Category: Research Funding

Highlights from the 21st Century Cures Act

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By Rachel Sachs

At the end of January, the House Energy & Commerce Committee released a discussion draft of the 21st Century Cures Act.  This document marks the beginning of the legislative phase of the 21st Century Cures Initiative, during which the Committee has held numerous roundtables and hearings and issued several white papers.  The first discussion draft of the Act, clocking in at nearly 400 pages (even with several sections “to be supplied”), is incredibly wide-ranging, including proposals that could affect every stage of the innovation process.

The discussion draft should be of interest to everyone in the health policy field.  One series of proposals is targeted at the NIH, including more support for the National Center for Advancing Translational Sciences and for the NIH’s BRAIN initiative.  Another set would act on the FDA, including one provision giving new drugs for unmet medical needs the option of 15 years of exclusivity.  This provision, based on the MODDERN Cures Act, is particularly likely to inspire a great deal of controversy and opposition.  The draft also contains a series of proposals designed to promote the development of new antibiotics, in keeping with President Obama’s recent focus on this issue.  Its attention to the use of social media by drug companies and to the FDA’s regulation of health-related software will be of interest to many, as well.

The proposed draft is much too long to catalog fully in this brief blog post, although those who are interested in a broader summary might enjoy the 13-page summary of the Act put out by the Committee, the Science summary by Kelly Servick and Jocelyn Kaiser, or Alexander Gaffney’s comprehensive Regulatory Explainer.  But I do want to highlight one section of the draft which deserves more attention than it has gotten: section 2021, which would create a national Medical Product Innovation Advisory Commission.

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The Revival of Phage Therapy to Fight Antimicrobial Resistance (AMR) – Part III: What about patent protection and alternative incentives?

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By Timo Minssen

In Part II of this blog on legal issues relating to the revival of phage therapy I discussed the US Supreme Court’s decisions in Myriad and Prometheus, which might present major obstacles to the patentability of phage-related technology (a more detailed analysis of the Myriad and Prometheus decisions is available here).

Yet, all is not lost. As indicated in Part II, Myriad does not directly affect the patentability of synthetically modified biological compounds and Prometheus would still allow patents on inventive applications of natural processes and correlations that add new features to “natural laws”. Thus there still seems to be considerable leeway for patenting within the area of page therapy.

One example, mentioned in a recent Nature article, could be the skillful selection and precise combination of different phages in order to attack one specific type of bacteria. Such selections, however, would face a tough battle to overcome the “additional features that add significantly more” and “not identical” thresholds set by Prometheus and Myriad. Another example with even better prospects for patentability relates to genetically modified phages that are – due to human intervention – enabled to target only specific bacteria. This technology was recently presented by MIT researchers at the 2014 American Society for Microbiology Meeting. The researchers led by Timothy Lu had genetically engineered phages that use a DNA-editing system called CRISPR to target and kill only antibiotic-resistant bacteria while leaving other susceptible cells untouched. The significant engineering and alteration of natural products and processes involved in such inventions would most likely meet both the Myriad and Prometheus standards.

Yet, while the USPTO has recently issued new patent eligibility guidance and the CAFC has begun to directly apply Prometheus and Myriad to reject patent claims in biotech cases (e.g. In re Roslin), many questions remain unsolved. In particular, it is still not sufficiently clear exactly how much modification is required to render a molecule or method sufficiently distinct from naturally occurring product and processes. And even if the patent-eligibility threshold could be met in extraordinarily circumstances, the claimed invention would still have to fulfil other patentability requirements such as novelty, non-obviousness and the written description-requirements. The threshold for these requirements, however, have been heightened in recent years (see e.g. KSR v. Teleflex (2007) , Nautilus (2014) etc.). Considering that phage therapy is almost a century old with a substantial common general knowledge and a state of the art employing routine methods, these crucial requirements might still prevent the patentability of many useful applications.

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The Revival of Phage Therapy to Fight Antimicrobial Resistance – Part II: What about patent protection and alternative incentives?

The Petrie-Flom Center Staff 4 Comments

By Timo Minssen

Three days ago I commented on a couple of legal issues raised in the recent Nature report “Phage therapy gets revitalized”  by Sara Reardon. One challenge concerns the reluctance of pharma companies to broadly invest in the development of phage therapies. As pointed out in the report, this does of course very much (but not only) relate to the question of patentability. Various aspects might present obstacles to the patentability of technology relating to phage therapy. To not complicate the discussion and considering recent developments I decided to focus on some of aspects under US patent law.

