FDA Lawyers Blog

On July 11, FLH Partner Brian J. Malkin will speak at Q1 Productions' Global Clinical Risk Management & Regulatory Policy Conference in Alexandria, VA on the topic "Developing a Single, Shared REMS in a Collaborative Setting". Joining Mr. Malkin will be Mark A. Collings, Ph.D., M.B.A., Director, REMS Programs, Pharmacovigilance and Risk Management, Endo Pharmaceuticals, Inc. The session description reads:

The FDA approves single, shared REMS for product categories with similar risk management programs to help relieve some of the burden on healthcare providers and pharmacies. The development of shared REMS requires that multiple companies and the FDA work in tandem to unify data and risk minimization systems, a model which some companies have also used to help organize risk management plans internationally. By ensuring a strong cross-corporate collaboration, internal regulatory policies, data analysis and communication systems can be synchronized internationally for advanced risk management programs.

• Structuring lines of communication within shared REMS network

• Establishing similar regulatory policies

• Utilizing data from all companies for international RMP development.

The Conference will be held on July 11-12, 2013. Throughout this two-day program, executive from industry will have an opportunity to discuss and debate the many challenges associated with profiling the risk of products as well as minimizing these risks and meeting regulatory expectations. As with all Q1 programs, the focus of the event will not only lie upon the educational content, but also providing attendees with an opportunity to network and build relationships across this highly dynamic and evolving market.

This week The Venture Café in Cambridge, Massachusetts hosts Life Sciences Week with a special focus on life science research in the Greater Boston area. Featured life science research speakers include Akrivis (ADAPT™ technology, which allows cancer detection much earlier by increasing the sensitivity of early biomarker detection and enabling the medical imaging of millimeter-sized cancer tumors), Human Metabolome Technologies (metabolome profiling for tumor metabolism, biomarker discovery and production optimization), Advirna (powerful and innovative tools to regulate activity of genes inside living cells), Heartbt Foundation (newly-developing non-profit seeking to boost translational research by bridging medical, professionals, big pharma, researchers, NGOs, the general public, and others), Extend Biosciences (startup biotech company with a platform technology that enables the development of long-acting peptide based drugs), Anchor Therapeutics (pre-clinical stage drug discovery company advancing Pepducin Technology, a novel approach towards allosteric modulators of G-protein coupled receptors), Lab Central (soon-to-open facility to house up to 65 scientists, in 20 individual lab stations and 9 private lab suites in the heart of Kendall Square), and Cellanyx (biomarker-based diagnostic biopsy test to determine oncogenic and metastatic potential for prostate tumors). The Venture Café will also host a Roundtable of Life Sciences Entrepreneurship in Massachusetts, featuring Peter Abair of the Massachusetts Biotechnology Council ("MassBio"), Pamela Norton from the Mass Life Sciences Center, and Peter Parker of Lab Central.

Written by Brian J. Malkin

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During Life Sciences Week, FLH Partner Brian J. Malkin will host Office Hours from 3-5pm EST along with several other service providers in the healthcare field. Mr. Malkin's Office Hours description reads:

Chat with Brian J. Malkin, Partner at Frommer Lawrence & Haug LLP. Brian specializes in FDA-regulated products, in particular pharmaceutical, biotechnology products and biosimilars. Discuss intellectual property, pathways for FDA approval, as well as life cycle management and due diligence investigations. Brian frequently speaks on a variety of IP- and FDA-oriented topics, and is editor of the FDA Lawyers Blog.

This blog is a second part to a blog here, describing Annual Conference for the Food and Drug Law Institute ("FDLI") held on April 23 and 24, 2013, its in Washington, D.C.

