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.”

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

The importance of meeting Supplier Management criteria

The importance of meeting Supplier Management criteria

Meeting the supplier management criteria set out by the FDA, the ISO and the QSR regulations is mandatory for the regulated industries. The qualification and assessment of suppliers is binding for the regulated industries. Being in compliance with the requirements for supplier management set out by the FDA and by the ISO and QSR standards is a sure means for organizations in the regulated industries to keep the costs of noncompliance down, as well as in helping to meet customer requirements and ensure control over their suppliers and the regulations.

This requirement assumes greater significance when the fact that the suppliers are exempt from the jurisdiction of both the FDA and the Notified Bodies in the EU is taken into consideration. It is only over the organizations that use suppliers that these agencies have authority. So, it is entirely up to the organizations in the regulated industries to ensure that they meet the criteria set out in these regulations and that they conform to the supplier qualification and assessment criteria. It is entirely their responsibility to ensure that they have sufficient control over the processes that go into supplier management. The core of this arrangement between the organization and the supplier is risk management, which is a very critical mitigation tool in the supplier management system.

Expected in all stages of the QSR and ISO

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Given that the full burden of ensuring supplier control rests on the organization; the latter has to ensure that it selects the supplier who is capable of thoroughly meeting the requirements set out by the regulatory agencies, on behalf of the organization. The supplier needs to be able to meet supplier qualification and assessment in accordance with the regulations set out by the QSR and ISO standards. However, contrary to popular belief, it is not necessary to spend a lot of money to ensure this. As many examples have shown, organizations can show compliance with ease with limited expenditure. How are they able do this?

A seminar on putting a compliant supplier management system in place

This is the essence of the learning from a two-day seminar that is being organized by GlobalCompliancePanel, a leading provider of professional trainings for the areas of regulatory compliance. This seminar will impart the skill and knowledge needed for putting in place a supplier management system that is both compliant with the ISO and QSR requirements and is yet cost-effective.

Jeff Kasoff, who is Director of Regulatory Affairs at Life-Tech, Inc., will be the Director of this seminar. Jeff has spent over 30 years in quality and regulatory management, during the course of which he has implemented and overseen quality system operations and assured compliance, at all sizes of company, from startups to those with more than $100 million in revenue. Please register for this seminar by logging on to The importance of meeting Supplier Management criteria. This course has been pre-approved by RAPS as eligible for up to 12 credits towards a participant’s RAC recertification upon full completion.

Complying with both QSR regulations and ISO standards

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Jeff will help participants of this seminar gain insights and knowledge of how to accomplish their supplier qualification and assessment that meet both the QSR regulations and ISO standards in a cost effective manner. In his review of the QSR and ISO requirements for supplier evaluation; Jeff will give a definition of the types of suppliers that require evaluation. He will also describe the QSR/ISO requirements for supplier assessment.

Considering the inescapable link between risk management and supplier qualification and assessment, the Director will show how to implement a risk management plan that is effective and will help participants to add value.

Discussion on the core areas of supplier management

Jeff will devote considerable attention to supplier nonconformance, a critical area of supplier management. He will put participants at ease with the ways of how and when to issue the Supplier Corrective Action Request, a highly detested action. Before it gets issued, all the options need to be exhausted. Jeff will show which these are, as well as the knack of handling supplier nonconformance adroitly. While highlighting the importance of collaboration in the approach towards suppliers; he will explain the kinds of behaviors that risk alienating or losing suppliers.

While putting in place a supplier management and system that meet all required regulations and guidance documents is imperative to all businesses; it is more so for outsourced processes such as contract manufacturing, sterilization and testing, and also for critical suppliers. By default, this system makes dependence on suppliers inevitable.

Yet, having in place a system which ensures that they have sufficient control over their suppliers, as well as to assure auditors and regulatory agencies that the product is safe and meets all the quality requirements, is a must for organizations. Jeff will show how this can be achieved. Ensuring the cost-effectiveness of the compliant supplier management program is very important. Not doing so eats into the gains of compliance and being in control. Jeff will teach ways by which this scenario can be avoided.

