Science, Cures and All the Stuff In Between

by Margaret Anderson

Reblogged from the Huffington Post

The outlook for science has never been brighter -- we know more today than ever before about the molecular basis for thousands of diseases that impact millions of patients -- but the outlook for funding has also never been bleaker. With the National Institutes of Health losing a billion and a half dollars to the sequester, traditional investors retreating from early-stage life sciences, industry pipelines shrinking, and philanthropy unable to fill the gap, we are poised to leave a huge scientific opportunity on the table, namely our ability to get important new medicines out of the lab and to patients more quickly. And with millions of lives hanging in the balance, it's just not something Americans can afford to do.

FasterCures hosted over 100 congressional staffers, patient advocates, academics and drug developers at a Capitol Hill briefing about the importance of federal support for translational research, the phase of medical R&D between the birth of a basic scientific discovery made in the lab and when a pharmaceutical company tests out a potential new therapy with patients -- or, more plainly put, "the stuff in between" microscope and marketplace.

"If there is anything I believe can bring this nation together for a cause, it is finding cures for disease," said House Majority Leader Eric Cantor in his opening remarks. "We ought to be about research regardless of political leaning, because it is an investment that yields tangible returns for our country." In an environment where Congress is laser-focused on coming up with ways to spend dollars more wisely, his advice to advocates was to emphasize the following message to policymakers:

1. It is good to be about cures. Disease touches us all, and as compassionate humans we should all care about improving and saving lives.
2. It is good for the economy to invest in research. This leads to job creation and global competitiveness.
3. It is a good way to bend the cost curve of health care. We have mounting deficits and debts, and we should prioritize the allocation of funds to areas like this that have the greatest impact.

"This is really an engineering problem," said NIH Director Francis Collins. "It's about taking a pipeline that is too long and too leaky and fixing it." With each new therapy that comes to market taking up to 15 years to develop and costing over $1 billion, it's more challenging than one might think to successfully navigate the translational stage. There is great momentum in science, and we are poised to start solving several of these problems, particularly with the existence of the new National Center for Advancing Translational Science (NCATS) at NIH, but if we don't continue to invest, we risk losing not only time and lives but a significant driver of economic health.

"NCATS is a unique new entity, purpose-built to solve this central paradox: that we know more than ever before about how the body works and what goes wrong in disease but lack the equivalent ability to fix it," said NCATS director Chris Austin. "We're designed to be perpendicular to how the rest of NIH works, and to focus not on what's different about disease but what is common." He went on to describe the institute as a biomedical equivalent to the Internet, focusing not on creating companies or products but on allowing people with ideas to share them in a more productive way, and de-risking investment by other sectors in this translational space.

Both Collins and Austin said that there is no better time to pursue translational research. Today, we know the molecular causes behind 4,500 diseases, yet there are drugs available to treat just 250 of them.

"We are operating on hope, not hype," said Sharon Terry of the Genetic Alliance. "The network solutions that have overtaken other industries and streamlined them are finally starting to hit the biomedical research space, and we can't afford to lose momentum." She specifically referenced networked programs such as the Clinical and Translational Science Awards at NIH that are significantly advancing systems improvement in the translational phase and are primed, with the proper support, to foster more integrated research across communities. A new report released this week by the Institute of Medicine goes into more detail.

"Industry can't do it alone. ... Companies depend on basic science coming from the NIH," Simeon Taylor of Bristol-Myers Squibb said. He described how Big Pharma relies on the basic science discoveries made early on in the lab to develop drugs for patients who need them so desperately. Just last week, BMS, along with eight other companies, provided 58 compounds for an NCATS pilot program called Discovering New Therapeutic Uses for Existing Molecules, designed to develop partnerships between pharmaceutical companies and the biomedical research community to advance the development of therapeutics.

I had the privilege of moderating this discussion. The message that rose the top was clear: Getting a therapy from discovery to patients requires partnership and collaboration across all sectors in the medical research ecosystem: scientists, regulators, industry, investors, and, most importantly, the patients themselves. An effective R&D system is an efficient one, but reengineering the system in the way we know is possible requires support, and with funding at critically low levels, the ripple effects could be devastating. If we are to improve our odds for success for our patients and for our economy, there is no time to waste.

