Paul Aliu, PharmD
Novartis, Chief Medical Office
Paul Aliu, PharmD
Global Head of Medical Governance
Novartis, Chief Medical OfficePaul Aliu is the Global Head of Medical Governance within the cross-divisional Chief Medical Office at Novartis with responsibility for the oversight, processes, training and systems for compassionate use/expanded access and post-registration medical programs (e.g. Investigator initiated trials, non-interventional studies, registries and Phase IV studies). He has worked in the pharmaceutical industry for over 15 years in multiple roles in drug development, medical affairs and program management covering therapeutic areas such as oncology, rheumatology, transplantation, infectious and tropical diseases. Prior to joining the pharmaceutical industry he trained and practiced as a clinical pharmacist.
He previously served as an industry representative on the WHO/Roll-Back Malaria (RBM) case management working group, managed alliances and partnership agreements (Public-Private & Private-Private) with multilateral organizations, NGOs, academic/research institutes; and participated in various round-table and stakeholder discussions on Global Health issues.
Center for Information and Study on Clinical Research Participation (CISCRP)
Center for Information and Study on Clinical Research Participation (CISCRP)Behtash Bahador is a Senior Manager for the Communicating Trial Results program at the non-profit Center for Information and Study on Clinical Research Participation (CISCRP). A Master of Science in Health Communication candidate at Tufts University in Boston, Behtash leads Quality and Compliance on CISCRP’s program that communicates clinical trial results in a way that is non-promotional, unbiased, and easy-to-understand for patients and the public. He works directly with a wide range of industry sponsors on global implementation and aligning best practices in health communication with regulatory and industry guidelines
Amy Barone, MD
FDA Office of Hematology and Oncology Products
Amy Barone, MD
FDA Office of Hematology and Oncology ProductsDr. Amy Barone is a pediatric oncologist who joined the US Food and Drug Administration (FDA) in January 2014 as a medical officer for the CNS, Pediatric Solid Tumors, and Rare Cancers. As a medical officer, she is responsible for the review and regulatory oversight of expanded access programs, numerous investigational new drug applications (INDs), new drug applications (NDAs), biologics license applications (BLAs), and single patient INDs. She is very involved in the FDA effort to move the field of oncology drug development for rare diseases forward.
Executive VP, Medicines Access
Inceptua LtdMark Corbett has over 20 years’ industry experience with the last 10 years specializing in the areas of pre-approval access and unlicensed medicines. Previously, he was Managing Director at Idis, Global Access, Senior Vice President at Clinigen Global Access Programs, Head of European Business Development at Idis and an Independent Pharmaceutical Consultant. Mark has extensive specialist knowledge and operational expertise gained through the implementation of more than 150 global access programs for a variety of biopharmaceutical companies across all stages of the drug lifecycle.
Anne Cropp, PharmD
Early Access Care, LLC
Anne Cropp, PharmD
Chief Scientific Officer
Early Access Care, LLCAnne Cropp, BSc, Pharm.D. BCAP serves as the Chief Scientific Officer for Early Access Care, providing consultative, functional and operational support to biopharmaceutical companies for Expanded Access / Compassionate Use of investigational drugs. Prior to founding Early Access Care, Anne was Vice President at Pfizer Inc. Anne has extensive experience in pharmaceutical and biopharmaceutical drug development. She has built high-performing, diverse leadership teams supporting pipeline products from Phase 1 through NDA/MAA submission. Her expertise spans drugs, biologics and devices . Anne is a globally recognized expert in Expanded Access US and ex-US and led the development and execution of the industry's first physician-entry request portal for a global pharmaceutical company. As a life long member of the scientific research community, Anne's passion has always been to bring solutions to advance patient care. Anne began as a pharmacist meeting the needs of hospitalized patients. She certified as a clinical pharmacologist and completed two post-doctoral research fellowships in cardiovascular clinical pharmacology. While at the NHLBI of the NIH she first experienced Expanded Access. Motivated by a desire to help patients, she founded Early Access Care.
