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Embracing Satan's PDUFA

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Monday’s public meeting on the FDA’s PDUFA VI Commitment Letter was a love-fest (mostly) – but as Theresa Mullin, Director of CDER’s Office of Strategic Programs, wisely noted, “the devil is in the details." Indeed.

The meeting was in three panels: Pre-Market Review and Post-Market Safety, Regulatory Decision Tools, and Administrative Enhancements.

A few highlights and comments.

Register for Aug 23 Lunch Briefing: 9 Biotech Value Killers - Daniels, Therasse


Opening up the day, CDER Director Dr. Janet Woodcock commented that, since the introduction of the first PDUFA in 1993, “It’s a brave new world.” That’s certainly true, but some have been braver than others. As Aldous Huxley wrote in his novel, A Brave New World, “Most human beings have an almost infinite capacity for taking things for granted.”

Janet (and later on others) spoke about PDUFA VI building a Patient-Focused Drug Development (PFDD) “Bridge.” In other words, moving from holding meetings to developing “fit-for-purpose tools to better reflect the benefit/risk calculus of patients.” A key point, via Janet, is that by doing this the FDA will not be the PFDD “bottleneck." The devil is in the details.

She also pointed to a fully operational and funded Sentinel as a linchpin not just for advancing safety specifically pharmacovigilance more broadly, but also for accelerating the ecosystem of Real World Evidence. RWE was a major focus of the meeting. More to follow. Albeit to say – the devil is in the details.

The factoid of the day was that in FY2015 (to date), the FDA has held over 3000 PDUFA meeting requests. Don’t even bother doing the math, because that doesn’t include the intense meeting preparation division staff must undertake prior to any actual face-to-face (or teleconference) encounters. This is a highly significant statistic insofar as there was much discussion on enhancing sponsor-agency communications. Is more always better? It depends. As Walter Gropius said, “Less is more. But more tastes better.” Communications must now be more about quality than quantity but, from a PDUFA perspective, how can that be measured?

In a small but important improvement, the FDA will provide 72-hour notice to all manufacturers whose products will appear in the quarterly FDAAA-mandated Public Notification of Emerging Postmarket Medical Device Signals (921 Safety Notices).

Per Sentinel, it was mentioned a number of times (including on an FDA slide) that it will be used to “inform important regulatory decisions.” Does this mean Sentinel data will be used “beyond risk” to also “inform” FDA thinking on potential new benefits? Will Sentinel become a tool for validating Real World Evidence? That was not discussed – but maybe it’s time for it to be put on the table, perhaps at one of the RWE meetings promised in the agency’s commitment letter.

As promised, the FDA will publish an updated version of its Structured Approach to Benefit/Risk Assessment in Drug Regulatory Decision-Making. It’s been an arduous journey. It’s important to remember that the key tenet of the PDUFA philosophy isn’t speed, but predictability, and the ability to understand regulatory decision-making (and, ultimately reproducibility) is crucial. This is important for many reasons, not the least of which are the ever-increasing costs of drug development.

There was (Finally! At last!) much discussion about “staff capacity.” Not just more, but better. Not just quantity, but quality. One specific conversation focused on the need for better reviewer understanding of and training in adaptive clinical trial design models. Let’s face it, there aren’t a lot of people inside the FDA considered expert in (among other things) Bayesian statistical design. Better MAPPS and SOPPS (Manual of Policies & Procedures, Standard Operating Policies & Procedures), that were promised, and they will help. There have to be internal rules of the 21st century regulatory road as well as external guidance but – the devil is in the details.

Some positive forward motion on biomarker prequalification. Among other things, the FDA will create a website listing the biomarkers it’s working on. Per the FDA presentation, this is designed to help stimulate further development. It’s a start and more needs to be done. (More, always more! But without the internal expertise, how is the FDA to parse its expert resources?) Maybe it’s time for a more serious discussion of intramural cooperation. FDA needs adequate resources – beyond PDUFA funding -- to provide advice and oversee review and decision-making. One solution is to partner with an external entity (an Intramural Biomarker Consortium-IBC) to develop early advice and serve as an expert sounding board for nascent biomarker efforts. The IBC could be a required or voluntary resource in the review process, especially for initial data package reviews. This approach would allow FDA staff to focus on their primary role of product review and regulatory oversight.

PDUFA is an important path, but it isn’t the only one.

All present (FDA staff, patient groups, industry representatives) were very excited about the opportunities of PFDD next steps and Real World Evidence. But how to get there? As BIO’s Kay Holcombe said, “We cannot put anecdotes on the drug label.” Real World Evidence is the new star on the precision medicine horizon. But the tool set for using this treasure trove of healthcare information is nascent and the tasks as are daunting as the opportunities. Patient passion is important to share. When combined with data and a more dispassionate understanding of regulatory paradigms, a patient-driven pathway can and must evolve into a tool used to impact regulatory decision-making. The devil is in the details.

The most decidedly unsexy but most honest and important part of the meeting came last – a discussion of “administrative enhancements” including electronic submissions and data standard activities, hiring capacity, and financial management. It’s important to note that PDUFA VI is the first time that “hiring capacity” has been directly addressed – and it’s about time. The FDA must have the firepower to not only retain but to aggressively recruit the best and the brightest. Again, it’s not just about body count (quantity) but quality. Quality is not what you put in. It’s what you get out. “Staff capacity” means more (a lot more) than PDUFA-measurable hiring numbers. We need a PDUFA quality metric.

In the immortal words of Admiral Hyman Rickover, “The devil is in the details, but so is salvation."

Register for Aug 23 Lunch Briefing: 9 Biotech Value Killers - Daniels, Therasse

 

This article originally appeared on DrugWonks.com and is shared here with permission.

Peter J. Pitts  is an authority on global regulatory policy issues and an Executive Partner at YourEncore. He is a former FDA Associate Commissioner, the Chief Regulatory Officer for Adherent Health Strategies, and the President of the Center for Medicine in the Public Interest, a policy institute he founded in 2004.


