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DIA's Content Currents provides you with new and important global regulatory developments and their impact on pharmaceutical, biotechnology, and medical product development.

EDITION PUBLISHED: April 11, 2014

SECTION 1 FDA GUIDANCES & MAPPS

CDER 2014 New & Revised Draft Guidance Agenda

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

CDRH FY 2014 Proposed Guidance Development

On April 9, 2014, FDA announced the establishment of a docket to receive suggestions, recommendations, and comments on innovative packaging, storage, and disposal systems, technologies or designs (‘‘designs’’) that could be used to prevent or deter misuse and abuse of opioid analgesics by patients and others. FDA is interested in receiving comments on new designs as well as enhancements to existing designs, and is particularly interested in comments from academic institutions, regulated industry, technology companies (e.g., those producing technologies for medication adherence, disposal, or tracking), healthcare professionals, patient representatives, clinical trial service providers, and other interested organizations.  Comments are requested by June 9, 2014.  [Federal Register]

On April 9, 2014, FDA announced the availability of a draft guidance entitled ‘‘Immunogenicity-Related Considerations for the Approval of Low Molecular Weight Heparin for NDAs and ANDAs.’’ This guidance discusses how applicants for low molecular weight heparin (LMWH) products should provide information on impurities and the potential impact on immunogenicity. To ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance, submit either electronic or written comments on the draft guidance by June 9, 2014. [Federal Register]

On April 9, 2014, FDA announced the extension to May 7, 2014, the comment period for the notice that appeared in the Federal Register of January 7, 2014 (79 FR 830). In the notice, FDA requested comments on a draft guidance document entitled ‘‘Blood Glucose Monitoring Test Systems for Prescription Point-of-Care Use.’’ The Agency is taking this action in response to requests for an extension to allow interested persons additional time to submit comments. [Federal Register]

On April 9, 2014, FDA announced the extension to May 7, 2014, the comment period for the notice that appeared in the Federal Register of January 7, 2014 (79 FR 830). In the notice, FDA requested comments on a draft guidance document entitled ‘‘Self-Monitoring Blood Glucose Test Systems for Over-the-Counter Use.’’ The Agency is taking this action in response to requests for an extension to allow interested persons additional time to submit comments. [Federal Register]

On April 9, 2014, FDA announced the availability of a draft guidance for industry, researchers, patient groups, and FDA staff entitled ‘‘Meetings With the Office of Orphan Products Development.’’ This draft guidance provides recommendations to industry, researchers, patient groups, and other stakeholders (collectively referred to as ‘‘stakeholders’’) interested in requesting a meeting with FDA’s Office of Orphan Products Development (OOPD) on issues related to orphan drug designation requests, humanitarian use device (HUD) designation requests, rare pediatric disease designation requests, funding opportunities through the Orphan Products Grants Program and the Pediatric Device Consortia Grants Program, and orphan product patient related topics of concern. This draft guidance document is intended to assist these groups with requesting, preparing, scheduling, conducting, and documenting meetings with OOPD. To ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance, submit either electronic or written comments on the draft guidance by June 9, 2014. [Federal Register]

On April 14, 2014, FDA announced that a proposed collection of information has been submitted to OMB relating to the Adverse Event Program for Medical Devices (Medical Product Safety Network).   FDA is seeking OMB clearance to continue to use electronic data collection to obtain the information on Form FDA 3500A (approved under OMB control number 0910–0291) related to medical devices and tissue products from the user facilities participating in MedSun, to obtain a demographic profile of the facilities, and for additional questions which will permit FDA to better understand the cause of reported adverse events. Participation in the program is voluntary and currently includes 250 facilities. In addition to collecting data on the electronic adverse event report form, MedSun collects additional information from participating sites about reported problems emerging from the MedSun hospitals. This data collection is also voluntary and is collected on the same Web site as the report information.  Comments may be submitted for consideration by May 14. 2014. [Federal Register]

SECTION 2 FDA NOTES & RELATED NEWS

Hamburg to PhRMA: If You Don't Have Quality, the Rest Doesn't Matter

Speaking at the PhRMA's annual conference in Washington, DC, Hamburg highlighted recent reasons for concern that quality standards are not being adhered to--as well as the agency's renewed focus on enforcing those standards.

