Purpose
- Regulatory Approval: 510(k) Clinical Data / PMA
- Reimbursement
- Marketing / Adoption
Study Rationale
- Study Hypothesis
- Study Endpoints (Primary & Secondary)
- Study Population (Type & Number)
- Study Enrollment/Consent
- Study Comparison Arm (Standard or Care?)
- Study Blinded (Sham)
- Randomized?
- Follow-up intervals (Efficacy vs. Safety)
- Safety Monitoring
Clinical Development Program
- Non Clinical: in-vitro / ex-vivo
- Pre-Clinical: Non GLP / GLP
- Clinical: FIH / Pilot / Pivotal / Post-marketing
Models
Bench
- Require building anatomic or physiological model
- Simplest method for testing concept
- May be used to ask fundamental question
Ex-vivo
- Allows for near-real anatomy/physiology testing
- Minimizes assumptions
- Useful for understanding fundamental problems
In-vivo
- Real anatomy/physiology
- May assume no pathologic disease present
- Large animal ideal for assessing feasibility & risk
Good Laboratory Practice (GLP)
- Guidelines for conducting and reporting non-clinical or pre-clinical studies to ensure quality and integrity of data
- FDA encourages but does not require
Good Clinical Practice (GCP)
- Guidelines for conducting and reporting an IDE clinical trials for investigators, sponsors, and IRB
- Absolutely FDA required!
Investigational Device Exemption (IDE)
- Required for clinical studies supporting a regulatory device submission
- Allows a pre-market device to be tested in humans such that the necessary safety and effectiveness data can be collected
- All PMA studies and 510(k) which requiring clinical data data must have an IDE prior to starting the trial
Off-Label Usage
- Device approval for specific indication but used by physician for any purpose deemed clinically appropriate
- Method to discover new applications for existing technologies
- Company cannot promote “off label” usage
- When “off-label” usage significant, companies often initiate trials using IDE to add additional indication
IDE Exemptions
Legally marketed device
- “on-label” use: 510(k) device not requiring clinical data
Diagnostic Devices
- Non-invasive
- Low risk sampling procedure
- Does not introduce energy into body
- Is not used as sole diagnostic tool
Consumer Preference Testing
- Combining features of approved devices
Patient Recruitment
- Inclusion Criteria: Characteristics or indications that a subject must have in order to participate in the trial
- Exclusion Criteria: Characteristics or contraindications that eliminate subjects from participate in a study
Human Subject Informed Consent
- Standard governing the protection of human subjects in clinical research
- History of abuse (Nazi Germany, Tuskegee)
- No investigator may involve human beings as a subject in research unless informed consent has been obtained: Informed & Consent
- Risk vs. Benefit Analysis
Institutional Review Board (IRB)
- Any board, committee, or other group formally designated by an institute to review and approve the initiation of biomedical research involving human subjects
- Primary purpose of review is to ensure the protection of the tights and welfare of human subjects
- Conduct ongoing reviews during the study
Cost
- Device (IDE and Standard)
- Performing the Procedure
- Evaluating the Clinical Result (Follow-up)
- Core Labs (Centralized Analysis): Unbiased 2nd Opinions
- Investigator/Institutional Fee
- Conduction the Trial (Training/Monitoring): CRO
- Patient Recruitment (Advertising, Compensation)
- In-house Management
- Institutional Review Board (IRB)
- Data Safety Monitoring Board (DSMB)
- Advisory Boards (Planning)
[Source: lecture by Todd Brinton]
