FDA Breaks New Ground With First Approved Gene Therapy for Cancer

FDA Breaks New Ground With First Approved Gene Therapy for Cancer.jpg

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.”

Read More: http://snip.ly/bunjk#http://www.philly.com/philly/business/fda-advisors-give-a-thumbs-up-to-glaxosmithklines-shingles-vaccine-20170913.html

It is important and necessary to document Software for FDA Submissions

It is important and necessary to document Software for FDA Submissions2Software project management has an important tool in the Agile methodology. The Agile methodology developed as a product of the gradual efforts at arriving at a team based methodology of iterative software development. Because of its close association with software, in terms of suitability; Agile is to software development what Lean is to manufacturing. Among the many areas in which the Agile methodology is very well suitable and adaptable; healthcare is one, since it uses software heavily.

The Agile methodology is effective in helping software project managers anticipate and address major logjams of software project management, such as vulnerability and unpredictability. By preventing project delays; Agile helps to cut costs. Since flexibility is an important characteristic of Agile; it has the ability to accommodate and take in many new changes that take place as the project develops.

Another major benefit of Agile is that it prevents piling of work at later stages of the project by reviewing project progress at every stage by validating roles, steps and processes functions. This is absolutely useful in the backdrop of severe constraints of time and money, because of which it is highly preferred and rated by Project Managers.

Is Agile perfect?

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All these terrific advantages notwithstanding; Agile is not perfect. It is not suited in every setting and in every situation. If Agile has to be efficient and deliver its results optimally; it has to work in conditions where there is complete, tightly knit team coordination. In the absence of very active and strong participation from the team leaders, subject matter experts and stakeholders; Agile can prove less than suitable or successful.

Agile’s suitability for the healthcare industry is well-established. However, Agile, being a highly teamwork-dependent initiative can fail to deliver in the absence of one or more situations in which it thrives. In the absence of complete confidence by those using it in the healthcare industry of its role in saving money, time and other resources; the Agile methodology can be less than useful

Get to learn the applicability of Agile methodology to healthcare

Business.

The ways by which the healthcare industry can adapt and optimize Agile methodology for its use and overcome the deficiencies and shortfalls of this methodology for enhanced performance will be the topic of a very interesting two-day seminar that is being organized by GlobalCompliancePanel, a leading provider of professional trainings for all the areas of regulatory compliance.

The Director of this seminar is Brian Shoemaker, who consults for healthcare products companies on computer system validation, software quality assurance, and electronic records and signatures, and has worked with companies in Germany and Switzerland as well as the U.S. Please register for this session by logging on to It is important and necessary to document Software for FDA Submissions. This seminar has been pre-approved by RAPS as eligible for up to 12 credits towards a participant’s RAC recertification upon full completion.

Clarity on the suitability of Agile to IEC 62304

It is important and necessary to document Software for FDA Submissions3

A criticism that does the rounds in healthcare software industry circles is that Agile, because of its lack of documentation, runs counter to the lifecycle standards mandated in IEC 62304. Brian will clarify on this area and explain how clear processes for quality management system, risk management process, software maintenance, configuration management, and problem resolution, which go into the IEC 62304 principles actually reinforce, rather than undermine the Agile methodology. The proof of this fact is that the AAMI Agile report (TIR 45) has stated that the proper application of Agile, with its emphasis on nimbleness and ongoing learning, into a quality system and safety risk management can blend with and expedite the fulfilment of regulatory expectations of well-documented development.

Contrary for popular belief, documentation in Agile actually helps in taking advantage of iterative development. How? Since the IEC 62304 does not specify any lifecycle model; documentation can grow out of the required iterative activities. Agile, by developing incrementally and preventing last minute anxieties and worries; is highly useful in many disciplines of healthcare such as hazard analysis. When risk management is included in iteration tasks; it becomes more robust and solid.

It is because of all these reasons that this is a session that professionals across a wide spectrum of positions such as Regulatory Specialists, Quality Assurance Specialists, Documentation Specialists, Test Managers, Software Team Leaders and Lead Developers, and Project and Program Managers ought not to miss out on.

They will get thoroughly familiarized on the applicability of the Agile methodology to software documentation for FDA submissions. Over these two days of this seminar, Brian will cover the following areas:

  • Agile vs IEC 62304: an apparent contradiction?
  • The role and value of documentation
  • The REAL regulatory requirements
  • Specific documents required for an FDA submission
  • Areas where most development processes bog down
  • Iteration – well suited for risk, usability, and design reviews
  • Key practices to bridge the Agile and regulated worlds
  • Agile is not only acceptable for medical device software, but can be clearly superior.