"We are … trying to really reshape some of how we address the quality issue, to try to incentivize better practice and more accountability around quality," she said. " … And I think this is the time when we need to be recommitting to it."

Part of that, she said, has involved creating a new Office of Pharmaceutical Quality and putting it on par organizationally with other critical offices, like those for new drugs and safety. Another part has been recognizing the need to become a global agency--one that not only has offices and inspectors on the ground around the world, but also has working relationships with counterpart regulators, harmonized regulatory approaches with those counterparts, and a new kind of partnership with the industry. More at link above. (Fierce Pharma)

Advisory Committee for Reproductive Health Drugs Changes Charter and Name
FDA) is amending the standing advisory committees’ regulations to change the name and function of the Advisory Committee for Reproductive Health Drugs. This action is being taken to reflect changes made to the charter for this advisory committee. The Agency decided that the name ‘‘Bone, Reproductive and Urologic Drugs Advisory Committee’’ more accurately describes the subject areas for which the committee is responsible. The committee reviews and evaluates data on the safety and effectiveness of marketed and investigational human drug products for use in the practice of osteoporosis and metabolic bone disease, obstetrics, gynecology, urology and related specialties, and makes appropriate recommendations to the Commissioner of Food and Drugs. (Federal Register)

Recognizing Those Who Strive to Vanquish Alzheimer’s Disease

Excerpts from Dr. Margaret Hamburg’s’ blog in the April 9 FDA Voice:  “Only last month new research was published that suggests that deaths due to Alzheimer’s disease in the United States actually have been severely under-recognized. By these new estimates, Alzheimer’s disease may rank as the third leading cause of death, trailing only behind heart disease and cancer. What’s even more alarming is that it is the only cause among the top 10 without a way to meaningfully prevent, treat, or slow its progression.

“Fortunately, in stark contrast to these disturbing figures, the commitment of the countless individuals entrenched in the fight against this epidemic remains resolute. The FDA shares in this determination and holds the need to facilitate the discovery of effective treatments for Alzheimer’s disease among its highest priorities.

“In February 2013, the FDA published a draft guidance document responding to the shifting focus of the research community towards the earlier stages of Alzheimer’s disease. The hope is that intervening earlier in the disease process (before the onset of dementia) may provide a greater opportunity to alter the course of the condition. Given that these are uncharted waters, it is essential that we provide as much clarity as possible. Of particular note, the guidance discusses the possible use of the accelerated approval pathway as a means of approving drugs for patients in the earliest stages of the disease.” More at link above.  (FDA.gov)

Solomon Becomes Permanent Regulatory Affairs Deputy
FDA named Steven Solomon as deputy associate commissioner for regulatory affairs for FDA's Office of Regulatory Affairs. Solomon has been acting deputy associate commissioner since January 2013. He will step down from his concurrent role as associate director for global operations and policy in the Office of Global Regulatory Operations and Policy, which houses the Office of Regulatory Affairs. (BioCentury)

FDA Stops Issuing National Health Related Items Code (NHRIC) and National Drug Code (NDC) Labeler Codes
This week, FDA announced it will no longer issue National Health Related Items Code (NHRIC) and National Drug Code (NDC) labeler codes to manufacturers for use with medical devices to conform with changes made under the Unique Device Identification (UDI) final rule.  The changes correspond to the compliance dates for UDI requirements, which will replace the NHRIC and NDC.  (FDA.gov)