Medical research investment: an opportunity for bipartisanship?

By Cecilia Arradaza, Director, Communications and Policy, FasterCures

May 29, 2013

Photo courtesy of the NIH

A few weeks ago, House Majority Leader Eric Cantor led a bipartisan group of members to the National Institutes of Health campus to learn more about their work, but also to discuss the future.  Reps. Earl Blumenauer (D-OR), Michael Burgess (R-TX), Renee Ellmers (R-NC), Eliot Engel (D-NY), Chaka Fattah (D-PA), Andy Harris (R-MD), Tim Murphy (R-PA), and Ted Yoho (R-FL) joined the Majority Leader.  Cheryl Jaeger, Megan Whittemore, and Matthew Zackon of Leader Cantor’s office and Karen Summar of Rep. Cathy McMorris Rodgers’s office also participated.

Photo courtesy of the NIH

Seeing is believing – walking through the NIH Clinical Center, the crown jewel of our nation’s research framework, provides you not only with hope for the future but a real sense of optimism that science matters and that it can make a difference in our lifetime. So to see our Members of Congress carve out time to visit the NIH provides the medical research advocacy community with great hope we can reaffirm our commitment to the biosciences.

At the Milken Institute Global Conference, on a panel with Senate Majority Leader Harry Reid and FasterCures founder Michael Milken, Leader Cantor said that “[we] believe that science should be a priority.” He added that he “hopes when [the Congress] gets to the point where there are issues that seem insurmountable, let’s go ahead and try and practice doing things together so that we can get some things done.”

He then cited the creation of the National Center for Advancing Translational Sciences (NCATS), as one of the demonstrable results of him working closely with Leader Reid. “Harry, when you and I did NCATS, it was a great example I think of the ways that we can work together without a lot of fanfare and just practicing and putting points on the board, and maybe we can work our way towards larger goals after that.”

Cantor then alluded to the May 9^th bipartisan trip to the NIH that he was organizing. “Not only will we spend a couple hours together one afternoon in Washington, but also perhaps learn something together we can practice trying to solve problems together,” he said.

During the panel discussion, Leader Reid concurred with Cantor’s statement on the critical importance of supporting science and noted that “if we’re going to make progress in some of the dreaded diseases we have, we have to step up as a federal government to do that. It’s going to take us working together to fund these programs so that our great scientists can continue doing what they know needs to be done. And we can’t do it on the cheap.”

Investing in our nation’s medical research infrastructure is an investment in our future.

From a dollars-and-cents vantage point, consider the economic payoff of the Human Genome Project: the U.S. government’s $4 billion investment in the Human Genome Project spurred an estimated $796 billion in economic growth from 2000-2010—a 141-fold return on investment, after adjusting for inflation.

And, we are living longer, more productive lives, due in large part to research advances that started at the NIH. For example, U.S. death rates from heart disease and stroke have fallen more than 60% in the last half-century.

The case for investing in science and medical research is clear. To punctuate this point, let’s turn to the cost of inaction. Well –

• talk to the brilliant PhD with bleak prospects for a career in science…
• talk to the employer who has lost his or her most productive and talented staffer because that person had to instead become a full-time caregiver…
• talk to the patient who just walked out of the doctor’s office after having been diagnosed with a debilitating disease for which there are no cures or meaningful treatment options…

Point punctuated.

Now to further underscore, consider the global environment we’re in. At a time when other countries are doubling down on their biomedical research spending, the U.S. is cutting our investment (figure 1) in the industry that promises more jobs, greater productivity, and lives saved.

With all these compelling arguments to back us up, let’s remind our elected officials of what’s at stake, and get them to act on making medical research a national priority.

Figure 1

Related links:

• Photos from Leader Cantor’s personal Flickr of his trip to the NIH
• Global Conference 2013 panel discussion with Leader Reid, Leader Cantor, and Michael Milken
• The impact of NIH research

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!

