New FDA Directions – Part 3 – Opioids

In this third, and final installment on new directions for FDA under the watch of a new Commissioner, we turn to the issue of opioids. When Dr. Robert Califf was being considered for confirmation as FDA Commissioner he faced a great deal of concerns from members of Congress about opioids, even facing a hold on his nomination over the issue. Just prior to his confirmation hearing, FDA issued an Opioid Action Plan  that included a range of policy options designed to mitigate some of the risks of abuse. 

However, likewise this year Dr. Scott Gottlieb made clear during this confirmation process – in answer to the same concerns being addressed by members of Congress – that addressing issues associated with the opioid epidemic in the U.S. were for him a top priority. Since then, FDA has added to the body of actions undertaken in 2016 with respect to opioids. Here is a brief overview of where we are.

  • Opening Salvo – Sworn in on May 11, Commissioner Gottlieb put up his first posting on the FDA blog – FDA Voice – outlining his first steps that he was taking to address the opioid crisis. He began laying out some policy parameters, advocating for opioid prescriptions to be given only for the durations of treatment that are needed – in other words not to set up prolonged use with a prescription that goes beyond the likely therapeutic need. As a practical matter, he stated that he was establishing an Opioid Policy Steering Committee that would assemble people within the agency to consider what additional tools or strategies are needed and posed three questions to the committee – (1) Are there circumstances under which FDA should require some form of mandatory education for healthcare professionals; (2) Should FDA take additional steps under risk management authorities such as requiring the dispensing of opioids in line with the clinical need and (3) is FDA using the proper framework for considering risk and abuse in the review process? What was not clear in the posting was which internal experts served on the committee, what the makeup was, procedures for meeting and making recommendations or a timeline. 
  • Product Removal – A few weeks later on June 8, FDA requested the removal from market of Opana ER (oxymorphone hydrochloride) based on the concern that the benefits no longer outweighed the risks and followed FDA’s review of postmarketing data which suggested a shift in the route of abuse for the product from nasal to injection.
  • Public Input on Deterrence – Five days later, Commissioner Gottlieb issued a statement announcing a public meeting to be held on July 10-11 focus a discussion on whether there is a secure understanding of whether abuse deterrent opioid products are having their intended impact – in other words – do we know if abuse deterrence deters? The transcript has been made available and is located here for the first day and here for the second, though not easy to find on the FDA site as the meeting is not posted in FDA’s Recent Meetings page.  
  • Next Steps – In his opening remarks to the meeting, the Commissioner gave a more complete picture of where the agency was heading by (1) looking at ways to address exposure of people to opioids – in other words – addressing the risk associated with their normal use – not opioids that are being diverted – to make sure that appropriate patients get prescribed the appropriate amount of medication for their specific clinical need, with an aim of getting fewer prescriptions written in the first lace; (2) addressing the way we go about assessing the risk and benefit of pain relief products – looking at both individual risks as well as societal risk, and (3) to look at training for prescribers and other healthcare providers involved in pain management and whether existing programs are appropriate. He also stated that FDA intends to update the existing REMS on extended release opioid products as well as on immediate release.

On July 13, the Commissioner reiterated the position that his highest priority as Commissioner is to reduce the scope of the epidemic of opioid addiction. Naturally FDA by itself cannot address the breadth and depth of this epidemic. That said, in terms of new directions, what has so far transpired is really an investigational or information gathering phase. It is almost the inverse of the announcement of an Opioid Action Plan made prior to the Califf confirmation when FDA issued several steps it was taking to address the issue. This approach is more of a process than an event.

Right now, FDA is soliciting input, conducting studies and assessing the status quo. Early indications for manufacturers are clear however, that the environment for approval and marketing is going to change and that abuse deterrence, while a factor, is not a comprehensive solution, but only a part of a greater piece of the landscape. Beyond the information gathering phase we now seem to be in, if there is to be meaningful impact, the next phase regarding opioids will be the enunciation of additional new policies and their execution. 


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Weekly Roundup 9.15.17

One season winds down, another offers hints of a beginning.

I am afraid that both the regular Weekly Roundup as well as regular postings have lately been interrupted by some domestic issues that came up for me as well as a heavy travel schedule.  Time to catch up – lots has happened though, and here are a few things I would bring to your attention while I get back on track:

  • Mobile Medical App Approved for Many Types of Substance Abuse – This week FDA approved the first mobile medical app for use in treating substance abuse disorders. Called Reset, the app is intended for use with outpatient therapy to treat involvement with alcohol, cocaine, marijuana and stimulants, though not for use in opioid dependence. According to the FDA press release, the app contains a patient application and a clinical dashboard, supporting patients in skills intended to support abstinence and increase retention of outpatient therapy programs and will be available as a prescription-only adjunct treatment. The devise was reviewed by the agency through the de novo premarket review pathway which is for low-to-moderate-risk devices that are novel and for which there is no legally marketed predicate device which can claim substantial equivalence. Developed by a company that specializes in prescription digital therapeutics, which it refers to as “eFormulations”.
  • Big Week in Oncology Medicines at FDA – Much to report here. Let’s first look to approvals. This week FDA announced approval of the first biosimilar to treat cancer. Amgen’s Mvasi (bevaczumab-awwb) (reference product is Avastin (bevaczumab)) was approved as a biosimilar, not as an interchangeable product, and is indicated for multiple types of cancer. The second piece of oncology news came from FDA in the form of an accelerated approval announcement regarding Aliqopa (copanlisib) for the treatment of adults with relapsed follicular lymphoma, a slow growing form of non-Hodgkin lymphoma that frequently recurs. Aliqopa was granted priority review and has orphan drug status. While that covers two approvals out of the pipeline this week Sandoz announced this week that FDA also accepted a proposed BLA for a biosimilar for Rituxan® (rituximab) for the treatment of blood cancers as well as rheumatoid arthritis. 
  • FDA’s OPDP Issues Second Letter of the Year! – Unsure as to the date of posting to the FDA site, but a Warning Letter was sent August 24 by FDA’s Office of Prescription Drug Promotion. It is notable because it is only the second letter of the year, and the first Warning Letter. The letter went to the manufacturer of an opioid product and addressed the content of a “professional detail aid” that enumerated the benefits and uses of the pain control medication while failing to mention any of the risks associated with the product use. While Warning Letters generally contain multiple violations, given the concern regarding the opioid abuse epidemic, and the dangers noted in the label which included a boxed warning and limitations on use, this letter was sent in regard only to the lack of risk information and failure to fully state the limitations of use. 
  • FDA AdComm Recos Approval for Shingles Treatment – On September 13, the Vaccines and Related Biological Products Advisory Committee met to consider an application from GlaxoSmithKline for its shingles vaccine candidate Shingrix and unanimously recommended approval. According to media reports, the action date for a decision by FDA on the recommendation is October. 

That’s it for me this week folks. Have a good weekend and more soon. 



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Weekly Roundup – 9.1.17

As hurricane season made itself felt this week, summer seems over in the east. Kids are back to school and this weekend summer whites and linens have their final appearance before next Memorial Day. Meanwhile it was a very busy week for FDA – there was an approval of the first treatment of Chagas disease as well as a new antibacterial drug. All in all it was actually a rather momentous week and we capture a portion of that here.

  • Approval of First Gene Therapy – “We are entering a new frontier in medical innovation…” began a quote from Commissioner Scott Gottlieb in the FDA press release this week announcing the approval of Kymriah (tisagenlecleucel) for the treatment of certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). Indeed the approval does usher in a new era where a treatment is manufactured using patient’s own modified T-Cells to kill cancer cells and where in the clinical trial that was the basis for approval, the overall remission rate within three months of treatment was 83 percent. The treatment carries the potential for severe side effects and carries a boxed warning for potential cytokine release syndrome (CRS) which can carry high fever and flu-like symptoms. FDA also announced the expanded approval of tocilzumab to treat CRS. You can see the company digital and multimedia press release here. 
  • FDA Takes Action on Stem Cell Therapies – From one avenue of promising therapeutic change to another, but with a different direction – FDA announced this week enforcement actions being taken vis a vis the use of unapproved stem cell therapies in the U.S. The agency announced it had issued a warning letter to one manufacturer and a seizure of product associated with another.  In a statement issued by FDA Commissioner Gottlieb, the agency also outlined new policy steps and enforcement vis a vis stem cell and regenerative therapies. In it, Commissioner Gottlieb makes clear that while stem cell and regenerative research holds a great deal of promise, the use of unapproved product stands to potentially harm patients and undermine the entire field of science, stating that the agency must separate “the promise from the unscrupulous hype” and that FDA would be stepping up enforcement while simultaneously working to ensure that responsible research is supported. 
  • Real World Evidence and Devices – Patient centeredness has many facets, and one of them that has been of growing interest has been the use of real world evidence in the approval of treatments for patients. Most of the focus has been on drugs, but this week FDA made available guidance – “Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices“. The guidance describes when such evidence can be used, and the criteria that must be fulfilled in order for there to be confidence int he data as well as providing some examples of actual use that have already led to FDA decisions. 

That’s it for me this Labor Day Weekend. Have a good one everyone. 


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My Moment with Jay Thomas

A departure from the usual FDA fare. The blog is meant to cover FDA issues, but also various aspects of communications. This is a story about media training, sort of.

Jay Thomas died yesterday. He was 69 years old. He had a lot of acting gigs and I always really liked him. But it may be less known that he was also a disc jockey and he had a popular morning show in Los Angeles.

On November 7, 1991, Magic Johnson announced that he had HIV. It was a momentous announcement, ushering in HIV awareness on a broader scale than ever before and to new audiences. In 1985 Rock Hudson had brought HIV into every living room in America, but during the years before that HIV was not really talked about and certainly not understood by anyone outside New York, San Francisco, or Los Angeles. In the years following the Rock Hudson milestone – AIDS was still perceived as a gay, white male disease. Magic Johnson’s announcement brought with it a vast expansion of HIV-related subject matter into straight world, into sports world and into African American world. It was a huge.