Internet Ad Spending Beats Broadcast TV Last Year
For the first time, it was reported by Mashable that revenue spent on advertising on the Internet surpassed that spent on broadcast television.  Not sure if that is true for the pharma industry or not, but it certainly signals that if not yet, it will likely be soon.  And for FDA, it signals that enforcement efforts, long centered by OPDP on broadcast, need to be approaching their efforts in new ways, taking into consideration a lot of new factors. (Eye on FDA)

SECTION 3 AGENCY AND ADVISORY COMMITTEE MEETINGS

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

Public Advisory Committee Meeting.  Neurological Devices Panel of the Medical Devices Advisory Committee. April 24, 2014. Gaithersburg, MD. The committee will discuss the current knowledge about the safety and effectiveness of aversive conditioning devices that are intended to deliver a noxious electrical stimulus to a patient to modify undesirable behavioral characteristics. FDA is convening this committee to seek clinical and scientific expert opinion on the risks and benefits of certain aversive conditioning devices based on available scientific data and information. [Federal Register]

Public Meeting. Meeting for Software Developers on the Common Formats for Patient Safety Data Collection and Event Reporting. April 25, 2014. Rockville, MD. Convened by AHRQ, this meeting is designed as an interactive forum where Patient Safety Organizations (PSOs – formed under the Patient Safety and Quality Improvement Act) and software developers can provide input on the formats. AHRQ especially requests participation by and input from those entities which have used AHRQ's technical specifications and implemented, or plan to implement, the formats electronically. (Federal Register)

Public Advisory Committee Meeting.  Risk Communications Advisory Committee. May 5-6, 2014.  FDA White Oak Campus, Silver Spring, MD.  The committee will meet to discuss methods for identifying the impact and increasing the reach of communications on topics of interest to consumers. The discussion will also address how FDA can evaluate whether its Consumer Updates (ForConsumers/ConsumerUpdates) are reaching the targeted population, and whether they are increasing awareness and understanding of the key risk messages. The discussion will also assess whether the communications are having the intended impact on knowledge, behaviors and/or outcomes. [Federal Register]

New: Public Workshop. Standards for the Interoperable Exchange of Information for Tracing of Human, Finished, Prescription Drugs, in Paper or Electronic Format.  May 8-9, 2014.  FDA White Oak Campus, Silver Spring, MD.  This public workshop will provide a forum for discussing the development of these standards in the Drug Supply Chain Security Act of 2013. [Federal Register]

Public Meeting on Patient-Focused Drug Development: Pulmonary Arterial Hypertension.  May 13, 2014.  FDA White Oak Campus, Silver Spring, MD.  Patient-Focused Drug Development is part of FDA’s performance commitments in the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). The public meeting is intended to allow FDA to obtain patients’ perspectives on the impact of pulmonary arterial hypertension on daily life, as well as their perspectives on the available therapies for pulmonary arterial hypertension.

Public Hearing. Generic Drug User Fee Amendments of 2012: Regulatory Science Initiatives.  May 16, 2014.  FDA White Oak Campus, Silver Spring, MD.  The 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 will take the information it obtains from the public hearing into account in developing the fiscal year (FY) 2015 Regulatory Science Plan. [Federal Register]

Public Meeting.  Study Approaches and Methods to Evaluate the Safety of Drugs and Biological Products During Pregnancy in the Post Approval Setting.  May 28-29, 2014. FDA White Oak Campus, Silver Spring, MD.  The purpose of the public meeting is to engage in constructive dialogue and information sharing among regulators, researchers, the pharmaceutical industry, public health agencies, health care providers, and the general public concerning challenges in designing and implementing pregnancy registries and other methods of evaluating the post-approval safety profile of drugs and biological products in pregnant women. The input from this meeting and public docket will be used to support the revision of a guidance for industry on establishing pregnancy exposure registries.  [Federal Register]

