Independent Review Commends CIRM's Achievements, but Calls on Institute to Revise Governance and Policies to Maintain Credibility and Impact

WASHINGTON — The California Institute for Regenerative Medicine (CIRM) has achieved many notable results during its first seven years and helped establish the state of California as an international hub of stem cell research and development, says a new report from the Institute of Medicine. However, CIRM's efforts could be enhanced by creating an external scientific advisory board to provide continuing and consistent strategic advice, by dealing more effectively with the perception of conflicts of interest on its governing board, and by reorganizing its governance to ensure that the board is able to provide independent oversight.

The IOM's review, commissioned by CIRM, noted that the organization rapidly built a high-quality research portfolio that has supported 40 patent applications and three license agreements and enabled grant recipients to attract over $1 billion in matching funds. While the study committee was not asked to evaluate the merits of individual projects that CIRM has funded or denied, it commended the institute for quickly implementing a thoughtful and effective grant-making process. At the same time, however, CIRM's leadership and California legislators need to consider changes in the institute's governance and operations given the rapid progress being made in the field, the institute's need for independent oversight, and the institute's desire to steer its efforts from basic research to clinical applications, the report says.

"Overall, CIRM has done a remarkably good job setting priorities and directing voter-approved funds to projects that have given California a prominent position in regenerative medicine," said committee chair Harold T. Shapiro, president emeritus and professor of economics and public affairs, Woodrow Wilson School of Public and International Affairs, Princeton University, Princeton, N.J. "The field has evolved since CIRM's inception, so we have recommended ways to help the maturing institute evolve as well, particularly in its governance structure, some of its administrative practices, and its use of external perspectives on strategic scientific priorities."

Members of CIRM's governing board -- the Independent Citizens' Oversight Committee (ICOC) -- should focus on strategic planning, oversight, and broad allocation of resources and program definition rather than daily operational activities, the report says. The institute's president and other staff members should have full responsibility for daily management and operations, particularly concerning review of grant applications and funding recommendations for projects within the parameters set by the board. These changes are necessary to alleviate concerns that in its current composition the ICOC cannot provide independent oversight of management decisions, the report says. In addition, a greater number of board members should have no direct stake in the institute's decisions, and no organization should be guaranteed a board seat.

These structural changes would require amendments to Proposition 71, the legislation that established CIRM, the committee acknowledged. The legislation gave the ICOC chair several operational responsibilities and stipulated that university leaders and others most knowledgeable with stem cell research and its potential applications were to occupy many of the board seats. These provisions protected CIRM from political interference in its early years, the report says, but they have since hindered efforts to resolve concerns about the board's influence and perceptions of conflicts of interest among members who stand to benefit from CIRM's funding decisions.

The grants working group -- an advisory team of scientists, patient advocates, and others that evaluates funding applications -- should be reconstituted to exclude ICOC members, the report says, and ICOC members of the working group who provide the insights of patients should be replaced by other patient advocates so that the group does not lose this critical perspective. ICOC should be the final step of approval for a slate of grants, but its members should not have the power to approve or veto individual applications. Because the committee was not charged with reviewing CIRM's past funding decisions, it did not identify any specific cases of conflict, but it concluded that the potential is real and is a widely held concern. In addition, the establishment of a new scientific advisory board would provide a source of expertise to help CIRM's president vet ideas for funding opportunities, decide which discoveries should progress to the clinic, and engage industry partners.

CIRM issued a new strategic plan this year that outlines a shift in emphasis from basic research and infrastructure building to translation and clinical applications, which the committee supports. Given this shift, the institute should increase industry representation on its governing board and advisory groups to leverage corporate expertise and resources, the report says. Enhanced industry representation is necessary if CIRM is to successfully encourage companies to make the large investments needed to develop new therapies.

CIRM should also develop a sustainability platform that would enable it to take full advantage of its accomplishments if its support from the state is not renewed. This platform should explain if and how the institute intends to obtain additional private or public funding and how such developments might affect the institute's ability to meet its obligations to the public as well as ICOC's role and structure, the committee said. CIRM's latest strategic plan does not address uncertainties about what will happen once all the voter-approved funds have been dispersed, creating challenges for researchers and the industry. Of the $3 billion provided by Proposition 71, $856 million remains to be either earmarked or expended.

The report was sponsored by the California Institute for Regenerative Medicine. Established in 1970 under the charter of the National Academy of Sciences, the Institute of Medicine provides objective, evidence-based advice to policymakers, health professionals, the private sector, and the public. The Institute of Medicine, National Academy of Sciences, National Academy of Engineering, and National Research Council together make up the private, nonprofit National Academies. For more information, visit http://national-academies.org or http://iom.edu.