#p4c2013

At the 2013 Partnering for Cures meeting, the panel “Reducing Drag: New Approaches to IP Negotiation and Technology Licensing” was moderated by FasterCures Senior Fellow Robert Cook-Deegan. He posed three questions to the panelists concerning IP negotiation: what would you like to fix, what do you not want to break, and what else should the audience hear. With a panel of voices from industry, patient groups, academia, consulting, and government, the answers were varied and highlighted many issues in IP negotiation faced by all.

Justin McCarthy, senior vice president and general counsel of Pfizer, began the conversation by describing the changing landscape of medical research and how this has an impact on IP negotiation. At Pfizer, there has been the realization that, “the days where a drug is wholly discovered, developed, manufactured, and commercialized within the four walls in one company is largely a thing of the past.” They have now moved toward models that involve more open approaches with a variety of institutions and look to collaborate in ways beyond the past fee-for-service model. These collaborative models have scientists working side by side to ensure that contributions are fair for everyone.

Josh Sommer, executive director of the Chordoma Foundation, has a unique point of view as an advocate for a disease with the potential for orphan drug status. For some companies, chordoma may be a path to market for drugs in their portfolio, but it must be an attractive path in order to get companies to invest. Many potential investors look for pre-clinical data to validate that their investment will be worthwhile. However, the system experiences a breakdown in getting these pre-clinical models to the companies. Academic institutions who own these models expect licensing fees, “reach through” rights, or other compensation, often causing negotiations to break down over time. Because of the difficulty of acquiring models and the collection costs, researchers will test on the limited models they already have access to, but often these sets are not representative of the entire disease. Testing in this way, as Sommer pointed out, is counterproductive from a scientific point of view and morally questionable from a patient perspective. “There’s nothing inventive about these models,” Sommer said. “They’re taken from human tumors donated by patients who are treated at these medical centers. They are not modified in any way; they’re simply sustained and they don’t embody intellectual property.” In addition, if patients do not demand royalties for the success of drugs from their clinical trial participation, why do academic centers expect royalties? Sommer’s solution for these issues is to treat all patient-derived models as a public resource, free to be used under the same terms by both commercial and academic institutions.

Voicing the concerns of academic institutions was Lita Nelsen, director of the Technology Licensing Office at the Massachusetts Institute of Technology (MIT). She was quick to point out that MIT already supports Sommer’s solution as all their mouse models are available non-exclusively for nonprofits and single payers alike. The difficulty in academia is in the translation of findings to inventions. “It is our experience that very, very seldom will an invention that comes out of our very fundamental research go directly to a large pharmaceutical company,” forcing researches to go the start-up company route in the biotech area. The other issue academics face is dealing with venture investors who will not invest in unrealistic agreements and often may want to take back the IP license after millions of dollars have been invested. There must be agreements in place that give investors reasonable assurance that their rights will not be taken from them in order for these collaborative efforts to be successful.

Many collaborative research efforts have been implemented with great success, as a collaboration among PATH, the U.S. Food and Drug Administration, and the National Institutes of Health (NIH) recently displayed. Steven Ferguson, deputy director of licensing and entrepreneurship in the Office of Technology Transfer at NIH, described how this collaboration was able to create a meningitis vaccine, which sold more than 100 million does in Africa and had a direct impact on the disease prevalence in areas where it was used. This work inspired the NIH to create a term sheet template for working with foundations and nonprofits who are working to commercialize technology, in an effort to ease many of the woes that would be faced. Ferguson emphasized the importance of flexibility that research institutions must have in order to work with nonprofits to take a product through to development.

Reiterating many of the themes mentioned by other panelists, David Clifford, founder of Avicenna, said that the future is in collaboration, not the current models. “Biology is very complex—it’s unlikely that we’re going to get to the next generation of IP discovery along the same mode that has taken us this far. It’s more likely that it’s going to be coalitions, consortia of people that are coming together to form new organizations or leveraging existing systems that really breed new generations of therapeutics.” Reevaluating the current models and asking for goals to allow for true partnering for cures is a space with many unanswered questions that need to be addressed.

Yet if that’s the case, then why is there so much uncertainty in the industry today? While investors are enjoying healthy returns, the industry needs to attract much more capital to deliver on its potential to save, extend, and improve lives. There are worrying signs that this isn’t happening, explained moderator Michael Milken, chairman of the Milken Institute and founder of FasterCures.