Symposium. 2014 Medical Countermeasures Initiative Regulatory Science Symposium. June 2-3, 2014. FDA White Oak Campus, Silver Spring, MD.  The symposium is intended to provide a forum for the exchange of scientific ideas for medical countermeasure development and evaluation, communicate progress on regulatory science efforts related to the development and advancement of medical countermeasures, facilitate innovative directions, and inform stakeholders on medical countermeasure-related scientific progress and accomplishments. [Federal Register]

New: Public workshop. Advancing the Development of Pediatric Therapeutics (ADEPT): Pediatric Bone Health. June 3, 2014. FDA White Oak Campus, Silver Spring, MD.  The purpose of this initial workshop is to provide a forum to consider issues related to advancing pediatric regulatory science in the evaluation of bone health in pediatric patients. [FDA.gov]

New: Public Workshop.  Immune Responses to Enzymes Replacement Therapies: Role of Immune Tolerance Induction.  FDA White Oak Campus, Silver Spring, MD. June 9, 2014. The workshop will provide a forum to discuss the role of immune tolerance induction in patients receiving replacement biological products. (FDA.gov)

Public meeting. Inborn Errors of Metabolism Patient-Focused Drug Development. June 10, 2014. FDA White Oak Campus, Silver Spring, MD.  This public meeting will address Patient-Focused Drug Development for neurological manifestations of inborn errors of metabolism. FDA is interested in obtaining patient input on the impact of the neurological manifestations of inborn errors of metabolism on daily life and patient views on treatment options. [FDA.gov]

Public Workshop. IOM Workshop: “Characterizing and Communicating Uncertainty in the Assessment of Benefits and Risks in Drug Regulatory Decision-Making.”  February 12-13, 2014.  The purpose of the workshop is twofold: To explore potential approaches to addressing and communicating uncertainty and to identify key considerations on developing, evaluating, and incorporating potential approaches for addressing uncertainty into the assessment of benefits and risks in the human drug review process. The format of the meeting consists of a series of presentations on topics related to uncertainty in the assessment of benefits and risks, followed by a discussion on those topics with invited panelists and audience members. This workshop satisfies an FDA commitment that is part of the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). Federal Register  Note: Due to impending inclement weather, DAY 2 of this meeting, February 13th, focusing on communication of uncertainty, will be postponed and rescheduled for the spring (tentatively April or May). The new workshop date will be announced via email and will be posted on the FDA and IOM meeting websites.

SECTION 4 OTHER REGULATORY AUTHORITIES & ORGANIZATIONS

Obama Bids Health Secretary Goodbye as He Names Successor
US Secretary of Health Kathleen Sebelius announced her resignation on April 10, 2014. President Barack Obama thanked Sebelius for helping pass the 2010 health-care law and named Sylvia Mathews Burwell, Office of Management and Budget Director, his new health secretary, calling her “a proven manager” who knows how “to deliver results.”  Among her credentials prior to serving in Washington, Burwell was president of the Walmart Foundation and the president of the Global Development Program of the Bill and Melinda Gates Foundation.  More at link above.  (Bloomberg News)
 
National Cancer Institute investigating trial outliers to resurrect 'failed' drugs

Last year the National Cancer Institute announced the launch of an initiative meant to examine failed clinical trials in search of "exceptional responders": patients who responded well to drugs that the bulk of the trial population didn't.

Now, after sifting through 10 years' worth of clinical trials, NCI's Cancer Diagnosis Program has identified about 100 of these unique patients so far, according to Bloomberg.

Memorial Sloan-Kettering Cancer Center in New York, Dana-Farber Cancer Institute and Massachusetts General Hospital in Boston and the Broad Institute in Cambridge, MA, have teamed up with NCI to look for these outlier patients and eventually establish a national database of patient data for researchers. More at link above. (FierceBiotech)


NIH stem-cell programme closes

Stem-cell researchers at the US National Institutes of Health (NIH) have been left frustrated and confused following the demise of the agency’s Center for Regenerative Medicine (CRM). The intramural programme’s founding director, stem-cell biologist Mahendra Rao, left the NIH, in Bethesda, Maryland, on 28 March and will join the not-for-profit New York Stem Cell Foundation (NYSCF) Research Institute. The centre’s website was taken down on 4 April.

