Once and Future AdComms Affecting Drug Applications

One area of FDA activity that I like to track is in relation to advisory committees which I think provide us with something of a surrogate marker for the pace at which FDA is considering and approving new drugs. To that end, I have a little database that tracks activity going back through 2009 – looking only at those advisory committees that are listed under the “Drugs” tab on the FDA website.

Let’s begin by looking back to last year, then we’ll look forward to this next month.

During the first quarter of 2015, FDA held 8 advisory committee meetings, 6 of which involved new drug or supplemental new drug applications. Of those 6 meetings, the advisory committees recommended approval in 5 of them. Of those 5, FDA went on to approve 4.

By contrast, we are off to a bit of a slower start in the first quarter of 2016. There have been a total of 4 AdComms this quarter, all of which involved new drug considerations. Of those, the Advisory Committees recommended approval in all four of them, including one which was a biosimilar to reference product Remicade and in one that occurred yesterday. With regard to approvals, FDA final decisions as of this writing and the PDUFA date for one of the products has been extended by the agency. As you know, FDA is not bound by the determination of an advisory committee.

As for upcoming meetings, in April there are three meetings scheduled to consider new drugs:

There will also be a meeting of a few device panels in April as well as the Pediatric Advisory Committee – All meetings are listed here.

At the conclusion of this quarter in a few days, we’ll review FDA press releases compared to last year at this time, including approval announcements.

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Weekly Roundup 3.25.16

Well the first quarter of 2016 has sure blown by. We had the earliest Equinox since the 19th Century. Someone flipped a switch and everything started blooming at once. The cherry blossoms got well underway this week.

And here is what was blooming this week involving FDA – there were a cluster of new approvals, coincidentally – all monoclonal antibodies:

  • Anthrax Treatment Approved – First off, on Monday the agency announced the Friday approval (unusual timing) of a new treatment for inhalation anthrax. The treatment, Anthim, is a monoclonal antibody that neutralizes toxins from the anthrax and was approved under the “Animal Rule” which allows the consideration of testing on animals in lieu of humans where it is not feasible to do them in humans. The company press release can be found here.
  • New Plaque Psoriasis Drug Approved - Taltz, a monoclonal antibody, was approved this week by FDA for the treatment of moderate to severe psoriasis, FDA announced. Taltz acts to counter the effects of psoriasis, an autoimmune disorder, by binding to the protein that causes inflammation in psoriasis and thereby inhibits an inflammatory response. For a more in-depth discussion about the clinical trials leading to the approval, see the company press release here.
  • New Asthma Option Approved – People suffering from severe asthma despite treatment now have a new option with the approval announcement from FDA this week of Cinqair. The treatment is administered by a health care professional once every four weeks and works by reducing the amount of blood eosinophils, a white blood cell that contributes to the development of asthma. The company press release can be found here.

That’s all for me this week – I’m taking a little Spring Break. Have a good weekend everyone.

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Patient Engagement Mainstreams

One Tuesday morning early in October in 1988, a large group of protestors appeared in front of the building that housed the Food and Drug Administration and effectively shut the agency down. There were hundreds of protesters present, organized by ACT-UP (the AIDS Coalition to Unleash Power) and scores were arrested by glove wearing policemen who told many FDA staffers coming to work to turn around and go home. Said one protester, “[w]e are here for all the people with AIDS but we are also here for all the other people with life-threatening illnesses who need drugs now.” The demands of the protesters were numerous, but chief among them was a greater say in the way clinical trials were conducted and the speed with which drugs were approved.

As a result, the approach to drug approvals changed and mechanisms formally introduced into the process that significantly cut down on the time that it took to get candidate therapies reviewed by the agency. AIDS activism – demanding a greater say – changed the system. The mold of patient advocacy changed shape.

Over the years, those pathways have been refined – Fast Track, Accelerated Approval and the like. But the bottom line is that the involvement of patients into the process changed the status quo and most would argue, for the better.

Patient engagement can mean many things. It can mean that when in a doctor’s office, a patient is engaged with his or her physician to ask important questions. Or it can mean that patients are in a position to have some input into the development of treatments for their conditions. This is about the latter.

In the fall of 2015, FDA formally embarked on the development of a new advisory committee – the Patient Engagement Advisory Committee - by announcing its formation in September and soliciting nominations for membership. According to the Federal Register notice, the agency said that the committee would provide input for FDA related to the development of guidance and policy documents, clinical trial registries, patient preference study design and other aspects related to medical devices. Last week FDA published notice that the committee would be formally added to the roster of Advisory Committees.

That is what is happening with devices. With respect to the development of medicines, FDA has been involved in a five year initiative to get patient input related to drug development though a series of public meetings and a list of the topics and meetings can be found here.

