[Press release] NIH to partner with biopharmaceutical companies and nonprofits to diagnose/treat diseases
The Accelerating Medicines Partnership (AMP) is a bold new venture between the NIH, 10 biopharmaceutical companies and several non-profit organizations to transform the current model for developing new diagnostics and treatments by jointly identifying and validating promising biological targets of disease. The ultimate goal is to increase the number of new diagnostics and therapies for patients and reduce the time and cost of developing them.
[At the risk of breaking copyright, this came via Twitter]
AMP will begin with three to five year pilot projects in three disease areas:
- Alzheimer’s disease
- type 2 diabetes
- autoimmune disorders of rheumatoid arthritis and systemic lupus erythematosus (lupus)
For each pilot, scientists from NIH and industry have developed research plans aimed at characterizing effective molecular indicators of disease called biomarkers and distinguishing biological targets most likely to respond to new therapies.
Through this cross-sector partnership, which will be managed through the Foundation for the NIH (FNIH), NIH and industry partners are sharing expertise and resources — $230 million — in an integrated governance structure that enables the best informed contributions to science from all participants. A critical component of the partnership is that industry partners have agreed to make the AMP data and analyses publicly accessible to the broad biomedical community. These pilot projects will set the stage for broadening AMP to other diseases and conditions.
Government Industry Non-Profit Organizations FDANIH AbbVieBiogen Idec
Johnson & Johnson
Alzheimer’s AssociationAmerican Diabetes Association
Lupus Foundation of America
Foundation for the NIH
Geoffrey Beene Foundation
Rheumatology Research Foundation
Budget: 5 years [$230 Million (Rounded) Total Project Funding]
($Millions) Total Project Total NIH Total Industry Alzheimer’s Disease 129.5 67.6 61.9 Type 2 Diabetes 58.4 30.4 28 Rheumatoid Arthritis and Lupus 41.6 20.9 20.7 Total 229.5 118.9 110.6
DNDi welcomes World Health Organization expert recommendations to begin negotiations for an R&D convention at the World Health Assembly
After a decade-long process of analysis and deliberations on ways to better address the health needs of developing countries, a recently released report of the Consultative Expert Working Group on Research and Development: Financing and Coordination (CEWG) will be discussed this week at the 65th World Health Assembly (agenda item 13.14). The Drugs for Neglected Diseases initiative (DNDi), a not-for-profit research and development (R&D) organization, welcomes the experts’ conclusion that ‘the time has now come for WHO Member States to begin a process leading to the negotiation of a binding agreement on R&D relevant to the health needs of developing countries’.
A decade ago, R&D for poverty-related neglected diseases was at a virtual standstill. Since then, there have been significant changes in the landscape for these diseases, with now some 150 new R&D projects, including for drugs, diagnostics, and vaccines, in the pipeline. This progress stems from international pressure and the engagement of new public and private actors and donors in both endemic and non-endemic countries, as well as initiatives such as product development partnerships (PDPs), which were set up to fill the gaps left by policy and market failures.
However, despite incremental progress, the essential health needs of the vast majority of the world’s population are still largely unmet, current R&D efforts are still too fragmented, and financing is still far too fragile. Genuine therapeutic breakthroughs that are adapted to the needs of patients in resource-limited settings and that have the potential to fundamentally transform the treatment of many neglected diseases, notably those with the highest death rates, have yet to make their way through costly clinical trials and reach patients in need.
- Expert Group Recommend That World Health Assembly Should Adopt An International Convention On Global Health R&D (medicalnewstoday.com)
- WHO: Sixty-fifth World Health Assembly (crofsblogs.typepad.com)
- Global Health R&D Needs To Be Harmonized (medicalnewstoday.com)
- World Health Assembly should adopt an international convention on global health R&D: Expert group (medicalxpress.com)
- An international treaty is needed to improve medical research worldwide (eurekalert.org)
- We need a global treaty on health research for the poor (scidev.net)
The traditional way of making medicines from ingredients mixed together in a factory may be joined by a new approach in which doctors administer the ingredients for a medicine separately to patients, and the ingredients combine to produce the medicine inside patients’ bodies.
That’s one promise from an emerging new field of chemistry, according to the scientist who founded it barely a decade ago. Carolyn Bertozzi, Ph.D., spoke on the topic – bioorthogonal chemistry – delivering the latest Kavli Foundation Innovations in Chemistry Lecture at the 243rd National Meeting & Exposition of the American Chemical Society (ACS).