NIH officials say that they are rethinking how they will conduct in-house stem-cell research. James Anderson, director of the NIH’s Division of Program Coordination, Planning, and Strategic Initiatives, which administered the CRM, says that the CRM will not continue in its current form. “The field is moving so fast that we need to rethink.” To that end, the NIH plans to hold a workshop in May to gather stem-cell researchers together and decide what to do with the programme and its remaining budget.

One option could be to allow CRM projects to be absorbed by the National Center for Advancing Translational Sciences, an NIH institute established in 2011 to translate basic research into therapies. But Anderson says that participants at the workshop will also discuss whether the NIH needs to replace the CRM with another dedicated stem-cell programme.  More at link above. (Nature)

GPhA and EGA Urge TTIP Negotiators to Adopt Sensible Measures to Streamline Drug Development and Approval Processes

In a joint letter sent April 3rd to the European Commission and the Office of the US Trade Representative, the Generic Pharmaceutical Association (GPhA) and European Generic medicines Association (EGA) recommended that Transatlantic Trade and Investment Partnership (TTIP) negotiators pursue a strategy of regulatory convergence. The letter focuses on specific ways that trade agreements can remove duplicative processes.

The letter reiterated five key recommendations shared by GPhA and EGA in an earlier presentation in Brussels during the fourth round of TTIP negotiations, including enhancing existing cooperation between the EMA and FDA on the convergence of biosimilar development pathways.  More at link above.  (GPhA)

NCPDP Issues Patient Safety Guidance on Dosing of Oral Liquid Medications

NCPDP (National Council for Prescription Drug Programs), the not‐for‐profit pharmacy standards development organization, announced today the availability of a patient safety white paper that provides specific industry guidance for standardizing the dosing designations and labeling of oral liquid medications. The white paper details patient risks associated with the variety of oral liquid dosing designations, prescribing practices and processing systems which can lead to dispensing and later administration errors that can harm patients ‐ especially pediatric patients who are often prescribed liquid medications.

The white paper includes specific calls to action for industry stakeholders, but broadly calls organizations to communicate, adopt and implement the recommendations; measure organization performance and stress accountability across the organization for adhering to the recommendations; and develop patient-centered communications and encourage pharmacist‐to‐patient conversations at the point of dispensing.  More at link above.  (PRWeb)

Application of New Cholesterol Guidelines to a Population-Based Sample

The 2013 guidelines of the American College of Cardiology and the American Heart Association (ACC–AHA) for the treatment of cholesterol expand the indications for statin therapy for the prevention of cardiovascular disease.

Using data from the National Health and Nutrition Examination Surveys of 2005 to 2010, we estimated the number, and summarized the risk-factor profile, of persons for whom statin therapy would be recommended (i.e., eligible persons) under the new ACC–AHA guidelines, as compared with the guidelines of the Third Adult Treatment Panel (ATP III) of the National Cholesterol Education Program, and extrapolated the results to a population of 115.4 million U.S. adults between the ages of 40 and 75 years.

The new ACC–AHA guidelines for the management of cholesterol would increase the number of adults who would be eligible for statin therapy by 12.8 million, with the increase seen mostly among older adults without cardiovascular disease. More at link above. (NEJM)

Big Pharma Opens New Chapter on Big Data Collaboration

In the course of one short week, no less than 3 different models have emerged for sharing big data in the pharmaceutical industry.

The highest profile of these ‒ called Project Data Sphere ‒ was announced earlier today with the official opening of an online resource to share clinical trial data for use in cancer research. The number of available data sets available today is fairly small (9), but the list of companies committed to providing data is impressive and includes AstraZeneca, Bayer, Celgene, Janssen Research and Development, Pfizer, Memorial Sloan Kettering Cancer Center and Sanofi U.S. The primary objective is to accelerate drug discovery and development. (See information at BioCentury also.)

