Weekly Roundup 5.25.18

It is Memorial Day Weekend!  The opening bell for summer vacations has arrived. It is time to do your wardrobe switch – get out the white shoes, sandals and linen clothing and put the corduroys away until Labor Day. Time to wince about last year’s bathing suit and the fact that it appears to have shrunk while stored away. And sorry to say this was a busy week, leaving no time for an intermittent posting between Weekly Roundups, but will shoot for at least one next week. Meanwhile a bit of what happened in this week as everyone is poised to get away or stay home and usher in the summer with a barbeque:

  • Gottlieb Signals New Facilitation of Gene Therapy –  In a speech given at the annual board meeting of the Alliance for Regenerative Medicine, Dr. Gottlieb stated that we have arrived “at a key point when it comes to cell and gene therapy” – moving from theory to therapy.  He pointed up that the agency has put into place a new policy framework in 2017 putting in place an informed approach for the development of new products through enhanced pathways offered by the agency, in particular providing the means for small institutions efficient means to meet requirements of the approval process. The field is growing rapidly – with three new approvals this past year in gene therapies and more than 500 active investigational new drug applications with the agency now. To facilitate rapid consideration he said that the agency will be issuing guidance documents, among which will be a pathway for accelerated approval endpoints for certain gene therapy products and that the first therapeutic category addressed will be for hemophilia. 
  • Congress Passes “Right to Try” – The House of Representatives passed the “Right to Try” legislation that provides an authorized pathway for patients with a terminal illness to seek direct access to drugs under investigation for safety and efficacy. Need for the legislation was perceived by many to be more political than practical given the already existing process of early access or compassionate use overseen by FDA. The new legislation removed federal oversight and Commissioner Gottlieb in prior Congressional testimony expressed reservations about it. The ultimate decision for expanded access under any pathway is  with the sponsoring pharmaceutical company that is investigating the drug. The bill heads to the White House for signature to become law.

Things to Keep an Eye on This Week

  • Not much going on that caught my eye. Congress is gone, no FDA Advisory Committee meetings. 

Regulatory Developments in Pharma/Biotech/Devices

Photograph by Lynne Bertram

 

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Weekly Roundup 5.18.18

There has been so much rain in the past week, it is difficult for those of us in Washington, D.C. to remember that we live in that Washington and not Washington state. It has been relentless and it continues. It has also been raining FDA -related actions this week, of which many blog posts could be written. But time, like the weather, is constraining so here is a bit of what happened in digest:

  • Migraine Preventive Gets FDA OK – FDA announced approval of a new biologic therapy for the preventive treatment of migraine in adults. During clinical trials Aimovig (erenunab-aooe) was found to reduce the number of days migraines were suffered by patients in clinical trials. Aimovig works as a calcitonin gene-related peptide receptor (CGRP-R) thought to play a role in migraine. An interesting side note in the wake of the national discussion on pricing that took place this week, the release from the company names the list price for the drug, outlines a rationale for the price set and discusses the patient assistance program. The company press release can be found here and the multi-media news release here
  • FDA Approves New Treatment for Opioid Addiction – A priority of FDA and of the Commissioner has been to make new treatments available for opioid withdrawal symptoms. This week Lucemyra (lofexidine hydrochloride) the first non-opioid product was approved for use by FDA for the mitigation of withdrawal symptoms. Only approved for use for a fourteen day period, the release from FDA cautions that it may not prevent withdrawal symptoms but may lessen severity of symptoms and is not a treatment for opioid use disorder, but is part of a broader treatment plan. Lucemyra is an oral, selective alpha 2-adrenergic receptor agonist reducing the release of nopinephrine which is believed to play a role in the symptoms. The treatment was granted priority review and fast track and will be required to engage in 15 postmarketing studies. On a related note, Commissioner Gottlieb posted on the FDA Voice blog an entry regarding actions and upcoming plans to address concerns that a balanced approach to the opioid misuse with respect to the needs of patients who require pain control. 
  • Trump Speech on Pharma Pricing and Aftermath – Technically last week, but continued into this week. The speech was to have been delivered in late April, was delayed, and then delivered on May 11. Despite the anticipation and build up to the speech by Administration officials, the actual speech resulted in a rise in pharma industry stocks. Nevertheless, there were follow up statements from HHS Secretary Azar  as well as Commissioner Gottlieb, the latter of which seemed to set up the subsequent FDA unveiling of a portion of the website devoted to listing names of pharma companies where there have been reported issues related to access for purposes of generic drug development the subject of a posting yesterday). And in addition, the Administration posted the blueprint for action which was less a plan than it was a compilation of topics for discussion called American Patients First

Things to Keep an Eye on This Week

Regulatory Developments in Pharma/Biotech/Devices

Photo by Shahar Ezra on Unsplash

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FDA and The Scarlet Letter

FDA apparently intends to implement today an idea that has been kicking around for a while to begin publishing the names of companies that have had letters of complaint lodged against them asserting that they are taking actions to block the development of generic drugs. There is no doubt that the arc of progress favors greater transparency on many levels in many areas. This may or may not be one of them. 

