In an effort to speed up the discovery of new medicines, the Innovative Medicines Initiative (IMI) has created the European Lead Factory, a Europe-wide public-private partnership that combines the Joint European Compound Collection, a comprehensive collection of candidate drug molecules, with an industry-like screening center.

The pharmaceutical companies involved have committed to contributing at least 300,000 chemical compounds from their corporate collections. Teams of universities and small and medium-sized enterprises will have access to this unique library of chemicals and are expected to add another 200,000 compounds. The screening center will build on industry’s expertise in high-throughput screening—a testing method in which robots test hundreds of thousands of unique chemicals for biological activity.

The European Lead Factory will provide to public partners an industry-like discovery platform to translate cutting-edge academic research into high-quality candidate drug molecules on a scale and speed not possible previously. Such candidates will be directly introduced to the drug development process for further refinement or will serve as research tools to improve understanding of disease mechanisms.

The total budget is $222 million: up to $105 million will be provided by the European Commission’s seventh Framework Program for Research (FP7) and up to $117 million will be provided in in-kind contributions by the participating companies.

The Center for Genomic Regulation (CRG), an international biomedical research institute of excellence supported by the Catalan government and the Spanish Ministry of Science and Innovation, is collaborating with Sanofi to share research programs using state-of-the-art experimental platforms and computational and bioinformatics approaches in computational biology, medical genetics and epigenetics, with special emphasis in genetic and rare diseases.

This strategic partnership is based on the cross-fertilization of R&D from both sides, including scientific resources, structures and information, technical equipment and human talent.

"An institute like the Center for Genomic Regulation must ensure that its research has a positive impact on human health and national economies. Yet, we cannot do this alone and we need to collaborate with strategic partners for this purpose,” said Dr. Luis Serrano, director of the Center for Genomic Regulation. “Sanofi will have increased access to globally cutting-edge research and the CRG will be linked to a major company that can ensure the translation of research into real-life solutions for patients."

The CRG and Sanofi have initiated a first set of four projects to discover innovative therapeutic approaches for infectious diseases, develop novel delivery systems using synthetic biology, decipher disease-relevant cellular trans-differentiation pathways and identify original targets from unexploited genomic transcription mechanisms. Additional projects will be selected and launched within the three-year agreement period.

The CRG's mission is to discover and advance knowledge for the benefit of society, public health and economic prosperity. It is committed to technology transfer and the return of knowledge to society. It has a dedicated technology transfer office and a team of professionals with experience in the biotechnology and pharmaceutical businesses, managing the intellectual property generated in the institute and facilitating its transfer to the companies within the sector as patents and licenses that enable the development of innovative products and services.

Federal laws that motivate or require drug and biologic developers to conduct pediatric studies have yielded beneficial information to guide the use of medications in children, according to a new report by the Institute of Medicine (IOM). Still, studies involving children continue to be limited, especially in certain areas such as medication use in newborns and long-term safety and effectiveness in children.

The report identifies ways Congress and the FDA could further improve the utility of clinical information obtained from pediatric studies, including expanding innovative strategies to research drugs and biologics in children, using the FDA's authority to require long-term pediatric studies of possible safety risks and giving the FDA flexibility to impose sanctions for unreasonably delayed studies.

Conducting research with children is inherently more difficult than with adults. Pediatric patients also offer drug companies a much smaller market and potential economic return. Clinicians often treat children with drugs that have been approved for use in adults but have not been studied with children, even though the drugs may have different risk/benefit profiles for pediatric patients.

Recognizing a shortage in knowledge of how medications affect children, Congress has sought to increase pediatric studies under two laws, the Best Pharmaceuticals for Children Act (BPCA), which offers companies economic incentives to study medications in children, and the Pediatric Research Equity Act (PREA), which requires such studies in specific situations. Both laws are due for reauthorization this year. As specified by Congress, the FDA asked the IOM to review certain aspects of studies that have been conducted under the laws.

The laws have had positive effects, according to the report. They have spurred the development of helpful information about the uses of therapies in pediatric patients and have expanded access to information from these studies, including FDA reviews of clinical data submitted by study sponsors.

The report suggested the FDA consider more frequent use of its authority to require sponsors to undertake long-term follow-up studies after products have been approved for market. Because children's bodies and minds are continuing to develop and because some therapies for chronic conditions may be used for many years, pediatric studies of drug safety and effectiveness over the long term are important but are not commonly required.