Working together towards cures for rare diseases

By Karlee Stewart, Communications Coordinator at FasterCures

7,000 different types of rare diseases and disorders

          1 in 10 Americans suffers from a rare disease

                    95% of rare diseases do not have a single FDA approved drug

                                        The numbers don’t add up.

“Take on the voice that I’m sharing with you today, reach out to your fellow patient advocates who frequently don’t see beyond their own situation and help them understand the bigger systematic challenges we all face.” – Hugh Hempel, The Addi and Cassi Fund 

On February 28, 2013 we joined friends and colleagues in the rare disease community to commemorate Rare Disease Day, the culmination of a week's worth of activities to bring widespread recognition of rare diseases as a global health challenge. 

This year, we were lucky enough to be a part of a major event here in DC kick-starting the week and celebrating the screening of Here.Us.Now a documentary highlighting the Hempel Family and their struggle to save their twin daughters, Addi and Cassi, who are living with Neiman Pick Type C. To learn more about the Hempel’s journey, check out their Time=Lives story.   

The documentary focuses on the pitfalls of an ineffective system for patients with rare diseases who have hit a wall in the search for treatment options or a cure. It features prominent members of the medical research community from academia, pharma, biotech, and patient groups calling for change in this uncommunicative and slow system.

Panel moderated by Margaret Anderson

Following the screening, we heard from National Center for Accelerating Translational Science (NCATS) Director Chris Austin during a panel moderated by Margaret Anderson. “The bad news is that there are at least 5,000 more disease like [Addi and Cassi’s] that when doctors like me see them in the clinic, all you can say is please come back to me when you need supportive care.”

Answers are what the Hempels, and others like them, are looking for. However, because rare disease research today is so siloed and disease-specific, communication between groups is limited, leading to occasional duplication of research efforts and time wasted in a world where there’s no time to waste.

“Reach out to your fellow advocates and rally them together for rare disease research,” Hugh Hempel urged the panel’s audience, “We find ourselves fighting over crumbs from funding from the NIH…let’s work together.” 

Hempel said the biggest issue that smaller, disease-specific groups have is not working together for one ultimate purpose, cures.

Besides an insightful talk and plans for collaboration between advocacy groups, here are some outcomes from Rare Disease Week: 

• Reg4All – a “community of people storing and sharing information about ourselves, our health, and our diseases– to improve health and speed therapies across all disease.” An award granted by the Sanofi US Collaborate Innovate challenge that allowed the Genetic Alliance, CFIDS Association of America, National Psoriasis Foundation and the Inflammatory Breast Cancer Research Foundation to build the registry. 

• Boehringer Ingelheim collaboration with PatientsLikeMe on the rare lung condition idiopathic pulmonary fibrosis (IPF.) The partnership will provide “a customized experience for its users that will allow them to monitor their health and progress over time and connect with other to learn more about the condition.” 

• United States Senate agreed to resolution designating February 28, 2013 as Rare Disease Day

FasterCures was pleased to participate in RDLA's 3rd Annual Rare Disease Cocktail Reception and Movie Screening

Make sure to join FasterCures on Wednesday, March 20 for our FREE Webinar, Building New Patient-Centered Research Networks: The T1D Exchange and Registries for All Diseases and learn about: 

• The T1D Exchange, which consists of an integrated clinic network of more than 65 clinics across the U.S., a biorepository, and Glu, an online community with mobile capabilities for people touched by type 1 diabetes. Its mission is to improve outcomes in the community by facilitating better care and accelerating new therapies on the path to a cure.
• Registries for All Diseases (Reg4All), which recently won the Sanofi Collaborate/Activate challenge – a cross-disease, crowdsourced registry that promises to break down the silos between diseases and gather information from patients in a standardized way.

Automatic budgets cuts could stall cancer progress

By Shawn Sullivan, Program Associate, FasterCures

“When I was growing up, cancer was a death sentence, and today my multiple myeloma is in remission because of a drug that was developed based on NIH-funded research,” said former nine-term U.S. Congressman M. Robert “Bob” Carr at a briefing earlier this month to unveil the American Association for Cancer Research’s Cancer Progress Report 2012.

