Wednesday, December 22, 2010
Thursday, December 16, 2010
Videos and photos of most panel and plenary sessions, as well as the 31 innovator presentations, are now available online. Please visit regularly over the next week as updates and outcomes are posted.
FDA Releases Guidance: FDA Commissioner Dr. Margaret Hamburg and Director of the Center for Drug Evaluation and Research Dr. Janet Woodcock released guidance for industry codevelopment of two or more investigational drugs for use in combination therapy.
Video: Margaret Anderson, Executive Director, FasterCures, opening remarks at Partnering for Cures 2010
Interview with Fox Business Network's Brian Sullivan: Finding Solutions for Medical Research: FDA Commissioner FDA Commissioner Margaret Hamburg and Milken Institute Chairman Michael Milken on the importance of bringing the medical industry and researchers together.
Video: Opening Plenary Session - Innovation: Where Is It Coming From?
Wednesday, December 15, 2010
FasterCures, a center of the Milken Institute, convened this two-day meeting to bring together nontraditional allies to jump-start agreements and program development necessary to turn scientific breakthroughs into effective therapies for patients.
“We are at a critical inflection point in current discussions within the biomedical research establishment about what actions need to be taken to push science toward cures where possible,” said Margaret Anderson, executive director, FasterCures. “Patients need to know that we, the collective ‘we’, are doing everything we can to get new preventive, diagnostic and treatment options through the pipeline and into the clinic to improve patient outcomes and quality of life.“
In a plenary session, News From the FDA: Drug Development in the Age of Targeted Therapy, FDA Commissioner Margaret Hamburg, M.D., and Center for Drug Evaluation and Research Director Janet Woodcock, M.D., presented a guidance for industry codevelopment of two or more unmarketed investigational drugs for use in combination to treat a variety of illnesses including cancer, cardiovascular and infectious diseases, among other important topics.
“To truly advance medical progress and see the return on our national investment in the sciences, we need to seamlessly pass the baton of innovation from one sector to the next -- from the laboratory bench, through the regulatory finish line and into the hands of patients,” said Commissioner Hamburg.
With 16 panels and more than 80 speakers addressing, the meeting addressed mission-critical issues in the life sciences – from innovation to regulation, from drug repositioning to de-risking investments, and from translational research to data networks. A separate track featured 31 innovator presentations: tangible case studies of cross-sector programs that are potentially transformative and scalable across diseases and sectors.
The meeting also featured a customized partnering system that connected participants from different sectors who shared similar goals or had complementary activities. The system allowed participants to schedule free, one-on-one consultations with pioneers in the field.
In 2009, participants at the inaugural Partnering for Cures meeting said it was "game-changing." A year later, that meeting has yielded tangible results including the development of a preclinical stage cancer program collaboration, the creation of innovative mechanisms to advance cancer research and a matching grant program between a pharmaceutical company and a medical research foundation.
Videos, photos, and session summaries are available online at http://www.partneringforcures.org/.
Sunday, December 12, 2010
Executive Director, FasterCures, The Center for Accelerating Medical Solutions
Every day we see stories in the media about the latest medical "breakthroughs" that could lead to treatments or cures for dreaded diseases. We are overwhelmed with snippets about stem cells, genes linked to Alzheimer's disease, autism and diabetes. We hear that cancer drugs are being tailored to treat an individual tumor. And yet, many breakthroughs like these have not borne fruit for patients.
Whether it's because science is hard and unpredictable, or that resources are limited, or there is lack of prioritization -- too many great scientific ideas sit at the laboratory bench waiting for translation. But, there are successful models that have turned a basic discovery into an effective therapeutic option. These models can provide pathways to a healthier future.
We are at a critical inflection point in current discussions within the biomedical research establishment about what actions need to be taken to push the science toward cures where possible. We need to take advantage of this moment, and we need to bring patients, the public and policymakers into the conversation. Next week from December 13-15 FasterCures is convening in New York City all of the sectors involved in medical research to do just that. Partnering for Cures is a meeting like no other, a place to forge collaborations and participate in outcome-focused dialogue about the challenges facing medical research. We have always maintained that each of the sectors plays a vital role, whether it is government, industry, philanthropy, academia, finance or the non-profits. And the need for their ability to seamlessly pass the baton from one to the other has never been greater or the stakes higher.
Successful models have integrated all of these sectors. Everyone needs to be at this table. Few life-saving therapies have come to market without the resources of private industry. Increasingly, patients have become more sophisticated and disease groups are ever innovating with new models for collaboration with academia and industry. The U.S. government is recognizing that it can play a special and critical role in providing an environment where successful partnerships can grow and proliferate. Partnering for Cures provides an opportunity for all sectors to productively collide, creating an ultimate open source opportunity to shine a light on these models so others can learn and build on them.
One area of keen interest to us is identifying solutions and models to cross the so-called "Valley of Death" -- an ever-widening gap in funding and support for the kind of research that moves basic science down the path toward treatments.
In a new report released today by FasterCures, "Crossing Over the Valley of Death," we highlight the productivity gap that currently exists today, how research moves from molecule to marketplace, how we can traverse the Valley of Death and what all of the sectors are doing towards that end. Many players are marching into that valley, but we are far from reaching the other side.
The need to keep marching with the resources required to make the passage is recognized by advisors to U.S. National Institutes of Health (NIH) Director Dr. Francis Collins, who have recommended that a new translational medicine and therapeutics development center be created by the federal agency post-haste. This proposed center would bring together existing NIH activities in translational research and medicine and allow greater coordination and collaboration internally as well as externally, and ultimately, we hope, produce greater outcomes.
