What They Said – 2nd Quarter 2017

During a somewhat slow week given the holiday that coincides with the end of the second quarter, this is a good time to look back at press releases for the quarter and the year so far.

A single press release tells us what is happening today for the agency. The aggregation of press releases tells us about trends with respect to events or emphasis by FDA. To that end, once a quarter I consult my database of FDA press releases to provide a look-back at what has been issued during the quarter and so far this year.

First looking to volume on a year-by-year basis, the overall trend is down. In 2015, the agency issued 161 releases and in 2016 that fell to 122. So far for this year the agency has issued a total of 53, which if you annualized it would project only 106 for the year unless the pace picks up.

Next just the quarterly numbers. Last we checked, during the first quarter of 2017  FDA sent out 23 releases – fewer than it had for the first quarters of either 2015 or 2016. For this second quarter of 2017, the pace has picked up a bit with 29 press releases issued by the agency. That said, the number for this quarter is fewer than were released during the same quarter last year (40), and even with that of 2015 for the same quarter (29). In short, this year FDA has said less than it has in either of the previous two for both the first and second quarters.


Of note this quarter, the number of General Announcements picked up, a reflection of new leadership announcing agency policy and direction with respect to orphan drugs, competition and opioids.

Content-wise the main focus is on the number of approval announcements by FDA. Overall approval announcements this quarter picked up with 16 announced, up over 14 from last quarter but of the 16, 11 were drug approval announcements (same as last quarter), though 3 of them this quarter involved label expansions and not new drugs.  Of the 16 approval announcements this quarter, there 5 were in the device category, an increase of 2 from the first quarter.

Overall, it would appear that approvals are not running high. Press releases about approvals tend to be, but are not exclusively, reflective of the approval of new molecular entities (NME). (Remember, 2015 was a banner year for approvals.) For 2017, while the number of approvals announced during the first quarter was slightly ahead of last year, the announcements fall short of those announced in 2015 during the same time period.

Here is a comparison of the releases during the first six months of the year for 2015-2017:

However, when compare where we are with respect to NME approvals looking at 2017 to 2016 and 2015 and  you will see that in fact FDA has already surpassed all of 2016 and is well ahead of where we were mid-year for 2015. By mid-year there were 23 NME approvals by FDA, compared to 22 for all of 2016. The banner year of 2015, which saw 45 new NME approvals had only 14 by mid-year. While there have been 22 press releases issued this year regarding drug approvals, several have involved label expansions. This year, the NME approvals are occurring but the press releases are not.

We’ll check in again at the end of the third quarter.

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What Do Patients Want?

Thirty years ago, HIV advocates elevated the notion of patient involvement in the development of new medicines to entirely unprecedented levels by demanding input into multiple aspects of clinical development of HIV treatments.  That included not only in the way that drug companies conducted trials, but in the way FDA evaluated them. The result was the development of enhanced pathways for approval and faster development. Recently there has been renewed effort to more broadly bring the patient voice into greater play. In fact, being “patient-centeredness” has become a common goal along the entire healthcare spectrum – from the provision of healthcare services to the comparison of medical treatments to considerations in developing new medicine.

FDA is getting on board. In a March 2017 Federal Register Notice,  FDA announced that the agency was opening a public docket to solicit input into the creation of an Office of Patient Affairs within the agency. This is not like it was three decades ago. There are a good number of highly active patient organizations that are involved in a spectrum of disease and condition-specific advocacy. There are diseases that are common such as cancer, a rare such as Duchenne muscular dystrophy. In other words, today patient involvement means a greater volume and range of involvement and issues if any organization is truly seeking to be more patient-centric. That naturally leads to the question – what do patients want?

RX4Good is an organization uniquely suited to finding out. Formed in 2009 the organization is aimed at helping companies not only achieve a greater understanding of patient need, but how they can respond thoughtfully and strategically to it. This month, RX4Good issued a report that compiled the results of a survey of patient organizations to assess what the priorities should be for FDA in developing an Office of Patient Affairs. The report contains a good deal of specific information, but also lays out the top three priorities identified by patient groups as FDA moves forward.

The most immediate priority for FDA’s new office expressed by 4 out of 5 the organizations surveyed was the desire for patients to have a seat at the table in the clinical development phase, particularly so that clinically meaningful endpoints translate into meaningful patient outcomes and to minimize the outcome that a trial may have a limited value to patients.

The second most relevant goal cited by just over half of the respondents was for the support of expanded access programs. And just behind this was expediting recruitment and the sharing of outcomes data.

The third area of concern was for the generation of greater amounts of communication and information.

In fact, there are currently a number of ways that patient organizations can interact with the FDA process – such as participation on an FDA advisory committee or workshop.  But it is another noteworthy aspect of this report that a clear majority of the advocates surveyed have not participated in FDA workshops or advisory committees.  While there was some awareness of patient-centered efforts on behalf of the agency, only a small percentage felt that they were very familiar with them. That means the agency has its work cut out on the communications and outreach front – the “if you build it they will come” approach won’t be enough.

You can download a full copy of the report at the RX4Good Website to see the results to the specific questions posed by the organization to patient groups. Clearly there are no quick fixes, but as the report points out, there are plenty of opportunities for FDA to capitalize on what it has already started with respect to patient centeredness while the agency takes in more information and suggestions through the docket on what it still needs to do.

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Healthcare Reform – Where Are We? – An Invitation

Over the past three decades, we have gone from Hillarycare to Obamacare to Trumpcare. The mere fact that healthcare is characterized by the names of public figures is testament to the extreme politicization of an important public policy issue.

