April 25, 2013

FDLI's Annual Meeting Features FDA Seeking More Resources and Regulatory Authority and More (Part 1 of 2)

Thumbnail image for FDLILogo.jpgOn April 23 and 24, 2013, the Food and Drug Law Institute ("FDLI") held its Annual Conference in Washington, D.C. As expected, FDLI's Conference featured the FDA Commissioner, Margaret A. Hamburg, M.D., presentations from leaders from all of FDA's Centers and the Chief Counsel, Elizabeth Dickinson, and leaders from industry, academia, and the legal bar. Unlike previous years, however, there were far fewer FDA attendees due to budget cuts, and FDA seemed more reluctant to use the Conference as a platform for announcing new policy or initiatives.

Hamburg kicked off the Conference with her recognition that FDA's budget was cut, along with other government agencies, noting that it would make times tight but that there are no planned furloughs. Hamburg said that this fiscal year, FDA lost about $209 million-$126 million in budget authority and $83 million in user fees. FDA will continue to collect user fees, but FDA cannot use them this fiscal year due to the sequesteration issue. Hamburg said that the reduced budget would mean reductions in programs but did not specify which ones. Yet, at the same time, Hamburg emphasized that FDA has been busy implementing its new regulatory authorities, including the new Center for Tobacco Products and authorities in the Food and Drug Administration Safety and Innovation Act ("FDASIA").

Hamburg stressed that it has become more and more apparent that its regulated industries need to build quality in their products. She highlighted that quality issues have caused two out of three drug shortages, and FDA has uncovered "shockingly unsafe drugs" at compounding pharmacies. Over the past several months, Hamburg explained, FDA has been inspecting compounding pharmacies and has found unidentified black particles in what were supposed to be sterile injectable products, rust and mold in sterile rooms, and products being processed with bare hands. At the same time, FDA has encountered increased resistance during its inspections, resulting in at least two cases needing administrative warrants. Hamburg said that FDA believes there should be a distinction between traditional and nontraditional compounding--traditional is individualized to patient; nontraditional is sterile product prepared out of state compounded and anticipated without a prescription. For nontraditional compounding, FDA has asked Congress for more clear FDA authority to monitor and examine records in the "patchwork" of compounding rules.

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April 22, 2013

Generic Oxycontin®--Abuse Resistance Required Says FDA

mortar and pestle.jpgThe U.S. Food and Drug Administration ("FDA") announced last Tuesday that it would not approve any generic versions of the original formulation of the prescription narcotic painkiller OxyContin® ("original Oxycontin®"). OxyContin® is a brand name for oxycodone hydrochloride, an opiate-based pain medication. Original Oxycontin® has been marketed by Purdue Pharma since 1995 and is notorious for its user misuse and abuse.

OxyContin® contains a large amount of oxycodone because it is designed to release the pain-relieving drug over an extended 12-hour period. However, original Oxycontin® can easily be crushed and then snorted or injected (or even sprinkled on food) to produce a rapid and intense euphoric high. The abuse of original OxyContin® in this manner can lead to addiction and dependence and has reportedly earned the product the nickname "hillbilly heroin." Its accessibility has magnified abuse rates; FDA reports that half a million people over age twelve began using original OxyContin® for non-medicinal purposes in 2008 alone. According to the Center for Disease Control, the death toll from prescription painkiller overdoses tripled in the first decade of the 21st century, and such overdoses "now kill more Americans than heroin and cocaine combined."

In addition to a patent for original OxyContin, which expired on Tuesday, Purdue Pharma also owns a patent for a reformulated, abuse-deterring version ("reformulated Oxycontin®"). This newer version was designed to resist being crushed and to form a gel that is difficult to inject when dissolved. Notably, FDA approved an updated label for this product last week, specifying the tablets' crush-resistant properties and warning of the fatal risks of misuse. (The label information is available here.) Purdue withdrew original OxyContin® from the market when its new version was approved in 2010 but retained the trade name.

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April 19, 2013

FLH Partner Brian J. Malkin Speaks at FDLI's Annual Conference on CBER Breakout Panel Next Week

FDLILogo.jpgNext 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!

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.

April 18, 2013

European Clinical Trials Report Issued by European Medicines Agency

EMA Logo.jpgThe European Medicines Agency ("EMA") has released their report giving detailed information regarding numbers of patients, sites and inspections with respect to pivotal clinical trials submitted in marketing authorization applications ("MAA") between January 2005 and December 2011.