To promote studies of newer and more widely used drugs that have not been adequately evaluated in newborns and premature infants, Congress could provide additional resources for short- and long-term neonatal drug studies through the existing BPCA program at the National Institutes of Health, the report said.

To improve the timeliness of pediatric studies, Congress could specify that sponsors submit their plans for pediatric studies at the end of phase II trials involving adults. Congress could consider providing the FDA with more flexibility to impose sanctions, including monetary penalties, for unreasonably delayed studies.

Eli Lilly has launched a global Innovation Starts Here initiative, which includes the Lilly Research Awards Program and the Lilly Innovation Fellowship Awards. Through both, Lilly scientists will collaborate with academic researchers on projects to aid in the advancement of Lilly's pipeline. These programs also will further Lilly's investment in R&D in Europe and the U.S.

The Innovation Fellowship Awards Program will foster post-doctoral career development through the selection of innovative research proposals. The awards establish a pre-competitive academic-industry training partnership in which a post-doctoral fellow and academic mentor are paired with a Lilly scientist to provide the industry resources to enable the advancement of the post-doctoral scientists' research proposal.

The Research Awards Program brings together scientists worldwide in an effort to further the advancement of research in therapeutic areas in which Lilly has expertise, including neuroscience, cancer, diabetes and immune system disorders. The program provides a pre-competitive environment in which scientists in academia gain invaluable access to tools to conduct basic research, and Lilly scientists receive critical information to help inform the future of drug discovery and development. Currently, Lilly is visiting academic institutions worldwide to invite them to participate.

A report by research group Kalorama Information has found more than a third of clinical trials conducted by pharmaceutical firms are now outsourced to vendors.

In her report, Outsourcing in Drug Development: The Contract Research (Clinical Trial) Market, Mary Anne Crandall wrote outsourcing clinical trials has moved from being a "should" to a "must."

Naturally, the amount spent on outsourcing also is increasing, up 6.6% to $36.6 billion in 2011 from $31.8 billion in 2009. Revenues allocated for outsourcing are expected to increase at an annual rate of 13.5% over 2009 to 2016, reaching almost $60.8 billion by 2016, according to the report, culled from publicly available information and interviews with CROs.

CROs held 68% of the outsourcing market in 2011, while academic medical centers had 26%. The remaining 6% is shared among other organizations, including managed site networks, clinical trial labs and niche competitors.

Crandall said one finding that shocked her was the number of trials being conducted outside the U.S. "I expected to see 60% or 70% of trials still being done in the U.S., and instead it was much less than that,” she said.

Crandall used the ClinicalTrials.gov registry, which at the time included 129,085 trials at 70,000 sites in 180 countries. The findings: approximately 44.1% of active investigative sites were located in the U.S.; 22.8% in Europe; 6.8% in Canada; 11.5% in Asia; 6% in the Middle East; 6% in the Pacific regions; and 8.8% in the rest of the world.

Some of the major trends the industry is seeing currently that will continue to have an impact on the contract drug development industry, according to the report, include: globalization of drug development; rapid technological change increasing the amount of data; biotechnology industry growth; increased regulatory scrutiny, contributing to the complexity of clinical trial conduct; the lack of minority clinical trial participation; the need for fast, efficient and cost-effective drug development; clinical trials budgeting; and clinical development opportunities in biosimilars.

Europe’s neuGRID, a cloud-computing infrastructure that stores and analyzes a vast database of 3D brain scans, is joining with Canada’s CBRAIN and the U.S.’s LONI to create outGRID, a global online system to centralize and boost Alzheimer’s research initiatives.

The convergence of e-infrastructures will be supported by the United Nations’ International Telecommunication Union (ITU) and will allow scientists worldwide to share their medical data while accessing the extensive information available online, helping them to draw more precise conclusions about the onset and development of Alzheimer’s.

"outGRID will allow for fully exploiting research efforts developed around the world, which are currently fragmented and often duplicated,” said Dr. Giovanni Frisoni, neuGRID and outGRID coordinator. “Most countries are devoting resources to neuroimaging studies on earlier Alzheimer’s diagnosis and the development of disease markers to be used in clinical drug trials, but a lack of coordination between these initiatives limits the prospect of making substantial scientific advancements."

