Amy Ritter was Scientific Editor, BioPharm International.

Amy Ritter, PhD

When should there be restrictions on the dissemination of basic research results? This question has arisen in the context of papers from independent laboratories submitted to the journals 

. Ron Fouchier and colleagues from the Erasmus Medical Center in Rotterdam, the Netherlands and a team headed by Yoshihiro Kawaoka of the University of Wisconsin-Madison, were both studying the H5N1 strain of flu virus, which causes avian flu. Avian flu can decimate flocks of poultry, but is rarely transmissible to humans. Nevertheless, because of the close proximity in which humans and poultry reside, there is concern that the virus could someday acquire the ability to infect mammals, and become the source of a pandemic. Both laboratories, working independently, identified mutations in H5N1 that allowed mammal-to-mammal transmission, using ferrets as the experimental model.

		In a rare response to those findings, the Department of Health and Human Services, acting on advice from The National Science Advisory Board for Biosecurity (NSABB) asked the authors of the papers and the editors of the journals to publish the papers in redacted form, where the conclusions would be published, but the methodology would not be made public. HHS also asked that language be added to the publications outlining the goals and potential health benefits of the research and detailing the safety measures taken to protect the laboratory workers and the public. In a 

 issued by the National Institutes of Health, the agency said “Recognizing the significant potential benefit of the information about the experimental details to the global influenza surveillance and research communities, the US government is working to establish a mechanism to allow secure access to the information to those with a legitimate need in order to achieve important public health goals. The US government is also developing a proposed oversight policy that would augment existing approaches to evaluating research that has the potential to be misused for harmful purposes.”

		The authors agreed to the government’s request, and went a step further. In open letters published in 

, the authors of the papers jointly called for a 60-day moratorium on the creation of new H5N1 strains, to allow time for public discourse on the merits of the research as well as its risk. In the letter, the authors state: “We recognize that we and the rest of the scientific community need to clearly explain the benefits of this important research and the measures taken to minimize its possible risks. We propose to do so in an international forum in which the scientific community comes together to discuss and debate these issues. We realize that organizations and governments around the world need time to find the best solutions for opportunities and challenges that stem from the work.”

		Scientists are divided on whether redaction of the data is really in the public interest. Nonetheless, the willingness to pause for an open discussion about the risks versus benefits of this type of research will go some way towards balancing public safety with public health goals.

Articles

When should there be restrictions on the dissemination of basic research results? This question has arisen in the context of papers from independent laboratories submitted to the journals Science and Nature.

Public Safety versus Public Disclosure

On Jan. 11, 2012, AstraZeneca and IMS Health announced a three-year collaboration to use real-world healthcare data from Europe to guide AstraZeneca’s discovery and clinical development programs. The partnership will give AstraZeneca access to de-identified electronic health records, which include clinical outcomes, economic, and treatment pattern data. In addition, the companies will jointly develop a customized research and data-analysis platform.

The information will be used to analyze how medicines that are already on the market are being used in real-world clinical applications, and to elucidate the standard of care and treatment patterns across several therapeutic areas, with an emphasis on chronic illnesses. AstraZeneca aims to use the analysis to allocate its R&D and clinical development resources more effectively. Martin Mackay, AstraZeneca’s president of R&D, said in a 

: “Our collaboration with IMS is a key milestone in our commitment to understand the impact of our medicines in the real world, beyond what we see in controlled clinical trials. This insight will help us and healthcare decision-makers to improve the treatment of disease and ensure effective use of medicines to minimize the burden on individuals and healthcare budgets.”

The collaboration with IMS Health to use European health information to analyze real-world healthcare trends follows a similar agreement between AstraZeneca and HealthCore, the health outcomes research subsidiary of WellPoint. The partnership with HealthCore was 

 in February 2011, and aims to analyze US healthcare data to find ways of improving overall patient health while lowering the total cost of care—especially in the treatment of chronic diseases.