 

 

Applied Statistics for FDA Process Validation

The pharmaceutical industry considers Applied Statistics for FDA Process Validation to be of very high importance. In 2011, the FDA set out this guidance for the industry. as part of this guidance, called “Process Validation: General Principles and Practices”, which sets the framework for Process Validation in the pharmaceutical industry, any organization in the pharmaceutical industry has to set up a three-stage process.

These are the three stages:

I.           Process Design

II.           Process Qualification, and

III.           Continued Process Verification.

Stage 1, or what is called the Process Design stage, is the stage in which the commercial manufacturing process is defined. This definition is based on knowledge gained through development and scale-up activities.

Stage 2, called the Process Qualification, is the stage in which an evaluation is made of the process design to determine if the process is capable of reproducible commercial manufacturing.

Stage 3, the Continued Process Verification, is meant for giving ongoing assurance during routine production to ensure that the process remains in a state of control.

A seminar on the ways implementing Applied Statistics for FDA Process Validation

GlobalCompliancePanel, a leading provider of professional trainings for the regulatory compliance areas, will be organizing a two-day seminar in which the ways of using Applied Statistics for FDA Process Validation will be taught. Richard Burdick, Emeritus Professor of Statistics, Arizona State University (ASU) and former Quality Engineering Director for Amgen, Inc., will be the Director of this seminar on applied statistics for FDA Process Validation.

In order to learn Applied Statistics for FDA Process Validation in-depth, please register by visiting Applied Statistics for FDA Process Validation. 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 detailed and methodical approach to implementing statistical methodologies

This two-day course on Applied Statistics for FDA Process Validation will focus on the ways by which a systematic approach to implementing statistical methodologies into a process validation program consistent with the FDA guidance can be established.

Beginning with a primer on statistics, Dr. Burdick will explain how the methods of Applied Statistics for FDA Process Validation seminar can be applied in each remaining chapter.

Dr. Burdick will next move on to explaining the two fundamental requirements for Process Validation, namely the application of statistics for setting specifications and assessing measurement systems (assays).

He well then show how to apply statistics through the three stages of process validation as defined by requirements in the process validation regulatory guidance documents.

Given that the methods taught through all these three stages are recommended by regulatory guidance documents; this seminar on Applied Statistics for FDA Process Validation will provide references to the specific citations in the guidance documents.

This seminar on Applied Statistics for FDA Process Validation will lead participants into ways of establishing a systematic approach to implementing statistical methodologies into a process development and validation program that is consistent with the FDA guidance.

All-round learning related to Applied Statistics for FDA Process Validation

Dr. Burdick will teach participants how to:

o  Apply statistics for setting specifications

o  Assess measurement systems (assays)

o  Use Design of Experiments (DOE)

o  Develop a control plan as part of a risk management strategy, and

o  Ensure process control/capability.

All concepts at this Applied Statistics for FDA Process Validation seminar are taught within the three-stage product cycle framework defined by requirements in the process validation regulatory guidance documents.

Although established for the pharmaceutical industry, this seminar on Applied Statistics for FDA Process Validation also provides a useful framework for other related industries.

In this important learning on Applied Statistics for FDA Process Validation; Dr. Burdick will cover the following areas:

o  Apply statistics to set specifications and validate measurement systems (assays)

o  Develop appropriate sample plans based on confidence and power

o  Implement suitable statistical methods into a process validation program for each of the three stages

o  Stage 1, Process Design: utilize risk management tools to identify and prioritize potential critical process parameters; and define critical process parameters and operating spaces for the commercial manufacturing process using design of experiments (DOE)

o  Stage 2, Process Qualification: assess scale effects while incorporating large (pilot and/or commercial) scale data; develop process performance qualification (PPQ) acceptance criteria by characterizing intra and inter-batch variability using process design data and batch homogeneity studies; and develop an appropriate sampling plan for PPQ

o  Stage 3, Continued Process Verification: develop a control plan as part of a risk management strategy; collect and analyze product and process data; and ensure your process is in (statistical) control and capable.

A Tour of the FDA 2017

Businessman uses smart watch and phone.