Harvard Medical School
Harvard Medical SchoolJonathan Darrow joined the faculty of Harvard Medical School and the Program on Regulation, Therapeutics, and Law (PORTAL) in 2016, following service as Senior Law Clerk to a judge on the United States Court of Appeals for the Federal Circuit, the court that decides all patent appeals involving pharmaceuticals, biologics, and medical devices. He holds degrees in genetics, law, and business from Cornell, Duke, and Boston College, respectively, as well as a research doctorate in pharmaceutical policy and intellectual property theory from Harvard, where he also completed the LL.M. program. After admission to the bar in 2001, Dr. Darrow worked on emerging company issues in the Silicon Valley and on pharmaceutical litigation matters at a large private firm in Washington, DC. He has since served on the faculties of four universities, and has supported various global heath projects at the Geneva campuses of the World Trade Organization, the World Health Organization, and the World Intellectual Property Organization. He is a co-author of Cyberlaw: Management & Entrepreneurship (2015) and The Legal and Ethical Environment of Business (2d ed. forthcoming 2018). His scholarship on health policy and intellectual property has appeared in the British Medical Journal, the New England Journal of Medicine; the Journal of Law, Medicine & Ethics; the Stanford Technology Law Review; the Yale Journal of Health Policy Law & Ethics, and the Harvard Journal of Law & Technology, among many others.
King & Spalding
King & SpaldingDavid Farber is a partner in the FDA and Life Sciences practice at King & Spalding, resident in the firm’s Washington, D.C. office, where he maintains a health care practice focused on government advocacy and regulatory counseling. A leading national expert on Medicare matters, he works regularly on Capitol Hill and with key regulatory agencies, particularly the Centers for Medicare & Medicaid Services and U.S. Food and Drug Administration, on legislative and regulatory advocacy and counseling matters. His regulatory experience complements and augments the firm’s FDA regulatory, life sciences and healthcare teams, all of which are recognized as leading practices by Chambers USA. David regularly speaks before national audiences on expanded access, health care reimbursement, secondary payer, and CMS and FDA regulatory matters.
Carrie Langlais Furr, PhD
Carrie Langlais Furr, PhD
VP Regulatory Affairs
AmpliPhi BioSciencesCarrie-Lynn Langlais Furr, PhD, RAC, is Vice President of Regulatory Affairs and Program Management at AmpliPhi Biosciences, a clinical-stage biotechnology company focused on the development of bacteriophage-based therapies for the treatment of antibiotic-resistant bacterial infections. Dr. Furr has over a decade of integrated product development and regulatory affairs experience. Prior to joining AmpliPhi, Dr. Furr served as Senior Director of Operations at Rho, Inc., a clinical research organization, where she also held leadership roles in regulatory affairs, integrated development, program management, and medical writing, which stemmed from her early years in regulatory operations at PPD. Dr. Furr’s experience includes providing strategic and operational support through all phases of clinical development, including formal interactions and negotiations with regulatory authorities as well significant contributions to FDA market approval of 5 drugs and a biologic-device combination product. She received a PhD from Texas A & M University in biochemistry and biophysics and studied the antibiotic mechanism of bacteriophages. With AmpliPhi, Dr. Furr is applying her bacteriophage basic research knowledge to treat patients with serious or life-threatening bacterial infections Under Expanded Access regulations, as well as under traditional development pathways.
MedSciCom LLCArt Gertel established an independent consultancy in 2012, MedSciCom, LLC, after 35 years serving in Senior Management positions in the pharmaceutical and related industries. He provides independent and collaborative strategic regulatory consulting - applying drug/device development, medical writing, bioethics, and DSMB expertise.
With a strong interest in Biomedical Ethics in the context of clinical trials, he has developed seminars on the topic and leads workshops, worldwide. Recently, Art co-chaired the development of CORE Reference, a medical writer’s guide to compliance with ICH E3. He has been involved in the development of clinical study protocol standards under the auspices of CDISC (Clinical Data Interchange Standards Consortium) and TransCelerate Biopharma, and is a Charter Member of the CDISC Glossary Group. He serves on the Advisory Board of an IRB, and chairs the Alliance for Clinical Research Excellence and Safety (ACRES) Global Ethical and Regulatory Innovation (GERI) Steering Committee. He has published on pre-approval access issues, and has served on ethics panels addressing issues of patient protections and integration of clinical practice into the new drug research and development process.
