3 Cool Things the British are Doing

All eyes were on the United Kingdom during the 30th Summer Olympic Games in London. The UK captivated the world, reminding us of its rich culture and history while delivering athletic thrills. Though this summer’s games provided reason enough to applaud the UK, its remarkable national commitment to  biomedical innovation should garner equal praise. Here are three reasons why:

1. The initiative to make all national health service (NHS) patient data available for research: In a move that will dramatically alter the landscape for medical and public health research in the UK, the British government plans to make electronic health records from the National Health Service (NHS) widely available for research by 2013. Prime Minister David Cameron announced the groundbreaking decision at the first Global Health Policy Summit held last month, saying the move is part of the UK’s effort to secure a global leadership position in biomedical research. The prime minister said his government will change the NHS data sharing policy from an opt-in basis to an opt-out standard, meaning anonymized patient data will be available for all patients who do not actively decline to participate. The opportunities this policy offers are vast, providing medical researchers access to patient data on an unprecedented scale and potentially delivering life-saving advances in our understanding of managing disease and preserving health.  


2. Leveraging olympic infrastructure for biomedical research: Creating a provident legacy from the London Olympic Games, the anti-doping facility, provided for the Games by GlaxoSmithKline and operated by King’s College, will be repurposed as a new medical research center focused on biomarker discovery and development of targeted therapies. Sponsored jointly by the United Kingdom’s Medical Research Council (MRC) and the Department of Health’s National Institute for Health Research (NIHR), the MRC-NIHR Phenome Centre will be a unique resource focused on analyzing the phenome, the collection of an organism’s expressed traits, including all the proteins and active cellular signaling pathways. Researchers will search for biomarkers that can help identify susceptibility to – and protection from – disease, and may lead to precisely targeted prevention and treatment strategies. 


3. Construction of the New Francis Crick Institute: Even as the massive undertaking of preparing for and hosting the Olympic Games was underway in London, construction of the new Francis Crick Institute, possibly Europe’s largest dedicated research laboratory building, was progressing simultaneously. Scheduled to open in 2015, the Francis Crick Institute will be home to 1,500 staff, including 1,250 scientists, and 120 research groups. The institute is the product of a six-way partnership between Cancer Research UK, the Medical Research Council, the Wellcome Trust, University College London, Imperial College London, and King’s College London. It is designed to attract leading scientific talent from around the world and will work collaboratively with United Kingdom hospitals and universities to study cancer, heart disease, infections, stroke, and immune disorders, illnesses linked to aging and obesity, and other medical issues. The Francis Crick Institute will play a key role in training the nation’s top scientists and in supporting the worldwide biomedical research mission. 

FasterCures has been following the UK’s innovation efforts with interest.  We were pleased to welcome Sir Michael Rawlins, Chairman of the National Institute of Health and Clinical Excellence (NICE), to last year’s Partnering for Cures meeting to talk about the difficult choices Brits have to make when it comes to deciding which new treatments and therapies to cover in a centralized, single-payer system.  This year, we’re happy to have Chas Bountra, Chief Scientist for the Structural Genomics Consortium at University of Oxford coming to discuss its efforts to spearhead "open access" chemistry partnership – a new model for pre-competitive drug discovery in which the public and private sectors collaborate to generate potent and selective pharmacological inhibitors of human proteins that regulate epigenetic signalling, and commit to make these reagents available without restriction on use.

 

Solutions not challenges

by Margaret Anderson, Executive Director, FasterCures

The President’s Council of Advisors on Science and Technology (PCAST) released its “Report to the President on Propelling Innovation in Drug Discovery, Development, and Evaluation” this week.

The punch line of the briefing came toward the end when Steve Galson from Amgen opined that “There are rooms of reports on innovation and the challenges – what will differentiate this one?” It was helpful to have it said out loud, because I felt a sense of déjà vu listening to the presentations and perspectives. Things may look tough now, but they’ll be much tougher if we don’t wake up and smell the coffee.

