Imagine the possibilities if we could only read your mind…or at least map your brain

"Imagine if no family had to feel helpless watching a loved one disappear behind the mask of Parkinson’s or struggle in the grip of epilepsy.  Imagine if we could reverse traumatic brain injury or PTSD for our veterans who are coming home…,” said President Barack Obama last week as he laid a grand, ambitious new government initiative to map the brain.

Obama said he will ask Congress for $100 million in 2014 to support the BRAIN (Brain Research through Advancing Innovative Neurotechnologies) initiative which allows us to "better understand how we think and how we learn and how we remember." Three government agencies will be involved: the National Institutes of Health, the Defense Advanced Research Projects Agency and the National Science Foundation.

“It's an audacious, bold idea,” said Francis  Collins, director of the NIH. "To understand how the human brain works is about the most audacious scientific project you can imagine," he said. "It's the most complicated structure in the known universe."

In many ways, this initiative is similar to the Human Genome Project which Collins led and completed in 2003, in its vision and ambition. But, unlike the Human Genome Project, the BRAIN initiative has not laid out its primary scientific goals. The lack of detail is worrying BRAIN skeptics and advocates alike. Some scientists were quick to question the motives behind this initiative, noting that a lot of great research on the brain is already underway.”

At a Q&A segment  following the President’s announcement, Collins said that these details would be hammered out by a “dream team” of 15 scientists who will hold their first meeting at the end of the month. This team is led by Cori Bargmann of Rockefeller University and William Newsome of Stanford University, will be charged with coming up with a plan, a time frame, specific goals and cost estimates for future budgets.

Additionally, Obama addressed how this initiative can drive growth and create new job opportunities. As the “rest of the world” is racing ahead in the quest for innovation, he expressed concern that we will lose a new generation of scientists because of uncertainty in research funding. We can’t afford to miss these opportunities, he said.

The President called on companies, research universities, foundations, and philanthropists to get involved.

What we took from sitting in the room with the leaders and innovators in science and research as the president spoke about the promise and potential of this new initiative, was a broader message about the power and value of investing in science, and the important role the federal government plays in ensuring scientific breakthroughs can improve health.  We took such enthusiasm and energy as our cue to be more determined than ever to advance medical progress.

Upcoming Milestones

• As part of this planning process, input will be sought broadly from the scientific community, patient advocates, and the general public.
• The working group will be asked to produce an interim report by fall 2013 that will contain specific recommendations on high priority  investments for Fiscal Year (FY) 2014.
• The final report will be delivered to the NIH Director in June 2014.

Key Resources:

• Remarks by the President on the BRAIN Initiative and American Innovation, April 2, 2013
• Video of Remarks by NIH Director Collins and the President, April 2, 2013
• Fact Sheet: BRAIN Initiative(Whitehouse.gov), April 2, 2013
• Video of Open for Questions with NIH Director Francis Collins(Whitehouse.gov), April 2, 2013 
• WhiteHouse.Gov Blog: BRAIN Initiative Challenges Researchers to Unlock Mysteries of Human Mind, April 2, 2013

The long-term effects of sequestration on medical research

“I worry about the treatments that we’re not going to discover as a result of these cuts. What does this mean downstream; what does it mean in terms of the unmet needs of patients who have diseases with no treatments or cures? The NIH investment is a very powerful driver to this entire enterprise.” 

- Margaret Anderson

Last week, FasterCures Executive Director Margaret Anderson appeared on the BioCentury broadcast with host Steve Usdin to examine the long-term ramifications on medical research and health outcomes if sequestration is not appealed. Watch the whole segment here. Anderson was joined on the Sunday, March 10 segment by Lynn Marquis, Director of the Coalition for the Life Sciences, who talked about the effects that these cuts will have on the scientific workforce.

“We’re going to lose an entire generation of scientists moving forward,” Marquis cautioned. “We’re already not seeing kids pursuing scientific careers because there is no future for them.”

Though optimistic about the possibility to reverse this alarming trajectory, Anderson and Marquis warned that medical science is an area in which the United States cannot afford to fall behind.  They emphasized the outsized impact that investments in research have on both human health and the health of our economy, and noted that it is hard to compare these types of cuts with others.

