Today we have the pleasure of celebrating the fact that we have reached the milestone of 200+ followers on WordPress. Since we started this blog, we have had such a great time connecting with everyone. we never expected to actually to connect with other people in the blogging community.
we are so incredibly thankful for each and every one of you who follows and comments on my blog posts. Please know that!
we would continue our blogging in these areas FDA Regulation, Medical Devices, Drugs and Biologics, Healthcare Compliance, Biotechnology, Clinical Research, Laboratory Compliance, Quality Management ,HIPAA Compliance ,OSHA Compliance, Risk Management, Trade and Logistics Compliance ,Banking and Financial Services, Auditing/Accounting & Tax, Packaging and Labeling, SOX Compliance, Environmental Compliance, Microsoft Excel Spreadsheet, Geology and Mining, Human Resources Compliance, Food Safety Compliance and etc.
The most critical aspect of risk management is the identification of potential areas of risk management. This helps the organization to stay focused on the areas in which it could possibly face risks, rather than taking an aimless view and shooting about in the dark.
In a very broad sense, the potential areas of risk management include all areas of a business, because simply no area of the business is exempt from a risk. Talk about finance, and it comes with a risk. What about manufacturing? And what about operations or marketing? How about human resources? In this very expansive sense, every area or activity of the business is among the potential areas of risk management.
Potential areas of risk management could lie simply anywhere
On top of these potential areas of risk management that each part of the business is prone to; there are also the other industry-related risks that inhere into any business. The risks of running, say, a firecracker business, are much higher than running a grocery store. So, potential areas of risk management should ideally include a very broad discussion on every aspect of risk management.
However, when one takes an overview of the potential areas of risk management instead of trying to break down the elements of each function in which there are potential areas of risk management; one can classify these among them:
Generic risks: As we have been discussing, any business, absolutely any business, comes with some degree of risk. And, each business comes with its own generic risk, such as falling short of funding at crunch times, core people leaving the organization at important times, logistics failures at critical times, and so on.
Product specific risks: As the title suggests, this kind of risk is specific to the product that the business deals with. Some products come with their unique risks, and hence, this kind of risk counts among the potential areas of risk management.
People-specific risks: These can happen in a business in which much depends on a few important people. The inefficiency or departure of such people could be among the potential areas of risk management for businesses or projects that are dependent on people.
Financial risks: Obviously among the top potential areas of risk management; financial risks come into play when the organization is not able to meet its bottom lines due to a variety of factors. Not getting funds on time, not getting payments from customers on time, not being able to service debts are some of the factors of financial risks.
Technology risks: Technology is a high area of risk, because it keeps changing at a breakneck speed. If organizations don’t keep up with the pace, technology risks could count among potential areas of risk management.
Market risks: Market risks are yet another of the potential areas of risk management because most businesses are run on the assumption or speculation that a market is going to grow at a certain rate or pace. If the estimate of this market goes wrong, it affects the business negatively.
Customer risks: The ultimate decider of the business is the customer. If a customer gets irate at a bad product or service and issues bad press, it could become one of the biggest of the potential areas of risk management.
Real estate risks: For some businesses, especially retail, the location of the business is a major factor. In many instances, the choice of location could often decide the fate of the business. Imagine setting up a high end retail store in the vicinity of a slum. Does that make sense? Yet, even if a business chooses the right location, it could sometimes be forced to relocate due to factors such as legal issues of the property, making this among the potential areas of risk management.
Finally, what needs to be said is that the list above is by no means a comprehensive one. The potential areas of risk management, as we have discussed at the beginning, are simply too many and too fluid and subjective. They could vary from market to market, product to product and business to business. A business that is perceptive about the market has to make the right assessment of the potential areas of risk management before it starts one. It should also be ready to face the potential areas of risk management if it is up against any factor that lies beyond its reach or forecast.
