Successful collaboration for research development between the NIH and patient organisations

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1 Successful collaboration for research development between the NIH and patient organisations Stephen C. Groft, Pharm.D. Office of Rare Diseases s of Health Department of Health and Human Services EURORDIS 10 th Anniversary Meeting Gaining Access to Research Resources Paris France May 4, 2007

2 Identifying Research Partners at the NIH, USA Office of Research on Women s Health Office of AIDS Research, Office of Dietary Supplements, Office of Behavioral and Social Sciences Research, Office of Disease Prevention, Office of Rare Diseases, OD Deputy and Associate Directors Administrative Offices Advisory Committee to the Director National Cancer Institute National Eye Institute National Heart Lung and Blood Institute on Aging Clinical Center Center for Information Technology Center for Scientific Review John E. Fogarty Center for Advanced Study in the Health Sciences on Alcohol Abuse and Alcoholism of Allergy and Infectious Disease of Arthritis and Musculoskeletal and Skin Diseases of Child Health and Human Development National Center for Complementary and Alternative Medicine National Center for Minority Health and Health Disparities National Center for Research Resources National Human Genome Research Institute on Drug Abuse of Deafness and Other Communication Disorders of Dental and Craniofacial Research of Diabetes and Digestive and Kidney Diseases of Biomedical Imaging and Bioengineering of Environmental Health Sciences of General Medical Sciences of Nursing Research of Mental Health of Neurological Disorders and Stroke National Library of Medicine

3 NIH Emphasis on Patient Advocacy Groups as Collaborators Institute of Medicine/National Academy of Sciences Publication - Opportunities and Public Needs: Improving Priority Setting and Public Input at NIH (1998) ih/ NIH Director - Enhancing Public Participation in NIH Activities (1998)

4 NIH Emphasis on Patient Advocacy Groups as Collaborators Advisory Councils/Special Committees Council of Public Representatives Institutional Review Boards Advice on NIH Research Priorities NHLBI, NINDS, NCI Conference for PAGs Identify Public Concerns Participate in Scientific Conferences Review of Disease-Specific Information Fact Sheets Policy Statements

5 Selected Tasks of Patient Advocacy Groups Establish Medical and Scientific Advisory Boards Support Research and Training Programs Provide Ready Access to Media Serve as Direct Links to Patients and Physicians Translate Research Results to Communities Organize Research Based Conferences and Meetings for Patients/Families/Caregivers

6 Activities of Patient Advocacy Groups Recruit Patients for Clinical Studies Identify Cohorts of Patients with Range of Phenotypic Expression Provide Developmental Assistance and Review Study Protocol Informed Consent Document Acquisition and Sharing of Patient Biomaterials Appearances Before Regulatory Agencies and Review Divisions

7 Needs of Patient Advocacy Groups Education of Leadership Science Behind Disease Knowledge of Cutting Edge Scientific Tools and Technology Knowledge of Government and Industry Research Structure, Activities, Requirements, Operating Environment, and Procedures Perspectives of Researchers Regulatory Requirements for Research Studies

8 ORD/NORD Regional Workshops for Patient Support Organizations A Model Program The Types of Research Patient Travel to Research and Treatment Sites Intramural/Extramural Research Programs Scientific Workshop Program NIH Patient Recruitment and Referral FDA Office of Orphan Products Development OOPD Grants Program and Orphan Designation Process Product Regulatory Evaluation and Research Patenting/Licensing of Genetic Material IRB/Human Subjects Protection/Ethics Issues in Research Implications of Genetic Testing

9 Selected Outcomes of the Scientific Conferences Program Identify Research Opportunities Establish Research Priorities Develop Program Announcements and Solicit R01 Applications Establish Diagnostic and Monitoring Criteria Develop Animal Models Support Registries - Patient and Tissue Develop Research Protocols, Collaborative Research Arrangements, and Plan Clinical Trials Disseminate Results to Targeted Professional and Voluntary Health Organizations Co-Sponsor with Patient Advocacy Groups, Academic Investigators, Industry, Foundations, Intramural and Extramural Research Programs

10 Meeting Patient and Family Needs Identify and Expand Worldwide Partnerships and Collaborations of Patient Advocacy Groups Identify Economic Impact of Rare Diseases on Families and Individuals Expand Training Programs on Living and Coping with Rare and Genetic Diseases Gaining Acceptance for Disabilities and Improving Educational Opportunities for Patients Maximize Access to Rehabilitation Therapies Physical, Hearing, Speech, Vocational, Occupational, Assure Worldwide Access to Safe and Effective Products for the Prevention, Diagnosis, and Treatment of Rare Diseases