“This is a powerful investment in the future - for patients, for the economy, for jobs, for industry. I don’t think it’s the same as cutting other programs,” Anderson said. Marquis agreed and offered a bit of hopefulness, “I think we see from both Republican and Democrats…there is huge support for NIH’s biomedical research, primarily because of the human health and economic benefits that we reap from our investment.”

So if Congress understands that medical research is vital to the future of our country, what can be done to ensure we don’t lose our preeminence in the life sciences?

Anderson thinks there’s hope for the NIH to avoid budget-desolation. “I’m an optimist. I think that people need to speak their mind to their members of Congress about how they feel about this investment.” She also spoke about the power of team science and cross-sector collaboration in moving promising research from lab to patient faster.

Just this year, NIH will be forced to reduce its spending by $1.6 billion, significantly slashing the United States’ ability to fund basic scientific research.  Yes, that’s billion with a capital B. Devastating to the medical research community – and more importantly, to the hundreds of thousands of patients suffering from diseases for which there are no cures and few treatment options – does not begin to describe the impact of sequestration.

What Can Be Done?

• Contact your Members of Congress and let them know that further cuts to the NIH and FDA will set us back on the path to new therapies and cures for disease, jeopardize our economic competiveness, and result in job loss in communities throughout our country. Share with them why a strong investment in medical research is important to you, your family, and your community.
• Submit your story about why medical research and protecting funding for new research matters to Time=Lives.
• Attend the Rally for Medical Research on April 8th here in Washington, DC. Even if you can't attend, check out their site for more way to get involved!

FasterCures Finds Outcomes, Metrics, and Strategic Leadership Define the Effectiveness of Venture Philanthropy Groups

Two new reports outline the practices and approaches of nonprofit funders of medical research

FasterCures today released two reports that paint a vivid picture of how medical research foundations, also known as venture philanthropy groups, have transformed the medical research enterprise. These entrepreneurial groups are accelerating medical research and development by tackling science where it needs the most effort and resources, and applying innovative business approaches in the pursuit for a cure.

Both resources tell a compelling narrative of how venture philanthropy groups are creating a culture in medical research that is mission-driven, results-oriented, and focused on the true bottom line: preventing, diagnosing, and curing disease.

• The first, Honest Brokers for Cures: How Venture Philanthropy Groups are Changing Biomedical Research, features insights from leaders of 20 venture philanthropy groups. This publication features valuable insights and perspectives from leaders of medical research foundations that are transforming the cure enterprise by virtue of how they find and fund research. In this report, FasterCures dissects the business model that has emerged from the shared sense of urgency and frustration stemming from these patient-driven organizations, and their laser-sharp focus on outcomes.

• The second, Measuring and Improving Impact: A Toolkit for Nonprofit Funders of Medical Research, is a how-to guide for foundations seeking to apply some of the best practices and lessons learned from venture philanthropy groups who’ve demonstrated their effectiveness.  It provides a common framework for assessing and improving organizational effectiveness, and a panoply of ideas, questions, and models to help guide new and emerging nonprofits with strategic and tactical choices.

Venture philanthropy groups play an outsized role in improving the medical research and development system to better meet the needs of patients. Since its inception, FasterCures has continued to shine a light on the successes of these foundations in an effort to amplify their lessons learned and sense of urgency to the rest of the medical research community.

“In the end, the most central characteristic of these groups is their close connection to the disease they are pursuing. For most, it is personal—either they or a family member or close friend is or has been affected by the disease,” said Margaret Anderson, executive director of FasterCures. “It spurs them to find the dollars to meet the challenges and change the trajectory of research. We can all benefit from the lessons they've learned.”


                                                  

To download these publications, visit http://www.fastercures.org/Publications/vp.php

 

A perfect storm brewing over science

A dynamic panel of experts painted a vivid picture of the tsunami of budget and fiscal issues that threaten federal science programs during a FasterCures Webinar on “The 113th Congress and Medical Research: A Perfect Storm Approaching?” moderated by FasterCures Executive Director Margaret Anderson. While the prospects for avoiding widespread cuts seem bleak, the speakers remained optimistic that a loud, unified, focused argument from the medical research community could have an impact right now.

