Appendix B

Summary of Questionnaires

As described in Appendix A, the committee solicited testimony in multiple forms from a variety stakeholders on topics related to the study charge. In addition to direct testimony given at three public meetings, the committee asked for comments through seven online questionnaires (see Boxes B-1 to B-7 for questionnaire items) from members of the Independent Citizens Oversight Committee (ICOC), the leadership of California Institute for Regenerative Medicine (CIRM)-funded institutions, principal investigators who were recipients of CIRM grants, investigators who applied for but did not receive CIRM funding, university technology transfer professionals, CIRM industry partners, CIRM international partners, and stakeholders and members of the general public.

Each questionnaire was posted on the committee’s Institute of Medicine (IOM) project website. IOM staff contacted individuals and groups in each category by e-mail to provide information about the study, the purpose of the questionnaires, and links to each questionnaire. Only aggregated responses—without information identifying individual respondents—were presented to the committee and placed in the public access file for the project.

The questionnaire responses were used to inform the committee’s deliberations. The results were not obtained through random sampling, but through targeting of specific audiences. The information obtained through these seven questionnaires was in no way determinative of or the sole basis for the committee’s recommendations.



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Appendix B Summary of Questionnaires A s described in Appendix A, the committee solicited testimony in multiple forms from a variety stakeholders on topics related to the study charge. In addition to direct testimony given at three public meetings, the committee asked for comments through seven online ques- tionnaires (see Boxes B-1 to B-7 for questionnaire items) from members of the Independent Citizens Oversight Committee (ICOC), the leadership of California Institute for Regenerative Medicine (CIRM)-funded institutions, principal investigators who were recipients of CIRM grants, investigators who applied for but did not receive CIRM funding, university technology transfer professionals, CIRM industry partners, CIRM international part- ners, and stakeholders and members of the general public. Each questionnaire was posted on the committee’s Institute of Medicine (IOM) project website. IOM staff contacted individuals and groups in each category by e-mail to provide information about the study, the purpose of the questionnaires, and links to each questionnaire. Only aggregated responses—without information identifying individual respondents—were presented to the committee and placed in the public access file for the project. The questionnaire responses were used to inform the committee’s delib- erations. The results were not obtained through random sampling, but through targeting of specific audiences. The information obtained through these seven questionnaires was in no way determinative of or the sole basis for the committee’s recommendations. 145

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146 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE DESCRIPTION OF QUESTIONNAIRES The committee received 220 responses in total, from 12 of 29 members of the ICOC, 8 of 18 invited individuals from the leadership of CIRM- funded institutions, 120 of 340 CIRM principal investigators, 30 investiga- tors not funded by CIRM, 4 of 21 technology transfer professionals, 1 of 20 individuals representing CIRM industry partners, and 45 individuals from the general public. Although these comments helped inform the com- mittee’s deliberations, the committee recognizes that the response rate to the questionnaires was low and that the responses cannot be considered representative of the targeted groups. The remainder of this appendix provides brief summaries and high- lights of the thoughts, perspectives, and concerns articulated by various respondents on CIRM’s organizational processes, its programmatic and scientific scope, its organizational and management systems, its funding model, and its intellectual property policies. RESPONSES TO QUESTIONNAIRE FOR MEMBERS OF THE INDEPENDENT CITIZENS OVERSIGHT COMMITTEE There is little question that members of the ICOC are proud of CIRM’s overall record of accomplishment in establishing California as one of the key worldwide hubs of research in regenerative medicine. Moreover, given the complex nature of mobilizing an expert staff to allocate thoughtfully funds entrusted to them by the state, they believe CIRM has done a remark- able job in a very short period of time. Those board members responding to the questionnaire did have some concerns, which focused on the work- ing relationship between the chair and the president, the size of the board (too large), and the challenge of meeting various state requirements that apply to all state agencies. Finally, for the most part, respondents thought the board operated effectively and focused on the right issues. As a group of respondents, they are aware of the transition challenges that lie ahead, including the potential expiration of state funds, the need to produce clini- cal outcomes, and the desire and need to work more effectively with indus- try. Most respondents believe that personal interests have not played a role in their work on the ICOC and that conflicts of interest have not affected the ICOC’s effectiveness. RESPONSES TO QUESTIONNAIRE FOR LEADERSHIP OF CIRM-FUNDED INSTITUTIONS To gauge the impact of CIRM and CIRM funding on research institu- tions in California, the committee selected those institutions with the high-

