Fulfilling the promise of bioscience

Bioscience is facing a Sputnik moment, according to Michael Milken, founder of FasterCures, as he opened the first session of the Partnering for Cures conference. The 1957 launch of Sputnik spurred the United States to focus on science and technology. Today, dramatic advances in genomics and disease research have brought the scientific community to the threshold of new discoveries, and he challenged the panelists in the Partnering for Cures opening session, “The (Near) Future of Bioscience and Health,” to explore what needs to change in bioscience in order to fulfill the promise.

Increased collaboration among stakeholders who don’t normally work together was seen by all to be a crucial factor in the future success of biomedical science. The U.S. Food and Drug Administration’s (FDA) Vicki Seyfert-Margolis said her agency is not just involved in regulation; rather, its role is to promote and drive innovation. She envisions a triangle composed of basic research, product development, and the FDA. “Information should move seamlessly between them,” she said.

Pfizer has been pursuing new collaborations as well, reported Mikael Dolsten, president of its worldwide research and development. “There has been a climate change in relation to regulatory agencies and academic partners,” he said. “I have been enthusiastic to see how many academic medical institutions were willing to take a new look at how to work together, work like one team to address different ways to treat disease,” he said.

Pfizer collaborated with the Cystic Fibrosis Foundation, Dolsten said, to define together a research plan to identify new molecules that will translate into successful compounds for patients with the disease. His company also has partnered with the Lupus Foundation in a similar way. If companies like Pfizer build on this, and others in the bioscience ecosystem see their success, he said, it will bring new interest into these partnerships.

New stakeholders and new voices must also be brought into the process, the panelists agreed. Risa Lavizzo-Mourey with the Robert Wood Johnson Foundation pointed to research conducted at the University of Miami into whether babies in neonatal units feel pain. Neuroscientists partnered with engineers to identify physical manifestations of pain. “The team brings the right kind of innovation to the table,” she said. “We don’t recognize all of the perspectives that we need.” Along those lines, she also identified the nurse scientist as a stakeholder who can help drive innovation, since these individuals are often closer to the patients and more familiar with their needs.

Funding is a problem at all levels. Milken noted that the FDA continues to be underfunded, even as the rest of the world falls back on the FDA for rulings on new drugs and devices when they run into problems in clinical trials. And with venture capital investments into the “valley of death” decreasing, patient collaboration becomes that much more important.

For Jay Schnitzer of the Defense Advanced Research Projects Agency, embracing risk is another key to success in bioscience. He said the field today is too risk averse. “We have to be willing to fail, to fail often, to know we fail and move on,” he said.

The decoding of the human genome is part of a “sea change” in biosciences, said Kenneth Davis of the Mt. Sinai Medical Center. Now, when some patients respond to a treatment, health records can be superimposed with genomic data to identify responders and find others with the same genomic makeup who can be used to test whether a new treatment will be effective. This precision medicine approach is being used with cancer research now, he said, and if more people contribute to biobanks and get behind genomics, he hopes more can be done with brain diseases.

A basic problem in healthcare today, Milken said, is that we spend more resources treating diseases than preventing them. Davis noted that Mt. Sinai is a fee-for-service medical system where payment is not keyed to prevention. But that is changing, he added: bundled payments, accountable care organizations, and other innovations are moving the U. S. healthcare system away from this fee-for-service approach.

In his conclusion, Milken cited notable medical advances. He noted that science averted a gloomy prediction of how widespread polio would be, and even in just a few decades, mobilized efforts have resulted in great strides in AIDS research and treatment. While the challenges are great in the future of bioscience and healthcare, Milken said that he has “a tremendous sense of optimism about the future.”

Are you ready? Partnering for Cures is here! Have you: • Updated your P4C Connect Profile? • Scheduled your partnering meetings with other attendees? • Set up sessions with leading experts from across the medical research field available for free, private one-on-one meetings? • Decided which presentations to attend & built your customized P4C schedule in your calendar of choice? Remember to download the Partnering for Cures partnering system P4C Connect app today! The partnering system also allows you to request free, private, 25-minute advice sessions on: Strategic Planning  •  Attracting Capital  •  Collaboration Structuring Interacting with the FDA  •  Communications& Marketing P4C Connect is accessible via your iPhone, iPad, or Android Device. You can download it from the iTunes App Store, Google Play Store, or scan the QR codes here. Android iPhone Remember to keep up with the conversation on twitter before and during the event with the hashtag #P4C2012

A Great Opportunity for Medical Research Philanthropy: Fund Team Science

by LaTese Briggs, Philanthropy Advisory Service Program Analyst, FasterCures

As seen in Philanthropy News Digest:

Today we are plagued by a variety of vexing and complex diseases. Many of us are affected, directly or indirectly, by cancer, HIV/AIDS, diabetes, and heart disease. While there is indeed an army of researchers working persistently to find cures and lifesaving treatments to these and other diseases, the rate at which they are making discoveries is impeded by limited resources and a reward system that discourages collaboration.

