Lonza news Tolvaptan identified as a treatment for autosomal dominant polycystic kidney disease

A phase 3 trial studying the effects of tolvaptan has found that the drug slowed the rate of decline in kidney function in patients with polycystic kidney disease…

Tolvaptan identified

A phase 3 trial studying the effects of tolvaptan has found that the drug slowed the rate of decline in kidney function in patients with the most common form of polycystic kidney disease, a condition with no cure.

This is the first treatment that targets a mechanism that directly contributes to the development and growth of the kidney cysts in autosomal dominant polycystic kidney disease

The results of the trial demonstrated tolvaptan’s ability to intervene in a way that slows kidney function decline in this population.

“This is the first treatment that targets a mechanism that directly contributes to the development and growth of the kidney cysts in autosomal dominant polycystic kidney disease,” says Dr Vicente Torres, director of Mayo Clinic’s Translational Polycystic Kidney Disease Center. “This in effect means it may delay the need for a kidney transplant or dialysis in patients with this disease.”

Autosomal dominant polycystic kidney disease is an inherited condition that affects 1 in every 500 to 1,000 individuals in the U.S. This disease is found in all races and sexes. Autosomal dominant polycystic kidney disease, which is the fourth most common cause of end-stage kidney disease, requires dialysis or a kidney transplant.

The disease causes a slow but relentless growth of cysts that damage the kidneys. In addition to negatively affecting the quality of life, the condition also causes hypertension and painful complications. The cysts, which can damage kidneys with their size, can develop in other organs, especially the liver.

Approximately half of individuals with autosomal dominant polycystic kidney disease eventually will require dialysis or kidney transplant by age 60.

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FDA approves first commercial product for peanut allergy prevention

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The approach towards preventing peanut allergies has changed dramatically in recent years. Now the US Food and Drug Administration has approved the first commercial product, called Hello, Peanut!, to help inform the public that early peanut introduction and regular consumption can reduce the risk of peanut allergy in young children.

The Hello, Peanut! introduction kit offers convenience in the form of packets of peanut powder blended with oat given in increasing quantities for seven days, as long as children tolerate it well. After which maintenance packets are recommended for use up to three times a week. The introduction kit is $25, and the maintenance kit sells for $20 for eight packets.

The FDA decision was informed by the landmark Learning Early About Peanut Allergy study published in 2015. It showed that high-risk children who regularly consumed peanut in infancy had far fewer peanut allergies by age 5 than their counterparts who avoided peanut over the same span of time. This understanding led to new guidelines published in 2017 by National Institutes of Health about giving peanut to babies to protect against peanut allergy.

Infants who have severe eczema or egg allergy are considered at high-risk of developing a peanut allergy. By offering peanut early in life – between 4-6 months of age – and continuing with regular consumption, we can prevent the onset of peanut allergy in many of these children. High-risk children should see their doctor before parents introduce peanut protein in any form. The physician may decide to do skin or blood testing.  If the test is negative, age-appropriate peanut products can be given at home. However, if a child tests positive, introduction to peanut is done under clinical supervision. If the child is deemed already allergic to peanuts, the guidelines recommend strict avoidance of peanut and ready access to epinephrine auto-injectors.

Read More: http://snip.ly/ktety#http://www.philly.com/philly/health/kids-families/fda-approves-first-commercial-product-for-peanut-allergy-prevention-20170926.html

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

Read More: http://snip.ly/jeird#https://www.lexology.com/library/detail.aspx?g=54c0daa5-aed0-4976-995b-5e0204c336c4

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.

 

Navigating the nuances of the Internet of Medical Things

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Earlier this year, Gartner released a forecast for the Internet of Things (IoT)predicting that 8.4 billion connected things will be in use worldwide in 2017 and that total spending on endpoints and services will reach almost $2 trillion this year. This forecast is certainly encouraging, but due to the prevalence and influence of the Internet of Things in almost all business arenas, it’s not wholly surprising. What is quite eye-opening is the massive growth potential of certain emerging sub-sectors of the IoT – with one of those being the Internet of Medical Things (IoMT).

The IoMT market, which stood at $22.5 billion in 2016, is expected to grow at an impressive compound annual growth rate of 26.2 percent to reach $72 billion by 2021, according to analyst and research firm Frost & Sullivan. For some perspective on that $72 billion projection, that’s roughly the same size as forecasts for the virtual reality market – which is generating a lot more buzz than the IoMT. And in addition to healthcare industry stalwarts such as Medtronic and Philips, tech giants like Apple, IBM and Cisco are currently developing applications for the IoMT. So, considering all of this economic promise for the IoMT, it’s worth taking a deeper look at the sector and the opportunities and challenges it presents.

 

Read More: http://snip.ly/qk357#https://medcitynews.com/2017/09/navigating-nuances-internet-medical-things/?rf=1

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”