Orkambi (lumacaftor/ivacaftor) reduced levels of the main biomarker of the lung disease cystic fibrosis and improved the nutritional status of children with the condition, according to a Phase 3 clinical trial.
Vertex conducted the Phase 3 trial (NCT02797132) of Orkambi to evaluate its effectiveness and safety in preschoolers with two copies of the CFTR gene’s F508del mutation. The 60 children were aged 2 to 5. Mutations of the gene cause CF by producing faulty versions of the CFTR protein.
An indication of Orkambi’s effectiveness in the trial was that it reduced the production of the children’s sweat chloride and improved their nutritional status.
A sweat test is the gold standard for diagnosing CF because people with the disease have more chloride in their sweat than those who don’t. As for nutrition, the thick mucus that CF produces in the digestive system can prevent patients from absorbing nutrients and fat properly, leading to difficulty gaining weight and slower growth. CF also produces the mucus in lungs and other organs.
The Phase 3 trial also showed that Orkambi was safe and that the children tolerated it well. Researchers reported no adverse events besides those seen in studies of patients aged 6 to 11.
Based on the promising results of the trial, Vertex plans to submit a New Drug Application on Orkambi to the U.S. Food and Drug Administration during the first quarter of 2018. It will also ask the European Medicines Agency to extend the therapy’s availability to very young children.
Another Phase 3 trial (NCT02412111) that Vertex conducted evaluated a combination of tezacaftor and Kalydeco’s ability to reduce respiratory problems in patients more than 12 years old.
The study included 151 participants at 68 sites in the United States, Canada, Australia, and the European Union. The patients had one copy of the F508del mutation and one copy of another CFTR mutation.
Eight weeks of treatment with the combo led to a negligible improvement in a measure of patients’ lung function known as forced expiratory volume in one second, or FEV1. This is the amount of air that people can forcefully blow out of their lungs in one second.
The combo did lead to a reduction in sweat chloride that was larger than Kalydeco generated alone, however.
Given the results, Vertex has decided not to continue pursuing regulatory approval for the combo. One reason is that most patients older than 12 are eligible to receive Kalydeco by itself.
The FDA is expected to make a decision by February 2018 on a related New Drug Application that Vertex has filed. That application involves using the tezacaftor-Kalydeco combo to treat patients aged 12 or older who carry two copies of an F508del mutation or one copy of an F508del mutation plus another mutation. The FDA is giving the request priority review.
European regulators are expected to decide whether to approve the combo therapy in the second half of 2018.
Vertex has completed enrolling children 12 to 24 months for another Phase 3 trial (NCT03277196) of Kalydeco. It will evaluate the therapy’s safety in children less than 2 years old with a CFTR gating mutation and an R117H mutation.
Muscat: The number of Influenza cases has dropped by almost a quarter compared to 2016, but the Ministry of Health has warned residents to
be vigilant as flu season approaches.
According to the latest report by the MoH, deaths as a result of influenza have reduced over the past three years, with 2017 season seeing eight cases of deaths so far compared to 2015 which recorded 25 deaths.
As a semi-tropical country, seasonal influenza viruses continue to appear throughout the year in the sultanate. The virus starts in early September and can continue until mid-May.
“In 2017, there were 952 cases until the tenth of October of influenza while 2016 saw 1492 cases of influenza,” the ministry said in a statement.
“The Sultanate has recorded 25 cases of deaths in 2015, six cases of deaths in 2016, and eight deaths in 2017, all linked to the flu and most of those cases were among the groups most at risk of high risk of complications of influenza. Despite the high number of cases, it is still within the normal range compared to previous years,” added the ministry.
CISOs need to be thinking about their answers to critical questions the CEO is likely to pose.
Information Security Media Group asked seven security experts what questions they believe CEOs should be asking CISOs, and what information CISOs should arm themselves with to be prepared to provide answers. Following are eight questions and the experts’ suggested responses.
We have been investing in cybersecurity for a few years now. Would you say our organization is secure?
