Look Who's Talking at Partnering for Cures 2013

Remember to visit us on our new blog: www.fastercures.tumblr.com Partnering for Cures is a meeting focused on innovation, science, and collaboration. It is a platform for big ideas, transformative programs, and tangible results brought forth by some of the most disruptive innovators of our time. A shared sense of urgency binds together meeting participants, because we all know that when it comes to the search for cures, "soon" is just not good enough. JOIN US!  Look who's talking at #P4C2013 Confirmed speakers »  Panel topics »   Partnering for Cures emphasized the urgency that time is lives... We have to be willing to approach [challenges] in a different way to get solutions."  – Academic scientist   It's a place to challenge conventional thinking."   – Start-up company executive   [Partnering for Cures] is the biggest gathering of patient advocacy groups, biotech companies, pharma companies, and regulators."   – Xconomy Have you registered yet?   http://www.partneringforcures.org »

Evaluating “Value”: How Do We Foster Innovation in Today’s Cost-Conscious Healthcare Environment?

Two out of three participants in a recent FasterCures workshop believe that patients’ needs and priorities are not driving decisions made by payers, innovators, and providers. To have a stronger grasp on the role the patient can and must play in reimbursement decisions, FasterCures and the Cystic Fibrosis Foundation convened a one-day multi-stakeholder workshop earlier this month titled, “Value and Innovation: What Will the New Day Look Like for Patients?”

Fifty-three leaders participated from across the four Ps – venture philanthropy patientorganizations from TRAIN (the Research Acceleration and Innovation Network), biotechnology and pharmaceutical company executives, payers (public and private), and provider organizations.

Anchoring the day-long conversation, the first of five sessions focused on the stakes for patients in a cost-constrained environment, and how patients and their advocates can help shape the dialogue and decisions about who will pay for development of new treatments and rising healthcare costs. An overview of the Affordable Care Act followed, providing important context to the conversation about curbing health care costs. Next, the group focused on resources and infrastructure for research being created by innovative patient foundations – and how these groups are doing more and more to transform both the path toward cure and care for their disease. The fourth session looked into the types and sources of evidence payers use and are looking for to make informed reimbursement decisions. Finally, participants dug into the meaning of the term “value” and how new payment and delivery models are driving the definition of value and affecting innovation.

Five themes surfaced during the dynamic discussion:

1. Patient-relevant outcomes will ultimately drive the definition of value: Patient engagement in all stages of drug and device research and development, care delivery and payment decisions is vital. Determining and measuring outcomes that are important to patients is an emerging area of research and practice that will require experimentation and refinement.
2. Innovators and patients need to understand the evidentiary needs of payers: Defining what evidence is needed and collecting it at the earliest possible point can help drive toward a shared understanding of value across all stakeholder groups. High quality evidence is needed to make regulatory and payment decisions and will ultimately be generated through both traditional and novel study designs and analytical methods.
3. Better data is needed to make better decisions: A lot of data and information is being collected and stored from multiple sources across multiple platforms. Agreeing on standards and facilitating stronger interoperability would enable sample sizes to grow and better evidence to be harvested for the benefit of all stakeholders.
4. All stakeholders agree that it’s imperative to remove waste from the healthcare system: Up to 50 percent of healthcare delivered is of uncertain value and this uncertainty impacts all stakeholders. Cost pressures can force payers into the uncomfortable role of being “watchdogs” for quality and the evidence available to make decisions is uneven and may be applied inconsistently by different payers in the system.
5. The concept of value is derived from the total cost of a patient’s care, not the cost of an individual product: There is a strong need to unite sources of information about costs of care delivered in different settings by different providers, including family caregivers, and to identify means to calculate savings achieved through prevention and avoidance of harms. Costs of individual products are important but not sufficient to inform decisions about effective care to achieve better health outcomes for the individual and the broader population.