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APPENDIX B 147 est CIRM funding levels and invited the deans of seven medical schools, research deans at three universities lacking medical schools, research direc- tors at two independent hospitals, and research directors at six independent research institutes to complete the questionnaire. Three respondents were members of the CIRM board, and all three felt they were able to balance their responsibilities as a board member with their institutional responsibili- ties. Respondents were unanimous in indicating that CIRM’s intellectual property policies had not influenced their decision to pursue CIRM funding. The greatest diversity of responses was elicited by the question: “In what ways can CIRM be improved?” Two respondents alluded to the need for greater transparency, and two felt that a more clearly articulated strategic plan was needed. Two expressed concerns about CIRM staff turnover and other indications of administrative “dysfunction.” One mentioned a need to involve clinicians earlier in the planning stages of research to “ascertain the realistic translational potential” of basic research projects. Overall, based on a fairly limited set of responses, CIRM appears to have had a positive impact on research institutions in California, especially with respect to increased space for stem cell research, enhanced training opportunities, and retention of faculty. Responses suggest that the overall institutional impact of CIRM has been a function of success in obtaining CIRM funding. There have clearly been big “winners” in this competition, and those institutions are the ones for which CIRM funding has had the largest positive impact, including recruiting faculty from outside Califor- nia and building entirely new buildings for stem cell research funded by a combination of CIRM facilities awards and private gifts. RESPONSES TO QUESTIONNAIRE FOR PRINCIPAL INVESTIGATORS FUNDED BY CIRM Most principal investigators who responded to the questionnaire felt that CIRM’s priorities were clearly articulated, that the requests for appli- cations (RFAs) were clear regarding the scope and purpose of the research, and that the pre-application review was helpful. Most also felt that the CIRM scientific staff were very helpful, although at times were somewhat inflexible regarding adaptation to emerging findings. Those principal inves- tigators that were also funded by the National Institutes of Health (NIH) felt that overall, the CIRM process compared favorably. On the other hand, some expressed the sentiment that the grant management process could be more flexible and streamlined and that the pre-application process could be more transparent.

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148 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE RESPONSES TO QUESTIONNAIRE FOR CALIFORNIA STEM CELL INVESTIGATORS WHO APPLIED FOR AND DID NOT RECEIVE FUNDING FROM CIRM Principal investigators that were not successful in obtaining funding from CIRM were more critical of CIRM’s processes. Even in this group, however, most respondents felt CIRM’s priorities were clear, as were the RFAs. This group of unsuccessful applicants for CIRM funding was less sanguine about the fairness of the overall program and the responsiveness of the CIRM staff. Most of these respondents felt that the feedback from CIRM staff and/or reviewers was not helpful and said they were unaware of the appeal process. RESPONSES TO QUESTIONNAIRE FOR TECHNOLOGY TRANSFER PROFESSIONALS Given that there were only four responses to this questionnaire, the committee could not draw definitive inferences. All respondents considered themselves to be familiar with CIRM’s intellectual property policies. Most offered unfavorable comments about these policies. They felt that the poli- cies are adequate but confusing, seem overly burdensome to licensees, and in some cases are too aggressive. Only one respondent felt that CIRM’s intellectual property policies were similar to other such policies. Others felt that CIRM’s policies are complex and “place more burdens on potential licensees.” One respondent indicated that “implementation guidelines useful for instructing practice are virtually nonexistent. For example, CIRM requires an annual utiliza- tion report but does not cite a due date or what kinds of information such a report should contain.” Most respondents have received invention disclosures involving CIRM- funded research; the number of invention disclosures received varied from 4 to 35. These respondents had filed patent applications for CIRM-funded inventions, and the number of inventions they sought to patent or license ranged from 4 to 12. These respondents all felt that CIRM policies were more complex to administer than others. None are currently in licensing discussions concerning any such inventions. Respondents made several suggestions for improving CIRM’s intellec- tual property policies. They include reducing burdens on grant recipients and potential licensees; clarifying regulations and policy definitions; clari- fying guidelines for implementation and for compliance; holding quarterly videoconferencing; networking to identify possible licensees; and modifying licensing sections, especially in drug pricing and royalty sharing with the state.