While the focus of the briefing was on advancements, such as those that helped Carr, the looming shadow of the sequestration and its deep cuts to both the National Institutes of Health (NIH) and National Cancer Institute (NCI) budgets dominated much of the discussion. Jon Retzlaff, managing director of AACR’s Office of Science Policy and Government Affairs, pointed out that “while the federal government has made large and lasting commitments to cancer research in the past, we are concerned that this commitment has been eroding for some time and is about to suffer a massive blow.”

To put this in perspective, Douglas Lowy, M.D., deputy director of NCI, noted that federal support is critical if we are to keep vital programs moving. The National Lung Screening Trial (NLST), developed by NCI, is a prime example. In this trial, a pool of more than 50,000 patients with a high risk of developing lung cancer went through a randomized trial of helical computerized topography scanning multiple times a year for seven years. This new type of scan caused a 20 percent reduction in mortality over a control group that just received a chest X-ray.

Additionally, Lowy pointed out the limitations NCI is facing. The FY12 budget of NCI was $5.07 billion, but a majority of funds are already committed to ongoing activities. In FY12, the NCI funded fewer than 15 percent of new grant applications. This flat funding, along with the uncertainty regarding the sequester in particular and Congress’ questionable commitment to future research funding in general, is causing a lot of reluctance within NCI to start up new large-scale projects such as the NLST.

Anna Barker, Ph.D., director of Transformative Healthcare Networks at Arizona State University and the former deputy director of the NCI, highlighted much of the hope that is being generated because of exciting new developments, such as the relatively inexpensive cost of gene mapping. It really “is a new day in cancer research,” she said. She described an exciting and ambitious community research project sponsored by NCI and the National Human Genome Research Institute called The Cancer Genome Atlas (TCGA). TCGA aims to analyze the genomic changes in a large number of cancers to discover the spectrum of genes implicated in each form of cancer, and to learn how specific combinations of genes work together in the cancer. This information could then be used to suggest new uses for existing drugs and development of new drugs. A project of this size and scope can only be done by the federal government. While expensive, the cost of cancer itself is much more profound, costing this country approximately $250 billion per year, and expected to rise significantly in the coming years.

To take advantage of the exciting discoveries being made in cancer research and treatment, panelists agreed that it is crucial to increase our nation’s commitment to medical research funding.

To conclude the meeting, Retzlaff previewed the Rally forMedical Research, to be held April 8, designed to reinforce the messages delivered at this briefing. It will be held in conjunction with AACR’s annual meeting.  

The IOM wants to know how you feel about CTSAs (say what?)

By Kristin Schneeman, Program Director, FasterCures

Have you ever heard of a CTSA?  How about NCATS?  Does this feel a little like a word game – how many acronyms can you make out of these letters?

CTSAs are Clinical and Translational Science Awards, a significant program at the National Institutes of Health to fund – you guessed it – translational and clinical science and resources (as distinct from the more basic discovery science NIH has historically supported) at the nation’s most prominent academic research institutions, 60 in all, across the entire country. NCATS is the National Center for Advancing Translational Science, the newest Center at NIH which was created to “catalyze the generation of innovative methods and technologies” to support the translation of promising basic discoveries into new diagnostics and therapeutics for patients.

Last fall, NCATS asked the Institute of Medicine to conduct a review of the CTSA program, which at $461 million is the largest component of NCATS’ $575 million budget and its assets. I attended the most recent committee meeting at the end of January, which focused to a significant degree on the CTSAs’ “community engagement.” There was not much clarity about what “communities” were supposed to be engaged – in most cases they seem to be the geographic communities in which the CTSAs are located, but in some cases they seem to be the patient communities that researchers are interested in studying, which may or may not be local. Community engagement activities and research were in previous years required components of CTSA applications but are no longer; that was the source of some consternation at the meeting, with speakers lamenting that relationships painstakingly built over the course of the first five plus years of the program were at risk (and that new ones would not be a priority) if they aren’t a requirement of funding. Among the most eloquent speakers was Bray Patrick-Lake of the Clinical Trials Transformation Initiative, herself a patient advocate, who said trust and relationship-building are long-term endeavors, and that academic institutions need to invest in patient groups and “not just come running when you need them for a grant.”

