Weekly Roundup 9.28.18

Greetings to you from Cape Cod. A little vacay time. And it was a good time to get out of Washington, D.C. as you may have noticed. Lots happening, challenging to any other news that might emerge during this time.  Still, there may not have been dramatic televised daytime hearings, but some things did happen in our corner of the world and here are a few of them.

  • Gottlieb Opens Flu Season – The flu season started this week and FDA Commissioner Gottlieb issued one of his many statements on the topic – and in fact, I know of some people who began the season early. He noted that the flu virus is a vaccination challenge given that the viruses can not only change from season to season, but during the season and that while during last year’s flu season there were less than optimal experiences with the efficacy of last year’s vaccine in the face of a season that had a high level of severity, it is nevertheless important (he got his flu shot for this year). Last year, for example, the strains identified for the vaccine were correct, but some changes in the makeup of the strains over time caused the vaccine to be less effective than in years past. He believes that there have been updates to the scientific approach taken to address these changes that reduce the chances of a repeat of last year’s issues. He dove into an extraordinary amount of detail regarding how flu vaccines are manufactured, but his bottom line is that vaccination remains the square root of effective flu season preparation. 
  • FDA New Draft Guidance on Adaptive Designs – FDA this week brought out two draft guidance documents designed to facilitate the development of clinical trials that examine multiple drugs for multiple issues and therefore establish principles moving forward more quickly than can be accomplished through a singular and sequential model of study.In FDA’s statement on the guidelines, Dr. Gottlieb stated that improving the approaches to drug development in this manner could speed up the discovery and development of new therapies, not only getting them into the hands of physicians and patients more quickly, but creating an environment with a greater potential for competition of compounds thereby bringing prices – as well as the cost of development – down. The two drafts “Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biosimilars” and “Adaptive Designs for Clinical Trials of Drugs and Biologics” were reported in the Federal Register and more can be seen in that publication here and here regarding the docket that is being opened offering the opportunity for public comment.
  • NSCLC Approval – In the past few years, there have been a number of advances in oncology, both blood-based cancers and solid tumors, reflected in the large number of approvals that have come through. This week FDA approved a new first-line oral, once daily treatment – a kinase inhibitor – for Non-Small Cell Lung Cancer (NSCLC) with three unique biomarker properties. Vizimpro (dacomitinib), per the company press release, the FDA had granted a priority review. 
  • DEA Re-Schedules Epilepsy Treatment Derived from Marijuna Plant– In 2015, legislation was signed into law that set a timeframe for the Drug Enforcement Agency to take action on scheduling drugs that had been approved by FDA. This week DEA issued an order for scheduling a recently FDA approved plant-derived cannabidiol  treatment for epilepsy listing it under Schedule V which is the lowest restriction level, meaning there is a proven medical use and a low abuse potential. The re-scheduling affects only one FDA-approved treatment and per the company release, all other non-FDA approved cannabidiol treatments are in Schedule 1. The company said  in its release that with the completion of the label, it hopes to make the medicine available in six weeks. 

Things to Keep and Eye on This Week

Regulatory Developments in Pharma/Biotech/Devices

Photo by Anna Sullivan on Unsplash

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Weekly Roundup 9.21.18

It has been a tumultuous few weeks. Despite my best intentions, there were no intervening posts from the last Roundup to this and last week’s had to be skipped due to lack of time. But trust me, I’m working on it.  The busy-ness just hasn’t let up and I have been on the run – so this week I will cover more than usual and go back a bit as well. 

  • E-Cigs and Kids – Well the big news this week as well as last involved FDA actions to stem the rising tide of use among teens. It began when last week the agency issued a press release and the Commissioner issued a statement, outlining regulatory steps that were being taken against both retailers and manufacturers. The agency said that it was the largest coordinated enforcement action taken in FDA’s history, missing more than 1,300 warning letters and fines to retailers involved in the illegal sale of e-cigarette products to minors. In addition, there were 12 letters issued to other online retailers who continued to market products that resembled kid-friendly food products. In an interview on the PBS News Hour, Dr. Gottlieb stated that in addition, FDA was taking action vis a vis the flavored products by having manufacturers submit documentation that these products provide a net public health benefit, and further that “all options were on the table. With e-cigarette use among teen skyrocketing, FDA also announced “The Real Cost” Youth E-Cigarette Prevention Campaign to educate youths that vaping is not a risk free activity. 
  • FDA Completes REMS Guidance for Opioids – The final Opioid Analgesic Risk Evaluation and Mitigation Strategy (REMS) was approved this week by FDA, including for the first time immediate-release opioid medicines used in the outpatient setting. The program requires that training be made available to health care providers – including nurses and pharmacists – who are involved in the management of patients with pain, though according to the release, there is no federal requirement that makes the training mandatory. Since 2012, education has been part of the REMS for extended release and long-acting products, but immediate-release opioids account for 90 percent of all opioid pain medications prescribed for outpatient use. The action expands the REMS coverage from 62 products to 347. 
  • FDA to Enhance Review of Low-Risk Medical Devices by Third Parties – The agency has taken many steps to facilitate the faster review of new products across the board, steps that have included enhanced review cycles, for example. And concentrating agency efforts on areas that present the greatest risk has been an on-going effort for some time. Last week FDA announced that it was going to put forth a framework to “better leverage reviews provided by experts in FDA-recognized third party review organizations” that would be equivalent in quality to FDA’s own type of review. The plan is for the agency to expend less time and effort on low-risk devices by accepting outside review for submissions that are eligible. FDA will review the recommendations of third parties and then make a final decision, but “generally will not review the submission itself.” The plan can be found here. The draft guidance can be found here. The news of the plan was released through an FDA in Brief notice