Like in Europe, the first door to patentability that phage-related technology would need to pass concerns patent eligibility. In the last years the US Supreme Court has rendered an astonishing number of fundamental patent-decisions, including not less than four (!) landmark judgments on patent eligibility, i.e. Bilski v. Kappos (2010), Mayo v. Prometheus (2012) , AMP v. Myriad (2013)  and Alice v. CLS (2014). Most relevant in this context are the decisions in Prometheus and Myriad.

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My Slate Article on the Importance of Replicating Science

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By Michelle Meyer

I have a long article in Slate (with Chris Chabris) on the importance of replicating science. We use a recent (and especially bitter) dispute over the failure to replicate a social psychology experiment as an occasion for discussing several things of much broader import, including:

  • The facts that replication, despite being a cornerstone of the scientific method, is rarely practiced (and even less frequently published) not only in psychology but across science, and that when such studies are conducted, they frequently fail to replicate the original findings (let this be a warning to those of you who, like me, cite empirical literature in your scholarship);
  • Why replications are so rarely conducted and published, relative to their importance (tl;dr: it’s the incentives, stupid);
  • Why it’s critical that this aspect of the academic research culture change (because academic science doesn’t only affect academic scientists; the rest of us have a stake in science, too, including those who fund it, those who help researchers produce it (i.e., human subjects), those who consume and build on it (other scholars and policy-makers), and all of us who are subject to myriad laws and policies informed by it); and
  • Some better and worse ways of facilitating that cultural change (among other things, we disagree with Daniel Kahneman’s most recent proposal for conducting replications).

How an IRB Could Have Legitimately Approved the Facebook Experiment—and Why that May Be a Good Thing

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Image courtest Flickr
Image courtesy Flickr

By Michelle Meyer

By now, most of you have probably heard—perhaps via your Facebook feed itself—that for one week in January of 2012, Facebook altered the algorithms it uses to determine which status updates appeared in the News Feed of 689,003 randomly-selected users (about 1 of every 2500 Facebook users). The results of this study—conducted by Adam Kramer of Facebook, Jamie Guillory of the University of California, San Francisco, and Jeffrey Hancock of Cornell—were just published in the Proceedings of the National Academy of Sciences (PNAS).

Although some have defended the study, most have criticized it as unethical, primarily because the closest that these 689,003 users came to giving voluntary, informed consent to participate was when they—and the rest of us—created a Facebook account and thereby agreed to Facebook’s Data Use Policy, which in its current iteration warns users that Facebook “may use the information we receive about you . . . for internal operations, including troubleshooting, data analysis, testing, research and service improvement.”

Some of the discussion has reflected quite a bit of misunderstanding about the applicability of federal research regulations and IRB review to various kinds of actors, about when informed consent is and isn’t required under those regulations, and about what the study itself entailed. In this post, after going over the details of the study, I explain (more or less in order):

  • How the federal regulations define “human subjects research” (HSR)
  • Why HSR conducted and funded solely by an entity like Facebook is not subject to the federal regulations
  • Why HSR conducted by academics at some institutions (like Cornell and UCSF) may be subject to IRB review, even when that research is not federally funded
  • Why involvement in the Facebook study by two academics nevertheless probably did not trigger Cornell’s and UCSF’s requirements of IRB review
  • Why an IRB—had one reviewed the study—might plausibly have approved the study with reduced (though not waived) informed consent requirements
  • And why we should think twice before holding academics to a higher standard than corporations

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“That’s a Lot of Marijuana”

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By Nadia N. Sawicki

Earlier this month, the Drug Enforcement Administration issued notice that it would be increasing the 2014 production quota for marijuana from 21 kilograms to 650 kilograms – an almost 3000% increase. In the words of DEA spokeswoman Barbara Carreno, “That’s a lot of marijuana.” This step, according to the National Institute on Drug Abuse (NIDA), was a necessary response to a dramatic increase in current and proposed marijuana research. Read More

A More Transparent System for Clinical Trials Data in Europe – Mind the Gaps!