On the first day, FLH Partner Brian J. Malkin spoke on a panel discussing the priorities for the Center for Biologics Evaluation and Research ("CBER"). The two key CBER presentations came from Diane Maloney, J.D.., Associate Director for Policy, and Mary Malarkey, Director, Office of Compliance and Biologics Quality. The panel also included Michael S. Reilly, Executive Director, Alliance for Safe Biologic Medicines, and Mark S. Robbins, Ph.D., Vice President, Clinical Regulatory Affairs, DiaMedica USA, Inc. and was moderated by Scott Cunningham, Partner, Covington & Burling LLP. A key issue raised by panel was the status of biosimilars. Reilly, for example, was concerned that there may be misinformation about biosimilars, especially because there is no public information concerning biosimilars applications being considered in the United States. Malkin asked when FDA planned to issue additional guidance, because FDA's guidance has so far been very general and concerns pre-investigational or pre-biosimilars application stages, not the key issues of interchangeability and naming that have been debated. Maloney explained that CBER and the Center for Drug Evaluation and Research ("CDER") have been discussing strategy, even though there still have been no filed biosimilar applications, and would issue additional guidances. At the same time, Malkin and others acknowledged that at the state level, legislators have been passing bills that specifically prohibit substitution of biosimilars for their referenced counterpart, unless FDA deems the biosimilar "interchangeable" and naming issues and more persist.

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A question was raised about a pending Citizen Petition that includes an argument that it was a taking for biosimilar applicants to file applications referencing biologic products before the passage of the Biologics Price Competition and Innovation Act ("BPCIA") (March 23, 2010). In this Petition, Abbott argued that before this date, when biologics license applications ("BLAs") were submitted, their sponsors had "reasonable, investment-backed expectations that the trade secrets in their applications would not be used to approve competing products." Abbott asserts, therefore, that FDA's use of trade secrets in these BLAs to support biosimilar approvals "would constitute a taking under the Fifth Amendment to the U.S. Constitution. FDA should not implement the BPCIA in any manner that would raise constitutional issue." While most panel members declined to respond, Malkin hazarded a guess that based on comments FDA has made (see for example here) and in the context of FDA's Citizen Petition response for a similar issue concerning 505(b)(2) new drug applications (where a similar takings argument has been made), FDA wanted to decide that there was no "taking," but FDA has not answered the petition in part because no biosimilar application has been filed to date.

On April 23, FLH Partner Brian J. Malkin will speak at FDLI's Annual Meeting in Washington, D.C. in the Concurrent Breakout Session for the Center for Biologics Evaluation and Research ("CBER"). During the Concurrent Breakout Session, representatives from each FDA center will discuss the three most important developments from last year and their three most important goals in 2013. Following the FDA center representative presentations, food and drug law stakeholders responding to their presentations. This year Diane Maloney, Esq., Associate Director for Policy, CBER, will be presenting for CBER, and Mr. Malkin will be responding as a food and drug law stakeholder. Scott Cunningham, Partner, Covington and Burling, will moderate the session. The Concurrent Breakout Sessions are always a big draw for attendees at FDLI's Annual Conference, and Mr. Malkin hopes to see you there and at the CBER Breakout Session.

Next week, FLH Partner Brian J. Malkin will speak at Center for Biologics Evaluation and Research ("CBER") Panel at FDLI's Annual Meeting held in Washington, D.C. on April 23-24. The CBER Panel will be held on the first day of the Conference, Tuesday, April 23, from 2-3:30 p.m. FDLI has posted the key two presentations by CBER Representatives Diane Maloney, Esq., Associate Director for Policy, and Mary Malarkey, Director, Office of Compliance and Biologics Quality. The panel will be moderated by Scott Cunningham, Partner, Covington & Burling LLP, and also includes Michael S. Reilly, Executive Director, Alliance for Safe Biologic Medicines, and Mark S. Robbins, Ph.D., Vice President, Clinical Regulatory Affairs, DiaMedica USA, Inc.

As demonstrated by the presentations already posted by Maloney and Malarkey, the panel has a lot of ground to cover, and we as panel members have been providing feedback for additional topics to address as time permits. All of us hope for a lively discussion following the initial presentations and hope to see you there!