A tour of the FDA is something like a snapshot of what the FDA does. The importance of the FDA can never be understated: It regulates products from the proverbial pin to airplane in the food, medical devices, pharma and healthcare industries, which touch almost every aspect of American lives. The products that the FDA regulates account for about a trillion dollars, which make up about a quarter of all goods traded in the US.

So, what is a tour of the FDA like?

A tour of the FDA helps to get a broad understanding of the this regulatory body and get some idea of the various departments it has, as well as the functions of these departments. To get an understanding of what the FDA does, a reference to its mission statement could give some direction:

“(The) FDA is responsible for protecting the public health by assuring the safety, efficacy and security of human and veterinary drugs, biological products, medical devices, our nation’s food supply, cosmetics, and products that emit radiation.” This is just the opening line of the FDA’s mission statement. Reference to its other statements, which have now included a reference to containing terrorism, will serve as a good guide to a tour of the FDA. In short, the FDA regulates nearly every item used and consumed by the American public.

The history of the FDA

The legal sanction for carrying out its mission is mandated by the Federal Food, Drug and Cosmetic Act (FD&C Act). An amazing fact that a tour of the FDA reveals is that it is one of the oldest regulatory bodies in the world, with its earliest jurisdiction having covered regulation of drugs in the year 1848. The Department of Agriculture, to which President James Polk appointed noted chemist Charles Wetherill, can be considered the earliest endeavor to regulate medical products of daily use in the US.

How is the FDA organized?

A tour of the FDA is incomplete without a reference to the way it is organized. Its structure consists of this hierarchy:

  • Office of the Commissioner
  • Office of Foods and Veterinary Medicine
  • Office of Global Regulatory Operations and Policy
  • Office of Medical Products and Tobacco
  • Office of Operations

Under these broad heads, a tour of the FDA shows the way into which it is divided into several offices and organizations. Important among these include:

  • Office of Regulatory Affairs (ORA)
  • Center for Food Safety and Applied Nutrition (CFSAN)
  • Center for Drug Evaluation and Research (CDER)
  • Center for Biologics Evaluation and Research (CBER)
  • Center for Devices and Radiological Health (CDRH)
  • Center for Devices and Radiological Health (CDRH)
  • National Center for Toxicological Research (NCTR)

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GlobalCompliancePanel announces Seasonal offers for Professionals with Flat 50% OFF on all Seminars

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Do celebrations need a cause and a reason? Yes, and GlobalCompliancePanel, a leading provider of professional trainings for the regulatory compliance areas, is having a solid cause and reason for doing so. It is celebrating the many years of its relationship with its customers spread all over the world by offering its trainings at a massive 50% discount!

Yes, that is right. GlobalCompliancePanel’s seminars will be available for a huge 50% discount till April 30. Regulatory professionals who want to augment their knowledge of regulatory compliance can now do so by paying just half the price of these trainings from GlobalCompliancePanel. All that is needed to do walk away with a rare offer such as this is to visit https://www.globalcompliancepanel.com/seminar?wordpress_SEO and use MGCP50 Promo Code.

This offer is valid till April 30, 2017. Regulatory professionals who want to take any of GlobalCompliancePanel’s trainings can book their trainings for an area of their interest by this date. From April 1 onwards, this offer will cease, meaning that the original price will apply from then.

So, why is GlobalCompliancePanel offering this discount? It is for a simple, but profound reason: It wants to thank its huge customer base for the support they have been extending to this company over the many years for which it has been in business. During the course of the 10 years for which GlobalCompliancePanel has been in business, it has trained thousands of regulatory compliance professionals from around the world.

These professionals, belonging to such varied geographies as the US and Japan and India and Canada, have been able to meet their regulatory compliance challenges on account of these trainings. These trainings are relevant, focused and valuable, and are from some of the best known regulatory compliance Experts found anywhere on this planet.

It is these trainings that have been hoping these professionals in the regulatory compliance arena gain more insights into regulations from the FDA, the EMA and other such bodies around the world. These trainings have been consistently helping them to meet these challenges, as they give them a better and sharper understanding of the implementing these requirements.

These regulatory requirements can pose hurdles to the most experienced and brightest of regulatory compliance professionals in the medical devices, pharmaceutical, life sciences and food and biologicals areas, but not to those who undertake professional trainings from GlobalCompliancePanel. GlobalCompliancePanel’s panel of experts is here to help them overcome these challenges and hurdles.

