Accelerating Cures by Building a More Effective and Efficient FDA

Kristin Schneeman, Program Director, FasterCures
A distinguished panel of observers from patient organizations and industry declared themselves to be in “violent agreement” on the urgent need to increase resources and build scientific capacity at the FDA, with one panelist calling it of “strategic importance to the country.” Among the points of agreement were:

• FDA suffers from an almost scandalous lack of resources. The agency that regulates 25 cents of every dollar U.S. consumers spend has a budget that is half what Americans spend every year on potato chips.
• Scientific capacity at the agency is very weak and is inhibiting the agency’s ability to evaluate new therapies moving through the pipeline.
• The agency badly needs to be able to collaborate meaningfully with other sectors – what some refer to as the “triple helix” of government, academia and industry – without being constantly “demonized” for conflict of interest.

Gail Cassell, Vice President of Scientific Affairs at Eli Lilly & Co., shared the results of an important review of FDA’s science capacity that she chaired in 2007 as a member of FDA’s Science Board, which put a bright spotlight on the resource constraints under which FDA operates and their serious consequences. “Chronic underfunding of FDA puts American lives at risk.”

Garry Neil, Corporate Vice President at Johnson & Johnson’s Corporate Office of Science and Technology, detailed the risky, unpredictable, and costly pathway to new drug approval, a process that hasn’t changed substantially in 50 years. He reiterated some oft-cited facts – that it costs well over $1 billion to develop an approved drug, and that despite exponential increases in investment by government and industry the number of new therapies approved every year has declined. “There is nothing black and white here. The whole process is about the progressive removal of uncertainty. And there will never be complete certainty.”

Ellen Sigal, Chair and Founder of Friends of Cancer Research and an advisor to many government cancer research initiatives, provided the view of many patient advocates who find FDA impenetrable, with no mechanism in place for patient input, a confusing structure, and a lack of expertise in the places where it’s needed. She said the patient voice is not being heard on Capitol Hill, that concerns (many of them legitimate) about safety were all but drowning out patients’ appetite for innovation, willingness to risk-share, and need for better drugs.

Jonathan Simons, President and CEO, Prostate Cancer Foundation, offered up some interesting models for addressing some of these challenges at FDA. One was for some kind of “honest broker knowledge exchange” within FDA that facilitates collaboration among stakeholders in the system, evaluates all data presented by stakeholders, and creates value through transmission of knowledge. He also proposed that FDA have a sort of “amicus curiae” process, as they do in the court system, to allow meaningful input by stakeholders other than regulated industry.

Cassell held up as a success story the development of anti-retrovirals to treat HIV – 29 new therapies approved in relatively short time, achieved when stakeholders worked together across sectors with optimal speed. Simons noted that a significant reason for this success was leadership – that Anthony Fauci at the National Institute of Allergy and Infectious Diseases had responsibility for addressing the crisis and invited in the patient community to provide guidance. Can this success be replicated? “It happened with HIV, it can happen again,” said Neil.

The panel ended on a surprisingly hopeful note, with most panelists echoing Sigal’s optimism that “there is a growing recognition that the agency needs more resources, needs collaboration, needs science, and that its science is different from NIH’s science.” All agreed that the public and Congress need to be better educated about the importance and impact of FDA – not only on health but on our economy and national security.

Health Reform: Where Do We Go From Here?

Cecilia Arradaza, Communications Director, FasterCures

With the health reform law in place, participants joining this year’s Milken Institute Global Conference had more questions than answers about its implications. A second panel on health reform convened Tuesday (Read Summary of Monday Health Reform Panel) looks at cost, coverage, and consequences.

“Cost comes from price [of health care] and utilization,” said Ceci Connolly who covers health policy for The Washington Post. Measures are in place to curb the soaring cost of health care services, like a measure that brings hospitalization costs down by lowering readmission rates by addressing preventable underlying causes. But, “we have a lot to do” when it comes to utilization. The law starts to modify behavior by changing incentives, but it requires broader public engagement.

Michael McCallister, President and CEO, Humana Inc. said that the power of consumers are great and can be leveraged with the right incentives, accurate information, and transparency throughout the health system.