Also notable at this meeting was the lack of focus on what might be done with the CTSAs as a network, as opposed to how the individual institutions are progressing and evolving. This does seem to have been taken up at previous IOM meetings as part of this review, but it was markedly absent during the discussions about future directions for the program that wrapped up this particular day. NCATS itself has made a principle for the next stage of the program that “the whole must be greater than the sum of its parts,” and many observers believe that great things could be achieved by connecting all these major institutions and allowing stakeholders both internal and external to better understand and access the expertise and resources that reside in them. There is a relatively new Coordinating Center which has begun working on developing shared resources such as IRBshare, Contractshare, and the Research Electronic Data Capture (REDCap) platform, which could benefit external stakeholders as well as the CTSAs themselves.

The IOM is accepting comments from the public about the CTSAs until March 1st.  We encourage you to take a look at the questions and to consider commenting.  Even if you aren’t familiar with the CTSAs, that might be worthy of comment in and of itself. Here are a few of the questions we’re focused on:

• Are potential stakeholders aware of the resources available through the CTSA Program and are there barriers to use of those resources?
• Have the CTSA institutions, individually and collectively, played an appropriate and adequate role in involving and interacting with community organizations and patient advocacy groups? 
• Does the balance of CTSA Program efforts across the continuum of research from first phase studies in humans to clinical trials to population-based research on health outcomes and comparative effectiveness need to shift?
• What do you see as successes, challenges, and future directions of the CTSA Program?

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: Michael Kaplan

“I’ve been a Type 1 diabetic since I was 12, so 31 years; and HIV positive for 20 years this March. I’ve been able to maintain good health, keep my viral load down, my t-cells up, which has allowed me to do the work I do.” – Michael Kaplan, President & CEO of AIDS United

It has been over thirty years since the emergence of the HIV/AIDS epidemic and thanks to incredible advancements in science, a diagnosis once tantamount to a death sentence is today managed in much the same way as a chronic disease. Great strides have been made in reducing the burden of HIV/AIDS, but this is a war still in progress.

A leading advocate for HIV/AIDS patients, policy, and research, Michael Kaplan is the president and CEO of AIDS United.  Born out of the merger of the National AIDS Fund and AIDS Action in late 2010, AIDS United’s mission is to end the AIDS epidemic in the United States by combining strategic grantmaking and capacity-building with national advocacy to ensure access to life-saving HIV/AIDS therapies and services, and advance key policy initiatives.

View Michael's Story Here

“As the successes of early treatment towards prevention merge with health care reform across the U.S.,” says Kaplan” the light at the end of the tunnel is only getting brighter.  I truly believe I’ll see the end of this epidemic in my lifetime."

Kaplan advocates for early testing and treatment as a way to dramatically decrease the number of new HIV/AIDS cases and help those already infected to start managing their illness as soon as possible. Like many currently incurable diseases, access to treatment is crucial. NIH research shows that the right drug cocktail can reduce a person’s ability to spread the virus to another by up to 96 percent.

Medical research discoveries from several fields have helped create many of the current medications HIV/AIDS patients depends upon today. Just as in the past, “fundamental basic research that is being done now is going to lead to things ten or fifteen years from now that we cannot predict,” said Dr. Anthony Fauci at a FasterCures' 2012 Celebration of Science event.

But with funding for research across all diseases at risk because of sequestration – looming, across-the-board budget cuts – support for the work of advocates like Michael and the science he helps to advance is more important than ever. Saving time in medical research means saving lives.  

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

-- VISIT the campaign Web site
-- LIKE the Facebook page
-- TWEET with us at #TimeEqLives
-- DOWNLOAD and SHARE the Message
-- TELL us your story

Relevant Links:
-- Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change

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