FDA
Investigational New Drug Application Resources
CDER
Investigational New Drug Applications
http://www.fda.gov/cder/regulatory/applications/ind_page_1.htm
IND Drug Review Process (flowchart)
http://www.fda.gov/cder/handbook/ind.htm
Guidance for Industry Q & A - Content and Format
of INDs for Phase 1 Studies of Drugs, Including
Well-Characterized, Therapeutic, Biotechnology-Derived
Products
http://www.fda.gov/cder/guidance/3591fnl.htm
Office of Drug Evaluation IV (ODE IV) Pre-Investigational
New Drug Application (IND) Consultation Program
http://www.fda.gov/cder/ode4/preind/default.htm
CBER (blood,
vaccines, cellular/gene therapy, tissue, devices)
Investigational New Drug Applications
http://www.fda.gov/cber/ind/ind.htm
CBER Frequently Asked Questions
http://www.fda.gov/cber/faq.htm
Guidance Documents
http://www.fda.gov/cber/guidelines.htm
FDA: GENERAL
RESOURCES
REGULATORY
Office of Orphan Products Development
http://www.fda.gov/orphan/
Manufacturers Assistance and Technical Training
Branch (MATTB)
The Manufacturers Assistance and Technical Training
Branch (MATTB) informs industry and trade associations
of the status of CBER policies and initiatives through
regular information dissemination and training.
MATTB also serves as the CBER focal point for industry
and trade associations to provide meeting support,
and coordinates external meetings with other FDA
Centers.
http://www.fda.gov/cber/manufacturer.htm
Draft Guidance for Industry: Information Program
on Clinical Trials for Serious or Life-Threatening
Diseases and Conditions (Revision 1) - 1/26/2004
- (PDF),
(Text)
Guidance for Industry: Fast Track Drug Development
Programs - Designation, Development, and Application
Review - 7/21/2004 - (PDF),
(Text)
Appendix 2 (PDF)
Appendix 3 - CDER
MAPP 6020.3 (PDF), CBER
SOPP 8405(Text)
Appendix 4 (PDF)
Draft Guidance for FDA Review Staff and Sponsors:
Content and Review of Chemistry, Manufacturing,
and Control (CMC) Information for Human Gene Therapy
Investigational New Drug Applications (INDs) - 11/8/2004
- (PDF),
(Text)
Draft Guidance for Reviewers: Instructions and
Template for Chemistry, Manufacturing, and Control
(CMC) Reviewers of Human Somatic Cell Therapy Investigational
New Drug Applications (INDs) - 8/15/2003 - (PDF),
(Text)
Draft Guidance for Clinical Trial Sponsors on the
Establishment and Operation of Clinical Trial Data
Monitoring Committees - 11/15/2001 - (PDF),
(Text)
Guidance for Human Somatic Cell Therapy and Gene
Therapy
http://www.fda.gov/cber/gdlns/somgene.pdf
- 1998
Guidance for Industry: Supplemental Guidance on
Testing for Replication Competent Retrovirus in
Retroviral Vector Based Gene Therapy Products and
During Follow-up of Patients in Clinical Trials
Using Retroviral Vectors
http://www.fda.gov/cber/gdlns/retrogt1000.pdf
- 2000
Points to Consider in the Characterization of Cell
Lines Used to Produce Biologicals
http://www.fda.gov/cber/gdlns/ptccell.pdf
Good Clinical Practice in FDA-Regulated Clinical
Trials
http://www.fda.gov/oc/gcp/default.htm
FUNDING
OPPORTUNITIES
Orphan Products Grant Program
http://www.fda.gov/orphan/grants/index.htm
NIH RESOURCES
NIH
CLINICAL TRIAL RESOURCES
Office of Biotechnology Activities
http://www4.od.nih.gov/oba/
Recombinant DNA and Gene Transfer
http://www4.od.nih.gov/oba/Rdna.htm
RAC – Recombinant DNA Advisory Committee
http://www4.od.nih.gov/oba/rac/aboutrdagt.htm
GeMCRIS – Genetic Modification Clinical Research
Information System
http://www4.od.nih.gov/oba/RAC/GeMCRIS/GeMCRIS.htm
