Link to RDCRC Slides

Rare Diseases Clinical Research Network: Rare Diseases Clinical Research Network: Pre-Application meetingPre-Application meeting

Preparing to submit a U54 Application for Preparing to submit a U54 Application for RDCRC (RFA-OD-08-001) RDCRC (RFA-OD-08-001)

andandDMCC (RFA-OD-08-002)DMCC (RFA-OD-08-002)

Monday, March 17, 2008Monday, March 17, 20081:00 PM EST1:00 PM EST

6001 Executive Blvd. Rockville, Maryland6001 Executive Blvd. Rockville, Maryland

Meeting AgendaMeeting Agenda

IntroductionIntroduction ((Dr. Stephen C. GroftDr. Stephen C. Groft))

Overview of RDCRN RFAsOverview of RDCRN RFAs 1) RDCRC RFA 1) RDCRC RFA ((Dr. Rashmi Gopal SrivastavaDr. Rashmi Gopal Srivastava))

2) DMCC RFA2) DMCC RFA ((Dr. David EcksteinDr. David Eckstein))

Q & A SessionQ & A Session Answer Questions Related to Submission of ApplicationAnswer Questions Related to Submission of Application- Live audience: use microphone- Live audience: use microphone- Video-Cast Audience: send email to - Video-Cast Audience: send email to “ “Live Event Feedback Form” (Live Event Feedback Form” ([email protected]@od.nih.gov))

Slides will be posted: Slides will be posted: http://http://rarediseasesinfo.nih.govrarediseasesinfo.nih.gov

mailto:[email protected]

http://rarediseasesinfo.nih.gov/

http://rarediseasesinfo.nih.gov/

RDCRN: GoalRDCRN: Goal

The Rare Diseases Clinical Research Network The Rare Diseases Clinical Research Network (RDCRN) should facilitate (RDCRN) should facilitate

• Identification of biomarkers for disease risk, disease Identification of biomarkers for disease risk, disease severity/activity, and severity/activity, and

• Identification of measures of clinical outcomes Identification of measures of clinical outcomes appropriate for applicability to clinical trialsappropriate for applicability to clinical trials

• Encourage development of new approaches to Encourage development of new approaches to diagnosis, prevention, and treatment of rare diagnosis, prevention, and treatment of rare diseases. diseases.

Rare Disease Clinical Research Rare Disease Clinical Research Network Network

• Reissuance of RDCRN RFAReissuance of RDCRN RFAss (two RFAs for two (two RFAs for two components of the same program)components of the same program)

• RDCRC RFA PartnersRDCRC RFA Partners *NCI, NHLBI, *NIA, *NIAAA, *NIAID, NIAMS, NICHD, *NCI, NHLBI, *NIA, *NIAAA, *NIAID, NIAMS, NICHD,

*NIDCR, NIDDK, NINDS*NIDCR, NIDDK, NINDS

• DMCC RFA PartnerDMCC RFA Partner NINDSNINDS

*New partners*New partners

RDCRN StructureRDCRN Structure

RDCRC 1

Rare Diseases ClinicalResearch Network

Data Management Coordinating Center

(DMCC)

Patient Advocacy Groups

RDCRC 2

NIH ICs Project ScientistsORD Program Staff

RDCRC 3

RDCRC 4

RDCRC 5

RDCRC 6

RDCRC 7

RDCRC 8

Rare Diseases Clinical Research Rare Diseases Clinical Research Consortia (RDCRC)Consortia (RDCRC)