The second model was voted on last week by the European Parliament in the form of legislation that is likely to take effect by 2016. The overwhelmingly positive vote (547 to 17) is also aimed at clinical trial data transparency, but only targets new trials after the law takes effect.

The third approach ‒ announced just yesterday ‒ is the 5-year commercial agreement between Genentech and PatientsLikeMe which is also targeting cancer research as the lead effort. The data in this case isn’t clinical trial data, but data provided by actual patients in the course of their current treatment for a wide range of conditions ‒ including cancer.  More at link above.  (Forbes)

The Imperative of Overcoming Barriers to the Conduct of Large, Simple Trials
Randomized clinical trials remain the most reliable means of identifying the drugs, devices, and treatment strategies that will improve human health. There is increasing interest in the possibility that “personalized” medicine can be evaluated in much smaller trials because the average treatment effect is expected to be larger in highly selected cohorts. Smaller, biomarker-driven trials can provide major insights into whom to treat and may be sufficient for selected disease states in which considerable treatment effects may be observed. However, a precise biological understanding of most chronic illnesses and biomarkers that might predict response has eluded investigators. Moreover, treatment effect sizes in chronic conditions are expected to be modest in most cases. As a result, determining the long-term balance of risk and benefit, particularly in comparative effectiveness trials, often requires large numbers of clinical events in representative populations. More at link above. (JAMA)

Payer Evidence Requirements for New Drugs: Trends and Impact

The call has been made—loud and clear: payers want more comparative evidence research (CER) for coverage decision making. However, the primary source for common research results—the biopharmaceutical industry—has been highly scrutinized by payers as a suitable source. Since it is unlikely that payers themselves will fund large-scale CER research, what can manufacturers do to make their research studies more palatable to payers? Katya Svoboda, MBA, MPH, Partner, Percipient LLC, Somerville, New Jersey, and Nathan White, CPC, director, NucleusX Market Access, Atlanta, Georgia, conducted a survey-based investigation to find out.

Overall, payer needs for HEOR are not being met. The gaps are the result of lack of credibility of manufacturer-generated data, payers having insufficient resources to conduct their own research or critically evaluate existing manufacturer-generated research, lack of value placed on humanistic data, and study populations that do not reflect health plan membership. Manufacturers, on the other hand, understand the importance of including outcomes end points, but need to weigh their risk in doing so, which include delays in submission timelines and the risk of negative outcomes.  More at link above.  (National Pharmaceutical Council)

Health Canada Consultation: DRAFT Guidance for Industry - Submission of Risk Management Plans and Follow-up Commitments

Health Canada has released the “DRAFT Guidance for Industry - Submission of Risk Management Plans and Follow-up Commitments” and will accept comments until May 26, 2014.

The objective of this document is to provide guidance to manufacturers on how to proceed when submitting Risk Management Plans RMPs and follow-up commitments with Health Canada. The principles and practices outlined in the document apply to pharmaceuticals, biologics and biotechnology-derived products for human use, within the scope of ICH E2E. The submission of RMPs for natural health products, medical devices (except when they are part of a combination product submission) and veterinary products are outside the scope of this guidance document.  More at link above. (Health Canada)

Health Canada to Post Drug Safety Reviews Online

Health Canada has begun posting summaries of drug safety reviews on its website with the goal of better informing the public about potential harms associated with certain medications. Federal Health Minister Rona Ambrose announced the program -- part of the "regulatory transparency and openness framework and action plan" -- Tuesday (4/8) in Ottawa.

"With this initiative, Canada is now a world leader in the posting of drug-safety reviews and post-market access to this information," Ambrose said.