NIH Guidelines for Research Involving Recombinant
DNA Molecules
http://www4.od.nih.gov/oba/rac/guidelines/guidelines.html
Office of Human Subjects Research
http://www.nihtraining.com/ohsrsite/
Bioethics Resources on the Web
http://www.nih.gov/sigs/bioethics/casestudies.html
Overall NINDS Clinical Trials Program Information
http://www.ninds.nih.gov/funding/research/clinical_research/index.htm
NINDS Clinical Trial Planning Grant
http://grants2.nih.gov/grants/guide/pa-files/PAR-03-051.html
NINDS Clinical Trial Planning Grant (R34) Program
http://grants.nih.gov/grants/guide/pa-files/PA-04-008.html
Pilot Studies for Clinical Trials in Neurological
Disorders http://grants.nih.gov/grants/guide/pa-files/PAR-03-174.html
NIH
TRAINING RESOURCES
Ruth L. Kirschstein National Research Service Awards
for Postdoctoral Fellowships in Muscle Disease Research
http://grants.nih.gov/grants/guide/pa-files/PA-05-052.html
NINDS Mentored Research Scientist Development Awards
in Translational Research
http://grants.nih.gov/grants/guide/pa-files/PAR-02-138.html
Mentored Clinical Investigator Career Development
Awards in Muscle Disease Research
http://grants.nih.gov/grants/guide/pa-files/PA-05-051.html
NIH
TRANSLATIONAL RESEARCH FUNDING OPPORTUNITIES
Overall NINDS Translational Research Program http://www.ninds.nih.gov/funding/research/translational/index.htm
NINDS Exploratory/Developmental Projects in Translational
Research http://grants.nih.gov/grants/guide/pa-files/PAR-02-138.html
NINDS Cooperative Program in Translational Research
http://grants.nih.gov/grants/guide/pa-files/PAR-02-139.html
NINDS Mentored Research Scientist Development Awards
in Translational Research http://grants.nih.gov/grants/guide/pa-files/PAR-02-140.html
Announcement of NINDS High Throughput Drug Screening
Service and Call for Assay Proposals http://grants1.nih.gov/grants/guide/notice-files/NOT-NS-04-005.html
Administrative Supplements for the Sharing and
Distribution of Mouse Genetic Models http://grants2.nih.gov/grants/guide/notice-files/NOT-NS-04-009.html
Novel Approaches to Enhance Animal Stem Cell Research
http://grants.nih.gov/grants/guide/pa-files/PA-04-125.html
Centers of Excellence in Translational Human Stem
Cell Research http://grants.nih.gov/grants/guide/rfa-files/RFA-NS-05-005.html
Small Business Innovation Research (SBIR) and Small
Business Technology Transfer (STTR) to Improve The
Chemistry and Targeted Delivery of RNAi Molecules
http://grants.nih.gov/grants/guide/pa-files/PA-05-041.html
NIH
GENERAL RESOURCES
Muscular Dystrophy Coordinating Committee
http://www.ninds.nih.gov/find_people/groups/mdcc/
CINCH (Consortium for Clinical Investigations
of
Neurological Channelopathies )
http://www.rarediseasesnetwork.org/cinch/index.htm
NINDS Human Genetics Resource Center
http://locus.umdnj.edu/ninds/
NINDS: Increasing Quality of Life in Mobility Disorders
http://grants.nih.gov/grants/guide/pa-files/PA-02-111.html
NINDS Institutional Center Core Grants to Support
Neuroscience Research http://grants.nih.gov/grants/guide/pa-files/PAR-02-059.html
Precursor Cells in Skeletal Muscle Repair and Hypertrophy
http://grants.nih.gov/grants/guide/pa-files/PA-02-136.html
Muscular Dystrophy: Pathogenesis and Therapies
http://grants.nih.gov/grants/guide/pa-files/PA-05-038.html
The SMA Project: A Collaborative Program to Accelerate
Therapeutics Development for Spinal Muscular Atrophy
(SMA)
http://www.smaproject.org/Solicitations/opensolicitations.htm
NIAMS, NICHD, NINDS: Support for Muscular Dystrophy
