The year began with a prediction from Senator Mitt Romney to pharmaceutical executives that “change is coming”. There have been high profile hearings on the topic in the Congress and legislation that moved in both the Senate and the House of Representatives. There have been initiatives by the Administration in the wake of a blueprint issued but which has mostly gone nowhere. And the FDA has generated record numbers of approvals in generic drugs and issued policy documents to address two means of importation from other countries. All in all, there has been more talk than action.
But what of the talk? What did the conversation reflect from the past year?
Perhaps one of the most striking answers to that question is related to the emergence of the Institute of Clinical and Economic Review – ICER – from perhaps what could be described as the background of the issue of pharmaceutical pricing to the forefront. A non-profit that has been around since 2006 with a goal of providing a model framework for evaluating medicines for their value versus their cost, ICER has been steadily growing its presence in the media around the issue for a while. But this year in particular, the organization is not only being mentioned more in media coverage on the topic of pharmaceutical pricing, it is having an impact in actual costs and in coverage decisions. In short, ICER had not only a quantitative expansion of presence, but a qualitative impact as well.
This did not happen by chance. Part of the reason for the increasing profile of ICER in the media is of course that pharmaceutical pricing was a premium topic last year. But it is more than that. ICER issues a press release at every juncture of its milestone-ridden process and sends out a weekly report that summarizes its activity as well as overviews media mentions for the organization. In short, the organization has carefully cultivated its media profile.
In a white paper published this week, my colleague Adam Silverstein and I examined the public discourse respecting pharmaceutical pricing in 2019. We look at media coverage of the issue – both the drivers of coverage and the stakeholders involved, in particular the ascent of ICER. You can download the paper here.
It is time to look at the forest rather than the trees. It has been a regular custom to occasionally look back and see what FDA has been saying, and how frequently the agency has been saying it and what the focus has been. During the entire year, we look at individual press releases, but let’s look at the entire body of communications. What is the same, and what has changed?
Naturally we start with a look at the total volume. This year FDA issued 272 press releases, down just a bit from the previous year of 289, but well ahead of the yearly output prior to that. It bears noting that 2019 began in the midst of the longest government shutdown due to an impasse on federal appropriations and which lasted until January 25.
Given that the volume has remained about the same this year over last, what about the subject matter? What did FDA talk about this year and were there any changes compared to years gone by?
Alerts – There were 13 press releases regarding alerts to consumers/patients issued this year, compared to 12 the previous year. This year there were fewer for drugs (4) and more for devices (8).
Approvals – Despite the fact that there were fewer new molecular entities approved during 2019 than in 2018, there were more press releases about approvals this year (92) over last (74). This year there were 64 press releases about drug approvals, 25 about devices and 2 in tobacco and 1 in gene therapy. Last year there were 47 drug approvals announced and 27 in the device category.
Legal Actions – This category includes issuing warning letters, seizures, and consent decrees and announcements were on par with last year – 36 in 2019 compared to 34 in 2018. However, the focus was different. This year there were more announcements regarding drug products over last year (17 in 2019, 8 in 2018) and less involving food (only 1 this year compared to 5 last year). There were also more involving devices this year compared to last (5 in 2019, only 1 in 2018).
Recalls – Were about the same – 3 this year compared to 4 last.
Rules and Guidance – There was an uptick in announcements of rules or guidance documents from 6 last year to 10 this year.
Commissioner Statements. All of the above categories are impacted to a degree by the presence of Commissioner Statements issued by FDA. Communications output from FDA changed dramatically once Commissioner Scott Gottlieb was at the helm of the agency when the number of annual press releases in 2018 increased a whopping 74 percent over the previous year and more than doubling the rate of 2016. This was largely due to the advent of statements issued from the Commissioner’s office. In 2018, there was an aggressive communications effort mounted from the Office of the Commissioner which began issuing statements from that office in the form of press releases. These offered a platform for the Commissioner to present a more personal point of view directly to stakeholders. Just prior to his departure, and since then, those types of statements continue to come out from the agency, but come from the heads of various divisions. Think of them like a tweet, but without character limits. Many of these showcased announcements of new rules, guidances and policy, while some were there to present a point of view about the agency’s plans or progress in a specific area of concern. The subject matter of these releases would not be captured in the numbers above. In particular, these communications devices are used to explain in greater detail or to spotlight a new guidance or rule that is going into effect.