Health Canada conducts a review of a drug when a safety issue is red-flagged to the department, said Dr. Supriya Sharma, a senior medical adviser at Health Canada. More at link above.  (Press – Canada)

WHO releases global hepatitis C treatment guidelines
The World Health Organization has issued its first guidelines for the treatment of hepatitis C in a bid to help improve access to more effective and safer medicines for patients worldwide. The agency strongly recommends HCV drugs from Gilead and J&J, antibody testing for hepatitis C and appropriate selection of treatments. The WHO is also urging pharmaceutical companies to reduce the price of HCV drugs, citing voluntary and compulsory licensing and tiered pricing as possible ways to achieve affordability. FoxNews.com/Reuters (4/9), Aidsmap (4/9) (via FDLI Smart Brief)

WHO Investigates Use of a Biological Qualifier for Biosimilars

The World Health Organization is still discussing several different options on how to name biosimilars, according to the recently published executive summary of its 57th Consultation on International Nonproprietary Names (INNs) for Pharmaceutical Substances.

The need for global harmonization with respect to the naming convention for biosimilars was highlighted and the possible use of a unique global ‘biological qualifier (BQ)’ for biosimilars was discussed.  Two schemes for obtaining and assigning an INN-BQ for biosimilars were presented.  More at link above.  (Generics and Biosimilars Initiative Online)

EMA announces final steps for its clinical-trial data policy

The European Medicines Agency (EMA) will launch a final round of targeted consultations with key stakeholders on its draft policy on proactive publication of and access to clinical-trial data at the beginning of May. This will give key stakeholders and the Agency the opportunity to address any outstanding issues before the final policy is presented to the EMA's Management Board for endorsement in June 2014.

This consultation is meant to clarify and fine-tune specific aspects and achieve the broadest possible consensus and understanding of the policy. The Agency will liaise shortly with organisations representing patients, academia, pharmaceutical industry, as well as European Union (EU) institutions. More at link above.  (EMA.europa.eu)

Specifications for additional efficacy studies for medicines published in the Official Journal of the EU

The European Commission has adopted a delegated act  specifying the situations where a post-authorisation efficacy study can be required by medicines regulatory authorities. The act was published on 10 April 2014 in the Official Journal of the European Union (EU) and will enter into force on 30 April 2014.

The aim is to enable regulators to request such studies when there are important questions about the efficacy of the medicine that can only be answered once the product is in general use, or when questions arise in the post-authorisation period.

The European Medicines Agency, in collaboration with the EU Member States, is developing a scientific guidance document that will describe the design of post-authorisation efficacy studies. More at link above. (EMA.europa.eu)

Registration opens for EMA workshop on the clinical investigation of medicines for the treatment of Alzheimer’s disease

The European Medicines Agency (EMA) is inviting expressions of interest in attending its workshop on the clinical investigation of new medicines for the treatment of Alzheimer’s disease on 24-25 November 2014.

The Agency is organising this workshop following the public-consultation exercise on its concept paper on the need for revision of the guideline on medicinal products for the treatment of Alzheimer’s disease and other dementias. The concept paper was released for public consultation from October 2013 to January 2014. Based on the comments received, the Agency is currently revising the guideline and expects to release a first draft for public consultation within the next few months.

The main goal of the workshop is to make sure that, while revising its guideline, the EMA can take the most up-to-date scientific developments in understanding and treating Alzheimer’s disease into consideration, as well as the positions of experts in the field on the main topics covered in the guideline. More at link above. (EMA.europa.eu)

MHRA accepting applications for early access scheme
The U.K.'s Medicines and Healthcare products Regulatory Agency is now accepting applications for its Early Access to Medicines Scheme, which will allow limited access to unapproved or off-label products based on quality, safety and efficacy data from Phase II or III testing. Both the BioIndustry Association (BIA) and the Association of the British Pharmaceutical Industry (ABPI) have supported the scheme. The scheme lacks financial support from government to pay for the drugs, which both groups say could lead to it being underused. (BioCentury)

French plan to push copycat biotech drugs worries Big Pharma

France is going out on a limb with a plan to push the use of cheap copies of biotech drugs, triggering alarm among companies in Europe's second-biggest pharmaceutical market behind Germany.