The health reform law has measures in place that will move the country toward “outcomes-driven care,” said Billy Tauzin, President and CEO of the Pharmaceutical Research and Manufacturers of America.

The question now, according to Connolly is “who in the industry is going to innovate?” She noted that “we will see creative, money-making ways” as implementation of different aspects of the law is actualized.

Tauzin said that the law contains protections for biopharmaceutical research if implemented right. To which Connolly pointed out that these protections, the issue of 12-year exclusivity for biosimilars is an ongoing argument among policymakers.

Among the law’s missing links, according to Connolly, was a delay in convening a new Medicare Commission (scheduled for 2014) to examine and improve the Medicaid dental coverage for adults, and no “death panels,” which were never “death panels” to begin with.

Tauzin said the law does not address the litigation system to stop ‘defensive medicine.” The fear of liability encourage healthcare providers to overutilize unnecessary health care service which lead to rising costs that do not necessarily bring better care.

Sandy Beaty, Senior Vice President, Public Affairs, Pfizer Inc., said that implementing this complex health reform law is a five to ten year process.

When panel moderator Sage Kelly of Jefferies & Company asked if timing was right, Tauzin said had the Congress and the President not acted, it would’ve take another 10 years to revisit the issue.

Medical Philanthropy: Investing in the Cure Enterprise

by Gillian Parrish, Manager of Alliance Development and Communications, FasterCures

Though philanthropic investment in medical research accounts for only three percent of overall spending, a pittance compared to government and industry funding, it plays a vital role in catalyzing progress. Unburdened by the demands of shareholders, and the legacy mandates of institutional funders like the NIH, nonprofit disease foundations have the flexibility to fund high risk, high reward research that disproportionately accelerates the pace of innovation.

The “Investing in the Cure Enterprise” panel Monday afternoon at the Milken Institute Global Conference explored what’s working in medical philanthropy and how to encourage more philanthropists to invest effectively in the development of new and better treatments for disease.

Melissa Stevens of FasterCures moderated the discussion, noting the important role that medical philanthropy plays in stimulating research in under-resourced disease areas, and bridging the so-called “valley of death” in funding, between basic discovery and later-stage, clinical research.

Panelists from across the philanthropy spectrum agreed that a mix of accountability and measurement in research, together with investments in process infrastructure, would speed the discovery and development of new treatments.

Though perhaps not as “sexy” as disease-specific investments, funding for the development of medical philanthropy process infrastructure—including the sharing of data and best practices across sectors and diseases—is critical to replicating success and learning from failure, noted Matthew Bishop of The Economist. Melanie Schnoll Begun, Managing Director, Morgan Stanley Smith Barney Philanthropic Services, echoed the importance of understanding failures, saying that often in medical R&D, “failures” are actually successes, in that they eliminate ineffective research options and point the way to better cures.

Jane Wales of the Global Philanthropy Forum talked about the growing convergence between the social sector and private sector. “Philanthropists are bringing the rigor of the private sector to the work they do in medical research,” she said, “and the most important thing they can do moving forward is to pass their values along to the generations that follow to drive sustainability towards long-term goals.”

Susan Axelrod of 23andMe, Inc., said: “Philanthropists like us have a high tolerance for risk. We’d rather push the envelope in dramatic ways, even if we might fail, than play it safe.”

Global Conference FasterCures Panels

Watch video of FasterCures' sessions from Monday covering health and medical research at the 2010 Milken Institute Global Conference.

Healthy Living Trends and Advances: Responding to Today’s Wellness Consumer
Watch a video of this session

Changing Our Health-Care Priorities: Incentivizing Prevention and Cures
Watch a video of this session

Designing Innovative Medical Research Systems: Patients Are the Hidden Asset
Watch a video of this session

Accessing Capital for Medical Innovations
Watch a video of this session

Rethinking Financing for Global Health
Watch video of this session

Medical Philanthropy: Investing in the Cure Enterprise
Watch a video of this session

Rethinking Financing for Global Health

by Loren Becker, Global Health Program Analyst, FasterCures
There is a vital need for creativity and innovation in our thinking about how to fund medical R&D targeting diseases of the developing world, according to a panel of experts from the financial, pharmaceutical, and nonprofit sectors. The discussion focused on innovative financing for global health research and development is part of the health track at the 2010 Milken Institute Global Conference.