Bertozzi explained that the techniques of bioorthogonal chemistry may fundamentally change the nature of drug development and diagnosis of disease, so that the active ingredients for medicines and substances to image diseased tissue are produced inside patients.
“Suppose a drug doesn’t reach diseased tissue in concentrations high enough to work,” Bertozzi said, citing one example of the potential of the new chemistry. “Maybe it is an oral drug that doesn’t get absorbed very well into the blood through the stomach. You can imagine a scenario in which doctors administer two parts of the molecule that makes up the drug. …
From the 2 March Science Daily article
Cases in which a newly approved drug is more effective than the cheaper alternatives already available are the exceptions rather than the rule.
This is the conclusion reached in a study by Mariam Ujeyl et al. in the current issue of Deutsches Ärzteblatt International.
Research into 39 proprietary medicinal products (PMPs) launched on the German market in 2009 and 2010 shows that there were frequently insufficient data available on efficacy when approval was granted. The legal requirements of the licensing procedure have never yet required direct data comparing a new drug to a commercially available drug.
The researchers’ evaluations also show that for around half of approvals the only trials presented compared the new drug with a placebo, not an effective comparator drug.
This can give rise to room for interpretation regarding pricing when new drugs are marketed. The authors do not even rule out the possibility that these more expensive PMPs may actually be inferior to the alternatives already on the market.
- Drugs: ‘New’ does not always mean ‘better’ (eurekalert.org)
Many NIH-funded clinical trials go unpublished over two years after completion (with ClinicalTrials.gov link for many trial study results)
[Flahiff's note: It is possible that many of these unpublished clinical trial results would have made a positive difference in many people's lives. These unpublished results have the potential of aiding many researchers. They can prevent unnecessary duplicate trials, point to areas needing more research, and potentially provide groundwork for collaboration.
On another note, it is good to see that published research papers are now more accessible to all. As of 2008, research papers based on NIH grants must be submitted to PubMed Central (PMC) when those papers are accepted for publication in a peer-reviewed journal. PMC will then make the papers freely available to the public within 12 months of publication.
I look forward to the day when all research papers are freely available to the public. There are a myriad of issues, as who pays for the publishing, the peer review process, and where the research papers should be "housed". However, I believe the more scientific research results are disseminated in easily accessible format, the more we can advance in technology applications and filling in knowledge gaps.]
In a study that investigates the challenges of disseminating clinical research findings in peer-reviewed biomedical journals, Yale School of Medicine researchers have found that fewer than half of a sample of trials primarily or partially funded by the National Institutes of Health (NIH) were published within 30 months of completing the clinical trial.
These findings appear in the January issue of the British Medical Journal, which focuses on the topic of unpublished evidence.
“When research findings are not disseminated, the scientific process is disrupted and leads to redundant efforts and misconceptions about clinical evidence,” said Dr. Joseph Ross, first author of the study and a Yale assistant professor of medicine. “Such inaction undermines both the trial in question and the evidence available in peer-reviewed medical literature. This has far-reaching implications for policy decisions, and even institutional review board assessments of risks and benefits associated with future research studies.”…
Ross said that there may be many reasons for lack of publication, such as not getting accepted by a journal or not prioritizing the dissemination of research findings. Still, he said, there are alternative methods for providing timely public access to study results, including the results database at ClinicalTrials.gov** that was created in response to Federal law.
[From the About Page at Clinical Trials.gov
US Public Law 110-85 (Food and Drug Administration Amendments Act of 2007 or FDAAA), Title VIII, Section 801 mandates that a "responsible party" (i.e., the study sponsor or designated principal investigator) register and report results of certain "applicable clinical trials" that were initiated or ongoing as of September 27, 2007...]
ClinicalTrials.gov offers up-to-date information for locating federally and privately supported clinical trials for a wide range of diseases and conditions.
ClinicalTrials.gov currently contains 118,682 trials sponsored by the National Institutes of Health, other federal agencies, and private industry.
**Here is how one can check for study results
(remember, researchers are not mandated to submit study results to ClinicalTrials.gov, they are voluntary)
ClinicalTrials.gov records with published results listed via the PubMed medical literature search service.
- Use the Advanced Search with the search phrase clinicaltrials.gov[si]
Use the Builder limit results by topics (as a disease, medical device), year(s), name of researcher/invesitator)
- Need help searching? PubMed has tutorials , including a YouTube at the Advanced Search Page
Ask for assistance from a reference librarian at your local public, academic, hospital, or medical library.
Many academic, hospital, and medical libraries offer at least basic search help to all. Call ahead and ask
about their services. You may be pleasantly surprised.
