Time=Lives Story of the Week: Jessica Foley

Encouraging the next generation of scientists

“What motivates me is working in a field where every day it's changing, it’s challenging, and it’s exciting.”

Meet Jessica Foley. She’s the Scientific Director at the Focused Ultrasound Foundation (FUSF) and a long-time advocate for greater use of the focused ultrasound, a revolutionary technology that allows for the treatment of numerous diseases without the danger of invasive surgery or a lengthy recovery time.

Watch Jessica’s story here.

Currently, MR-guided focused ultrasound surgery has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of uterine fibroids, and has obtained CE-mark approval in Europe for uterine fibroids and pain from bone metastases.  However, the possibilities for this technology are countless - with ongoing clinical trials for breast tumors and brain tumors, and plans to begin additional research into it's impact on several other kinds of tumors, stroke, and epilepsy.

For over 12 years, Jessica has had a rich career as a scientist and researcher in this novel field. Before joining FUSF, she worked with InSightec and Medtronic, and also served as a 2011-2012 AAAS Science and Technology Policy Fellow at the National Science Foundation.

We met up with Jessica at last year’s Celebration of Science to talk about what she envisions for future generations of scientists and researchers and the challenges they may face with today’s economic shortcomings. She emphasized the need to elevate the role of science in our society so we can attract and engage tomorrow's innovators.

"Years ago [the public] seemed to get it," said Jessica, who holds a Bachelor’s in Biomedical Engineering from Duke University and a PhD in Bioengineering (emphasis in focused ultrasound) from the University of Washington.  "And maybe not everyone gets it now, but I hope that through all sorts of projects we're trying to work on we can get to that point again where kids want to grow up and be scientists and engineers."

To learn more about the Focused Ultrasound Foundation visit http://www.fusfoundation.org.

And make sure to check out the TIME=LIVES campaign for more stories about the power and promise of medical research, and to help spread the message that research matters and needs to be a national priority.

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.

Time=Lives Story of the Week: Derrick and Meredith Day

“I think I’m the same, except I just can’t see.” 

– Derrick Day, 6 years old

Derrick (6) and Meredith’s (4) story came to Time=Lives via the Foundation Fighting Blindness, an organization dedicated to driving research to prevent, treat and cure people affected by retinal degenerative diseases.

View Derrick & Meredith’s story here.

Born legally blind, the siblings suffer from a rare inherited eye disease called Leber’s Congenital Amaurosis, a disorder that can also create central nervous system abnormalities.

Seemingly simple tasks for a sight-abled person become difficult hurdles or even impossibilities when disease has robbed someone of their sight. Derrick’s dad dreams of a day when his son will be able to drive a car. “I never thought about Derrick’s not being able to drive,” said his mom. “When we got to the stop sign, he presented the question to me: Mom, how are we going to make that sign braille so that I can drive?”

Whether it’s as simple as experiencing a rainbow, or as complex as driving a car, the only way for kids like Derrick and Meredith to be able to achieve these seemingly insurmountable tasks is to be able to see. “And the only way that we can have them see,” says their mom, “is with research, and by funding scientists to find a cure.”

The good news is that retinal disease science is advancing. For example, just yesterday the Foundation Fighting Blindness reported that the Argus II retinal prosthesis, a device that can restore some vision to people who are blind from advanced retinitis pigmentosa (RP), received U.S. market approval from the Food and Drug Administration (FDA). More than 20 years of research went into the development of Argus II, with early support from the Foundation.

Also, earlier this month The Washington University School of Medicine created an innovative method for treating vision-robbing diseases using genetic reprogramming. And while their research is at an early stage in mice, it has revealed valuable clues about how to potentially save vision in people.

But promising research like Washington University is conducting wouldn’t be possible without federal funding.  And with sequestration – mandatory, across-the-board budget cuts – looming, the researchers who study diseases like Leber’s Congenital Amaurosis and others could face debilitating cuts to their research programs that would, at best, slow innovation and progress, and at worst, cause entire labs to shut down.

