Medivir delays start of pivotal study of remetinostat in cutaneous

UV light and radiotherapy and topical chemotherapy—all of which have tolerability issues.

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A year ago, Medivir was riding high after reporting midstage data that encouraged it to press ahead with a pivotal trial of its HDAC inhibitor remetinostat in cutaneous T-cell lymphoma (CTCL). It’s hit a snag though—it can’t agree on a design for the study with the FDA.

The Swedish biotech issued a statement this morning saying it had “decided to continue the discussions” with the agency on the design, and that delay means it won’t be ready to start the study this year as planned.

Its shares went on the slide as investors digested the update and tried to work out the implications, with the stock trading down more than 11% at the time of writing. Medivir bought the rights to remetinostat from TetraLogic Pharma for $12 million upfront in November 2016, and the drug is currently its lead pipeline candidate.

Christine Lind, Medivir’s CEO, said “it is of utmost importance that we design a phase 3 study that meets the expectations of the regulatory agencies. Further discussions with the FDA are needed to ensure that we can initiate a pivotal study that will allow us to bring this drug through approval and to patients.”

Medivir is hoping to open up a new CTCL market in the US for the drug worth an estimated $900 million, based on around 15,000 potential patients. It hopes it could provide an active treatment for early-stage CTCL patients who are currently just monitored until their symptoms progress sufficiently to need treatment with topical steroids, UV light and radiotherapy and topical chemotherapy—all of which have tolerability issues.

Last year all seemed to be on track, with Medivir reporting phase 2 data with remetinostat that showed a 40% response rate in 60 patients with a slow-growing form of CTCL called mycosis fungoides (MF), with the highest responses seen in patients treated with a 1% gel formulation of the drug.

The delay is the second pipeline setback for Medivir in recent months, coming after Johnson & Johnson pulled the plug on a hepatitis C virus program partnered with the Swedish biotech after deciding there was little market opportunity. It also lost former chief medical officer John Öhd who departed the company in March who has yet to be replaced. The company’s share price has been cut in half over the last 12 months.

FDA Final Guidance for [Industry]

If a firm disseminates HCEI to this audience, FDA does not intend to consider such information false or misleading.

FDA recently announced the availability of an important guidance for industry. The document, “Drug and Device Manufacturer Communications With Payors, Formulary Committees, and Similar Entities–Questions and Answers,” updates an Obama era-draft that outlines how companies can communicate off-label health care economic information about their products to drug purchasers like a health plan or a hospital.

Guidance

The guidance, “Drug and Device Manufacturer Communications With Payors, Formulary Committees, and Similar Entities–Questions and Answers,” provides answers to common questions regarding firms’ communications of HCEI (healthcare economic information) about their approved prescription drugs to payors. The guidance also provides answers to common questions regarding firms’ communications of HCEI about their approved or cleared medical devices to payors. In addition, the guidance addresses common questions relating to firms’ dissemination to payors of information about medical products that are not yet approved or cleared for any use and about unapproved uses of approved/cleared medical products.

There are two changes of note in the document. First, the guidance is expanded and includes medical devices along with drugs. Additionally, the guidance applies to communication about unapproved products or unapproved uses of already clearly drugs or devices.

According to FDA Commissioner Scott Gottlieb: “Taking into consideration the many thoughtful comments from stakeholders on our draft guidance, our final guidance now includes recommendations that are designed to enable truthful, non-misleading and appropriate company communications with insurers across a product’s lifecycle. The goal is to advance public health benefits such as increased cost savings from informed and appropriate coverage and reimbursement decisions. In this way, we can help ensure patients have more timely access to cutting-edge medical technologies. We can facilitate access by helping to reduce the overall cost of providing these benefits to patients. And in promoting access, we will advance important public health goals.”

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The guidance specifically addresses how the FDA intends to implement its language for HCEI disseminated in accordance with section 502(a) of the FD&C Act. In particular, FDA notes that an appropriate audience under this section of the law is HCEI that relates to an approved indication and is based on competent and reliable scientific evidence. If a firm disseminates HCEI to this audience, FDA does not intend to consider such information false or misleading.