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By Timo Minssen

Following the approval of the European Parliament (EP) earlier last month, the Council of the European Union (the Council) adopted on 14 April 2014 a “Regulation on clinical trials on medicinal products for human use” repealing Directive 2001/20/EC.  As described in a press-release, the new law:

“aims to remedy the shortcomings of the existing Clinical Trials Directive by setting up a uniform framework for the authorization of clinical trials by all the member states concerned with a given single assessment outcome. Simplified reporting procedures, and the possibility for the Commission to do checks, are among the law’s key innovations.”

Moreover, and very importantly, the Regulation seeks to improve transparency by requiring pharmaceutical companies and academic researchers to publish the results of all their European clinical trials in a publicly-accessible EU database. In contrast to earlier stipulations which only obliged sponsor to publish the end-results of their clinical trials, the new law requires full clinical study reports to be published after a decision on – or withdrawal of – marketing authorization applications. Sponsors who do not comply with these requirements will face fines.

These groundbreaking changes will enter into force 20 days after publication in the Official Journal of the EU. However, it will first apply six months after a new EU portal for the submission of data on clinical trials and the above mentioned EU database have become fully functional. Since this is expected to take at least two years, the Regulation will apply in 2016 at the earliest (with an opt-out choice available until 2018).

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New regulatory pathways and incentives for sustainable antibiotics: Recent European & US Initiatives

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By Timo Minssen

Please find attached a ppt presentation on “New regulatory pathways and incentives for sustainable antibiotics: Recent European & US Initiatives” given on March 7, 2014 at the Broad Institute of MIT and Harvard.  The presentation was followed by a discussion moderated by US patent attorney Melissa Hunter-Ensor, Partner at Saul Ewing, Boston.

I started out by emphasizing increasing problems of antimicrobial resistance (AMR) on a global level, providing new statistics and facts. This was followed by a discussion of main reasons for these alarming developments, such as inappropriate use in agriculture and medicine, insufficient precautions, lack of education, climate change, travel behavior, insufficient collaboration and funding of R&D, scientific complexities, and the problem that incentives provided by the traditional innovation system model often fail in the case of antibiotics.

Next the presentation focused on a variety of solution models that could be discussed to fight AMR. These include both conservational and preventive approaches comprising use limitations, increased public awareness, and better hygiene, but also reactive push & pull strategies, such as increased investments, new collaborative models for R&D in antibiotics, prizes, “sui generis” IP-related incentives, regulatory responses and new pathways for approval.

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Clinical Trials of Primary Care Drugs: Could Smaller Be Better?

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By Kate Greenwood

Cross-Posted at Heath Reform Watch

Lately it seems that each passing day brings another article about the cost of orphan drugs.  Earlier this week at FiercePharma, Tracy Staton reported that the United Kingdom’s National Institute for Health and Clinical Excellence (NICE) has asked Alexion Pharmaceuticals to justify the price of its drug Soliris which is, per Staton, “the most expensive drug in the world” at around $569,000 a year.  Specifically, NICE seeks “‘clarification from the company on aspects of the manufacturing, research and development costs’” of the drug.  According to Staton, this latest development in a review process characterized by “halting progress” is “a departure from NICE’s usual calculations, which typically focus on quality-of-life years and the like.”

Pushback by NICE and other payers notwithstanding, the orphan drug market is growing.  As I blogged about here, in 2013 EvaluatePharma estimated that “the worldwide orphan drug market is set to grow to $127 [billion], a compound annual growth rate of +7.4% per year between 2012 and 2018[,]” which “is double that of the overall prescription drug market, excluding generics, which is set to grow at +3.7% per year.”  In a recent article in the New England Journal of Medicine, venture-capital investors Robert Kocher and Bryan Roberts note that “more than half of the 139 drugs approved by the FDA since 2009 are for orphan diseases” and suggest that there is a risk of “systematically underinvesting in other important areas of medicine.”

Kocher and Roberts’ explain that one reason that orphan drugs attract investment is that their development costs are low.  The problem or potential problem of underinvestment in diseases like depression and diabetes could therefore be addressed, they contend, by bringing the cost of developing treatments for these common conditions in line with the cost of developing treatments for rare diseases.  And, they argue, one promising approach to doing so is to reduce clinical trial costs by reducing the size of clinical trials. Read More