Things to Keep an Eye on This Week

Regulatory Developments in Pharma/Biotech and Devices

Photo by Lee Pigott on Unsplash


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Weekly Roundup 9.7.18

BAM!  Traffic has returned. So have we all from the beach or the mountains or somewhere in between. Back-to and First-Day-of-school pictures have dominated Facebook postings. A promise has been extended by weather folks that the high  temperatures and humidity will both drop. Hurricanes have begun spinning their way across the Atlantic – and in political news. I took last week off for the end of summer and a birthday and so this week will incorporate a few things that happened from last week. And next week there will be postings between the Weekly Roundups as we get back to a more normal routine. So here is a bit of what happened. 

  • OPDP Issues Fourth Letter of the Year – Yes, the rest of us were on vacation, but the eerily quiet Office of Prescription Drug Promotion (OPDP) was not and the agency posted the fourth letter of the year that had been sent out mid-month. The letter was an Untitled Letter and as has become the near-norm, it went to a company that you would not find in the top-50 list of companies in terms of size. The product in question is indicated for treatment of moderate to severe vasomotor symptoms and moderate to severe vulvar and vaginal atrophy due to menopause and was a topical gel that came with a boxed warning on the label, but the agency made no mention of risk in the violation portion of the letter. The communications vehicle in question was a “sell sheet” that contained language that the product represented the treatment with the “lowest effective dose” for the indicated uses when FDA said that this was not the case. You can see the letter and sell sheet here. Unsubstantiated claims are a relatively rare violation.
  • Sweet Spot on Sugar – Finding the best way to design the nutrition label on the food we purchase is the product of a long and negotiated effort. This week FDA Commissioner Scott Gottlieb issued a statement to discuss the treatment of “added sugars” and what that means. He stated that while the old version of the label listed only the total grams of sugar without distinguishing between naturally occurring sugars and those that are added to the product, the new final rule does and includes not only sugar, but honey and maple syrup among others, noting that added sugars can contribute more than 10 percent of a person’s daily calorie intake. The new label will not only note the added sugar, but will include an added daily value for added sugar for the consumer to make assessments. However that may be confusing when the product is a single ingredient product such as syrup or honey. For those type of products then as of now FDA is suggesting the label include extra information to clarify any reference to “added sugar” to be included in a sort of footnote so as not to confuse consumers. FDA anticipates issuing the final guidance by January 2020. 
  • Compounding (Again) – The agency announced that it was issuing a revised draft of the Memorandum of Understanding (MOU) between FDA and the states to address outstanding issues related to the regulation of compounded drugs, taking steps to address concerns that have been raised by pharmacies regarding a 2015 draft. The concerns raised centered on the potential for restrictive distribution policies laid out would impede patient access to compounded drugs for which there is a legitimate medical need. FDA liberalized the provision that was of concern in a way that would allow more compounding pharmacists to distribute compounded drugs. The agency also signaled that in the coming months there would be several more actions in relation to compounding to shore up safety with regard to their development and use and cited as an example the existence of unsanitary conditions in settings were drugs are produced. 

Things to Keep an Eye on This Week

Regulatory Developments in Pharma/Biotech/Devices

Photo by Joe Leahy on Unsplash



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Weekly Roundup 8.24.18

This is it. We are in the home stretch. Summer, at least the one defined by the school year, is almost over.  Even though it is many years for me that I have been ruled by that calendar, it will always be the one most real for me. So even though Summer officially ends much later in September, really, this is it. We get one more week of respite from traffic before the Labor Day weekend signals the return to regular routine. And even though it is a time that is less productive than other parts of the year, here are a few things that happened this week of note. 