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APPENDIX B 149 RESPONSES TO QUESTIONNAIRE FOR INDUSTRY PARTNERS The committee received only one response to this questionnaire and did not believe it was appropriate to draw even very tentative inferences from that response. RESPONSES TO QUESTIONNAIRE FOR THE GENERAL PUBLIC The committee was eager to receive input from the California public and grateful to those that took advantage of the open questionnaire. How- ever, the committee recognizes the difficulty of assessing this input given the small number of responses and with little knowledge of the backgrounds of the respondents themselves. Most respondents to the public questionnaire felt that CIRM took a leadership role in establishing infrastructures for biomedical research, providing funding for a research area that lacked federal financial support, attracting and retaining distinguished stem cell scientists, creating impor- tant partnerships with many countries, promoting collaboration among researchers around the world, and stimulating similar programs in other states. Several made comments, however, about CIRM’s having spent too much on infrastructure and basic research with no clinical outcomes/prod- ucts. Some respondents felt the California taxpayers were “misled” and their money “wasted.” There was some expression of concern that CIRM was providing insufficient support for work with human embryonic stem cells. There were many complaints about transparency. Most respondents felt that information on CIRM’s website was suffi- cient, useful, and up to date, and the monthly newsletter and e-mail updates were very helpful. Others felt that the materials were too scientific for the public to understand.

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150 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE BOX B-1 Questionnaire for Members of the Independent Citizens Oversight Committee (ICOC) The California Institute for Regenerative Medicine (CIRM) has asked the Institute of Medicine (IOM) to provide an independent assessment of CIRM’s programs, operations, and performance. The IOM Committee on a Review of the California Institute for Regenerative Medicine will as- sess the organization’s initial processes, its programmatic and scientific scope, organizational and management systems, funding model, and intellectual property policies. To help the committee address its charge, it would like to obtain input from members of the Independent Citizens Oversight Committee (ICOC). Question 1: What are CIRM’s most significant accomplishments? Question 2: What features of CIRM have contributed most to its successes? Question 3: What features have been most challenging or have impeded accomplishments? Question 4: What do you think are CIRM’s greatest challenges and op- portunities in the future? Question 5: How well do the varying constituents of the ICOC work together? Question 6: In what ways does the ICOC agree on the direction the organization should take? In what ways do they disagree? Question 7: In what ways are you productively involved in strategic planning for CIRM? Question 8: Are there elements of the strategic planning that you are not part of? Should you be? Question 9: Do you find the work of the ICOC (including sub-committees, work groups, etc.) appropriate, and do ICOC members have appropri- ate input to board decisions?