Workshops and Research Conferences
http://grants1.nih.gov/grants/guide/pa-files/par-03-176.html
DHHS, NIH, NINDS: Assay Development for High Throughput
Molecular Screening (R03/R21)
http://grants.nih.gov/grants/guide/rfa-files/rfa-rm-06-004.html
CENTERS FOR
DISEASE CONTROL
Duchenne/Becker Muscular Dystrophy Projects Page
(MD StarNet, Newborn screening, Family Needs Assessment
and Cardiac Health in Female Carriers)
http://www.cdc.gov/ncbddd/duchenne/cdc.htm
BURROUGHS
WELLCOME FUND RESOURCES
Clinical Scientists Awards in Translational Research
http://www.bwfund.org/programs/translational/clinical_scientists_main.html
HUMAN
SUBJECT PROTECTION
ADVERSE
EVENT REPORTING
Gene Therapy (FDA)
http://www.fda.gov/cber/ltr/gt110599.htm
Gene Therapy (NIH and FDA): GeMCRIS – Genetic
Modification Clinical Research Information System
http://www4.od.nih.gov/oba/RAC/GeMCRIS/GeMCRIS.htm
MedWatch: The FDA Safety Information and Adverse
Event Reporting Program(Post-marketing surveillance)
http://www.fda.gov/medwatch/
GENERAL
HUMAN SUBJECT PROTECTION
NIH – Office of Human Subjects Research
http://www.nihtraining.com/ohsrsite/
NIH – Bioethics Resources on the Web
http://www.nih.gov/sigs/bioethics/casestudies.html
DHHS - Office for Human Research Protections
http://ohrp.osophs.dhhs.gov/
FDA - Guidance for Institutional Review Boards
and Clinical Investigators
http://www.fda.gov/oc/ohrt/irbs/default.htm
FDA - A Guide to Informed Consent
http://www.fda.gov/oc/ohrt/irbs/informedconsent.html
The Belmont Report: Ethical Principles and Guidelines
for the Protection of Human
Subjects of Research
http://ohsr.od.nih.gov/mpa/belmont.php3
Nuremberg Code
http://ohsr.od.nih.gov/nuremberg.php3
Helsinki Declaration
http://ohsr.od.nih.gov/helsinki.php3
Federal Policy for the Protection of Human Subjects
http://ohrp.osophs.dhhs.gov/humansubjects/guidance/45cfr46.htm
ANIMAL
WELFARE
USDA Animal Welfare Act
http://www.aphis.usda.gov/ac/awa.html
Institutional Animal Care and Use Committees (IACUC)
http://www.iacuc.org/
NIH - Office of Animal Care and Use (OACU)
http://oacu.od.nih.gov/
American College of Laboratory Animal Medicine
(ACLAM)
http://www.aclam.org/
Association for the Assessment and Accreditation
of Laboratory Animal Care International
http://www.aaalac.org/
TISSUE
AND REAGENT BANKS
Brain and Tissue Bank Resources
http://www.ninds.nih.gov/find_people/voluntary_orgs/vol_org_sub_BB.htm
Cord Blood Registry
http://www.cordblood.com
BrainNet Europe
http://www.brainnet-europe.org
Medical Solutions Biomaterials Resource
http://www.biomaterialsresource.com/
Facioscapulohumeral Muscular Dystrophy (FSHD) Tissue
Bank
http://www.mda.org/research/ct-fshtissue.html
Morris Neuromuscular Antibody Collection
www.rjah.nhs.uk/cind/morrisge/mabs.htm
ANIMAL
MODELS
JACKSON LABORATORIES
Neuromuscular Defects
http://jaxmice.jax.org/list/ra121.html
OTHER
SOURCES
Amyotrophic lateral sclerosis (ALS) – SOD1
rat
http://www.taconic.com/emerging/002148.htm
REGISTRIES
National Registry of Veterans with Amyotrophic
Lateral Sclerosis
http://www.va.gov/durham/alsregistry.asp
ALS Patient Care Database
http://www.umassmed.edu/outcomes/als/
The Myositis Association
http://www.myositis.org/patient_registry/index.html
The International Spinal Muscular Atrophy Patient
Registry
http://www.fsma.org/registry2002.shtml
National Registry of Myotonic Dystrophy & FSHD
Patients and Family Members
http://www.urmc.rochester.edu/nihregistry/
North American Charcot-Marie-Tooth disease (CMT)
Database
http://www.med.wayne.edu/neurology/clin_programs/labs/CMT/index.htm
