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7 Toward Developing a Cultural, Legal, and Behavioral Framework for Precompetitive Collaboration key Points Raised by Speakers · Diminishing returns in drug development are creating powerful forces for change. · Establishing IP-free zones would open new areas of R&D to precompetitive collaboration. · Leadership from both NIH and the pharmaceutical industry will be essential to drive needed institutional and cultural changes. “Precompetitive collaboration is not a new concept,” said Stephen Eck of Eli Lilly and Company. “It’s happening, and it delivers results. So the real question is how can we make that happen on a larger scale?” FACING THE PRObLEMS William Spencer urged a continuing focus on the basic problem: the number of drugs coming out of the pharmaceutical industry is declining while research costs are going up. It is taking too long to develop drugs and R&D costs are not covering new drug delivery. According to Garry Neil, the high costs of drug development—between $1.2 billion and $1.8 billion per drug—are being driven by biological complexity and by the high rate of failure during development. Only one out of ten drugs pays back its devel- opment costs, he said. Viewed in that light, some of the issues discussed 49

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50 ESTABLISHING PRECOMPETITIVE COLLABORATIONS at the workshop are of less importance, Spencer said. “When the surgeon walks in the room and says ‘It’s malignant,’ you don’t give a damn about privacy at that point. [The patient only cares about] how are you going to solve that problem for me?” Spencer said that a leader from the pharmaceutical industry needs to stand up and say, “We’ve got an issue.” The NIH needs to play a role similar to that played by the Department of Defense with SEMATECH, he continued. The industry needs to convince the federal government that it will get products into the market faster and with less expense through a cooperative effort between government and industry. With universities, the desire to protect IP was solved in the SEMATECH case by sending them money. The important issue is to move from basic discoveries to something that will help patients, said Spencer, and “patents in that process are not very important.” Neil cited as major problems an inadequate clinical research infrastruc- ture and antiquated regulatory science. Attention needs to be devoted to creating the right infrastructure to move faster on the clinical side. Addi- tionally, there needs to be a refocusing of research efforts in human systems as “animal models are very imperfect examples of human biology, let alone human disease.” Sharon Terry, president and CEO of Genetic Alliance, expressed the opinion that a crisis is needed to drive the necessary changes, because change is difficult. The current crisis is that “the public understands that we’ve spent a lot of money on research, and although it’s a tiny amount of money when we look at other industries . . . we don’t see a lot of results. We might have hyped things in earlier years without meaning or wanting to, and this stuff takes a long time.” In order to move forward and develop products, the shared understanding of where and what people can make money on needs to change, she said, which is hard for companies, for aca- demia, and even for not-for-profit foundations such as the one she heads. A lot of issues facing the field today are transactional in nature, said Geoff Ginsburg. When potential collaborative agreements enter into the legal department, the process slows noticeably. There is a disconnect which needs to be addressed where IP protections can be overvalued while the value of the collaboration itself may be overlooked. Ginsburg supported the idea that studies of disease biology and biospecimens should be IP-free. “There’s nothing in a biospecimen that is worth patenting. It’s what you do with them,” he said. “If we could lay down ground rules and say getting specimens and data out into collaboration or to the public domain is not an IP event, to me that changes the dynamics of the transaction.” Terry also called attention to the cultural dimensions of change, which means putting new incentives in place.

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51 CULTURAL, LEGAL, AND BEHAVIORAL FRAMEWORK DRAWING THE LINE bETWEEN PRECOMPETITIvE AND COMPETITIvE EFFORTS In drawing the line between precompetitive and competitive R&D, Eck pointed out that one possibility is for everything to be precompetitive before the point of a clinical proof of concept. Another possibility is that even establishing the proof of concept could be a precompetitive and col- laborative activity. Several speakers spoke in favor of this idea, with Kelly Edwards noting that “we all want to accelerate science, we all want to get to health impact, and we all are struggling with the accrual problem, the patient recruitment problem, we’re all struggling with funding. So why not include clinical proof of concept?” Eck pointed out, though, that if the proof of concept succeeded, the drug would immediately become generic with no one to pay for phase III development. Martin Yuille observed that the Innovative Medicines Initiative (IMI) in Europe has an operational way of defining the boundary between pre- competitive and competitive. People from different companies formulate particular research topics, which then generate responses from academic consortia to work with industry on the topics. Issues such as IP protections are handled by the industry group, so they are no longer issues by the time the collaboration begins. Sally John agreed that the IMI model has advantages, because industry can collaborate on what the bottlenecks are and make in-kind contributions to a project, which enables it to be a much more active participant. However some of the calls for precompetitive collaborations have been less successful than others, and some issues still need to be overcome as a collaboration proceeds. As Aled Edwards pointed out, this would include reevaluating the prohibition of an applicant sitting down to develop the plan in conjunction with the potential collaborating industry. Yuille responded by saying that the IMI is modifying its procedures on the basis of previous experiences, so that problems that have arisen are trying to be corrected. “It’s an ongoing process of trying to improve what you’re doing.” Stephen Friend asked specifically what needs to be changed to move the boundary between precompetitive and competitive R&D. Are the issues legal, ethical, economic, institutional, or cultural? “How do we exit the world that we’re living in and get to this world that we might want to live in?” Clayton emphasized that other areas of biology have already made this transition. The Human Genome Project and the Human HapMap Project both had to negotiate the terms of what was precompetitive and what was competitive. “It’s a good idea to learn from past experience.” It also helps to have strong leaders, she said.