Furthermore, HCEI should clearly and prominently present the information discussed in the guidance, including study design and methodology, generalizability, limitations, sensitivity analyses, and information relevant to providing a balanced and complete presentation. If HCEI includes material differences from the FDA approved labeling (e.g., new or increased risks, different dosing/use regimens, different endpoints, more-limited/targeted patient populations), it must present “a conspicuous and prominent statement describing any material differences between the health care economic information and the labeling approved for the drug”.

What does the medical device/biotechnology auditor look for and why?

How to maintain cleanliness in these areas and keep them presentable for the auditor.

Audits -both internal and external -are a vital tool for ensuring that a medical device or biotechnology product and its manufacturing operations meet prescribed regulatory requirements. Medical device and biotechnology auditors have guidelines on how to establish that the manufacturer has met standards such as ISO 13485, ISO 14971, or regulations such as 21 CFR Part 820.

Apart from going by these guidelines; the auditor also uses her intuition and observation gained from personal experiences to ensure that the facility she has visited is putting in place the right mechanisms to ensure what a state of control looks like. Given that a medical device audit can happen at just about any time; it is imperative for an organization in the medical device industry to be prepared for an audit any time, all the time.

What are the points that the manufacturer has to bear in mind to ensure compliance, so that the medical device audit goes on smoothly without events? What processes and methods should it follow to pass a medical device audit? These are critical questions that the manufacturer needs to have answers for.

Thorough learning on how to think like the auditor

A webinar from Compliance4All, a leading provider of professional trainings for all the areas of regulatory compliance, will equip participants with the knowledge needed to clear a medical device audit. José Mora, a Principal Consultant specializing in Manufacturing Engineering and Quality Systems, will be the speaker at this very important webinar. Over the more than 30 years that José worked in the medical device industry, he specialized in manufacturing, process development, tooling, and Quality Systems; he introduced process performance, problem solving, and Quality System methodologies.

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His medical device experience includes surgical instruments, PTA & PTCA dilatation and guiding catheters, plastic surgery implants and tissue expanders, urology implants and devices for the treatment of incontinence, delivery systems for brachytherapy, orthopedic implants and instruments, and vascular surgery grafts and textiles.

You can gain the benefit of his experience by registering for this webinar at http://bit.ly/2laaQ7G

The mental checklist

The important learning of this webinar is that José will provide insight into the auditor’s way of thinking. He will explain the kinds of clues an auditor might use in determining what areas to focus on. Rather that offer a heads-up on the regulations and standards relating to medical device audits; José will give knowledge of the general mental checklist.

Adhering to this checklist, an intuitive one created more out of the expert’s knowledge and experience rather than one gleaned from textbooks, will go a long way in determining how well an audit goes. He will also explain what kind of culture and environment a medical device organization’s management has to inculcate and encourage, so that the medical device auditor gains a better than normal perception of the organization.

An eye on the visual clues

An experienced auditor who knows her job does not need to look too far or deep to discover anomalies. The checklist she has in her mind is enough for her to match the facility’s requirements with those in it. At this webinar, José will bring his years of experience in helping participants understand how to frame a mental checklist of their own, so that they map it with that of the auditor.

He will emphasize the importance of housekeeping, proper maintenance, orderliness, and following safety rules. He will also highlight the importance of objects that serve as visual indicators in the facility, and will show how to follow them. Generally, in most medical device facilities, utility rooms and custodial closets are dumping places for several items. An experienced auditor is aware that what a medical facility hides in these places are a greater indicator of the facility’s orderliness than what are kept in the clean and presentable areas. The expert at this webinar will show how to maintain cleanliness in these areas and keep them presentable for the auditor.

José will cover these areas at this webinar:

  • What the auditor is looking for and why
  • The first five minutes
  • Touring the plant and manufacturing area
  • Entering and touring the warehouse
  • Entering and touring a cleanroom
  • Entering and touring lab areas
  • Reviewing documents
  • Reviewing the quality system
  • Summary

This course offers immense benefit to those in charge of handling auditors. These include Managers, Supervisors, Directors, and Vice-Presidents.