He has been active in the establishment of standards of authorship for AMWA, EMWA, and ISMPP, as well as CDISC data transformation standards for protocols, registries, and health records. Art is a founding member of GAPP (Global Alliance of Publication Professionals) – a rapid-response task force with a remit to address misconceptions about authorship. As a Registered Agent with FDA, Art represents ex-US biopharmaceutical companies in FDA interactions.
Art is Past President of the American Medical Writers Association (AMWA) and a Fellow of both AMWA and the European Medical Writers Association (EMWA), continuing to serve on committees and lead workshops and plenary sessions. He received AMWA’s Swanberg and Eric Martin Awards.
Formerly at FDA, Office of Health and Constituent Affairs
Formerly at FDA, Office of Health and Constituent AffairsRichard Klein recently left FDA after more than 41 years with the agency. He served as director of the FDA’s Patient Liaison Program in the agency’s Office of Health and Constituent Affairs, the primary agency interface with patients and patient advocate communities. He interacted extensively with outside communities and within the agency’s scientific and policy offices to advocate for patient interests, and facilitate patient engagement. He actively addressed issues and concerns of patients in a variety of areas, including treatment access to unapproved drugs, product safety, and clinical trial design.
Mr. Klein has worked in various capacities at FDA providing him with a well-rounded understanding of the regulatory issues that affect patients. He participated in the development of revised expanded access regulations and guidelines, and led the creation of the FDA expanded access website, played an active role in the development of the streamlined application for individual patient access, the exemption from full board IRB review for Individual Patient IND expanded access, and the Expanded Access Navigator. He also worked to create an expanded access full-board IRB waiver for Individual INDs to facilitate and speed the review process.
Prior to working in patient engagement, he helped to develop policies and regulations for the protection of human research subjects, and provided guidance for institutional review boards (IRBs).
Michael Kurilla, MD PhD
National Center for Advancing Translational Sciences (NCATS)-NIH
Michael Kurilla, MD PhD
Director of the Division of Clinical Innovation
National Center for Advancing Translational Sciences (NCATS)-NIHMichael Kurilla is the director of the Division of Clinical Innovation at NCATS (National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)). In this capacity, he oversees the Clinical and Translational Science Awards (CTSA) Program, which supports innovative solutions to advance the efficiency, quality and impact of translational science, with the ultimate goal of getting more treatments to more patients more quickly. Prior to joining NCATS, Kurilla served as the director of the Office of Biodefense Research Resources and Translational Research within the National Institute of Allergy and Infectious Diseases (NIAID), where he focused on translational efforts toward infectious disease product development, including vaccines, therapeutics and diagnostics, with emphasis on biodefense and emerging infectious disease threats. Prior to joining NIAID in 2003, Kurilla was an associate director for infectious diseases at Wyeth. He also worked in antimicrobials at DuPont and on clinical microbiology and molecular pathology at the University of Virginia Health Sciences Center.
Kurilla received his M.D. and his Ph.D. in microbiology and immunology from Duke University. He was a postdoctoral research fellow at Harvard Medical School and completed a residency in pathology at Brigham and Women’s Hospital. He received a B.S. in chemistry from the California Institute of Technology.
Hank Mansbach, MD
Hank Mansbach, MD
VP Clinical Development
UltragenyxHank Mansbach is a neurologist who, over his 19 years in the pharma/biotech industry, has served in leadership roles in the development and commercialization of numerous novel therapeutic agents. He is currently VP, Clinical Development and Ultra Programs for Ultragenyx, a clinical-stage biopharmaceutical company focused on novel treatments for rare and ultra-rare genetic diseases. One of the Ultra Programs he oversees at Ultragenyx is the individual and cohort expanded access program.
Richard Moscicki, MD
Pharmaceutical Research and Manufacturers of America (PhRMA)
Richard Moscicki, MD
Executive VP, Chief Medical Officer
Pharmaceutical Research and Manufacturers of America (PhRMA)Richard Moscicki (Mo-shis-ke), M.D., is the Executive Vice President for Science and Regulatory Advocacy and the Chief Medical Officer at Pharmaceutical Research and Manufacturers of America (PhRMA).