• We heard about how innovation has played a huge role in advancing the health of our nation, but that the system is under stress.
• We heard about the key role the FDA plays in influencing the ecosystem’s behavior, but even with changes at the agency, more needs to be done.
• We heard about how collaboration is central to future discovery, but the system doesn’t consistently incent it. 
• We heard about unmet need in the form of Alzheimer’s, treatment for psychiatric diseases, cancer, and heart disease and stroke, but that we have been making some inroads with treatments for diseases like HIV/AIDS. 
• We heard about how the entire system is struggling for many reasons – funding, patent cliffs, low-hanging fruit being picked, and overall costs of drug development rising, but that there are byways being built. 
• We heard how patients and venture philanthropy are central to solving the problem, but there aren’t groups in existence capable of taking on all the challenges. 
• We heard there has been progress – at times breathtaking progress – but that there is still a long way to go to reap the benefit of the scientific promise that lies ahead.
• We heard how we need money to do all of this, but we don’t know exactly where it’s going to come from, especially in these tight times.
• And we heard we’d better get moving.

There is a shift underway; during the briefing we heard people like Todd Sherer, who is the CEO of the Michael J. Fox Foundation for Parkinson’s Research, saying he’s optimistic based on things he’s seeing. And Bob Beall, president and CEO of the Cystic Fibrosis Foundation, talking about the game-changing approval of Kalydeco and the need to look at the PCAST report with the express purpose of figuring out what recommendations would speed things up. There was some discussion of consumers and patients needing a seat at the table. Well, we learned doing our report “Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change” that you don’t get handed a seat at the table, you earn it. And you earn it by getting smart about the system and what solutions look like. I’d like to see us moving beyond giving patients lip service to ensuring we’re having a real dialogue with them about what they are already doing, and what is possible if and when they are engaged. As Sherer reminded us, “patients want to be part of a solution.”

We know current resources are threatened by the possibility of sequestration, which would automatically cut the federal budget in January 2013. Future resources to address these pressing needs would also be slashed.

The PCAST report cites two primary needs: 

• better methodologies and tools to translate basic biological insights to validated therapeutic targets and leads, and
• the need to have a more efficient clinical trial system, as it currently constitutes 40 percent of the biopharmaceutical R&D budget.

It posits recommendations in areas of improving drug discovery and development; improving drug evaluation; monitoring and communication about benefits and risks; improving FDA management; and economic incentives. There’s a timeframe for action and a list of steps to be taken. We need to assess how this gets done, which entities can and should do it, in what timeframe, and also look at what agencies like the FDA are already beginning to do under the recently enacted PDUFA V agreement, and perhaps most importantly how to get the resources to do it. We were probably all raised with the phrase “money doesn’t grow on trees,” so we need to figure out creative ideas that will focus attention on the imperative for funding.

By focusing on specific solutions and not just the challenges, this report is different than many before it – but it can’t be put on a shelf, action must be taken. As my husband often reminds me, “How do you eat an elephant? One bite at a time.”

NCATS a Reality

On Sept. 14, FasterCures’ Executive Director Margaret Anderson joined fellow leaders in medical research for the first meeting of the National Center for Advancing Translational Sciences (NCATS) AdvisoryCouncil and the Cures Acceleration Network (CAN) Review Board. FasterCures has been a strong supporter of the establishment of NCATS and CAN and their goal of saving time and effort in the pursuit and conduct of medical research and development across disease areas.

The NCATS Advisory Council is made up of 18 appointed members, and the CAN Review Board is comprised of 24 appointed members. They will both meet on a regular basis to provide guidance, consult, and makes recommendations to improve the translation of basic science into clinical application.

The meeting began with an overview of NCATS’ mission and its role in translational research at NIH, budget details, recent science advances, and major center milestones. Then NIH Director Francis Collins announced Christopher P. Austin as the first NCATS director. "There is no way any one scientific discipline can accomplish what we want to do; we only can be successful if we do this together," Austin said.