While there is a common misconception that most biomedical discoveries that lead to new treatments originate in the laboratories of major pharmaceutical companies, many groundbreaking discoveries are actually made in the labs of academic researchers. These labs are usually severely underfunded, and the intrinsic reward system and career growth opportunities for researchers almost exclusively hinges on the amount of grant funding they are able to secure. In a constrained and highly competitive funding environment, this often indirectly discourages collaboration among researchers, thus slowing progress on finding answers to complex questions in medical research.

Unfortunately, the development of new technologies such as genome sequencing has led the academic research community to realize that many diseases are more complex than originally thought. To tackle the biological complexity of these diseases, researchers across different disciplines need to work together. But to enable this type of "team science," we need to reconsider how the structure of philanthropic research funding affects the research itself and acknowledge that in order to improve the current research paradigm, we need to change the way academic research is funded.

Recognizing this, a new model for medical research philanthropy with an emphasis on scientific collaboration is being brought to fruition at institutions such as the Howard Hughes Medical Institute, the Scripps Research Institute, and the Broad Institute of Harvard and MIT. Each of these institutes initially was funded by seed money from private donors, and each has gone on to become a dramatically successful nonprofit research organization at the forefront of biomedical science. And because they unite multiple academic institutions and bring together experts from various disciplines to work on high-risk, high-impact projects in the absence of traditional funding barriers, they promote collaboration by their very design. The results speak for themselves. Some of their contributions to biomedicine to date include identifying new genetic risk factors for diseases such as schizophrenia, bipolar disorder, and autism; the classification of human cancers by their genomic alterations rather than by their location in the body; and the identification of key genes that regulate stem-cell development. These initial successes have attracted more traditional capital (including public-sector investments) to the field and promoted additional giving by other philanthropic organizations, further expanding the pool of capital available for high-impact research.

But you don't need tens of millions to found a new institute to make an impact in the team science arena. Donors and foundations can choose instead to fund interdisciplinary teams or multi-center collaborations focused on biomedical discoveries with the potential to lead to new treatment options and cures. Philanthropic gifts can also be directed to organizations like the Melanoma Research Alliance, the Michael J. Fox Foundation, and the Burroughs Wellcome Fund that already fund team science.

Philanthropic opportunities in medical research — funding team science among them — will be a hot topic at our annual Partnering for Cures meeting, November 28-30, in New York City. At this year's meeting, more than seven hundred venture capitalists, philanthropists, policy makers, biotechnology and pharmaceutical company executives, patient advocates, and medical researchers will gather for a series of engaging panels and one-on-one meetings, with the ultimate goal of advancing the field of biomedical research and finding cures to a range of diseases. Partnering for Cures is uniquely positioned to introduce new and emerging foundations and philanthropists to the fundamentals of medical philanthropy and to provide more seasoned givers with a range of due-diligence opportunities. We hope you'll join us in further exploring how we can all work together to facilitate the flow of philanthropic capital into medical research in the most impactful ways possible.

About FasterCures and the Philanthropy Advisory Service

The FasterCures Philanthropy Advisory Service (PAS) was specifically created to help philanthropists make informed investment decisions on giving to biomedical research. Since its inception, PAS has successfully channeled funding to a number of high-impact collaborative biomedical research projects and has created a pair of giving guides: Getting Started: The Medical Research and Development Primer and Giving Smarter: Building a High-Impact Medical Philanthropy Portfolio.