Dr. Moscicki came to PhRMA in 2017 after serving as the Deputy Center Director for Science Operations for the U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) since 2013. While at FDA, Dr. Moscicki brought executive direction of Center operations and leadership in overseeing the development, implementation, and direction of CDER’s programs. Previous positions include serving as Chief Medical Officer at Genzyme Corporation from 1992 to 2011, where he was responsible for worldwide global regulatory and pharmacovigilance matters, as well as all aspects of clinical research and medical affairs for the company. He served as the senior vice president and head of Clinical Development at Sanofi-Genzyme from 2011-2013.
Dr. Moscicki received his medical degree from Northwestern University Medical School. He is board certified in internal medicine, diagnostic and laboratory immunology, and allergy and immunology. He completed his residency in internal medicine, followed by a fellowship at Massachusetts General.
Hospital (MGH) in clinical immunology and immunopathology. He remained on staff at MGH and on
the faculty of Harvard Medical School from 1979 until 2013.
Richard Payne, MD
Center for Practical Bioethics
Richard Payne, MD
John B. Francis Chair
Center for Practical BioethicsRichard Payne, MD, is the John B. Francis Chair in Bioethics at the Center for Practical Bioethics in Kansas City, Missouri. As of July 1, 2017, he is the Esther Colliflower Professor of Medicine and Divinity (Emeritus) at Duke Divinity School, Duke University. At the Center Dr. Payne is the Medical Director for a pain advocacy program, Pain Alliance to Institute a National Strategy (PAINS) and co-edits PAINS policy and educational briefs. He is also directing projects addressing the ethics of conflict of interest in public-private partnerships, and the role of African-American faith communities in addressing disparities in access to palliative and hospice care, including the role of faith communities in promoting advance care planning. Dr. Payne travels extensively lecturing on bioethics and palliative care for the Center.
Dr. Payne created the Initiative to Improve Palliative Care for African-Americans (IIPCA) in 1999 as a 501c3) organization to promote education and research to improve access to pain management, palliative and end-of-life care for African-American patients. He also directed the creation a palliative care curriculum for clinicians to address the needs of African-American patients (APPEAL), which has been taught widely throughout the country. Dr. Payne created and directed a community-based palliative care program, The Harlem Palliative Care Network to address needs of medically underserved patients and families in New York City. This program eventually created the first inpatient hospice unit for the Harlem community, located in North General Hospital.
Center for Medicine in the Public Interest (CMPI)
Center for Medicine in the Public Interest (CMPI)Peter Pitts is President of the Center for Medicine in the Public Interest and Chief Regulatory Officer for Adherent Health. A former member of the United States Senior Executive Service (SES), Peter was FDA’s Associate Commissioner for External Relations, serving as senior communications and policy adviser to the Commissioner. He supervised FDA's Office of Public Affairs, Office of the Ombudsman, Office of Special Health Issues, Office of Executive Secretariat, and Advisory Committee Oversight and Management. In 2010, he was named by Modern Healthcare magazine as one of the 300 “most powerful people in American healthcare.” He is a Visiting Lecturer at the École Supérieure des Sciences Économiques et Commerciales (Paris and Singapore), and has served as an adjunct professor at Indiana University’s School of Public and Environmental Affairs and Butler University. Peter is a widely published author and commentator on matters of FDA policy and process, healthcare technology assessment and reimbursement issues, real world evidence, off label-communications, pharmacovigilance, patient-focused drug development, GMP standards, drug safety, personalized medicine, clinical trial transparency, and drug importation. He serves on the boards of several policy institutes and foundations.
Jess Rabourn, CFA
Jess Rabourn, CFA
Founder and CEO
WideTrialJess Rabourn is the founder and CEO of WideTrial, an early-stage company that serves as a third-party sponsor of multi-site, large-group Expanded Access programs. Since 2010 he has been a nationally recognized speaker on pre-approval access and, through his consulting company and non-profit projects, has provided substantial education and policy support on the topic. He is a member of the American Academy of Neurology and was lead author of the Expanded Access section of the submitted draft for FDA's Guidance on ALS Drug Development. Prior to this work, Jess spent 15 years in the investment management industry at firms in San Francisco and New York. He earned the Chartered Financial Analyst designation in 2004.
