Stem Cell Research: Identifying emerging high priority policy issues

Transcription

1 The state stem cell agency Stem Cell Research: Identifying emerging high priority policy issues Ellen G. Feigal, M.D. SVP, Research and Development National Cancer Policy Summit Washington, DC November 4,

2 California Institute for Regenerative Medicine to establish, innovate, AND deliver California taxpayer supported research institute proposition 71 approved by voters (2004) $3 B state obligation bonds fund stem cell research in California Collaboratively developing scientific, regulatory and commercial pathways and funding research to produce stem cell-based therapeutics to improve the lives of patients with serious medical conditions 2

3 Partnerships across public-private sector to facilitate pathway into the clinic Community, industry, academia support, patient advocate partnership Stimulus to academic, biotech sector; build research excellence, collaborations Research capacity Support best scientists, science; translation of discovery to clinical opportunity; partnerships enhancing the best - critical to success Gov t Bonds Collaborations leveraged funding BioPharma Clinical trials Investing in intellectual capital for the long-tem Driver: improved patient outcomes, quality of life, reduced health care costs; commercialization 3

4 CIRM s Vision and Strategy Mission To support and advance stem cell research and regenerative medicine under the highest ethical and medical standards for the discovery and development of cures, therapies, diagnostics, and research technologies to relieve human suffering from chronic disease and injury Explore ( ) Fund broad number of diseases and projects Establish foundation for leadership in stem cell research Focus ( ) Prioritize projects and investments Drive clinical trials for patients to generate preliminary evidence of therapeutic benefit Develop partnerships Deliver (2016+) Facilitate commercialization of therapies Advance therapies to patients Enable business model for stem cell-based therapies 4

5 CIRM activities toward our mission 600 awards > 60 institutions and organizations for training, basic, tools n tech, translation/development Hundreds of collaborations between academic researchers, across state, US, and internationally Co-funding with 21 countries,states,foundations,nih Productive interactions with regulatory agencies, alliance for regenerative medicine, prof societies 70 programs moving towards or in clinic; collaborations, leverage expertise and $ with companies 21 development teams: 7 collaborative funding partners; 1 collaboration with disease foundation; 5 have companies as PI/co-PI; 3 founded companies 5

6 Driving partnerships in translational research towards patients Innovation and solutions Academia Research infrastructure Research capacity Innovation IP Regulatory experience and focus Industry Manufacturing Standards Focus milestones, gono-go decisions Relevance competition IP utilization Delivery of cell therapies Clinical Medicine Patients Clinical infrastructure Monitoring long term Clinical trial capacity 6

7 CIRM s collaborative funding network enables research world-wide UK Spain Andalusia Scotland Germany France India China Japan Canada AHF Keystone NYSCF NIH Maryland JDRF MDA Australia Victoria Argentina Brazil 7

8 Tool and Technologies program address challenges in translation of stem cell therapies to clinic Replacing Diseased or Injured Tissue Lasting, functional engraftment in vivo Clinically compatible technologies to achieve cell survival, engraftment, integration Predictive Preclinical Modeling Reflect human physiology, xenograft retention Persistence and Proliferation Cell Tracking (e.g. imaging) Efficient Processes Cost of Goods, reproducibility (e.g. markers, characterization tools, small molecule substitutes for differentiation factors) Delivery devices 8

9 Early Translational program to develop evidence of POC and development candidate Basic Research Candidate Discovery Research Preclinical Research POC Select Development Candidate Preclinical Dev. File IND Phase 1 Clinical Research Program Goal: Enable the research to translate promising, innovative stem cell discoveries Rationale: Therapeutic hypothesis testing Outcomes: Within 3 years, Achieve in vitro or in vivo proof of concept or Achieve a development candidate ready to move into INDenabling preclinical development Phase 2 Clinical Research Complete Clinical Trial 9

10 Development programs move into clinic trials and consider follow-on financing Basic Research Candidate Discovery Research Preclinical Research Preclinical Dev. Phase 1 Clinical Research Phase1/2, 2 Clinical Research Program Goal: Enable preclinical dev to file IND to enter FIH and/or to complete clinical trial; for Strategic Partnership to attract industry engagement and investment Outcomes: Within 4 years, Complete IND enabling studies to file IND to enter FIH and/or Complete clinical trial to establish feasible dose, delivery that is safe with evidence of biologic activity and/or clinical parameters of preliminary efficacy 10