The call to cut waste out of the health care system and streamline the process of finding, delivering, and paying for the best possible therapy was a resounding theme.  Patient groups may also find themselves in the unfamiliar position of having to advocate for reducing inefficient treatments and care for their own patients out of the system.

Participants concurred that innovation and value aren’t mutually exclusive concepts and in fact need to be mutually reinforcing. Balancing the real-world competitive market factors for innovators and payers while striving to meet patient needs will require transparency, flexibility, communication, and – above all – trust within and between stakeholder groups.

These are issues of growing concern to many organizations with diverse constituencies and you’ll be hearing more about them. FasterCures will issue a detailed report on the workshop and its outcomes and an upcoming blog post will focus on recommendations generated at the workshop.

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

FasterCures and Friends of Cancer Research Host Capitol Hill Briefing

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

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

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

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

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

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

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

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

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

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

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

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

* A video of this briefing is now available.

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

Funding for Translational Research at NIH

This month, The National Institutes of Health will be accepting its first round of applications for a new funding opportunity focused on supporting collaborative translational research projects.  First announced last fall, this U01 research program – Opportunities for Collaborative Research at the NIH Clinical Center – is designed to provide extramural (non-NIH) investigators a mechanism to establish collaborations with NIH intramural investigators and to take advantage of the unique research opportunities available at the NIH Clinical Center.

It is being offered by 12 NIH institutions and is intended to facilitate the translation of basic biological discoveries into therapeutic candidates for clinical testing.

With the creation of this funding opportunity and the establishment of NIH’s newest Center, NCATS, NIH continues to push forward its vision to transform the translational process for the benefit of patients. Recognizing the critical advancements that have been made in the understanding of basic disease biology, the agency continues to look for ways to infuse new and innovative thinking into not only the scientific discovery process, but also the process of getting important new medicines from discovery to patient.

We’re excited to see the increased focus on collaboration – for entry to the program, extramural research projects must have a collaborating investigator in the NIH Intramural Program as well as a “Collaboration Plan” – and eagerly await the first crop of awardees.

Fully utilizing the NIH Clinical Center was one of the recommendations that FasterCures had put forward in 2008 through its blue-ribbon task force focused on the NIH Intramural Research Program led by Nobel Laureate and FasterCures board member Dr. David Baltimore. In fact, of the five recommendations the task force presented, three have been acted on, with outcomes that will save lives by shortening the time it takes to turn breakthroughs into medical solutions.

To learn more about the application process for this grant program, watch the pre-application webinar or visit the official announcement page.

Resources:
Sequestration Station – Check out FasterCures’ Sequestration Station for more information on how sequestration will affect the National Institutes of Health.

Top 10 Medical Research Trends to Watch in 2013

By Margaret Anderson, Executive Director of FasterCures

As seen in The Huffington Post

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

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

2. FDA: Recalibrating the benefit-risk equation. 

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

Saying thank you to FDA's Janet Woodcock

3. Financial innovation: Attack of the megafund. 

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

4. Reimbursement: Can value drive innovation? 

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

5. Venture philanthropy: Playing in the major leagues.

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

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

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

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

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

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

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

9. Collaboration: Writing the playbook. 

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

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

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

Using IP Strategies to Break Down Barriers to Progress

Intellectual property (IP) protection is critical to driving investment in new products that can help patients as well as create economic value. Some maintain that IP protection can make scientific collaboration, technology transfer, and commercialization more challenging. At Partnering for Cures, five leaders in biotechnology, science, and intellectual property joined together to discuss how IP could aid the pace of innovation and technology to get to patients who most need it.

Moderator Maria Freire of the Foundation for the NIH began by pointing out that some people “go into panic attacks when having to deal with intellectual property … it seems like a big hurdle between basic science research done in universities or companies and getting it to the market.” However, this is not a new issue. The biggest change is that, within the last 10 years, new players have emerged: venture philanthropies. Freire referenced FasterCures’ newest publication, Unlocking Intellectual Property: Principles for Responsible Negotiation, which serves as a useful tool for all parties in biomedical research, in particular these new nonprofit disease groups and philanthropists.

