The FasterCures Track: March 2014 - Bringing innovators into the spotlight

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The FasterCures Track
One of the most popular features on FasterCuresTRAIN Central Station is the Innovator Spotlight, which catalyzes the innovative activities of participating disease research organizations. Each month we feature an innovator in venture philanthropy focused on establishing new efforts to accelerate medical solutions.

We've expanded this section to include a new series that profiles individuals who power some of the most creative consortia in medical research. It is part of FasterCures' larger Consortia-pedia project, which dissects the research-by-collaboration model to understand what's working and aims to establish an operational framework that can be applied across any multi-stakeholder collaboration.
  • Laura Esserman of I-SPY 2, which is bringing better breast cancer therapies to market faster; and
  • Michel Goldman of the Innovation Medicines Initiative (IMI) a €2 billion investment made jointly by the European Union and the European Federation of Pharmaceutical Industries.

In the spirit of innovation and progress, I'm happy to share that George Washington University's School of Public Health received an $80 million boost in grants from the Milken Institute, the Milken Family Foundation and Sumner Redstone's foundation in what GWU President Steven Knapp called "a pivotal moment in bringing philanthropy to bear on some of the most challenging health issues that humankind faces right now." This continues the longtime commitment of the Institute to improving lives around the world by enhancing health. Learn more about the Milken Institute School of Public Health at George Washington University.
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Margaret Anderson
Executive Director

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Program Highlights

FasterCures has a number of programs focused on bringing efficiency to the medical research process by identifying and eliminating the roadblocks that slow medical research down. This month, we're featuring updates on our efforts on our Benefit-Risk, TRAIN (The Research Acceleration and Innovation Network), and PAS (Philanthropy Advisory Service) programs.

FDA initiatives incorporate patient input and benefit-risk framework into real-life review process
What's on the regulator's mind?

Evaluating the balance between the benefits and risks of products under review is among the most important elements regulators juggle across the lifecycle of a product and is receiving intense focus at the moment. In a recent Webinar hosted by FasterCures, Andrea Tan and Pujita Vaidya from the U.S. Food and Drug Administration (FDA) reviewed the status of two new efforts: a structured framework for assessing benefits and risks of products and a Patient-Focused Drug Development Initiative that provides patients a pathway for providing input to regulators on the specifics of their conditions and current treatment options. Following the FDA's presentation, Eric Gascho of the National Health Council (NHC) discussed a new Patient Information Tool NHC developed to help patient communities gather the kind of information important to both regulators and developers. View the Webinar slides, archive, and summary.

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Five things you should know about benefit-risk

K. Kimberly McCleary, director of strategic initiatives and the lead for FasterCures benefit-risk program writes about the need for greater consistency and transparency in benefit-risk assessment is gaining a lot of attention and outlines five things you need to know.

Register for March 26 Webinar on crowdsourcing DOs & DONTs from experts who've posed CHALLENGES and gotten RESULTS
Sage Webinar graphic The Dialogue on Reverse Engineering Assessment and Methods (DREAM) project is a challenge-based initiative designed to foster the development of predictive models that allow scientists to better understand human disease. In 2013, after seven years and 27 challenges, DREAM merged with Sage Bionetworks to launch DREAM8.5, a series of three computational challenges that seek to crowd-source solutions to complex biomedical questions in cancer, rheumatoid arthritis, and Alzheimer's disease.

By fostering scientific collaboration through shared data, the DREAM8.5 challenges seek to advance the field of systems biology and provide a meaningful impact to both discovery and clinical research. In this Webinar, to be held March 26, from 1 to 2 p.m. Eastern, you will hear from Stephen Friend and John Wilbanks of Sage about the status of these challenges, the problems they are trying to solve, and how to get involved. This free Webinar is part of 
FasterCures' Webinar series designed to spotlight innovative approaches to disease research. Register today.

Want to know who is checking out our Webinars? The first two Webinars in our series this year have attracted 580 unique registrants from 13 countries, including 38 states and territories. They identify as belonging to the following sectors: Webinar reg sector2

Presentations focus on changing relationships among universities and venture philanthropies
AUTM logo

At the recent Association of University Technology Managers (AUTM) annual meeting in San Francisco, Margaret Anderson spoke on a panel about intellectual property negotiations between universities and foundation funders of research, and Kristin Schneeman, program director, participated in a discussion about how universities can identify and most effectively work with venture philanthropies. AUTM's more than 3,200 members represent managers of intellectual property from more than 300 universities, research institutions, and teaching hospitals as well as numerous businesses and government organizations.