The desperate global need for (medical diagnostics)

This new list is a great step forward in making the most crucial diagnostic tests available globally.

Prince, a three-year-old boy, is brought to the emergency room in Monrovia, Liberia, with fever and a decreased level of consciousness. He is critically ill. He is treated for malaria.

Time is the only way to tell if this was the right treatment choice — but time may not be on his side. An astute nurse asks a visiting doctor if she can borrow a blood glucose monitor and nails the diagnosis: Type 1 diabetes, requiring urgent administration of insulin to save the boy’s life.

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While Prince was lucky to get a correct diagnosis on time, blood glucose monitors are rarely available in resource-limited clinics in sub-Saharan Africa.

Situations like this occur daily in low-resource settings: A sick patient, a clinical suspicion, but no confirmatory testing. As a result, many patients are misdiagnosed, receive unnecessary or harmful treatments, or die.

Thankfully, blood glucose measurement — an important laboratory test — just made it onto the first edition of the WHO Essential Diagnostics List (EDL).

This new list is a great step forward in making the most crucial diagnostic tests available globally.

However, future editions of this list will need to cover an even broader range of tests.

Beyond the ‘Big Three’

The WHO EDL, published on May 16, 2018, is a first step towards addressing the “diagnostics desert” that health-care providers and patients alike still face in many resource-limited settings.

WHO and its expert committee carefully put together a priority list of essential “in vitro” (laboratory-based) diagnostics to guide governments and health-care stakeholders on what tests to make available. The aim was to address the highest priority health-care needs.

Like its 40-year-old WHO sibling, the Essential Medicines List, this list promises to have a big impact on the availability of laboratory-based diagnostics globally, and on the quality of health-care delivery and population health.

WHO and its expert committee should be applauded for this essential accomplishment, including the effort to look beyond the traditional “Big Three” infections (TB, HIV and malaria).

While the list still has an obvious focus on infectious diseases, the consideration of essential non-communicable diseases (NCD) diagnostics is timely, if not overdue.

Read more at http://bit.ly/2JJu7ay

What is GDPR and How to Comply and How to Protect Privacy?

When not implemented properly, can lead to havoc for the company.

A core law concerning the protection of data of all people living in the European Union (EU) is the General Data Protection Regulation (GDPR). The GDPR, which is codified as Regulation (EU) 2016/679, has been formulated to fulfil two important purposes:

  • Placing the control of personal data in the hands of citizens and residents of the EU
  • Easing the regulatory process and environment and standardizing and unifying data protection regulations across the EU with the aim of making global business within the EU easier.

The legislative and secretarial bodies of the EU, i.e., the European Parliament, the Council of the European Union and the European Commission, consolidate and strengthen all aspects relating to data protection for all individuals within the European Union (EU) through the GDPR regulation.  The GDPR also addresses the area of export of personal data to regions outside the EU.

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Gain complete understanding of the GDPR

With the GDPR now being the law that companies that want to do business in the EU have to comply with; it is necessary for them to get proper and thorough understanding of all its elements. This learning is what a webinar from Compliance4All, a leading provider of professional trainings for all the areas of regulatory compliance, will be offering.

The speaker at this webinar is Derk Yntema, who has over 15 years of experience in ICT and security-management and has demonstrated capacity to implement innovative security programs that drive awareness towards information security and strengthen organizations and proven knowledge of privacy legislation and helping companies towards privacy compliance. Please visit http://bit.ly/2JurJbE to enroll for this webinar and get a full and proper understanding of the GDPR and how it matters to your business.

Explanation of the advantages and disadvantages of the GDPR

Derk will explain all the aspects of the GDPR, including its benefits and drawbacks. Among the advantages it carries are the greater and clearer insight it offers into Personally Identifiable Information (PII) processing within the company, the boost and synchrony it gives to security controls across the 27 EU members, the increased customer confidence that accrue from stronger safeguards for data protection, and the simplification of the process of doing business in the EU.

However, the GDPR, when not implemented properly, can lead to havoc for the company. It can result in unimaginably high levels of penalties, up to two percent of violating companies’ worldwide revenues, fines of up to € 20 million, complicated lawsuits, loss of reputation, and many liability cases.

