Last month, the National Institutes of Health, 10 biopharmaceutical companies and several nonprofit organizations launched an unprecedented partnership to transform the current model for identifying and validating the most promising biological targets of disease for new diagnostics and drug development.
According to a new report released last month by the trade group Pharmaceutical Research and Manufacturers of America (PhRMA), the biopharmaceutical pipeline is innovative and robust, with a high percentage of potential first-in-class medicines (meaning a new treatment where nothing currently exists) targeting diseases with limited treatment options. In addition to identifying medicines in development for conditions and diseases such as septic shock, ovarian cancer, sickle cell disease, and Lou Gehrig’s disease (amyotrophic lateral sclerosis), which haven’t had any new product approvals in the last ten years, the report offers positive news for the rare disease community: one third of the products currently in clinical development have a rare disease designation by the U.S. Food and Drug Administration (FDA).
The National Institutes of Health recently unveiled a collaborative program that will match researchers with a selection of pharmaceutical industry compounds to help scientists explore new treatments for patients. NIH’s new National Center for Advancing Translational Sciences (NCATS) has partnered initially with Pfizer, AstraZeneca, and Eli Lilly and Company which have agreed to make dozens of their compounds available for this initiative’s pilot phase.
Over the past decade, collaborative research efforts to support the discovery and development of medicines has increased dramatically. Last month, the National Institutes of Health and Eli Lilly and Company announced a new collaboration: they will generate a publicly available resource to profile the effects of thousands of approved and investigational medicines in a variety of advanced disease-relevant testing systems . In-depth knowledge of the biological profiles of these medicines may enable researchers to better predict treatment outcomes, improve drug development, and lead to more specific and effective approaches.
Biomarker Bulletin is an occasionally recurring update of news focused on biomarkers aggregated at BiomarkerCommons.org. Biomarkers are physical, functional or biochemical indicators of normal physiological or disease processes. The individualization of disease management — personalized medicine — is dependent on developing biomarkers that promote specific clinical domains, including early detection, risk, diagnosis, prognosis and predicted response to therapy.
- Biomarker Commons Named World Companion Diagnostics Summit Media Partner
Diagnostics used to select patients for treatment with a particular therapeutic or determine what and/or how treatment will be administered have been termed companion diagnostics. Companion diagnostics hold great promise for personalized medicine. A companion diagnostic is a biomarker(s) used in a specific context that provides biological and/or clinical information that enables better decision making about the development and use of a potential therapeutic. Given the significance of companion diagnostics, I’m proud to announce Biomarker Commons’ first media partnership with the 4th World Companion Diagnostics Summit.
- Personalized Medicine or Patient-centered Care?
Personalized medicine is a term used in science and medicine that holds significant promise of administering medicines specifically tailored to an individual’s genome or metabolism. However, an editorial published yesterday in the Canadian Medical Association Journal (CMAJ) suggests that the term creates an image for the public that is completely opposite of science and technology and sets up unrealistic expectations.
- FDA, EMA Seek Input on Companion Diagnostics, Genomic Biomarkers
Regulators in the U.S. and Europe have each posted requests for public comment on issues affecting the development of medicines by drug companies.
- FDA Issues New Guidelines on Clinical and Nonclinical Genomic Biomarkers
The U.S. Food and Drug Administration (FDA) has issued new guidelines on biomarkers related to drug or biotechnology product development. The guidance was developed within the Efficacy Working Group of the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH).
- Neuroimaging Identifies an Endophenotype and Candidate Biomarker for Autism
In response to facial expression of emotional, a similar pattern of brain activity is observed in both people with autism and their unaffected siblings. Researchers from the University of Cambridge recently used functional magnetic resonance imaging (fMRI), a specialized MRI scan used to measure the change in blood flow related to neural activity in the brain, to show that reduced activity in areas of the brain associated with empathy and face processing is a candidate biomarker for familial risk of autism. The findings were published online recently in the journal Translational Psychiatry.