  • FDA Announces Effort to Devise a Framework for Opioid Guidelines – The Commissioner issued a statement this week announcing that the agency has awarded a contract to the National Academies of Sciences, Engineering, and Medicine (NASEM) to assess the evidence that would be needed to inform future clinical practice guidelines for opioid analgesic prescribing are sufficient and to identify the research necessary to generate needed evidence. The scope of the work includes the commencement of a consensus study to identify where evidence-based clinical practice guidelines would best inform prescribers as well as a gap analysis of existing guidelines by, among other things, conducting a series of meetings and public workshops to engage contributing stakeholders in the analysis.  
  • First Drug Approval for Rare Disease of the Cornea – Oxervate (cenegermin) won approval from FDA this week for the treatment of neurotrophic keratitis, a condition affecting the cornea in less than five in 10,000 individuals. It is, however, a degenerative disease that has a huge impact on the patient resulting in the loss of corneal sensation and progressive damage that in turn can mean thinning of the cornea, ulceration and perforation. Until now, treatment was palliative and involved surgery. Oxervate, a first-in-class recombinant human nerve growth factors, is a topical eye drop and is the first topical biologic approved in ophthalmology. Oxervate was a Priority Review, with Orphan Drug Designation, Fast Track and Breakthrough Therapy Designation. The company release can be found here
  • FDA Extends Expiration Date on EpiPen – In the face of reported shortages for EpiPen and coincident with back-to-school time, FDA announced this week that it was extending the expiration date of some lots of the product by a period of four months and has put up a web page where consumers can check the numbers of the batches for dates. While it brings some relief to the shortage, along with the recent approval of a generic demonstrates the types of actions FDA can take to impact the status quo with regard to both supply and price. It also leads one to wonder what are the criteria for deciding what dates can be extended and how much wiggle room there is around a stated expiration date. 

Things to Keep an Eye on This Week

Regulatory Developments in Pharma/Biotech/Devices 

Photo by David Tostado on Unsplash

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Mid-Year Check on the Pace of New Drug Approvals at FDA

At the mid-year point, I usually do a check-in to see how we are doing on new drug approvals and how it compares to years gone by. I am quite tardy checking in on that front, so let’s get it done.

To set a little context, last year (2017) with 46 approvals of new molecular entities (NMEs) at FDA, it was a record year. However, it followed a year (2016) that was a little on the slow side with only 22 which, in turn, had followed another record year in (2015) with 45 approvals. So one year up, one year down, one year up.

Everyone is always interested in the number of new drugs being approved at FDA, but this year may be of particular interest because it comes in the wake of a broad policy push to lower the regulatory burden and to improve the pathway to support innovation. That is manifest not only in the passage and implementation of the 21st Century Cures Act, but in the repeated statements by the new Commissioner aiming at “modernizing” the approval process without sacrificing the agency’s gold standard for safety. It will be some time before that can be fully assessed, but in the meantime, many mechanisms have been put into place – such as Breakthrough Therapy designation, to enhance the regulatory process. One of our first insights into the effectiveness of these efforts is with the rate of new approvals for NMEs.

So at the first half of the year, things are coming along and we do not appear to be having the kind of dip we saw in 2016. In fact, FDA is at a slightly lower level of approvals than the previous year, but still going at a good clip.  As of June 30, 2017, FDA stood at 23 approvals of new molecular entities, comprising exactly half of what would be the total for the year – 46. As of June 30, 2018, the number stood at 20, only two less than were approved the entire year of 2016.  

The number so far is high and if we keep the pace, the year should reflect a healthy amount of approvals. Will we do it?  A few “surrogate endpoints” to look at to see how we are doing.

First, are there many advisory committees scheduled to hear drug NDAs and are they for NME? On that front, it does not look so hot right now – the advisory committee schedule page does not list any adcomms for drugs right now. Any that come much later in the year would likely represent PDUFA dates that will fall in 2019.

The other place to look is at known PDUFA dates. I run a little database on PDUFA dates gleaned from public sources such as company press releases. For the balance of the year, I have 42 PDUFA dates listed, but only 24 of them are for NDAs, as opposed to applications that are supplemental. That is not likely the entire universe of dates between now and the end of the year, only the ones that are apparent to me. Therefore the number is likely larger.

One final note given my lag in timing – since the mid-year mark, we have seen a healthy number of approvals. So in the end, we are likely to have a year that is on par, if not exceeding, last year’s number of NME approvals. Whether that is the effect of a healthy pipeline, the enhanced policy efforts in regulating the approval process or a combination of both still remains to be seen. 


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