FDA Finalizes Guidance on Interoperable Medical Devices

On September 6, 2017, FDA finalized a guidance document entitled “Design Considerations and Pre-Market Submission Recommendations for Interoperable Medical Devices” (“Final Guidance”). In the Final Guidance, the agency outlines design considerations for manufacturers when developing interoperable medical devices, as well as recommendations about information to include in premarket submissions and device labeling. Interoperability of devices can encourage the availability and sharing of information across systems, even when products from different manufacturers are used. A draft of this guidance was issued on January 26, 2016.

The Final Guidance defines “interoperable medical devices” as medical devices “that have the ability to exchange and use information through an electronic interface with another medical/non-medical product, system, or device.” These functions can consist of a one-way data transmission to another device or product, or more complex interactions in which command and control is exercised over another device. An “electronic interface” is defined as the medium by which systems communicate with each other, and includes both the type of connection and the information content.

According to the Final Guidance, the agency considers the management of risks associated with an electronic interface incorporated into a medical device to be part of a comprehensive quality system under 21 C.F.R. Part 820. Manufacturers of interoperable medical devices should perform a risk analysis and conduct appropriate testing addressing the risks associated with interoperability, the anticipated users, reasonably foreseeable misuse, and reasonably foreseeable combinations of events that can result in a hazardous situation. In particular, the Final Guidance identifies the following considerations that manufacturers should take into account and “appropriately tailor[]” to the device’s interface technology, intended use, and use environments

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FDA Breaks New Ground With First Approved Gene Therapy for Cancer

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When oncologist Dr. Carl June heard the Food and Drug Administration’s decision to bring the first gene therapy to market in the US, he pinched himself, hard.

“It was so improbable that this would ever be a commercially approved therapy,” he said, voice breaking with emotion.

June was referring to a revolutionary cancer therapy that he helped bring from lab bench to market. Co-developed with the drug giant Novartis, the therapy, CAR-T, genetically alters a patient’s own immune cells to target and destroy cancer cells.

Recently, in a historic decision, the FDA threw their support behind Kymriah (tisagenlecleucel), a “living drug” that is designed to treat blood and bone marrow cancer in children that, even with aggressive chemotherapy, is often lethal.

An entire process rather than a packaged pill, the therapy harvests a patient’s own immune cells—T cells that patrol and destroy abnormal cells—retrains them with extra bits of genetic code, and turns them into torpedoes aimed at cancerous cells once reintroduced into patients’ bodies.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Dr. Scott Gottlieb in a statement, adding that the therapy is “the first gene therapy available in the United States.”

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FDA advisors give a thumbs-up to GlaxoSmithKline’s shingles vaccine

FDA advisors give a thumbs-up to GlaxoSmithKline's.jpg

GlaxoSmithKline’s shingles Shingrix vaccine received an unanimous vote of support by a Food and Drug Administration advisory committee Wednesday on safety and effectiveness to be used in adults 50 and older.

A decision by the FDA to commercialize Shingrix is expected later this year. The agency usually follows the recommendations of its advisory panels.

GSK said in June that the vaccine produced a strong immune response in adults 65 and older who had previously been vaccinated against shingles with Merck’s vaccine, Zostavax. Scientific data published in the New England Journal of Medicine showed that the effectiveness of Merck’s vaccine wanes over time, while GSK’s vaccine appeared to have longer-lasting protection.

GSK said data show that people who received Merck’s vaccine, the only one approved now for the herpes zoster (shingles) vaccine, can later receive the Shingrix vaccine safely and effectively.

“The risk of developing shingles increases with age and it is estimated that up to one in three people in the United States will develop shingles,” said Emmanuel Hanon, GSK head of vaccines research and development. “Today’s vote brings us one step closer to approval of Shingrix, which is specifically designed to overcome age-related weakening of the immune system.”

 

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Understanding the FDA’s GMP Expectations for Phase I and First-in-Man Clinical Trials

The establishment of the initial safety of a drug is the primary reason for which early clinical trials are conducted. Phase I of drug development consists of:

o  Research and drug discovery

o  Preclinical development, of which initial IND, or first in humans (also called first in man), is part

o  Clinical development, which consists of Phase I, Phase 2 and Phase 3 clinical pharmacology studies

Phase I studies are designed mainly for investigating the following qualities of an investigational drug in humans:

o  The extent to which it is safe and tolerable: (if possible, identify its Maximum Tolerated Dose (MTD)

o  Pharmacokinetics

o  Pharmacodynamics of an investigational drug in humans.

The ultimate goal of these Phase I and later studies is to ensure that the right drug is given to the right patient in the right dose, at the right time. These clinical studies are carried out to determine which dose or dosing regimen of the target drug achieves all its objectives for all the populations groups for which it is being developed.

Rationale for a small sample

Generally, these studies are carried out on a small sample of subjects of normal health. These early clinical trials normally use lower doses of the drug product. On account of this, the organization or laboratory carrying out these early stage clinical studies uses only small amounts of investigational material.

This is because of two reasons: The small sample is chosen in such a fashion that it is a representation of the whole study in relation to various parameters, and two, choosing a small sample substantially reduces the costs associated with a largescale study. It also reduces the regulatory burden during these early stages. Keeping in mind these factors, the FDA has established guidelines by which early stage investigational products can be allowed to be manufactured under less stringent GMPs.

Not meeting regulatory requirements makes the exercise futile

Without an understanding of the FDA’s guidelines for these GMPs, the whole exercise of early or Phase I studies becomes wasteful and meaningless. All along, the FDA’s guidelines have to be consistently and accurately adhered to in order to meet compliance requirements, without which the clinical trial and its results do not get approved.

What are the steps that a clinical or pharmaceutical organization need to take in order to ensure that these GMPs for Phase I clinical trials are being met? All the aspects of this topic will be covered in depth over two days of intense learning that will be imparted at a seminar from GlobalCompliancePanel, a leading provider of professional trainings for the areas of regulatory compliance.

Peggy Berry, the President and CEO at Synergy Consulting where she provides consulting services to companies in all aspects of drug development, will be the Director of this seminar. All that is needed to gain from the rich wealth of Peggy’s experience is to register for this seminar by logging on to Good Manufacturing Practices . This course has been pre-approved by RAPS as eligible for up to 12 credits towards a participant’s RAC recertification upon full completion.

A complete explanation of the FDA’s GMP requirements

This seminar is being organized to help participants gain an understanding of the requirements for advancing drugs from research into early clinical development and the minimum FDA requirements for Phase I GMPs. She will help them learn practical applications for implementing Phase I manufacturing strategies to meet FDA requirements.

Towards this end, Peggy will discuss these topics to lay the foundation and basis for advancing drugs into clinical development from research and providing required information to the FDA regarding these products on Day 1:

o  Moving a Product out of R&D

o  CMC Requirements for an IND Study

o  Good Manufacturing Practices: Basics for Beginners

o  Raw Material Management

Taking off from these topics, Peggy will focus on the topics relating to the requirements for early stage products of different types and for vendor selection and management on Day 2, which include:

o  GMPs for Phase I IND products

o  GMPs for Combination Products and 505(b)(2) Products

o  Process Validation for Early Stage GMP

o  Outsourcing Early Stage Manufacturing.