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FDLI's Annual Conference is the largest (750+ attendees expected) and longest running conference for the FDA-regulated industry, which now includes tobacco products. FDLI's program brings together experts from the Federal Government, industry, private sector bar, consulting organizations, and academia in one place to discuss legal, regulatory, policy, and economic issues falling in FDA's vast authority. FDA Commissioner, Margaret A. Hamburg, M.D., will provide the Keynote Address the first day, with other plenary sessions featuring FDA's top regulatory and compliance officials, breakouts for all of FDA's centers and a sessions featuring compliance and liability issues. The next day features top cases in food and drug law, concurrent breakouts in emerging issues, an address from FDA's Chief Counsel, Elizabeth Dickinson, and plenary sessions in interagency dynamics and the role of the media in food and drug law, regulation, and policy.

Over the past months, there has been a lot of speculation (see recent blogs here , here, and here) whether the White House's proposed budget would cause a sequester situation for FDA, resulting in potential layoffs or program cuts, in an era of new user fees for generic drugs and biosimilar biological products. While initial reports and temporary budget fixes (called continuing resolutions) appeared to keep FDA's user fees intact and available for use, FDA's Commissioner, Margaret A. Hamburg, M.D., recently reported to members of a biotechnology trade association, the Massachusetts Biotechnology Council ("MassBio"), that it was not clear what would happen with user fees in the new federal budget.

Released on April 10, the White House's proposed fiscal year 2014 budget is a mixed bag that has been called a "political document rather than a serious piece of legislation" with a "series of bargaining positions" that "would bleed pharma." On the one hand, the plan would appear to confirm that FDA's user fees would not be sequestered, given that it supported the $4.7 billion in total program budget requested by FDA, which included user fees that would help fund over 90 percent of the requested increases. On the other hand, the budget includes a myriad of proposals that would change the way the government pays for medical care and products. For example, Medicare (senior citizens' drug coverage) Part D manufacturer discounts for branded drugs would be increased from 50% to 75% in 2015 (rather than 2020) and low-income individuals would be pushed more to generic drugs by increasing certain copayments for branded drugs and lowering certain copayments for generic drugs.

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Many of the more controversial proposals were nestled in a document called "Reducing the Deficit in a Smart and Balanced Way". Here, the White House proposes, among other things, several items to purportedly lower drug costs, including: 1) authorizing the Federal Trade Commission to stop companies from entering into certain "pay-for-delay" agreements (see below) and 2) beginning in 2014, to reduce biologic product exclusivity from 12 years to 7 years and prohibit additional periods of exclusivity for minor changes to product formulations. These two items could open up some unanticipated debate regarding the White House's budget.

The United Kingdom's ("UK's") method of controlling the prices its Department of Health pays for innovative medicines has been, up until now, the Pharmaceutical Price Regulation Scheme ("PPRS"). The PPRS has been a voluntary scheme run over many years between the Department of Health and the branded pharmaceutical industry represented by the Association of the British Pharmaceutical Industry ("ABPI"). Its objective was to deliver the provision of safe and effective medicines at reasonable prices to the National Health Service ("NHS") while promoting innovation, the rapid uptake of new clinically and cost effective medicines, and in a sustainable manner.

Over recent years, the National Institute for Health and Care Excellence ("NICE") has been taking on a more visible and key role. Its job has been to improve outcomes for people using the NHS by producing evidence-based guidance by way of advice to healthcare professionals and to develop quality standards and measures for those providing health care services and also providing information to practitioners and managers in both health and social care.

Written by Howard E. Rosenberg, Ph.D.

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The UK Government's response to the Health Select Committee report of 2012-2013 on NICE was to re-establish NICE as a new statutory body giving it a key position in the healthcare system. It gives NICE the role of deciding the value-based pricing of medicines. Thus, value-based pricing will replace the current PPRS, when it expires in January 2014.

On April 1, FDA issued a Federal Register Notice announcing a new draft biosimilars guidance, "Formal Meetings Between the FDA and Biosimilar Biological Product Sponsors or Applicants". This is the latest in FDA's new biosimilar guidances for 2013, which FDA has announced in earlier meetings this year would be coming to help spur the filing of a biosimilars application, which FDA has called 351(k) applications based on the section in the Public Health Service Act ("PHS Act"). As of a few weeks ago at the Massachusetts Biotechnology Association's ("MassBio's") Annual Meeting, which we blogged on here, FDA's Commissioner, Margaret A. Hamburg, M.D., continued to report that FDA has not received a single 351(k) application to date.