“The world’s economy is $72 trillion, but the revenue from biotech is less than $200 million,” said Milken. “The promise of the biotech/bioscience industry is so dramatic that it’s hard to believe how relatively small this industry is.”

To be sure, the recent recession created turmoil for the industry, which saw a heavy reduction in both private capital and government investments. But there’s something of a silver lining in the economic downturn that could help the industry, explained Robert Hugin, CEO and chairman of Celgene.

“One of the positive impacts of the Great Recession is that it pushed the need for integrated solutions and efficient investing,” said Hugin. “If companies aren’t able to make transformational change in patients’ lives, they won’t get investment.” In other words, the constriction of investment capital has focused the industry on providing solutions to some of the world’s most immediate challenges.

Richard Pops, chairman and CEO of Alkermes, advised biotech companies – particularly start-ups – to take a pragmatic view of the motivations of investors. “My sensibility is forged in the red-hot fires of investors, who don’t care about philanthropy,” he said. “They care about the rate of return.”

It’s a simple concept – that investors typically place financial return above social return – but for an industry known for long product cycles, it’s a crucial insight for solving the capital conundrum. Speaking of his own company, Celgene, Hugin said, “We raised a billion dollars before we ever made a dime. It took 15 years.”

And it’s not just the venture capital market that faces headwinds; philanthropic giving and government grants are on the wane, too. “Philanthropy is not the way to go in R&D,” said Christopher Egerton-Warburton, partner at Lion’s Head Global Partners. “Grants alone are not enough. There is a critical need to get more capital in this space.”

At least part of the solution, said Hugin, is that biotech companies need to start looking at building partnerships with their peers. “We had no choice but to collaborate, but it was the best business decision for us,” he said.

Milken echoed this point, encouraging the industry to pool its resources more effectively, particularly as it relates to finding cures. An important part of FasterCures’ work, he said, is “to do everything we can to strengthen the disease-specific network.”

The panel also covered two areas of great promise. First was crowd-sourcing, which can harness global intellectual and investment capital through the right combination of incentives and passion. Second was the increasing role of companies outside the health industry. Milken cited the $500 million initiative by Google founder Larry Page and its potential impact on the industry. Egerton-Warburton added that “Google can bring so much more than just cash” – it can also bring in human capital and provide vision and leadership for specific endeavors. In other words, the biotech community could benefit greatly from the experience and expertise of other sectors.

Stephen Ubl, president and CEO of AdvaMed, underlined the dramatic shifts in the ecosystem for innovation as a system “under duress, with a 40 percent decrease in venture capital for devices from 2007, at the same time as a 75 percent longer review time at the FDA.” He stated that this makes for a very difficult environment, particularly for startups with potentially the most innovative devices. He said that payment was equally difficult, and that the right quality questions were not being asked in the assessment of devices. For example, the test for hip replacements is whether the patient lives or dies from the procedure and if the patient is readmitted to the hospital within 30 days of the procedure, instead of asking who is the patient and how can medical technology improve his or her quality of life? Is the patient a 60-year-old athlete or an 85-year-old frail person? The panel agreed that quality standards must keep pace with scientific advances.

Robert Beall, president and CEO of the Cystic Fibrosis Foundation, discussed the importance of patient organizations in driving value considerations, with the recent example of CFF’s involvement with the development of Kalydeco, the first treatment that addressed the cause of the disease. He stated that patient groups have the ability to provide the data to the system on what outcomes are the most valuable to patients. Beall explained that “patient groups have a responsibility to develop data to show value of effective therapies,” not just for U.S. Food and Drug Administration (FDA) approval but also with patient-reported outcomes spanning years, and it is critical that patients have a seat at the table with payers and industry to contribute to value judgment decisions.

From the payer perspective, Jo Carol Hiatt, chair of the National Product Council at Kaiser Permanente, explained that there are areas for improvement in patient-reported outcomes, including in ensuring consistency, and in the fact that many outcomes are researcher-defined, instead of patient-defined. With their top concern being improved patient outcomes, she expressed her desire to have the patient community input to inform value decisions.

The sentiment was echoed by Shari Ling, deputy chief medical officer at the Centers for Medicare & Medicaid Services (CMS): “What those outcomes should be and what is meaningful, we can only know by involving patients in those conversations.” She explained that the goal at CMS is to drive higher quality for lower overall cost. 