In the media there has been some question as to whether or not this is “shaming”  pharmaceutical companies who have refused samples to those seeking to develop generic competition. It raises some important questions. Just a few, for example, is publication of the names of these companies an effective means of deterring this delay?  Is it needed? And is publication of a one-sided allegation advisable?

Is it effective?  Time will of course tell. But a lot of information gets published on the FDA website that is unpleasant. If a manufacturer does not meet current good manufacturing processes, they are sent a letter from FDA demanding compliance. The letter and the damaging information are there for all to see.  Does that action deter manufacturers to the point that compliance is enhanced?  One could argue it does, but nevertheless, the letters are still issued on a regular basis. And before FDA’s Office of Prescription Drug Promotion dramatically slowed down its enforcement to a trickle, letters were also published on the FDA site that cited regulatory violations for what the agency considered inappropriate promotion. So the idea of publishing violations on the FDA site is not new. Is it a deterrent? FDA’s OPDP seems to have given up. Meanwhile violations of good manufacturing practices still occur. Would there be more if the letters were not published?  We don’t know.

Is it needed?  FDA has cited a number of complaints regarding manufacturers who have not provided samples. But are the letters aimed at many or a few manufacturers? Do they involve many or a few products?  It isn’t entirely clear. That said, FDA has said there are 150 such letters – reportedly impacting about 50 brands. And still last year FDA approved over 1000 generic drugs – a record year. The year before that – at 813 – was a record year as well. So while there may be some bumps in the road with some manufacturers, the numbers indicate a healthy pipeline of generics and 50 is not a very large number in comparison. So it is logical to ask the question, is this a solution in search of a problem?

Is it advisable?  That invites lengthy discourse. Obviously FDA thinks so. But what is being provided is one-sided story. A manufacturer submits one side of the story. FDA publishes it. But is a manufacturer going to be permitted to submit and publish their rationale for delay?  Are there circumstances that might justify a point of view for delay?  Moreover, does this really address the issue of pricing. Yes, the more generics that exist, the more prices come down. But is this addressing the issue of pharmaceutical pricing on a broad scale, or is it a nibble at the issue from the edge?  Generics, after all, come on to the market when a patent has expired. The most expensive drugs that are on the market often, though not always, tend to be those which have been newly approved. This may address a part of the pricing issue, but actually it would appear to be a relatively small part. 

President Trump this week gave a speech this week on pharmaceutical pricing that followed a campaign alleging that industry was “getting away with murder” that was accompanied by a blueprint entitled “Putting Patients First“. But the blueprint – which implies a plan – while many pages was long on ideas and short on action. After the speech, pharma industry stocks shot up. And for those advocates demanding action, the plan was more like Putting Patience First. As a first step out of the starting gate, publication of these letters is a small and incremental effort and yet as the first, it is going to get a lot of attention. But as to the substance, it is painting the pharmaceutical industry with a broad brush stroke when generic approvals have been at record levels and it certainly does not go to the heart of the matter of pricing. 

Photo by Larry Costales on Unsplash

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Weekly Roundup 5.11.18

Soon school will be out, rush hour will be less hectic, vacations long planned will be taken. Pollen counts are high and thunderstorms threaten to materialize nearly every other day. Washington is moving at high speed though the gravity of politics outweighs that of policy by far. In the meantime, here is some of what happened this week and some to look forward to next week in this Weekly Roundup. 