RFA-OD-08-001

Dr. Rashmi Gopal-Srivastava

NIH Office of Rare Diseases

National Institute of Mental Health

National Institute of Neurological Disorders and Stroke

National Library of Medicine

The View of the NIHThe View of the NIH

National Cancer Institute

National Institute on Aging

National Heart Lung and Blood Institute

National Eye Institute

Clinical Center Center for Scientific Review

Center for Information Technology

National Institute of Environmental Health Sciences

National Institute of General Medical Sciences

National Institute of Deafness and Other Communication Disorders

National Institute of Dental and Craniofacial Research

John E. Fogarty Center for Advanced Study in the Health Sciences

National Center for Complementary and Alternative Medicine

National Center for Minority Health and Health Disparities

National Center for Research Resources

National Institute of Nursing Research

National Institute of Arthritis and Musculoskeletal and Skin Diseases

National Institute of Allergy and Infectious Disease

National Institute on Alcohol Abuse and Alcoholism

National Institute on Drug Abuse

National Institute of Biomedical Imaging and Bioengineering

National Human Genome Research Institute

National Institute of Diabetes and Digestive and Kidney Diseases

National Institute of Child Health and Human Development

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, OPASI

Office of NIH DirectorDeputy and Associate

DirectorsAdministrative Offices

Advisory Committee to the Director

RDCRC RFA: IC Program ContactRDCRC RFA: IC Program Contact

• NCI: Elizabeth Read-Connole, Ph.D.NCI: Elizabeth Read-Connole, Ph.D.• NHLBI: 1) For rare heart diseases: Sonia I. Skarlatos, Ph.D., FAHA NHLBI: 1) For rare heart diseases: Sonia I. Skarlatos, Ph.D., FAHA

2) For rare lung diseases: Hannah H. Peavy, M.D. 3) For rare blood 2) For rare lung diseases: Hannah H. Peavy, M.D. 3) For rare blood diseases: Andrei L. Kindzelski, M.D., Ph.D.diseases: Andrei L. Kindzelski, M.D., Ph.D.

• NIA: Susan Nayfield, MDNIA: Susan Nayfield, MD• NIAAA: Svetlana Radaeva, Ph.D.NIAAA: Svetlana Radaeva, Ph.D.• NIAID: Josiah Wedgwood MD PhDNIAID: Josiah Wedgwood MD PhD• NIAMS: James Witter MD, PhDNIAMS: James Witter MD, PhD• NICHD: Mary Lou Oster-Granite, Ph. D.NICHD: Mary Lou Oster-Granite, Ph. D.• NIDCR: Yasaman Shirazi, Ph.D.NIDCR: Yasaman Shirazi, Ph.D.• NIDDK: Catherine McKeon, Ph.D. NIDDK: Catherine McKeon, Ph.D. • NINDS: 1) Danilo A. Tagle, Ph.D. 2) Randall R. Stewart, Ph.D. NINDS: 1) Danilo A. Tagle, Ph.D. 2) Randall R. Stewart, Ph.D.

NoteNote: Also see GM contacts and : Also see GM contacts and “IC-Specific Research Area “IC-Specific Research Area of Interest”of Interest”

http://grants.nih.gov/grants/guide/contacts/rfa-od-08-001_contacts.htmhttp://grants.nih.gov/grants/guide/contacts/rfa-od-08-001_contacts.htm

http://grants.nih.gov/grants/guide/contacts/rfa-od-08-001_contacts.htm

Meeting Agenda: RDCRC RFAMeeting Agenda: RDCRC RFA

OverviewOverview

• Time LinesTime Lines

• RFA PurposeRFA Purpose

• RequirementsRequirements

• Review of Applications Review of Applications

• RemindersReminders

Your Questions?Your Questions?

Timeline for RFA-OD-08-001Timeline for RFA-OD-08-001

• Letter of Intent (optional)Letter of Intent (optional) July 20, 2008 July 20, 2008

• Application ReceiptApplication Receipt August 20, 2008 August 20, 2008

• Peer ReviewPeer Review January-February, 2009 January-February, 2009

• Council ReviewCouncil Review May, 2009 May, 2009

• Earliest Anticipated July, 2009Earliest Anticipated July, 2009

Award DateAward Date

RDCRC Letter of Intent: ContentRDCRC Letter of Intent: Content

• Draft Consortia (RDCRC) TitleDraft Consortia (RDCRC) Title

• RFA ID: RFA ID: OD-08-001, Rare Diseases Clinical Research OD-08-001, Rare Diseases Clinical Research

Consortia for RDCRNConsortia for RDCRN

• Principal Investigator, with contact infoPrincipal Investigator, with contact info

• Other Key PersonnelOther Key Personnel

• Participating InstitutionsParticipating Institutions

• (Optional: Brief description of projects)(Optional: Brief description of projects)

Submit to: Submit to: Rashmi Gopal SrivastavaRashmi Gopal Srivastava ([email protected])([email protected])


OverviewOverview




• Review of ApplicationsReview of Applications



Purpose of RDCRC RFAPurpose of RDCRC RFA

The purpose of the RDCRC is to facilitate clinical research The purpose of the RDCRC is to facilitate clinical research in rare diseases through support for in rare diseases through support for