Want to know more about sequestration? Visit FasterCures’ Sequestration Station for facts, forecasts, and ways to add your voice to the fight for funds. Medical research matters, and must be supported. Derrick, Meredith, and millions of others suffering from debilitating diseases are counting on it.

See more stories about the power and promise of medical research, and tell us why medical research matters to you.

Here's how to get involved:

-- VISIT the campaign Web site
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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

Cutting-Edge Science, Collaboration, and Sustained Funding Needed to Get New Medicines from Lab to Patient

FasterCures and Friends of Cancer Research Host Capitol Hill Briefing

“While I can tell you there’s never been a more exciting time for science, I can also tell you there’s never been a more stressful time,” said Francis Collins, director of the National Institutes of Health (NIH), addressing the issue at hand – tightening fiscal resources that threaten the research and development ecosystem to fully deliver science’s full potential to improve health and well-being. This was the main topic of a Capitol Hill briefing co-hosted by FasterCures and Friends of Cancer Research.

Moderated by FasterCures Executive Director Margaret Anderson, Collins and other medical research leaders representing distinct sectors provided a glimpse into what it takes to turn a scientific discovery into a safe and effective therapy that will improve, and maybe even save, patients’ lives. More than 300 policy decision makers, advocates, and key legislative staffers participated.

“There is no one in this room who hasn’t been touched by disease, whether it’s yourself or a loved one,” said Ellen Sigal, chair and founder of Friends of Cancer Research, opening the discussion. “We cannot let the FDA or NIH become a victim of political polarization.” The discussion kicked off with video highlights from a viral campaign, Time=Lives, that featured images and stories of patients, families, researchers, and executives, reminding everyone in the room about what’s at stake.

Onyx CEO Anthony Coles explains impact
of public investment on drug development 

Underscoring the role of public investment in basic research by the NIH, N. Anthony Coles, president and CEO of Onyx Pharmaceuticals, noted that pharmaceutical companies depend solely on this research to understand biology and disease origins. “Companies like Onyx then take these findings, advance the research, and move it toward delivery to patients,” he said.

Improving and accelerating research and development requires all stakeholders in the medical research ecosystem to work together – including federal agencies, industry, academia, and patient groups. Collaboration – existing collaborative efforts, and the need for even more partnerships – was a resounding theme throughout the discussion.

Disease research organizations feel the excitement for an improved system. “This is the most robust pipeline we’ve seen. Now is not the time to step back from working together, but to do more,” said Deborah Brooks, co-founder of the Michael J. Fox Foundation for Parkinson’s Research.

“The FDA is the final common pathway to translating science to patients,” said Margaret Hamburg, commissioner of the U.S. Food and Drug Administration (FDA). “If the FDA is not fully funded and supported, the ecosystem will not function optimally. When we engage early with the scientific community, we’re able to cut five years off of the drug development process.”

Roy Jensen, director of the University of Kansas Cancer Center, is already seeing how limited resources are impacting research progress. “We’re not even funding a fraction of the best science. We’re starting to cut to the bone – scientists are having to close labs, they aren’t able to train the next generation… It’s fundamentally altering our infrastructure,” said Jensen.

The importance of supporting the next generation of brilliance and innovation weighs heavily on the minds at the NIH, and researchers are hopeful for the next group of young investigators. “It is very tough right now to be a grad student, or a post-doc. As they look at the landscape of this country, they wonder if there’s room for them,” Collins said.

Coles echoed Collins’ concern with a sobering concept about the future of scientific innovation: “What answers won’t we have in 10 years if we don’t fund this research?”

“The decisions we make today will have implications long into the future. Unlike delaying construction of a bridge that can be resumed in a few years, if we lose a generation of scientists, there’s no way to rebuild that human capital quickly,” said Mike Milken, founder of FasterCures and chairman of the Milken Institute. “We fully understand the need for overall budget restraint in Washington; but short-term cuts in bioscience only assure a less-healthy America down the road. Modest investments in prevention and cures today will help avoid the catastrophic costs of care tomorrow.”