Faculty of 1000 (F1000) is set to launch F1000 Research, a new fully open access publishing program across biology and medicine, later this year.

The program will address the major issues afflicting scientific publishing today: timely dissemination of research, peer review and sharing of data. Diverging from traditional journal publishing, F1000 Research will offer immediate publication; open, post-publication peer review; open revisioning of work including ongoing updates; and encouragement of raw data deposition and publication.

In addition, F1000 Research will accept a broad range of article formats and will encourage content types now routinely rejected, such as negative results, case studies, thought experiments, preliminary analyses and incomplete data sets.

"The open access model has addressed effectively the issue of inadequate access to research findings," Vitek Tracz, chairman and founder of F1000. "It did not address the major issues around communicating the research finding: the delays in access, the inadequacies of peer review and the complexities of data publishing. It is up to collaboration between researchers and publishers to come up with a solution, and we are determined to be a part of it."

F1000 was conceived in 2002 as a collaboration of 1000 international faculty members. It identifies and evaluates the most important articles in biology and medical research publications.

America’s biopharmaceutical research companies are working on 54 medicines to treat chronic obstructive pulmonary disease (COPD), according to a report by the Pharmaceutical Research and Manufacturers of America (PhRMA).

These therapies are in late-stage development, meaning they are either in clinical trials or awaiting review by the FDA.

Today, more than 13 million American adults suffer from COPD, one of the most common lung diseases in the U.S. A chronic, progressive lower respiratory disease, COPD is characterized by obstructed airflow to the lungs that interferes with normal breathing.

America’s biopharmaceutical researchers are exploring various new ways to attack COPD. Examples include: An adult stem cell therapy that targets a protein in the blood often elevated in COPD; a monoclonal antibody that acts on IL-1 receptors involved in inflammatory conditions; and a medicine that targets the underlying inflammation in COPD.

More than 120,000 Americans die from COPD each year, making it the third leading cause of death in the U.S. COPD costs the nation nearly $50 billion annually, including direct and indirect healthcare costs, according to the National Institutes of Health.

The Association of Clinical Trials Organizations (ACTO) says the 2010 crisis brought on by Russia’s new regulations and the transfer of approval authority from Roszdravnadzor to the Health Ministry has been resolved.

Reflecting this conclusion is the 2011 annual data, as well as the resolution of many issues presented in the new legislature, such as the ban on importing registered medicinal products, trial participant insurance issues and medical institute re-accreditation.

ACTO said Russia approved 567 clinical trials in 2011, a decrease of 1.7% from 2009 but an increase of 17.6% from 2010, when new regulations resulted in an unusually low number of requests.

Of the 567 approvals, 370 were international multicenter clinical trials—a record high since recorded statistics began in 2004 and a 6.3% increase from 2009. Other types approved in 2011 included 35 local trials with foreign sponsors (+9.4%), 19 bioequivalence studies with foreign sponsors (+137.5%), 80 local trials with local sponsors (-28.6%) and 63 bioequivalent studies with local sponsors (-18.2%).

Despite the unusually low numbers in 2010, there was still a notable decrease in all local sponsor trial and study approvals from 2010 (200) to 2011 (143), the lowest in the last seven years. This decrease could be in correlation with the sudden increase in international multicenter trials, which accounted for 65.3% of the 2011 total.

Breaking down 2011, the fourth quarter was by far the most productive, with 234 approvals—nearly double the activity in any other quarter. More specifically, of the 19 approved bioequivalence studies with foreign sponsors in 2011, 16 were approved in the fourth quarter. This sudden increase could be due to the eight-month ban on imported registered medical products, which was lifted in June 2011. Since the time between requesting and gaining approval for trials is so long, trials registered in the summer would not be approved until the fourth quarter.

ACTO said Russian clinical trials are still suffering from regulatory and administrative issues. The timeframe for issuing approvals has always been difficult and has become exacerbated under the Health Ministry. After six months of monitoring, ACTO found the Health Ministry has increased waiting time by 30%, and in some cases the waiting time is actually two to four times longer than designated in the law. ACTO suggested adopting new regulations that clearly specify timelines or, instead, removing the requirement to re-file documents to conduct trials (which forces international trials to apply twice for approval).

Transparency Life Sciences (TLS), the first drug development company based on open innovation, has launched, introducing a prototype of its crowdsourced web platform that allows patients, physicians, researchers and other stakeholders to contribute to the design of clinical studies.