The insights that Derk will offer at this webinar will help participants understand how to comply with the regulation in such a manner that their organizations fully derive the benefits of this regulation and neutralize the negative effects of not doing it right.

He will cover the following areas:

  • What is Privacy?
  • How to Protect Privacy
  • What is PII?
  • What is in the GDPR (General Data Protection Regulation)
  • How to Comply.

This webinar is of very high value to professionals such as Board of Directors, Supervisory Board, CxO’s and Compliance Managers/Officers.

http://data.consilium.europa.eu/doc/document/ST-9565-2015-INIT/en/pdf

http://mlawgroup.de/news/publications/detail.php?we_objectID=227

http://www.eugdpr.org/

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How to Comply with Both in the same Organization

A leading provider of professional trainings in all the areas of regulatory compliance.

Medical devices being a global market; it is not surprising to see many medical device companies marketing their products in both the US and the European Union (EU), two huge markets for these products. Given that these are two different markets; each has its own regulatory requirements. These requirements focus mainly on its Quality Management Systems (QMS). The QMS for the two markets are described in two documents: one by the international ISO standard 13485 and the other by FDA Quality System Regulation (21CFR 820).

On the surface, these two systems appear similar as they have many features in common. But as one delves a little into them, it turns out that they are not identical. A regulatory professional who markets medical devices into these two markets is likely to face crucial questions:

·        Is it possible, and is it permitted to have a QMS on the same site that complies with both requirements?

·        Should one QMS comply with both, and should that be put in place, or is it necessary to have two QMS coexist on the same site?

Thorough learning of the ISO and FDA QMS requirements

Answering questions such as these requires a thorough understanding of the background of both these documents, their legal status, what is expected by the regulatory authorities, and some basic concepts in quality and medical devices quality in specific. This is the learning that a webinar from Compliance4All, a leading provider of professional trainings in all the areas of regulatory compliance, will impart.

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At this webinar, Compliance4All brings the owner of ELC Consulting Services, Eyal Lerner, as expert. Eyal’s organization offers the pharmaceutical and medical devices industries support in all quality related issues, such as Preparation for inspections, CAPA and RCA, quality manual QSM, self-inspection (internal audits), compliance gap analysis, 3rd party audits (FDF and API’s), Management Review, GMP, GLP, MD: ISO 13485:2016 adjustments, DHF, DMR, V&V, 510k submission preparation and ISO 17025 (lab accreditation) and Validation. Eyal has preformed more than 100 audits in various facilities around the world as a lead auditor and auditor.

Please visit http://bit.ly/2JDpIWO to enroll for this webinar and have important doubts about the implementation of the QSRs of the ISO 13485 and 21 CFR 820 regulations clarified.

Offering insights into the two regulations

At this webinar, Eyal will give clarity to participants on how to understand what is required by both. It is necessary for regulatory professionals to understand semantic and substantial differences between the two documents. To give an example, while the ISO requires what it calls a Quality Manual (QM), the FDA QSR does not find mention of such a term. An in-depth look at the policies and Standard Operating Procedures facilitates the ease of handling of such terms. Eyal will focus on other issues that require more attention. He will explain the main features of these areas and describe what should be done from the perspective of a site or a manufacturer which wants to comply with both QMS.

The objective of this learning session is to offer a clear understanding to organizations in the medical devices industry that either develop or plan to market medical devices to USA and Europe of how to plan and implement a QMS that complies with both regulatory authorities. Eyal will help them ask themselves how to get the QMS right and make it work efficiently.

This session is aimed at professionals who are involved in implementing their organizations’ QMS, and includes Medical Devices Quality Personal (QA and QC), Laboratory Managers, R&D Researchers of Medical Devices and Combined Products, Quality Control Staff, and Regulatory Affairs (RA) Staff.