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52 ESTABLISHING PRECOMPETITIVE COLLABORATIONS MOvING FORWARD Neil said that leadership has to come from many sources: policy mak- ers, the patient advocacy community, the FDA, Health and Human Services, the Centers for Medicare and Medicaid Services, and other regulatory agencies. “All these people need to be at the table to talk about how we’re really going to get after this. Because this really matters. It matters to public health, it matters to issues of access and affordability of care, . . . an aging population that’s going to need more of what we can deliver, and how we’re going to be able to continue to be competitive as a country.” The challenge with drug development, said Terry, is to move collabora- tion to the point that it is dealing with products that could change lives. This requires leadership, by both NIH and the pharmaceutical industry. She suggested that “it has to be NIH, with probably a pharma leader who is visionary and willing to risk a great deal, standing up and saying, ‘We are going to change the culture.’” A participant noted that “if NIH and [the pharmaceutical industry] got together . . . the academic research enterprise will follow.” Thomas Insel challenged the Roundtable on Translating Genomic- Based Research for Health to create a vision that can be shared by all stakeholder groups, including patient groups. Many resources can be shared beyond data. For example, “there are lots of wonderful small molecules, and lots of drugs that people try that don’t work, that pharma has on the shelf. . . . Often it’s because they have some kind of off-target indications, but their off-target indication may be exactly the on-target indication for somebody somewhere else.” Enabling the sharing of other kinds of resources could allow the development of a “safe haven” and a different culture which could then forge a path for more controversial items. Eck agreed and pointed out that in-house software tools may be easier to make available because there are fewer obstacles to overcome in order to do so. “There’s no competitive disadvantage to us in making these tools widely available,” he said. SHARING bIOSPECIMENS AND DATA To enable the sharing of biospecimens and data in a precompetitive manner, several potential paths were espoused by participants. Terry sug- gested that it may be necessary to have NIH make it a requirement of aca- demic investigators to deposit these items into a public commons. Edwards suggested as an alternative, the negotiation of what will be shared on a project by project basis. Eck pointed out that a neutral third party may be required to decide which materials should be open access and which should remain under wraps. John said that PIs should have a say over what hap-

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53 CULTURAL, LEGAL, AND BEHAVIORAL FRAMEWORK pens to their samples. “The people who collected the samples, who have done all the work, who collected all the baseline data, they are the people who will be best able to interpret results.” Ellen Wright Clayton reminded the group that “one of the things that needs to change is the idea that what’s yours is mine and what’s mine is not yours.” People have a tendency to say that they “own” certain objects or ideas, but a better model than an owner- ship model, according to Clayton, is a stewardship model in which institu- tions have responsibilities toward samples and the donors of those samples. Faculty members and their students are stakeholders in these decisions, Frazier said. They have to benefit for them to accept whatever model is adopted. Kelly Edwards agreed, saying that “it doesn’t help us to have good guys and bad guys. I think we have to be honest about the history that we’re coming from and focus on the process of getting [samples and data] shared. The present task is to work toward equity, if not equality, in sharing benefits and risks “so that there’s something in it for everyone.” FINAL THOuGHTS Eck summed up by identifying the ingredients needed to move forward, including incentives, appropriate forms of consent, funding, governance, a focus on specific topics to engage upon, and strong leadership. Spencer added that the leader must convince the government to engage in a coop- erative effort that will shorten the time and reduce the cost of bringing a new drug to market. Flexibility in program design is also a key element he said. “If you set a program that isn’t exactly right, you’ve got to be willing to make a change and sell it to the people who funded you.” Neil reminded the group that just before he was a medical student, the cure rate for childhood leukemia was about 15 percent. Today it is 85 per- cent. The tools and drugs available today can produce that kind of progress in many other areas through a coordinated effort among academic centers, NIH funding, and industry, he said. “We need to remember what we can do when we get together with leadership, with the contemporary tools that we have, and with the highly targeted therapies that we have now. If we can address the infrastructure, the regulatory science, and the coordination, it would be very achievable to make significant progress on many of the big diseases that face us.”

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