• collaborative clinical research in rare diseases, including collaborative clinical research in rare diseases, including longitudinal studies of individuals with rare diseases, longitudinal studies of individuals with rare diseases, clinical studies and/or phase I , II and II/III trials; clinical studies and/or phase I , II and II/III trials;

• training of investigators in clinical research of rare training of investigators in clinical research of rare diseases; diseases;

• pilot/demonstration projects pilot/demonstration projects

• access to information related to rare diseases for basic access to information related to rare diseases for basic and clinical researchers, academic and practicing and clinical researchers, academic and practicing physicians, patients, and the lay public. (Website physicians, patients, and the lay public. (Website resource for education and research in rare diseases)resource for education and research in rare diseases)


OverviewOverview




• Review of Applications Review of Applications



RDCRC: Required Components/ActivityRDCRC: Required Components/Activity

Both New and Renewal applications Both New and Renewal applications focused on at least focused on at least three rare diseasesthree rare diseases will be accepted. will be accepted.

Required components of an RDCRC (U54) application Required components of an RDCRC (U54) application include include • A minimum of two clinical research projects A minimum of two clinical research projects

(at least one of them must be a longitudinal study)(at least one of them must be a longitudinal study)• At least one pilot/demonstration project At least one pilot/demonstration project • A training (career development) component A training (career development) component • A website for educational and research resources in A website for educational and research resources in

rare diseases rare diseases • An administrative unit andAn administrative unit and• Collaboration with patient support organization(s) Collaboration with patient support organization(s)

Each RDCRC will…..Each RDCRC will…..

• Perform collaborative clinical research focused on a Perform collaborative clinical research focused on a group of group of at least three rare diseasesat least three rare diseases that are relevant that are relevant to the interests of the participating NIH ICs. to the interests of the participating NIH ICs.

• Consist of a consortium of clinical investigators, Consist of a consortium of clinical investigators, institutions, and relevant organizations, including institutions, and relevant organizations, including patient support organizationspatient support organizations

• Train new investigators in rare diseases research, Train new investigators in rare diseases research, and and

• Provide content for an internet resource site on rare Provide content for an internet resource site on rare diseases. diseases.

Each RDCRC application must include the Each RDCRC application must include the following components and activities:following components and activities:

A Description ofA Description of Overall Clinical Research Program, Overall Clinical Research Program, Leadership & ResourcesLeadership & Resources::

• RDCRC Program Introduction and Statement of RDCRC Program Introduction and Statement of ObjectivesObjectives

• RDCRC Scientific and Administrative LeadershipRDCRC Scientific and Administrative Leadership• Multidisciplinary Team Involving Patient Support Multidisciplinary Team Involving Patient Support

Organizations and collaborationsOrganizations and collaborations• Environment and ResourcesEnvironment and Resources• Institutional Commitment Institutional Commitment • Access to Rare Diseases Patient PopulationAccess to Rare Diseases Patient Population• Data and Research Resources Sharing PlanData and Research Resources Sharing Plan

1) Clinical Research Projects for 1) Clinical Research Projects for Observational/Longitudinal Studies and/or Observational/Longitudinal Studies and/or clinical trialsclinical trials

(At least two projects are required and one of them (At least two projects are required and one of them must be a longitudinal study)must be a longitudinal study)

• Describe the rationale for the planned clinical studies Describe the rationale for the planned clinical studies and longitudinal assessment of subjects. Strategies and longitudinal assessment of subjects. Strategies for recruitment, retention, assessment, and analysis for recruitment, retention, assessment, and analysis must be included. must be included.

• The clinical studies could include strategies for The clinical studies could include strategies for assessing current therapeutic interventions, and assessing current therapeutic interventions, and phase I, II or II/III clinical trials. phase I, II or II/III clinical trials.

Clinical Research Projects: Longitudinal Clinical Research Projects: Longitudinal StudiesStudies

• Applicants must propose clinical research projects Applicants must propose clinical research projects that can characterize and more completely define the that can characterize and more completely define the disease and its course for the rare diseases. These, disease and its course for the rare diseases. These, in general, will be observational (non-interventional), in general, will be observational (non-interventional), such as longitudinal or natural history studies of such as longitudinal or natural history studies of patients with the given disease. patients with the given disease.

• The study design and objectives should take in to The study design and objectives should take in to consideration what information regarding the rare consideration what information regarding the rare disease population would be needed in order to disease population would be needed in order to pursue clinical trials for that rare disease. pursue clinical trials for that rare disease.