Sudip Parikh, vice president and general manager of Battelle Health & Analytics and a former senior staffer on the Senate Appropriations Committee, walked participants through how the budget process is supposed to work and how it’s currently working – or not working, as the case may be. An across-the-board spending cut or “sequester” is set to take place on March 1, the government is operating on a continuing resolution set to expire at the end of March, the government could shut down if that is not resolved, and the national debt ceiling looms once again in mid-May. The budget process is about setting priorities, Parikh said, and the current breakdown is not only in the budget process but also in priority-setting. Priorities within the Labor-HHS appropriation, for example, which includes the National Institutes of Health (NIH), are competing head-to-head this year – medical research, Title I education spending, Pell grants – and they can’t all win. He reminded listeners that this is a long-term discussion and that spending levels set now will set the stage for years to come.

Carrie Wolinetz, associate vice president for federal relations at the Association of American Universities, as well as president of United for Medical Research, took participants on a tour of the members of Congress involved in decisionmaking about all these budget matters, including the leadership of appropriations subcommittees with responsibility for NIH’s and the Food and Drug Administration’s (FDA) budgets as well as the Senate and House leadership responsible for overall budget negotiations. While decisions will be made at the end of the day by those top negotiators, they do still rely on input from the committees. Many of these members can be considered strong supporters of biomedical research, but in this year what has been historically a bipartisan priority is being consumed by the macro-level fiscal arguments. Still, Wolinetz argued that it is critical to continue to cultivate champions, including new ones such as Representatives Kevin Yoder, Ed Markey, Eric Cantor, and Steve Stivers, to replace old friends like Arlen Specter, John Porter, and Tom Harkin who are gone or will soon retire.

Ceci Connolly, managing director of PricewaterhouseCoopers’ (PwC) Health Research Institute and former Washington Post journalist, gave participants a flavor of the political environment in which budget negotiations are taking place this year. In addition to the unusual confluence of the budget, the sequester, and the debt ceiling, this is the beginning of President Obama’s second term, and Connolly noted that second terms can be a “double-edged sword.” On the one hand, they can liberate an Administration to take risks; on the other hand, they can cause the Administration to over-reach. And presidents don’t really have four years in which to accomplish their goals; they really have 12-18 months before attention turns to the next election. The Obama Administration, she said, is trying to get past the fiscal issues to what they consider their potential legacy issues of immigration reform and gun control. The advice she extended to medical research advocates was to focus on the compelling argument that medical research creates jobs and leverages significant economic activity, noting that in a recent PwC survey, 69 percent of Americans agreed that biomedical research is important to economic growth. She also recommended advocates expand the community, and build new alliances and relationships with others who benefit from research investments, including industry.

Parikh urged advocates to come up with an “ask” that’s clear and common across all stakeholders. “This isn’t just about the next three months; we need to set up for the next two years. The Fiscal Year 14 process is underway.”

FasterCures’ Sequestration Station and United for Medical Research’s Web site provide information and resources about sequestration and its impacts on research and the economy.

View an archive of this Webinar here.

The World According to BIO CEOs

By Kristin Schneeman. Program Director, FasterCures

We look forward every year to attending the BIO CEO & Investor Conference in New York in February, because it gives us a window into what the issues of interest and concern to the biotech community are, as we’re planning our own activities for the year.

Here are a few nuggets picked up at this year’s conference:

• Reimbursement continues to be a key and thorny issue – and not everyone in the industry is dealing with it.  Companies and deal-makers noted that even in early-stage programs, commercial considerations are “at the table,” and that today you need to have not only a biological and clinical hypothesis in early research but a commercial hypothesis as well.  However, almost immediately after a fascinating panel discussion about “Reimbursement in an ACA World,” which catalogued some of the fundamental ways in which the world of not just healthcare but research is changing, a panel of very accomplished investors was asked how reimbursement factored into their world view right now – and they all fell back on the conventional wisdom that “truly innovative products will always get paid for.” One investor in a later panel mentioned that “the Street is ‘catching on’ to reimbursement” – just catching on?


• Everyone seems to love the FDA this year.  Investors described the regulatory environment as “favorable,” friendlier,” and companies praised the new accelerated approval and breakthrough designation initiatives – while noting that they remain something of a “black box.”  


• Rare diseases are hot, with companies and investors.  They see that these products can command high prices.  Personalized medicine is causing common diseases to be redefined as subtypes that in many cases could be considered “rare,” and clearly companies were embracing this line of thinking.  Hepatitis C, on everyone’s lips last year, was almost nowhere to be found – instead we heard about multiple myeloma, lysosomal storage disorders, and “genetically defined cancers.”  This is great news for patients with rare diseases, but how long will the fad last?  What happens to patients then?  And, as one speaker noted, “just having orphan designation isn’t a get-out-of-jail-free card on pricing anymore.”