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APPENDIX B 151 Question 10: Are ICOC meetings focused on the right issues and do they make good use of time? Question 11: Is the required time commitment as an ICOC member appropriate? Question 12: Do you feel valued as an ICOC member? How has this been demonstrated (or not)? Question 13: In what ways do the structure and composition of the ICOC impact its effectiveness? Question 14: How effectively does the ICOC work with CIRM Board Chair and President? Are roles and responsibilities clearly defined? Question 15: What is your perspective on the balance of responsibilities between the Chairperson and the President? Question 16: What would you change to make the ICOC more effective? Question 17: Do you believe that board members, in their work on the ICOC, are influenced by their personal or private interests, apart from and potentially different from the broader interests of the people of California as sponsors of CIRM? Please elaborate. Question 18: Does conflict of interest affect the ICOC’s effectiveness? Question 19: What are the strengths of the ICOC organizational struc- ture? Weaknesses? Question 20: How does the structure, level of transparency, and over- sight of the ICOC contribute to the effectiveness of CIRM (e.g., driving excellent science, achieving clinical outcomes, engaging the scientific community and other stakeholders, and improving California’s status in the field)? Question 21: Please feel free to share any further thoughts with us. You may also e-mail documents or other information to jxi@nas.edu.

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152 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE BOX B-2 Questionnaire for Leadership of CIRM-funded Institutions The California Institute for Regenerative Medicine (CIRM) has asked the Institute of Medicine (IOM) to provide an independent assessment of CIRM’s programs, operations, and performance. The IOM Committee on a Review of the California Institute for Regenerative Medicine will as- sess the organization’s initial processes, its programmatic and scientific scope, organizational and management systems, funding model, and intellectual property policies. To help the committee address its charge, the leadership from institu- tions and organizations that receive funding from CIRM are invited to share their thoughts and concerns about CIRM. Question 1: How has CIRM funding affected your ability to increase space for stem cell research? Question 2: How has CIRM funding enabled you to recruit new faculty from outside California? Question 3: How has CIRM funding enabled you to retain faculty who might otherwise be recruited outside California, including both senior faculty and trainees who have joined your faculty? Question 4: How has CIRM funding provided new training opportunities for students; postdoctoral fellows? Question 5: How has CIRM funding affected your ability to raise phil- anthropic funds? Question 6: How has CIRM funding affected your faculty’s ability to obtain National Institutes of Health grant funding? Question 7: Given that CIRM funding likely provides lower indirect costs than National Institutes of Health funding, what effect has this had on overall financial status of the research enterprise? Question 8: What has been the effect of CIRM funding on setting re- search directions and priorities? Question 9: If you are a member of the CIRM Board, how have you balanced your responsibilities as a member of the board with your responsibilities as a leader of your own institution? Question 10: In what ways can CIRM be improved? Question 11: Have CIRM intellectual property policies influenced your decision to pursue CIRM funding? Question 12: What type of organization do you represent (e.g., medi- cal school, research institute, hospital, university without a medical school)? Question 13: If you have additional thoughts about how CIRM can im- prove its programs and initiatives to meet its scientific goals or would like to share information related to the committee’s work, please use the space provided below to do so. You may also e-mail documents or articles to support your testimony to jxi@nas.edu.

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APPENDIX B 153 BOX B-3 Questionnaire for CIRM Principal Investigators The California Institute for Regenerative Medicine (CIRM) has asked the Institute of Medicine (IOM) to provide an independent assessment of CIRM’s programs, operations, and performance. The IOM Committee on a Review of the California Institute for Regenerative Medicine will as- sess the organization’s initial processes, its programmatic and scientific scope, organizational and management systems, funding model, and intellectual property policies. To help the committee address its charge, investigators who receive funding from CIRM are invited to share their thoughts and concerns about CIRM. Question 1: Are CIRM funding priorities clearly articulated? Question 2: Are CIRM-initiated requests for applications clear regarding scope and purpose of the research for which they were requesting proposals? Question 3: Was the pre-application and proposal review process help- ful and fair? Question 4: Was CIRM program staff accessible, responsive, and help- ful once a grant was awarded? Question 5: Was CIRM willing to allow appropriate modification of re- search aims and budget allocations when results [in your lab or else- where] indicated some change in direction was advisable? Question 6: How does the CIRM proposal and grants management process compare to other agencies such as the National Institutes of Health? Is it more or less efficient or about the same? Question 7: In what ways can CIRM be improved? Question 8: If you have additional thoughts about how CIRM can im- prove its programs and initiatives to meet its scientific goals or would like to share information related to the committee’s work, please use the space provided below to do so. You may also e-mail documents or articles to support your testimony to jxi@nas.edu.

