections in the region are from group A.  More at link above.  (NIH.gov)


SECTION 3 AGENCY AND ADVISORY COMMITTEE MEETINGS

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

New: FDA Webinar. Inclusion of Minorities in Clinical Trials (FDASIA 907). October 7, 2014. 1:00 – 2:00 PM EDT.  (Online). Increasing participation in clinical trials  is a multi-faceted challenge that will require collaboration of our federal partners, industry, health care professionals, patient advocacy groups and community-based organizations.  This meeting is the first in an ongoing discussion with stakeholder groups regarding the agency’s efforts in this area. Register by Oct. 6. [FDA.gov]

Public Workshop. Additive Manufacturing of Medical Devices: An Interactive Discussion on the Technical Considerations of 3–D Printing. October 8-9, 2014. White Oak Campus, Silver Spring, MD.  The purpose of this workshop is to provide a forum for FDA, medical device manufactures, additive manufacturing companies, and academia to discuss technical challenges and solutions of 3–D printing. The Agency would like input regarding technical assessments that should be considered for additively manufactured devices to provide a transparent evaluation process for future submissions. [Federal Register]

New:  Public Workshop (and Request for Comments). Collaborative Approaches for Medical Device and Healthcare Cybersecurity. October 21, 2014. National Intellectual Property Rights Coordination Center Auditorium, Arlington, VA. FDA, in collaboration with other stakeholders within the Department of Health and Human Services (HHS) and the Department of Homeland Security (DHS), seeks broad input from the Healthcare and Public Health (HPH) Sector on medical device and healthcare cybersecurity. The vision for this public workshop is to catalyze collaboration among all HPH stakeholders. [Federal Register]

Public Meeting.  Patient-Focused Drug Development and Scientific Workshop on Female Sexual Dysfunction.   October 27-28, 2014.  White Oak Campus, Silver Spring, MD.  FDA is conducting a Patient-Focused Drug Development public meeting and scientific workshop on Female Sexual Dysfunction (FSD).  [Federal Register]

Day 1 – October 27th Patient-Focused Drug Development Public Meeting
FDA is interested in obtaining patient input on:

  1. The impact of the most common form of FSD, female sexual interest/arousal disorder (FSIAD) on daily life
  2. Patients’ views on currently available therapies to treat the condition

Day 2 – October 28th Scientific Workshop
The scientific workshop will include discussion on scientific challenges related to:

  1. Diagnosis of the condition for clinical trials and in clinical practice
  2. Ensuring valid patient-reported outcome measures for the key efficacy endpoints used in clinical trials

Public Meeting.  Development and Regulation of Abuse-Deterrent Opioid Medications.  October 30-31, 2014.  Silver Spring, MD.  The purpose of the meeting is to discuss the development, assessment, and regulation of abuse-deterrent formulations of opioid medications. The meeting will focus on scientific and technical issues related to the development and in vitro assessment of these products, as well as FDA’s approach towards assessing the benefits and risks of all opioid medications, including those will abuse-deterrent properties. [FDA.gov]

Public Advisory Committee Meeting. Risk Communications Advisory Committee. November 3-4, 2014. White Oak Campus, Silver Spring, MD.  The Risk Communication Advisory Committee will discuss methods for effective risk communication with a focus on messages about the importance of eating adequate amounts of fish, while avoiding certain fish with higher amounts of methyl-mercury. These messages are especially important for women who are pregnant or nursing, or for anyone who prepares food for young children. [Federal Register]

Clinical Investigator Training Course. November 4-6, 2014. College Park, MD.  This training course, a co-sponsored event of FDA CDER and Duke University Office of Continuing Medical Education, is intended to provide clinical investigators with expertise in the design, conduct, and analysis of clinical trials; improve the quality of clinical trials; and enhance the safety of trial participants.  [Federal Register]

Public Advisory Committee Meeting. Cellular, Tissue and Gene Therapies Advisory Committee. November 6, 2014. White Oak Campus, Silver Spring, MD. The committee will discuss the draft guidance for industry entitled ‘‘Design and Analysis of Shedding Studies for Virus or Bacteria-Based Gene Therapy and Oncolytic Products’’ and the Dear Gene Therapy IND or Master File Sponsor Letter.  [Federal Register]