- Many NIH-funded clinical trials go unpublished over 2 years after completion (eurekalert.org)
- The White House Calls for Information on Public Access to Publications and Data (The Scholarly Kitchen)
- How to obtain free/low cost medical and scientific articles(jflahiff.wordpress.com)
- Patients want to understand the medical literature (with links to resources for patients) (jflahiff.wordpress.com)
- Missing trial data threatens the integrity of medicine (eurekalert.org)
- Poor patient recruitment cited in call for trial disclosures (fiercebiotechit.com)
- A Present for NIH: President Signs Law Creating New Translational Center (news.sciencemag.org)
- NIH and Non-profits Sign Research and Development Agreement (kauffman.org)
- NIH establishes National Center for Advancing Translational Sciences (jflahiff.wordpress.com)
In a move to re-engineer the process of translating scientific discoveries into new drugs, diagnostics, and devices, the National Institutes of Health has established the National Center for Advancing Translational Sciences (NCATS). The action was made possible by Congress’ approval of a fiscal year 2012 spending bill and the president’s signing of the bill, which includes the establishment of NCATS with a budget of $575 million.
NCATS will serve as the nation’s hub for catalyzing innovations in translational science. Working closely with partners in the regulatory, academic, nonprofit, and private sectors, NCATS will strive to identify and overcome hurdles that slow the development of effective treatments and cures.
“Congressional support for the National Center for Advancing Translational Sciences marks a major milestone in mobilizing the community effort required to revolutionize the science of translation,” said NIH Director Dr. Francis S. Collins, M.D., Ph.D. “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.”
A prime example of the type of innovative projects that will be led by NCATS is the new initiative between NIH, the Defense Advanced Research Projects Agency, and the U.S. Food and Drug Administration to develop cutting-edge chip technology. This new technology will allow researchers to screen for safe and effective drugs far more swiftly and efficiently than current methods. A great deal of time and money can be saved testing drug safety and effectiveness much earlier in the process.
To meet the goals of NCATS, NIH is reorganizing a wide range of preclinical and clinical translational science capabilities within NIH into an integrated scientific enterprise with new leadership and a bold new agenda. While the effort to recruit an NCATS director continues, organizational changes and realignment of resources will move forward under the leadership of Acting Director Thomas R. Insel, M.D., and Acting Deputy Director Kathy Hudson, Ph.D. Dr. Insel is the director of the National Institutes of Mental Health and Dr. Hudson is the deputy director for science, outreach, and policy at the National Institutes of Health.
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
The budget for NCATS is primarily a reallocation of funds from programs previously located in the NIH Office of the Director, National Human Genome Research Institute, and National Center for Research Resources. NIH is committed to both basic and applied research and has maintained a relatively stable ratio of funding across these two areas of focus. The funding ratio will not be disturbed by the establishment of this new center.
The formation of NCATS has been a methodical process highlighted by the recommendation of the NIH Scientific Management Review Board in December 2010 to create a new center dedicated to advancing translational science. This recommendation was followed by a year of intensive feedback and expert insight from all sectors of translational science through advisory meetings and extensive public consultation.
“I am deeply grateful for the expertise and insight provided by the many researchers, industry executives, patients, voluntary organizations, and NIH staff that helped NIH evaluate NCATS’ purpose and crystallize its vision,” said Dr. Collins.
To learn more about the impetus and development of NCATS, go to:
- NCATS web page: http://www.nih.gov/about/director/ncats/index.htm.
- NCATS on the Feedback NIH website: http://feedback.nih.gov/index.php/category/ncats/
About the National Institutes of Health (NIH): NIH, the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visitwww.nih.gov.
- NIH Director Jumps the Gun With Memo Announcing New Center (news.sciencemag.org)
- NIH Chided on Translational Center, Warned of More Budget Cuts (news.sciencemag.org)
- NIH centre faces spell in limbo (nature.com)
- White House Boosts Translational Medicine, Drug Chip Project (news.sciencemag.org)
- US biomedical and energy budgets inch toward resolution (blogs.nature.com)
- Senate Panel Trims NIH Budget By $190 Million (news.sciencemag.org)
- New drug-discovery center lost in translation (fiercebiotechresearch.com)
- NIH launches $140M drug tox project, hunts for translational R&D chief (fiercebiotechresearch.com)
- Departing Director Reflects on NCRR Breakup (news.sciencemag.org)
- IEA Places 18 Employees within the National Institutes of Health (NIH) (prnewswire.com)