What we do see about Commissioner Statements is the biggest difference between last year and this is the volume. While Dr. Gottlieb was still Commissioner in 2018, FDA issued 126 Statements from the Commissioner – a number that dropped to 89 for this year, many of which were statements not from the Office of the Commissioner, but from individual division heads within FDA. This is likely the biggest change of all in FDA’s communications as perceived through the issuance of press releases.
Spanish or No? FDA issues some press releases in both English and Spanish. It is not entirely clear, at least to this observer, when that happens and when it doesn’t. It has been difficult to discern a pattern, but last year there were 39 releases in Spanish as well as English, or 13 percent, which compares 32 releases this year which is about 11 percent.
FDA in Brief. One other thing of note. In 2017 FDA began issuing notices called “FDA in Brief” which are mini-missives to announce recent agency activities, but not up to a press release. One would think media and FDA beat reporters likely follow these. It is a mechanism FDA is using with greater frequency. In 2017, there were about 7 issued each month; in 2018 it went up to an average of 8 issued each month and in 2019 it was an average of 9.5.
The output from FDA in 2020 will be determined by the presence of a new commissioner, the lack of a government shutdown, and perhaps by the election cycle. We’ll keep an eye out.
Happy New Year! It is 2020 – the year of clear vision. Let’s look back for a bit as we plunge forward.
FDA is a giant agency where a lot happens. Narrowing down the actions of significance is therefore a very subjective experience. Even limiting the subject matter to those actions which affect medicine, as opposed to food, devices and cosmetics, there is still a very wide bandwidth of activity and impact. And everyone gets to have their point of view. Having been involved in writing this blog since 2006, here is mine. Some of these noted items are things FDA has done – great milestones and achievements – some involve things the agency has not done – and others may involve situations that are evolving around the agency.
Fewer NMEs, But Interesting Approvals – At 48, there were fewer New Molecular Entities (NME’s) approved in 2019 than in the previous year when there were a whopping 59, but it was still more than in 2017, 2016 and 2015, thanks to a spate of approvals in December. That said, there were a number of interesting developments within the greater span of approvals, including some NMEs. These included the approval of two new treatments for Sickle Cell Disease, the approval of the first Gene Therapy (and incidentally the most expensive drug to be on the market); the approval of the third cancer treatment based on the presence of a genetic characteristic rather than tumor type; the approval of two new anti-depressants in a category that has long been without advance; and in vaccines, the approval of the first preventive for dengue as well as one for Ebola and the first test to detect Zika virus antibodies.
Three Commissioners in One Year – FDA started the year with Dr. Scott Gottlieb at the helm who surprised everyone with a departure in the Spring. In turn, he was replaced by the solid choice of Dr. Ned Sharpless who worked as Acting Commissioner for most of the year. Another surprise, despite the support of many past FDA commissioners, Dr. Sharpless was not appointed FDA Commissioner. Rather Dr. Stephen Hahn was sworn in as the 24th Commissioner of FDA. How the advent of a new commissioner will impact policy is anyone’s guess. During the confirmation process there was some question as to the commitment he has on the FDA’s efforts vis a vis the vaping industry, but as we learned during the Gottlieb confirmation process, concerns raised before don’t always materialize as deficits after confirmation. Even with the advent of a new commissioner, expect the media shadow of Dr. Gottlieb to extend to future FDA activities and policy announcements.
FDA Moves on Importation – Apart from generics, this is the other area where the agency can contribute to the effort to bring down the price of prescription drugs. It was an historic first in many respects – the first time the agency facilitated the pathway for the importation of drugs by issuing a draft guidance document and a rule. The former sets up a regulatory pathway for states and other entities to import drugs from Canada and the latter devises a means by which manufacturers can import drugs from outside the U.S. in order to lower the price in the U.S. though it remains a mystery as to why any manufacturer would likely do such a thing. The other historic thing about the announcement from FDA was that it was the second time the agency politicized the policy announcement by linking it to the Administration – the first being in September. For thoughts on that see the previous posting.