In addition to the arrival of new players, Stephen Johnson of Kirkland & Ellis LLP and One Mind for Research emphasized that the IP landscape has changed due to the arrival of new technology. In the past, IP focus was on patents, but the “focus has moved away from patents and toward data,” he said. He used to see resistance to sharing data among pharmaceutical and biotechnology companies. Now, “there is acceptance of the pre-competitive space,” and companies increasingly embrace the opportunity to work together. “The future of creative collaborations will be balancing openness with incentives,” he suggested. Freire summarized that companies are more willing to share all data in the beginning, but once there is true innovation, they will then put protection around it. Johnson agreed with her assessment and advocated for processes that promote transparency. He said that one of the reasons that companies would be reluctant to share data is that they are worried that someone else who has access to the data is smarter than them.

Stephen Friend of Sage Bionetworks gave an example of another hindrance, stating that “many companies don’t feel like they can share what they’re talking about … and it’s hard to get things financed without a clear IP strategy. Many worry that someone else could come in, grab the idea, and take advantage of them.” He supported extending the pre-competitive space: “Too many post-docs think they are working on the next billion-dollar drug long before it is.” He cited the successful collaboration of Merck, Pfizer, and Lilly, who generated data in China and agreed that they would share the data among themselves for one year and then make the data publicly available.

Teresa Stanek Rea said that the U.S. Patent and Trademark Office also has a cooperative approach: “We are trying to collaborate with companies to find out what they need to do their job.” Like industry, the Patent and Trademark Office is trying to be more precise and more efficient in what they do and sees itself as the innovation agency in the U.S. government. “We are an agency in the throes of change, just as you are,” she said. Rea noted that the America Invents Act, puts forth “great provisions that help the user community because it takes a second look at issued patents, and whether the patent should have been granted.” Rea believes that the act should minimize litigation and not inhibit research.

Steven Tregay of FORMA Therapeutics brought the conversation back to venture philanthropies and emphasized the importance of being “cognizant of whether the patent that covers the product can actually be translated into treatment.” The key is helping people understand the value for society versus owning one possible combination that may be pertinent or may never be turned into a drug. He cited the success of the CoMMpass project of the Multiple Myeloma Research Foundation, a collaborative study that brought together a network to decide how IP will be shared and who has access to it. Tregay has also worked with the Leukemia & Lymphoma Society (LLS), which aggregates data from many organizations at once. LLS is concerned with creating value to patients and creating a path forward, and it shouldn’t be burdened with the “nightmare of bringing universities together,” he said. “That is the power of these disease foundations – that they are really laser sharp in their approach,” said Freire. “They don’t want to fix the world; they want to fix something for that indication. The traditional paradigm may not necessarily apply.”

Friend agreed that traditional ways of doing business may stymie dialogue and interaction. “We are at a spectacular time where we have tools, new approaches in order to innovate, and yet the way we have structured our incentives and our rewards around sharing, around who is getting credit, et cetera, is basically independent to that.” He cited the success of CommonMind, a public-private pre-competitive consortium that generates and analyzes large-scale genomic data from human subjects with neuropsychiatric disease and makes the data and results broadly available to the public. Friend said that university tech-transfer offices had a hard time at first agreeing to share data being generated, but that “the data required to build the models needed to develop the drugs had to be accessible in order to innovate.” The parties created collaboration agreements that allowed investigator data to be shared with others and not kept to themselves. “We must come up with incentives and rewards that allow the data to get out there,” he said.