FDA 483 Response: Four Tips to Avoid a Warning Letter

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The receipt of a 483 at the conclusion of a facility inspection notifies a company of conditions the FDA believes are in violation of the Food, Drug & Cosmetic Act. If 483s are not properly addressed, the FDA can take further enforcement action to protect the public health – including issuing Warning Letters. Warning Letters not only indicate potential risks to patients, but also bring unwanted publicity, erode patient trust, and potentially restrict the approval of future products significantly impacting revenue. Therefore, companies must know how to mount a competent response.

In a recent webinar hosted by YourEncore, we outlined four steps medical devices companies can take after receiving a 483 to successfully allay FDA concerns and prevent further enforcement actions.

  1. Understand the process. Once an inspection takes place and deficiencies are found by the FDA, a Form 483 is issued. It is important to respond to the FDA within 15 days to ensure that the FDA considers your response before taking further enforcement action. The response should detail how the company is addressing the FDA concerns and specifically how these problems will be avoided in the future.  How these issues are addressed will determine whether or not the FDA will take additional action.

  2. Address and avoid common problems. Deficiencies tend to fall into three main areas: insufficient complaint handling, poor Corrective and Preventative Action (CAPA) procedures, and deficient Critical to Quality (CTQ) control in the supply chain. In a follow-up inspection, the FDA will look for data to prove that the company has found and fixed deficiencies, so they won’t happen again.

  3. Provide competent responses. A good response must convey a sense of urgency to address the problem. Lay out a specific plan with action items, dates and commitments. The timetable should provide scientific justifications. The plan also should include a commitment to allocate the necessary resources to make the needed corrections. This includes not just addressing the individual item listed by FDA, but also larger systemic issues. Outline a commitment to long-term quality and speak about changes as enhancements to existing policies and procedures.

  4. Speak to issues. The most important thing in communicating with FDA is to show why the problem won’t reoccur, so document the root cause and corrective action. Speak to the specific concerns of FDA. FDA wants to know exactly what’s being done and who is doing it. Clearly document the situation, include specifics, and explain how the corrective action will address the problem. 

Regulatory inspections are required as part of FDA oversight of medical device companies. Knowing how to handle 483s quickly and thoroughly can have a major impact on the success of your company.

To listen to the FDA 483 Response Best Practices webinar, click here. To talk with a YourEncore expert who can guide you through the FDA process, contact Mary Ellen Schipp (Maryellen.Schipp@yourencore.com).

About Victor Chance:Mr. Chance is a Strategic Advisor and member of YourEncore’s Medical Devices Practice. Previously, he was the Vice President of External Operations and Chief Supply Chain Chief Procurement Officer for J&J Medical Devices and Diagnostics, and he also served as Worldwide Vice President of Operations for J&J’s Cordis franchise.

About Judith Meritz, J.D.:Ms. Meritz is a Strategic Advisor and member of YourEncore’s Medical Devices Practice. Previously, she was the Associate General Counsel at Covidien/Medtronic and specializes in compliance counseling concerning FDA and other international regulatory authority matters including inspections, enforcement actions, product approval strategies, marketing and advertising programs, and complaint handling.

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Drug Pricing – What’s the Answer to High Cost Off-Patent Medicines?

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In the wake of the Turing, Valeant, and Mylan EpiPen pricing controversies, YourEncore Insights coordinated a conversation with two top experts to discuss potential solutions to some drug pricing dilemmas.  I interviewed Peter J. Pitts, former FDA Associate Commissioner and President of the Center for Medicine in the Public Interest, and Dr. Tim Franson, YourEncore’s Chief Medical Officer and former VP of Global Regulatory Affairs for Eli Lilly and Company.

The transcript is provided below: 

Combs:  In the U.S. there is a lot of news and attention being brought to Mylan recently with their EpiPen pricing, which has increased 500% to 600% in the last eight years according to sources.  Many are calling for price control, including politicians, but Peter Pitts, what say you? 

Pitts:  The right answer is to drive competition.  Competition is what lowers prices.  And in the U.S. when it comes to generic drugs and devices, it lowers prices quite significantly.  When you have a product like EpiPen that has been off patent for a while but has no generic competition for a number of reasons, the company is free to price the product however they see fit.  So I think it’s an opportunity for the FDA to put senior resources against applications, like Teva’s, that introduce competition to Mylan’s EpiPen, while ensuring they’re brought to market safely and effectively and lower cost.  At the end of the day, minus competition you’re not going to really have any on prices, whether it’s generic or on-patent competition within the same therapeutic category.

Combs:  Is there anything specifically FDA could do to help foster competition, particularly as it relates to high quality generics?

Pitts:  Yes. Last March, Rob Califf, the FDA commissioner, said the agency was going to prioritize generic applications for products that don’t currently have generic competition, from two to three years to six months.  That’s something the FDA said a couple of months ago, and the Mylan thing happened subsequent, so I would hope the FDA is beginning, with its new GDUFA funding, to really get serious about that.  But, you can’t just push the button and have things change; these things are a process.  It’s also important for people to understand that when products don’t come to the market as quickly as they’d like, or if they don’t come to the market at all, it’s not necessarily the FDA’s fault.  There are lots of things that go on that aren’t seen by the public, because some information is confidential, proprietary to the company.  If the company wants to share it, that would be permitted, and I think they probably should answer some questions.  But at the end of the day, the best thing the FDA can do is really work with companies to help them solve problems and get these products to the market as quickly as they can.

Combs: To your point around FDA involvement, with any organization there are resource constraints, and the FDA is no different.  There’s always more needs than there are people, so where does high-quality generics fit into the broader to-do list of the FDA? 

Pitts:  High-quality generics is very high on the list of things the FDA wants to do, needs to do, and in fact is doing.  And this is especially true for first-to-market generics because not only is it an issue of price competition and access, it’s also an issue of avoiding drug shortages.  When you only have one manufacturer for a product, if that manufacturer has manufacturing issues, drug shortages can occur and that’s a public health crisis as well as a pricing crisis, which is really what everybody wants to talk about.  All of these things places high-quality generics and certainly first-to-market generics, very high up on the list of FDA’s priorities.