As we noted in a previous blog there has been an increase in concern amongst regulators and the public about how well clinical trials are conducted from an ethical and scientific/organizational standpoint, and especially with regard to good clinical practice ("GCP") compliance. An applicant has to provide information in every MAA regarding the location, conduct and ethical standards applied in respect of the clinical trials conducted in third countries.

The report relates mainly to new applications (485), line extensions (95), and variations where new clinical trial information was provided (97). Generic applications are included as part of the new applications, but they generally do not add much to the number of patients, because these applications are mainly based on small bioequivalence trials, but they do provide information on the locations where these trials were conducted.

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April 16, 2013

Myriad Hits Supreme Court with Oral Arguments

supreme court.jpgOn April 15, the U.S. Supreme Court heard oral arguments in Assoc. for Molecular Pathology v. Myriad Genetics, Inc. et al., the famous case concerning the patent-eligibility of human gene patents. At issue is the validity of Myriad's patents on the human genes, BRCA1 and BRCA2, but ruling would surely cover the patent eligibility of all animal and plant genes and impact various biotechnology industries. Additional details may be found in previous blogs, for instance here and here.

The Justices appeared skeptical of Myriad's argument that isolated DNA is patent-eligible subject matter. Isolated DNA is DNA that is cut from a chromosome, resulting in an "isolated" piece of DNA that has the same nucleotide sequence as the naturally-occurring genomic DNA.

The Justices compared Myriad's isolated DNA to the discovery of plants with medicinal properties. Justice Breyer commented that it was long-standing patent law that while particular applications of such a plant can be patented, the plant cannot be. If someone discovers a medicinal plant "he gets a patent on the process, on the use of the thing, but not the thing itself." Justice Sotomayor expressed her understanding that to obtain a patent "you had to take something and add to what nature does" and wondered, "how do you add to nature when all you are doing is copying its sequence?"

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April 12, 2013

Biosimilars and "Pay-for-Delay" Settlements on the Table in White House's Proposed Budget

Obamabudget.jpgOver 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.

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.

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April 10, 2013

Orphan Drug Prices Challenged in Europe - Will the U.S. Follow?

eurosdollars.jpgOn April 7, Simeon Bennett from Bloomberg reported that individual members of the European Union are attempting to control the cost of medical care by containing the reimbursement of drugs intended for smaller patient populations, generally called orphan drugs. In Europe, orphan drugs are defined as a medicine to treat no more than 5 in 10,000 inhabitants. Many of these drugs undergo a centralized approval process via the European Medicines Agency (see related blog resource page here.) In practice, however, these drugs may only reach the market when each member state decides that its national health system will reimburse for the drug. For example, 35 orphan drugs reached the market in Belgium, 44 in the Netherlands, and 28 in Sweden in 2008. 35 such drugs reached the market in France and 23 in Italy in 2007.

According to Yann Le Cam, CEO of Eurodis, a French patient advocacy group for patients with rare diseases, "The price of orphan medicinal products is under much more debate. We have seen countries which were providing good access to orphan medicinal products now questioning the continuation of reimbursement."

Some examples provided in the Bloomberg report included the Netherlands demanding price reductions for certain therapies such as Sanofi's Myozyme® (alglucosidase alfa), an enzyme replacement therapy for patients with Pompe disease, which costs 700,000 euros ($909,000). As we previously reported from MassBio's Annual Meeting, Myozyme® was the largest research and development effort in the history of Genzyme, which was later acquired by Sanofi, and the result of a concerned father of two children with Pompe's disease pushing the promising therapy along to help it reach the public. Another example mentioned in the report included Ireland recommending against the government paying for Vertex Pharmaceuticals, Inc.'s Kalydeco® (ivacaftor) for cystic fibrosis until the company significantly reduced the price for the drug product. Yet another example was the rejection by the United Kingdom ("UK") to expand use of Alexion Pharmaceuticals, Inc.'s drug Soliris® (eculizumab) for two blood disorders, despite the recommendation for this use by an advisory panel. Instead, the government referred the matter to its National Institute for Health and Care Excellence, which we recently blogged on here, as an instrument to encourage value-based medicine in the UK.

