Approvals in the Crazy Year of 2020

Each year in January, it is relevant to look back on the activities of FDA during the course of the previous year and look at how it was the same or different from prior years, and to look at trends in new direction. This past year, needless to say was a doozy on all fronts. Let’s begin by looking at approvals.

One might have surmised that the advent of the COVID-era might have thrown off new approvals and that NMEs approved would go diminish. That, however, was not really the case. During 2020, FDA was able to approve 53 – not surpassing the 2018 all time record of 59, but certainly an admirable second place.

One has to also take into account that the approval mechanism at FDA was particularly busy this year. The agency had a number of Emergency Use Authorization (EUA) applications to consider, many of which were for devices (tests) but some for therapeutics and two for vaccines that included the staging of two advisory committee meetings within a week of one another, resulting in the authorization of two mRNA vaccines – a platform never before commercially available. While not full licensure, the mechanisms for approval did utilize agency resources.

Notably, the 2020 NME approvals included 22 related to oncology, with 2 new GIST treatments, 3 in breast cancer and 2 approvals in prostate cancer. There were 2 new treatments for Ebola as well as an approval for a diagnostic in Alzheimer’s. And there were at least 14 approvals for conditions considered rare.

The number of FDA Advisory Committee meetings held for 2020 to consider new drug or biologic applications numbered 19, down from the previous year when there were 23 which was down from 2018 when there were 28. That may be less a reflection on the impact of COVID than it is the fact that so many investigational compounds come through the system with an enhanced status that may diminish the need for advisory committee input.

It was certainly reasonable on the part of many to expect that FDA’s capacity for keeping the pipeline moving through the regulatory process might have been negatively affected by the COVID-19 pandemic. Certainly the shift to virtual advisory committees must have been a challenge (and trying at times to listen to) as was the shift to remote work for employees, but in the end, the agency and the pipeline delivered the second highest number of NMEs to date, while some PDUFA dates have been missed or postponed.

Looking ahead, the interesting question for the year to come will be whether or not the pandemic will have affected the completion of clinical trials for new investigative compounds that were underway or recruiting during 2020 and what impact, if any, that will have on future numbers of approvals and NMEs.

Happy New Year, by the way.

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Vaccine Development and Approval in a Time of Pandemic

Sometimes in the midst of events, perspective is elusive. Today the Food and Drug Administration will hold a meeting of the Vaccines and Related Biologics Advisory Committee (VRBAC) to consider the application for an Emergency Use Authorization (EUA) submitted by Pfizer/BioNTech for their investigative vaccine for COVID-19. Usually prior to an FDA Advisory Committee meeting where a product is being considered for approval, the posting of the FDA Reviewer comments is greatly anticipated for what it will say about the potential shortcomings of the investigative compound under consideration. The review that was posted regarding the vaccine candidate was extremely positive. Three other countries (United Kingdom, Bahrain and Canada) have already approved the vaccine for emergency use.

We are at a point in time in the United States where this could not come soon enough. We are running out of intensive care beds in hospitals across the nation. Yesterday the U.S. set a world record with over 3000 deaths, nearly as many people dying daily as died in the 9/11 attack. And there were approximately 220,000 new cases reported yesterday and the day before alone. Worse, there is complacency among some about these numbers and their impact on individual human lives. It is as if the lives, and the process for the development of a solution, have been taken for granted. Experience has taught us well that should not be the case.

It is not even a year ago that this virus emerged to threaten pandemic, lives and livelihoods. Within that short amount of time, research identified the virus, routes of transmission and began exploring therapeutics to treat and pathways to prevention. Phase 1 trials of vaccine candidates were designed and executed while plans were underway to engage in Phase 3 trials involving tens of thousands of people around the world. Study subjects had to be administered vaccine or placebo in huge numbers, wait and receive a second dose, and then to wait and see if it worked to prevent illness. All of this has brought us to this day to a VRBAC meeting to consider an EUA for a candidate vaccine for the first of at least two applications, with more to follow.