Combs:  And I think I’ll bring Tim into this conversation as well.  Tim obviously has a lot of experience in industry as well.  I suspect that in your career some products in your portfolios faced some price scrutiny, just as probably every pharma company has.  What’s your perspective on drug pricing and the public response?

Franson:  Well, that’s an interesting question, Jim.  I like Peter’s responses, and I do think that when these issues like with Mylan or Turing come up, they’re clearly not mainstream.  They’re not representative of what general pricing issues are.  But they raise some interesting discussions about pricing as a surrogate for trust and transparency.  And certainly although pricing has been quite in play in the political environment, on the transparency piece I guess I’ve never understood why industry has not taken a much higher profile approach to the value of their benefit over risk for certain products.  In other words, the last big pricing brouhaha we saw in the press several years ago was with the new hepatitis C compounds.  And on the face they look excessive in price, but if you looked at the overall value, you were talking about transitioning patients from multiple courses of interferon or other long term treatments to something that over six months would cure them, an absolute cure.  Now, what is the value of that?  And how can one avoid minimizing that value by focusing only on price?  So I think there are some very important examples we can work with to help us re-achieve price-value balance.  And to me, the balance comes back to very objectively assessing benefit and risk for patients.

Pitts:   That’s exactly right.  It comes down to getting the right medicine to the right patient at the right time.  The hepatitis C situation is a great example, because now people are being cured twice as regularly with half the amount of treatment, and the value to the U.S. healthcare system is going to be enormous.  It’s going to far outweigh the short-term cost, and that’s what many analyses have shown.  Of course, the initial out-of-the box reaction was, “Oh my gosh, these drugs are so expensive.”  But to Tim’s point, we need to talk about the value in the long term rather than the price in the short term.

Combs:  And so, Peter, there are a lot of calls right now for price controls, but what is the problem with controls?  Why not go that direction?

Pitts:  Price control would make the problem worse.  Price controls stifle innovation.  And without innovation you don’t get new products.  And without new products you don’t have competition.  I think that’s the last thing that we want.  It’s a very facile, soundbite, political solution, unfortunately in the political season to say price controls.  But that’s what happens when companies have a strategy of raising prices to increase their profits and also to allow insurance companies to demand a larger discount.  It’s an ecosystem of pricing and value.  Right now people are only focusing on the pharmaceutical industry, but to Tim’s point, whether you’re looking at Mylan or you’re looking at Turing Pharmaceuticals, these are not innovator companies.  These are generic companies.  And generic drugs in the U.S. are cheaper, are less expensive than the same generic product in Canada or Europe.  People forget that.  And when they think about the price of drugs, they also generally mean their copay at point of dispensation at the pharmacy.  And that is something that is not necessarily a pharmaceutical issue; it’s as much an insurance issue as anything else.  It’s easy to say “price controls,” it resonates and people like to hear it, it creates a bad guy that you can aim at, but it would only make the problem worse and also stifles innovation which would be the ultimate sacrifice.

Combs:  Peter, you supervised the FDA’s Office of Public Affairs and have some background in external relations.  What would be your counsel to companies that are considering a price increase?  There are many justifiable reasons for raising pricing in any industry, but is every pharma company going to face the same kind of scrutiny?  Are there any lessons learned or any guidance you would give to not just Mylan but also the broader industry as they think about their pricing going forward?

Pitts:  That’s about a five-hour conversation.  But I think the quick answer is:  Pay attention to what you’re saying, and certainly in a political season.  When Heather Bresch, the president of Mylan, says “Hey, I’ve got a business to run,” that’s not going to calm people’s anger. It’s only going to fan the flames.  People don’t understand the pharmaceutical business. That’s not their responsibility.  But it is the responsibility of the pharmaceutical industry to prioritize being in the public health business rather than being in the profit-making company business.  They’re both important and they’re both legitimate, but I think the pharmaceutical industry needs to stand up and become the poster child for responsible players in healthcare as opposed to being painted as the profiteering bad guys.

Combs:  Excellent.  Well with that, I think we can wrap up here.  But before we do, any kind of parting thoughts, Tim, that you’d like us to end on?

Franson:  I’ll just offer one.  And that is reflecting back on when I started in the field of medicine over three decades ago, I’m trying to imagine if innovation was stifled then by price controls what a different world we’d be in.  We wouldn’t be looking at the way we treat ulcer disease now with pills instead of major surgery.  We would not see all the innovations with devices, robotic surgery.  And I think we should be very cautious when we talk about price controls and the potential chilling effect on innovations of any of those kind of measures.  We’ve done pretty well as a society in terms of improving health and survival via policy support for innovations, and let’s focus on those successes and how we got there.

Combs:  Excellent.  And I’ll ask the same question to you, Peter, before we wrap up.  Anything you’d like to close with?

Pitts:  Amen to that.  We’ve got to protect sustainable innovation.  We’ve got to insert competition into the marketplace any number of different ways, and the FDA plays an important role in that, and transparency is a key part to understanding how the whole system works.

Are You Ready for EU MDR?

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Back in 2010 when European regulators announced plans to overhaul legislation on how the European Union oversees medical devices, the industry knew that it would take time for changes to take effect. With the EU reaching agreement on the regulation, it appears that the final Medical Device Regulation (EU MDR) may take effect as early as January 2017. 

I sat down with Jon Lange, a Principal in the Advisory Services practice of EY and its EU MDR lead, to outline how medical devices companies can prepare. Interestingly, during the webinar, only 26 percent of participants said they had completed an assessment of their EU MDR readiness, while 74 percent said EU MDR readiness either wasn’t on their radar or they were waiting until the regulations were final before beginning major prep.

There are two areas in which medical devices companies must prepare for EU MDR—strategy and compliance—and doing the strategic work early will position medical devices companies favorably.