Marjorie Speers, PhD
Clinical Research Pathways
Marjorie Speers, PhD
Clinical Research PathwaysDr. Marjorie Speers is Executive Director of Clinical Research Pathways (formerly WCG Foundation), a public charity, since 2015. Clinical Research Pathways promotes compassion, inclusion and education in research. Clinical Research Pathways helps physicians and institutional review boards streamline the Food and Drug Administration's expanded access process to make it easier for desperately ill patients to try experimental medicines. Previously, beginning in 2001, Dr. Speers was the inaugural president and CEO of the Association for the Accreditation of Human Research Protection Programs (AAHRPP) until her retirement in 2013. From 1999-2001, she was the Acting Executive Director of the National Bioethics Advisory Commission, where she oversaw the Commission’s work and report on the oversight system for research involving human participants. Prior to that, she was the Deputy Associate Director for Science at the Centers for Disease Control and Prevention (CDC) where she oversaw all domestic and international research involving human participants. Dr. Speers was on the faculty at the University of Texas Medical Branch prior to joining CDC. As an epidemiologist, her research focused on risk factors for cardiovascular disease and cancer prevention.
BioCenturySteve Usdin has been Washington Editor of BioCentury since 1993, and has spent the past 25 years in the nation's capital covering political and policy issues affecting the life sciences sector. He also is BioCentury Senior Editor responsible for coverage of social issues involving biotechnology, as well as the former host of BioCentury This Week, BioCentury's public affairs television program. Steve’s reporting about biotechnology and biomedical policy has been cited in The Economist, The Wall Street Journal, the Washington Post, New Scientist and other publications. In 2012, the FDA Alumni Association named Steve the Harvey W. Wiley Lecturer, making him the first journalist to receive the Wiley Award. His book, “Engineering Communism: How Two Americans Spied for Stalin and Founded the Soviet Silicon Valley,” was published in 2005 by Yale University Press.
Executive VP, Early Access Programs
Bionical GroupTom Watson has partnered for the last 5 years with pharma and biotech organizations to design their strategy for Pre-approval Access and develop global programs, allowing patients to gain access to treatments that would otherwise be unavailable to them whilst supporting traditional development pathways. Within this time Tom has been involved in setting up and running over 200 global programs all tailored to the needs of the asset and the company. His role as Head of U.S. Business Development for IDIS Managed Access, part of the Clinigen Group, provided a platform to help patients around the world whilst developing thinking and strategy for many of the Top 20 Pharma companies in this important area.
Previously to working in this space, Tom spent 14 years within the pharmaceutical industry fulfilling a range of leadership roles including Head of Marketing for a multinational pharma company. Within this time, he led a wide range of global pre-launch and launch activities for treatments addressing areas of high unmet medical need.
Tom is a member of the New York University School of Medicine Working Group on Compassionate Use and Pre- Approval Access. He is deeply committed to helping companies with promising medicines in development and the patients they serve.
University of Michigan Medical System
Adjunct Associate Clinical Professor, Co-Chair, Expanded Access Oversight Committee
University of Michigan Medical SystemKevin Weatherwax directs the staff of the MICHR IND/IDE Assistance Program (MIAP) at the University of Michigan Health System (UMHS). He also Co-Chair’s the UMHS Expanded Access Oversight Committee to develop/enhance internal process and infrastructure to support treating physicians interested in providing access to investigational products to patients with no alternatives. Kevin has been instrumental in the development of institutional policy and processes as it relates to FDA regulated research at the University of Michigan. Under his direction, the MIAP team has consulted with over 500 investigators, including device, drug and biologics development along with regulatory support for IND/IDE clinical studies. With a multidisciplinary support focus, MIAP has interfaced with hundreds of faculty and staff across many Schools, Departments and Divisions at the University of Michigan.