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The Guidance focuses on formal meetings for 351(k) applications and the associated requirements or performance goals from the Biosimilar User Fee Act of 2012 ("BsUFA"), which was enacted as part of the Food and Drug Administration Safety and Innovation Act ("FDASIA"). In particular, the Guidance discusses the principles of good meeting management practices ("GMMPs") and describes standardized procedures for requesting, preparing, scheduling, conducting, and documenting such formal meetings.

On March 14-15, the Massachusetts Biotechnology Council ("MassBio") held its Annual Meeting in Cambridge, Massachusetts. The Meeting also featured a Keynote from FDA Commissioner Margaret A. Hamburg, M.D. (see related blog here). Key themes at the Meeting were the importance of the Cambridge/Boston biotechnology community for advancing new therapies and the unique resources available in the area that have made it an industry leader. Some of the Cambridge/Boston advantages discussed were the intellectual research capital (local universities such as Harvard and Massachusetts Institute of Technology), venture capital, and local biotechnology businesses, such as Biogen Idec and Genzyme, as well as other biotechnology companies that now have offices in the Cambridge/Boston area and are seeking partnerships to develop new products, such as AstraZeneca, Pfizer, Merck, Novo Nordisk, and Sanofi.

On the second day, Hamburg described here "special affection" for the Cambridge/Boston region dating back to her days at Harvard, saying that she hopes D.C. "would be as efficient and congenial as here." Hamburg said that the Cambridge/Boston region is a life sciences enterprise fueled by top notch research and medical care with the top five NIH-funded hospitals and a "biotech supercluster second to none" with "a remarkable 500 biotech and pharma companies here, and some thirty venture capital firms."

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Hamburg described FDA as striving for true collaboration and regulatory flexibility with industry, including MassBio, and has been hearing that industry wants more clarity, certainty, transparency with decisions. Hamburg said that FDA is trying to have creative approaches--not a one size-fits-all approach. To this end, Hamburg described approaches that FDA has taken with four new products from the Massachusetts area: 1) Inclusig® for two rare forms of leukemia, 2) Juxtapid® (an orphan drug), 3) Linzess® for irritable bowl syndrome, and 4) Kalydeco® for cystic fibrosis. In addition, Hamburg highlighted new provisions in the Food and Drug Administration Safety and Innovation Act ("FDASIA") for expedited approvals, citing 31 breakthrough therapy designation requests, of which 9 have been granted, 10 denied, 11 pending, and 1 withdrawn. To help with more companies taking advantage of this new process, FDA will be publishing a new guidance shortly, Hamburg announced.

On March 14-15, the Massachusetts Biotechnology Council ("MassBio") held its Annual Meeting in Cambridge, Massachusetts. The Meeting featured key topics such as biosimilars and a Keynote from John Crowley, Chairman and CEO of Amicus Therapeutics.

On the first day of the conference, Crowley exemplified many of the speakers' entry in biotechnology, which originated with a family member or friend with a disease requiring development of a biotechnology product. For Crowley, it was his two children Megan and Patrick, were diagnosed with a severe neuromuscular disorder, Glycogen storage disease type II, known as Pompe's disease. Rather that sitting still to wait for a cure, Crowley became involved in the process, first moving to Princeton, New Jersey, to be close to doctors specializing in the disease and leaving his job with Bristol-Myers Squibb. He later took a position as CEO of Novazyme Pharmaceuticals, a biotechnology research company located in Oklahoma City founded by Dr. William Canfield, which was conducting research on a new experimental treatment for the disease. Novazyme was acquired by Genzyme Corporation, which was then the world's third largest biotechnology company. Crowley was put in charge of Genzyme's global Pompe program, becoming the largest research and development effort in the company's history.