• China is attracting not just big pharma companies but smaller biotechs as well – and not only to take advantage of cheaper gene sequencing or clinical research capacity, but to manufacture and sell products as well as to raise capital.  While doing business in China is not an easy row to hoe, you could hear the excitement of the panelists talking about this new frontier for U.S. companies.  Many U.S.-educated Chinese scientists and businesspeople are returning home to make their careers.  Will development of the academic infrastructure to fuel homegrown science and companies be far behind? 


• Sadly, there was almost no discussion about the impact of impending budget cuts on NIH, which fuels much of the science these companies thrive on, or FDA, which must have the capacity to regulate and approve their products. Their own user fees, which go directly to FDA review of their products, are threatened with sequester – where is the outcry?  Besides a promising few talking about the value of these agencies, the policy agenda seemed largely focused on repealing IPAB, the Independent Payment Advisory Board, from the Affordable Care Act.  

As always, an informative and thought-provoking two days, interspersed with fascinating glimpses of the products coming down the pipelines of dozens of companies.

Relevant postings:
--- Medical research delivers cures, saves lives and – oh, by the way – is pretty great for our economy too
--- Cutting-Edge Science, Collaboration, and Sustained Funding Needed to Get New Medicines from Lab to Patient
--- The Next Big Thing In Biotech: BIO CEO Conference
--- Are Drugs for Ultra-Rare Diseases the Future of Biotech? Francois Nader of NPS Weighs In

Medical research delivers cures, saves lives and – oh, by the way – is pretty great for our economy too

Over the past few weeks, Washington D.C. has been abuzz with conversation about the value of medical research to our national economy, collective health, and global competitiveness. Leading up to our own Capitol Hill briefing with Friends of Cancer Research last Wednesday – “The Blueprint of Medical Research: How New Medicines Get from the Lab to the Patient” – FasterCures has attended several events focused on the impact of research and research funding on our nation’s well-being.

We’ve heard unequivocally that scientific opportunity – particularly when it comes to our understanding of disease biology – has never been greater, but the outlook for funding has also never been more worrisome.  If we don’t increase our investment in medical research, we’re effectively cutting it, risking not only a major engine that fuels our economy, but also the lives of hundreds of thousands of people living with diseases that have few or no meaningful treatment options.

The budgetary decisions we make today are decisions that won’t just have an impact in our lifetimes, but will alter the course of the next generation of patients, family members, and young scientists.  Here are some of the things we’ve heard around town: 

• “If scientists can take a few minutes to put down their microscopes and pick up a microphone it will make a real difference.  As we stand at this budget crossroads, we need your help to make the case.”
• Congressman Ed Markey (D-MA) at American Cancer Society’s Cancer Action Network briefing on Capitol Hill, February 5, 2013

• “The FDA is really a bargain when you look at scope of activities that have exploded without parallel budget increases. FDA costs only about $8 per American per year, and we are determined to maximize the dollars that we have. Drugs are available in the U.S. faster than anywhere else in the world, and with 39 new approvals in 2012, including a number of new advances in personalized medicine, we are making real progress. Sequestration would result in a significant reduction of domestic and international inspections, which makes for a tough environment in which to recruit and retain top people. We cannot walk away from our responsibilities."
• U.S. Food and Drug Administration (FDA) Commissioner Margaret Hamburg at Alliance for a Stronger FDA meeting, February 5, 2013

• The average per capita National Institutes of Health investment is just under $100/citizen/year, and less than 1% of federal budget. Yet these investments are having an outsized impact on our economy and health. The more stories that scientists and patients can tell in their own words, the more likely it is that the message will resonate with our policymakers. 
• National Institute of Health’s Scientific Management and Review Board (SMRB) discussion from January 14, 2013 board meeting

• “We have come so far in cancer science, especially in my field of immunotherapy. We now live in an age where it is possible to take the army in own bodies, and train it to fight against our particular disease. But scientists like me would not be where we are if not for the investments that were made in us at the start of our careers.  If the budget of the NIH is cut any further we will be faced with having to tell the patients who rely on our science: I’m sorry, there’s nothing more we can do.  No one wants to be the giver or receiver of this heartbreaking message.”
• Helen Sabzevari, global head of oncology-immunotherapy for EMD Serono Inc. at American Cancer Society’s Cancer Action Network briefing on Capitol Hill, February 5, 2013