We need to support this recommendation, which if implemented would shine a light on the critically needed but under-resourced and under-appreciated area of translational research. In our jump to embrace this incredible opportunity, however, we need to ensure that Peter is not robbed to pay Paul. NIH's other strengths include supporting the nation's basic biomedical research enterprise, and that focus cannot be lost or diminished in our impatience with the pace of progress. Basic science is still as important as ever, but we also want and deserve concrete outcomes.
Dr. Robert Beall, President and CEO of the Cystic Fibrosis Foundation often talks about the Foundation's own model for de-risking research as providing "more shots on goal." Never has the need been greater to have all of the sectors implement that same approach, to take bold steps to move forward swiftly.
Our nation's wise and prolonged investment in basic science has produced discoveries that now need translation. Knowledge gained from basic discoveries allows us to take more strategic and informed shots on goal. This new and important focus on translation no doubt makes some in the research community uncomfortable, as they worry about focus, changing research priorities and competition over scarce dollars.
We recognize those concerns, but at the same time, the need for accountability and outcomes prevail. Patients need to know that we, the collective "we" in the medical research system, are doing everything we can to get new preventive, diagnostic and treatment options through the pipeline and into the clinic. The alternative to this change is the status quo -- 15 years for an intervention to go from bench to bedside. Clearly that isn't acceptable. Is it?
Join us at Partnering for Cures, December 13-15 in New York City. .
Thursday, December 9, 2010
Key points raised during the forum include:
- The lack of communication between different sectors of the medical field despite the ever-growing state of health information technology.
- The current efforts by the ONC to establish standardized electronic health record (EHR) systems as a means of enhancing the quality of clinical care and
- Utilizing patient data to advance research and development.
- Addressing the challenges of patient privacy and security in implementing standardized EHR systems.
- The need for patients to have access to information in EHR systems, not only clinicians and providers.
- The importance of health information technology encompassing clinical research moving forward, creating effective avenues towards scientific innovation.
This effort is part of FasterCures’ Think Research program that supports the utilization of medical records and clinical datasets towards research on the progression of diseases and the development of treatment methods for them. An updated version of the FasterCures’ white paper entitled “Think Research: Using Electronic Medical Records to Bridge Patient Care and Research” will be released in early 2011.
Tuesday, November 30, 2010
Late-Breaking Partnering for Cures Addition: Hamburg and Woodcock to Discuss Drug Development in the Age of Targeted Therapy
With less than two weeks until Partnering for Cures, we have plenty to look forward to:
- a dynamic program (12 panels and four plenary sessions) with an exemplary speaker roster.
- a customized partnering system that eases the process of finding the right contacts and establishing meaningful relationships (already live and active!).
- a new addition to the program, the Expert Consultations component allows participants to schedule free, one-on-one consultations with scientific, technical, and financial leaders.
- an innovator presentation track featuring 31 case studies of cross-sector collaborations.
- focused networking opportunities according to specific therapeutic affinity areas.
Tuesday, November 16, 2010
The Department of Health and Human Services recently released approximately $1 billion in funds through a new program called the Qualifying Therapeutic Discovery Project (QTDP). Included as part of the Patient Protection and Affordable Care Act (also known as the Healthcare Reform Law), QTDP is unlike traditional NIH peer-reviewed projects as it uses an expedited review process to fund small biotechnology companies producing potential products in the biomedical pipeline. It specifically directs funding to companies developing products that will treat unmet medical needs, reduce long term health care costs, or advance the goal of curing cancer within 30 years.
Approximately 3000 companies from 47 states and the District of Columbia received funding through the program through either tax credits or grants, with California topping the list with nearly $300 million in awards followed by Massachusetts with $125 million. Projects ranged across the spectrum of diseases including approximately 30 research programs in Parkinson’s disease, 60 in Alzheimer’s, 80 in diabetes, and more than 100 in cancers.
Quantifying the impact of this funding on the medical research paradigm will be challenging to measure in the short-term. Here’s hoping that this funding boost will provide financial incentives for small biotech companies to pursue development of much needed therapeutics, and spur greater efforts that will accelerate translation of scientific discovery into clinical products.
At FasterCures, we firmly believe that patients need results-oriented research that may be high-risk but with the potential of high rewards, similar to those supported by this new effort. After all, the real value of our national investment in scientific discovery should be measured in terms of improved patient outcomes: better health, improved quality of life, and overall wellness.
Wednesday, November 10, 2010
Advancing medical progress can sometimes be akin to putting together 3,000-piece 3d puzzle. Each piece is critical and must be in its appropriate place. In medical research, we have many of the most critical pieces such as technological achievements in molecular and cellular biology that are revolutionizing research capabilities and expanding our understanding of disease.
- Once hailed as the pinnacle aspiration of clinical genomics, the $1000 genome now seems to be right around the corner.
- The application of protein sciences, aptly named proteomics, has demonstrated clinical effectiveness in evaluating for heart damage, as in the measurement of troponin, and in identifying responders to targeted treatments like Herceptin in certain breast cancers.
- Advances in bioinformatics are providing researchers the ability to integrate large and complex datasets and model molecular networks.
- And just this month, researchers presented a novel breakthrough using RNA to reengineer human fibroblast cells to pluripotent stem cells in a manner that is almost 100 times more efficient than gene transfer and does not alter the cell’s genome, bringing about tremendous potential for using differentiated stem cells for cellular modeling of disease or treatment response.