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Through these efforts, an experimental enzyme replacement therapy was developed, and Megan and Patrick Crowley received the therapy, which Crowley credits with saving his children's lives. Crowley went on to become President and CEO of Orexigen Therapeutics and was named the President and CEO of Amicus Therapeutics, based in Cranbury, New Jersey, which he helped take public in 2007. Crowley's efforts were documented in a Wall Street Journal article and other publications, which ultimately resulted in Harrison Ford working to bring the story to life in a major motion picture, Extraordinary Measures.

FLH Partner Brian J. Malkin will attend the Massachusett's Biotechnology Council's ("MassBio's") Annual Meeting in Cambridge, Massachusetts on March 14-15, 2013. FLH is a member of MassBio, reflecting FLH's commitment to the development and promotion of new biological and related products. Each year, the MassBio Annual Meeting focuses on the most timely and critical challenges facing the Massachusetts biotechnology industry. The meeting program is pulled together by a Steering Committee of leaders in the industry and the agenda encompasses keynote presentations, panel discussions, interactive working sessions, and extensive networking opportunities for all MassBio members. This year, keynote presentations feature John Crowley, Chairman & CEO of Amicus Therapeutics, Inc. and FDA Commissioner Margaret A. Hamburg, M.D. Key topics of interest include personalized medicine and companion diagnostics, biosimilars, RNA therapeutics, healthcare reimbursement strategies, research resource sharing opportunities and a variety of orphan drug candidate topics. Mr. Malkin looks forward to seeing you and catching up on the latest biotechnology developments with some of the best biotechnology leaders in the Massachusetts area and beyond.

On February 13 and 14, 2013, Leerink Swann ("Leerink") held its annual Global Healthcare Conference at the Waldorf-Astoria Hotel in New York, New York. Each year the Conference highlights emerging themes and controversies in healthcare, where Leerink's equity analysts present surveys and moderate discussions with MEDACorp Key Opinion Leaders and industry specialists to provide unique and timely insights. MEDACorp is Leerink's network of 30,000 healthcare professionals including key opinion leaders, practitioners, clinicians, and hospital administrators. In between the main panels, senior management from some of the companies identified by Leerink as the most prominent and promising deliver "fireside chats" and presentations with company updates, including discussions of new products in their pipeline under review. This year the sole sponsor of Leerink's Global Healthcare Conference was Latham & Watkins LLP.

Headlining the Conference was a Keynote Address entitled "Innovating Medical Product Development - FDA and Other Forward Looking Trends" delivered by Vicki L. Seyfert-Margolis, Ph.D. Seyfert-Margolis just recently was the Senior Advisor for Science Innovation and Policy in FDA's Office of the Commissioner. Seyfert-Margolis is currently Chief Science and Strategy Officer for Precision Health, which provides services, infrastructure, and technologies to companies developing personalized medicines.

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Seyfert-Margolis worked for about three and a half years in FDA's Office of the Commissioner, where she led the effort to help develop a more coherent policy for companion diagnostic and personalized medicine, including developing guidances for personalized medicine such as "In Vitro Companion Diagnostic Devices" and "Qualification Process for
Drug Development Tools". While at FDA, Seyfert-Margolis looked at why companies were not seeing as many returns from their investments in research and development, noting that it was not FDA's "fault" for less new chemical entity filings, because FDA can only review or approve products that are filed in new applications.

This Thursday, FLH Partner Brian J. Malkin will host Legal Office Hours at The Venture Café in Cambridge, Massachusetts. Mr. Malkin's Legal Office Hours description reads:

Learn how you can protect your start-up with patent and trademark protection from intellectual property Partner Brian J. Malkin, Frommer Lawrence & Haug LLP. Mr. Malkin is an expert on FDA-regulated products, in particular pharmaceutical/biotechnology products and biosimilars, and can discuss pathways for FDA approval, as well as life cycle management and due diligence investigations. Mr. Malkin recently published a chapter on biosimilars, has a primer on the drug and biologics approval process, frequently speaks on a variety of IP- and FDA-oriented topics, and is the editor of FDA Lawyers Blog, a blog that focuses on legal and scientific developments for FDA-regulated products.