About LaTese Briggs

LaTese Briggs is the Philanthropy Advisory Service (PAS) program analyst at FasterCures. Briggs previously served as a pharmaceutical market analyst for Decision Resources, a Boston-based research and consulting firm serving the biopharmaceutical industry. In that capacity, she provided expert analytics on the state of research and clinical development, including research challenges, market drivers, and unmet patient needs in the infectious disease space. She is trained as a biochemist and completed her doctoral studies at the University of Maryland Baltimore County and her postdoctoral training at Harvard University/Broad Institute, where she focused on chemical biology and early drug discovery. In addition, she has received a number of honors, including being named a Gates Millennium Scholar, and has authored several scientific articles.

Releasing Unlocking IP: Principles for Responsible Negotiation

By Elizabeth West, Program Manager, FasterCures

Readers of the FasterCures blog are well aware of the importance of reducing cost, time, and inefficiency in the biomedical research system. One area consistently raised to us as having unnecessarily high transaction costs - both human and financial - is intellectual property (IP) negotiation.

We've long heard how IP can be a roadblock to innovation, but since it's a necessary and critical "cost" of doing business in drug development, we began to wonder if it might be possible to create an agreed-upon culture of responsibility in the practice of negotiating IP for medical research collaborations.

Last week we released Unlocking IP: Principles for Responsible Negotiation to serve as a set of guiding principles and points to consider when engaging in intellectual property negotiations involving biomedical R&D. The principles are a useful tool for all parties in biomedical research, in particular disease foundations, nonprofit disease groups, and philanthropists negotiating IP with academic, industrial, and nonprofit partners. The document includes principles for before, during, and after negotiations, for both new and seasoned organizations engaging in IP negotiations.

Some of the highlights include:

• Do not let fear paralyze you…If the desired result [of a deal] is based on research impact as well as monetary returns, not doing the deal or slowing progress is a far bigger loss to the patient and the public than lower potential economic return.
• Make sure you have the right people at the table at the right time…Think through which stakeholders are needed to  foster innovation, and only exclude a stakeholder group—especially nonprofits—after careful thought.
• Build in “use it or lose it” requirements (interruption licenses)….If you have invested in the IP and the owner is not exercising the rights to use the invention and make it widely available, or if they are using it in a way that impedes progress, ensure you have a way to take it to a party where its value can be maximized.

The principles emerged from a small, expert-led working group convened by FasterCures in July 2012. In addition to developing the principles, the group came to some broader conclusions regarding the intellectual property ecosystem. In particular, it found that there is a fundamentally new landscape in biomedical research resulting from the emergence of venture philanthropy and patient advocacy groups. These groups tend to want to enhance the freedom to operate, versus the limiting model more prevalent in the current environment.  A key conclusion of the working group is that an IP management system is needed that addresses this emerging ecosystem, not reflects the current one. We welcome all of your thoughts on the fundamentals of a forward-looking IP system.

The Webinar – directly from the experts

On Nov. 16, we continued the conversation on IP and the principles through the TRAIN (The Research Acceleration and Innovation Network) Webinar series. The Webinar (available to view here), moderated by Margaret Anderson, was led by an expert panel of individuals with in-depth experience in intellectual property negotiation from a variety of viewpoints.

Maria Freire, of the Foundation for the NIH, laid out the main findings of the working group and gave a detailed overview of the principles themselves, as well as spoke of the increased sense of urgency and interest of many groups in deals that “move towards the goal.” Robert Cook-Deegan of Duke University’s Institute for Genome Sciences and Policy provided the audience with the broader landscape of intellectual property negotiation successes and controversies, as well as analyzed the elements of success through an in-depth case study of Cystic Fibrosis Foundation and the CFTR gene patent. David Lubitz, of Schaner&Lubitz, PLLC, which provides council for many disease foundations, gave a thorough overview of the viewpoint of disease foundations and venture philanthropy groups, including funding goals, trends, and useful IP tools, including  interruption licenses. Many of the questions during the Q&A period focused on the replicability of the Cystic Fibrosis Foundation model, which the panelists agreed would require a foundation with financial resources, technical expertise, and potential commercial partners.

More on IP at Partnering for Cures

We will have more on this topic at our annual conference, Partnering for Cures, in New York City, Nov. 28-30. The IP-focused panel, License to drive (innovation): IP strategies to support, not slow, progress, will explore the evolving IP law and procedures, the IP implications of the movement toward more precompetitive collaboration in biology, examples of responsible IP management practices, and how IP decisions can impact follow-on innovation and patient access. We invite you to join us there!