The meeting also included a brief history of CAN’s establishment, its functions, and its activities. “Collaboration, coordination, communication, and policy all will play a crucial role in our work to advance new treatments and cures for patients,” emphasized Freda Lewis-Hall, CAN Review Board chair.

The Division of Clinical Innovation led presentations focused on the Clinical and Translational Science Awards (CTSA) program and participated in a discussion of its goals, achievements, and coordination. Austin delivered the final presentation of the day, which focused on the Division of Pre-Clinical Innovation.

In response to a question from a council member about getting medicines to patients faster, Austin replied: “Through the CTSAs. Combining the power of these two groups, we can do some magical things."

Relevant Resources on NCATS and CAN:

Archive andsummary of ‘A Virtual Town Hall with NCATS Leadership’

Factsheet about NCATS

“A Discussion onInfusing Life into the Valley of Death”

Keeping pace with globalization: A question of public health

One-quarter of every dollar spent in this country goes to products regulated by the U.S. Food and Drug Administration (FDA). Think about that for a minute. Every meal you eat, every medicine you take, every cosmetic you use, even the food you feed your pet was vetted for safety by FDA experts. But in an increasingly globalized world, the agency’s responsibilities are quickly outpacing its resources.

In addition to all the food, drugs, biologics, medical devices, cosmetics, dietary supplements, and veterinary products made in the United States, FDA also regulates products coming into the U.S. from more than 300,000 facilities in 150 countries around the world.

However, we could be heading toward some risky compromises– and fast. Consider this:

• 80 percent of the active pharmaceutical ingredients in medications sold in the U.S. are manufactured elsewhere.
• Nearly two-thirds of the fruits and vegetables consumed domestically come from outside of this country.
• Half of all medical devices used in the U.S. are imported.
• Food and medical device imports are growing by an average of 10 percent per year, and pharmaceutical product imports are increasing by nearly 13 percent per year.

“While foreign manufacturing facilities have increased dramatically, FDA’s budget and staffing have remained consistent,” noted Marcie McClintic Coates of Mylan, one of the largest generics and specialty pharmaceutical companies in the world, at a Capitol Hill briefing that FasterCures Executive Director Margaret Anderson moderated earlier this week. “Because their resources for international inspections are so limited, FDA has not visited some of our facilities in more than nine years.”

Panelist Ron Guido of Johnson & Johnson pointed out that counterfeiting is the largest growing industry in the world and the crime of 21st century. “Up to 10 percent of the pharmaceuticals coming to the U.S. may be counterfeit. To put that into perspective, 1 percent equals 40 million prescriptions.”

The challenges of globalization are perhaps most visible in the food industry. “Food imports have almost doubled in the past 10 years,” said Caroline Smith DeWaal of the Center for Science in the Public Interest, “and FDA doesn’t have the capacity or inspection structure to keep up with the needs of consumers, which raises frightening possibilities.”

Congress has passed two new laws aimed at helping FDA handle its responsibilities: The Food and Safety Modernization Act and the Food and Drug Administration Safety and Innovation Act. But the agency is not yet receiving adequate funds to implement either law. And sequestration would further compromise FDA’s public health mission, making an already tenuous situation worse. 

For a resource-intensive agency that spends 80 percent of its budget on people (inspectors, researchers, etc.), cuts could have especially far-reaching effects, impacting the products we use every day and expect to be safe and effective. Even though the agency collaborates with foreign governments, “there is no inspection like an FDA inspection,” noted McClintic. “The bar is higher.”

For more information on strengthening the FDA, visit the Alliance for a Stronger FDA at: http://strengthenfda.org/

Science matters

Our economy, global competitiveness, and, most importantly, our health depend greatly on whether we prioritize our investment in and commitment to science. That sense of urgency and the powerful voice of patients was at the heart of our three-day event, Celebration of Science on September 7-9, held at George Washington University and the National Institutes of Health (NIH). As FasterCures Chairman Mike Milken put it in a Wall Street Journal opinion piece, “[September 7] in Washington, a town not known these days for widespread consensus, a remarkable gathering of leaders begins a weekend of events devoted to a single proposition: Science matters.”