• Projects for time period of five years.Projects for time period of five years.

Clinical Research ProjectsClinical Research Projects: : Longitudinal Longitudinal Studies (Cont.)Studies (Cont.)

• Applicants should approach a longitudinal study with the Applicants should approach a longitudinal study with the question: What knowledge/tools are needed regarding the question: What knowledge/tools are needed regarding the rare disease in order to design efficient efficacy trials for this rare disease in order to design efficient efficacy trials for this rare disease? rare disease?

• Even if there are no treatments currently proposed for the Even if there are no treatments currently proposed for the rare diseases under study, a longitudinal study should be rare diseases under study, a longitudinal study should be designed with the intention that if a treatment was suddenly designed with the intention that if a treatment was suddenly available, what knowledge (outcome measures, features of available, what knowledge (outcome measures, features of disease course, markers of disease or subpopulations of the disease course, markers of disease or subpopulations of the rare disease that may alter disease course, etc.) about the rare disease that may alter disease course, etc.) about the rare disease over time would be important to have to design rare disease over time would be important to have to design an appropriate treatment (efficacy) trial. an appropriate treatment (efficacy) trial.

2) Pilot/Demonstration Projects2) Pilot/Demonstration Projects

(At least one project is required)(At least one project is required)

• Propose pilot projects that generate feasibility data and have Propose pilot projects that generate feasibility data and have the most promising clinical research potentialthe most promising clinical research potential. .

• To support studies of a limited duration, of 2 years or less. To support studies of a limited duration, of 2 years or less.

• Include description and provide rationale for the planned pilot Include description and provide rationale for the planned pilot projects. projects.

• Must be maintained throughout the entire term of the Must be maintained throughout the entire term of the cooperative agreement award.cooperative agreement award.

• Examples also include development of novel laboratory Examples also include development of novel laboratory assays and clinical instruments.assays and clinical instruments.

• Depending on the state of knowledge of the particular Depending on the state of knowledge of the particular diseases, the pilot studies could include strategies for diseases, the pilot studies could include strategies for assessing current therapeutic interventions, phase I, II or II/III assessing current therapeutic interventions, phase I, II or II/III clinical trials. clinical trials.

3) Training (career development) 3) Training (career development) ComponentComponent

• Each application must include a plan for training.Each application must include a plan for training.

• The RDCRC should provide a unique environment The RDCRC should provide a unique environment for clinical research that will prepare new scientists for clinical research that will prepare new scientists for careers in this field. for careers in this field.

• A minimum of $50,000 direct costs per yearA minimum of $50,000 direct costs per year from the from the RDCRC budget must be dedicated to this programRDCRC budget must be dedicated to this program

• Include criteria and process of selecting candidates.Include criteria and process of selecting candidates.

• Include a list and short description of potential Include a list and short description of potential candidates. candidates.

4) Website resource for education and 4) Website resource for education and research in rare diseases:research in rare diseases:• Describe resources to be included in a web site for Describe resources to be included in a web site for

education and research in rare diseases (should education and research in rare diseases (should include links or materials for lay public, patients, include links or materials for lay public, patients, basic and clinical researchers, and clinicians). basic and clinical researchers, and clinicians).

• Examples: contacts for animal modelsExamples: contacts for animal models;; availability of availability of tissue, serum, specimens, DNA, etc; antibodies and tissue, serum, specimens, DNA, etc; antibodies and research reagents; genetic resources; registries; research reagents; genetic resources; registries; education materials; and/or diagnostic flow charts. education materials; and/or diagnostic flow charts.

• Each RDCRC and the DMCC must agree to work Each RDCRC and the DMCC must agree to work cooperatively to develop the web site resource and cooperatively to develop the web site resource and provide content related to the RDCRC’s specific rare provide content related to the RDCRC’s specific rare diseasesdiseases..