Consequently, the day of the announcement, I was busy. I was the Director of Client Services at AIDS Project Los Angeles. In the morning ABC News called me to say someone big was coming out with HIV and asked if I knew who it was. I said I did not. They were dubious. In any case, a few hours later the news broke. In the afternoon, every major news outlet and camera was in our conference room and I held the kind of press conference where microphones are all taped to a podium and where flashbulbs went off like strobe lights in a disco. You learn a lot during those moments, which is why I enjoy doing media training to this day.

What this all has to do with Jay Thomas is that the next morning I was on his radio show. It was a live show in the morning hours in Los Angeles. He and everyone in the studio was very nice. I got there early – 5 AM actually. There was a woman he worked with and we all got acquainted and then we put on our headphones for the top of the show. We were all drinking beverages – mine was definitely coffee. I had done so many interviews the day before, I was losing my voice.

He came over and introduced himself. He was very pleasant – smart, warm, personable. He was quick. He was the kind of person with whom you immediately felt comfortable, as if you had met before, even though you hadn’t.

The show began. Jay did his greeting and then an intro for the show. He reported that the day before Magic Johnson had announced that he had HIV. He had some more to say about that. Then he said that he had Mark Senak from AIDS Project Los Angeles as a guest to talk about the announcement.

He looked at me. I looked at him. The woman took a sip of her coffee. Then he began the interview by asking me -“So Mark – what’s your HIV status.” I looked at him. He looked at me. In a very visible gesture, the woman he worked with came close to spraying  out her coffee onto the console.  I still looked at him. He still looked at me. Drivers all over Los Angeles were waiting to hear if I was HIV positive or negative. He noticed. He was swift – this man. He was quick on his feet.

“Maybe that’s a bad question,” he said – or something to that effect. I recovered somewhat. I wanted to say that it wasn’t a bad question if he was interested in dating me, but I didn’t have the chutzpah for that. So I simply explained that taking an HIV test and sharing the results was a very personal decision, and that I wasn’t making mine then and there. On the air. With a LOT of Los Angeles listening. It actually turned into kind of a teachable moment.

Then we went on and had an interview.

I do a lot of media training and I really enjoy it actually. Most media trainers are former journalists, but I was the guy in the interview seat for many years and in many hot spots. This was the most unusual. I always use this as an example of how a media interview can get unexpectedly personal. And you have to prepare for that.

But today the bottom line is – a nice guy has gone. He had a great career and was very talented. And he gave me a very fine memory and provided me with a teachable moment then, and now. Bye Jay and thanks. You probably did not give me a moment’s thought after that, I’ve remembered you all of my life.

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Weekly Roundup 8.11.17

I’ve decided to bring back the Weekly Roundup. I have missed it and especially now with the prospect of a good deal of change in healthcare in general and FDA in particular, much more is happening than I would ever have time to write about in regular every day blog postings.  The Roundup affords an opportunity to take note of some milestones that are either interesting or should provoke some thought for stakeholders in healthcare. I strayed, but now return – back in the saddle.

And so here is a bit of what happened this week that was noteworthy:

  • New ALS Drug to Market – During the last week of July, award winning playwright, actor and director Sam Shepard died of complications from amyotrohphic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease. This week the first new drug in more than twenty years, Radicava (edaravone) became available for patients following the May 5 approval by FDA. ALS is a degenerative disease of the nerve cells that control voluntary functions of the body and is estimated to affect 12,000-15,000 people in the U.S.  Radicava is an intravenous treatment and according to the company’s multimedia news release can be administered in a number of infusion settings.
  • Expansion of FDA’s Tobacco Education Campaign – The Real Cost campaign was launched by FDA in February  2014 aimed at youth who were smoking or trying to quit smoking and has developed an array of resources to support it. Employing multiple media platforms, including social and traditional, FDA points to follow up research that demonstrates that the first two years of the campaign resulted in a significant decrease in young people taking up smoking, or quitting once they had. This week the agency announced an expansion of the campaign to now include e-cigarettes, citing estimates that the number of middle and high school students who have become users. The agency said that the expanded campaign was part of a comprehensive plan aimed at tobacco announced in late July.
  • FDA to Hold Public Meeting on Risk/Benefit – The agency announced this week a public meeting to be held on September 18, 2017 for the purpose of discussing topics related to structured assessment of benefits and risks in drug regulatory decision-making. Among other things, the meeting will focus on approaches to incorporating patient perspectives as well as methods to structured benefit risk assessment. The format will be comprised of a number of presentations and panels. Registration deadline is September 11 and the agency has set up a docket for comments at For those considering attending, the meeting is to be held at the FDA’s campus in Silver Spring (which means go early and wear comfortable shoes).

That’s it for me this week. I hope you enjoy the return of the Weekly Roundup and have a good weekend.

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