The Utah Dystrophinopathy Project
http://dystrophy.genetics.utah.edu/
Dysferlin Deficiency
http://www.jain-foundation.org
CLINICAL
STUDIES AND DATABASES
CIDD (Clinical Investigation of Duchenne Dystrophy);
Clinical research study group that conducted natural
history and therapeutic studies between 1979 and
1987
http://archneur.ama-assn.org/cgi/content/abstract/44/8/808
Leiden Mutation Databases
http://www.dmd.nl/
Human Gene Mutation Database
http://archive.uwcm.ac.uk/uwcm/mg/hgmd0.html
MD STARnet
http://www.cdc.gov/ncbddd/duchenne/MD%20STARNet%20Factsheet.pdf
and http://www.cdc.gov/ncbddd/duchenne/cdc.htm
Utah dystrophinopathy Project
http://dystrophy.genetics.utah.edu
NIDRR Rehabilitation Research & Training Center
in Neuromuscular Diseases (RRTC/ NMD): Projects
http://nmdinfo.net/program.html
CINCH (Consortium for Clinical Investigations of
Neurological Channelopathies )
http://www.rarediseasesnetwork.org/cinch/index.htm
PRACTICE
PARAMETERS AND CONSENSUS STATEMENTS
Pulmonary
Respiratory Care of the Patient With Duchenne Muscular
Dystrophy. American Thoracic Society Consensus Statement
– March 2004.
http://www.thoracic.org/sections/publications/statements/pages/respiratory-disease-pediatric/duchenne1-10.html
Steroids
Corticosteroid Treatment of Duchenne Muscular Dystrophy.
Report of the Quality Standards Subcommittee of
the American Academy of Neurology and the Practice
Committee of the Child Neurology Society –
January 2005.
http://www.neurology.org/cgi/reprint/64/1/13.pdf
Treatment of Duchenne Muscular Dystrophy: Defining
the gold standards of management in the use of corticosteroids.
Report on the 124 ENMC International Workshop –
April 2004.
Glucocorticoid corticosteroids for Duchenne muscular
dystrophy. Cochrane Database System Review. –
2005.
http://www.mrw.interscience.wiley.com/cochrane/clsysrev/articles/CD003725/frame.html
Cardicac
The management of cardiac involvement in muscular
dystrophy and myotonic dystrophy. 107th
ENMC International Workshop – June 2002.http://download.journals.elsevierhealth.com/pdfs/journals/0960-8966/PIIS0960896602002134.pdf
PUBLICATIONS
Cornetta K and Smith F. Regulatory Issues for Clinical
Gene Therapy Trials. Human Gene Therapy. 2002;13:1143-1149.
Escolar D, Henricson K, Pasquali L, Gorni K, and
Hoffman E. Collaborative translational research
leading to multicenter clinical trials in Duchenne
muscular dystrophy: the Cooperative International
Neuromuscular Research Group (CINRG). Neuromuscular
Disorders. 2002; S147-S154.
Grilley B, and Gee A. Gene transfer: Regulatory
issues and their impact on the clinical investigator
and the good manufacturing production facility.
Cytotherapy. 2003;5(3):197-207.
Hibberd PL, Weiner DL. Monitoring participant safety
in phase I and II interventional trials: Options
and controversies. J Investig Med. 2004 Nov;52(7):446-52.
DiMasi J, Hansen R, and Grabowski H. The price
of innovation: New estimates of drug development
costs. Journal of Health Economics. 2003; 22:151-185.
http://www.cptech.org/ip/health/econ/dimasi2003.pdf
NEUROLOGY
(index to neuromuscular disease)
www.neurology.org/collections/index.shtml#neuromuscular_disease
www.neurology.org/cgi/collection/anterior_nerve_cell_disease
www.neurology.org/cgi/collection/myasthenia
www.neurology.org/cgi/collection/muscle_disease
www.neurology.org/cgi/collection/lamberteaton_syndrome
www.neurology.org/cgi/collection/amyotrophic_lateral_sclerosis_
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