By contrast, looking back to the AIDS epidemic, the first really effective therapeutic took a decade and a half, an extremely painful and bleak time for people who were infected and their care partners. There has been criticism of the governmental response in COVID-19, but the AIDS epidemic began in 1981 and it was not until 1985 that President Reagan referred publicly to the epidemic. By the end of 1985, there had been a cumulative total of over 44,000 cases diagnosed in the United States with over 24,000 dead. After nearly 40 years, a vaccine still eludes us.

It is not simply the speed of the COVID-19 research response that has been breathtaking, but there has been a dogged determination in overcoming substantial obstacles that were in the way to get us to this point – practical, political, bureaucratic and even financial barriers – that frankly merit our admiration at the accomplishment.

While the speed and process for approval have been a point of concern and criticism for many, there is also the prospect of what weighs against those concerns. As of yesterday over 286,000 people have died from COVID-19. Many others suffer lingering illness. Economies are strained. The global pandemic out of control, but particularly in the U.S. These things are a heavy counterweight. We should not lose sight of it. FDA will likely make its decision in very short order after the conclusion of the meeting.

In the coming days and weeks, as people are considering their personal decisions about whether to vaccinate, other people will be getting this jab and will experience adverse events and reactions. Many of these will be reported on in the media and exploited by some with political agendas as well as vaccine deniers, all of which will be discouraging to those who are fence sitting about whether to take a vaccine. There will be problems in distribution, in uptake and in administration. In some respects, all of these are good problems to have because they come to us solely as a result of the fact that we have viable choices. And there is much for an individual to weigh – from their own health status to that of those with whom they live, among other things.

Speaking for myself, roll up a van to my front door right now. My sleeve is pulled up and my arm is out there and I’m waiting. And as I do so, I’m thanking my lucky stars that this time, it was not a fifteen-year wait.

Photo by Elena Mozhvilo on Unsplash

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COVID-19 Vaccinations – Where a Commitment to Diversity is Essential

In my last posting on preparedness for a post-vaccination world I touched on some of the questions facing employers in general – policy questions that should be considered now for the eventualities of later. But digging a little deeper, there are also questions to consider with respect to support for ensuring access to vaccines by communities of color. All employers have a stake in the issue of enhancing access for minorities to vaccination, but large multi-state employers, those that may be smaller/regional employers with a disproportionate share of minority employees, and of course minority owned businesses all are enhanced stakeholders. Particularly for those businesses that have supported initiatives for diversity and inclusion as a response to the issues of social justice, this is where the rubber meets the road.

Here is the issue. First of all disparities in healthcare have had an overwhelming impact on minority outcomes and causing a disproportionate impact of burden across many disease states. COVID-19 has cast a particularly harsh light on this fact where minorities in the U.S. are dying at younger agents and in greater numbers than non-minorities. Conversely, while vaccine hesitancy has been identified as a factor in the uptake of vaccines, there is a higher reserve of mistrust regarding vaccines on the part of African-Americans. With higher and harsher incidence, and higher mistrust, access to, and education about, COVID-19 vaccines is an imperative – both ethically and from a public health perspective.

However, once a COVID-19 vaccine, or multiple vaccines, are licensed and approved, distribution of the vaccines will be guided by states. The Centers for Disease Control (CDC) directed U.S. health jurisdictions to develop plans for distribution which were submitted and reviewed by the agency. A recent review of these state plans by the Kaiser Family Foundation (KFF) found the following:

  • 53 percent of the states had “at least one mention incorporating racial and/or ethnic minorities or health equity considerations in their targeting of priority populations”, meaning that nearly half do not;
  • In identifying providers for vaccinations, only one-fourth of the state plans discuss making use of providers needed to reach racial minorities;
  • Only half of the plans address communications approaches/needs for outreach to minorities or vulnerable populations;
  • Only one-third of the plans mentions the consideration for addressing vaccine misinformation, but even those that did lacked specific strategies for countering misinformation
  • In addition, states indicated that there were limitations in collecting ethnicity data on vaccinated individuals.

That there are deficits in these plans comes as no surprise given the gravity of the situation and the compressed timeframe for response. So what should businesses be considering? Given the high level of need and the considerable gaps there are some public affairs opportunities that may serve to help the situation.