11 Coordinated Efforts for Successful Orphan Product Development/Rare Diseases Research Pharmaceutical, Biotechnology, and Medical Devices Industries Academic and Private Foundation Research Communities-Multidisciplinary Research Efforts Medical Specialty Societies and Healthcare Providers Patient Advocacy Groups Federal Government Regulatory Reimbursement Research Health Care Services Prevention

12 How Can YOU Help? Societal Needs and Values Mobilize Health Professionals and Society to Take Action Obtain Cooperative and Collaborative Support From All Nations and Partners in Health Research and Product Development Improve Health Literacy of Populations to Enable Information-Based Decision Making Reduce Disparities in Health with Ready Access to Information and Interventions for Diagnosis, Prevention, and Treatment of Rare Diseases

13 How Can YOU Help? Research Needs Provide Global Access to Clinical Studies and Clinical Trials Develop Globalization of Research Efforts and Common Protocols with Multidisciplinary Research Teams Continue Efforts for Harmonization of Research Data for Regulatory Purposes Establish Better Definitions of Patient Responders with Development of Appropriate Biomarkers and Surrogate Endpoints for Safety and Efficacy Expand Global Linkages of Patient Advocacy Group Networks Develop Inclusive Web-Based Inventory of Global Rare Diseases Research/Intervention Activities and Information Resources

14 Office of Rare Diseases (ORD) - Collaborative Clinical Research Programs Intramural Research and Training Programs Bench to Bedside Research Program with Extramural Research Program Clinical and Biochemical Genetics Training Program (NHGRI) Extramural Research Program Scientific Conferences Rare Diseases Clinical Research Network (RDCRN) CETT Genetic Testing Program International Congress on Rare Diseases and Orphan Drugs (ICORD 2007 September 14-15, 15, Brussels) Information Development and Dissemination Activities Trans-NIH Working Group on Rare Diseases Research Inventory of Bio-specimen Collection, Storage, and Distribution Systems (RAND Corporation) OTT/ORD Technology Transfer Activities

15 Information Development, Dissemination, and Education Activities National Library of Medicine Gateway NORD, Genetic Alliance, DIRLINE > 1200 PAG Clinical Trials.gov 5,429 Studies Recruiting for 875 Rare Diseases (11,676 Total for 1,024 Rare Diseases) Pub Med/MEDLINE 4800 Journals from 70 Countries, 750 Million Searches/year Gene Tests (1294 Diseases) (Euro Gen Test Links)) 1134 Clinics Diseases Clinical Tests 606 Laboratories Diseases Research Only Laboratories Seminars for PAG Leadership National Coalition for Health Professional Education in Genetics (NCHPEG)

16 Technology Transfer Activities- OTT/ORD Office of Technology Transfer (Neglected Diseases, Rare Diseases) Available Technology from Government ( >500 technologies) and Not for Profit Organizations - Not-for for-profit Academic Research Centers and Others Web site: /raredisease_ovrvw.asp Press release: htm

17 Human Biospecimen Collections > 307 million specimens stored in the United States Accumulating at a rate of > 20 million per year > 30 million specimens have been collected specifically for research purposes: Stored at >300 bio-repositories in the United States > 250 million specimens originally collected for diagnostic or therapeutic reasons Stored in pathology labs

18 The Genetic and Rare Diseases Information Center (NHGRI/ORD) >18,000 Inquiries ( ) > 4,640 Rare Diseases or Conditions > 6,740 Related Terms and Links Toll-free (USA) International Access Number: Fax:

19 ORD Website Rare Diseases Information Pub Med, NLM Gateway Research and Clinical Trials - CRISP, ClinicalTrials.gov Patient Support Groups DIRLINE, NORD, Genetic Alliance, > 1200 Patient Advocacy Groups, Patient Travel & Lodging Genetics Information Gene Tests, OMIM, NCHPEG Research Resources Scientific Workshops, Archived Reports Website Trends

20 Office of Rare Diseases - Staff Ms. Mary Demory Ms. Marita Eddy (Angel Flight) Dr. John Ferguson (Consultant) Dr. Stephen C. Groft Dr. Rashmi Gopal-Srivastava Mr. Christopher Griffin Ms. Henrietta Hyatt-Knorr Ms. Sharon Macauley Ms. Geraldine Pollen (Consultant) Dr. Giovanna Spinella (Consultant) Dr. William Gahl (Clinical Director, NHGRI)

21 Office of Rare Diseases s of Health 6100 Executive Boulevard Room 3B-01, MSC Bethesda, MD Voice: Fax: ORD@nih.gov Website:

Rare Diseases Collaborative Research and Development Activities

Rare Diseases Collaborative Research and Development Activities Stephen C. Groft, Pharm.D. Office of Rare Diseases s of Health Department of Health and Human Services American Academy of Dermatology 65