Moderator Hannah Kettler, Senior Program Officer and Health Economist at The Bill & Melinda Gates Foundation laid the groundwork for the discussion by invoking the name of the late C.K. Prahalad, who was a leading thinker on the concept of the market at the bottom of the wealth pyramid. Although Prahalad, who died last week, did not focus specifically on health, Kettler drew the connection between his broader concept and the market for new medical tools that target diseases disproportionately affecting the poor in developing countries. The question, according to Kettler, was how to attract more investment of resources, financial and otherwise, from a broader set of actors to help meet the health needs of the poor.

Holly Wong, Vice President of Public Policy at the International AIDS Vaccine Initiative pointed to a proposed product development partnership (PDP) financing facility that would rely on a donor-backer bond issue to generate upfront funding while reinvesting some of the proceeds from product sales into the development of new products. The idea, according to Wong, would build more sustainability into the model and allow for cross-subsidization by directing proceeds from one disease area to potentially fund R&D in another.

Eric Easom, Program Leader for Neglected Diseases at Anacor Pharmaceuticals, represented the perspective of a small, privately-held biotechnology company that is collaborating with several PDPs. Easom said that, for smaller companies, “push” funding, which provides capital to companies looking to invest in R&D for developing country needs, is preferable to “pull” funding, which rewards companies for their successful investments.

Stephen Sands, Vice Chairman of Investment Banking and Global Co-Head, Lazard Healthcare, proposed making the investment climate for neglected diseases look more like the one for orphan diseases, which are very attractive to investors because of preferential payment and taxation environments and active patient populations. Sands felt that the credit markets and venture philanthropy models could provide a source of capital that is better aligned with the high risk nature of investment in medical research than debt instruments such bonds.

The audience also brought a variety of perspectives to the discussion. Robert Sebbag of Sanofi-Aventis provided insight on how major pharmaceutical companies make decisions about whether and how to get involved in global health R&D. When asked about how we can ensure products developed for chronic diseases that affect people in both the developed and developing worlds (e.g., diabetes) are accessible to poor populations, Kettler said efforts to make anti-retroviral drugs for HIV/AIDS more accessible as a potential model; however, she also cautioned that affordability issues need to be addressed early in the R&D process in order to ensure that products are accessible to poor patients in the developing world.

Changing Our Healthcare Priorities: Incentivizing Prevention and Cures

by Cecilia Arradaza, Communications Director, FasterCures

Health reform legislation is a positive step toward providing more Americans with insurance, but there's a big omission: The law doesn't do enough to promote prevention, innovation and cures. In a session moderated by CNBC’s Maria Bartiromo, panelists discussed taking a broader and more holistic approach to what the concept of health care really means.

At least 70 percent of health spending goes toward preventable diseases, most of which are caused or made worse by lifestyle choices. Obesity is linked to several serious medical conditions – from heart disease to cancer – and its consequences are grave.

Every imperative to lose weight is there. But obesity presents an accumulation of complex socioeconomic factors that influence lifestyle choices. For example, in many communities, especially urban neighborhoods, options for healthy choices are limited, not easily affordable, or in some instances, completely unavailable making healthy choices difficult or impossible.

Prevention, said Wayne Gattinella of WebMD, is a “concept without a champion.” Health insurers, pharmaceutical companies and even health-care providers are just not incentivized to promote it. Jay Gellert of Health Net said it’s time to wage a war against obesity, the same way we fought the war against smoking. According to Gellert, “right now, we are fighting this war with squirt guns.”

Ardis Hoven of the American Medical Association called for educating the public in a meaningful way and ensuring that available information is trustworthy and reliable.

Quickly woven into the conversation were the likely suspects of the ever-expanding waistline – Pepsi, Coca-Cola, McDonalds. Are they to take the brunt of the blame? Perhaps. But the larger question remains: Who’s in charge of helping Americans improve their lifestyles, make healthier choices, and enhance productivity?