  • Large employers may wish to review state plans in which they are doing business to assess the deficits that may exist with respect to their employees with particular regard to minority issues related to vaccination. A list of the state plans compiled by KFF can be found here.
  • Identify the gaps in planning and communications that might be addressed through employer-based programs:
    • Development of mechanisms to address vaccine misinformation and enhance education – or alternatively programs that curate third-party information and make it available;
    • Assess potential of public/private partnerships that might address state or regional planning and communications deficits;
  • Perform company-specific research of employee knowledge, attitudes and beliefs around vaccination to inform messaging and program development;
  • Leverage public affairs clout to pressure state health officials to address gaps in planning and communications vis a vis minorities;
  • Consider public statements of support (op-eds, paid advertising) for addressing healthcare disparities in COVID care and vaccination programs and seek and enter into coalitions with other employers.

How well we come out of this will depend on how well we manage the vaccination of enough people to actually make a difference in the course of the pandemic. Anything that falls short for any of us, falls short for all of us.

Photo by Clay Banks on Unsplash

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As the Prospect of a Vaccine Approaches, Business and Communications Challenges Are Just Around the Corner

Coincident to COVID-19 case levels in Europe and the United States entering new and serious levels comes welcome news on the development of a vaccine. Last week Pfizer and BioNTech announced findings from the first interim analysis of the Phase III studies of their candidate vaccine for COVID-19 demonstrating high efficacy levels followed this week by a similar announcement by Moderna. It was a much-craved dose of good news, spawning broad optimism during what is an extremely challenging phase of the pandemic. And while there are many questions left – from efficacy to safety to distribution – given the fact that both of these companies have stated that they will have doses ready in 2020 there appears more of a possibility that there will be more than one vaccine soon – even beginning in December.

But first there will be regulatory rites of passage. During the October 30 meeting of the CDC Advisory Committee on Immunization Practices (ACIP) which focused solely on COVID-19 vaccine development, FDA stated the prior to any approval for an Emergency Use Authorization (EUA) that the agency would reconvene the Vaccines and Related Biologics Advisory Committee tor review data submitted in support of such licensure. It would appear plausible based on the information gleaned from the data announcements that there could be a filing for an EUA very shortly which could in turn mean that limited vaccinations could start being administered very soon. Because the regulatory oversight of COVID became highly politicized, and after some missteps by FDA, some states have indicated that any federal decision will be reviewed, but that does not seem likely to present much a barrier in terms of timeline, if at all.

While vaccination may actually begin in December, the number of doses will be limited. In addition, the process of vaccination takes time. These two particular vaccines are two-dose vaccines administered weeks apart. Vaccination may start next month, but the effects will not occur until after a period following the second dose which would occur in 2021.

Nevertheless, the real prospect of a vaccine roll out raises some very practical and important questions for businesses now. This is true for business to consumer or business to business concerns – whether manufacturers or in the service sector. Vaccination for COVID-19 not only requires business planning, but communications planning in support of the decisions. Vaccination will occur in phases, with more and more people being able to access as increasing doses become available. While the initial vaccines may be utilized with very specific populations, over time there will be increasing numbers of people rolling up their sleeves. A post-vaccine world begs some specific questions:

  • First and foremost – will businesses require a vaccine of employees? And related – will businesses seek to set themselves apart by making it known that their environmental risk is being addressed with mandatory vaccination of staff? This is particularly relevant for retail establishments for example.
  • On the heels of that, will there be circumstances where access to goods and services by consumers is reliant on vaccination? This is particularly relevant for the travel and hospitality industries, but perhaps also for some medical services.
  • Related to both of these first two questions – what sort of documentation will be necessary for an individual – either employee or customer – to present evidence of inoculation? And also how will those who do not vaccinate because of medical or religious reasons be considered?
  • What are the HIPAA and privacy implications, if any, related to all of these questions?
  • What role, if any, will employers play in the facilitation of vaccinations? Currently many employers sponsor vaccines for the flu – will there be a role for COVID vaccination support? Several of the vaccine candidates are administered in two doses, several weeks apart. Will employees need assistance or support in accessing vaccines (and keeping track of which vaccine they have received), particularly if vaccinations are being required?
  • Surveys have indicated that many are reticent about taking a vaccine – will that change when vaccination moves from the theoretical to the real? Will employers have a role in educating people about the safety and efficacy of vaccines, particularly if there are circumstances where vaccinations are being required?
  • Where vaccines are required as a pre-condition to access to either employment or goods and services how will this be balanced by the fact that vaccination of the population will be a process, not an event, and that there may be many people who for one reason or another, are unable to access vaccination?
  • All of these questions may likely have different answers in different regions of the U.S. Companies that are national in scope may have to formulate responses that take into account local or regional conditions. What are the implications of uneven vaccine implementation by region and geography?
  • Finally, among the many unknowns connected with the mass production of a vaccine only developed and researched in the past few months is the long-term efficacy and safety. What is the contingency plan needed around diminishing effectiveness?

