RESPONDING TO FRUSTRATION1

Joshua Boger, Vertex Pharmaceuticals, reminded the workshop participants that the legislation leading to CAN resulted from frustration with the status quo. He cautioned that CAN will be tempted to do the same thing over and over while expecting different results, which, as Albert Einstein noted, is one definition of insanity. “CAN needs to have a very low tolerance for the status quo, and frankly for consensus, which is the basis of the status quo,” said Boger. It should instead search for companies, individuals, and collaborators who are outside the mainstream, he said.

The small amount of funding initially allocated to CAN is likely to force it to work on tools rather than specific diseases, Boger said. But he added that tools are best developed in the context of a specific project. “Technology is rarely the problem,” he said. “We need to close the application gap.” He commented that individual projects are the best way to create system change.

Boger also expressed concern about a misalignment of goals among funders. Investors do not necessarily have the goal of creating cures. In that sense, the best co-investor is often a patient group, he said, because those are the groups most closely aligned to the mission of CAN.

Finally, he reminded the workshop participants of how difficult it will be to achieve the goals of CAN. “With all due respect, [designing] a Mach 20 aircraft is easy compared to a typical drug.” Ninety-nine out of 100 drug development projects fail to make a significant medical impact. CAN’s portfolio therefore needs to focus not only on cures but on transforming the process that will lead to cures. Good project management is necessary but not sufficient. “Expect most projects to fail. Don’t be defensive about that. Don’t over promise, and therefore you won’t have to fear people or the Congress. They can handle the truth.”

CAN should not be used to convert academic researchers into translational scientists, Boger said. But it can increase knowledge about the constraints on either side. In this way, it can help reshape the engagement of academic investigators with translational work.

The essential ingredient of successful drug development is personal passion sustained over long periods of time, Boger said. “All successful projects fail at least once. That should be built into the process. I know of no exceptions to that rule for any successful drug. They have all failed once. If the projects are set up to weed out failures, it will weed out successes.” Even spectacular failures, if done in good faith, can amount to wins. “Insist on great science, but insist on projects that can only come about through challenging the existing process.”

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1 This section is based on remarks by Joshua Boger, Founder, Vertex Pharmaceuticals.



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