AstraZeneca and MRC form Landmark Collaboration

Global Healthcare on the Ground: Merck and AstraZeneca Tackle Cervical Cancer and Tuberculosis

On Jan. 11, 2012, AstraZeneca and IMS Health announced a three-year collaboration to use real-world healthcare data from Europe to inform AstraZeneca's discovery and clinical development programs.

AstraZeneca and IMS Health Announce Data and Research Collaboration

The US Pharmacopeia (USP) announced in a 

 a draft standard contained in the proposed 

. The draft standard contains best practices for ensuring that drugs can be traced to their original manufacturer, are not counterfeited or adulterated, and can be transported to their intended destination without compromising quality.

The proposed standard contains a section on importation that includes best practices for supply-chain risk management, development of effective supplier partnerships, and building a supply-chain quality system. Another section covers counterfeiting, including a definition of counterfeiting and best practices to combat counterfeit drugs and medicines. Included in the best-practices section are discussions of anticounterfeiting strategies such as the use of tamper-resistant packaging technologies and barcoding, as well as guidance for combating illegal Internet pharmacies. Finally, the guidance contains a section on diversion and theft.

In USP’s press release, Dr. Praveen Tyle, chief science officer of USP, says, “There is incentive for all players in the pharmaceutical industry—large and small companies, regulators and standards-setting bodies—to come to some agreement on hot-button issues such as track-and-trace technology and, at the larger level, to codify what constitutes a solid, universal approach to global supply-chain integrity.”

USP is seeking feedback on the draft standard, which will be posted in the 

 38 (2), March–April 2012. The draft chapter and public comments will be the focus of the USP Supply Chain Integrity Workshop to be held on May 22–23, 2012, in Rockville, Maryland. The workshop will provide further opportunity to discuss the proposed standard and to decide if further information should be added.

The US Pharmacopeia announced a draft standard containing best practices for ensuring that drugs can be traced to their original manufacturer, are not counterfeited or adulterated, and can be transported to their intended destination without compromising quality.

USP Announces Proposed Standard for Supply-Chain Integrity

 from Jones Lang LaSalle, a financial and professional services firm specializing in real estate services and investment management, looks at the world in terms of life-sciences investment, and finds some not-so-surprising trends over the past 10 years. The report ranked areas of the world according to how much direct investment they received in life sciences, and compared the rankings made between 2003–2006 with those made between 2007–2010. Those time periods were chosen to represent periods before and after the world-wide economic slowdown. Not surprisingly, on both lists, the US was number one, with Ireland, China, and Singapore also in the top 5 in both analyses. However, there have been some changes over the past few years. Puerto Rico, which ranked number 8 on the 2003–2006 list, failed to make the top 10 ranking in the 2007–2010 list. And Canada and Brazil, two non-European countries, replaced Sweden and France as top 10 recipients of life-sciences investments. India, ranked at number 9 in the 2003–2006 analysis, moved up to number 4 in the more recent ranking.

		Despite the economic slowdown after 2006, the total number of dollars invested in US life sciences far outstripped the rest of the world. The US saw investments of $73.3 billion between 2007–2010, up from $38.7 between 2003–2006, an increase of $34.6 billion. In contrast, the next runner up, China, saw an increase in investments of $10.1 billion, to reach $29.8 billion in 2007–2010. Most other areas of the world witnessed much more modest increases, or declines in investment. For instance, Ireland, which still made the top 10, witnessed a decline in investments of $21.1 billion. This shows that despite the global economic slowdown and all the wailing and gnashing of teeth over the movement of facilities to Asia, the US is still receiving a huge amount of life-sciences investment. Those of us in the US should consider ourselves lucky.