Consider that the number of new drugs being submitted to the FDA for approval has fallen from 53 drugs in 1996 to 19 just last year. Very few drugs (only about 8%) make it from pre-clinical testing all the way to market and among the reasons for a drug’s failure to move ahead early is toxicity concerns in animal models during early stage testing.
Recently, I participated in a panel that focused on this issue as part of the Brookings Institute and Friends of Cancer Research Conference on Clinical Cancer Research. The discussion focused on the value 21st century technologies could add to improving drug design, screening, and approval – values that benefit both the drug developers and the patients receiving promising new therapies. In an issue brief the panel put together to complement the discussion, we highlight two case studies (an integrated approach to organ injury and oncology drug-induced cardiovascular toxicity) that may help shed some light on real-world implications of safety assessments.
Among the key concepts from the panel and echoed throughout the day-long conference were:
- Current in vivo models for drug toxicity testing have changed very little in decades and have not taken into account advances in molecular and systems biology.
- Animal models were inadequate predictors of toxicity responses in humans, both in terms of generating evidence for biologically accurate mechanisms of drug toxicity, as well as identifying concerns for low-incidence, but dangerous, toxicity responses in our diverse human population.
We await anxiously where this conversation may lead and look to the FDA to start picking up these puzzle pieces - genomics, proteomics, bioinformatics, and other scientific breakthroughs – and begin piecing them together to improve patient outcomes. Drug toxicity profiling, as technical as it may be, is a critical piece to help complete the cure puzzle.
Adam Clark joined FasterCures in September to lead its scientific and federal affairs programs.
Tuesday, October 12, 2010
"…One thing is clear: if we are to solve the most-pressing public health problems we face today, we need new approaches, new collaborations and new ways to take advantage of 21st century technologies," said FDA Commissioner Margaret Hamburg last week in a speech at the National Press Club that outlined the agency’s vision for advancing regulatory science.
These days it seems, each time anyone – government official, patient advocate, legislator – says anything about the FDA, it often starts with, “The FDA needs…” That’s because this agency, tasked with regulating nearly 25 percent of consumer spending in America, needs so much just to be able to complete its enormous tasks at hand: ensuring safe and effective products, and promoting public health. We all know and publicly acknowledge the gravity of the need. Let’s act and begin addressing it.
At FasterCures, we are committed to improving health outcomes, a better quality of life, and greater productivity for all by ensuring that effective treatment options are accessible and available when disease strikes. But we cannot accelerate medical progress if we do not have a robust, scientifically sound FDA. The real potential of scientific breakthroughs and cutting edge medical solutions to improve the lives of patients, even cure disease, will not be actualized without appropriately resourced review and regulatory processes.
In the report Commissioner Hamburg released last week, Advancing Regulatory Science for Public Health, the FDA outlined its priorities for its new effort, articulating what a focused agenda and targeted investment in regulatory science may bring. We all know too well the consequences of inaction. Here are just a couple of examples from the report where regulatory science can truly impact the research and development paradigm.
- Accelerating Delivery of New Medical Treatments to Patients. The report states that we can modernize product development and develop new tools, standards, assays, disease models and science-based pathways to improve the speed, efficiency, predictability, capacity and quality of the entire process, from development to evaluation to manufacturing.
For example, we can have more adaptive clinical trials like the I-SPY 2 Trial launched in March 2010. This innovative trial design individually targets drugs in development to the biology of each woman’s tumor using specific genetic or biological markers, known as “biomarkers.” This allows scientists to efficiently test the most promising products in women with higher risk, rapidly growing breast cancers, and more quickly eliminating ineffective treatments and drugs.
- Enhancing Safety and Health Through Informatics. FDA houses the largest known repository of clinical data — unique, high-quality data on the safety, efficacy and performance of drugs, biologics and devices, both before and after approval. But the FDA lacks the hardware and software necessary to translate this data into valuable information that could help address key questions.
For example, with the appropriate informatics framework in place, we would be able to look at 15 studies of HIV drugs at once to analyze which products are effective for which patient, and detect a new or rare safety risk by capturing safety information from millions of medical records in months instead of years.
- Leadership, coordination, strategic planning, and transparency to support science and innovation
- Support for mission-critical applied research, both at FDA and collaboratively
- Support for scientific excellence, professional development, and a learning organization
- Recruitment and retention of outstanding scientists
FDA has outlined its future “must do” list. Now it’s time to provide FDA with the resources it needs to do its job and to do it well. The billions of dollars of public and private investment in medical research can only reach its full potential if we have an FDA that’s fully equipped to carry the baton of innovation from the research community over the regulatory “finish line” and into the hands of patients.
Tuesday, September 28, 2010
by Margaret Anderson, Executive Director, FasterCures
Characterizing the abyss separating basic and clinical research with tangible and intangible landmarks, we held our fifth annual blue-sky brainstorming session in partnership with Esquire magazine. FasterCures hosted a provocative and productive discussion last week on “Crossing the Valley of Death” with a group of thinkers and innovators from across the medical research spectrum. Participants represented large pharmaceutical companies, small biotechnology companies, venture capital funds, universities, and nonprofit foundations that fund research. There was broad agreement on the significant challenges we all face – scientific, financial, and cultural – in moving promising research across the valley and that we are at an inflection point where more action and less talk is required.
The objectives of the session were:
- To hear about new models of R&D collaboration and highlight lessons learned from them, and
- To identify broader applications of existing models and opportunities for new collaboration mechanisms.