Alan Russell, professor at Carnegie Mellon University and chief innovation officer and executive vice president of Allegheny Health Network, expressed his desire for more disruptive models for the innovation-to-adoption timeline, saying “We’ve been ‘almost there…three to five years off’ forever! It’s time to find the missing pieces that can accelerate the process.”

A recurring theme throughout the discussion was that the key to investing for impact in the medical research space is heavily dependent upon investment selection and the quality of due diligence. Nancy Brown, CEO of the American Heart Association (AHA), explained that for every major investment, the association sets up a team of experts to review and vet each opportunity. She also added that AHA has an oversight advisory group that ensures that the opportunities that are reviewed are aligned to the overall mission and vision of the organization.

George Weiss, founder of the Orphan Disease Pathway Project, pointed out that even though we are talking about philanthropy, there are many principles from business and investment in capital markets that can be extrapolated, including the necessity of high-quality due diligence and advisory groups. “As a businessman, I learned to pay attention to what doesn’t work, and this is one of the key transferrable principles that can have real impact in this space.” This principle is important because there is a lot of science out there that does not work or needs to be modified for higher impact. Weiss shared that he has come across a number of scientists that are afraid to change their research because they are afraid of losing their funding.

Weiss and others on the panel agreed that it is important to create a new incentive structure that will reward researchers for moving away from the safety of how they have conducted science throughout most of their careers toward cutting-edge research that could have a major impact on accelerating medical progress. Brown added, “We know that there are great ideas out there, but we also know that if we box in [researchers] too tight we could lose these great ideas.”

Brown went on to explain that once the good ideas have been identified and selected for investment, AHA has a robust evaluation system in place to measure impact. Using metrics such as milestones; timelines; follow-on funding; level of collaboration; and direct changes to standards of prevention, screening, and care, AHA is able to measure progress on specific outcomes as well as impact. Bob More, head of venture investing at the Bill & Melinda Gates Foundation, added, “Everything we do, we are measuring how those dollars are impacting the goal, which in our case is the impact that we are having on the incidence and prevalence of the disease that we target.”

In response to the question of whether there is a “one size fits all” approach to evaluation, Brown said, “Our evaluation is very methodical, thoughtful, and cutting-edge, but it is specific to the type of grant that we are looking at.”

I later asked the panel to comment on the role of venture capital strategies in philanthropy. Ronald Harrington, chairman of The Harrington Family Foundation and chairman of BioMotiv, shared his opinion that “the venture capital model is not working,” at least not as robustly as it does in other markets. Harrington described the innovative model that is The Harrington Project for Discovery & Development. This is a $250 million national initiative created to support the discovery and development of therapeutic breakthroughs by physician-scientists. This model integrates two nonprofit initiatives – The Harrington Discovery Institute and the Innovation Support Center – with a for-profit accelerator, BioMotiv. The nonprofit components are dedicated to supporting physician-scientists by providing funding, mentorship, business support, and other resources that will enable these researchers to transform their research into high-impact therapies that can significantly improve the lives of patients. BioMotiv uses innovative business models to accelerate commercialization of breakthrough medical discoveries supported by The Harrington Discovery Institute and the Innovation Support Center.

When asked about opportunities for return on philanthropic investments, Harrington explained that in The Harrington Project model there is an opportunity for return on investment (ROI), which is dictated by the performance of both the nonprofit and for-profit components of the initiative. Similar to The Harrington Project and many others, the AHA has also set up an accelerator program so that the organization can obtain a financial ROI, and they also use ROI from the accelerator as a performance metric.

There were a range of opinions displayed as the panel discussed financial returns on philanthropy. Weiss explained that he is not an opponent of the “evergreen model,” but shared his preference for setting up philanthropic organizations “where there is no profit mode and are just aiming to help people.” More commented, “I don’t think that a dollar is noble or not noble in its use… There is a role for both [for-profit and nonprofit initiatives].” Brown explained, “It’s not about making money for the sake of making money. Until these diseases are eradicated, our organizations are entirely devoted to investing every dollar that we have into the cause. Until these diseases are gone, we need more money to be able to fund more projects to be able to have a greater impact.”

More elegantly concluded the discussion on this topic by saying: “The paranoia about money is misplaced. [Instead] there should be more paranoia about accomplishing something. I tell organizations, ‘Don’t worry about making money; worry about doing what you do and the money will come,’ and it is the same for nonprofit organizations.”