The Venture Café was created to provide a resource for the Boston entrepreneurial and innovation communities. Our mission is to enable fresh and useful conversations.

Cambridge is a fountain of innovative spirit, spirit that needs a framework to reach its full potential. The Venture Café serves as a nexus for helping innovators and entrepreneurs find one another and collaborate to bring their dreams to reality.

Even in this digital world, it's important to have a physical space. Shared physical spaces provide common meeting ground and a forum for semi-serendipitous encounters that often foster brainstorming and drive creativity. Meeting in person establishes the trust that's so crucial to working together, particularly on risky, underfunded projects. The Venture Café can provide the framework upon which numerous experimental "applications" can be nurtured and launched.

Frommer Lawrence & Haug LLP is proud to announce that the Food and Drug Law Institute ("FDLI") just published the FDLI Primer, "The Drug/Biologics Approval Process" authored by FLH Partner Brian J. Malkin and Associate Scot Pittman. Mr. Malkin has been a member of the Primer Committee for several years now. Recently, FDLI decided to make each Primer separately available for purchase ($119 for nonmembers, $95 members) rather than an entire series for its predecessor FDLI Monograph publication. FDLI asked Mr. Malkin to author this Primer based on a previous FDLI publication authored by Geoffrey M. Levitt, Senior Vice President and Associate General Counsel, Regulatory and Policy, Pfizer, Inc. Updates in the FDLI Primer include adaptations based on recent FDA legislation, including the latest user fees acts for generic drugs and biosimilars and the Food and Drug Administration Safety and Innovation Act ("FSASIA"). According to FDLI, Primers are:

[E]xtensively researched, referenced, and edited by some of the most experienced and respected professionals in the field. ... The Primers are designed to provide you with information and proprietary analysis to enable you to advise your clients or help your company comply with vexing issues, regulations, and guidance.

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For this particular FDLI Primer, FDLI writes:

This publication will explain, in practical terms, the approval processes for drugs and biologics. It will describe the various FDA premarket requirements and pathways for drug and biologics application reviews, including changes enacted under the Food and Drug Administration Safety and Innovation Act (FDASIA). Topics addressed will include the New Drug Application (NDA) process, non-NDA routes to market, generic drugs and the abbreviated new drug application process, as well as over-the-counter drugs and biologics. With this Primer, pharmaceutical stakeholders will feel confident that they have a helpful overview to support successfully navigating the FDA drug and biologics review processes.

On January 28, The New York Times reported that biotechnology companies are actively lobbying state legislatures to limit access to biosimilar versions, i.e., "highly similar" versions of previously-approved, innovator biological products ("biologics"). According to the author, Andrew Pollack, Amgen and Genentech are proposing bills that would make it more difficult for pharmacists to substitute biosimilar versions for the innovator's products, unless FDA determines that a particular biosimilar version is "interchangeable" with the innovator's product.

For instance, the Virginia House of Delegates reportedly already passed such a bill last week by a 91-to-6 vote. Other bills in the works require patient consent for substitution, pharmacist notification of the patient's physician if a switch is made, and for both the pharmacist and patient's physician to maintain records of any such substitutions for years.

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The Generic Pharmaceutical Association ("GPhA") and insurers generally accept that biosimilar substitution for a biologic should follow similar methods as with drugs only if deemed interchangeable by FDA but find that many of the bills go further to discourage use of biosimilars. "All of these things are put in there for a chilling effect on these biosimilars," commented Brynna M. Clark, Director of State Affairs for GPhA, adding that many of the limits "don't sound too onerous but undermine confidence in these drugs and are burdensome." GPhA and insurers would prefer that legislatures leave biosimilar regulation to FDA, which has been entrusted with using its regulatory prowess to determine the necessary requirements for biosimilars and "interchangeable" biosimilars, as well as when to waive those requirements based on what is know about a particular biosimilar product.