 

Partnering for Cures is less than two weeks away

Partnering for Cures is less than 2 weeks away and the FasterCures team is buzzing with excitement.  Not only does this year’s meeting offer

• Our best, most engaging PROGRAM yet
• Over 85 fantastic SPEAKERS from across the medical research enterprise
• Numerous onsite PARTNERING opportunities to find and share ideas with like-minded people and organizations

But we are also proud to be featuring 30 PRESENTATIONS by some of the most innovative cross-sector medical research collaborations out there. You won’t want to miss them describe their efforts to commercialize early-stage discoveries, share data, develop novel research tools, repurpose assets, and create innovative financial models to drive progress.

For example, did you know . . . 

• The Clinical Trials Transformation Initiative co-founded by the U.S. Food and Drug Administration (FDA) and Duke University, is identifying practices that through broad adoption will increase the quality and efficiency of clinical trials.
• Lawrence Livermore National Laboratory and IBM Research are working together on a simulation of the whole human heart using the world’s highest performance supercomputer, a project which stands to significantly accelerate new discoveries in cardiovascular therapy
• New York Genome Center is developing the infrastructure and practices to share data and repurpose research assets across eleven major academic medical and research centers with the aim of speeding the translation and commercialization of early-stage discoveries through large-scale genomics analyses
• Code-N, a silicon valley life sciences “Big Data” start-up, is working to empower scientists to analyze data simultaneously “in the cloud” to make compound-gene-protein-disease connections that will open the door to new scientific discovery
• Virtual biotech Phoenix Nest, created to find treatments for the ultra-rare pediatric disease Sanfilippo Syndrome, was built through collaboration between leading academics, experienced researchers and nonprofit disease foundations who met at last year’s Partnering for Cures

To learn more about these and other collaborations featured at the meeting, click here.

We’ll see you in a few weeks!

Keep up with Partnering for Cures conference updates by following us at:

     @FasterCures
    @FC_P4C 
    #P4C2012

Need Cures? Who Ya Gonna Call?

By Margaret Anderson, Executive Director, FasterCures
As seen on HuffingtonPost 

Do you know anyone whose life hasn't been touched by disease? As I get older I become more aware that the frenetic life I lead and call normal could be interrupted in the blink of an eye.

We all know the drill. People in our lives are just going about their business when something goes amiss -- a twitch in their leg, some dizziness, a lump, a pain, an abnormal result, an accident, an ambulance, a hospitalization, a doctor's visit, a test, a diagnosis. A new reality. A replacement of what once was to a new normal.

I can conjure up times in my life that disease has interrupted life just like that. Here are just a few that were easy to recall; I am sure you have just as many.

• "I didn't see this coming -- I thought it was my heart medication." -- My father after his leukemia diagnosis.


• "This week, I found a lump in my breast." -- My friend when she put her hand on my arm after I asked what's new.


• "My dad is at the end of life after battling Alzheimer's." -- My colleague after her dad began his final days.


• "My doctor had told me I would be dead in three years." -- My friend recounting how in the earlier days of the AIDS epidemic there was no prospect of survival.


• "I also realize that while I have what may be the nastiest cancer out there, I have it easy compared to others." -- My friend describing his fight against pancreatic cancer.

That moment when you or someone you love become a patient, the patient. And then what?

Well, hopefully the wonders of science and medical research will have led to good therapeutic options for you. At our recent Celebration of Science event, we witnessed scientific discovery literally unfold on the stage of the National Institutes of Health as scientists and patients told breathtaking stories of research triumph. Here's a glimpse of how scientific discoveries have improved health and saved lives. It's remarkable. But, consider that of the 4,000 diseases for which we have the exact molecular basis, only 250 have treatments available.

So, while we need to celebrate the success stories in medical research that allow us to carry on our lives -- be those successes through prevention, diagnostics, devices, or medical intervention -- we have more work to do. We must ensure that we continue to have a robust flow of scientific discoveries that we can then translate into better health.

The well-being of too many of our friends and family depends on this.

The pathway to treatments and cures is littered with failure, lack of funding, scientific and regulatory challenges, reimbursement issues, health care delivery issues, and if that is not enough there are immediate challenges like the impending fiscal cliff and sequestration. Decisions that our reelected President Barack Obama and the U.S. Congress will make could substantially impact the future of treatments and cures.

I could cite data to show how medical research matters.

• It saves lives.
• It creates jobs.
• It maintains us leadership in the global economy.
• People's lives depend on it.