Israel Bryski, vice president, technology risk, Goldman Sachs: To pre-empt this question, the CISO should have a conversation early on with the CEO to determine the organization’s risk appetite. This will allow the CISO to align and prioritize security initiatives accordingly.
We are in the business of information and technology risk management, so the “Are we secure?” question is somewhat misguided. The question should be: “Are we managing risk according to our risk profile?” To answer this, the CISO should be able to easily demonstrate, based on a recent risk assessment, how the various cybersecurity initiatives and projects are in fact reducing risk, shrinking the attack surface of the organization and aligning the security program with the firm’s overall risk profile.
We have a board meeting next week. Can you talk about cybersecurity in a way they will understand?
Mischel Kwon, former director of US-CERT and deputy CISO for the Department of Justice; currently CEO of MKACyber: CISOs should be able to confidently say “absolutely” to this question. They should be able to speak with the board in a very businesslike way and articulate what they are doing with the company’s money and how they are protecting the company and its assets.
The key to being able to speak to the board is to base their program on a business-focused model. That business model shows their capability founded on their maturity, and that maturity is based on the probability of detecting specific types of attacks. These are the type of attacks that are most likely to happen to them, and this is the risk to the business, its goals and its reputation that these attacks bring.
Do you have enough money to do what you need to do?
Tim Youngblood, CISO, McDonald’s: Depending on where CISO sits, this can be a hairy topic. That can be a difficult conversation to say “I’m not getting enough.” It’s not easy if the CIO is in the room.
The best way to answer that is, “We may have current risks we are really well-funded to address, but there may be future risks we’ll need to fund and we still have some work to figure that piece out.”
A CEO is not going to write you a blank check. The CEO is going to look at the CFO and CIO and say, “The CISO needs money. You take it out of your budget and make it happen.” There is not an extra pot of money waiting for anyone, so making the clear case for why it is needed is key.
Is this really worth the investment?
Heath Renfrow, CISO at U.S. Army Medicine: The best thing a CISO can do when asked this question is have multiple options they can present to the CEO. Explain to them: Here’s the full issue. This is the total cost to fix this issue. This is what we believe the cost will be if this issue doesn’t go away and how much it will be should the vulnerability be exploited.
As an example, we didn’t know not know where our protected health Information and personal identifying information resided across all systems when I first got to Army Medicine. It would be a huge HIPAA concern if we got hit on that, or if there was a leak or a violation. It could have cost millions of dollars and many jobs. I tied in the overall cost and broke it down to how much it would be per end-user device to address it and it came out to be an about $3.43 per end-user device. Then I tied in all the results of HIPAA violations in the past few years and the fines associated with them. You get your senior leaders attention real quick with that approach.
Rick Howard, CSO, Palo Alto Networks, adds: Questions like this are sure to arise as corporate leadership attempts to understand the business risk associated with a cyberattack. As a result, CIO/CISOs should be prepared to explain the total cost of a potential breach. Everything from business disruption and loss of customers to consequential legal fees and remediation can rack up the bill more quickly than leadership may realize.
FDA commissioner Scott Gottlieb on Monday explained to attendees of RAPS’ Regulatory Convergence conference some steps FDA is taking to make the clinical end of drug development more efficient and effective.
Opening with a discussion of the ways in which the gap of time between the discovery of the science behind new treatments and the adoption of such treatments has been shrinking, Gottlieb outlined a few of the ways in which the agency is modernizing its approach to collecting and evaluating clinical information.
And on a day when the discussion of how much it costs to develop a new oncology drug is being hotly debated with the release of a new study, Gottlieb also discussed how the costs of drug development “are also high, and growing.
“There’s been criticism of the various estimates of how much it costs to develop a new drug,” he said, according to the transcript of his speech. “Moreover, on a relative basis, in many cases the costs of early stage drug development has grown at a proportionally faster rate than the cost of late stage drug development. In other words, inflation in early stage drug trials is rising faster than inflation in late stage development.