* A video of this briefing is now available.

From L to R: Margaret Anderson, Roy Jensen, Deborah Brooks, Tony Coles, Ellen Sigal, Margaret Hamburg, Mike Milken, Francis Collins

Top 10 Medical Research Trends to Watch in 2013

By Margaret Anderson, Executive Director of FasterCures

As seen in The Huffington Post

1. Federal funding: Fasten your seatbelts, it's going to be a bumpy ride.

Congress has pushed the date of the "sequester" off another two months, delaying the prospect of automatic 8.2 percent cuts in the budgets of NIH, FDA, and other federal science programs. But a sequester (or other cuts) could still happen. Consider that current funding for these programs expires in March, the FY14 budgets are still being drafted, and there will likely be a fight over the debt ceiling. Advocates need to keep making noise to make a compelling case for the critical importance of medical research. The health of our citizens and the economic and fiscal health of the nation are at stake. Resources are available on our Sequestration Station website; also check out our Time Equals Lives social media campaign, where you can contribute your story to show our leaders why medical research matters, because we will all be patients eventually.

2. FDA: Recalibrating the benefit-risk equation. 

Patient groups have been saying for years that people living with a disease are often willing to accept more risk than the FDA is generally willing to tolerate in a new product. Well, now the agency can do something about it. In 2012, the latest reauthorization of PDUFA created a new "Patient-Focused Drug Development" initiative at the FDA, which allows the FDA to formally consult with patients -- initially in 20 disease areas -- about what their priorities are and what tradeoffs they are willing to accept. It may not sound revolutionary, but it could represent the beginning of a paradigm shift in the regulatory process if they get the process right, and if we don't get bogged down in fighting over what the 20 disease areas are. This will be an important initiative to track and be active in this year, and an important area for the FDA to focus on across its enters.
By the way, we heard at Partnering for Cures that nobody thanks the FDA -- so here we go! Way to go, FDA, on a second year in a row of 30-plus new drug approvals. If they do it again in 2013, is it a trend?

Saying thank you to FDA's Janet Woodcock

3. Financial innovation: Attack of the megafund. 

In October 2012, MIT finance professor Andrew Lo and colleagues proposed creation of a $30 billion "megafund" to bridge the translational research-finance gap by using financial engineering to securitize a highly diversified portfolio of therapies in development, an idea FasterCures got a preview of at its Financial Innovations Lab in July 2011. The model securitizes a portfolio of research assets as collateral to raise funds in the capital markets using a mix of debt and equity that appeal to a variety of investors' risk-reward ratios. Is this the kind of large-scale solution required for the big problem of financing risky research, particularly at the early stages? Lo plans a conference in 2013 to further develop the idea. Stay tuned for more work in this area by FasterCures and the Milken Institute in 2013, because this is one of the issues we are hearing the most consternation about.

4. Reimbursement: Can value drive innovation? 

Concerns in the pharma industry about the impact of reimbursement on their upstream R&D strategies are escalating, as efforts to rein in health-care costs are gaining steam. But do medical progress and the quality of care have to be sacrificed on the altar of cost-cutting? We heard some interesting discussion in 2012 from former pharma exec Frank Douglas and his new Austen BioInnovation Institute in Akron about the concept of "value-driven engineering" in medical devices, based on the principles of clinical utility, reduced complexity, and cost savings and efficiency. Douglas believes this framework can be applied to drug development as well. Can we shorten the time from molecule to hu(man) to ultimately drive down costs for payers? Will a focus on value be an innovation killer, as some fear, or can it be a disruptive driver for companies to tackle risky areas of high unmet need and first-in-class therapies? What is the methodology for deciding value? Where do patients fit into the equation? We look forward to more broad-based discussions of -- and hopefully more answers to -- these critical issues in 2013, as reimbursement could be our next cliff, if we survive the fiscal one.

5. Venture philanthropy: Playing in the major leagues.

FasterCures has been tracking and supporting the rise of venture philanthropy in medical research for almost a decade, and these strategic, patient-driven research funders have hit the big leagues and gotten the attention of the large pharma companies. Small companies have found them valuable sources of seed capital and other non-financial assets for some time, but large pharma has been slower to understand what they bring to the table. 2012 saw the Cystic Fibrosis Foundation (fresh off a big success investing in Vertex's newly-approved drug Kalydeco) invest $58 million in Pfizer's research into therapies targeting the most common CF subtype (yes, you read that right -- the foundation is funding Pfizer), and Sanofi awarded $300,000 to a team of patient groups to create a cross-disease registry to accelerate translational research. Companies like Merck Serono and Celgene are working with nonprofits like the National MS Society and the Leukemia and Lymphoma Society as "talent scouts" of sorts to identify promising early-stage research. As foundation head Bonnie Addario advised a company CEO at Partnering for Cures in November, "Put us on your boards -- we put you on ours!" We call that speaking truth to power.

6. Academic research: Let's see you do that again. 

Industry and investors are becoming increasingly frustrated with the lack of reproducibility of positive results from academic science published in peer-reviewed journals. In 2012, Amgen could not reproduce the results of 47 out of 53 so-called "landmark" oncology studies, and in 2011 Bayer discontinued 65 percent of its target validation projects when its results did not match published experiments. Some patient groups, like the ALS Therapy Development Institute and the Chordoma Foundation, have made replication of published results and validation of research products a core piece of their missions. Some investors are funding their own "killer experiments" before making investment decisions, and efforts like Science Exchange's Reproducibility Initiative are cropping up to find ways to systematically address the disincentives for academic researchers to validate their findings. Expect this debate to get louder in 2013 -- and hopefully coalesce around some productive solutions.

7. Data standards: Coming soon to a therapeutic area near you. 

We hear over and over again that medical research desperately needs standards for collecting and analyzing data, but it's always seemed like a bite too big to chew. But the Critical Path Institute and CDISC are stepping up to the plate with CFAST, the Coalition for the Advancement of Standards and Therapies, in partnership with the FDA and the new industry consortium TransCelerate. We first heard about it at Partnering for Cures 2011, and it formally launched in October 2012, but it already has standards in seven therapeutic areas (out of 55 targeted) in various states of development. Watch for accelerated progress in 2013.

8. The other translation gap: Turning outputs into outcomes. 

FasterCures has spent a lot of time focusing on the "first translation gap," between basic scientific discoveries and research in humans subjects; somewhat outside our scope has been "T2," the gap between approval of a treatment and its adoption into widespread use to improve patient outcomes, which can take as long as the R&D process. Many others have analyzed this problem, but the ImproveCareNow network has gone from analysis to action, creating a rapid learning system that is speeding the adoption of evidence-based care practices for kids with Crohn's disease and ulcerative colitis at almost 50 care centers across the country and improving their health dramatically. The Kauffman Foundation is working to scale and replicate their success through its Health Network Trust initiative. We hope 2013 will see this model launched in other disease areas.

9. Collaboration: Writing the playbook. 

There now seems to be broad acceptance of the idea that collaboration among R&D stakeholders (government, academia, industry, nonprofits) is required if we are going to make progress in an increasingly complex scientific and business environment. There are many experiments in collaboration going on, and many successful outcomes that can be highlighted. It is time for more systematic analysis of what's working and what's not. While these efforts vary widely depending on the goals and the collaborators, can we draw some common lessons about, for example, what are fertile areas for collaboration, what one should look for in partners, what are the pitfalls to avoid? After all, as Luke Timmerman of Xconomy wrote recently, what we're after isn't collaboration for its own sake, it's a "creative rethinking of the biomedical R&D continuum," and "while those [collaborative] efforts are encouraging, this really should be a national conversation that involves a whole lot more players." Will 2013 be the year we start trying to know what we know, and perhaps even start redrawing that linear R&D pipeline we all know so well?