  • FDA Moves on Stem Cell Clinics  – The agency announced this week that it was seeking permanent injunctions against two stem cell clinics marketing unapproved stem cell products to patients – one in Florida and one in California. FDA found that the clinics were offering treatment for a wide variety of conditions including Parkinson’s disease, AML, COPD, cystic fibrosis among others. FDA is seeking permanent injunctions against the clinics. Stem cell operations offering hope to patients with life-threatening conditions are not a new phenomenon. And the number of practitioners is high – as evidenced in this 2016 survey  published in Cell Stem Cell. While FDA’s action is aimed at only two, it begs the larger question about the operations of many other clinics in the U.S. 
  • Non-surgical Device for GI Bleeds Approved – FDA approved a non-surgical treatment for upper (esophagus, stomach and small intestine) and lower (large intestine and rectum) bleeds of the gastrointestinal tract the agency announced this week. The treatment is an aerosolized spray delivered during an endoscopic procedure and is not intended for use in all kinds of bleeds, including for patients with bleeds derived from large veins developed as a result of conditions such as alcoholic liver disease. The spray is a device that was approved under FDA’s De Novo pathway, a risk-based classification for devices without a predicate device already on the market and which is regarded to present low to moderate risk. The company release can be found here
  • More Action on e-Liquids – Following up on prior action against marketers, FDA this week announced four new warning letters aimed at manufacturers selling “e-liquids” – that which goes into an e-cigarette and contains nicotine – in a way that makes the product resemble products designed for children such as soda, cereal and pancakes. In addition one of the manufacturers was cited for selling such products directly to minors. In the announcement about the action, FDA made the point that apart from presenting a danger to minors, such marketing techniques undermine the legitimate potential role for e-cigarettes to support tobacco cessation by adults. It was unclear from the release if this action is part of a continuing string designed to inhibit such marketing practices or if the letters of the past two weeks are meant to address the entire scope of the problem. The manufacturers have 15 days to respond to the warning letters. 

Things to Keep an Eye on This Week

Regulatory Developments in Pharma/Biotech/Devices

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Pharma, FDA and Web Site Mobilization

In 2009 FDA set up the framework outlining its approach to regulating promotional speech by pharmaceutical companies by posing five questions for discussion during a two-day public meeting the agency held in Washington, D.C.  Rather than issue a single guidance document that outlined responses to those questions, what followed many years later were a few guidance documents specific to digital platforms – one on correcting third party misinformation and one on use of platforms with character space limitations. And in the course of addressing broader topics, there were other guidance documents that encompassed digital as well. An overview of FDA social media actions can be found on a tab set up on the blog site here

With respect to character space limitations, the guidance document provided examples of branded communications where space would be limited – one for ostensibly for a platform like twitter and one for an online advertisement.  There was no mention of creating a web site for mobile viewing on a small screen. 

One of the few areas that provides any insight into the advance-regulatory thinking of FDA’s Office of Prescription Drug Promotion (OPDP) is looking at the research it conducts. In 2017 FDA completed a research project that analyzed promotional content regarding prescription drugs on mobile devices – i.e., tablets and phones, which was published in the Journal of Medical Internet Research (JMIR) in April of 2017. That effort examined 51 mobile communications that were DTC.  The study had a particular emphasis on looking at the presentation of benefit and risks with regard to presence, place and prominence. Of these 51 advertisements, 35 of them linked to branded web sites. Of those sites, the researchers found multiple approaches to presenting risk and benefit information.  

While fully trusting the research I engaged in a bit of my own that differed from the methodology used in the OPDP research and simplifying it greatly with perhaps a related, but different objective. I merely wanted to examine the approaches with respect to the landing pages of branded sites (not the advertisement that leads people there). I took a list of 20 top-selling drugs and visited their branded websites using my phone and my iPad. I looked to see if indication was mentioned on the landing page and how the risk information was positioned.

What I found indeed confirms the conclusions of the OPDP research – the approach is not uniform, though it does cover the bases. Unlike the last time I did a check on top-selling pharma branded sites, this time all of the ones I looked at were mobile sites, readable in the small screen. But the approach in design was often different. Many, for example, did not mention an indication on the landing page – leaving you to either click on prescribing information or scroll to find it. Likewise, risk information is often either click or scroll. And it happens in different combinations. Some with indication on landing and risk on scroll, others with risk on landing and indication on scroll or click. In other words, any lack of stated FDA preference or guidance has meant that different approaches are being taken. It is a little mystifying why OPDP would conduct research to discover what common sense would bear out. 

For many, access to the Internet is actually reliant on online access via smart phones, and in particular, this is true for younger Americans, non-whites and lower income individuals, according to a Pew Research Center Fact Sheet dated February 2018

It is not entirely clear how interested FDA is in this topic. While they have put some effort at research on the issue, a regulatory approach to this does not merit a decade of thought. Perhaps they are just taking the “risk-based approach” and assessing that any risk here is negligible, which would be hard to argue with. But if that is not the case, lacking any standards from the agency, companies will do the best they can. That is likely sufficient from a patient safety point of view, but at some point the agency could decide that a single approach is necessary. When that happens, everyone else will be wrong. 

 

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