All panelists underscored that collaboration across sectors – health, policy, business – will be key.

The other side of coin is finding real cures for disease, said Michael Milken, chairman of the Milken Institute and FasterCures. Breakthrough cures will pay tremendous dividends, but we will have to channel capital correctly and streamline the process of translating discoveries into treatments. Milken cited the successful management of polio in the 1950s, and urged policymakers to learn from history.

David Brennan, CEO of AstraZeneca said that it’s “about the art of what’s possible.” The pharmaceutical industry has been treatment-oriented, and for most diseases, there’s limited understanding of prevention.

America’s leadership in medical innovation is now being challenged on a global scale, and the U.S. should look to science parks in China, a biopolis in Singapore and better science and math education in Asian nations to find best practices. Innovation will thrive in an environment where it’s appropriately incentivized and rewarded.

Milken noted that if the U.S. continues to misallocate resources significantly, our future in science will be in question. “What are we as a government going to stand for?”

Accessing "Smart Money" for Medical Innovations

By Kristin Schneeman, Program Director, FasterCures

A panel of international financial innovators in health and nutrition agreed today that there is an urgent need to bridge a widening funding gap between early-stage research and product development, not just with more money but with “smart money.” In describing their models, they demonstrated that government and nonprofit intermediaries can be ideally positioned to play that role, if they take novel and thoughtful approaches.

Moderator Margaret Anderson, Executive Director of FasterCures and Glenn Yago, Executive Director of Financial Research at the Milken Institute laid out the challenges and the urgency in addressing them. The funding “valley of death” in medical research is real and widening; companies and venture capital are increasingly risk-averse, and information gaps between academia and industry are growing. The slow progress in addressing poverty and disease around the world is inhibiting global growth.

Noting that Germany suffers from a lack of entrepreneurialism, especially in life sciences, Stephan Gutzeit, Executive Director of Stiftung Charité, outlined his organization’s model. The foundation makes grants to academic labs, and then when the work is advanced enough to build a start-up company around, a for-profit venture arm invests in those companies, providing wrap-around support to move ideas along. Gutzeit echoed the words of Scott Johnson, founder of the Myelin Repair Foundation, from an earlier panel, when he said that nonprofits can play a unique role as “systems integrators” to bring together the players in the research and development system.

Ora Dar, Head of the Life Sciences Sector of Israel’s Office of the Chief Scientist briefed the group on a new program, the Life Sciences Fund, the Israeli government has embarked on to support its vibrant start-up community, which the government already supports with direct grants to young companies. Four management teams were selected and are in the process of raising funds. In addition to investing directly in all four funds, the government will provide upside sharing of its net profits and downside protection to other investors.

Craig Courtney, Special Advisor, Innovative Finance for the Global Alliance for Improved Nutrition infused the discussion with his learnings from nutrition. GAIN works to address constraints in the market and draw private capital in to improving global food markets. In all cases GAIN is creating partnerships, leveraging its global scale, and de-risking investment by private sources.

While experiences differ from country to country, and between organizations addressing developed world and developing world needs, many of the funding challenges are strikingly similar, and there is a need for more intermediaries to help bridge gaps and address market constraints. One panelist noted that in many cases the models being discussed weren’t terribly novel from a financial markets perspective. Several panelists appealed for a thoughtful role for government and not a knee-jerk reaction to merely inject more capital into the market.

Designing Innovative Medical Research Systems: Patients Are the Hidden Asset

by Gillian Parrish, Manager of Alliance Development and Communications, FasterCures
A panel of experts from across the medical research spectrum who convened today at the Milken Institute’s Global Conference said that while the capital for medical innovation is there, how those funds are allocated will dictate progress towards cures. The discussion—designing innovative medical research systems—explored new models emerging from the biotech and pharmaceutical industries that are clearing the way for faster medical progress.

Moderator Debra Lappin, President of the Council for American Medical Innovation, set the stage by outlining the tectonic shifts in medical research: from new and emerging interdependence of sectors, to flattening of federal funds through NIH, to a newly risk-averse environment in which high risk, high reward research is the first to be cut.