Public Workshop. Brain-Computer Interface Devices for Patients With Paralysis and Amputation. November 21, 2014.  White Oak Campus, Silver Spring, MD.  The purpose of this workshop is to obtain public feedback on scientific, clinical, and regulatory considerations associated with BCI (Brain-Computer Interface) devices. Ideas and suggestions generated during this workshop may facilitate development of draft guidance to provide our initial thoughts regarding the content of premarket submissions for emerging BCI technologies to help speed development and approval of future submissions.  [Federal Register]


SECTION 4 OTHER REGULATORY AUTHORITIES & ORGANIZATIONS

American Association for Cancer Research Releases 2014 Cancer Progress Report: Research is Transforming Lives
Research continues to fuel significant advances against cancer. This progress has been spurred by federal investments in the National Institutes of Health (NIH) and National Cancer Institute (NCI), according to the American Association for Cancer Research's (AACR) fourth annual Cancer Progress Report, released September 16, 2014.

The AACR Cancer Progress Report 2014 is a comprehensive educational tool that chronicles the progress that has been made against cancer; details how federal investment in the NIH and NCI is transforming lives.  It calls on the administration and Congress to prioritize the growth of the NIH and NCI budgets at a predictable, robust pace by providing annual budget increases at least comparable to the biomedical inflation rate.

The report states that although extraordinary advances are being made against cancer, the disease remains a major health care challenge and a huge financial burden, both nationally and internationally.

Moreover, because most cancer diagnoses occur in those who are 65 years of age and older, a segment of the U.S. population that is expected to double by 2060, it is predicted that the number of cancer diagnoses will increase dramatically in the future. More at link above.  (AACR.org)

Cancer Progress Requires Integrating Data And Therapeutics – AACR Report
Executive Summary: As genomic analysis produces ever-increasing amounts of data on cancer biology, continued progress in oncology will require advances in managing and analyzing data and new approaches to clinical trial design to increase efficiency and address development of treatment resistance to molecularly targeted therapies, the American Association for Cancer Research asserts in its annual Cancer Progress report.

Continued progress against cancer will require the ability to creatively manage and exploit the gusher of data coming from advanced genomic technologies into both therapeutic development and cancer care, the AACR emphasizes in the report.

“Technological advances in DNA sequencing have dramatically increased the number of known cancer-associated genomic alterations,” and continued progress “will multiply many times over the number of molecules that could provide a potential target for anticancer therapies.”

What’s needed now is much more work in computational biology, report concludes, as well as innovative trial designs like umbrella and basket trials.  More at link above. (The Pink Sheet, paid subscription required.)

NIH awards $35 Million for Centers for Collaborative Research in Fragile X
The National Institutes of Health is making funding awards of $35 million over the next five years to support the Centers for Collaborative Research in Fragile X program. Investigators at these centers will seek to better understand Fragile X-associated disorders and work toward developing effective treatments.

Fragile X syndrome, Fragile X-associated tremor/ataxia syndrome (FXTAS), and Fragile X-associated primary ovarian insufficiency (FXPOI), along with other related conditions, can cause major health problems of concern to individuals and families.

Fragile X syndrome is the most common form of inherited intellectual and developmental disabilities, affecting approximately 1 in 4,000 males and 1 in 8,000 females. People with Fragile X syndrome may have disabilities ranging from mild to severe, as well as emotional and behavioral problems. As many as 30 to 50 percent of people with Fragile X syndrome also have features of autism spectrum disorders.  More at link above.  (NIH.gov)

NIH Funds Next Phase of Tissue Chip for Drug Screening Program
The National Institutes of Health will award funds to support the next phase of its Tissue Chip for Drug Screening program to improve ways of predicting drug safety and effectiveness. Researchers will collaborate over three years to refine existing 3-D human tissue chips and combine them into an integrated system that can mimic the complex functions of the human body. Led by the National Center for Advancing Translational Sciences (NCATS), the program will support 11 institutions at $17 million in 2014 with additional support over the remaining two years if funds are available.

“The development of tissue chips is a remarkable marriage of biology and engineering, and has the potential to transform preclinical testing of candidate treatments, providing valuable tools for biomedical research,” said NIH Director Francis S. Collins, M.D., Ph.D.