Times have changed from 1988. But a generation following the protest of that Tuesday in October, it would appear that there are more ways and more opportunities for patients to have direct input into the development of treatments. And though the list of demands from that day have yet to be fully addressed, formalizing pathways for greater patient involvement is a start. And it’s not just for patients with life-threatening illnesses, it is for all of us.

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Weekly Roundup 3.18.16

St. Patrick’s Day is behind us, Easter is before us. And yet, there is a forecast of snow for our area here in the mid-Atlantic looming before us for the weekend. Yes, it is that delightful time of the year when on a recent warm day edging into the mid-seventies you might be inspired to plant your herbs even though something tells you it is too early, and then Mother Nature reminds you that in fact, it was.

Still, we persevere and here are a few things that happened this week I wanted to convey:

  • FDA Starts New Twitter Feed – This week the agency added yet another twitter feed to its assembly of twittering pronouncements called FDA Patient Network – a/k/a @FDA_Patient_Network – making of a total of 18 FDA Twitter feeds. As of this writing, the feed had only two tweets, but the description on the home page for the feed states that they will cover a “range of FDA-specific topics and conducts numerous activities that are of interest to patients” – which seems pretty broad, but hey the more, the merrier. And if you are interested, the @eyeonfda twitter feed has a list to which you can subscribe where you can see all FDA tweets (and feeds) and what FDA is saying.
  • FDA Provides Alteration to Approving GenericsRecently you may have noticed many headlines reporting where the price of a single available generic product were raised astronomically to much public criticism. This week, FDA responded by issuing a new policy that will speed up approvals for generic drugs and prioritize approvals where there is only one product available. This could dramatically speed up the number of competitive generics available thereby making such pricing actions less practical.
  • Expanded Use of Biologic for Use in Lung Cancer - FDA announced expanded approval of Xalkori (crizotinib) for use non-small cell lung cancer for treating tumors that have an ROS-1 gene alteration. As noted in the agency’s press release one of the things that makes lung cancer so difficult to treat is the existence of different mutations. This is the first treatment approved by the agency for treating this particular mutation. The expanded use application had FDA breakthrough status, priority review as well as being designated orphan status. You can review the meaning of these status designations here. You can see the company press release here.

That’s it for me this week folks. Have a great weekend.

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Testimonials – The Pitfalls

People like to get information from people they trust. Moreover, a hallmark of digital communications has been a turning away from large, institutionalized sources for information and turning instead to hear from individuals.

Pew Research has shown that especially for people with chronic conditions, there is a reliance on getting information and support from friends and family and one quarter say that they seek information from people who have the same health condition that they do. In other words, there appears to be good reason for the use of testimonials by patients when seeking to promote a medical product. A patient like me who has faced what I faced and now has been through the experience tells me what it is like, and I may take great stock in what they say.

But while it may make sense, there may be inherent regulatory risks in going down that path – risks that are peculiar to use of testimonials. Why?

Part of the appeal in having a patient describe their experience using a medical product is that they provide a real, first-person description of how the product worked for them and the difference it made for them with respect to their condition. That is also the drawback.

Communications about the use of a medicine are supposed to reflect the label that FDA approved for the product. Going outside of that parameter risks an FDA regulatory action like a warning or untitled letter from the Office of Prescription Drug Promotion (OPDP).

I went through my database of OPDP Warning and Untitled Letters which includes letters from 2004 through today and tracks multiple characteristics of the letters, including descriptions of the communications vehicle (brochure, video, e.g.) and of course, the violations cited. The data base profiles more than 300 letters and over 1000 violations. While not a separate field, when a testimonial has been involved it is noted in the file.

I was able to identify 12 instances in which FDA sent a letter regarding a communication that involved a patient testimonial – in 3 of those instances the patient was also a celebrity spokesperson.

It is noteworthy that the most common violation cited in OPDP letters generally is the minimization or omission of risk information – by far. However, when it comes specifically to patient testimonials, the most common violation was the overstatement of efficacy, involving 11 of the 12 letters.

The basis for that is likely that when a person describes in subjective terms their experience with a medication, it usually includes a reference to the impact the use had on their lives. Phrases such as it “literally changed my life” and “restored my confidence” or describing in personal terms the use of the medication for which evidence does not exist to demonstrate that everyone would have such an experience.

Almost all of the communications vehicles involved video, but it also involved some print.

It also bears reminding that OPDP usually looks not only to an individual statement or action (though that can suffice) but also the entirety of the presentation.

This is not to say that testimonials are bad or should not be attempted, but it does say that one should take care to examine the presentation carefully, with particular sensitivity to the issue of overstatement of efficacy and to take note of historical examples where FDA has acted.

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