While concerns about gaps have opened up, overall, the medical research system has worked spectacularly well, said Chris Coburn, Executive Director, Cleveland Clinic Innovations. Coburn said “easy” achievements have already been accomplished, thus moving the needle becomes that much more complicated.

Technology, said Usama Malik, VP and Head of Worldwide Innovation, Pfizer, is the lynchpin that will decentralize industry and empower consumers. And technology is what’s needed to cultivate open innovation, which he said, is the model of the future, enabling a free flow of ideas allowing the system to pick the best/most impactful ones earlier on.

The group also discuss the growing disconnect between academia and industry, and the critical need to better bridge innovation from universities into commercial markets.

Scott Johnson, President and Founder, Myelin Repair Foundation, said that we need to reframe academic model to produce results more relevant to commercial industry—over 800,000 academic medical papers are published each year and industry has a hard time wading through them to find gems; we need apples-to-apples comparison.

“Top-down solutions won’t work,” Johnson added. “ You can’t force progress through policy, but you can incent collaboration.”

Despite wide agreement amongst insiders that public/private collaboration is essential, externally it is conflict of interest, rather than confluence of interest, that—unfortunately—makes headlines.

Among the factors that have led to an unclear regulatory pathway is a lack of understanding of the relevance of science in commercial marketplace, said Lesa Mitchell, VP, Advancing Innovation, Ewing Marion Kauffman Foundation.

A better understanding of the tradeoff and dynamic tension between risks and rewards, particularly from the patient’s perspective, is key to accelerating medical progress.

Healthy Living Trends and Advances: Responding to Today’s Wellness Consumer

by Angelo Bouselli, Senior Communications Director, FasterCures

Among the early panels that kicked off the 2010 Milken Institute Global Conference was one focused, appropriately, on healthy living trends affecting aging baby boomers. Brian Wood, Managing Director at Imperial Capital moderated the discussion, on how clinical information and public education can influence a person’s behavior and their healthy living decisions.

“There is a need in this country to educate people on the food they eat, the diet supplements they take, and the overall need to increase physical activity in their daily life,” said Pierre Fitzgibbon, President and CEO, Atrium Innovations.”

The diet supplement industry is now a $25 billion industry in the U.S. and has seen an eight percent growth in the past year.

“There has been a healthy living shift in the way we look at diet supplements. This shift is being driven by the anti-aging movement, the rising cost of healthcare, and the overall focus on avoiding sickness,” said Guru Ramanathan, Chief Innovation Officer and Senior Vice President, GNC. “But diet supplements need to be coupled with changes in a person’s diet and daily exercise.”

“One of the biggest changes to nutrition in the U.S. happened between 1975 and 1985 with the introduction of high fructose corn syrup to the diets of millions of American’s. Corn syrup is used in many foods to soften texture and enhance flavor and corn syrup is one of the major causes of obesity in this country,” said David Heber.

Clinical research and public education are helping to advance the health living lifestyle movement forward. Product label claims and warnings, good manufacturing practices, and adverse effect reports are working to educate the public in decisions related to personalized nutrition.

According to Heber, “giving information to the public is not going to necessarily drive them to modify their unhealthy behavior, but personalizing the information for public consumption will have a greater effect on behavior changes.” Heber added, “99.9 percent of people are identical in genetic make-up, it’s a balanced nutrition and a healthy natural lifestyle that separate us apart.

There is an emerging trend and demand for healthier and more active lifestyles, including yoga. Trevor Tice, Founder and CEO of CorePower Yoga, a chain of over 30 yoga studios said “there are currently 15.8 million American’s that do yoga annually and spending on yoga fitness has doubled since 2004.”

Infusing Life into the Valley of Death

Medical research leaders and patient advocates yesterday participated in a forum that spotlighted the Cures Acceleration Network (CAN) provision of the Patient Protection and Affordable Care Act signed into law by President Obama on March 23, 2010. Speakers and participants were eager to discuss CAN and its potential to transform the medical research enterprise by supporting efforts specifically designed to bridge a gap in the therapeutic development pipeline between basic and clinical research.