In my conversation with Jon Lange, we outline the following tips:

  • Address strategic issues early. On the day regulations are published, medical devices executives will have so much to do to be compliant that the strategic aspects will fall to the wayside. Laying the business strategy early allows businesses to be in the most competitive position to move forward when regulations are published.

  • Use EU MDR as an opportunity to fundamentally transform your business. Ask questions such as: How can we enhance our brand? How will this shape our portfolio? Will this present M&A opportunities? Should we cull our SKUs?

  • Understand your risk. It’s important to map out the risk across your business, including in R&D, Supply Chain, Regulatory Affairs, Quality Assurance and Commercial. EU MDR will touch each of these areas.

  • Gather the facts. The most important aspect of planning is getting the facts. Once you have a good understanding of your risks and gaps, how the legislation is likely to impact your current product line, and the opportunities EU MDR will present, you can start mapping an EU MDR readiness plan to take to your leadership team.

  • Don’t wait for 100% clarity. The time to act is now.

To listen to the EU Medical Device Regulation webinar, click here. To talk with a YourEncore expert, contact Mary Ellen Schipp (maryellen.schipp@yourencore.com) .

Pharm-ers Almanac 2016: The Butterfly Effect

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There is a saying, coined by the late Edward Lorenz, an early pioneer of the chaos theory, that “the flap of a butterfly’s wings in Brazil can set off a tornado in Texas.” This observation is known as the butterfly effect: the concept that small changes can have large downstream effects.

Today, the life sciences industry is operating at the tip of a butterfly’s wings.

This evolving milieu is characterized by a shifting regulatory environment, calls for pricing reform, mounting pressure to innovate, concerns over data quality and drug safety, and worries about the industry’s reputation.

Life sciences experts at YourEncore have identified six areas in the pharmaceutical industry that are particularly susceptible to the butterfly effect. These areas are poised for the kind of disruption that initially may seem insignificant, but may have a large and lasting ripple effect.

Each of these are discussed in detail, along with our point of view (POV) in Pharm-ers Almanac 2016: The Butterfly Effect.

 

Biosimilars Take Flight

Biologics are among the most expensive drugs in the pharmaceutical industry. With eleven biologics losing their patents this year, many biosimilars are expected to enter the market. Despite a discounted rate of 20-30 percent, a 2014 Rand analysis predicts that biosimilars will only decrease U.S. spending on biologics by about 4 percent, begging the question: is the manufacture of biosimilars a necessary evolution of the market or a distraction from big pharmaceutical’s innovation and investment in other new drugs to address unmet needs?

 

FDA Nets Drug-Device Combination Products

The popularity of drug-device combination products has been soaring. The market for combinations of drugs, biologics, and devices is expected to grow to $31 billion by 2019. However, growth in this sector is threatened by a lack of alignment with FDA, in which there are countless examples of combination products taking upward of five years to receive approval. How can drug and med device companies best operate within the regulatory framework today and in the future to deliver innovative solutions to patients in a reasonable time frame?

 

Price Puddling

With the price hike of Mylan's EpiPen making recent headlines, this hot topic is leaving the pharmaceutical industry in a difficult position. How do companies balance the thirst for innovation by patients and the value their medicines deliver with the cost of delivering that innovation and building a sustainable business?

 

CROs vs. Cocooning

Nearly two-thirds of pharmaceutical and biotech companies spent between $10 million and $50 million in 2014-15 for outsourcing. However, issues with quality have some pharmaceutical companies talking about retaining in-house resources, with nearly 35-55 percent of sponsors reporting moderate to significant dissatisfaction with a contracted project. What’s the cause of this? Does the success of a CRO engagement begin with the selection process, or does it come down to management?

 

Regulatory Wing Clipping

Legislative and regulatory changes in the pharmaceutical industry are in their chrysalis stage, and soon will be ready to radically emerge and make their impact on developers. By the looks of it, we should expect an invasion of new regulations and their interpretation, which will slow down the rate of approvals. Are all these upcoming regulations a barrier or an opportunity?

 

Inspection Migration

The rising costs of drugs and biologics calls for increased drug imports, and worries about supply chain quality are putting oversight of drug development, manufacturing, and marketing by regulators under a magnifying class. How can the pharmaceutical industry prepare for increased quality oversight?

 

Individually, any of these trends can be disruptive to a drug developer. In aggregate, they can pose a significant butterfly effect on the industry. While developers cannot stem the tide of change, YourEncore can help you anticipate and prepare for these changes.

Read more about what YourEncore experts have to say about the changing life sciences industry by downloading the highly anticipated Pharm-ers Almanac 2016: The Butterfly Effect.

 

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Considering Biosimilars – 3 Key Areas to Assess in Your Strategy

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Innovators simply cannot afford to ignore biosimilars. These products have the potential to create significant clinical and financial advantages for both patients and payers.

Originator firms must figure out how to integrate the presence of biosimilar products into a therapeutic franchise portfolio strategy — even if the innovator elects not to manufacture a biosimilar. To start, originators must develop a strategy for biosimilar products as part of building and maintaining a therapeutic franchise. This requires defining the portfolio effects of biologically similar therapies on lifecycle management as well as clinical migration. The originator needs to assess three areas:

1. Whether there is a franchise commitment to a therapeutic category

2. Whether presence in that segment is “once and done” with a specific product

3. Determine the appropriate response for the launch of biosimilar agents

Key to building this strategy is a firm understanding of how the clinical, regulatory, and commercial environment for biosimilar products is evolving. Equally important is establishing scenarios that lead to developing follow-on therapies that are clinically and economically differentiated from the originator and the biosimilar products.

Sponsors also should look at partnership opportunities available to bundle a biosimilar with a follow-on innovative therapy. Such bundles may lead to lower prices and entry into new therapeutic categories, giving sponsors the headroom needed to generate new therapies.

All this requires knowledgeable pharmaceutical executives with experience modeling the pathways to secure clinical differentiation, achieving expedited regulatory review, and building sustained value in therapeutic franchise portfolios.