This trend has been being witnessed from the time GlobalCompliancePanel entered the line of professional trainings. Any wonder then, that no fewer than 50,000 professionals have benefited from these trainings? What could be a better way of thanking such a huge base of customers than with this offer? GlobalCompliancePanel believes that a celebration should also be useful, and this is that this offer is!

Hurry up and enroll today. Happy learning!

 

 

Article on FDA 21 CFR Part 11 Compliance

FDA-regulated industries electronic signatures and other records are considered authentic. From 2007, a strong body of opinion has emerged challenging the stringency of these requirements, but nothing major has been diluted from these.

The regulations under FDA 21 CFR Part 11 Compliance set out criteria that the Food and Drug Agency (FDA) considers in order to deem electronic signatures authentic. The electronic records, electronic signatures, and handwritten signatures executed to electronic records of several FDA 21 CFR Part 11 Compliance sets out benchmarks by which FDA-regulated industries have to be compliant with the standards set out in FDA 21 CFR Part 11 Compliance to prove that these are authentic, safe and trustworthy. The operative factor is that the FDA has to consider these signatures as being on par with those done on paper.

Which industries are included in FDA 21 CFR Part 11 Compliance?

FDA 21 CFR Part 11 Compliance applies to nearly all FDA-regulated industries, including but not restricted to:

  • Medical device manufacturers
  • Drug makers
  • CROs
  • Biotech companies, and
  • Biologics developers

The Aim of FDA 21 CFR Part 11 Compliance

The aim of FDA 21 CFR Part 11 Compliance is to ensure that specified FDA-regulated industries such as those mentioned above (with specific exceptions) implement controls -which could include audits, audit trails, documentation, system validations, and electronic signatures -for software and systems involved in processing electronic data that are:

  • Required to be maintained by the FDA predicate rules or
  • Used to demonstrate compliance to a predicate rule. The FDA describes a predicate rule as any requirement set forth in the Federal Food, Drug and Cosmetic Act, the Public Health Service Act, or any FDA regulation other than Part 11. FDA 21 CFR Part 11 Compliance also applies to submissions made to the FDA in electronic format, such as a new drug application.

Which industries are exempt from FDA 21 CFR Part 11 Compliance?

Interestingly, exceptions are allowed within the same industry, based on the format of filing. For example, while FDA 21 CFR Part 11 Compliance applies to submissions made to the FDA in electronic format; it does not apply to a paper submission for the same made in electronic format, such as fax.

Also, FDA 21 CFR Part 11 compliance is not required for record retention for trace backs by food manufacturers. Similar to the logic used in the mode of filing as noted above; most food manufacturers are not otherwise explicitly required to keep detailed records, but when organizations keep electronic documentation for HACCP and similar requirements; this documentation must meet these requirements.

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FDA guidance

FDA guidance

AnFDA guidance, as the title suggests, is a set of suggestions from the FDA on a number of its governing areas. The FDA, being a regulatory body sets standards for almost every conceivable area. It is almost impossible to list the number of guidances the FDA has issued.

Nonbinding in nature

Guidances are suggestions for best practices and methods, and are not a set of rules. The outstanding characteristic of FDA guidances is that they are nonbinding. The FDA website states as much: “This guidance represents the Food and Drug Administration’s (FDA’s) current thinking on this topic. It does not create or confer any rights for or on any person and does not operate to bind FDA or the public. You can use an alternative approach if it satisfies the requirements of the applicable statutes and regulations. If you want to discuss an alternative approach, contact the FDA staff responsible for implementing this guidance.”

This note is to be found in guidances the FDA issues in every of its governing areas. As can be seen from the above explanation; guidances are open to interpretation, and the individual, group of individuals, organizations or laboratories that come under these guidances are free to offer their own perspective. They can discuss industry-specific guidances with the FDA and sort out matters whenever disagreements arise.

General areas for FDA guidance

These are the broad areas in which the FDA has issued guidances:

  • Food
  • Drugs
  • Medical Devices
  • Vaccines, Blood & Biologics
  • Animal & Veterinary             Cosmetics
  • Radiation-Emitting Products
  • Regulatory
  • Tobacco Products

References:

http://www.fda.gov/regulatoryinformation/guidances/default.htm

http://google2.fda.gov/search?site=FDAgov-Guidance-All&restrictBox=FDAgov-Guidance-All&restrictBox=FDAgov-Guidance-Food&q=&btnG=Search&client=FDAgov&output=xml_no_dtd&proxystylesheet=FDAgov-Guidance&ie=UTF-8&oe=UTF-8

 

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