Approximately 80 percent of candidate drugs fail in human clinical trials because they are found to be unsafe or ineffective. More than 30 percent of promising medications fail due to toxicity, despite promising preclinical studies in animal and cell models. These models can be costly and poor predictors of drug response in humans.  More at link above. (NIH.gov)

NIH exceptional responders to cancer therapy study launched
The Exceptional Responders Initiative, a study to investigate the molecular factors of tumors associated with exceptional treatment responses of cancer patients to drug therapies, was launched September 24, 2014, by the National Cancer Institute (NCI). Scientists will attempt to identify the molecular features of tumors that predict whether or not a particular drug or class of drugs will be beneficial. Investigators will examine tumor specimens from patients in clinical trials who achieved an exceptional response relative to other trial participants, or other patients who achieved an exceptional and unexpected response to a non-investigational therapy.

In this study, some of the tissue and clinical data from exceptional responders will be obtained from NCI-supported trials as well as potentially other clinical trials. The remaining samples and data will come from standard therapy settings, such as community practice, where there are reliable outcome data, and from pharmaceutical industry trials or other sources. Consequently, letters of solicitation are being sent to cancer centers and others clinicians nationwide to ask them to assist in this effort.  More at link above. (NIH.gov)

NIH announces network to accelerate medicines for rheumatoid arthritis and lupus
The National Institutes of Health has awarded grants to 11 research groups across the United States to establish the Accelerating Medicines Partnership in Rheumatoid Arthritis and Lupus (AMP RA/Lupus) Network. Launched in February of this year, the NIH AMP Program is a public-private partnership developed to transform the current model for identifying and validating the most promising biological targets for the development of new drugs and diagnostics. Through a competitive process, the AMP RA/Lupus Network Leadership Center and Research Sites were selected, and $6 million of first-year funding was awarded on Sept. 24, 2014. The network will implement the goals of the broader AMP RA/Lupus Program.

Over five years, the AMP RA/Lupus Network will analyze the interplay among biological pathways, including at the single cell level, in tissues of patients with RA and lupus. The goal is to integrate data from multiple genome-wide analytic approaches to generate a comprehensive understanding of the mechanisms of tissue damage in RA and lupus. More at link above. (NIH.gov)

PCORI Chief Science Officer to Retire
Patient-Centered Outcomes Research Institute (PCORI) Chief Science Officer Bryan Luce, PhD, MBA, who led the development and implementation of PCORI’s comparative clinical effectiveness research (CER) agenda over the past 18 months, today announced his intention to retire within the next six to 12 months. PCORI will initiate a search for his replacement immediately.

“As PCORI’s first Chief Science Officer, Bryan brought his scientific insights and leadership skills to support rapid growth in our scientific staff, and to develop structures and processes for building and managing our research portfolio of patient-centered CER projects,” said PCORI Executive Director Joe Selby, MD, MPH.

“Thanks in large part to Bryan’s leadership, we have a vibrant science department and a large and growing research portfolio that will improve outcomes of importance to patients.” More at link above. (PCORI.org)

Engaging Patients: Interviews with Patients, Providers and Communities Across the Country
Health Affairs has partnered with the Patient Centered Outcomes Research Institute (PCORI) to produce three videos about ways patients and practitioners are incorporating patient engagement in health care decisions.  “Teaching them, soliciting their input, and communicating with family-members and other caregivers right from the start – can result in better, more efficient health care outcomes,” says John Dimsdale, who hosts and reports the videos, in a September 25, 2014 blog.

“I was privileged to report on and host these videos on the new era of patient engagement. I interviewed nearly two dozen people across the country – patients, doctors, researchers, care-providers, academics and community activists – all of whom have inspiring stories to tell about the benefits of focusing on patient centered outcomes. Time constraints meant that many of their important points landed on the cutting-room floor during the video editing process. It’s another privilege to be able to offer some of those additional insights in this blog.”  More at link above.  (Health Affairs.org)

High-Tech Matchmaking: PCORI Rewards Apps that Connect Patients and Researchers
Announced by PCORI (Patient Centered Outcomes Research Institute) on September 23, 2014:

To speed development of apps and websites that can help bring the voice of patients and others into the research process, we launched the 2014 PCORI Matchmaking App Challenge last March. We have just announced the winners at the Health 2.0 Fall Conference in Santa Clara, California.