This is an excerpt from Pharm-ers Almanac 2016: The Butterfly Effect. Read the full report here:

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About the Author: David Wierz is a YourEncore expert, who regularly consults on commercial strategy for biosimilar products as well as biologics, vaccines and small molecules. He has 25 years of experience working with physician groups, payers and life science companies, including Wyeth (Pfizer), Novartis, Baxalta and Takeda Oncology to name a few.

Navigating Drug-Device Combination Product Approval

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The popularity of drug-device combination products has been soaring. These products — which comprise combinations of drugs, biologics and devices — had sales reaching $22 billion in 2014, and the market is expected to grow to $31 billion by 2019.1

However, growth in this sector is threatened by a lack of alignment within the FDA, which is slow to approve drug-device combination products.  For those considering developing a combination product, I thought it helpful to provide an FDA primer to help people navigate the approval process and understand where the challenges are.    

Medical device products range from drug-coated stents and prefilled syringes, to antibodies combined with drugs for targeted cancer therapy, and products that mimic or replace organs, such as an artificial pancreas.

Combination products pose unique challenges because they may involve new, complex technologies and because their review at the FDA usually involves the expertise of more than one FDA center. With regard to medical devices, how the product can be used is as important as the product itself.

These products are reviewed by the medical product center at the FDA that is  responsible for the constituent part providing  the product’s primary mode of action.  Manufacturers of combination products have to  submit applications through more than one center for regulatory approval, a confusing and delay-prone process. Also, differences in communication, policies, practices, systems, and application types can be challenging when the centers work together.

The FDA’s Office of Combination Products (OCP) oversees and coordinates this activity. OCP was created over 10 years ago with a mandate to ensure timely and consistent premarket and postmarket regulation of combination products. While OCP is charged to be the mediator and has been successful in getting the centers to talk, it has no authority to direct an outcome. When disagreements arise, OCP has no mechanism to facilitate or resolve them.

With little alignment in the FDA when it comes to the approval of combination products, there are countless examples of combination products taking five years or more to receive approval.

When looked at through the devices lens, a combination product has to meet a drug standard, which often requires large clinical studies and is not what people in the medical device industry are accustomed to. When looked at through the drug lens, it would be inappropriate to lower the standard just because there is a device delivery system.

Development of combination products often involves expanded preclinical study programs, including human factor testing to determine if patients use a device delivery system appropriately. The questions addressed by human factor studies overlap with those addressed by medication error assessments, an area of user-product interaction evaluation commonly applied to drugs.

The FDA has recommended that manufacturers and clinical researchers design human factor studies to put products through real-life circumstances with close monitoring and integration with patients. According to the FDA, studies of combination products should be conducted as part of the product design controls process, and before commencing major clinical trials. One step forward is a recent draft guidance that explains human factor studies recommended for combination products.2

With no internal alignment in the FDA around how centers can work together, it’s not surprising that there is also confusion about how to appropriate user fees, which should be used to ensure that the FDA reviews a combination product in a reasonable amount of time. With combination products, the user fee isn’t apportioned to the respective product review centers — drugs and devices. Therefore, they tend to fall to the bottom of the prioritization heap.

Another significant challenge is that manufacturing controls and quality systems vary considerably for drug and device companies, as do product labeling and postmarketing requirements. Also, it is unclear how the revision of medical device regulations by the EU authorities will impact the development and authorization of combination products.

The question is how drug and medical device companies can best operate within the regulatory framework today and in the future to deliver innovative solutions to patients in a reasonable timeframe. Until there is more alignment within the FDA, companies will need to build both drug and device expertise across regulatory, quality, clinical, and the supply chain to work with two reviewing centers simultaneously and synthesize two sets of regulatory requirements. 


Sources:
(1) “Global Markets for Drug-Device Combinations,” BCC Research, January 2015.
(2) Human Factors Studies and Related Clinical Study Considerations in Combination Product Design and Development, FDA.

This is an excerpt from Pharm-ers Almanac 2016: The Butterfly Effect. Read the full report here:

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About the Author:Judith Meritz, J.D. is a Strategic Advisor in YourEncore’s Medical Devices and Diagnostics Practice and was previously the Associate General Counsel at Covidien/Medtronic. Ms. Meritz specializes in compliance counseling for FDA and other international regulatory authority matters for medtech companies.

Leading the Bristol-Myers Squibb Transformation (Part 1) (Ep. 1 of The Pharmcast)

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We are pleased to announce that YourEncore has officially joined the podcasting ranks with the launch of the YourEncore Pharmcast, your weekly dose of wisdom from top life science minds.   Each week we will speak with top industry experts in our network or beyond about hot industry topics, leadership, and the path to success. 

For our maiden voyage of the Pharmcast, we talk with a trio of former Bristol-Myers Squibb executives, who developed and delivered BMS’ “String of Pearls” strategy that delivered first-class medicines to patients and positioned BMS as a clear leader in the immuno-oncology space.

What follows is a tremendous case study on leadership, portfolio decisions, organizational transformation, and most importantly, developing life-changing medicine for patients.

Jim Cornelius, a current YourEncore board member and former BMS CEO, gathered some of his former colleagues: Dr. Elliott Sigal, BMS’ former Chief Scientific Officer, and John Celentano, a former SVP and also a YourEncore board member, to share their story of the BMS transformation.

We begin the conversation with Jim Cornelius, who was a recent addition to the BMS board, and in quick order, found himself offered the CEO position for Bristol.  What was originally thought to be an interim position turned out to be a three-year assignment for Cornelius, as he and his senior management team broke with conventional wisdom to spin off businesses, cut costs, and focus its development on a few winning  ideas.

What followed was a large scale corporate transformation that helped catapult the immuno-oncology revolution, delivered first in class medicines, and ultimately shaped the industry and patients lives for years to come.

We broke this discussion into two episodes, so if you like what you've heard, check out part II here

The Pharmcast is now available for download on iTunes, Google Play, and Stitcher. If you like what you hear, share it with your friends and write a review or give us a rating on your podcast store. 

