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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Weekly Roundup 8.17.18

The vacation continues here in Washington, D.C. Traffic is lighter than a feather. Heat and thunderstorms continue and some of the leaves on trees have had it and are falling early, not in autumnal fashion, but as dried up little tired things – perhaps they were the earliest to appear in Spring and are now calling it a season. Tomatoes are still in their full glory which coincides nicely with bumper crops of basil. In the meantime, there was a little official life going on and here is some of it:

  • FDA Pronouncement on Opioids and Animal Care – Commissioner Gottlieb released a statement this week addressing the agency’s ongoing efforts to curb abuses in opioids but this time in relation to veterinary medicine. FDA produced a new resource to inform vets regarding potential abuse by humans with pets, designed to provide context around possible diversion by humans who are exploiting an animal in order to obtain opioids. The agency is recommending the use of alternatives to opioids where appropriate and advising vets on the development of plans should they encounter a situation where there is suspected diversion. The resource, however, refers vets to local police for information and does not provide a blueprint, guide or considerations for development of such a plan.  
  • EpiPen Generic Approved – As headlines appeared regarding an Epipen shortage, FDA this week approved a generic version of the combination product auto-injector of epinephrine for use in the emergency treatment of allergic reactions. While FDA does not have authority over pricing of the products it regulates, the approval and introduction into the marketplace is one means by which FDA may influence pricing. According to the agency’s press release, this is the first approval of a generic version of the most widely prescribed epinephrine auto-injector in the US.. He added that the agency is committed to the development of generics, particularly of hard-to-copy products and would be advancing new guidance in this space as well as prioritizing review of generic applications. Last year FDA approved a record number of generic drug applications. 
  • FDA, NIH on Modernizing Gene Therapy – In the wake of the approvals of the first three gene therapy treatments by FDA this year, the heads of FDA and NIH announced reforms to the review of gene therapy research designed to maintain safety while streamlining overnight, a regulatory reform theme during the past two years. Drs. Gottlieb and Collins stated that they would seek to reduce duplicative reporting requirements made by their agencies on research and that NIH would be dropping several of its review procedures though research will continue to be subject to FDA clinical trial regulations. To provide more thorough context and history regarding the gene therapy research and regulation, the pair published an article in the New England Journal of Medicine

Things to Keep an Eye on This Week

Regulatory Developments in Pharma/Biotech/Devices

Photo by Annie Spratt on Unsplash

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Weekly Roundup 8.10.18

The summer creaks onward. In less than a month it will be Labor Day. I don’t know about you, but I find that almost incomprehensible. The folks on Capitol Hill are gone, which makes traffic here almost bearable. We like it that way. You can keep them all home if you like. One note related to the posting last week regarding the increase in the number of Commissioner Statements from FDA – this week there were none!  That hasn’t happened since May. In spite of the quiet, some notable things have happened, so let’s saddle up and round up some news.

  • FDA Approves First DTC App for Prevention of Pregnancy – It is perhaps an historic first for Direct-to-Consumer as well as medical app support as well as contraception that FDA approved today a mobile app to support women in contraception to prevent pregnancy. The app works by providing support in the monitoring of conditions that would signal a likelihood of fertility on certain days – a method of contraception referred to in the FDA release as fertility awareness.  Clinical studies to evaluate the efficacy of the app involved over 15,000 women who used the app for an average of eight months. Remember when you used your phone to make phone calls? 
  • Approval in Non-Hodgkin Lymphoma – Non-Hodgkin lymphoma is a blood cancer that begins in the white blood cells. Two types of disease are Mycosis fungicides (MF) and Sezary syndrome (SS), both rare and difficult to treat. FDA announced approval of Poteligeo, a monoclonal antibody, with an indication to treat both of these types of non-Hodgkin lymphoma for patients with relapsed or refractory forms of MF/SS where there has been a prior systemic treatment . Poteligeo was given Priority Review and had Breakthrough Therapy status as well as Orphan Drug designation. The company press release on the approval can be found here
  • New Generic Regulatory Pathway Sees First FDA Approval – FDA has very limited authority to impact the price of medications through its regulation of the pharmaceutical industry. However, the more generics that are on the market for specific conditions, then the more pricing pressure there is on manufacturers and generally the lower the price will go. With some treatment categories experiencing a lack of available generics, a new Competitive Generic Therapy Designation was put into place to expedite the development and review of a generic drug where there is a lack of competition. This week saw the first approval under that designation with the FDA approval of a generic potassium chloride oral solution for the treatment and prevention of hypokalemia (low potassium blood levels) in patients who are on diuretics.  Ultimately pricing impact will depend on the number of drugs that utilize this category as well as the number of conditions affected. Time will tell. 
  • FDA Expands Valsartan Recall – Back in July, FDA issued a press release announcing a recall involving some valsartan products prescribed for the treatment of high blood pressure. Since that time there have been a number of updates to the initial recall. The focus has been the presence of NDMA in some active pharmaceutical ingredient.  The update from this week includes updated lists of products that are under recall as well as a list of products that are not under the recall. Both lists can be accessed in the most recent update. FDA is continuing its investigation and will provide further updates. 

Things to Keep an Eye on This Week

  • No hearings
  • No AdComms
  • No FDA meetings
  • It’s August…..

Regulatory Developments in Pharma/Biotech/Devices


Photo by Jez Timms on Unsplash

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