10 themes that emerged from the discussion:
- A fundamental restructuring of the system is what's needed if the biopharmaceutical industry is to survive and thrive and patients are to benefit from innovation. The rest of the world is innovating outside the constraints of the U.S. system.
- We need to redefine the process to make it cheaper, more sustainable.
- We need to get beyond buzzwords like “open innovation” and “collaborative research,” and dig in to create standards for intellectual property, precompetitive research, collaborative contracting, or we will never be able to scale up models that exist.
- We need to find ways to increase the output of valuable intellectual property, not just any intellectual property.
- While we can’t predict the successful business models of the future, we can create the conditions that will allow for disruptive innovation.
- We need to change the terms of the conversation – we need to talk more about capital efficiency and not stop at building capital, address the issue of patents but also really focus on productivity.
- Decision-making in the biotechnology and pharmaceutical industries is not entirely rational or evidence-driven -- how can we change that?
- There is a crying need for better communication to and understanding by the public and policymakers about the process and roles of the players in medical research. We need to elevate success stories of collaboration as a means to thread the needle more and create a fabric of innovation. It’s time these models transcend the patchwork of case studies and best practices and become the overarching approach that’s sorely missing.
- We need to have the right incentives in place. Universities need to incentivize their faculty towards commercialization and collaboration with industry. They need to change their internal metrics of success. Federal and state policies need to create incentives for the results we want to see.
- We need to rationalize the allocation of our resources -- money, time, and human capital.
A variety of actionable suggestions, for FasterCures and other players, also emerged throughout the day. Stay tuned, we’ll summarize those in another blog post.
We will also be producing a meeting report on the topics that were discussed throughout the day. We expect the discussion to inform the program at this year’s Partnering for Cures conference in New York on December 14-15. And the action items will feed into FasterCures’ strategic planning for next year.
Most importantly, this was an opportunity to have a candid, honest discussion of where medical research is and where we need to go, among those steeped in the system but with the foresight and will power necessary to infuse life into the valley of death. Onward.
Tuesday, September 21, 2010
Last week, an eight member working group of the National Institutes of Health’s (NIH) Scientific Management Review Board (SMRB) issued recommendations for improving the fiscal sustainability and utilization of the NIH Clinical Center, which comprises almost 6,000 scientists and constitutes nearly ten percent of the NIH’s budget.
After over a year of deliberation – and consultation with dozens of internal and external stakeholders from research hospital administrators to potential external users of the Clinical Center to key NIH investigators and advisors – the group, chaired by University of Pennsylvania School of Medicine’s Executive Vice President Arthur Rubenstein, offered three core recommendations:
- That the NIH Clinical Center expand its vision and role to serve as a state-of-the-art national resource for both internal and external investigator use.
- That the governance structure of the Clinical Center be modified to facilitate the development and implementation of an overall strategic vision for clinical research, including eliminating oversight by the NIH Steering Committee and establishing a new governing board comprised of Institute and Center directors.
- That the Clinical Center maintain a stable, responsive budget underpinned by priority setting and funded as a line item in the Office of the Director appropriation.
In May of this year – together with 86 other patient organizations – FasterCures again urged the SMRB to open up clinical center facilities to other researchers through a joint letter to the board.
As our nation’s crown research jewel, the Clinical Center features some of the greatest scientific minds using the most advanced medical technologies in the world. It ignites hope and has a distinguished history of discovery, yet remains underutilized due to fiscal and governance constraints. The workgroup’s recommendations chart a path forward for advancing the cause of clinical research, both within and beyond the agency.
We hope to see the NIH leadership act upon these recommendations and continue the focus on advancing translational and clinical research in the interest of public health. They have already demonstrated a commitment to integrating efforts, collaborating across sectors, and working together to meet the goal of getting therapies to patients faster.
Relevant FasterCures Resources:
Tuesday, September 14, 2010
According to the Centers for Disease Control and Prevention, at least 14 million children in this country have a brain disorder for which there is no treatment or cure. This figure represents 17 percent of children between birth and 19 years of age.
The Children’s Neurobiological Solutions Foundation (CNS) recently convened a meeting that brought together policy makers, researchers, nonprofit organizations, and industry to address the barriers to clinical trials and treatments for children affected by neurological conditions. We participated at that meeting.
According to CNS, when research into a potential new treatment advances to the clinical stage, obstacles arise. Among the reasons are that few clinicians are experienced in devising pediatric clinical trials for brain disorders, the potential risks of pediatric trials discourage institutional review boards and scare drug companies, and biomarkers or imaging technologies readily used for adult trials have not been adapted to pediatric populations.
CNS is hoping to create the Centers for Excellence for Pediatric Neurological Disorders, a network of locations across the U.S. focused on conducting pediatric brain clinical trials. Because of its specialty focus, testing of treatments for pediatric neurological disorders would be accelerated, significantly enhancing the possibility that laboratory discoveries are translated into safe and effective treatments. The proposed network would be organized and managed under several Centers of Excellence – a hub and spokes system that would be populated by academic child neurologists across the United States. The network would also be charged with:
- Providing training for child neurologists and their physician and nurse colleagues in clinical trial design management, and interpretation;
- Exploiting existing infrastructure for clinical trials design, execution, and, evaluation, as well as creating infrastructure where needed;
- Evaluating emerging basic science discoveries as potential treatments;
- With colleagues at the FDA and industry, assessing the feasibility of developing discoveries into drugs or other therapeutic modalities;
- Enlisting international partnerships whenever necessary to advance the mission; and
- Communicating the results of studies to colleagues and to patients and their advocates, including assembling a searchable accessible database.