It may seem like we are a far way away from having to address these types of questions, given the fact that there is no approved vaccine yet and there are considerable distribution obstacles associated with the administration of COVID-19 vaccines to the general public. The fact that vaccination may begin as soon as December does not mean that answers to these questions are needed now. But the process for answering them is. A post-vaccine environment is going to demand business plans and external and internal communications plans that support and consider multiple scenarios and contingencies. If we have learned one thing in this pandemic, reality overtakes everything else – and the environment shifts rapidly. Public facing companies need to act now to consider strategic challenges and support responses with communications approaches that protect and preserve not only credibility, but the well-being and good will of their stakeholders.

Photo by Simon Berger on Unsplash

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FDA AdComm Agenda Covers the Bases

First of all, we are having a bit of a crisis with our crisis. As we appear to be moving into yet another wave of COVID-19 infections, and as research into a vaccine is aggressively underway, we also see serious drawbacks. Apart from the increasing numbers of cases in the United States, there has been a strong perception that the process has been politicized which in turn has brought on an increasing skepticism or crisis in confidence on the part of the public to embrace the outcome. And now, three agencies that have seen an erosion in public confidence – FDA, CDC and NIH, will participate in an FDA Advisory Committee meeting that will discuss vaccine development.

The agenda has been posted for the October 22 meeting, and the discussion will be available to view on YouTube, according to FDA Commissioner Hahn. One had to wonder what the meeting would actually be discussing since there was no candidate vaccine to consider for approval and given the fact that FDA has already issued guidance to provide a framework of understanding for how the agency will approach approval.

The order of the day includes presentations from all three stakeholder agencies – CDC, FDA and NIH and appears to be designed to add a little more meat to the bones on how each will play a role and to lay out some of the mechanics of the process. in the day they will establish a level playing field for understanding COVID-19, followed by the various key agencies explaining their part in the process, beginning with NIH, followed by BARDA with CDC discussing that agency’s role in monitoring use after either early use authorizations (EUA) or final licensure and CBER will follow up describing post-marketing surveillance systems. Another CDC presentation will discuss vaccine distribution and tracking, which is an important topic about which there are many questions, followed by a discussion on “vaccine confidence” presented by the Reagan-Udall Foundation. There will be a specific discussion of the manufacturing and control as well as the clinical considerations for EUAs and licensure. In short, broadly all the categories in which there are questions seem to represented in the agenda.

Apparently as well, they are expecting a higher degree of public participation in the Open Public Comment period, usually reserved as a 60-minute window, but which is cast for 90 minutes for Thursday. Presumably if that is the case, participation will be limited to a set number of minutes. The number of comments added to the docket that had been set up for the meeting numbered 34 as of yesterday and included two stakeholder companies engaged in clinical trials.

Hopefully what will come out of the discussion is not only the broad understanding of the background and mechanics of approval and distribution, but answers to a good number of the questions regarding the nuances of actual approval, distribution and uptake. Whatever is going to be offered up during the presentation from the Reagan-Udall Foundation about confidence, this meeting will actually be an important factor related to the confidence levels of people, particularly health care workers, in taking a vaccine once there is one to take. This meeting cannot settle for perfunctory presentations and must take the questions that were raised in the docket comments, and those that will be raised during the Open Public Comment period and respond to them in some way subsequent to the meeting. We are in unprecedented times that require unprecedented transparency, planning and sound communications that has so far been largely lacking. Meetings are often perceived as an event – but this one is the beginning of a process.

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