The government quietly introduced the measure allowing pharmacists to substitute prescribed brand-name biotech drugs with cheaper, similar versions in its 2014 healthcare budget.

The move, which still needs a decree to come into effect, could cut the cost of providing complex biological medicines to patients with conditions like cancer, kidney disease and rheumatoid arthritis. But it poses a threat to makers of original products that are particularly reliant on sales of such drugs, such as Roche and Amgen. More at link above. (Reuters via FDLI Smart Brief)


SECTION 5 LEGAL, POLICY, AND COMPLIANCE NEWS

Eshoo Introduces Biomedical Research Funding Bill

Rep. Anna Eshoo (D-Calif.) introduced in the U.S. House of Representatives a bill that would create a mandatory trust fund for biomedical research to increase funding for NIH, the Centers for Disease Control and Prevention, the Department of Defense Health Program (DHP) and the medical and prosthetics research program at the Department of Veteran Affairs. The bill would set fiscal year funding for these agencies and programs at 105% over the prior fiscal year, with an adjustment for GDP-indexed inflation. The bill, H.R. 4384, was referred to four house committees.

An identical senate bill, S. 2115, was referred to the Senate Health, Education, Labor and Pensions (HELP) Committee last month. (BioCentury)

Judge Likely to Boot Massachusetts Ban on New Opiate Zohydro
After Massachusetts Governor Deval Patrick banned Zogenix's powerful new FDA-approved painkiller, Zohydro, the company filed a lawsuit arguing the ban was unconstitutional. On Tuesday, federal district court judge Rya Zobel said she was inclined to agree with Zogenix and was leaning towards granting a preliminary injunction that would allow Zohydro to be sold in Massachusetts.

Zogenix filed a lawsuit in a Boston federal court earlier this week blasting Patrick for taking a unilateral action to ban Zohydro without alerting the company first. The lawsuit alleged that Patrick failed to respond to Zogenix's request for a meeting to discuss the drug.

Patrick has said he banned Zohydro because it's a pure opiate that lacks abuse-deterrent features that would stop addicts from crushing it to get high. He's far from the only state legislator to have an issue with the drug, which was approved by the FDA last fall: More than half of the states' attorneys general have asked the agency to withdraw Zohydro from the market, and Vermont recently passed an emergency order putting restrictions on how physicians prescribe it. But FDA Commissioner Margaret Hamburg continues to defend the drug, saying it was proven safe and effective and fills an important niche for the treatment of patients with severe chronic pain. More at link above.  (Fierce Pharma)

With Abuse and Overdose Rates at an All-Time High, Harkin and Alexander Announce the HELP Committee Prescription Drug Abuse Working Group

With prescription drug overdose death rates in the United States more than tripling since 1990 and prescription opioid abuse an ever-growing problem, Senate Health, Education, Labor, and Pensions (HELP) Committee Chairman Tom Harkin (D-IA) and Ranking Member Lamar Alexander (R-TN) today announced the establishment of the HELP Committee Prescription Drug Abuse Working Group.

The bipartisan staff Working Group will examine the characteristics and challenges of prescription drug abuse; what the federal government, state governments, public health groups, health care providers, law enforcement, and others are doing to address it; and consider ways to further address the issue. Harkin and Alexander have invited all members of the HELP Committee to join and contribute to the Working Group.

In the coming weeks and months, the HELP Committee Prescription Drug Working Group will convene a series of meetings to examine a wide range of topics around the issue of prescription drug abuse and misuse, including perspectives from states and local entities, private coalition efforts, federal officials, and innovators developing novel technologies for combating abuse, among other issues. (Senate.gov)


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.