At FasterCures, we are firm believers that expediting cures requires collaboration. It was affirming to hear the enthusiasm of participants at this meeting all eager to see this network come to fruition and deliver on the promise of a more connected clinical trial system for children with neurological conditions.
At the upcoming Partnering for Cures meeting, we are specifically spotlighting innovative, cross-sector collaborations that are advancing medical progress. A call for applications for innovator presentations is now underway. Our goal is to feature up to 30 of the most forward-thinking, transformative efforts. To learn more, visit http://www.partneringforcures.org/.
Monday, August 30, 2010
By Margaret Anderson
Executive Director, FasterCures
On August 23, a federal judge blocked NIH from funding human embryonic stem cell research, ruling that the support violates the rider (the Dickey Wicker amendment) of the Health and Human Services appropriations bill written by Congress in 1996. The rider prohibits the use of taxpayer money for research “in which a human embryo or embryos are destroyed, discarded, or knowingly subjected to risk of injury or death greater than that allowed for research on fetuses in utero.”
In 1999, the Department of Health and Human Services General Counsel determined that this law does not prevent NIH from supporting research that uses embryonic stem cells derived—using private funds—from embryos destined to be destroyed by those no longer needing them for infertility treatment. That ruling was expectedly controversial but it set the wheels in motion for U.S. scientists to explore how these most versatile of human cells could be used to safely test new drugs, restore worn and torn tissue, and replace diseased cells with healthy cells.
In 2001, President Bush halted research on all but a few embryonic stem cell lines. Last year, President Obama lifted some restrictions, but only after a clear and publicly vetted set of guidelines was in place for proceeding with scientific work.
Although the private sector is always free to conduct this type of research, most agree that we need the rigor, stature, and transparency of federal funding for the field to move forward.
At the bench, there are many ways to read the intent of Congress. Judge Lamberth read the intent of Congress in a way that would prohibit federal funding for such research.
If you visit the NIH’s Stem Cell information page today you will read that:
“Pursuant to a court order issued August 23, 2010, NIH is not accepting submissions of information about human embryonic stem cell lines for NIH review. All review of human embryonic stem cell lines under the NIH Guidelines is suspended. The February 23, 2010, proposal to revise the Guidelines is also suspended.”
The impact of this decision cannot be understated. Millions of research dollars and the grant review process have been frozen. Policymakers on both sides of the aisle have spoken out in support of this area of research, given that specific safeguards are in place.
The public opinion on pursuing this research is clear. The scientific need to pursue these avenues is clear. If, as the judge in this case asserts, the 1996 amendment is in the way, then it needs to be reexamined.
A June 2008 Time magazine poll conducted by the SBRI research group found that 73 percent of Americans support embryonic stem cell research using cells derived from embryos about to be discarded by couples after infertility treatment. A majority of Americans support embryonic stem cell research as long as there are strict guidelines and systems of oversight in place, which there are.
In addition to extensive ethics review at NIH, every institution receiving federal funding for this research—that could mean the academic medical center down the street from you—has several committees in place to review this area of research to ensure it is conducted ethically and with only the highest scientific justification. Citizens sit on these committees, as well as scientists, physicians, lawyers, and ethicists.
The solution now is for Congress to craft a new policy that allows the research to proceed, with federal dollars and with appropriate oversight. In the absence of a legislative change, the lawyers will continue to battle, which will take valuable time, while patients and their families sit by helplessly, waiting for a political miracle.
Thursday, August 19, 2010
While we focus on improving the efficiencies of the system that discovers treatments and cures for disease, there are untold numbers of people taking a medical treatment journey right now.
For the kids partaking in the 28th year of Camp Fantastic in Virginia this week, they get to focus more on the fun, and less on the challenges of coping with a cancer diagnosis and with treatment. Camp Fantastic is a program of a nonprofit called Special Love that gives cancer families support.
I learned of this amazing place from Kathy Russell who has been involved since its beginning and who also runs the Children’s Inn at NIH. The NIH Children’s Inn helps families with kids in treatment at the NIH Clinical Center get a bit of normalcy in their lives by providing a warm atmosphere for them to stay in versus an isolating hotel room. The overriding philosophy is that families make a key difference in the lives of their sick children. The work they do represents the full spectrum of NIH investment – from bench to bedside.
By the end of my chat with Kathy, after I dabbed my eyes, I was ready to pack my bags and tell everyone I knew to come with me to help prepare Camp Fantastic and allow kids there to take a break from cancer and be just kids. This year there will be nearly 100 kids at Camp Fantastic between ages 7-17. Usually one-half of them are in active treatment and there are upwards of 60 medical professionals (in addition to countless other folks) who volunteer their time before and during the camp. They literally set up a mini-hospital on-site because it’s in a remote location and far from a hospital with specialized pediatric oncology services. She told me of how kids get their bloodwork done in the am, and a van takes the samples into NIH to do labs and then turns back around with results and medication.
Every year, at least one child is usually transported from the camp in an ambulance or a helicopter to a hospital for further treatment, requiring diligent attention to medical details as well as a carefully thought through psychosocial plan of action to share that news with the other campers. Families are communicated with each day, and as you’d imagine many are nervous and excited about their kids being there. Some children participate while in their final stages of life. Their families make the ultimate sacrifice by being selfless enough to share their children with others, so that their kids can live out their final hopes and dreams – the same dreams we all have. To do the things we dream of doing, especially those seemingly simple things associated with summertime.