10. Innovation: Who's outside the box (you tell us). 

While there's no shortage of brilliant scientists and discoveries in medical research, it can be notoriously hard to move them forward in the expensive environments of academia and industry, where risk is not necessarily rewarded. We are seeing more efforts to try to bust out of this box in ways that are more common in other fields. Philanthropist Peter Thiel, who started out by giving young people $100,000 to skip college and work on their ideas, has established Breakout Labs to fund early-stage companies with radical ideas. The National Institutes of Health is getting into the action with its "skip the post-doc" Early Independence Awards to help free exceptional young minds from the academic treadmill. Jimmy Lin's Rare Genomics Institute is crowdfunding genomic sequencing for patients with rare diseases -- and there's much discussion about crowdfunding's utility in life sciences. Crowdsourcing is gaining traction in life sciences, from FoldIt's online protein folding games to Transparency Life Sciences' crowdsourced clinical study design. Who will be redefining "the box" in 2013? You tell us -- send us your ideas at info@fastercures.org.

Catalyzing drug development for the team sport of translational science

Today there are many efforts under way to create drug development tools – from therapeutic area data standards to preclinical safety biomarkers to patient-reported outcomes instruments – needed by the field involving the pre-competitive sharing of data and expertise and leading to the development of standards that are then qualified by the Food and Drug Administration (FDA) and other regulatory bodies.

At this year’s Partnering for Cures meeting, a panel of experts discussed the role of rules, tools, and data pools in the team sport that is translational science. The panel agreed that the efficiency of the drug development process needs to be improved, and the key to doing this will be to reform the system so that the rules are the same for all players involved.

Carolyn Compton discussed the role of the Critical Path Institute (C-Path) in this reformative process. Compton explained that one of the primary goals of C-Path is to form consortia around standards creation to streamline the rules so that they are applicable to all sponsors submitting new drug applications to the FDA. The development of these standards, she noted, will increase the workflow efficiency of both the sponsors and the FDA.

Eric Perakslis of FDA reiterated the need for this type of streamlining. Perakslis explained that currently the system by which submitted applications are checked for completion can take months. In addition, the task of reviewing data that have been collected and measured in numerous ways significantly hinders the workflow. He argued that the creation of thoughtful and meaningful data standards would go a long way in streamlining the review process.

George Vradenburg of USAgainstAlzheimer's pointed out that the value to patients, taxpayers, and industry of modestly compressing drug pipelines will result in billions of dollars in savings; however, he said that we spend too much time discussing the “trivial underbrush” and that we really need to set priorities that will create real value and “aim at those like a laser light.” Vradenburg also highlighted that the path to alleviate some of the process congestion will require a “focus on some big implementation steps with clear action goals that can be taken on jointly by the team,” which includes government agencies, industry, research communities, and patient advocacy organizations.

Some of the major implementation changes that Vradenburg referred to are evident in precompetitive research initiatives among leading biopharmaceutical companies. Marc Bonnefoi of Sanofi US explained that TransCelerate BioPharma and Project Data Sphere, initiated by the CEO Roundtable on Cancer’s Life Sciences Consortium, are examples of successful precompetitive research initiatives among pharmaceutical companies where data are shared with the goal of using data more efficiently to improve the quality of clinical studies and accelerate drug development. Bonnefoi stressed, however, that the success of these precompetitive initiatives is highly dependent on contribution from each organization’s leadership team to support the aims of the initiatives.

Dana Ball of T1D Exchange redirected the discussion toward asking the right questions of the data that we have: “Data for the sake of having data is not helpful. We have to think … down the line to ask what problems we are trying to solve with this information; all of this will [determine] the tools that we will need to build solutions to these problems and the rules [of using] the data pools.”

Panelists agreed that the primary limitation now is not the data; but rather the handling and interpreting of the data. In order to address this problem, there needs to be a major investment in infrastructure. As Compton said, “[This type of investment] is not sexy, but it is absolutely necessary.” We need this infrastructure to make the data powerful enough to turn it into medical solutions for patients.