I ended the panel by offering the audience three pithy takeaways:

Money doesn’t grow on trees

If you are not keeping score, then you are just practicing

At the 2013 Partnering for Cures panel “The New Value Proposition for Academic Science,” moderator Roxanne Khamsi, chief news editor of Nature Medicine, opened the conversation with a discussion on partnerships between academia and industry, and value expectations of both groups. Christopher Austin, director of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health, explained that NCATS not only serves as a catalyst for collaboration between academia and industry but also supplies an “adaptor language” to these groups who often use the same words but speak different languages. He said that in general both parties are interested in the value proposition of patient benefit; however, the challenge is that each group is also interested in a proximate outcome that is distinct from patient outcome. Academics are interested in tenure, publishing, and keeping the doors of their labs open by securing funding; whereas companies want to sell products that benefit patients while making a reasonable amount of money to satisfy shareholders. NCATS can adhere to these additional interests by providing funding for academic researchers to conduct translational research and proof-of-concept studies. Funding these types of high-risk, high-reward projects in the translational stage mitigates some of the risk, thereby attracting industry partners to projects that they would otherwise be apprehensive about pursuing.

Pearl Huang, vice president at GlaxoSmithKline, added her perspective on partnerships. She explained that the Discovery Partnerships with Academia program, which she heads, focuses on embarking on long-term, highly structured collaborations with academics with “very defined tasks and shared goals.” She agreed with Austin that bringing the two cultures together can be a challenge and welcomed that challenge: “There is nothing more exciting than when two people are trying to solve the same problem but going at it differently. The two will often disagree, but through that conflict and conflict resolution is where innovation occurs.”

Khamsi looked to Louis DeGennaro, executive vice president and chief mission officer of the Leukemia & Lymphoma Society (LLS), to provide a nonprofit perspective on the value proposition for academic science. Khamsi posed the question of how LLS compares two commonly used funding mechanisms – gifts/grants and sponsored research –in terms of extracting value. DeGennaro explained that there is higher expectation of accountability and interactivity when a nonprofit like LLS funds a project as sponsored research. It is more likely that there will be milestones and timelines in place. On the contrary, funding through gift streams allows for more degrees of freedom and in some cases additional creativity, but there is significantly less accountability. “LLS is focused on putting in place more projects that are structured and go through the sponsored research stream,” he said.

The only academic scientist on the panel, Conor Evans, assistant professor of Harvard Medical School, who benefits from both types of funding, added, “Sponsored research keeps us focused on the prize… Gifts give us the time to find and brand things that people may not have otherwise discovered on the other track.” He said that from a training perspective both mechanisms of funding are important in terms of value. He explained that in his lab, graduate students and post-docs that work on projects funded through either mechanism have the opportunity to learn about the demands and expectations of each type of funding from one another. “From a training standpoint and research standpoint, and to delivering, I think that having both together is a really important aspect,” said Evans.

Austin added to the discussion by pointing out that the funding mechanism that is most suitable for academic science really depends on the question that you are asking: “In the fundamental science it has to be supported by grants because you don’t know what you are looking for. You generally know what questions that you want to answer, but it is not as if you know specifically the direction that you are going.”

When asked specifically what role academic scientists should play in the changing landscape of drug discovery, there was a clear consensus among the panelists that academics should not assume the role of big pharma.

Jack Tillman, executive director of Emory Innovations at Emory University, said, “There is great opportunity in the potential for academic scientists in helping to commercialize therapies. They are great seeds of execution, but we need people who really understand and are immersed in the drug development process.” Evans agreed that it would be helpful for him as an academic to have a better understanding of regulatory expectations at the beginning of a project, rather than trying to retrofit the project once it reaches the commercialization pathway.

DeGennaro proposed that “academic scientists should enhance their understanding of drug development, but it should not supersede their primary focus of the science.” He went on to explain that LLS works to not only help educate scientists about the regulatory pathway but also to connect them with key nodes along the pathway, such as contract research organizations, that are necessary for U.S. Food and Drug Administration filings.

Overall, the panelists agreed that partnerships with academics are of growing importance as they continue to play a role in the clinical development research continuum; however, academic scientists cannot and should not replace the role of pharma. Instead, there needs to be a greater focus on educating academic scientists about drug development and regulatory science to ensure maximum value from innovation and technologies born from academic labs. According to Huang, “You need 50 skill sets minimal to get from idea to medicine.” Thus, diverse partnerships are important, as they are what can truly catalyze innovation. It is critical for all stakeholders to respect cultural differences among partners and have mechanisms in place to bridge the cultural divides that can often lead to miscommunication.