Joseph DeSimone of the Frank Hawkins Kenan Institute of Private Enterprise at the University of North Carolina agreed that “partnerships are what work the best, and new connections should always be made,” but cautioned that “without really clear IP, it’s getting increasingly hard to get things financed. Having a really clear IP strategy and path to market is going to be increasingly important.” His university has a conflict of interest committee that meets with him and his students who start companies. He believes that transparency of partnerships makes it more successful: “If you are open to that kind of openness, it can be powerful to enable these kinds of connections.”

In closing, the panelists discussed the future of intellectual property protection with regards to innovation. There was a consensus that patenting had gotten more difficult, and Freire ended by saying “Let’s not rediscover wheels. If you can put something in a box, it’s a lot easier. If you want to think outside the box, just make sure that what you have already doesn’t fit in an existing box.”

Related resource:
Unlocking Intellectual Property: Principles for Responsible Negotiation

Improving patient outcomes through technology

We hear a lot these days about how data sharing and collaboration have great potential to reduce the cost of healthcare and improve outcomes for patients – but who is actually doing it? What is at stake, what are the barriers, and what are potentially scalable solutions?

A Partnering for Cures panel focused on a case example of a patient-centric demonstration project within the ImproveCareNow Network, which includes 44 pediatric gastrointestinal care centers and more than 13,000 patients, and has succeeded in improving remission rates by sharing best practices among care teams across sites. Moderator Dominique Pahud of the Ewing Marion Kauffman Foundation introduced the goals, design, and participants leading the technology intervention.

The project features a collaboration among entrepreneurs, patients, physicians, designers, and researchers to provide a backbone for pediatric gastrointestinal care centers that delivers a full integration of technology-based solutions, including electronic health records, passive monitoring, and patient-recorded outcomes. The collaboration is employing a suite of technology interventions at eight different centers within the ImproveCareNow Network, representing 2,400 patients.

Each panelist discussed his or her contributions to the collaboration, with Richard Colletti of the University of Vermont School of Medicine introducing the operations and goals. Colletti highlighted that the remission rate of pediatric gastrointestinal patients increased from 50 percent to 75 percent since the establishment of the network in 2007, noting that “if we had done this with a new drug, it would be a drug that everyone would want to use.”

Peter Margolis of the University of Cincinnati School of Medicine discussed the founding of the network and the establishment of the revenue model, which relies on care centers paying to participate. He also highlighted the collaborative learning system of the network, which is based on a “steal shamelessly, share seamlessly” ethos.

Designers that were involved in creating the technology backbone also spoke on the panel, including John Chaffins of Lybba. Chaffins described the method that Lybba used to design apps, which was based on avatars that were used to solidify the concept of a “patient,” which he said “became a tool for thinking about the kind of design choices you are making … and establishing a common language” in the collaboration.

Anmol Madan of Ginger.io addressed his biggest challenges in being a health-focused entrepreneur, such as getting access to healthcare providers in order to understand their technology needs. He also commented that “the biggest value [of participating in the collaboration] was getting the validation we need to go from an interesting technology to a commercial product.”

Finally, John Wilbanks of the Ewing Marion Kauffman Foundation returned the conversation to the 10,000-foot level to discuss the challenges and opportunities for collaboration among multiple players in the biomedical space. As the developer of the intellectual property framework for the collaboration, he spoke of the need for an organizational structure that makes collaboration possible, including a “framework that allows for the creation of both private and public value.”

The panelists highlighted a range of organizational incentives for joining the project, and invited the audience to join the movement toward open, value-adding collaboration.

Fulfilling the promise of bioscience

Bioscience is facing a Sputnik moment, according to Michael Milken, founder of FasterCures, as he opened the first session of the Partnering for Cures conference. The 1957 launch of Sputnik spurred the United States to focus on science and technology. Today, dramatic advances in genomics and disease research have brought the scientific community to the threshold of new discoveries, and he challenged the panelists in the Partnering for Cures opening session, “The (Near) Future of Bioscience and Health,” to explore what needs to change in bioscience in order to fulfill the promise.