 

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Leading the Bristol-Myers Squibb Transformation (Part 2) (Ep. 2 of The Pharmcast)

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A continuation of our maiden voyage of the Pharmcast, we talk with a trio of former Bristol-Myers Squibb executives, who developed and delivered BMS’ “String of Pearls” strategy, that delivered first-class medicines to patients and positioned BMS as a clear leader in the immuno-oncology space.

In part 2 of the roundtable, we start off with John Celentano, who was responsible for delivering a large organizational transformation to fund reinvestment in their innovation pipeline.

With the "string of pearls" strategy in place, it was time to figure out how BMS was going to pay for their acquisitions, and continue to fund the development of new products.

Knowing they needed to fundamentally change how they ran the business, the BMS board set out with something they referred to as "the productivity transformative initiaitive."

After restructuring the way the company ran, by shrinking the company and increasing productivity, the board members of BMS set their sites on investing in innovative products and technologies - primarily biologics. At the time, only about 6-7% of major drus were biologics. But, the BMS executives estimated that in the future, the biologic market would near 25%.

In what was, at the time, a make-or-break decision, the BMS executives made a leap-of-faith, $2.3 billion acquisition of a small company called "Medarex," a company that focused on immuno-oncology medicines.

Despite originally being heavily criticized by leaders in the oncology field for the alliance, BMS was well on their way to becoming a leader in the immuno-oncology space. 

What follows was a decade of successes, adding $100 billion in shareholder value to the company, and ultimately getting life-changing drugs out into the market. 

The Pharmcast is now available for download on iTunes, Google Play, and Stitcher. If you like what you hear, share it with your friends and write a review or give us a rating on your podcast store. 


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9 Biotech Valuation Killers (Ep. 3 of The Pharmcast)

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This week in the Pharmcast, we discuss "9 Biotech Valuation Killers" with Dr. Brian Daniels, the former SVP of Global Development for Bristol-Myers Squibb and now Venture Partner with 5AM Ventures, and Dr. Don Therasse, former VP of Global Patient Safety and Medical Affairs for Eli Lilly & Company.  

According to a global study by Mergermarket and Reed Smith, 94 percent of senior life sciences executives plan to make an acquisition in 2016, so biotech firms need to do everything possible to make sure they are properly positioned.

These gentlemen have evaluated hundreds of assets throughout their careers, so we asked them to share their best advice to biotechs looking to attract big pharma suitors and at maximum valuations.

The first rule is to have good science. This is a given. After that, follow nine rules to maximize valuations, which Drs. Daniels and Therasse present in episode 3 of The Pharmcast.

The Pharmcast is now available for download on iTunes, Google Play, and Stitcher. If you like what you hear, share it with your friends and write a review. 


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Billion Dollar Bets: Lessons on Innovation from a BMS Chief Scientific Officer, Dr. Elliott Sigal (Ep. 4 on The Pharmcast)

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Each week in The Pharmcast, we talk with top industry experts from our network or beyond about hot industry topics, leadership, and the path to success. This week in The Pharmcast, we catch up with Dr. Elliott Sigal, the former Chief Scientific Officer of Bristol-Myers Squibb.

In 2012, Dr. Sigal was called by Scrip Pharma Intelligence "The Best R&D Chief in Pharma," largely for helping deliver BMS' "String of Pearls" strategy that helped position BMS as a major player in the immuno-oncology space.

As referenced in both Episode 1 and Episode 2 of The Pharmcast,  Dr. Sigal, through internal development and making sizable asset acquisitions, developed a pipeline of first-in-class medicines that helped transform the company, the industry, and most importantly, the lives of hundreds of thousands if not millions – of patients. 

When asked what his "secret sauce" to success was, he said it was not just one thing, but the culmination of many things.  Below are the three of the things Dr. Sigal attributed to his success.

1) Team. Despite being called "The Best R&D Chief in Pharma," he acknowledges that this was the recognition of something greater: his team. 

2) Experience. Sigal's unique background, which combined engineering and medicine as a Physician Scientist, brought him an interesting, advantageous outlook on the large organizational issues in the pharmaceutical industry. 

3) Focus. Dr. Sigal felt he and his team benefited from having focus:  a focus on the patient, a focus on the changing landscape of medicine, and a focus of his time on areas where he uniquely could add value.

Join us in Episode 4 of The Pharmcast, as we talk more with Dr. Sigal about leadership, portfolio decisions and what made him successful in his journey. 

The Pharmcast is now available for download on iTunes, Google Play, and Stitcher. If you like what you hear, share it with your friends and write a review or give us a rating on your podcast store. 


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Holiday Wish-Lists and Watch-Outs From Regulatory Leaders (Ep. 5 on The Pharmcast)

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 This week in The Pharmcast, we asked two of our top regulatory policy Experts to give us their holiday wish-lists and top things to watch for in 2017.

Leading the discussion is Dr. Tim Franson, who is the former VP of Global Regulatory Affairs and Global Patient Safety for Eli Lilly & Company, current Chairman of the Board for the Critical Path Institute, and YourEncore's Chief Medical Officer.

Joining him is Peter J. Pitts, former FDA Associate Commissioner and current President for the Center for Medicine in the Public Interest, a policy institute he founded in 2004. Peter was named one of the "300 Most Powerful People in American Healthcare" by Modern Healthcare Magazine, and frequently contributes to national media outlets including CNBC, Fox News and the Wall Street Journal.

As Dr. Franson and Peter discuss what they're hoping for under the tree this year and what situations could potentially turn into lumps of coal – they fixate on the letter "P" and all the changes, from "people" to "policy," that could be coming to the industry in 2017.

Join us in Episode 5 of The Pharmcast as Dr. Franson and Peter begin to dissect these questions and what they're wishing for, among these and several other P-related hot topics for 2017.

The Pharmcast is now available for download on iTunes, Google Play, and Stitcher. If you like what you hear, share it with your friends and write a review or give us a rating on your podcast store. 