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12 In addition to funding, CIRM helps teams build product development experience Programs driven by science and evidence, and regulatory considerations needed on development pathway Prior to award Go, no go, progress milestones, and success criteria During research review preclinical/clinical protocols, regulatory strategy, prep for interactions with FDA, attend team meetings Education and training of teams through CIRM/FDA webinars, roundtables, conferences, seminars 12

13 CIRM works with FDA and other agencies on regulatory pathways for cell therapy Regulatory Pathways: International Workshop on Cell Therapies CIRM-led workshop Sept 2013; N. American, European, and Japanese regulatory frameworks for developing cell-based therapies CIRM webinars, roundtables and workshops topics: cell characterization, preclinical animal studies, imaging technology, immune response, scaffolding, clinical trials 13

14 Goal of CIRM s hipsc initiative is to establish a broadly accessible resource Establish high quality disease-specific hipsc resource in California, accessible broadly by researchers in academia and industry - Prevalent, genetically complex diseases - Tissue donor consent - Tissue donor medical information CIRM hpsc Repository Disease modeling and target discovery Drug discovery and development hpsc from California investigators 14

15 Main activities of the resource collecting, deriving, storing and distributing Tissue Collection for Disease Modeling - Consent donors - Collect medical info - Code medical info - Collect tissues hipsc Derivation - Preserve and expand primary source cells - Derive & characterize hipsc hpsc Repository - Store & distribute hipsc - Store & distribute primary source cells - Store & make available coded medical info - Store and distribute addit. hpsc from California - Maintain ability to re-contact, if consented - Assurance from viewers of private tissue donor information to not seek identity - Facilitate re-contact, if consented 15

16 Building quality clinical capacity: Stem cell clinic network solicitation Oct 25 th CLINICAL TRIALS: Develop resources for effective and efficient design and execution of clinical trials for investigational stem cell therapies DELIVERY OF THERAPIES: Become a center of excellence for delivery of stem cell-based therapies that have been proven safe and effective DATA AND INFORMATION: Centralize information about clinical trial experience and outcomes, and data to inform research, clinical, regulatory and reimbursement decisions INFORM THE PUBLIC: Education, outreach and training about clinical trials and available therapies, and potential dangers of unproven procedures HEALTHCARE ECONOMICS: Develop evidence base to support the development of sustainable business models, including reimbursement strategies 16

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18 Enable expanded access to materials and data while ensuring protections and accountability Rapidly expanding distribution of biological materials, associated data Procedures, policies, contract mechanisms provide a foundation ensuring societal benefits of research Consideration should be given to policy initiatives that: o Evaluate existing procedures and mechanisms for enabling expanded access while ensuring protections and accountability o Engage commercial and consumer stewards of materials and data, aimed at identifying consensus approaches for advancing research and commercial development while ensuring protections and accountability 18

19 Material and Information Exchange 19

20 Optimize mechanisms for clinical trial review, oversight, monitoring and info sharing o What are the best models for improving efficacy and harmonization? o What role can sponsors and/or funding agencies play in network governance to support efficiency, efficacy and harmonization? o Can patient information needs be incorporated into the overall system of review, oversight and monitoring? o Are there specific regulatory barriers that impede efforts aimed at efficiency, efficacy, harmonization and patient learning? o How can identified barriers be addressed? 20

21 Funding, regulatory approaches should encourage and foster innovation, not be risk averse Examples to accelerate, move faster Risk taking approach of accelerated approval accelerated approval with a surrogate marker; requires confirmatory trial Breakthrough designation requires highly promising data at phase 1 smaller data set, may be faster Do disease-based approaches slow innovative technologies? Does separation of biologics, drugs, and devices add undue uncertainty and burden on product development? In general, regulatory agencies and research institutions take a reductionist, segmented approach Need to develop cross-functional approach 21

22 Wednesday, November 06,