In the Matchmaking App Challenge, PCORI invited developers to submit novel and creative submissions of fully functioning, polished, ready-to-publish apps or mobile websites to facilitate partnerships for conducting patient-centered outcomes research. In true PCORI fashion, developers were required to work with patients and others in the design of their apps.

The first prize of $100,000 went to Sean Ahrens, the founder of Crohnology.com, who entered an open source, web-based, mobile-responsive application called PatientPowered.us. It is a network that connects patients with others who care about solving their health problems. Patients, health professionals, and scientists propose research ideas, in text or video, and everyone comments on those ideas, eventually building collaborations.  More at title link above.  (PCORI.org)


Characterizing and Communicating Uncertainty in the Assessment of Benefits and Risks of Pharmaceutical Products: Workshop Summary
Despite the extensive body of evidence that informs regulatory decisions on pharmaceutical products, significant uncertainties persist. As a result, regulatory reviewers are consistently required to draw conclusions about a drug’s safety and efficacy from imperfect data. Efforts are underway within the drug development community to enhance the evaluation and communication of the benefits and risks associated with pharmaceutical products, aimed at increasing the predictability, transparency, and efficiency of pharmaceutical regulatory decision making.

On February 12 and May 12, 2014, the Institute of Medicine’s (IOM’s) Forum on Drug Discovery, Development, and Translation held public workshops at FDA Headquarters in White Oak, MD, to advance the development of more systematic and structured approaches to characterize and communicate the sources of uncertainty in the assessment of benefits and risks, and to consider their implications for pharmaceutical regulatory decisions. This report is a summary of the February 12 and May 12, 2014 workshops. (Institute of Medicine)


Integrating Research and Practice: Health System Leaders Working Toward High-Value Care: Workshop Summary
Driven by robust innovation in treatments and interventions, and by increased insights about how people vary in what works best for them, traditional approaches to clinical research are straining to keep pace with the demands. The gap might even be growing between the evidence we have and the evidence we need for best care.  Improvements in electronic records and computing power, as well as novel research designs offer the prospect of drawing real-world practice and new evidence development much closer together.

In April and June 2014 the Institute of Medicine’s (IOM) Roundtable on Value & Science-Driven Health Care convened two workshops aimed at accelerating progress toward real-time knowledge generation through the seamless integration of clinical practice and research, one of the fundamental concepts of a continuously learning health system.

This publication summarizes the presentations and discussions that occurred during the two workshops, highlighting the key lessons presented, practical strategies, and the needs and opportunities for future leadership. More at link above.  (Institute of Medicine)

USP endorses INN suffix for biologics
The U.S. Pharmacopeial Convention (USP) endorsed a draft World Health Organization (WHO) proposal to create a unique suffix for biologic drugs, including biosimilars.

A WHO working group proposed a voluntary scheme to create unique identification codes, called "biological qualifiers," that would be distinct from international non-proprietary names (INN). The BQs would be random four-letter codes that would be linked in a WHO-maintained database to the product's INN, trade name, name and address of the manufacturer, and regulatory status. According to the WHO, the scheme could facilitate decision making about substitution and interchangeability and help regulators and physicians track patients' responses to products with the same INN.

In comments submitted to WHO, USP suggested that the BQ system be applied to all biologics, not just biosimilars. BQ codes could be assigned retrospectively.

The endorsement comes as FDA is finalizing its biosimilars naming policy in anticipation of regulatory decisions on at least two pending biosimilar applications.  (BioCentury)

Ebola outbreak: EMA to review experimental medicines to support treatment decisions
EMA) has started to review available information on Ebola treatments currently under development. The goal is to provide an overview of the current state of knowledge about the various experimental medicines to support decision-making by health authorities.

At the moment, there are no approved medicines to protect from or treat Ebola. Medicines against this disease are still at an early stage of development. Some experimental treatments against Ebola have reportedly shown encouraging results in the laboratory or in animals, but they have not yet been fully studied in people.