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From Big Pharma to Biotech C-Suite: Industry and Career Insights from Dr. June Almenoff (Ep. 6 on The Pharmcast)

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This week on The Pharmcast, we talk with Dr. June Almenoff, former President, Chief Medical Officer and Board Member of a biotech, which was recently acquired in 2014 by Actavis for $1.2 billion.

With an M.D. and Ph.D., Dr. Almenoff began her career as a faculty member at Duke University before landing a position at GlaxoSmithKline (GSK) in their medical R&D division and scientific licensing.  However, after 13 years at GSK,  a new opportunity presented itself in 2010 when Dr. Almenoff was invited to head up a small biopharma company as President & CMO, which she would soon lead into a $1.2 billion acquisition.

Almenoff helped the company successfully progress its Irritable Bowel Syndrome drug from Phase 2 through the FDA submission, which the FDA approved in 2015 on its first review cycle.

Everyone talks about learning from failure, but from our discussion with Dr. Almenoff, we learn from success.  I asked Dr. Almenoff about her keys to success, drug development trends, and the lessons she's learned across her 'big pharma' and biotech career.

The Pharmcast is now available for download on iTunes, Google Play, and Stitcher. If you like what you hear, share it with your friends and write a review or give us a rating on your podcast store. 

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Bioethics in Biopharma: Lessons on Safety, Big Data and Bioethics from Dr. Don Therasse  (Ep. 7 on The Pharmcast)

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This week on The Pharmcast, we talk with Dr. Don Therasse, former VP of Global Patient Safety for Eli Lilly & Company, and current Strategic Advisor for YourEncore.

With over 26 years of experience working with Lilly, Therasse held a diverse set of roles ranging from clinical research physician, to medical director, to medical affairs leader, and finally to patient safety leader.  Beyond drug safety, Don Therasse is a thought leader in bioethics and is credited with building Lilly's internal bioethics group.

In our conversation with Dr. Therasse, we explore experiencing a major patient safety event, building a bioethics program, the promise (and limitations) of big data in patient safety, and success formulas for transitioning into new departments within a large biopharma company. 

If you have topics you would like YourEncore to explore on future episodes of The Pharmcast, please email Pharmcast@yourencore.com with feedback or suggestions.

The Pharmcast is now available for download on iTunes, Google Play, and Stitcher. If you like what you hear, share it with your friends and write a review or give us a rating on your podcast store. 

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FDA 483 Responses: Avoiding Common Pitfalls and Deficiencies (Ep. 8 on The Pharmcast)

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This week on The Pharmcast, we explore best practices and common pitfalls in FDA 483 Responses. 

In 2015, there were an average of 175 inspections and 10 warning letters issued per month by the Center for Devices and Radiological Health alone.  Regulatory inspections and 483 findings are a part of being a life sciences company.  However, how companies respond can have a major impact on patient safety and business performance.

To share best practices, we held a special presentation with two of our top medical device experts, Victor Chance and Judy Meritz, to outline what they’ve learned over the course of their 20+ year careers in the medical devices industry.

Victor Chance is the former VP of External Operations and Chief Supply Chain Chief Procurement Officer for J&J Medical Devices and Diagnostics. Victor also served as Worldwide Vice President of Operations for J&J’s Cordis franchise and was responsible for directing the company’s global manufacturing, supply chain management and strategic sourcing functions. 

Judy Meritz, J.D., was the Associate General Counsel at Covidien/Medtronic and specializes in compliance counseling concerning FDA and other international regulatory authority matters including inspections, enforcement actions, product approval strategies, marketing and advertising programs, and complaint handling. 

We join the presentation with Victor Chance, who outlines his success roadmap to an effective 483 response.  If you would like to see the recorded presentation, you can watch here.   

If you have topics you would like YourEncore to explore on future episodes of The Pharmcast, please email Pharmcast@yourencore.com with feedback or suggestions.

The Pharmcast is now available for download on iTunes, Google Play, and Stitcher. If you like what you hear, share it with your friends and write a review or give us a rating on your podcast store. 

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No Surprises: Improving Medical Devices Due Diligence (Ep. 9 on The Pharmcast)

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This week on The Pharmcast, we discuss areas often overlooked in due diligence efforts that can lead to expensive or even show-stopper surprises once the acquisition is complete and later development and commercial efforts are underway, with two former Johnson & Johnson medical devices executives, Donna Godward and Dr. Janet Vargo.  Understanding what to look for (or present) during due diligence will reduce risks and lead to a better evaluation for the transaction.

Acquiring and selling innovative medical devices or diagnostics is standard business for small and large device companies – but doing it well is not standard business. 

Novel medical products offer the possibility of true breakthroughs in medical care but also come with the risks of developing, manufacturing, and commercializing a one-of-a-kind product where quality, regulatory, clinical and commercial pathways are yet to be paved. 

While licensing new technology and acquiring new businesses are standard business in the industry, many acquisitions do not live up to the anticipated value proposition.  Improving the due diligence efforts within a company, large or small, can provide better understanding of the value and the ultimate success of the acquisition

 

About Donna Godward:  Donna Godward is a Strategic Advisor to YourEncore and a member of the Medical Devices Practice.  Prior to assuming her current role in 2016, Donna Godward served as Chief Quality Officer, Medical Devices & Diagnostics (MD&D), for Johnson & Johnson.  Donna has extensive experience in due diligence in licensing and acquisitions. 

About Janet Vargo, Ph.D.:  Dr. Vargo is a Strategic Advisor to YourEncore and a member of the Medical Devices Practice.  Prior to joining YourEncore, Dr. Vargo served as Board Member and Head of Clinical and Regulatory Affairs for Mentor, LLC, a Johnson & Johnson Company, and she advised on clinical trial design, device and biologics safety analyses, due diligence efforts, and regulatory strategy for new-to-the-world products for the entire Johnson & Johnson Medical Device and Diagnostic sector. 

