FDA Credibility on the Line

In normal times, when there is no pandemic threatening our lives and livelihoods, it is essential for FDA to maintain the traditional “gold standard” in relation to the decision-making process for the approval of new treatments. That standard is even more important now, as we approach a time when critical clinical data will be emerging around the various vaccine candidates now being studied. That is especially so since a successful vaccine – and the generous uptake of that vaccine by a skeptical public – is a cornerstone to getting out of this mess.

That is why – pre-pandemic – I wrote this blog posting in December – Remaining Aloof from Politics – citing the action by the FDA press office to issue a press release that took on a political cast, using the name of the president in the headline and sounding like a cheerleader rather than an independent government agency when appearing so might compromise the agency’s all important credibility.

Since then, and almost at the outset of the pandemic, a number of FDA actions have caused further concern for the agency’s credibility.

Now, over the weekend, FDA issued an emergency use authorization for the use of convalescent plasma with the issuance of a ham-handed, politicized headline on the agency’s press release – “FDA Issues Emergency Use Authorization for Convalescent Plasma as Potential Promising COVID-19 Treatment, Another Achievement in Administration’s Fight Against Pandemic“.

The approach in this approval and the communications around it raises concern as we enter a phase in the process whereby critical data from vaccine trials will begin to be collected and preliminary effects assessed. Already there are many within the public who are not part of the anti-vax movement who are skeptical about the vaccine. One recent poll by Gallup showed that one-third of Americans say they will not take the vaccine while less than half indicated they would in an NBC poll. Muddying the waters even further are the tweets insinuating the existence of a “deep state” effort to stymie progress.

It is in our public health interest, as well as our collective economic interest, to have viable vaccines not only developed, but utilized by the public. That means that as much as possible, there must be confidence in the end product, supported by the process of assessment and approval. There must be confidence in FDA that is supported, not inhibited by the Executive Branch. Credibility is nearly as much as a key factor as the science. Even the appearance of political pressure for a specified outcome should be avoided at all costs. We can only hope that when the time comes, the Administration and the FDA both do a better job of it than they have with the issuance of this emergency use authorization.

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What They Said – A Look Back at FDA Press Releases First Half of 2020

Each year, midway through it all, I take a look back to see what FDA has been talking about and compare it to the year before to see how things may have changed. I’m a little late at getting to it this year due to all the COVID-19 distraction. But it is not likely to surprise anyone that the first half of 2020 has caused not only an increase in communication from FDA, but a focus as well.

During the first half of the year, FDA issued a record number of press releases and nearly two-thirds (64 percent) were about COVID-19. The first release related to the pandemic appeared on February 24 to inform us about what steps the agency would be taking with respect to foreign inspections. FDA acted early and relatively often after that. The agency now issues daily updates on the topic.

FDA issued a record number of press statements, two-thirds of which were about COVID-19

Let’s first examine the profile of all the releases, and then focus on those just about COVID-19.

Approvals – There were 54 press statements regarding approvals so far in 2020, comparing to 42 approvals announced in 2019. The big difference, however, is that a big slice of approvals in 2020 involved Emergency Use Authorizations (EUAs) for COVID related products. In fact, there were 16 EUA announcements in 2020, which if you subtract that from the total of 54, leaves you with 38 non-COVID announcements which would lag just a bit behind last year.

The breakdown of those approvals between drugs and devices is also skewed this year due to the fact that the overwhelming number of EUAs about which statements were issued involved devices – usually diagnostic testing associated with COVID. Fully 15 out of the total of 16 EUA announcements involved devices – with only one involving a drug announcement (the EUA for remdesevir). So for the total number of approvals, including EUAs in 2020, the split among the total of 54 approval announcements in2020 were 33 drugs and 21 devices, comparing to 2019 when there were 29 drug approval announcements, 12 devices and 1 gene therapy. If you back out the COVID-related EUAs for 2020, it would be 32 drug approval announcements with only 9 device approvals for 2020, which would have it behind last year at this point.

Alerts – Normally there are not too many alerts in a given year, but 2020 has been an alarming year to say the least. The agency sent out 9 alerts during the first half of the year, 4 of which were related to COVID-19, including one for the off-label use of anti-malaria medications. During 2019, the agency had issued 10 alerts related releases. And so the impact of COVID-19 on alerts did not increase the volume, but were involved in the subject matter of almost half of them.

Guidance/Policy Announcements – There has been more guidance from the agency this year, again due to COVID-19. During 2020, the agency issued 13 releases involving guidance announcements or policy changes, 11 of which were related to COVID-19 compared to the first half of the previous year when there were only 5 Guidance document-related releases. The non-COVID related guidance for 2020 was in relation to e-cigarettes and were issued during the first quarter. Everything subsequent was COVID related, indicating how the pandemic has consumed the focus of the agency.