5) RDCRC Administrative Unit5) RDCRC Administrative Unit

An Administrative Unit is required. It will be responsible for An Administrative Unit is required. It will be responsible for

• Overall administration of the RDCRC, Coordinating activities for Overall administration of the RDCRC, Coordinating activities for RDCRC, Coordinating Network connections (DMCC, NIH ICs, ORD, RDCRC, Coordinating Network connections (DMCC, NIH ICs, ORD, PAG)PAG)

Describe Describe --the chain of responsibility for decision-making and administration.--the chain of responsibility for decision-making and administration. --a plan for communication (meetings, conference calls etc) and --a plan for communication (meetings, conference calls etc) and

participation of all personnel within the consortium. participation of all personnel within the consortium.This unit must include This unit must include

• A clinical investigator A clinical investigator

• Identify a Co-PI on the application who will be responsible for Identify a Co-PI on the application who will be responsible for assisting the RDCRC Director (PI of the application)assisting the RDCRC Director (PI of the application)

• Describe the biostatistical support for the RDCRC in this section. Describe the biostatistical support for the RDCRC in this section. The biostatistician will provide statistical support for protocol The biostatistician will provide statistical support for protocol development and assist in study designs.development and assist in study designs.

6) Collaboration with Patient Advocacy 6) Collaboration with Patient Advocacy GroupGroup

• Include a plan to fully incorporate the relevant Patient Include a plan to fully incorporate the relevant Patient Support Organization within RDCRC structure and Support Organization within RDCRC structure and interactions (conference calls, meetings etc). interactions (conference calls, meetings etc).

• Describe their participation across the planned Describe their participation across the planned objectives (e.g. in addressing clinical design, objectives (e.g. in addressing clinical design, recruitment and education). recruitment and education).

• The proposed activities should be appropriate for the The proposed activities should be appropriate for the level of the Patient Support Organizations.level of the Patient Support Organizations.

Renewal Applications: Additional Renewal Applications: Additional Material RequiredMaterial Required

• All applications for renewal must provide information All applications for renewal must provide information documenting the impact of the clinical research from documenting the impact of the clinical research from the original application. the original application.

• See See Section V. Application ReviewSection V. Application Review Information for Information for the review criteria to be addressed.the review criteria to be addressed.

SummarySummary

An RDCRC must include the following:An RDCRC must include the following:1. Clinical Research Projects for Observational/Longitudinal 1. Clinical Research Projects for Observational/Longitudinal

StudiesStudies and/or Clinical trials (At least two projects are and/or Clinical trials (At least two projects are required, one of which must be a longitudinal study)required, one of which must be a longitudinal study)

2. Pilot/Demonstration Projects (At least one project is 2. Pilot/Demonstration Projects (At least one project is required)required)

3. Training (career development) Component 3. Training (career development) Component 4. Website resource for education and research in rare 4. Website resource for education and research in rare

diseases diseases 5. RDCRC Administrative Unit 5. RDCRC Administrative Unit 6. Collaboration with Patient Advocacy Group6. Collaboration with Patient Advocacy Group andand

A description ofA description of Overall Clinical Research Program, Overall Clinical Research Program, Leadership & ResourcesLeadership & Resources

Remember….It is a Network of ConsortiaRemember….It is a Network of Consortia

• You will propose the direction and organization of You will propose the direction and organization of your RDCRCyour RDCRC

• Awarded RDCRCs will be formed into a Network Awarded RDCRCs will be formed into a Network (RDCRN) including a DMCC(RDCRN) including a DMCC

• Oversight will be from a steering committee (SCOversight will be from a steering committee (SC))

Steering Committee:Steering Committee:

PurposePurpose• Review, facilitate and establish all Network Review, facilitate and establish all Network

procedures and functions through monthly procedures and functions through monthly teleconference calls and face to face meetings, teleconference calls and face to face meetings, schedule & plan agendasschedule & plan agendas

See RFA for detailsSee RFA for details

OrganizationOrganization• PIs for RDCRC, DMCC PI, NIH IC Project Scientists, ORD PIs for RDCRC, DMCC PI, NIH IC Project Scientists, ORD

Program Staff, and CPAG ChairProgram Staff, and CPAG Chair

• Other (non-voting) members (program Officials form IC and Other (non-voting) members (program Officials form IC and ORD)ORD)

• One PI elected as chairpersonOne PI elected as chairperson

Steering Committee OrganizationSteering Committee Organization

RDCRC PIs

RDCRN Steering Committee

DMCC PI

CPAG ChairNIH ICs Project Scientists

ORD Program Staff


OverviewOverview


• RFA Purpose & RationaleRFA Purpose & Rationale





Review of Applications:Review of Applications:

• Special Emphasis Panel (SEP) Special Emphasis Panel (SEP)