The speaker at this webinar will cover the following areas:

·        Introduction

·        Brief review of regulations and standard: status and history

·        ISO 13485:2016 short review

  • Principals
  • Quality management system
  • Management responsibility
  • Resource management
  • Product realization
  • Measurement, analysis and improvement

·        FDA QSR (21CFR820) short review

  • Quality System Requirements
  • Design Controls
  • Document Controls
  • Purchasing Controls
  • Identification and Traceability
  • Production and Process Controls
  • Acceptance activities
  • Nonconforming Product
  • Corrective and Preventive Action
  • Labeling and Packaging Control
  • Handling, Storage, Distribution, and Installation
  • Records
  • Servicing
  • Statistical Techniques

·        Comparison between both QMS

  • Common features
  • Variances between both

·        Terms/ vocabulary

  • Comparison between terms

·        Summary.

 

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How to Effectively Manage CROs/Vendors used in the [Pharmaceutical Industry]

It is imperative for pharmaceutical companies that outsource to get a thorough understanding of the elements of a sound outsourcing system.

Today’s globalized and interconnected world has made outsourcing indispensable. Outsourcing works to the benefit of both parties. Those who outsource can let other, independent companies, usually those that have specialized in this function, to take over several of their secondary processes, so that they can concentrate on the more productive and business-yielding activities. At the other end, the outsourced company earns substantial revenues by carrying out outsourced activities.

In the pharmaceutical industry, it is necessary and important to employ efficient and accurate strategies for this important activity. When projects are outsourced without proper safeguards and monitoring going into them, the outsourcing company has higher chances of inviting FDA actions such as Warning Letters, 483’s, etc.

Putting a selection process in place is very important

Companies should put in place robust structures that help them select vendors successfully and avoid scenarios such as these. These systems should also ensure that other critical aspects of outsourcing activities in GXP areas such as contract design, quality monitoring and risk minimization remain compliant. It is imperative for pharmaceutical companies that outsource to get a thorough understanding of the elements of a sound outsourcing system.

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Complete learning on how to select CROs/Vendors

A webinar from Compliance4All, a leading provider of professional trainings for all the areas of regulatory compliance, will educate participants on the elements of managing and avoiding common pitfalls with CROs/Vendors.

Laura Brown, an independent Pharmaceutical Project Management and Training Consultant and is Course Director for the M. Sc. in Clinical Research at University of Cardiff’s School of Pharmacy; will be the speaker at this course.

Dr. Brown has over 20 years’ experience of managing clinical research projects in the pharmaceutical industry. She has worked in Project Management for several companies including Wellcome, Hoechst Marion Roussel, Good Clinical Research Practices and Phoenix International. She is also co-author of two books on Project Management, including Pharmaceutical Project Management, a 2004 publication.

Please enroll for this webinar by visiting http://bit.ly/2JJHZVz

Learning on all elements of CROs/vendor outsourcing

At this webinar, Dr. Brown will combine theory and practical examples to offer a crucial overview of a sponsor’s responsibilities, which will give them the knowledge needed to ensure quality oversight.

In the course of this session, Dr. Brown will cover the following areas:

  • Understand how to Effectively Manage CROs/Vendors used in the Pharmaceutical Industry
  • Build an Understanding of your Responsibilities as the Sponsor or the Vendor: Identifying the Right Level of Management and Oversight
  • Discover Tools and Processes to Manage CROs/other Vendors
  • Gain an insight for CROs/other Vendors of what Sponsors Expect and how to Meet their Expectations
  • Measuring CRO Performance including Metrics and Key Performance Indicators
  • Managing and avoiding common pitfalls with CROs/Vendors.

Personnel who work in areas that involve one or more aspects of outsourcing CRO’s/vendors, such as Vendor Management, Global QA/Compliance, Contracts and Outsourcing, VPs, Directors, Outsourcing Department Staff, Purchasing, Finance and Contract, Management Staff, Managers and other personnel involved in selecting and managing cross, will benefit from this course.

Dr. Brown’s webinar is also aimed at helping personnel involved in vendor/CRO/CMO oversight (including those working in Clinical Research, Regulatory Affairs Pharmacovigilance, and Manufacturing) in the pharmaceutical, biotechnology, medical device and biologic industries who use Contract Research Organizations (CROs/CMO).

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