TLS’ goal is to develop therapies for significant unmet medical needs by acquiring promising drug compounds and testing them in clinical trials that leverage 21st century information technology to achieve unprecedented productivity.

"The communications and information revolutions have transformed nearly everything around us, yet the design and execution of clinical trials have changed little in the past 40 years,” said Tomasz Sablinski, M.D., Ph.D., founding CEO of TLS. “Transparency Life Sciences intends to use its own pipeline of compounds to demonstrate that an open innovation approach to drug development can deliver high-quality results that facilitate regulatory review and are more patient-centric. And we believe that our approach can accomplish this faster and at a much lower cost than conventional clinical studies."

Transparency’s approach is based on three principles. First, collaborative intelligence, also known as crowdsourcing, will be employed for the clinical protocol design phase, with the participation of medical experts, front-line physicians, patients and others, resulting in protocols that are focused on parameters most relevant to clinical decision-making and practice.

Second, Transparency is leveraging contemporary health information and communications technologies to implement patient-centric clinical trials that will reduce burdens on subjects and sponsors and enhance data quality. Third, TLS intends to be a leader in demonstrating how transparency throughout the clinical trial process can enhance drug development.

Clinical protocols for Transparency’s first three repurposed compounds are now available for collaborative input on the TLS crowdsourced web platform. The first is for the widely used anti-hypertensive drug lisinopril, which animal studies suggest may have efficacy in treating multiple sclerosis (MS). Transparency is in late-stage negotiations for an exclusive option to license lisinopril for development as a new treatment for MS.

Second is the phase II protocol design for sulodexide, a heparin-like compound that has demonstrated potential in animal models of peripheral vascular disease. The third protocol is for a phase Il trial of low-dose naltrexone as a potential treatment for inflammatory bowel disease.

No one knows exactly how many people currently participate in clinical trials around the world. Commercial research companies and drug sponsors keep their own databases of patients, but there is no central repository. Nor are federally-funded organizations such as the National Institutes of Health required to keep any such statistics.

But recommendations in the report recently issued by the Presidential Commission for the Study of Bioethical Issues could change that. One of the 14 recommendations in "Moral Science: Protecting Participants in Human Subjects Research" called for each federal department or agency supporting research with human subjects to maintain a core set of data for its research programs that includes the title and lead investigator of each project, the location of each study and the dollar amount appropriated for the research.

"We have more information on animals involved in research than we do people,” said bioethicist Art Caplan, the Emanuel & Robert Hart director of the Center for Bioethics and Sidney D. Caplan professor of medical ethics at the University of Pennsylvania. “No one really knows anything about who’s involved in human research."

A particularly stark example: During its research, the commission requested information from 18 agencies that conduct most federal human subjects research, and many could not provide basic data about the research they support. The Pentagon, for example, needed more than seven months to prepare information on specific studies supported by the Department of Defense.

In its report, the commission found the federal government supported more than 55,000 projects involving human subjects around the globe in fiscal 2010, mostly in health-related research, but also in education, engineering and social science.

The report said each office should develop or improve publicly available electronic systems or release information through a government-wide system. The commission suggested the Office for Human Research Protections should administer a central web-based portal linking to each individual department or agency system. In addition, the government should consider developing a unified federal research database.

Another key recommendation: agencies that fund research should study methods for compensating volunteers injured during trials. Since the benefits of research help to improve society, the commission argued, participants injured during studies should not have to personally bear the costs of treating their injuries, which is the case now.

Thirteen pharmaceutical companies, the U.S., U.K. and U.A.E governments, the Bill & Melinda Gates Foundation, the World Bank and other global health organizations will join forces to eliminate or control 10 neglected tropical diseases (NTDs) by the end of the decade.

Partners pledged to bring a unique focus to defeating these diseases and to work together to improve the lives of the 1.4 billion people worldwide affected by NTDs, most among the world's poorest.

In the largest coordinated effort to date to combat NTDs, the group said it plans to: sustain or expand existing drug donation programs to meet demand through 2020, share expertise and compounds to accelerate R&D of new drugs and provide more than $785 million to support R&D efforts and strengthen drug distribution and implementation programs.

The partners also endorsed the "London Declaration on Neglected Tropical Diseases," in which they pledged new levels of collaborative effort and tracking of progress. The Gates Foundation announced a five-year, $363 million commitment to support NTD product and operational research.