• Organized by CSR (Organized by CSR (Dr. Fernando Arena, Dr. Fernando Arena, Scientific Review OfficerScientific Review Officer))

• Panel will review only responses to this RFAPanel will review only responses to this RFA

• Special instructions given to reviewersSpecial instructions given to reviewers

The Peer Review ProcessThe Peer Review Process

• Will be driven by number of applications Will be driven by number of applications receivedreceived

• Will use expedited review process (triage)Will use expedited review process (triage)

• Will generate a summary statement for each Will generate a summary statement for each applicationapplication

Review Criteria for RDCRCReview Criteria for RDCRC

• Following few slides are just samples of Following few slides are just samples of review criteria and do not include ALL review review criteria and do not include ALL review criteriacriteria

• See RFA for See RFA for allall review criteria review criteria

Review Criteria: Clinical Research Review Criteria: Clinical Research ProjectsProjects

• Significance Significance

• ApproachApproach

• InnovationInnovation

• InvestigatorsInvestigators

• EnvironmentEnvironment

Review criteria: Pilot/ Demonstration Review criteria: Pilot/ Demonstration Projects Projects

• Does the proposed plan for the training component Does the proposed plan for the training component address attracting new ideas and pilot studies within address attracting new ideas and pilot studies within RDCRC institutions? RDCRC institutions?

• Does the plan address reviewing and funding a Does the plan address reviewing and funding a spectrum of pilot projects with clinical research spectrum of pilot projects with clinical research potential in rare diseases? potential in rare diseases?

Review Criteria: Training (Career Review Criteria: Training (Career Development) ComponentDevelopment) Component

• Is the plan for training new investigators adequate and Is the plan for training new investigators adequate and appropriate? Does the plan describe how promising appropriate? Does the plan describe how promising candidates for clinical research in rare diseases will be candidates for clinical research in rare diseases will be selected? selected?

• Are environments within RDCRC appropriate for supporting Are environments within RDCRC appropriate for supporting the training of new investigators of the rare diseases under the training of new investigators of the rare diseases under study? study?

• Does the proposed plan address how the investigators will Does the proposed plan address how the investigators will seek out and include qualified women and minorities for seek out and include qualified women and minorities for participation in the proposed program? participation in the proposed program?

For renewal applicationsFor renewal applications: Remember to provide current status : Remember to provide current status and clinical research activities of individuals who have been and clinical research activities of individuals who have been supported by the training program. supported by the training program.

Review Criteria:Review Criteria: RDCRC Administrative RDCRC Administrative UnitUnit

• Are the plan(s) for overall administration of RDCRC, Are the plan(s) for overall administration of RDCRC, coordination of clinical research and collaborations coordination of clinical research and collaborations presented and sufficient for the requirements of the presented and sufficient for the requirements of the proposed RDCRC? proposed RDCRC?

• Is the plan for communication and participation of all Is the plan for communication and participation of all personnel within the consortium well described?personnel within the consortium well described?

Review Criteria: Research Program Review Criteria: Research Program Organization and Capability Organization and Capability

Leadership:Leadership:

• Are the scientific qualifications and involvement of Are the scientific qualifications and involvement of the PI (RDCRC Director) and Co-PI as well as the PI (RDCRC Director) and Co-PI as well as his/her scientific and administrative leadership his/her scientific and administrative leadership capabilities and time commitment presented and capabilities and time commitment presented and sufficient for the requirements of the proposed sufficient for the requirements of the proposed RDCRC? RDCRC?

Rare Diseases Patient Population:Rare Diseases Patient Population:

• Is the access to rare diseases patients and Is the access to rare diseases patients and populations for conducting clinical research adequate populations for conducting clinical research adequate to ensure likely success of the goals of the program? to ensure likely success of the goals of the program?

Research Program Organization and Capability (Cont.)Research Program Organization and Capability (Cont.)

Institutional Commitment:Institutional Commitment: • Is there institutional commitment to establishing the RDCRC Is there institutional commitment to establishing the RDCRC

as an integral part of its overall clinical research environment? as an integral part of its overall clinical research environment? • Is there commitment from the institutional leadership to Is there commitment from the institutional leadership to

protect the time of the investigators to pursue clinical research protect the time of the investigators to pursue clinical research and mitigate the demands of providing patient care? and mitigate the demands of providing patient care?