The election’s impact on medical research

The Partnering for Cures panel “Election 2012: What Does it Mean for Medical Research?,” offered an inside look into the implications of the November election outcomes for biomedical research and innovation. In an animated discussion, panelists covered some of the hottest issues, ranging from funding for the Food and Drug Administration (FDA) and National Institutes of Health (NIH) to sequestration to the importance of maintaining U.S. competitiveness in healthcare innovation.

Neera Tanden of the Center for American Progress emphasized the need for Americans to maintain their competitive, innovative, and economic edge in the healthcare sector. In recognizing that “the president is extremely mindful of NIH’s role and its role in America’s competitiveness,” Tanden identified not only the need to solve the fiscal crisis, which has created a “high level of uncertainty that is unhelpful,” but also the strong connection between the budget and long-term American competitiveness.

Cheryl Jaeger of House Majority Leader Eric Cantor’s office drew an optimistic picture of possible bipartisan actions that could take place with the new Congress that takes office in January. Jaeger argued that while opinions may differ on entitlement spending, “there really is bipartisan agreement on the importance of funding NIH and FDA. If there is one area where both sides of the aisle come together, it is on medical research.” The challenge lies not in realizing the crucial necessity of these organizations for medical research, but in understanding how “we can grow the economy and how to make sure that individuals recognize that these programs are the highest priority within the funding infrastructure.”

Wendell Primus of House Minority Leader Nancy Pelosi’s office focused largely on the government’s need to raise more revenue in general and to take care of the large wave of baby boomers that are set to retire in the next decade. “We are not going to take Medicaid and Medicare spending down enough to increase NIH budgets – it ain’t going to happen. If we don’t raise revenue, the NIH budget will continue to go down in real terms,” he argued. For Primus, Obama’s re-election has meant the chance to raise government revenue and bring the NIH budget back to a place where it is not losing value due to stagnant funding levels and a loss of purchasing power over time.

Scott Gottlieb of the American Enterprise Institute pointed out that company formation has been limited, VCs are consolidating, and funds for startups are beginning to dry out. Gottlieb argued that the NIH is only part of the ecosystem when it comes to innovation, and there is a need to “look at what’s happening on the policy front and look at the other components: the capital formation, what’s happening with reimbursement, immigration policy, [as] these are exceedingly important to biomedical innovation.”

Moderator Greg Simon of Poliwogg posed a final question to the panelists about sequestration and if a substantial deal will be made before the “fiscal cliff” deadline. The verdict was largely optimistic, with Jaeger and Tanden believing a solution was “hopeful,” Primus stating that a solution will be reached “because we have to,” and Gottlieb suggesting that most of the major issues will likely be punted to the next Congress.

The FDA: Balancing innovation and safety

If you talk about the need to speed up the approval process for new drugs and devices, the U.S. Food and Drug Administration is often the first criticized for the time lag to new treatments. But, as the Partnering for Cures session, “What You Probably Don’t Know about Innovation at FDA” made clear, the agency is trying to become more innovative and approve new treatments more quickly and efficiently, but there are obstacles.

The biggest danger, said Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, is “where public criticism and controversy outweigh science.” Deliberations should be made based on the latest science, she said, but controversy affects how the agency works and creates what she called a “circling of the wagons,” a “let’s not be too innovative” mindset.

She noted that the agency approved 10 cancer drugs in the last fiscal year and is on track to do so again this year, but it is work the FDA should not be doing alone. “It is the job of the larger biomedical community in concert with FDA” to get new drugs on the market, she said.

Some drug makers are wary of using innovative approaches with the FDA. “Once we get to the clinical study, if we want to be innovative, we have to do the traditional approach alongside, sort of carrying two tracks to get the product to market,” said Joseph DeGeorge of Merck Research Laboratories.