 

The ingredients of a truly effective internal audit program

 

AFirst Sliden internal audit program of an organization’s Quality Management Systems is a core requirement from both the ISO and the FDA. At its barest, an internal audit system can be understood to be an assessment or inspection of an organization’s Quality System. Audits are a very beneficial and positive tool for continually ensuring that an organization’s internal management systems are up to the mark and keep meeting regulatory requirements on a consistent basis.

An internal audit program is the most effective and useful tool in helping an organization discover its loopholes and deficiencies in the Quality System, which is absolutely essential to help it pass regulatory hurdles. The purposes of an internal audit, as set out by ISO 9001, which describes Quality Management Systems audits, are to ensure that:

  • The Quality Systems of the organization comply with the requirements of ISO 9001 and its latest versions
  • They show compliance with the requirements of other regulatory bodies such as the FDA
  • They comply with the quality requirements the organization has
  • The organization makes effective implementation and maintenance of the QMS.

ISO 9001 does not require the internal audit procedure to be fully documented. An organization has to only, to use the ISO’s own words, “maintain an audit program” and retain the documents relating to the audit and its results and information contained in them.

Multifold benefits of auditing

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Auditing, both external and internal, bring many benefits. Besides meeting the purposes as set out by ISO 9001 and others such as the FDA; auditing helps organizations:

  • Identify the areas in which they need to make improvements
  • Apply best practice relevant to the particular industry
  • Take preventive actions that could nip potential problems in the bud
  • Ensure greater customer satisfaction.

Compliance with audit requirements is considered drudgery

Despite the noble intentions behind internal and external audits; three fourths of organizations consider audits as being very cumbersome, unpleasant and baneful. Most of them carry out audits with no conviction. They carry them out only because they have no other choice. Most businesses fail to see the value behind the efforts needed for carrying out audits that meet regulatory requirements.

Carrying out audits without any sense of purpose results in more drudgery and unpleasantness. The result of carrying out audits for the sake of it, rather than with purpose is there to see: More Warning Letters, 483’s, penalties and other actions such as even Consent Decree, from the FDA.

Learn to carry out effective audits more willingly and effectively

The ways by which organizations can overcome the mental barrier to carrying out audits and put in place a well-planned, compliant audit program that reduces the risk of noncompliance will be the teaching a two-day seminar from GlobalCompliancePanel, a leading provider of professional trainings for the regulated industries, will be offering.

Susanne Manz, Quality and Compliance Expert/Auditor for Medical Devices, Manz Consulting, Inc., who is an accomplished leader in the medical device industry with emphasis on quality, compliance, and Six Sigma, will be the Director of this seminar. To enroll for this webinar and gain complete understanding of the means for planning and implementing a Quality Systems audit that meets regulatory requirements,

Please register by logging on to http://www.globalcompliancepanel.com/control/globalseminars/~product_id=900854SEMINAR?wordpress_SEO. This seminar has been pre-approved by RAPS as eligible for up to 12 credits towards a participant’s RAC recertification upon full completion.

A complete understanding of the rationale and ways of carrying out compliant audits

This seminar will be highly useful in helping audit professionals in organizations devise an effective audit program with is compliant. It will put participants on the path to a thorough quality audit program that meets regulatory requirements. Such a well devised quality audit program is a powerful tool in helping organizations to understand, reduce and communicate risks relating to quality and compliance.

Susanne will open the participants’ minds to continuous improvement, which starts with the awareness of issues and opportunities. The absence of an effective audit program deprives management of an understanding of the issues within their Quality System. As a result, management is oblivious to the lacunae in the Quality System and the risk bring for the organization. Such a situation denies the management the opportunity to assess the impact a faulty Quality Management System audit has on product quality and the risk it poses to the customer. Susanne will draw participants’ attention and focus on the red-flags and explain to them the ways by which they can reduce compliance and quality risk.

This seminar will consist primarily of lectures, interactive discussions and practical exercises. Susanne will equip the participants with the tools and techniques they need for ensuring compliance of their Quality Systems audits. They will walk away with an understanding of the key concepts and their practices, along with the ways of implementing them and use them as reference for future use

 

Article on “A Tour of the FDA”

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)

Want to know more: Article on “A Tour of the FDA”