CBD Enforcement – CBD is everywhere. Everywhere geographically, everywhere in the span of products – teas, salves, dummies, hand cream, vape pens – you name it, CBD is likely there leading many stakeholders to label it the “Wild West” to signify that it is without oversight. In the wake of the Farm Bill which made legal the growth of hemp, FDA issued a press release in an attempt to preserve the agency’s regulatory authority over its use as an ingredient in supplements, drugs or food. That was despite the fact that the market was already running amok with products containing CBD. After his departure from FDA, Dr. Gottlieb penned an op-ed in The Washington Post outlining what FDA needed to do as the use of CBD was “getting out of hand.” During the course of the year, FDA issued a spate of warning letters to multiple manufacturers of a range of products including food producers warning them against the use of CBD. For an overview of FDA activity on this front, see “CBD and FDA – Where Are We?” Since that posting, FDA has issued additional letters. Can FDA send enough such letters to actually chill the CBD-related product market? Certainly the agency is under a measure of scrutiny given the opioid and vaping epidemics that are also high profile.
Record Number of Generic Approvals – The agency approved a record number of new generic drugs – one of the few contributions that FDA can make to impact the price of pharmaceuticals. The agency approved 1171 new drugs during the fiscal year. This follows on the heels of two notable years of approvals in 2017 and 2018 Notable announcements included the approval of a generic version of Advair Diskus, valsartan, naloxone nasal spray to treat opioid overdose, Gilenya – an oral treatment for multiple sclerosis, and the approval of a generic Eliquis in the atrial fibrillation treatment space. In addition, FDA issued multiple press releases during the course of the year providing updates on its progress to enhance the approval of generic drugs. With the backlog clearing out, it will remain to be seen if the pace continues in the new year.
And for 2020? Look for a likely uptick in attention to gene therapies as experience with those approved becomes more apparent and those in the pipeline get closer to the market. Watch what happens on the e-cigarette front given the concerns on the topic raised at the time of the new commissioner’s confirmation and monitor how FDA responds to the most recent criticisms of how the agency has handled the opioid epidemic. Probably best not to look for any further guidance on social media and communications by pharma as most of the research directed by OPDP is looking to traditional communications vehicles. As patients and the public have moved into social and digital for media consumption, OPDP remains with its head stuck in the sands of broadcast DTC.
Yesterday FDA issued a press release regarding the much-anticipated actions related to the importation of prescription drugs. It is the aim that a proposed rule and a draft guidance will provide two means of impacting the price of medicines through different avenues of importation. The outcome is not certain and there is a long process ahead in relation to achieving any progress through these means. In other words, the pudding which will be the proof is far from being served up and there is plenty to discuss related to the merits and eventual impact of these actions, whatever headlines may say.
That said, the unusual thing about the agency’s press release was the headline – “Trump Administration takes historic steps to lower U.S. prescription drug prices” – noteworthy on a few counts, but mostly so because it mentions the name of the administration.
I went to my database of FDA press releases and did a search on the names of past presidents. Not one came up. This would appear to the be first time that the agency has issued a press release in this manner. If so, it is an unfortunate precedent.
Is there any real harm? Not in an earth shattering way. But in a highly polarized political environment such as we are in (yesterday there was a vote of impeachment), for a federal agency to engage in any communication that appears to be politically motivated may impact perceptions of impartiality. There are some agencies where there might be particular need to stay away from any political scent in its communications. FDA is one of them.
Several years ago when Plan B was applying for over-the-counter status, the stakes were highly political. Conservatives generally opposed the application while progressives generally lauded it. As the regulatory agency in charge, FDA had to ensure that any decision it was making was not based on politics but on data and science. That is one of the gold standards that must be maintained and preserved to reinforce public confidence in the actions that they agency takes.
It is not the rule, but the exception for there to be political aspects of a regulatory deliberation by FDA either related to an approval or a policy. But it does happen from time to time. For that reason, the agency should strive at all times to maintain the appearance of being separated from the fray and potential bias that can be associated with being political. For that reason if no other, the press release issued by FDA was a mistake – one the agency should take pains not to repeat.