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154 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE BOX B-4 Questionnaire for California Stem Cell Investigators Who Applied for and Did Not Receive Funding from CIRM The California Institute for Regenerative Medicine (CIRM) has asked the Institute of Medicine (IOM) to provide an independent assessment of CIRM’s programs, operations, and performance. The IOM Committee on a Review of the California Institute for Regenerative Medicine will as- sess the organization’s initial processes, its programmatic and scientific scope, organizational and management systems, funding model, and intellectual property policies. To help the committee address its charge, investigators who applied for but did not receive funding from CIRM are invited to share their thoughts about CIRM. Question 1: Are CIRM funding priorities clearly articulated? Question 2: Are CIRM-initiated requests for applications clear regarding scope and purpose of the research for which CIRM was requesting proposals? Question 3: Was the pre-application and proposal review process help- ful and fair? Question 4: Was CIRM program staff accessible, responsive, and help- ful in providing feedback regarding the specific aims of the RFA prior to submission? Question 5: Were reviewer critiques helpful in revising proposals for a second submission? Was CIRM program staff accessible, responsive, and helpful in providing additional feedback that helped to understand the critiques? Question 6: Is it possible to appeal a funding decision? If so, was the process for appeal clearly defined and did it appear to be a fair process? Question 7: How does the CIRM proposal process compare to other agencies such as the National Institutes of Health? Is it more or less efficient or about the same? Question 8: In what ways can the CIRM-initiated RFA proposal process be improved? Question 9: If you have additional thoughts about how CIRM can im- prove its programs and initiatives to meet its scientific goals or would like to share information related to the committee’s work, please use the space provided below to do so. You may also e-mail documents or articles to support your testimony to jxi@nas.edu.

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APPENDIX B 155 BOX B-5 Questionnaire for the General Public The California Institute for Regenerative Medicine (CIRM) has asked the Institute of Medicine (IOM) to provide an independent assessment of CIRM’s programs, operations, and performance. In 2005, CIRM was cre- ated and began its operations following the 2004 adoption of Proposition 71 (the California Stem Cell Research and Cures Act). Proposition 71 charged CIRM with determining the most effective means of distribut- ing $3 billion in state funds over at least 10 years in order to accelerate the science of regenerative medicine and its translation into health and economic benefits for the State of California. The IOM Committee on a Review of the California Institute for Re- generative Medicine will assess the organization’s initial processes, its programmatic and scientific scope, organizational and management systems, funding model, and intellectual property policies. To help the committee address its charge, members of the public are invited to share their thoughts, perspectives and/or concerns about CIRM. Question 1: Please share your opinion about the continued importance of stem cell research and the role of CIRM’s work/contribution in this area. Question 2: What particular or specific impact has CIRM had on the biomedical research community both in California and elsewhere since its inception in 2005? Question 3: If you are an individual or know an individual with a chronic, debilitating disease what do you see as potentially the biggest benefit you or they might receive from CIRM’s work? Question 4: Do you think CIRM has provided to the public transparent, up-to-date, and sufficient information about its organization and activi- ties? Do you find the information helpful? Question 5: How should information about CIRM’s organization, activi- ties, and programs be shared with the public? Question 6: What is your opinion about CIRM’s portfolio of projects and its grant programs? Do you think it is necessary that CIRM meet its scientific goal of developing stem cell and related research for the diagnosis, prevention and treatment of disease and injury within its initial timeline of 10 years? Question 7: If you have additional thoughts about how CIRM can im- prove its programs and initiatives to meet its scientific goals or would like to share information related to the committee’s work, please use the space provided below to do so. You may also e-mail documents or articles to support your testimony to jxi@nas.edu.