Tweet about it! Copy + Paste from your @handle
• Is #crowdsourcing the answer 2 our biggest scientific challenges? Hear from experts @Sagebio on 3/26 @FasterCures

• "My PATIENTS motivate me." Check out @DrLauraEsserman's Q&A on bringing #breastcancer therapies to market faster

• "Addressing this great challenge does indeed keep me highly motivated." @IMI_JU @MichelGoldman @fastercures

• Join @FasterCures on 3/25 for tweet chat at 1PM with @wilbanks @maxlittle @grapealope about harnessing consumer tech 2 fight disease #fastercures

Introducing venture philanthropy to family offices
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Family offices have shown a growing interest in how they can help to fill the early-stage financing gap in medical R&D. Two recent FasterCures presentations aimed to introduce this community to patient-driven venture philanthropy models. On Feb. 19, Melissa Stevens, deputy executive director of FasterCures, and Rich Ditizio, executive director of business and program development at the Milken Institute, led a discussion on venture philanthropy with six prominent family offices in Houston who are interested in establishing a local fund to finance promising research from Houston research institutes. And, on Feb. 20, Stevens moderated a session on "Innovative Development Models: Family Office, Patient Advocacy Groups, and Venture Philanthropy" at the Texas Life Science Forum.

Learn more about FasterCures Programs | Upcoming and past speaking engagements
In the News
Science philanthropy pushes the frontiers of discovery

Philanthropists including Michael Milken, Bill and Melinda Gates, Debra Black, Ronald Perelman, Eli Broad and Paul Allen increasingly step in to fund cutting-edge scientific research where government funding falls short. Philanthropic investment in science has an outsized, transformative impact -- in biomedical research, we're seeing philanthropy funding translational science and accelerating the process of turning basic discoveries into treatments. Experts warn that philanthropy is a complement to, not a substitute for, government funding. Read the New York Times article.

Article highlights sense of urgency that drives patients

A Philadelphia Inquirer article features Emily's Entourage, a foundation founded three years ago by 29-year-old Emily Kramer-Golinkoff, which has raised about $630,000 for cystic fibrosis research through an annual event and an aggressive social media campaign. Kramer-Golinkoff has a rare form of cystic fibrosis and is seeking a cure through the foundation's efforts. Similar family-run foundations have sprung up over the last five years, as patients and their families work to personalize and accelerate research, said Cecilia Arradaza, FasterCures' managing director for communications and policy.

Blog Briefs
BRAIN Initiative: meet Human Genome Project

FasterCures Senior Fellow Robert Cook Deegan outlines six reasons that differentiate the BRAIN initiative from the Human Genome Project. Among the key differentiators are flat funding for biomedical research, the profusion of diverse private firms engaged in bioinformatics, imaging, management of Big Data and instruments of many kinds and degrees of complexity, and an atomized media world that seems far less attentive to science. In this piece, Cook Deegan presents a strong case for galvanizing support for the BRAIN initiative amid a tough fiscal, political and commercial climate. Read the blog.

R&D productivity: Analysis of recent reports on drug and medical development

How are we doing in developing new therapeutic interventions? A few years ago, Bernard Munos noted in Nature that the number of truly innovative new medicines approved by the FDA has declined substantially despite continued increases in R&D spending, raising the current cost of each new molecular entity to approximately $1.8 billion. In an effort to track where the field stands and where it is going, Timothy Swope, FasterCures research policy analyst, in a new blog post, looks at three reports that examine aspects of the drug and medical device regulatory landscape in an effort to identify trends and understand their impact.

Tools You Can Use

The R&D pipeline is currently a lengthy and iterative process of winnowing thousands of potential treatments down to a small number of compounds that prove safe and effective in treating disease. Discovery is largely funded by public sources, such as the National Insitutes of Health (NIH) and the Department of Defense; clinical research is largely funded by industry. The image below contains data from the NIH and appears in the FasterCures report "Measuring and Improving Impact: A Toolkit for Nonprofit Funders of Medical Research."

R&D pipeline graphic
Until next issue

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