• "The National Institutes of Health (NIH) supported more than 402,000 jobs and $57.8 billion in economic output nationwide in 2012 alone. If Congress fails to prevent the 5.1 percent automatic, across-the-board spending cuts slated for March 1, the nation's life sciences sector could lose 20,500 jobs and $3 billion in economic input."
• Feb. 6 statement by United for Medical Research 

Currently, only five cents of every U.S. health dollar goes to biomedical research; however, 1 in every 3 Americans is living with a deadly or debilitating disease for which there are no cures. The U.S. needs strong investment in biomedical research to deliver cures, save lives, and maintain our global competitiveness. There’s no time to waste.

Relevant links:
-- ACS CAN Report Highlights Critical Need for Sustained Funding for Cancer Research
-- Videocast of Jan. 14 SMRB meeting
-- The State of the FDA—February 2013
-- Report from United for Medical Research about the Economic Impact of Sequester on Life Sciences Sector
-- Sequestration Station
-- Time=Lives

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

Sequestration will be devastating to the cure enterprise

By Margaret Anderson
Executive Director, FasterCures

For months, the defense industry has taken the Hill and media outlets by storm detailing the potentially catastrophic impact “sequestration” will have on national security, defense jobs, and our economy. The “S” word stands for automatic spending cuts that will take into effect if Congress does not reach a budget agreement to reduce the deficit by the end of the year.

Members of Congress have expressed concern over the potential cuts to defense programs, and even called for carve outs to protect the Pentagon funding. There has been little mention, however, of the impact sequestration would have on nondefense programs, such as medical research at the National Institutes of Health (NIH), or education, which are also subject to the same across-the-board cuts imposed by the Budget Control Act of 2011.

Last week, we learned a little more about the impact of the sequester’s $1.2 trillion cuts on nondefense programs because of a report released by Senator Tom Harkin (D-IA). The report details the “destructive impacts on the whole array of Federal activities that promote and protect the middle class in this country – everything from education to job training, medical research, child care, worker safety, food safety, national parks, border security and safe air travel." The report even indicates that the “economic effects of cuts to nondefense programs could be worse than cuts to Pentagon spending.”

There is chilling evidence of what we can expect to happen to medical research at NIH should sequestration take place:

• NIH would issue about 700 fewer grants to medical researchers in Fiscal Year 2013, which means 700 fewer opportunities to find medical treatments and cures;
  
• The total cut to NIH would be $2.4 billion;
  
• The National Cancer Institute alone would be cut by $396 million;
  
• The National Center for Advancing Translational Sciences would lose nearly $45 million; and
  
• All 50 states would be expected to suffer from an across-the-board cut.
  

The report also illustrates how cuts to NIH will result in not only job loss (NIH currently supports approximately 432,000 nationwide), but also a reduction in medical innovation.

At FasterCures, we are deeply concerned by these numbers and urge members of Congress to identify a solution to sequestration before we see a decline in medical research. The ripple effects will be felt far and wide – in every university, every medical research setting, and every household that’s been struck by disease.

We know today that investments in scientific research, particularly in bioscience, not only save and improve lives, but also offer the greatest potential returns and serve as the best economic stimulus any nation can make. Nations that lead the world in bioscience will be the global leaders of the 21st century. No field has greater potential to improve health, create jobs, and stimulate the economy; and no field provides greater returns on investment. 

Important references:

• Research!America’s Sequestration Report: http://www.researchamerica.org/uploads/0725REAPpresentation.pdf
  
• Leadership in Decline: Assessing U.S. International Competitiveness in Biomedical Research: http://www.unitedformedicalresearch.com/wp-content/uploads/2012/05/Leadership-in-Decline-Assessing-US-International-Competitiveness-in-Biomedical-Research.pdf
  
• The Global Biomedical Industry: Preserving U.S. Leadership: http://www.milkeninstitute.org/publications/publications.taf?function=detail&ID=38801285&cat
  
• FASEB’s NIH State Information Factsheets: http://www.faseb.org/Policy-and-Government-Affairs/Advocacy-on-Capitol-Hill/Explore-Resources/NIH-State-Information-Factsheets.aspx