The FDA fast-tracked approval of Spinraza to treat spinal muscular atrophy through the agency's orphan drug program, and doctors say they are gratified to finally be able to give patients some hope. But the high price tags of orphan drugs concern patient advocates, and a recent study showed that some drugmakers may be manipulating the system.
Partnerships are an accepted way to tackle biomedical R&D challenges. Now, we need to analyze their effectiveness and heed the lessons we've learned for the next generation of productive collaboration. Read Executive Director Margaret Anderson's three lessons learned on the path to Collaboration 2.0.
If you were looking for organizations that have patient registries and that partner with industry, where would you search? FasterCures has a resource for that! The Patients Count Network digital directory is a diverse community of patient-centered foundations, from household names to founder-led startups. Create a profile today to increase your organization's visibility with potential partners.
Globally accessible cloud computing platforms are changing the drug development process by allowing real-time data collection from disparate locations and enabling collaboration, says Accenture Life Sciences managing director Kevin Julian. Cloud and related technologies also improve clinical trial recruitment, improve the efficiency of data collection and analysis, and cut costs.
IBM CEO Ginni Rometty described in her HIMSS17 keynote address how big data and predictive analytics are already transforming health care, and said that health technology must be developed in "an ethical and enduring way." Rometty emphasized that technology is being developed to augment -- not replace -- human intelligence.
The NIH's National Institute of Allergy and Infectious Diseases has launched an early-stage study to test the safety of its AGS-v vaccine for mosquito-borne infectious diseases such as dengue, malaria, Zika virus and West Nile virus. AGS-v was created using four synthetic proteins from mosquito salivary glands that should elicit an allergic response from the inoculated person, and researchers will also verify if mosquitoes that bite a vaccinated person experience early demise or diminished capacity for reproduction.
The Department of Defense's Defense Advanced Research Projects Agency awarded Ceres Nanoscience a $750,000 grant to support development of its Nanotrap technology to detect Zika and other pathogens. Ceres will collaborate with George Mason University to develop the platform, which will allow for more sensitive detection and more accurate diagnosis.
Harvard University chemical biologist Greg Verdine has co-founded 10 startups, worked at three venture capital firms and run early-stage biotechnology companies, and now he plans to retire from Harvard in the near future to take the helm of FogPharma full time. The company is developing what it calls cell-penetrating mini-proteins to deliver drugs to otherwise inaccessible targets.
Gilead Sciences bought Sarepta Therapeutics' priority review voucher for $125 million, giving Gilead a potential market advantage in the future, but the price is lower than some similar transactions. Leerink analyst Joseph Schwartz said drugmakers might begin struggling to justify "increasingly astronomical" upfront payments for a faster FDA review.
Backed by venture capital firm Sofinnova Ventures, Dauntless Pharmaceuticals is beginning clinical trials for its first investigational drug, an endocrine cancer treatment delivered noninvasively. Early development is outsourced, and each experimental drug is advanced as a standalone asset by a separate business entity.
Federal legislation intended to help terminally ill patients gain access to drugs that haven't been through the entire FDA approval process could have a chance of getting through Congress this year. Lobbying in support of the Right to Try legislation is picking up, and Vice President Mike Pence supports the idea.
Before Michael Gustafson died of medulloblastoma in 2013, he decided to donate his tissue in the hopes that cancer researchers could help others with the condition. He and his family started the Swifty Foundation to raise funds for pediatric brain cancer research and to support tissue donation for pediatric cancer research.
FasterCures is an action tank that works across sectors and diseases to improve the effectiveness and efficiency of the medical research enterprise. FasterCures, a center of the Milken Institute, is nonpartisan and independent of interest groups.
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