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The system of FDA Advisory Committees is in place so that when FDA needs advice – either on policy development, reviewing an issue with an already approved medicine, or regarding new drug approvals – the agency has a lot of expertise to consult. There are 31 advisory committees on hand organized by subject matter jurisdiction and expertise and 18 of them are under the category of Human Drugs. The Committees are comprised of a range of experts – from clinicians to practitioners to experts in clinical trial design and statistics. There is room for a patient and a representative from industry. They are summoned by the agency when considering a new drug or supplemental new drug application and generally meet about two months before the date set by the Prescription Drug User Fee Act (PDUFA) – also popularly known as the “PDUFA date”. They are not employed for use in every single approval decision, but in circumstances where the agency wants their input, usually the discretion of the division leader over the particular therapeutic category.
The agency has been asking for less advice than usual from the Human Drugs Advisory Committees – scheduling just 23 meetings this year, 19 of which were related to the approval of a new medicine . Granted, the year is not over, but given the organizational needs of holding such a meeting, FDA usually schedules them a few months in advance. There are unlikely to be any new ones scheduled for 2019, particularly given the holiday season will soon be upon us.
One possible reason for the need to consult with experts less frequently could be the emergence and uptake of Breakthrough Therapy status. This designation involves a more frequent and high level of consultation between the agency and the sponsor of a new drug application. Begun in 2012, the number of drugs that have been approved having had Breakthrough Therapy status has increased from 3 drugs in 2013 to 38 applications in 2018. The enhanced interaction between the drug sponsor and the agency may address many of the issues for which the agency would have otherwise sought consultation with experts.
Of course, another possibility is that there were fewer drugs to review. The number of NMEs approved so far this year is certainly less than it was last year. In 2016, there were only 22 NMEs approved, and 22 advisory committee meetings held that examined new drug applications. However in 2018 there were many more NMEs approved, but not many more advisory committee meetings considering NDAs for approval. This year, the number of NMEs has fallen, and so have the AdComms.
Whatever the cause, the notion that there are fewer AdComms may make lead some to feel there is less transparency to the process of approval. While they are long, and sometimes quite arduous to sit through, they are a setting in which one gets to see deliberation in action with respect to the approval process. You may not agree with everything, but you get to see it and hear it. Moreover, there is an opportunity for public input. There is a patient representative on each panel, as well as a non-voting industry representative. And perhaps most importantly in that regard, there is the Open Public Comment period during which patients, investigators and other interested stakeholders can share their outlook, experience and opinion and a docket is opened to receive comments. And it all happens before the media, who report on it in articles and on social media. Finally, if you miss it, there is both a transcript and a webcast. They are a complete learning experience for the observer, from providing insights into how FDA reviews safety and efficacy – how it develops concerns – what constitutes strength in clinical review and what spells weakness. We’ll have to see how next year shapes up.
And speaking of advisory committees, according to FDA’s current roster listings, there are currently vacancies on some of them.
Arthritis Drugs Advisory Committee – 2 vacancies
Bone, Reproductive and Urologic Drugs Advisory Committee – 2 vacancies
My name is Mark Senak. I’m a lawyer and I work at the international communications firm FleishmanHillard in New York. For the past several years, I have been consulting with pharmaceutical and biologic companies that are engaged in the process of bringing new drugs to market.
I am an authority on regulatory aspects of communications and medical products, with particular emphasis on pre-approval communications; strategist to help pharma and biotech companies prepare best case for advisory committee approval; and counselor in issues and crisis management. I am a frequent speaker on various aspects of same - drug development, promotion, reimbursement and new media in a highly regulated environment. Author of books, newspaper and magazine pieces related to drug marketing and promotion as well as HIV specialty pieces. And of course... blogger!
About This Blog
Eye on FDA is published by Mark Senak of FleishmanHillard's Washington, D.C. office. The thoughts and ideas in this blog and postings are strictly my own and are not screened by my employer. Everything posted on this blog is my personal opinion and does not necessarily represent the views of FleishmanHillard or its clients.