We face a major paradox -- that the potential of science is greater than ever but the outlook for funding has never been bleaker. If an agreement on how to prevent sequestration doesn't happen, here's what we'd see:

• U.S. government science funding programs would see their budgets cut by 8.2 percent in 2013.
• $2.52 billion decline in funding at the NIH.
• 2,300 fewer NIH grants -- representing almost a quarter of new and competing grants.
• Total employment supported by NIH awards would fall by 33,704.
• The Food and Drug Administration (FDA) will lose $318 million from its budget, which wasn't adequate to begin with.
• Virtually all user fees paid to the agency to ensure swift application reviews would be considered part of the sequester.
• About 80 percent of the agency's budget is devoted to personnel, so cuts would mean reduced staffing levels or imposing furlough days

So, given that the need is great, the promise is there, and the arguments in support of this funding are strong, now what?

1. Get educated. Go to our website called Sequestration Station to get smart on the issues and see how you can get involved.
2. Tell us your story. Be a part of our new social media campaign called Time Equals Lives. We are collecting personal stories -- from patients and scientists, researchers and caregivers, industry executives and academics -- to make a compelling case about the critical importance of medical research. We'll share these stories with leaders and decision makers to remind them of what is at stake.

Because when you or a loved one needed a cure, we want to make sure you knew you'd have someone to call on.

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For more by Margaret Anderson, click here.
Follow FasterCures on Twitter: www.twitter.com/fastercures

Novel Funding for Bioscience Companies

By Karlee Stewert, Communications Coordinator at FasterCures 



FasterCures Executive Director Margaret Anderson joined the Mid-Atlantic Bio Conference this fall for a discussion on novel funding sources for bioscience companies, a major concern plaguing the biotech and medical research industries. The panel discussed best practices to attract funding, how to develop important relationships with possible funders, and the opportunities crowdsourcing can bring to a small organization. “The biggest concern plaguing bioscience industry is funding,” moderator Peter Ginsberg, North Carolina Biotechnology Center’s VP for Business and Technology Development, pointed out, “the way a company approaches funding is the key to bringing in resources.”

“Building relationships is an obvious but important move,” Anderson recommended. “Start a conversation with a potential partner about something innovative you can do together.” Fellow panelist, John Hollway, president of Pragmatos Consulting, emphasized Anderson's point and warned that "the time to build a relationship with the federal government isn't when you’re applying, it's before." 

Crowdfunding, rallying support from others who can pool their resources rather than seeking out a major donor, has quickly become one of the most popular methods for small businesses to fund their endeavors. This approach to funding has permeated the biotech industry with organizations like Cloud-Based Quality Assurance for Cancer Treatment and myHealthPal, which use the vast opportunities the Internet provides to build their vision, raising funds and gaining access to their target audience.

Research organizations seeking funding from the National Institutes of Health (NIH) for their projects may unfortunately  be faced with the reality of sequestration of critical capital to create life-saving products and treatments. If sequestration goes into effect in January 2013, NIH will face a drastic funding cut. Anderson stressed the importance of scientists and biotech speaking out and making their stories heard by those in Congress: “I encourage you to understand what this means and make your voice heard. Talk to your members about what they can do and why sequestration matters. Speak to Congress about what it means to be a scientist, and tell them how much of an investment the federal government has made in you.”

To start to compensate for the uncertainty of federal funding, venture philanthropy groups will be looked on even more to step up and provide funding that the medical research community now relies heavily on.

Anderson recommended utilizing your members’ extended networks – both on and offline, ‘friends-of-friends’ connections —  building strong relationships with the funders can mean the difference between struggle and survival for your company.

And because this IS a FasterCures blog, we’d be remiss not to remind you of a great opportunity to find nontraditional allies who could be key to helping you advance your R&D goals – Partnering for Cures. This meeting, to be held November 28-30 in New York City is a unique opportunity for biotechnology companies to network and find true partners. This meeting brings together more than 800 leaders from all sectors of the medical research enterprise, across therapeutic areas. It is focused on solutions and making collaboration happen - and it works because the participants are there to get things done. A special rate is set for start-up and emerging biotech companies.

For more information on unique approaches to funding, check out these resources:

• FasterCures’ Fixes in Financing
• Crowdfunding: Maximizing the Promise and Minimizing the Peril
• crowdsourcing.org