John Walsh, president and CEO of the Alpha-1 Foundation, shared how his work did not stop with funding research, creating patient registries, and designing clinical trials. The foundation was also steeped in working closely with payers to make a strong case for the value proposition to ensure coverage of therapies in the pipeline. In addition, he talked about how his patient-driven organization also worked closely with specialty medical groups to draft guidelines for effective delivery of care.

This holistic approach, he said, is driven by meaningful data from patients and about patients.

At Cincinnati Children's Hospital Medical Center, Peter Margolis, a professor of pediatrics and director of research, echoed the need for quality data. Better outcomes, he noted, can come from systemic improvements not just transformative outputs. We have to “act our way into a new system,” he said, not just engage in “an analytical exercise” but instead “a concerted effort to use data and bring together stakeholders.”

There’s “amazing power of right networks coming together,” said Ben Heywood, co-founder and president of PatientsLikeMe. There’s real opportunity to measure and understand unmet needs of patients in the “context of me” and then take these insights and present it scientifically to inform both research and care delivery.

Studying the range of outcomes that matters to patients is the raison d’etre of the Patient-Centered Outcomes Research Institute. Its executive director, Joe V. Selby, said that once a range of outcomes are collected, we need tools to present this in ways that clinicians can use. “We need to compare effectiveness of therapies and care delivery approaches, but these data should be comparisons that matter to patients,” he said.

The institute has approved more than $303 million since 2012 to support patient-centered comparative effectiveness research and is on track to commit more than $400 million in research support in 2013.

Mark Wagar, president of Heritage Medical Systems, said that payers and providers are paying close attention to data and to best practices (like that of Alpha 1 and Cincinnati Children’s). “If there is anything that really works that improves outcomes and lowers readmissions, we want it tomorrow,” said Wagar.

The FasterCures Consortia-pedia project continues to unearth a menu of objectives that these consortia are pursuing, and surprisingly, has so far found very little overlap among its growing list of 350 consortia. Not only does this exemplify the multiple opportunities for collaboration, but it also serves as evidence that collaboration is no longer an option, but a necessity.

Panelists shared on-the-ground insight during a discussion at Partnering for Cures on “The Art and Science of Multi-stakeholder Collaboration,” featuring panelists representing different stakeholders who have initiated, managed, and participated in consortium activities, and moderated by Beth Meagher, director, Strategy & Operations, Deloitte Consulting LLP. All of the panelists agreed that there is a science and process for making these partnerships successful, but there are also over-arching challenges.

If there were a playbook for starting a consortium, the panelists voiced the following steps:

Define the objectives

You first need to “identify the key problem worth solving,” said Maria Freire, president, Foundation for the NIH (FNIH). Pinpointing a problem that multiple organizations share helps  to define the common goal and intent of the collaboration.

“Start with the end-in-mind as the focus,” advised Walter Capone, chief operating officer of the Multiple Myeloma Research Foundation. “Focus on collective interests, not individual interests. For us, [the focus is] the patient.”

Gigi Hirsch, executive director of the Center for Biomedical Innovation at MIT, urged the audience to make sure the objective is “unique, not a duplication of other efforts” and think about “formal or informal links with other collaborations” if there are similar efforts.

In starting a consortia, Michael Rosenblatt, executive vice president and chief medical officer of Merck, advised the audience to “focus on innovation.”

Develop and pursue a research strategy

“You need the right people,” said Hirsch. Include representatives that know how to align the consortium’s research objectives with the external and internal priorities of their own organization. And, reward those who participate in consortium activities so that the partnership isn’t a “volunteer hobby.” Rosenblatt added that it is important to make sure that the right people “have the skill sets that they want to use” so that they know they are contributing to the partnership.

Freire emphasized the importance of identifying a neutral convener who will be able to help the group develop milestones, achieve consensus, and create a nimble infrastructure. Hirsch added that this infrastructure should be truly collaborative and well-networked.

When developing a consortia, Capone said that identifying the “success” factor that can be measured during, and at the end, of the collaboration is vital from the beginning.

Address the challenges

Conceptualizing a partnership is easy, but putting it into practice has some challenges. There’s the perception of “consortium-fatigue,” which James C. Greenwood, president & CEO of the Biotechnology Industry Organization, attributed to a “lack of coordination and too much redundancy.” Greenwood added that there’s a need to educate stakeholders about these activities and to also include government and Congress, as these latter groups can further incentivize these models of collaboration. As detailed in the FasterCures Consortia-pedia project, the largest third-party biomedical consortium conveners in the United States – FNIH, Critical Path Institute, and Reagan-Udall Foundation for the U.S. Food and Drug Administration – were all created under government or Congressional direction.