Senator Arlen Specter (D-PA) introduced CAN to bridge this gap, often referred to as the “Valley of Death,” where research lies dormant and ideas come to a halt because the necessary next steps to take basic research discoveries and turn it into a safe and effective therapy are not taken. The forum was convened by Parkinson’s Action Network and FasterCures.

According to Amy Rick, CEO of PAN, “the business of taking a discovery and pursuing it for its full therapeutic potential is dramatically different than the business of basic research.” It includes considerations like FDA, legal issues, intellectual property, and the timing structure and milestones are different, as well. Appropriate incentives need to be in place to fill the valley of death.

CAN, as authorized, will be established within the Office of the Director of National Institutes of Health (NIH) and would award grants through NIH to biotech companies, universities, and patient advocacy groups. John Schall, deputy CEO of PAN, provided details of this provision, including:

• CAN is designed to accelerate the development of high need cures, including through the development of medical products and behavioral therapies. “Medical product” in this context means a drug, device, biological product, or any combination of the three.
• A diverse, 24-person board will be convened to advise the Director of the NIH on the conduct of the activities of the CAN. This board will represent multiple sectors including at least four individuals from the venture capital community and at least eight patient advocates.
• CAN is subject to the appropriation of funds.

John Myers, Senior Health and Science Policy Adviser to Sen. Arlen Specter (D-PA), encouraged patient advocacy groups to stay involved throughout the appropriations process already underway and build on the momentum of the health reform law, to ensure the potential of the CAN provision can be realized. Learn about how you can support this effort.

The forum, moderated by Margaret Anderson, Executive Director of FasterCures, elevated questions and discussion items that will likely continue as the process to implement the new health reform law unfolds.

This conversation will continue online at FasterCures TRAIN Central Station. Join the discussion.

Health Reform Act Creates Cures Acceleration Network to Speed Researchers along the Path to New and Better Treatments for Disease

The Patient Protection and Affordable Care Act which President Obama signed into law on March 23rd included a little-discussed, but important provision creating a Cures Acceleration Network (CAN) at the National Institutes of Health (NIH), which will work to reduce the time it takes to move new drugs and therapies from the microscope to the marketplace.

This provision, introduced by Senator Arlen Specter (D-PA) as an amendment to the original Senate bill in 2009, creates new funding mechanisms at the NIH through which contracts will be awarded to accelerate the development of “high need cures” – defined as drugs, biological products, or devices:
• That are a priority to diagnose, mitigate, prevent, or treat harm from any disease or condition
• For which the incentives of the commercial market are unlikely to result in its adequate or timely development.

It will establish CAN within the Office of the Director of NIH and authorize grants expected to more quickly move discoveries from the lab through the development, testing and regulatory review process, into the hands of the patients that need them. This is an important step forward in bridging the ever-widening gap between basic and clinical research, known as the “valley of death” – where promising ideas for new treatments for deadly and debilitating diseases are often slowed and sometimes dropped before they can be fully explored.

FasterCures is committed to helping move basic science down the path towards treatments, and looks forward to supporting NIH in this effort. As we noted in our report “Entrepreneurs For Cures: The Critical Need for Innovative Approaches to Disease Research,” the true measure of success will be in lives saved and suffering diminished.

To learn more, participate in the forum:
Infusing Life into the Valley of Death
Wednesday, April 7, 2010, 8:30-10am
1101 New York Ave. NW Suite 620
Washington, DC 20005
Click here for additional information and to register.

Parkinson's Action Network and FasterCures invite you to participate in a forum about the Cure Acceleration Network, a provision in the health reform law, to learn how it can bridge the chasm between a basic scientific discovery and its application as a medical solution, known as the "Valley of Death." Speakers include John Myers, Office of Sen. Arlen Specter (D-PA); Amy Rick, CEO, Parkinson’s Action Network; John Schall, Deputy CEO, Parkinson’s Action Network; and Margaret Anderson, Executive Director, FasterCures. RSVP by Monday, April 5 to Angelo Bouselli abouselli@fastercures.org or 202.336.8900.