To guide the effort against NTDs, the World Health Organization (WHO) unveiled a roadmap for implementation that sets targets for what can be achieved by the end of the decade.

"The efforts of WHO, researchers, partners and the contributions of industry have changed the face of NTDs. These ancient diseases are now being brought to their knees with stunning speed," said Dr. Margaret Chan, director-general of the WHO. "I am confident almost all of these diseases can be eliminated or controlled by the end of this decade."

New commitments from partners will close the funding gap to eradicate Guinea worm disease and expedite progress toward the 2020 goals of eliminating lymphatic filariasis, blinding trachoma, sleeping sickness and leprosy, and control of soil-transmitted helminthes, schistosomiasis, river blindness, Chagas disease and visceral leishmaniasis.

Speaking on behalf of the CEOs of the 13 pharmaceutical companies involved, Sir Andrew Witty, CEO of GlaxoSmithKline, said, "Many companies and organizations have worked for decades to fight these horrific diseases. But no one company or organization can do it alone. We pledge to work hand-in-hand to revolutionize the way we fight these diseases now and in the future."

With new and existing pledges totaled, companies will donate an average of 1.4 billion treatments each year to those in need, according to the International Federation of Pharmaceutical Manufacturers & Associations (IFPMA). In addition, new R&D collaborative efforts and access agreements with 11 companies and the R&D organization Drugs for Neglected Diseases initiative (DNDi) are providing unprecedented access to compound libraries that could lead to new treatments.

HUYA Bioscience International has formed a strategic partnership with the Tianjin Institute of Pharmaceutical Research (TIPR). The collaborative agreement will promote advancement of China’s drug innovations.

HUYA and TIPR will collaborate to promote drug development in China and worldwide. HUYA will have the first opportunity to evaluate certain R&D projects conducted at TIPR and help globalize select programs from TIPR.

South San Francisco-based Janssen Alzheimer Immunotherapy together with Pfizer, its collaborator on the Alzheimer's Immunotherapy Program (AIP), and the Geoffrey Beene Gives Back Alzheimer's Initiative have introduced the Alzheimer's Challenge 2012, calling for inventive concepts to help improve the diagnostic identification and tracking of Alzheimer's disease.

The challenge seeks the development of simple, cost-effective, consistent tools to easily assess memory, mood, thinking and activity level over time to help improve diagnosis and monitoring of people with Alzheimer's disease.

The challenge supports the U.S. Department of Health and Human Services (HHS) call to harness new thinking to deliver better care and better health at lower cost and provides an entrepreneurial springboard to harness new approaches to improve Alzheimer's care.

"In the United States, more than five million people have Alzheimer's disease—that's one in every eight people aged 65 and over. The odds increase to nearly one-in-two for people who are 85 years old," said Suzanne Blaug, head of Janssen Alzheimer Immunotherapy. "As we look toward the implementation of the first-ever National Plan to Address Alzheimer's Disease, the United States is taking critical steps to address the enormous toll that Alzheimer's is placing on families, their caregivers and the health care system. We're eager to collaborate with others, as it will take many of us working together to address one of the largest health care challenges of our time."

The challenge includes awards totaling $300,000: $25,000 to five finalists and $175,000 to one winner. "We believe someone out there has the answer and shares our sense of urgency," said Meryl Comer, president of The Geoffrey Beene Foundation Alzheimer's Initiative.

Five finalists will be selected and announced by April 16, each being awarded $25,000. Concept refinement will be completed by mid June. The winner will be announced at the end of June.

The Alzheimer's Immunotherapy Program of Janssen Alzheimer Immunotherapy and Pfizer is an equal collaboration, its research focusing on the beta amyloid hypothesis. Scientific evidence supports the idea that preventing the accumulation and/or promoting the removal of beta-amyloid may have the potential to slow the progression of Alzheimer's and help preserve function in people with the disease. This theory is being tested in clinical trials.

Today, 282 medicines to treat children and adolescents are in clinical trials or under review by the FDA, according to a report by Pharmaceutical Research and Manufacturers of America (PhRMA).

Of the 282, 54 are for cancer, 49 for infectious diseases, 48 for genetic disorders and 25 for neurologic disorders. In addition to developing new medicines, researches are testing existing medicines to determine safe and effective dosage levels for children.