Legal – These announcements include warnings to companies and court actions such as consent decrees. During the first half of the year, FDA issued releases about 14 legal actions, somewhat less than last year when it issued 19. Of the 14 issued this year, half of them were COVID related in some way, including the announcement regarding the agency’s revocation of the EUA for use of hydroxychloroquine as a treatment for COVID. A sidenote – it does not appear the agency issued a release regarding the issuance of the EUA for hydroxychloroquine. The legal announcements were almost entirely centered on drug related products, with 3 related to tobacco and only 1 for a device.

General Announcements – This is the largest category and the place where “other” goes, i.e. anything not in the above categories. It is the place for agency updates on various topics and providing information that is not specific to an agency action. It includes statements from the commissioner and various division heads. During the first six months of the year, FDA issued 51 general announcements, 30 of which were related to COVID. These covered subjects ranging from calls for blood donations to updates on policy developments to statements regarding the passage of legislation. This compares to 114 that were issued during 2019. While that would seem to be a lot fewer, in fact there has been a real diversion to talking about COVID. In addition to these general announcements, FDA has issued 72 Coronavirus Daily Updates which carry a miscellany of compiled updates on the agency’s activities that are not circulated via a stand-alone press release.

In short, the volume of what is coming out of FDA has increased significantly. This happened when Dr. Gottlieb took over the agency and began handing out Commissioner Statements like crazy. It has happened again because public health circumstances. But there is more to communications than volume, and in an upcoming posting, I’ll take a look at content and assess how well FDA is doing with regard to the content.

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The Fourth Quarter Will Have Little Oxygen – Implications for Healthcare Milestones

Given the state of things in 2020, a lot of us are already thinking about when it will end. Moreover when it does end, it will be good riddance. That said, this difficult year will not go quietly into the night. For those seeking to have a share of voice during the final quarter of the year, they may find it drowned out by circumstances and events.

First of all there is the obvious factor of the election cycle that means that the fourth quarter of the year will be different, and more intense than the previous three. Leading up to the election there is not only going to be the usual focus on political issues and the electoral milestones such as debate between the candidates. It is also likely that we going to witness an unprecedented stream of drama – as we have seen with suggestions of election postponement this week. That alone promises a good deal of media attention – both traditional and social. Additionally, the stakes along so many lines are high and intense. That includes the issues themselves – healthcare and the handling of the pandemic to diversity and social justice. As well, the balance of power is very much more in play this season with a much stronger possibility than there was at the beginning of the year that the Senate could change hands.

Secondly there is the pandemic itself, likely to be a frequent subject in the swirl mentioned above in addition to producing its own news. Transmission in the U.S. is out of control and a de facto policy that appears to favor waiting it all out in the hopes of a coming vaccine – a course that appears costly in human and economic terms. Not only will media be covering the political aspect of COVID already mentioned, but the deliberations that have begun as back to school decisions are made, or not made, among various jurisdictions. And it is during the fourth quarter that we will see the fallout from those actions. And that doesn’t even get into other ongoing debates, such as the existence and enforcement of mask mandates and new efforts to utilize old medicines.

Thirdly, there is the economy. The stunning negative developments are jaw-dropping and recovery is not only uncertain at this juncture, but given the transmission rates seen in most of the country, there could be further retrenchment. While another extensive shutdown may not happen, there will certainly be discussion for the need and that discussion itself may have an impact.

Finally, there is that vaccine thing and the data and regulatory milestones associated with it as well as public discussions about safety and efficacy. Vaccine candidates are already entering Phase III trials and are being conducted in impressive numbers of people. Preliminary results from those trials are going to start coming in during the fourth quarter. Not only will that be happening on the COVID-19 front, but there will also be the possible emergence of new treatments as FDA gets closer to a decision on approving the use of a treatment with convalescent plasma and wider use of such a treatment would be the subject of media scrutiny.

A smart colleague of mine did some analyses of media coverage during the early days of the pandemic. Needless to say, the events of this year have commanded (and demanded) a huge amount of attention. The final quarter of the year stands poised to really suck out almost all of the oxygen in the room, so to speak, with implications in healthcare for data milestones of non-COVID-related treatments as well as corresponding regulatory developments, such as approvals or FDA acceptances of new drug applications and, in turn, presents a challenge for medical meetings occurring during the second half of the year as well as any launches of new medicines.

Anyone seeking to gain some earned media coverage should at the very least be considering a strategy, is possible, that combines a social presence with some paid promotion. It at least will afford a pathway for gaining some of our attention. Holding that attention will be another thing.