Stories like this remind me of the passion and dedication of the medical research community, of the care providing community, and of the volunteer community. It’s a reality check for me, and for those of us who work on policy-related issues. These kids and their families point out the obvious – that time is of the essence in all we are doing to get to faster cures.
It can be hard to make the FasterCures message personal at times as we deal with mostly macro-level issues, but hearing about Camp Fantastic reminded me why we do what we do. Because it’s summer vacation time, and every kid (and for that matter, every grown-up) deserves a shot at creating their own lazy crazy hazy days of summer memories.
Monday, July 26, 2010
Be a part of an effort that brings together people with the expertise, experience, and creativity needed to transform the medical research system. Join us at Partnering for Cures to be held December 14 and 15 in New York.
This year’s program will feature cutting-edge discussions about key topics in medical research – from translational research to data frameworks, from regulatory science to new business models to accelerate therapeutic development.
Among the speakers will be medical research decision and policy makers, high-profile philanthropists, leaders of innovative nonprofit foundations, forward-thinking industry executives, and scientists engaged in some of the most ground-breaking research on the globe. Confirmed speakers include:
- Margaret Hamburg
Commissioner, U.S. Food and Drug Administration
- Carolyn M. Clancy
Director, Agency for Healthcare Research and Quality
- Delos (Toby) Cosgrove, M.D.
Chief Executive Officer and President, Cleveland Clinic
- Jesse Dylan
Founder, Lybba.org; Filmmaker, Creative Director and CEO, FreeForm
- Maria C. Freire
President, The Albert and Mary Lasker Foundation
- Stephen H. Friend
President, CEO, and Co-Founder, Sage Bionetworks
- Jeff Hammerbacher
Vice President, Products, Cloudera
- Isaac (Zak) S. Kohane
Harvard Medical School, Children's Hospital Medical Center
- Edison T. Liu, M.D.
Executive Director, Genome Institute of Singapore
- Susan Love
President, Dr. Susan Love Research Foundation
The Partnering for Cures agenda will feature:
- One-on-One Partnering Meetings. Connect with nontraditional allies to explore collaboration and develop new strategies.
- Therapeutic Affinity Roundtables. Engage with leaders in your field through focused, informal networking opportunities.
- Innovator Presentations. Learn first-hand about multi-sector collaborations, novel research portfolios, and effective partnerships at dozens of sessions featuring innovators on the frontlines.
- Hot-Button Panels. Participate in dynamic, candid, cutting-edge discussions about key topics in medical research including translational research, shelved compounds, data networks, and many more.
- Expert Consultations (new addition!). Access technical and scientific experts on-site to help you address some of your organization’s mission-critical challenges.
Tuesday, July 6, 2010
How appropriations can make a crucial difference in advancing medical progress
The Cures Acceleration Network (CAN) – authorized through the Patient Protection and Affordable Care Act or health reform law – will allow the National Institutes of Health to seek out and support biotech companies, universities, and patient advocacy groups that are pursuing promising, innovative research. CAN promises to establish a new funding mechanism that promotes outcomes-driven research. As authorized, CAN would have a budget of $500 million 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, and
- for which the incentives of the commercial market are unlikely to result in adequate or timely development.
But without appropriations, CAN will remain an unrealized promise.
We urge all in the patient advocacy community to support establishment of CAN within the NIH Office of the Director, as called for by the health reform law, with appropriated budget of $50-$150 million. Opportunities to change the trajectory of medical research across sectors and for all diseases do not come often. Consider reaching out to Members of House and Senate Appropriations Committees and their staff to voice your support for the Cures Acceleration Network
Thursday, June 3, 2010
Building a 21st Century FDA: Getting new treatments out of the pipeline and into the hands of patients requires more than just good research
By Gillian Parrish, Manager, Alliance Development and Communications
Every day, hundreds of thousands of lives are indefinitely on hold – patients waiting for effective treatment options to become available, families waiting for their loved ones to start having a better quality of life, the list goes on. At a standstill – and that’s if they’re lucky – because research that promises to curb their disease has yet to translate into new drugs or devices they can utilize.
Often, when a new molecular entity or biomarker is discovered that has promising implications for disease treatment, it gets waylaid for years in a needless web of outdated regulatory processes that are under-resourced and ill-equipped for modern scientific review. According to a report by Nature Reviews Drug Discovery, the number of new molecular entities approved by the FDA has dropped steeply in the last 15 years – from 53 in 1996 to 19 in 2009, over a period when the budget for National Institutes of Health (NIH) nearly doubled. And most patients living with serious illness can’t wait the 10-15 years it takes to move a new discovery through the current development, testing, and regulatory review process.
While increasing funding for NIH research is a critical step in finding cures, it’s only part of the equation. The remaining part depends on improving the regulatory process, building capacity—both financial and scientific—at the Food and Drug Administration (FDA) so it can carry the baton of innovation from the research community across the regulatory “finish line” to patients. One vehicle for speeding review and access is the Prescription Drug User Fee Act (PDUFA), which is due for its fifth Congressional reauthorization in 2012.