We celebrated because we’ve come so far – the scientific and technological advances of the past few decades have transformed the way we live, what we know about disease, how we’re searching for treatments, and how we’re delivering them to patients. Throughout the three days, we saw glimpses of the transformative power of science and research:

• Progress against HIV. First, from the vantage points of many forces in HIV advocacy – including Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases, and Earvin “Magic” Johnson, chairman and CEO of Magic Johnson Enterprises – two people who defied the odds of a disease that when it burst onto the scene 30 years ago was considered a death sentence. This progress was further crystallized as we heard at the NIH about “Yesterday’s Progess and Tomorrow’s Promise” from Timothy Ray Brown, the “Berlin patient,” the first person who has lived to tell the story of how he once had HIV.
• The power of the human genome. One of the largest collaborative efforts in science, which was led by NIH Director Dr. Francis Collins, is the sequencing of the human genome. Scientists were able to piece together, in order, the 3.2 billion nucleotides that encode our DNA, which is the blueprint for the cells of our body. Due to an incredible surge in the power of sequencing technology and plummeting costs, the idea that we may integrate sequencing into clinical care is closer than ever before. On the campus of NIH, we saw how the future of medicine is taking shape – today – thanks to the family of the 16-year-old Beery twins, Alexis and Noah – whose doctor used genetics to unlock the mystery of the twins’ affliction. What they learned led to a precision treatment approach for the twins’ rare muscular disorder, and they are leading virtually normal teenage lives today as a result.

We also heard about the development of the therapy Kalydeco, which treats cystic fibrosis patients with a specific gene mutation. Kalydeco, developed by Vertex Pharmaceuticals and the Cystic Fibrosis Foundation, is stunning proof that if you can get government, academia, nonprofit, pharmaceutical companies, and patients to work together for a common goal… you can literally change the world.

• Advances in rehabilitation medicine. We heard great optimism from Captain Dan Berschinski (retired), who lost both of his legs well above the knee and part of his hand in an attack in Afghanistan. He stood proudly in front of 1,000 leaders from across sectors, bluntly challenging us to stay focused on making medical research a priority, and in particular on his fellow veterans whose injuries are not as visible as his own, such as traumatic brain injury and depression. His remarks were further underscored by Paul Pasquina from Walter Reed and Geoff Ling from the Defense Advanced Research Projects Agency (DARPA), who showed us the vital importance of this research in treating combat-related injuries.
• Better understanding of the crafty cancer cell. Cancer researchers are in relentless pursuit of therapies for all unmet needs. We heard from the world’s leading investigators about progress in areas such as molecularly targeted cancer drugs, which give patients substantially greater hope than was thought possible even a few years ago. New understanding of the genetic events that contribute to cancer, the survival strategies of tumors and the body's immune responses, adoptive immunotherapy, and other treatment options emerging from their laboratories are achieving increasing success in holding back cancer progression and turning once-fatal diseases into treatable conditions. Susan Lindquist, an MIT researcher, described how a protein’s unique structure is critical to its function and how disruptions to that structure can lead to serious health conditions, such as cancer.

We also heard from Winter Vinecki, a 13-year-old triathlete, who is racing to find a cure for prostate cancer after it claimed the life of her father a few years ago. As crafty as the cancer cell might be, we know that determined advocates like Winter will not rest until we win.

These are among the many patient and researcher stories that came to life in discussions about the power of science. Transformative solutions to many of our most significant challenges are possible – but only if we make science a national priority and reaffirm our commitment to supporting it.

Scientific breakthroughs, combined with public-health advances, underlie what is arguably the greatest accomplishment in human history: World-wide average life expectancy has more than doubled since 1900. This has fostered major social benefits and economic growth in this country and many others.

However, this growth is in jeopardy because of the threat of sequestration, the automatic budget cuts that will happen soon if Congress and the White House do not act. Many in the scientific community are concerned it will cause irreparable harm and cast a pall on the future of medical research and patients waiting for treatments and cures. Last week the Office of Management and Budget (OMB) released a report that laid out the “devastating impact” sequestration would have on scientific research.