“By front-loading the cost of drug discovery, the broader biomedical community is making it harder to advance new ideas. It’s economically harder to capitalize the cost of an early stage drug program, relative to funding a later stage project. So frontloading the costs are a recipe for reducing the amount of new ideas that can be advanced.”
Biosimilars can be described as near-copies of an original pharmaceutical product that another company may have manufactured. These products are versions of an original or innovative product, but are officially approved.
There is a misconception that they are similar to or are the same as generics, but this is not so in reality. Generics differ from a biosimilars in one major sense: While a biosimilar is only highly similar to the original product (called the reference product); a generic is the exact replica of the original, whose chemical structure it takes. Further, there is a difference in the ways they are manufactured, their complexity (generics are small and biosimilars, big), and the data each is required to furnish for gaining approval.
The regulatory requirements for gaining approval for biosimilars are considered complex. Different approaches are used in different regions around the world.
Exploration of all aspects of biosimilars
Professionals in the areas of biological sciences, who deal with biologics, need to have complete knowledge of the whole host of issues associated with biosimilars, ranging from their manufacturing process to regulatory guidelines. Given the complex nature of the science and the approval process; lack of in-depth knowledge can result in their application getting rejected by the regulatory authorities.
It is to help avoid these situations that GlobalCompliancePanel, a leading provider of professional trainings for all the areas of regulatory compliance, will be organizing a two-day seminar. The aim of this seminar is to offer complete understanding of the topic of biosimilars, from its clinical aspects to what it takes to gain approval, and a whole host of other issues relating to biosimilars.
The Director of this seminar is Salma Michor, Founder and CEO of Michor Consulting Schweiz GmbH. Salma teaches regulatory affairs and clinical strategies at the University of Krems, Austria, and is an independent expert to the European Commission. Michor Consulting Schweiz’s clients include Johnson & Johnson, Novartis, Shire, Pfizer and Colgate Palmolive. To gain the immense benefits this seminar offers, please register by visiting All about biosimilars –from development to registrationThis course has been pre-approved by RAPS as eligible for up to 12 credits towards a participant’s RAC recertification upon full completion.
Discussion of all the areas relating to biosimilars
The FDA’s approval process for biosimilars is quite complex. If a biosimilar product has to gain FDA approval, it has to show that it is highly similar to an FDA-approved reference product. It has to show that the biosimilar product has no substantial deviation from the reference product in terms of effectiveness and safety. If there are differences, the company has to show that these are within the permissible limits allowed for the product.
The Affordable Care Act of 2010 made a few amendments into the way the Public Health Service Act (PHS Act) creates an abbreviated licensure pathway for biosimilars which are “biosimilar” to or are “interchangeable” with an FDA-approved biological product. This has to be in the manner described in the Biologics Price Competition and Innovation Act (BPCI Act).
An interchangeable biosimilar product is one that can be interchangeable with the reference product. That is, it can be substituted for the reference product without the advice of the prescriber. This means that the interchangeable biosimilar has met the FDA-approved reference product’s standards, as well as additional standards required for it to show interchangeability.
Complete understanding of the process relating to biosimilars
Over the two days of this seminar, Salma will help participants understand all the aspects relating to biosimilars. She will explain the challenges and choices that professionals in the field of biosimilars are up against. Biosimilar legislation in the US and the EU, the ways of establishing Target Product Profile (TPP) for a biosimilar, preclinical aspects, and quality, stability and preclinical testing will all be covered.
The Director will also lead participants into an exploration of the Phase I and Phase III clinical aspects relating to biosimilars, the existing guidelines on clinical testing and safety, registration process under the EMA, and market access for biosimilars in the US and Europe. She will intersperse these topics with relevant case studies.
This two-day seminar will offer value to those who deal with biosimilars in their work. These include Regulatory Affairs, Medical Officers and Clinical Trial Managers.
Salma will cover the following areas at this seminar: