By Alexander J. Varond –
The Institute of Medicine (“IOM”) recently issued a paper titled “Accelerating the Development of New Drugs and Diagnostics: Maximizing the Impact of the Cures Acceleration Network: Workshop Summary.”
The IOM paper summarizes the “Maximizing the Goals of the Cures Acceleration Network to Accelerate the Development of New Drugs and Diagnostics” workshop led by the Forum on Drug Discovery, Development, and Translation of IOM on June 4-5, 2012, in Washington, DC. The one-and-a-half day workshop brought together representatives from federal agencies, the private sector, advocacy groups, and academia to discuss an initiative called the Cures Acceleration Network (“CAN”), which was originally authorized in the Patient Protection and Affordable Care Act of 2010 and was later placed in the National Center for Advancing Translational Sciences (“NCATS”) within the National Institutes of Health (“NIH”) in 2012.
The overall purpose of the workshop and paper was to help inform the community, NCATS, and the Cures Accelerated Network Review Board (“the CAN Board”) about ways to increase CAN’s impact and accelerate and expand the availability of cures and promote human health.
The workshop objectives were to:
- Identify and catalog potential tools, methods, and approaches that hold promise for accelerating translational science;
- Discuss the authorities conferred to CAN and identify strategies for effectively using those authorities;
- Explore promising models for public-private collaborations that could be strengthened or facilitated by activities under CAN; and
- Identify barriers and potential solutions to facilitate coordination of activities under CAN with FDA regulatory review process and timelines.
Background on CAN. CAN was established in 2010 to award grants and contracts to eligible entities “to accelerate the development of high need cures, including through the development of medical products and behavioral therapies.” A “high need cure” is defined as a drug, device, or biologic, that, as determined by NIH, “is a priority to diagnose, mitigate, prevent, or treat harm from any disease or condition,” and “for which the incentives of the commercial market are unlikely to result in its adequate or timely development.”
The intended functions of CAN include conducting and supporting “revolutionary advances in basic research;” providing needed resources for entities, such as government agencies, biotechnology companies, or academic research institutions, to develop high need cures; and facilitating FDA’s review of the high need cures funded by CAN. The goal of CAN’s involvement in FDA review matters is to expedite “the development and approval of countermeasures and products.”
CAN grants can be made available to any government, private, or non-profit entity for the purpose of, among other things, promoting innovation and helping the recipient to establish protocols that comply with FDA’s standards and permit the recipient to meet regulatory requirements “at all stages of development, manufacturing, review, approval, and safety surveillance of a medical product.” Awards can be granted in amounts not to exceed $15,000,000 per project per fiscal year.
IOM’s paper repeatedly emphases that although CAN does not yet have the funding to support major projects, it can be a crucial catalyst for innovation within NCATS and industry, generally. In its first years, the key to the program’s success is finding ways to “catalyze interesting, novel collaborations, get work done faster, and make a real difference in patients’ lives.”
An Overview of the IOM Paper. Chapter 1 gives a summary of CAN and discusses an overview of the workshop themes. Chapter 2 provides analysis related to differing approaches to accelerating translational science and reviews several successful drug development projects as case studies. Chapters 3 and 4 discuss two unique features of CAN: (1) the authority to require funds to be matched one-to-three from other sources (a requirement that can be waived), and (2) the authority to use “other transactional authority” (a more flexible form of contracting that contributes to the ongoing success of Defense Advanced Research Project Agency (“DARPA”) and avoids some traditional government regulations and policies such as Federal Acquisition Regulations (“FAR”), provisions of the Bayh-Dole Act, and NIH peer-review requirements whereby allowing federal agencies to partner with organizations, and particularly large companies, that have concerns about the standard federal contracting process). Chapter 5 examines how CAN relates to the broader drug development framework. And the final chapter, Chapter 6, concludes by highlighting several actions CAN should take to create major impacts in the development of cures.
Key Takeaways from the IOM Paper and Workshop. A central function of CAN is to “facilitate review in the [FDA] for the high need cures funded by CAN.” One of the ways CAN may be able to help regulatory science and FDA review is to address gaps in regulatory science that could greatly improve product development. More specifically, CAN could be used to further develop the regulatory science toolbox and create a more efficient pathway to develop and evaluate products. The paper notes that the workshop participants repeatedly emphasized the catalytic function CAN must play due to its limited initial funding and acknowledged several steps that CAN could enable, including “building precompetitive cross-cutting consortia, developing better evaluative tools and measure, and supporting relevant data gathering and sharing initiatives.” The following areas were identified as areas where CAN could also potentially help, such as discovering and implementing “new endpoints, new trial designs, better biomarkers to divide diseases into subsets according to prognostic or response predictors, patient-reported outcomes that have credibility, and natural history studies to understand disease course, particularly for rare diseases.”
The key, after all, is for the program to find “a ‘sweet spot’ where industry is not investing, but promising opportunities exist.” And to do this CAN must “cut through red tape, create culture change, and create new tools and new processes that will make a demonstrable difference.”