“Health authorities or practitioners who need to take a decision whether or not to use an experimental Ebola treatment in a patient are currently lacking independent information,” explains Professor Guido Rasi, EMA Executive Director. “I have therefore asked the EMA Committee for Medicinal Products for Human Use, CHMP, to scrutinize all the available information about experimental treatments and compile everything we know to date about their efficacy, safety and quality. This will facilitate evidence-based decision-making.”  More at link above. (EMA.europa.eu)

Avoiding Duplication of Clinical Trials in Children
Proposed single development plan for tetanus-diphtheria-pertussis vaccines is released for public consultation: The European Medicines Agency is proposing a single development plan that pharmaceutical companies should follow when developing new vaccines to protect children against diphtheria, tetanus and acellular pertussis (whooping cough) across the European Union (EU). The proposal aims to harmonise the way these vaccines are being developed in order to avoid the duplication of similar clinical trials and the unnecessary exposure of children to clinical testing.  More at link above. (EMA.europa.eu)

Patients to discuss benefit-risk evaluation of medicines with the Committee for Medicinal Products for Human Use
EMA launches pilot project to integrate patients’ unique and critical views into CHMP discussions: The European Medicines Agency (EMA) has launched a pilot project to involve patients in the assessment of the benefits and risks of medicines in its Committee for Medicinal Products for Human Use (CHMP).

Listening to patients enriches the scientific assessment of a medicine with new ways of thinking about benefit and risk from the patient perspective. This pilot project marks the next step in bringing patients’ views and values to the assessment of medicines throughout their lifecycle.

As part of the pilot project, patients will be invited to present their views on medicines for which there is an unmet medical need and where the Committee still has concerns or doubts. Patients may also be invited to give their views in cases where the Committee is considering whether to recommend the withdrawal, suspension or revocation of a marketing authorisation, or a restriction of indication of an authorised medicine.  More at link above.  (EMA.europa.eu)

Australia TGA Opens Consultation on Adoption of European Union Guidelines

The Australian Therapeutic Good Administration (TGA) has opened a consultation on whether or not certain European Union guidelines should be adopted in Australia.  The TGA website lists those guidelines that are recommended for adoption and those that are recommended for non-adoption.  Certain quality guidelines, biological medicines guidelines, clinical safety and effectiveness guidelines, nonclinical guidelines, and pharmacovigilance guidelines are recommend for adoption.  See full listings and discussion at link above. (TGA.gov.au)

New TGA Web Service Helps Consumer Reporting of Side Effects
The TGA has launched a new web-based service to make it easier for consumers to report adverse events associated with medicines and vaccines.

It is generally acknowledged that adverse events are under-reported around the world, with estimates that 90-95% of adverse events are not reported to regulators. The new web-based reporting service for consumers is one of a range of initiatives being implemented by the TGA to encourage reporting among this group. More at link above. (TGA.gov.au)


SECTION 5 LEGAL AND COMPLIANCE

Sunshine Database: Trade Groups Call for Transparency
With just one week left before the launch of the Open Payments database – which will reveal how much money doctors receive from drug and device makers – three industry trade groups, PhRMA, BIO, and AdvaMed, are complaining they have not had an opportunity to review important background information about relationships with physicians.  They have also reiterated concerns that CMS has yet to explain why one-third of the payment information submitted by drug/device makers and group purchasing organizations has been removed from the database.  Their concern is that the absence of this data may affect the accuracy, completeness, and usefulness of the overall data.

The database, which will be launched on Sept. 30, was created under the Sunshine Act provision of the Affordable Care Act and aims to mitigate concerns that industry payments to doctors may unduly influence medical practice and research. Initially, the database will display payments made in the last five months of 2013 and will be updated going forward.  More at link above.  (Pharmalot, Wall Street Journal)

Physician Payments Sunshine Act: Bipartisan Members of Congress Introduce Bill to Exclude Educational Materials From Reporting, Including CME and Medical Texts
Two bipartisan efforts in Congress underscore the importance of up-to-date medical education. On Thursday, Reps. Michael Burgess (R-TX) and Frank Pallone (D-NJ) sent a letter to the Centers for Medicare and Medicaid Services (CMS), to voice concern about the agency's plan to eliminate the continuing medical education (CME) exemption from the Sunshine Act. On Friday, Reps. Burgess and Allyson Schwartz (D-PA) introduced bipartisan legislation (H.R. 5539) to exempt medical textbooks and journals, as well as indirect payments that pharmaceutical and device manufacturers offer to CME providers, from Sunshine Act reporting requirements.  More at link above. (Policy and Medicine)