Many challenges to new drug approval start well before a drug finds its way to the FDA. Jonathan Leff of Warburg Pincus noted that venture capital investment in life sciences has been decreasing dramatically, and in the first nine months of this year, the number of first-time companies receiving venture capital investment was the lowest it has been since 1995. Those venture capital firms that are investing, he added, are shifting their investments to other countries or in less innovative products. “We have tremendous opportunity to translate science that is happening into cures, but the system isn’t working as well as it needs to,” he said. “We are not getting the throughput. We need to address the issues of translating scientific discoveries to new therapies.”

Some stakeholders are finding ways to assist the FDA in the process. Richard Insel of JDRF said his organization decided it had a role in educating the agency in novel treatments and their development. And when JDRF wanted to work on an artificial pancreas, there was no regulatory guidance. So with the FDA’s knowledge, the group convened opinion leaders and created draft guidance that was submitted to the FDA. This helped the FDA draft its own regulatory guidance for the artificial pancreas, which just came out in final form.

Frank Douglas with Austen BioInnovation Institute said his organization decided to bring the FDA in earlier in the process and found that things went smoother. “We spent a lot of time trying to figure out how to approach the FDA to make them feel they were in a safe harbor for collaboration,” he said.

The panelists agreed that bringing patient foundations, as well as the patients and families, into the regulatory process to discuss risks and benefits of individual drugs is vital. “We need to hear from affected communities about what tradeoffs they think are acceptable,” said Woodcock. “That should be our guiding light.”

So what would help the FDA improve the process, moderator Margaret Anderson of FasterCures asked the group.

The innovative spirit that is evident at the highest levels of the FDA need to be transitioned to the practitioners of the review process, said Insel.

Douglas noted that as the FDA collaborates more, it should identify other bodies that can be brought in to help with reviews.

Woodcock noted that the workload at the FDA continues to be a problem. It is hard to feel innovative when you are facing a mountain of paperwork, she said.

Anderson noted that the Alliance for a Stronger FDA, which she urged attendees to support, is focused on increasing the agency’s budget.

One panelist answered the question with praise. “The FDA environment, in terms of helping to advance and stimulate innovation, is better today than it has been in a while, and that is a credit to the leadership,” said Leff.

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.”

FasterCures launches Sequestration Station

FasterCures today launched Sequestration Station – an online destination for relevant and up-to-date news and resources about how sequestration could impact medical research.

The prospect of automatic spending cuts, or sequestration, which will take effect in January 2013 if Congress does nothing, threatens the future of U.S. leadership in medical research and development and will delay or permanently remove access to life-saving medical treatments. Sequestration would slash federal investments in critical health, scientific, medical, and biological research aimed at discovering treatments, moving safe and effective new medicines to market, and creating innovations to grow our economy.

According to a report released by the Office of Management and Budget, sequestration would have a "devastating impact" on scientific research. Here’s how this will impact two agencies critical to advancing life-saving therapies:

• The National Institutes of Health (NIH) will face an 8.2 percent cut, resulting in a $2.518 billion loss. In addition to the discretionary cuts, an additional $150 million in mandatory budget authority (for diabetes research) will be subject to a 7.6 percent cut, equal to $11 million, making the total cut to NIH equal to $2.529 billion.
• $3.873 billion of FDA's budget is subject to an 8.2 percent reduction (merely $67 million of the agency's budget is exempt from the sequester). That cut means that sequestration will erase $318 million from the FDA's budget. Virtually all user fees that were active in FY 12 are considered part of the sequester. Such a loss will devastate the agency, cripple its ability to do its job, and put millions of Americans at greater risk from unsafe food and drugs that come from both inside our borders and abroad

At stake: our economy, national productivity, global competitiveness, and progress against life-threatening disease. Making medical research a national priority benefits all of us.

We’re concerned and you should be, too. But there is something you can do. 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 competitiveness, and result in loss of jobs in communities throughout the country. Share with them why a strong investment in medical research is important to you, your family, and your community.

Medical research matters – we must continue to fund it.