The advocacy community’s role in reauthorizing an effective PDUFA was the focus of a panel hosted by BIO and PhRMA last week. Panelists Marc Boutin (National Health Council), Marcie Bough (American Pharmacists Association) and Jeff Allen (Friends of Cancer Research) called upon advocates to step up and be disruptive, working collaboratively with each other and with industry to speed product reviews and ensure the right balance between safety and access. Margaret Anderson (FasterCures) who moderated the discussion summarized the key themes at the end of the briefing:
- Making sure the patient perspective is heard throughout the reauthorization process
- Applauding FDA’s efforts to be more transparent, and taking them up on their willingness to have a two-way dialogue
- Working together across sectors and diseases to find and leverage commonalities like:
- Improving REMS – Creating a standard Risk Evaluation and Mitigation Strategies (REMS) template, involving patients in the evaluation process, and establishing clearer metrics
- Incorporating Regulatory Science – Though user fees aren’t typically applied to scientific programs, reviewing drugs is a complex process that requires a complex way of thinking, and process science should be incorporated into PDUFA to increase efficiencies
- Advancing Public Education – Helping patients and consumers understand what FDA can do for them, and mobilizing them to act
- Balancing Fees and Appropriations – The appropriated base of human drug review has not kept pace with FDA’s workload (currently ~65% is paid for by user fees); a consistent, multi-year funding approach is necessary to operate a modern-scientifically based regulatory program
Tuesday, June 1, 2010
A week and a half ago I was gearing up to moderate a panel discussion at the Biotechnology Industry Organization’s annual convention in Chicago about venture philanthropy and the impact it’s having in accelerating the development of new treatments for patients. We’ve chaired panels similar to this one at BIO in the past, highlighting the financial resources patient organizations can bring to the table and the partnerships they’re increasingly forming with companies to develop products. This year we decided to focus in a little more on the non-financial assets these organizations also bring -- the “patient capital” in the form of tissue banks, medical data, and participation in clinical trials – as well as their ability to serve as “systems integrators” bringing all of the stakeholders together and smoothing over the information and funding gaps in the research process.
We assembled a phenomenal panel - passionate patient advocates, but also serious and successful R&D professionals -
- Howard Fillit, Executive Director of the Alzheimer’s Drug Discovery Foundation (ADDF);
- Dick Insel, Vice President for Research at the Juvenile Diabetes Research Foundation (JDRF);
- James O’Leary, Chief Innovation Officer at Genetic Alliance (GA);
- Louise Perkins, Chief Scientific Officer at the Multiple Myeloma Research Foundation (MMRF); and
- Steve Perrin, Chief Executive Officer and Chief Scientific Officer at the ALS Therapy Development Institute (ALS TDI).
Among the many impressive nuggets I jotted down during the session:
- MMRF has demonstrated its ability to activate clinical trials 30-40% faster than the industry standard, through its powerful consortium of 13 leading academic research institutions.
- JDRF is pursuing more than 20 development efforts in partnership with companies -- many of which have been picked up by large industry players – including novel agreements with Johnson & Johnson and Novartis to help those companies fill their product pipelines.
- ADDF has been instrumental in the creation of several startup companies that have gone on to leverage enormous amounts of follow-on investment.
- GA is providing resources to help patient groups in rare genetic diseases marshall the patient assets they need to help them engage companies in developing treatments for those diseases.
- ALS TDI has created a new model, a “nonprofit biotech company,” that is shaving millions of dollars off the development process and de-risking industry investment “by keeping patients in mind from the very start.”
- At the end of the session, I offered a question for further reflection and discussion: What are the implications of putting the patient (intermediated by organizations like these) at the center of the research and development process – not as research subjects or consumers, but as partners and decisionmakers?
Among their frustrations was the lack of true communication among patients, their physicians, and particularly the companies developing drugs to help them. “They should pay us to come talk to them,” commented one. She may have said it half in jest, but I suggest we take that idea seriously. And invite the FDA to listen in.
Kudos to Maureen Martino for trying to present a “human face [to] the drug industry.” We’ve been trying to do the same thing for a number of years by supporting the work of innovators like our BIO panelists, who are the trusted intermediaries for meaningful participation by patients in the research process. As a participant at a recent FasterCures meeting has said, “Patients are the only unique resource in the entire continuum … they can create the ultimate bargaining unit.” Where’s Norma Rae when you need her?
Our nation’s research crown jewel ignites hope – it features some of the greatest scientific minds using the most advanced medical technologies. And yet, when we checked on its utilization a year and a half ago as part of our Task Force on NIH’s Intramural Research Program, it was far from fully utilized. Recognizing this untapped potential, the SMRB invited experts (such as Robert Califf, Art Levine, Bill Crowley and Samuel Silverstein, among others) to advise them on opportunities and challenges for expanding the use of the Clinical Center to external researchers.
They agreed that while intellectual property and conflict of interest issues would need to be addressed and operationalized, there was no major impediment to outside investigators using the clinical center, and noted that many academics and nonprofit disease research foundations were, in fact, ready and eager to start utilizing the Center’s many training and research resources immediately.
Last year, FasterCures issued a white paper developed by our Task Force on NIH’s Intramural Research Program that recommended an enhanced and expanded role for and use of the NIH Clinical Center so we were particularly pleased the SMRB decided to take this issue on. And we were not alone. In advance of the SMRB meeting, we circulated a letter within the patient advocacy community to gauge support for the premise that the Clinical Center be made available to the external research community. Within days, 86 other organizations signed-on. In the letter to Director Collins and the SMRB, the patient community recommends the NIH:
- Create streamlined mechanisms by which external researchers can more fully use the Clinical Center for projects in collaboration with the IRP (for example, giving the Clinical Center and/or Institutes the flexibility and authority to negotiate broader collaborative agreements or public-private partnerships, taking into consideration ethics rules and intellectual property rights).
- Explore the possibility of the Clinical Center controlling a pool of funds to make use of the facility feasible for investigators who otherwise could not afford it (for example, through a program similar to the existing Bench-to-Bedside Awards).
