Lecture 2 - Regulatory Pathways for Devices

Legislative Branch

Writes the Laws

Executive Branch

• Charged with implementing statutes/laws through regulations

• RDA is an Executive agency

• Enforces Laws

Judical Branch

• Determines if laws are constitutional

• Uses case law

Branches of HHS – Health and Human Services Department

• PHS - Public Health Service

• FDA

• CDC

• National Institute of Health

Branches Of FDA

• CDRH – Center for Devices and Radiological Health → Regulates medical devices and radiation-emitting products.

• CDER – Center for Drug Evaluation and Research → Regulates prescription, over-the-counter drugs, and biologics.

• CBER – Center for Biologics Evaluation and Research → Regulates biologics (e.g., vaccines, blood products, gene therapies).

• OCE – Oncology Center of Excellence → Focuses on cancer-related drugs, biologics, and devices to streamline cancer product development.

When should a company submit an NDA vs a BLA to the FDA?

• NDA (New Drug Application): Submit for small-molecule drugs (traditional chemically synthesized drugs).

• BLA (Biologics License Application): Submit for biological products (e.g., vaccines, blood components, monoclonal antibodies, gene & cell therapies).

What FDA submission is used if there’s a predicate device already on the market?

• Submit a 510(k) Premarket Notification

• Used when a new device is shown to be substantially equivalent to a legally marketed predicate device.

What FDA submission is required if there is no predicate device on the market?

• De Novo Request → for low-to-moderate risk devices without a predicate.

• PMA (Premarket Approval) → for high-risk (Class III) devices requiring evidence of safety & effectiveness.

What is a drug (according to the FDA)?

• Articles intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease in humans or animals.

• Articles (other than food) intended to affect the structure or function of the body.

What is a biologic (according to the FDA)?

• A wide range of products such as vaccines, blood & blood components, gene therapies, tissues, and proteins (including monoclonal antibodies).

• Derived from living organisms (humans, animals, microorganisms).

• Regulated under a BLA (Biologics License Application).

What is a medical device (according to the FDA)?

• An instrument, apparatus, machine, implant, in vitro reagent, or similar article…

• Intended for diagnosis, cure, mitigation, treatment, or prevention of disease, or to affect the structure/function of the body.

• Does not achieve its primary intended purpose by chemical action within or on the body and is not dependent on being metabolized.

1976 MEDICAL DEVICE AMENDMENTS

Established device safety and
performance/effectiveness requirements
• Established FDA pre-market review
• Established the Medical Device Classification
• 2 Routes to Market
• Premarket notification: 510(k)
• Premarket Approval
• Established Investigational Device Exemptions
(IDEs)

What is a Guidance Document

A statement describing the FDA’s current thinking on a topic and provides recommendations

What is a Predicate

something on the market before 1976 or has a 510(k)

What is an Institutional Review Board (IRB)?

• A committee that reviews and monitors research involving human subjects.

• Ensures ethical standards, participant rights, and safety are protected.

• Must approve studies before they begin.

What is a Class I medical device?

• Low risk to the patient.

• Subject to general controls (e.g., good manufacturing practices, labeling).

• Often exempt from premarket notification [510(k)].

• Examples: Bandages, manual stethoscopes, exam gloves.

What is a Class II medical device?

• Moderate risk to the patient.

• Requires general + special controls (e.g., performance standards, postmarket surveillance).

• Usually requires 510(k) clearance.

• Examples: Infusion pumps, powered wheelchairs, blood pressure cuffs.

What is a Class III medical device?

• High risk to the patient.

• Requires Premarket Approval (PMA) with clinical evidence of safety & effectiveness.

• Life-supporting, life-sustaining, or implantable devices.

• Examples: Pacemakers, heart valves, deep-brain stimulators.

What is a 510(k) Premarket Notification?

• Submission to the FDA to demonstrate that a new device is substantially equivalent to a legally marketed predicate device.

• Usually required for Class II and some Class I devices.

• Does not require clinical trials in most cases.

What does substantial equivalence mean in FDA device regulation?

• A new device is considered substantially equivalent if it has the same intended use and similar technological characteristics as a legally marketed predicate device.

• Demonstrating this allows the device to go through a 510(k) submission instead of a PMA.

What are Humanitarian Use Devices (HUDs)?

• Medical devices intended to treat or diagnose conditions affecting fewer than 8,000 patients per year in the U.S.

• Can receive Humanitarian Device Exemption (HDE) from the FDA.

• Less stringent effectiveness requirements, but must still demonstrate safety.

What is a De Novo request?

• FDA pathway for low-to-moderate risk devices that have no predicate.

• Establishes a new device classification.

• Allows the device to be marketed with general and special controls.

• Often used when a 510(k) submission is not possible.

What is an IDE (Investigational Device Exemption)?

• Allows a medical device to be used in a clinical study to collect safety and effectiveness data.

• Required for significant risk devices before FDA approval (510(k), PMA, or De Novo).

• Ensures human subjects are protected and studies are regulated ethically.

What is a Breakthrough Therapy Designation?

• FDA designation for drugs or biologics that treat serious or life-threatening conditions.

• Preliminary clinical evidence indicates the therapy may be substantially better than existing treatments.

• Provides expedited development and review to get therapies to patients faster.

What is 3rd Party Review in FDA device regulation?

• FDA-accredited independent organizations review lower-risk devices.

• Sponsor submits one payment to the 3rd party (not FDA).

• 3rd party has more frequent access to FDA for guidance.

• They assess submission completeness and forward it to FDA with a recommendation.

• FDA has 30 days to review and respond.