House passes bill to repeal, refund medical device tax
The U.S. House of Representatives last week passed a jobs package that would repeal the medical device tax and issue refunds for all levies paid since the 2.3% excise tax went into effect last year. More at link above.  (Mass Device, subscription required)

A Bill Would Speed DEA Listings for Controlled Substances
For the second time this year, a bill has been introduced in Congress to streamline the process used by the U.S. Drug Enforcement Agency to place prescription drugs that are controlled substances on a list of medicines for which distribution is restricted.  The “Regulatory Transparency, Patient Access, and Effective Drug Enforcement Act of 2014” was introduced in the Senate by Senator Orrin Hatch (R-Utah).

The goal is to get these medicines to the market faster, since controlled substances must first be placed on the DEA list before they can reach pharmacy shelves. Drug makers have complained that the process can be lengthy and hamper their ability to take advantage of an important five-year marketing exclusivity period following FDA approval in which generic competition can be kept at bay.  More at link above. (WSJ Pharmalot)

Dems propose increased NIH funding
Reps. Rosa DeLauro (D-Conn.) and Brian Higgins (D-N.Y.) introduced a bill in the U.S. House of Representatives that would lift the cap on NIH funding due to sequestration.

The Accelerating Biomedical Research Act, H.R. 5580, would allow annual appropriations to NIH in excess of $29.9 billion to trigger a budget cap increase. If the bill is enacted, the agency's funding could increase incrementally, with 10% hikes in the first two years followed by 5% bumps thereafter.

According to its sponsors, the bill is designed to restore NIH purchasing power to 2003 levels, adjusted for inflation.  (BioCentury)

House Dems, Funding Advocates Push NIH Funding Bump Despite GOP Opposition
9/24, InsideHealthPolicy.com) reports “...A coalition of advocates for biomedical research joined key House Democrats this week in pushing for increased funding for the National Institutes of Health and warned that the United States will fall behind in biomedical innovation if Congress continues to cut or flat-line the agency's budget...Yet House Republicans seemed to nix the idea of increasing NIH funding...The GOP members proposed instead that the administration pull funds from other organizations, like the ACA-created Patient-Centered Outcomes Research Institute, to bolster funding for NIH.” More at link above.  (Inside Health Policy, paid subscription required)

OIG calls for federal healthcare coupon safeguards

HHS's Office of the Inspector General said current safeguards are insufficient to prevent consumers from using coupons to fund drug copayments to federal healthcare programs such as Medicare Part D.

In a report, OIG found that although manufacturers took precautions to prevent the improper use, coupons could still be used to pay for Medicare Part D drugs. The report said manufacturers are at risk of violating anti-kickback laws and may be subject to sanctions if they do not take appropriate steps to prevent unpermitted use.  More at link above.  (BioCentury)


SECTION 6 SOURCES REVIEWED FOR THIS NEWSLETTER

A partial listing of sources reviewed for this newsletter:  AdvaMed Smartbrief; AHRQ Newsletter; Alzheimers Association; Alzheimers Research Forum Newsletter; BioCentury; Biopharma Reporter; BIOtechNow; CDISC Monthly Newsletter; CER Daily Newsfeed (NPC); Daily Dose (Becker); DIA Daily; Drug Daily Bulletin;  EMA website; EP Vantage; Evaluate Pharma; Eye on FDA; FDA.gov; FDA Law Blog; Federal Register Table of Contents; Fierce Medical Devices; Fierce Pharma; Fierce Vaccines; FDLI Smartbrief; Genomeweb; Health Industry Washington Watch; Government Health IT; Health IT Security; Institute of Medicine News; MedCityNews; Medical Device Daily; Medical Device & Diagnostic Industry; MedPage Today; NPC Bulletin; Nutra Ingredients USA; Pharmabiz; Pharmafile; Pharma IQ; PharmaTimes; PhRMA website; PM Live; Policy and Medicine (newsletter); Regulatory Focus; RegLink News; US FDA Daily Digest Bulletin.