Increased collaboration among stakeholders who don’t normally work together was seen by all to be a crucial factor in the future success of biomedical science. The U.S. Food and Drug Administration’s (FDA) Vicki Seyfert-Margolis said her agency is not just involved in regulation; rather, its role is to promote and drive innovation. She envisions a triangle composed of basic research, product development, and the FDA. “Information should move seamlessly between them,” she said.

Pfizer has been pursuing new collaborations as well, reported Mikael Dolsten, president of its worldwide research and development. “There has been a climate change in relation to regulatory agencies and academic partners,” he said. “I have been enthusiastic to see how many academic medical institutions were willing to take a new look at how to work together, work like one team to address different ways to treat disease,” he said.

Pfizer collaborated with the Cystic Fibrosis Foundation, Dolsten said, to define together a research plan to identify new molecules that will translate into successful compounds for patients with the disease. His company also has partnered with the Lupus Foundation in a similar way. If companies like Pfizer build on this, and others in the bioscience ecosystem see their success, he said, it will bring new interest into these partnerships.

New stakeholders and new voices must also be brought into the process, the panelists agreed. Risa Lavizzo-Mourey with the Robert Wood Johnson Foundation pointed to research conducted at the University of Miami into whether babies in neonatal units feel pain. Neuroscientists partnered with engineers to identify physical manifestations of pain. “The team brings the right kind of innovation to the table,” she said. “We don’t recognize all of the perspectives that we need.” Along those lines, she also identified the nurse scientist as a stakeholder who can help drive innovation, since these individuals are often closer to the patients and more familiar with their needs.

Funding is a problem at all levels. Milken noted that the FDA continues to be underfunded, even as the rest of the world falls back on the FDA for rulings on new drugs and devices when they run into problems in clinical trials. And with venture capital investments into the “valley of death” decreasing, patient collaboration becomes that much more important.

For Jay Schnitzer of the Defense Advanced Research Projects Agency, embracing risk is another key to success in bioscience. He said the field today is too risk averse. “We have to be willing to fail, to fail often, to know we fail and move on,” he said.

The decoding of the human genome is part of a “sea change” in biosciences, said Kenneth Davis of the Mt. Sinai Medical Center. Now, when some patients respond to a treatment, health records can be superimposed with genomic data to identify responders and find others with the same genomic makeup who can be used to test whether a new treatment will be effective. This precision medicine approach is being used with cancer research now, he said, and if more people contribute to biobanks and get behind genomics, he hopes more can be done with brain diseases.

A basic problem in healthcare today, Milken said, is that we spend more resources treating diseases than preventing them. Davis noted that Mt. Sinai is a fee-for-service medical system where payment is not keyed to prevention. But that is changing, he added: bundled payments, accountable care organizations, and other innovations are moving the U. S. healthcare system away from this fee-for-service approach.

In his conclusion, Milken cited notable medical advances. He noted that science averted a gloomy prediction of how widespread polio would be, and even in just a few decades, mobilized efforts have resulted in great strides in AIDS research and treatment. While the challenges are great in the future of bioscience and healthcare, Milken said that he has “a tremendous sense of optimism about the future.”

Are you ready? Partnering for Cures is here! Have you: • Updated your P4C Connect Profile? • Scheduled your partnering meetings with other attendees? • Set up sessions with leading experts from across the medical research field available for free, private one-on-one meetings? • Decided which presentations to attend & built your customized P4C schedule in your calendar of choice? Remember to download the Partnering for Cures partnering system P4C Connect app today! The partnering system also allows you to request free, private, 25-minute advice sessions on: Strategic Planning  •  Attracting Capital  •  Collaboration Structuring Interacting with the FDA  •  Communications& Marketing P4C Connect is accessible via your iPhone, iPad, or Android Device. You can download it from the iTunes App Store, Google Play Store, or scan the QR codes here. Android iPhone Remember to keep up with the conversation on twitter before and during the event with the hashtag #P4C2012

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”