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April 8, 2013

OTC Nicotine Replacement Therapies - Citizen Petition Answered

nicotine.jpgOn April 1, FDA provided a combined response to three citizen petitions concerning the regulation of over-the-counter ("OTC") nicotine replacement therapy ("NRT") drug products. The petitions were submitted by the Commissioner of Health, New York State Department of Health (Docket No. FDA-2008-P-0116), the Director, Legal Resource Center for Tobacco Regulation, Litigation & Advocacy, University of Maryland Francis King Carey School of Law on behalf of the Association for the Treatment of Tobacco Use and Dependence ("ATTUD") and the Society for Research on Nicotine and Tobacco ("SRNT") (FDA Docket No. FDA-2008-P-0116), and four not-for-profit organizations, the American Cancer Society Cancer Action Network, the American Lung Association, the Campaign for Tobacco-Free Kids, and the American Legacy Foundation (FDA Docket No. FDA-2010-P-0454). For additional background on this topic generally, please see an earlier blog that we posted here about a NRT workshop that FDA held in October 2010 addressing the topics in these petitions.


The petitioners asked FDA to consider modifying the labeling of OTC NRTs, certain policy changes, and allowing greater access to the products. While the requests varied, a common theme was that the petitioners wanted consumers to have greater access to NRTs in smaller packages that would permit more flexible use of the products, other than the typical up-to-12-weeks smoking cessation program scenario contemplated by the clinical studies that supported the use of these products. For instance, some of the petitioners suggested that the labeling should include additional risk/benefit information concerning NRTs versus continued cigarette (or other tobacco product) use, including possible concomitant use of cigarettes and NRTs, as well as longer-term NRT use to reduce and perhaps ultimately eliminate an individual's cigarette use. The not-for-profit petitioners wanted FDA to make the development of NRTs a priority within FDA, including certain collaborations with manufacturers of NRTs, such as the development of appropriate trial designs, and to transfer evaluation of NRTs from FDA's drug division for addiction drug products to its division for oncology drug products.

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April 5, 2013

Clinical Trials Transparency

clinical trials.jpg
For some time now, there have been strident calls for the publishing of all clinical trial data. The pressure has arisen due to revelations that companies may have hidden crucial clinical data that might have shown that the drug being tested was not as efficacious or even as safe as they appeared from quoted trial results (see, for instance, a previous blog here). The campaign group AllTrials has brought together several people and groups (including for example David Tovey, editor The Cochrane Library; Ben Goldacre (book Bad Pharma); Carl Heneghan, Centre for Evidence-Based Medicine, University of Oxford) because, as they put it:

Around half of all clinical trials have not been published; some trials have not even been registered. If action is not taken urgently, information on what was done and what was found in trials could be lost forever, leading to bad treatment decisions, missed opportunities for good medicine, and trials being repeated unnecessarily.

Key publications like the British Medical Journal and research bodies such as the Medical Research Council and the Wellcome Trust agree, the trust encouraging its grant recipients to release their trial data.

The European Medicines Agency ("EMA") has been looking at this for some time, with the same aim in mind and is trying to develop a workable policy to enable the data to be published. Indeed it is thought that in the next few weeks, major players in the United Kingdom's medical community will meet to try and take things further in a more practical manner.

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April 3, 2013

UK's NICE To Have the Crucial Role in the Value-Based Pricing of Medicines from 2014

Bristish Notes.pngThe 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.

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.

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April 1, 2013

Biosimilar Meeting Guidance Issued by FDA

dna.jpgOn 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.

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.

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March 29, 2013

Judge Pender Featured at ITC Trial Lawyer's Association Luncheon

ITC Building.pngOn March 27, FLH attorneys Brian Malkin and Christopher Gosselin attended the International Trade Commission ("ITC") Trial Lawyer's Association ("ITCTLA") luncheon with administrative law judge ("ALJ") Thomas B. Pender. Judge Pender joined the ITC in October, 2011, and is one of six ALJs at the Commission.

Judge Pender arrived at the luncheon with a number of themes that he wanted to discuss. Chief among them is a concern shared by many other ALJs and district court judges about the scope of electronic discovery in today's litigation. He urged the practitioners in the room not to lose control of the paper, and to reign in the costs and scope of electronic discovery. In Pender's experience, less than 1% of all discovery becomes an exhibit, and less than 5% of those exhibits are ever argued. In addition to paring back discovery, Pender would like to see fewer patents and patent claims being asserted by complainants. Ultimately, Pender would like to see more efforts taken to reduce the cost of litigation at the ITC, and hopes that more streamlined cases will allow him to finish an Initial Determination in a year or less.