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156 THE CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE BOX B-6 Questionnaire for Technology Transfer Professionals The California Institute for Regenerative Medicine (CIRM) has asked the Institute of Medicine (IOM) to provide an independent assessment of CIRM’s programs, operations, and performance. The IOM Committee on a Review of the California Institute for Regenerative Medicine will as- sess the organization’s initial processes, its programmatic and scientific scope, organizational and management systems, funding model, and intellectual property policies. To help the committee address its charge, the committee has a very particular interest in how technology transfer professionals at institutions that perform CIRM-funded research assess CIRM’s intellectual property policies. Question 1: Are you familiar with the intellectual property (IP) policies of CIRM? If so, please complete the remaining questions. Question 2: Overall what do you think of the CIRM IP policies? Question 3: Do CIRM’s IP policies differ in important respects from those of other sponsors of research? If so, please explain. Question 4: Compared to other sponsors of research, does CIRM pro- vide adequate support for the technology transfer activities of its grantees? Question 5: Has your office received any invention disclosures involving CIRM-funded research in your institution? Question 6: How many such disclosures has your office received? Question 7: Have you filed patent applications or sought to license IP rights for any CIRM-funded inventions? Question 8: How many CIRM-funded inventions have you sought to patent and/or license? Question 9: Are you currently in licensing discussion concerning any such inventions? Question 10: Have the CIRM IP policies played a significant role in considerations of how your office will handle inventions arising from CIRM-funded research? Question 11: To your knowledge, have the CIRM IP policies had any impact on your ability to find licensees for CIRM-funded inventions? Question 12: How in your view could CIRM IP policies be enhanced or improved? Question 13: Please feel free to share any further thoughts you might have on IP policies and technology transfer related to CIRM-funded research. You may also e-mail documents or other information to jxi@ nas.edu.

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APPENDIX B 157 BOX B-7 Questionnaire for CIRM Industry Partners The California Institute for Regenerative Medicine (CIRM) has asked the Institute of Medicine (IOM) to provide an independent assessment of CIRM’s programs, operations, and performance. The IOM Committee on a Review of the California Institute for Regenerative Medicine will as- sess the organization’s initial processes, its programmatic and scientific scope, organizational and management systems, funding model, and intellectual property policies. To help the committee address its charge, entities in current partner- ship with CIRM are invited to share their thoughts and concerns about CIRM. Question 1: What is the nature of your partnership(s) with CIRM? How/ why was the partnership initiated? Who initiated it? If the partnership involves collaborating with academic scientists in California, how criti- cal was CIRM to forming the partnership? Question 2: Was the process of assembling the partnership easy to ar- range relative to other partnerships in which you have participated? Question 3: How critical is the research being conducted to your busi- ness? Does the partnership fill a gap in funding unmet by other sources? Question 4: What, if anything, is unique about partnering with CIRM? Question 5: Are you satisfied with the ongoing management of the part- nership? How could management be improved? Question 6: Would you expect your partnerships with CIRM to grow, remain at current levels or decline? Question 7: Have the intellectual property (IP) and revenue sharing policies and the Consumer Access requirements of CIRM been a significant consideration in entering into partnership with CIRM? What do you think of those policies? Have you licensed CIRM-funded IP? Are you in discussions to license IP arising from your partnership with CIRM or other CIRM-funded research? Question 8: How could existing CIRM programs and processes be en- hanced or improved? Question 9: Have CIRM funding and projects enhanced the biotech industry in California? How could CIRM engage/support industry better? Question 10: If you have additional thoughts about how CIRM can im- prove its programs and initiatives to meet its scientific goals or would like to share information related to the committee’s work, please use the space provided below to do so. You may also e-mail documents or articles to support your testimony to jxi@nas.edu.

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