• Will trainees training be supported? Is the institutional Will trainees training be supported? Is the institutional leadership committed to this program and its goals in terms of leadership committed to this program and its goals in terms of providing specific assets specifically for the program, such as providing specific assets specifically for the program, such as faculty support, specific equipment, dedicated space, or faculty support, specific equipment, dedicated space, or financial support as a few examples? financial support as a few examples?

• Do the plans for integrating the activities of RDCRC clinical Do the plans for integrating the activities of RDCRC clinical research projects, as well as integrating rare diseases research projects, as well as integrating rare diseases research with existing institutional resources (e.g., use of research with existing institutional resources (e.g., use of clinical data and safety management systems, biostatistical clinical data and safety management systems, biostatistical support, etc.), give confidence and sufficient evidence that support, etc.), give confidence and sufficient evidence that such efforts are likely to be effective?such efforts are likely to be effective?

Research Program Organization and Capability (Cont.)Research Program Organization and Capability (Cont.)

Multidisciplinary Team Involving Patient Support Multidisciplinary Team Involving Patient Support Organizations Collaborations:Organizations Collaborations:

• Is there a plan to fully incorporate the relevant Patient Support Is there a plan to fully incorporate the relevant Patient Support Organization within RDCRC structure and interactions Organization within RDCRC structure and interactions (conference calls, meetings etc)? (conference calls, meetings etc)?

• Is Patient Support Organization participation described across Is Patient Support Organization participation described across the planned objectives (e.g. in addressing clinical design, the planned objectives (e.g. in addressing clinical design, recruitment and education)? Are proposed activities recruitment and education)? Are proposed activities appropriate for the level of the Patient Support Organizations?appropriate for the level of the Patient Support Organizations?

• Are the abilities and availabilities of the investigators to Are the abilities and availabilities of the investigators to interact with other participating sites and with the NIH in interact with other participating sites and with the NIH in sharing information, participating in committees etc. sharing information, participating in committees etc.

Review Criteria: Progress for Renewal Review Criteria: Progress for Renewal ApplicationsApplications

• Are the progress and achievements specific to the Are the progress and achievements specific to the application and relevant to clinical research since the application and relevant to clinical research since the previous competitive review? previous competitive review?

• Has adequate progress been demonstrated on Has adequate progress been demonstrated on clinical research projects that are ongoing? Are there clinical research projects that are ongoing? Are there any difficulties in achieving the previously proposed any difficulties in achieving the previously proposed specific aims addressed? specific aims addressed?

• Is it evident that the investigative team established a Is it evident that the investigative team established a productive working relationship among themselves productive working relationship among themselves and with the Patient Support Organizations during and with the Patient Support Organizations during the past performance period? the past performance period?

Additional Review CriteriaAdditional Review Criteria

• Protection of Human Subjects from Research Protection of Human Subjects from Research RiskRisk

• Inclusion of Women, Minorities and Children in Inclusion of Women, Minorities and Children in ResearchResearch

• BiohazardsBiohazards

Review and SelectionReview and Selection

The following will be considered in the selection The following will be considered in the selection process (funding decision):process (funding decision):

• Scientific merit of the proposed RDCRC as Scientific merit of the proposed RDCRC as determined by peer reviewdetermined by peer review

• Availability of fundsAvailability of funds

• Relevance to NIH IC and ORD program prioritiesRelevance to NIH IC and ORD program priorities


OverviewOverview


• RFA Purpose & RationaleRFA Purpose & Rationale





Read and Comply with RFA InstructionsRead and Comply with RFA Instructions

• Remember, this is a paper submission.Remember, this is a paper submission.

• Include all required and recommended components.Include all required and recommended components.

• Follow the suggestions on page recommendations (limits) for Follow the suggestions on page recommendations (limits) for each component.each component.

• Follow the Follow the NEW NIH instructions on AppendixNEW NIH instructions on Appendix materials materials (consult the URL in the RFA and send (consult the URL in the RFA and send CDsCDs of the appendix of the appendix material).material).

• Read Terms and Conditions of Cooperative Agreement.Read Terms and Conditions of Cooperative Agreement.

• Read the review criteria carefully to understand the basis for Read the review criteria carefully to understand the basis for the evaluation. the evaluation. For renewal applications:For renewal applications: documentation of documentation of accomplished clinical research goals. accomplished clinical research goals.