Judge Pender also suggested, on a related note, that big firms make an effort to send their associates "to the podium," both to give the associates valuable experience, and to reduce the cost of a trial. In his experience, well-prepared associates perform as well or better than partners who had less time to prepare. Pender cautioned parties to think twice before betting an entire case on one witness, and suggested that secondary witnesses could and should be handled by associates.

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March 27, 2013

Supreme Court Hears AndroGel® Reverse Payments Case

supreme court.jpgOn March 25, 2013, the U.S. Supreme Court heard oral argument in the Federal Trade Commission's ("FTC's") case challenging the Hatch-Waxman patent settlements Solvay (now owned by Abbot Laboratories) entered into with Watson Pharmaceuticals, Par Pharmaceutical, and Paddock Laboratories resolving their disputes involving Solvay's testosterone-replacement drug AndroGel®. The so-called reverse-payment settlements at issue in FTC v. Actavis, Inc., Sup. Ct. No. 12-416 ("AndroGel") involved the generic manufacturers' agreements to abandon their patent challenges and delay generic entry for nine years. The settlements also involved Solvay making certain payments to the generic manufacturers in return for backup manufacturing and marketing support. For additional background information, please see some of our more recent blogs here, here, and here.

The issue before the Court is whether reverse-payment settlements are per se lawful unless the underlying patent litigation was a sham or the patent was obtained by fraud (as the Eleventh and other circuits have held), or instead are presumptively anticompetitive and unlawful (as the Third Circuit held in K-Dur).

Deputy U.S. Solicitor General Malcolm L. Stewart argued on behalf of the FTC that the Court should adopt a "quick look" rule of reason analysis under the antitrust laws whereby reverse-payment settlements will be presumptively anticompetitive unless defendants can show that the payment from the brand to the generic was for a purpose other than delaying generic entry, or the payment offered some pro-competitive benefit. Notably, this quick look approach was adopted by the Third Circuit in K-Dur. Counsel for the respondent drug companies argued that the Court should adopt the "scope of the patent defense" applied by the Second, Eleventh, and Federal Circuits finding these agreements to be lawful absent sham litigation or fraud in obtaining the patent.

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March 20, 2013

OGD Director Resigns and Is Replaced by Kathleen Uhl, M.D.

Thumbnail image for FDA logo.jpgIn a surprising turn of events, last week FDA's new Office of Generic Drugs ("OGD") Director Gregory P. Geba, M.D., M.P.H., voluntarily stepped down from his post after only about eight months after being announced as the new Director. Rather than hiring from within, FDA hired Geba from Sanofi US, where he had previously most recently served as Deputy Chief Medical Officer. FDA said then, "He [Geba] joins OGD at an opportune time to lead our expanding generic program into a reorganization of both structure and process to improve coordination, communication, and efficiency, as well as enhance the Office's ability to ensure that all generic drugs--which make up nearly 80 percent of prescriptions filled in the United States--are safe, effective, of high quality, and interchangeable with the brand name drug product/reference listed drug." Around this same time, FDA moved OGD to the same organizational level as the Office of New Drugs ("OND"), called a "Super office," signaling that the two offices now had the same reporting structure directly to the Director of the Center for Drug Evaluation and Research ("CDER"), Janet Woodcock, M.D., rather than a sub-office, as had previously been the case.

According to reports of an e-mail that Geba sent to FDA staff on March 13, Geba cited the movement of OGD's chemistry divisions into a new Office of Pharmaceutical Quality as one of the lead reasons for his resignation, as well as the relocation of his family to the Washington area. Geba reportedly wrote: "As I see it, two of the original reasons I came to [OGD] . . . would be challenged by resources needed for application to other extremely important efforts of the chemistry group in moving to [the Office of Pharmaceutical Quality." The e-mail explained that while Geba generally supported the transfer of the chemistry group to the Office of Pharmaceutical Quality. Geba thought, however, that the move could make it difficult for him to achieve his goals to approve generic versions of inhalers, topical creams, and other complex drugs, as well as addressing the importance of pill size, shape, color, and other characteristics to patients.

Geba helped FDA with its initial phases for implementing the Generic Drug User Fee Act ("GDUFA"). According to Geba, since GDUFA, OGD decreased the abbreviated new drug application ("ANDA") backlog by nearly 600 applications and approved nearly 200 ANDAs since October 2012.

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March 19, 2013

MassBio Features Hamburg Keynote at Annual Meeting - Part 2 of 2

monoclonal antibodies.pngOn 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."

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.

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