It will be good to see 2020 step aside.

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Look Back – Drug Approvals First Half of 2020 Amidst the COVID Challenge

As the year began, no one really thought that within a few months, the systems upon which we rely on for just about everything would be so severely disrupted and tested by the presence of something we cannot even see with the naked eye. But of course, that is what happened. By March we were remaining at home, including people who worked at FDA who assess new drugs for approval. What would the impact be on the approval process?

In response to written questions submitted in March, FDA indicated that the situation was fluid and could not say with certainty whether new approvals might be delayed, and also indicated that the agency was considering virtual advisory committee meetings. In April the agency reinforced the potential for disruption when it issued a statement saying that while it was meeting its PDUFA date obligations, it was uncertain that the agency could sustain its current work performance given the demands of the pandemic.

Now after six months of the COVID-19 pandemic, one might have expected that the approval of new medicines by the Food and Drug Administration might have been negatively impacted as a result. In fact, did everything that was going on – or more to the point not going on – did all that make a dent?

Looking to the approvals of new molecular entities, it appears not. In fact, so far, 2020 is doing rather well. Below is a chart comparing each year since 2015 at the 6-month mark of the hear. Not only is 2020 doing well, it is doing better by mid-year mark than any of the previous 5 years.

Mid-year comparisons are not always telling. For example, in 2018, there were only 20 approvals as of June 30, behind the number of the previous year when there were 23, but by year end new approvals in 2018 set a record at 59, far outpacing the previous year.

Another factor that might have adversely impacted the number of approvals for this year (though to a far lesser degree) are the number of FDA Advisory Committee meetings. Last year by June 30, FDA had held 9 meetings to consider new approvals. This year there were meetings either postponed to a date uncertain or cancelled altogether, while a few have been held virtually. During the first half of 2020, FDA AdComms were scheduled to discuss 8 NDAs, BLAs or sBLAs.

As far as upcoming PDUFA dates, I have fewer on my list for the second half of this year than I had last year at 45 NDAs or BLAs, at least 11 of which are Priority Review. In any case, with the number of approvals during the first half of the year, it suggests that this year will be at least on par with prior years.

While approvals are going along at a good clip during the first half despite COVID-19 challenges, this is occurring in an environment where patients are consuming fewer health services. That includes prescription medicines fueled in part by the fact that physician/patient interactions declined. Either because they have lost insurance and therefore their access, or because they feel unsafe in a medical care setting out of fear of COVID-19, patients are making fewer visits, getting less care, and engaging in diagnostics less. In the end, that leaves this a good news/bad news proposition. In spite of the challenges posed by the shutdown and distancing, the agency is nevertheless still approving NMEs at a healthy pace. But conditions in the greater environment mean that launch and uptake of these newly approved medicines will not be as robust as they might have been under different circumstances.

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FDA to Hold Virtual AdComm

I don’t usually publish the scheduling of an AdComm with a blog posting. But this one merits one.

With fewer than the usual minimum of fifteen days’ notice, FDA has scheduled an FDA Advisory Committee meeting of the Cardiovascular and Renal Drugs Advisory Committee to discuss a new drug application (NDA) for terlipressin, lyophilized power for solution for injection for the proposed indication of the treatment of hepatorenal syndrome. The meeting will be held on July 15 from 8 AM to 5 PM and it will be a virtual meeting.

Normally before an advisory committee meeting, FDA opens a docket for the submission of public comment prior to the meeting at www.regulations.gov – and while they are opening one for this meeting, it will only accept comments until the end of day July 14, 2020, but only those comments submitted before July 10 will be submitted to the committee for review.

The reason for the lack of time between publication of the notice and holding the actual meeting was stated to be unspecified “technical problems”. The agency sates that it will make the background materials available no later than 2 business days prior to the meeting. Hopefully no technical problems there. These should be found on the FDA website at the Cardiovascular and Renal Drugs Advisory Committee meetings page.

The agency states in the Federal Register notice that the meeting will be conducted via an “online teleconferencing platform” but does not say which one. The notice refers people with questions to the standard FDA Advisory Committee webpage about in-person meetings. So you can find out how you get lunch in an in-person meeting, but there is no information about how to access the virtual meeting or how it will work. Bad show, FDA. Presumably this will become apparent in the meeting materials that will be posted prior to the meeting.

We are in the seventh month of this pandemic and it has been clear for at least five of them that virtual advisory committee meetings were likely. FDA needs to deliver a better product here for the sake of credibility in the approval process moving forward.

Photo by Philipp Katzenberger on Unsplash

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