Monday, May 24, 2010
What would you do if you made something no one can afford but can’t live without? Biotech companies working on developing drugs for diseases of poverty face this daunting question every day. Pursuing an idea that could potentially save millions of lives from malaria, tuberculosis or other neglected diseases requires a substantial financial investment with little to no financial returns. This was at the heart of an innovative financing discussions at the Partnering for Global Health Forum and the Rethinking Financing for Global Health panel at the Milken Institute Global Conference.
According to experts on these panels, this challenge could be addressed through push or pull mechanisms
- “Push mechanisms” incentivize investments by subsidizing (or directly paying) biotechs to conduct R&D through product development partnerships, tax credits, and grants. These offer the least risky scenario for biotechs, but are costly for donors and require them to take on the full risk of failure.
- “Pull mechanisms” create a guaranteed market, for example, or increase the value of a more marketable product. These typically require companies to take on more risk at the onset but also provide a financial reward for success when a product is approved and reaches the market. If the reward is priced high enough, investors might see the up-front investment as being more worthwhile. For cash-strapped biotechs, one solution might be to establish interim rewards, granted for reaching specific milestones in the development process (i.e., completion of a Phase 2 trial). Panelists at the Forum suggested that such a system might increase the willingness of companies to shoulder some of the burden.
What is clear is that this kind of constructive and creative thinking is vital to curing diseases of the developing world.
- Rethinking Financing for Global Health panel at the Milken Institute Global Conference.
- Archived Webcast of Partnering for Global Health Forum
Scientists at Penn State University think you can and they are developing a diagnostic test that detects a unique odor emitted by even asymptomatic malaria patients. A German research team is trying to create a vaccine delivery platform that activates when it comes into contact with human sweat. Who comes up with these novel, transformative ideas? They emerge when you incentivize creative, nontraditional solutions to age-old challenges.
There are no stupid ideas when developing a strategy to fight ancient diseases like malaria, recently identified as the cause of King Tut’s death, or tuberculosis, which has been found in 9,000-year-old skeletons. That is the basis for the Gates Foundation’s Grand Challenges Explorations program, whose most recent grants were announced May 10. Awarded twice a year, the $100,000 grants are meant to provide creative problem solvers with enough funding to figure out whether their crazy theories could actually work. If successful, grantees could be eligible for up to $1 million more to further develop their ideas.
The Grand Challenges Explorations program is a great example of how foundations and philanthropists are able to take risks that private companies and governments frequently avoid, a theme that often emerges in our medical philanthropy work at FasterCures. Unburdened by the demands of shareholders or legislative mandates, philanthropic donors have the flexibility to fund high risk, high reward research that disproportionately accelerates the pace of innovation.
Even as the Gates Foundation announced this round of grants, applications were rolling in for the fifth round, which will be awarded in November. We are always excited to see what transformative idea will receive funding next in an environment where scientists are encouraged to think outside the box in proposing solutions.
Monday, May 10, 2010
Briefing with NIH Director Francis S. Collins: Leveraging Federal Investment to Speed the Development of Promising Therapies for Patients
- Francis S. Collins, M.D., Ph.D., Director, National Institutes of Health
- Senators Richard J. Durbin and Richard C. Shelby
- Robert J. Beall, Ph.D., President and Chief Executive Officer, Cystic Fibrosis Foundation
- Moderator: Margaret Anderson, Executive Director, FasterCures / The Center for Accelerating Medical Solutions
WHERE: Dirksen Senate Office Building, G-11
WHY: “The Cystic Fibrosis Foundation has shown the way, has lit up the path… and what’s been learned from CF can be extrapolated, generalized, to hundreds of other diseases.”- Francis S. Collins, M.D., Ph.D., Director of the National Institutes of Health
The past few decades have brought exciting scientific breakthroughs necessary to understand, diagnose, and treat many diseases. However, the ability to translate exciting advancements into treatments that can help patients severely lags behind the pace of innovation. On average, it takes 15 years to turn a scientific discovery into a viable therapy. For the millions of Americans who live with chronic and fatal diseases, this is simply too long to wait.
Fifty years ago, people with cystic fibrosis did not live long enough to attend grade school, but today, there are more than 30 drugs in a CF drug development pipeline and the median life expectancy for someone with the disease is 37 years.
NIH Director Francis S. Collins, Dr. Robert J. Beall of the Cystic Fibrosis Foundation, and Margaret Anderson of FasterCures will address:
- What lessons can be learned from the cystic fibrosis successes that can map the way for other diseases?
- How can federal investment at the NIH and other agencies be leveraged to answer important scientific questions in a way that accelerates the discovery and development of medical solutions for deadly and debilitating diseases?
- How can we bridge the “Valley of Death” between basic science discoveries and the creation of new therapies for patients?
Wednesday, May 5, 2010
A year ago, FasterCures launched the Philanthropy Advisory Service (PAS) to establish clear and transparent evaluation of nonprofit activities in medical research—helping philanthropists better understand and evaluate the R&D landscape, and strategically guide their investment dollars in high-impact areas. In its initial phase, PAS features the latest medical research developments and objective analysis of key nonprofit disease research organizations in Alzheimer’s disease, malaria, multiple sclerosis, and tuberculosis.
In developing this resource, FasterCures created an evaluation tool to help measure the performance of such organizations, looking at everything from accountability to collaboration to overall contributions to the field. With a year into this effort, we’re taking another look at the framework to learn how we can make it an even more effective tool—not just for use by philanthropists looking to evaluate investment opportunities, but also for organizations seeking ways enhance their effectiveness.