Progress we lauded during the Celebration of Science may be halted if federal dollars are cut to the NIH, Food and Drug Administration, and other agencies involved in medical research and advancement.

The Celebration of Science showed why science matters – because of the innovative research being performed in labs in every state, because of the treatments and cures reaching bedsides for a variety of diseases, and because all sectors concerned with medical research can come together with a focus on patients. There is nothing that matters more.

3 Reasons You Can't Miss Partnering for Cures

1. It's all about solutions. This meeting is designed to get things done - from dynamic panel discussions about transformative ideas and successful models, to the dozens of case study presentations about innovative paths toward meeting R&D goals.
2. It's all about collaboration. No other meeting of this scale is solely dedicated to forging strategic cross-sector collaborations. You will learn about innovative partnerships that are paving the path toward a more effective and efficient R&D system, and find partners who could make a meaningful difference.
3. It's YOUR meeting. Whether you're looking for an investor or a scientific partner, a customized partnering system allows you to easily connect with potential allies from all sectors of medical research. Experts will be available on-site for one-on-one, free consultation sessions to help address your challenges. It is a unique opportunity to get a pulse on trends and best practices that matter to you and what you do.

Look who's speaking:

Francis Collins
Director, National Institutes of Health

Janet Woodcock
Director, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

William Chin
Executive Dean for Research, Harvard Medical School

Frank L. Douglas
President and CEO, Austen BioInnovation Institute

Regis B. Kelly
Director, The California Institute for Quantitative Biosciences, University of California

Stelios Papadopoulos
Co-Founder and Chairman, Exelixis

Kim J. Popovits
President and CEO, Genomic Health, Inc.

Jay Schnitzer
Director, Defense Sciences Office, Defense Advanced Research Projects Agency

Scott Johnson
President, CEO and Founder, Myelin Repair Foundation

Todd Sherer
CEO, Michael J. Fox Foundation

... and many more

Learn more about Partnering for Cures

Join >800 forward-thinking leaders:

Biotech and Pharma Senior Executives

• Business Development Teams
• Advocacy and Alliance Leaders
• Federal Affairs Directors

Academic Medical Center Leaders

• Research Hospital Executives
• Institute Directors
• Principal Investigators

Life Science Investors

• Venture Capitalists
• Medical Philanthropists
• Wealth Advisors

Medical Research Innovators

• Patient Advocates
• Disease Foundation Executives
• Venture Philanthropists

Government Decision Makers

• Federal Funders
• Federal Researchers and Regulators
• Research Policy Makers

... and many more

Introducing Fixes in Financing: Novel R&D Funding Models

by Elizabeth West, Program Manager, FasterCures

If you could buy a revenue-securitized bond to fund cancer research, would you? What about financing a clinical trial for a rare disease through crowdfunding? Thanks to a growing field of disease-focused venture philanthropies and nonprofits, and the emergence of creative new models for financing medical research, these scenarios could soon be reality.

Here at FasterCures we have spent the last few years examining, tracking, and highlighting the innovative funding mechanisms that are accelerating medical progress. These include pre-competitive partnerships to de-risk research investment, methods of diversifying research portfolios, bridge funding, and government-backed ventures. To highlight these emerging models – including those that reflect the priorities of medical research finance 2.0 – we recently launched a new monthly series called Fixes in Financing: Novel R&D Funding Models. The series will depict financing structures that are active, launching, or conceptual, and analyze them using a uniform set of metrics that includes investment focus, stage of research, and return on investment (financial, societal, and hybrid returns).

Check out the inaugural case, Breakout Labs, a revolving fund that gives small grants to newly formed companies to fund ground-breaking ideas. And let us know if you have an original funding concept we can feature in the series by sending an email to Elizabeth West (ewest@fastercures.org). We’d love to hear your ideas from both within and beyond the biomedical research sector! 

Finance, especially creative finance, can be value-adding for research, not just another burden or obstacle between promising science and proven therapies.