If you have topics you would like YourEncore to explore on future episodes of The Pharmcast, please email Pharmcast@yourencore.com with feedback or suggestions.

The Pharmcast is now available for download on iTunes, Google Play, and Stitcher. If you like what you hear, share it with your friends and write a review or give us a rating on your podcast store. 

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Patient Engagement: Putting Rare Disease Patients First (Ep. 10 on The Pharmcast)

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This week on The Pharmcast, we turn our attention to the topic of rare disease, and more specifically: how to engage and educate rare disease patients to advance development. It's estimated that nearly 30 million Americans, or nearly 1 in 10 people,  are living with a rare disease - and almost 95% of rare diseases don't have any FDA-approved treatment. 

One of the unique challenges and perhaps keys to rare disease development is patient engagement. Patient populations are small by definition. Roughly 50% of patients are children, and benefit-risk equations are completely different from those of more traditional diseases. To learn more about engaging rare disease patients, we sit down with Dr. Lorna Speid, to discuss her stories of rare disease drug development, outline the unique challenges of engaging the rare disease population, and her advice to developers.

Dr. Lorna Speid is the author of Clinical Trials: What Patients and Healthy Volunteers Need to  Know, and founder of the nonprofit organization, Putting Rare Disease Patients First. With an extensive background in R&D, Dr. Speid has dedicated her career to working within the rare disease space - particularly in drug development, clinical trials and patient engagement. Dr. Speid earned her Ph.D. in Safety Assessment of Medicines, Pre and Post Marketing, and has served on the NIH Committee for Therapeutics for Rare and Neglected Diseases. 

Dr. Speid began her nonprofit in 2014, after spending a year writing her book, aiming to empower and inform patients and professionals alike. With hard-to-diagnose diseases, Dr. Speid felt especially passionate about helping patients understand their disease and the pharmaceutical industry - informing them on everything from the drug development process, to genomics, to natural history, and clinical trials - through easily accessible information such as webinars and blogs. The nonprofit also uses social media to share information and offer support to patients - which is especially important in a time when inaccurate information can spread like wildfire. 

If you have topics you would like YourEncore to explore on future episodes of The Pharmcast, please email Pharmcast@yourencore.com with feedback or suggestions.

The Pharmcast is now available for download on iTunes, Google Play, and Stitcher. If you like what you hear, share it with your friends and write a review or give us a rating on your podcast store. 

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Medtech Valuation Killers: 10 Regulatory & Commercialization Traps to Avoid (Ep. 11 on The Pharmcast)

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This week on The Pharmcast, we talk with Dr. Janet Vargo and Minnie Baylor-Henry on medtech valuation killers, and the 10 regulatory and commercialization traps to avoid. Dr. Vargo penned an article on the topic for Med Device Online, and joins us today to share the common ways value can be destroyed.

Dr. Vargo is the former Head of Clinical and Regulatory Affairs for Mentor, LLC, a Johnson & Johnson Company, and she advised on clinical trial design, device and biologics safety analyses, due diligence efforts, and regulatory strategy for new-to-the-world products for the entire Johnson & Johnson Medical Device and Diagnostic sector.

Joining her is Minnie Baylor-Henry, former Worldwide Vice-President for Regulatory Affairs for Johnson & Johnson’s (J&J) Medical Devices & Diagnostics sector as well. 

After evaluating dozens of innovative new medical device products as executives at Johnson & Johnson, and now with YourEncore, Dr. Vargo and Minnie Baylor-Henry has seen firsthand the common mistakes that small companies make.  In this session, they will outline the regulatory and commercialization pitfalls to avoid that will improve your speed to market, valuation, and interest from large medical devices companies.  

If you have topics you would like YourEncore to explore on future episodes of The Pharmcast, please email Pharmcast@yourencore.com with feedback or suggestions.

The Pharmcast is now available for download on iTunes, Google Play, and Stitcher. If you like what you hear, share it with your friends and write a review or give us a rating on your podcast store. 

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NDA Mistakes to Avoid: An Interview with Dr. Joseph Lamendola (Ep. 12 on The Pharmcast)

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This week on the Pharmcast, we talk with Dr. Joseph Lamendola on four New Drug Application (NDA) mistakes to avoid.

Dr. Lamendola is the former VP of U.S. Regulatory Affairs for Bristol-Myers Squibb and Schering-Plough, and now serves as the SVP and leader of YourEncore's Biopharma practice.

Joe has over 20 drug approvals to his name, so we ask Joe to share his life lessons on how to work with the FDA to receive NDA approval and ultimately get life-changing therapies to patients.

If you have topics you would like YourEncore to explore on future episodes of The Pharmcast, please email Pharmcast@yourencore.com with feedback or suggestions.

The Pharmcast is now available for download on iTunes, Google Play, and Stitcher. If you like what you hear, share it with your friends and write a review or give us a rating on your podcast store. 

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Executive Briefing: EU Medical Device Regulation | 5 Ways EU MDR Will Impact Your Business [White Paper]

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The European Union has agreed on a new Medical Device Regulation meant to improve patient safety and data  transparency. The regulation is extensive and impactful – it touches many functions within the company and  companies that fail to comply will not be allowed to sell products into the EU. With the legislation expected to  publish in 2017, companies will have three years to bring medical devices into compliance and, under a separate new regulation for IVD’s, five years for these products.

With the EU finally reaching the agreement, the compliance clock is ticking. Although many functions are impacted, regulatory leaders will most likely be responsible for driving corporate compliance.  If you’re just starting the process, download Executive Briefing: EU Medical Device Regulation to learn five ways EU MDR will impact your business. 

Download Executive Briefing: EU MDR | Five Ways EU MDR Will Impact Your Business

About the Author

Patrice Napoda is a former Director of International Regulatory Affairs for Johnson & Johnson’s Ethicon franchise, current YourEncore expert, and frequent speaker on EU MDR readiness.  For the past two years, Patrice has helped Fortune 500 medical device companies assess their compliance risk and prepare for the final EU Medical Device Regulation. 

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