A newly released report from Jones Lang LaSalle, a financial and professional services firm specializing in real estate services and investment management, looks at the world in terms of life-sciences investment, and finds some not-so-surprising trends over the past 10 years. The report ranked

US Still Strong but Europe Waning in Life-Sciences Investment

The National Institutes of Health (NIH) announced in a 

 the establishment of the National Center for Advancing Translational Sciences (NCATS), a center dedicated to the translation of scientific discoveries into new drugs, diagnostics, and devices. The establishment of the new center came as a result of Congress’ approval of the fiscal year 2012 spending bill and the president’s signing of the bill, which contained a budget of $575 million for NCATS.

The budget for NCATS is primarily derived from a reallocation of funds from the NIH Office of the Director, the National Human Genome Research Institute, and the National Center for Research Resources. According to the release, NIH does not expect the establishment of NCATS to adversely affect funding for basic or applied research.

The following programs will comprise NCATS:

Bridging Interventional Development Gaps, which makes available critical resources needed for the development of new therapeutic agents

Clinical and Translational Science Awards, which fund a national consortium of medical research institutions working together to improve the way clinical and translational research is conducted nationwide

Cures Acceleration Network, which enables NCATS to fund research in new and innovative ways

FDA-NIH Regulatory Science, which is an interagency partnership that aims to accelerate the development and use of better tools, standards and approaches for developing and evaluating diagnostic and therapeutic products 

Office of Rare Diseases Research, which coordinates and supports rare diseases research

Components of the Molecular Libraries, which is an initiative that provides researchers with access to the large-scale screening capacity necessary to identify compounds that can be used as chemical probes to validate new therapeutic targets 

Therapeutics for Rare and Neglected Diseases, which is a program to encourage and speed the development of new drugs for rare and neglected diseases.

NCATS aims to be a national center for catalyzing innovations in translational science, and will collaborate with partners in regulatory, academic, nonprofit, and private sectors to identify and overcome hurdles that slow the development of effective treatments and cures. NIH Director Francis S. Collins, said in the release, “Patients suffering from debilitating and life-threatening diseases do not have the luxury to wait the 13 years it currently takes to translate new scientific discoveries into treatments that could save or improve the quality of their lives. The entire community must work together to forge a new paradigm, and NCATS aims to catalyze this effort.”

The National Institutes of Health has established the National Center for Advancing Translational Sciences, a center dedicated to the translation of scientific discoveries into new drugs, diagnostics, and devices.

NIH Establishes National Center for Advancing Translational Sciences

If a clinical trial is run, but the results are never published, what benefit is there to patients, physicians, or the public?

		The British Medical Journal (BMJ) took on this topic by publishing several articles that demonstrate how haphazard and incomplete the public disclosure of clinical trial data is. The FDA Amendments Act of 2007 made publication of a results summary on ClinicalTrials.gov within 12 months of trial completion mandatory for all eligible trials in the US initiated or ongoing as of September 2007. In a 

 published in the January issue of BMJ, Prayle and colleagues  looked at studies completed between Jan 1, 2009 and Dec. 31, 2009, and found that only 22% had been reported on ClinicalTrials.gov by January of 2011. A second 

 by Ross and colleagues looked at NIH-funded trials, and found that of 635 trials completed by December 2008, only 46% had been published in a peer-reviewed journal 30 months later.

		The omission of data can affect how the remaining data are interpreted. Conclusions about a particular drug’s efficacy are sometimes based on meta-analyses, where data from several independent studies are integrated to get a better idea of treatment effects. A 

 by Hart and colleagues reanalyzed 41 meta-analyses of efficacy incorporating unpublished data from FDA, and found the outcome to be the same in only 3 cases.

, Richard Lehman, senior research fellow in the department of primary care at the University of Oxford and Elizabeth Loder, clinical epidemiology editor at BMJ underscore the seriousness of these lapses. “We are not dealing here with trial design, hidden bias, or problems of data analysis-we are talking simply about the absence of the data,” they write. “And this is no academic matter, because missing data about harm in trials can harm patients, and incomplete data about benefit can lead to futile costs to health systems. Moreover, researchers or others who deliberately conceal trial results have breached their ethical duty to trial participants.”

NIH Establishes National Center for Advancing Translational Science