Another point of disagreement among many consortia members is ownership of the consortium’s products. Capone stated that each stakeholder needs to “give up a little bit of IP,” as the partnership should be thought of as an investment in a shared “resource for research.”

As resources continue to decline, research-by-consortium will increasingly be the model of collaboration needed to protect the pipeline of cures for patients who need them the most. The Consortia-pedia project aims to help those seeking strategies for collaboration by providing a framework for consortium management, and highlighting the output and outcomes from existing collaborations. It is indeed a science, not an art.

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As prices climb and common conditions are divided into “rare” subtypes by new testing paradigms, concerns emerge about the sustainability of these patterns. A panel at the 2013 Partnering for Cures meeting convened to discuss this topic. Moderator Matthew Herper, senior editor of Forbes magazine, launched the discussion with a question for Matthew Perry, portfolio manager for venture capital firm BVF Partners L.P.: “What has made the companies developing orphan products so appealing?” Perry commented on both the economic and humanitarian benefits. “It’s been great for investors. Historically the cost of treating patients with rare conditions has been so high and their prognosis so poor that you could raise funds with the promise of changing lives. It has opened the whole field in a great way.”

Sharon Hesterlee, vice president for research at Parent Project Muscular Dystrophy (PPMD), agreed. “For many years, we couldn’t get in the door with our request on behalf of boys whose lives were cut short by Duchenne muscular dystrophy. Genzyme paved the way for interacting directly with patients with rare disorders. Patient engagement is especially important in areas where companies don’t have access to patients or clinical experts. Organizations like ours also de-risk development of drugs by providing direct funding and natural history data. Hopefully these supports can help bring costs down.”

J. Russell Teagarden, senior vice president for medical and scientific affairs at the National Organization for Rare Disorders, expressed the need for the next generation of orphan drug legislation to address access issues. “It’s a cruel hoax if these new therapies are out there but patients can’t afford to access them. We need an inventive way to deal with cost structures because payers large and small are shifting costs to the individual. It’s not sustainable the way it is now.”

Two pharmaceutical company executives weighed in. “The real issue is that most rare disorders will not have a ‘magic bullet’ therapy,” said Eugene Williams, CEO of DART Therapeutics. “We have to think about multiple therapies to change the course of a disease. We have to smooth the pathway, lower the costs, and integrate the needs of biotech with patients.” Alexion Pharmaceuticals’ vice president for global clinical operations, Mike Collins, expanded on the need to become more efficient. “There’s currently an arms race to collect more and more data, which we need to scale back. Patient groups helping us optimize our clinical programs and provide content expertise can cut time and costs. Serendipity helps, too.”

Panelists shared frustration with the path to marketing approval not always being clear in rare disease because many conditions are not well understood, and the right clinical endpoints to demonstrate effectiveness can be elusive. Hesterlee talked about the value of PPMD’s patient registry. “We realized that we could never have enough natural history data about patients with Duchenne. Changes in standard of care therapy changed the natural history of the disease. We recognized that we had to keep up with those changes.” Teagarden emphasized the need to include “day in the life” illness experience measures in natural history data collection. “When I worked at Medco and had to review data about new therapies, I remember receiving some bizarre measures for treatment effectiveness. Measures based on what’s important day-to-day to patients will help payers allocate resources more effectively.”

The tension between regulatory and reimbursement decisions came up several times in this panel’s discussion and at other Partnering for Cures sessions. The panelists discussed the current environment trending toward development in a small, narrowly defined population and then expanding to see who else might benefit. Perry emphasized the appeal to investors: “A little bit of money in rare disorders might yield big advances. By testing with a small number of patients, they can test a hypothesis.”

The ability to link data sets between different disease states was key. Herper asked if the expansion of markets beyond the original patient population intended for a product might lead to push-back from insurers. Teagarden nodded vigorously. “Suspicions about pricing have less to do with the primary disease and are generally rooted in what will happen if a treatment gets beyond that population. While few payers strictly limit access based on the labeled indication, cost is a factor in how much flexibility there is. You’ll see more paperwork requirements to secure payment authorization as prices rise and clinical use expands.”