Application Preparation Application Preparation

• Read and follow the instructions in PHS 398 Read and follow the instructions in PHS 398 ((Revised 11/2007Revised 11/2007) and “Other Submission ) and “Other Submission Requirements”.Requirements”.

• Make sure your application is complete. No Make sure your application is complete. No supplemental material will be accepted after the supplemental material will be accepted after the receipt date.receipt date.

• If human subjects are involved include a targeted If human subjects are involved include a targeted planned enrollment table.planned enrollment table.

• Don’t forget data and safety monitoring requirementsDon’t forget data and safety monitoring requirements

• Have a statistician assess statistical power for all Have a statistician assess statistical power for all studiesstudies

RDCRN (RDCRCs + DMCC)RDCRN (RDCRCs + DMCC)

• The RDCRN will require cooperation among the PIs The RDCRN will require cooperation among the PIs (Directors) of the RDCRCs and their collaborators, (Directors) of the RDCRCs and their collaborators, the PI (Director) of the DMCC, the participating IC the PI (Director) of the DMCC, the participating IC Project Scientists, and the ORD Program Staff to Project Scientists, and the ORD Program Staff to maximize their effectiveness. maximize their effectiveness.

• A number of issues need to be addressed in the A number of issues need to be addressed in the cooperative agreement applicationscooperative agreement applications (including those (including those highlighted in Organization of the Rare Diseases highlighted in Organization of the Rare Diseases Clinical Research Network and under Clinical Research Network and under Cooperative Cooperative Agreement Terms and ConditionsAgreement Terms and Conditions.).)

Updates for RDCRC RFAUpdates for RDCRC RFA

• Addendum:Addendum: revised the restriction to “More than revised the restriction to “More than one application per organization/Institution may be one application per organization/Institution may be submitted as long as rare diseases represented in submitted as long as rare diseases represented in the applications are distinctly different”. the applications are distinctly different”.

• Clarification on page limitations:Clarification on page limitations:

--for longitudinal/observational clinical projects page --for longitudinal/observational clinical projects page limit applies to Research Plan only; and limit applies to Research Plan only; and

--for other three components (pilot project, training --for other three components (pilot project, training component and administrative unit) it applies to Plan/component and administrative unit) it applies to Plan/Examples only.Examples only.

SummarySummary

• RDCRC RFA PageRDCRC RFA Page

http://grants.nih.gov/grants/guide/rfa-files/RFA-OD-08-001.htmlhttp://grants.nih.gov/grants/guide/rfa-files/RFA-OD-08-001.html

• DMCC RFA PageDMCC RFA Page

http://grants.nih.gov/grants/guide/rfa-files/RFA-OD-08-002.htmlhttp://grants.nih.gov/grants/guide/rfa-files/RFA-OD-08-002.html

• IC Program and GM Contacts and “Research IC Program and GM Contacts and “Research Areas of Interest”Areas of Interest”

http://grants.nih.gov/grants/guide/contacts/rfa-od-08-001_contacts.htmhttp://grants.nih.gov/grants/guide/contacts/rfa-od-08-001_contacts.htm

http://grants.nih.gov/grants/guide/rfa-files/RFA-OD-08-001.html

http://grants.nih.gov/grants/guide/rfa-files/RFA-OD-08-002.html

http://grants.nih.gov/grants/guide/contacts/rfa-od-08-001_contacts.htm

Meeting open forMeeting open forQuestionsQuestions

• Start with DMCC Start with DMCC

• Follow with RDCRCFollow with RDCRC

Q & A SessionQ & A Session

• Answer QuestionsAnswer Questions

- Live audience: use microphoneLive audience: use microphone

- Video-Cast Audience: send email to - Video-Cast Audience: send email to

“ “Live Event Feedback Form”Live Event Feedback Form” ( ([email protected]@od.nih.gov))


NIH Office of Rare Diseases: ContactNIH Office of Rare Diseases: Contact

6100 Executive Boulevard 6100 Executive Boulevard

Room 3B-01, MSC - 7518Room 3B-01, MSC - 7518

Bethesda, MD 20892-7518Bethesda, MD 20892-7518

Voice: 301-402-4336Voice: 301-402-4336

Fax: 301-480-9655Fax: 301-480-9655

E-mail: E-mail: [email protected]@nih.gov

Website: Website: http://rarediseases.info.nih.gov/http://rarediseases.info.nih.gov/


http://rarediseases.info.nih.gov/

Link to RDCRC Slides

Documents