The length of the effort to reform healthcare in the United States has been long, arduous, politically overcharged and above all, elusive. While a strong healthcare system is challenging, other countries seem to do a good job – or at least not such a bad job. Yet progress has been so painstaking and the discussion so exhausting, it may seem as if we have lost sight of the actual goal. And yet the subject is critically importance important both for society and for individuals who are ill or at risk – or who simply will need insurance because they are no longer able or wanting to work.

In the past weeks, media have carried stories about insurers pulling out of markets leaving many with limited or no options. We have heard that uncertainty around the future of access to subsidies is causing further erosion to the existing infrastructure. At the same time, we hear that prospects for a replacement for existing law are uncertain, both in terms of timing and impact.

On June 21 at the National Press Club in Washington, D.C., I am scheduled to moderate a panel discussion called “Healthcare in the Trump Era, Politics, Policy and People”. The aim is to discuss where we are, where we want to go, and what keeps us from getting there, looking at it through with balanced perspective and to have a genuinely sane discussion of the issues.

Politics – policy and people. It is impossible to talk about healthcare reform without talking about the politics – a favorite topic in Washington, D.C. Has the content of any proposal become completely subservient to the politics? Can we get past it? The policy elements – what are the essential aspects of healthcare reform that need to be addressed in legislation in order to make the effort meaningful? And finally, what is the impact of all of this on-going political debate and policy deliberation on every day lives? Does this only affect the uninsured or does it matter how you get your insurance?  These are the aspects we will be talking about.

To have this discussion, we have a panel comprised especially of people who can talk politics, policy and people in a bi-partisan way:

  • Jim Gerlach, CEO & President, Business-Industry Political Action Committee (BIPAC) and former Republican member of Congress representing the Sixth District of Pennsylvania;
  • Allyson Schwartz – CEO & President of the Better Medicare Alliance (BMA) and former Democratic member of Congress representing the Thirteenth District of Pennsyvlania;
  • Matt Salo – Executive Director of the National Association of Medicaid Directors.

You can join us in person at the National Press Club if you are local, or by Webinar if you are not. We will discuss relevant questions related to healthcare reform and will be reserving time for questions from the audiences, both live and virtual. Please join me. Thanks.

You can RSVP by clicking on the invite above or by emailing emma.cummingskrueger@fleishman.com

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A Memoriam and A Reckoning

Thirty years ago today, the man seated at the left died of complications from AIDS. The man standing on the right is me. This photograph was taken six weeks before he died. We were just 31 years old. Last week, the Food and Drug Administration for the first time approved a cancer therapy for an indication not based on the part of the body being affected, but rather for use with any solid tumor possessing a specific genetic feature.  The treatment is commonly referred to as an immunotherapy. The two events, thirty years apart, are linked.

Three decades is a long time. In the years just after a loss, the pain is very fresh and raw and the overwhelming feeling is one of sadness. Over time, the sadness never goes away, but it shares the space with other emotions. Perspective comes into play, thankfully.

During the cab ride to the hospital that last time, he reached across the seat and said that he wished we could get married. I replied that we would never live to see that day. A few days later he was gone.

Afterwards in 1993, I attended the IXth International AIDS Conference in Berlin, Germany where they keynote speaker stated that in order for the epidemic to change course a treatment would have to be oral, effective and cheap and that nothing like that was on the horizon.  I was present again in 1996 at plenary session for the XI International AIDS Conference in Vancouver when speaking of the potential promise of new treatments, Dr. David Ho uttered the words “eradication of the virus” and you could hear a pin drop in the room.

Joe died at thirty-one years of age. He is young, frozen in time. I have aged thirty more years – also something I did not entirely anticipate.

When the epidemic began, science and medicine knew very little about viruses and very little about the immune system. Many people would die while we would learn about both.

And so there are two points to make.

The first is that there are moments in life where something is seemingly impossible – where you are so caught up in what is happening in the moment that it seems as if it will never end and that things will never change. It does end and things do change though. Good happens if you are patient for it. You will see things you never expected you would live to see.

And the second is that but for the ugly, ugly thing that was the AIDS epidemic, it is highly doubtful we would have drugs that are administered to people that help their own immune systems fight cancer – a therapy held out as hope, but which many did not think would come to pass.

A Memoriam. A Reckoning.

Photograph by Janet Beller

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OPDP Sends First Letter of 2017

To put it mildly, enforcement by the Office of Prescription Drug Promotion (OPDP) has been lagging. Warning and Untitled letters from this office numbered in the triple digits twenty years ago. In the past three years, it did not break a dozen. Last year was poised to be the lightest on record, but in December OPDP suddenly came forth with several letters within a month. In the five months since, there has been an overwhelming lack of activity.

Today there was a blip. OPDP posted an Untitled Letter for a DTC Broadcast television advertisement to market a drug with an indication for weight loss. The letter noted that the drug has several warnings and contraindications noted in the label which also includes a boxed warning. In issuing the letter, the only violation that was the subject of the FDA’s enforcement was with regard to the minimization of risk information, which is by and far the most common violation of any cited by the agency when issuing such letters.

There are two issues with respect to risk that are of note. The list of contraindications spans a number of conditions and situations as well as a number of potential adverse reactions. FDA asserted that while contraindication was included as part of the ad, the full spectrum was not present. While there was a statement that the product was not something to be considered for use by everyone as well as one stating that “other side effects may occur” such umbrella statements were not deemed sufficient by the agency to cover the range of risks and contraindications from the label.

In addition, there was an issue with regard to risk presentation. In a March blog posting we looked at DTC advertising and some of the risks associated with it in light of research FDA has undertaken with regard to how risk is being conveyed. Specifically the agency has been on the hunt for anything that might compromise clarity and therefore comprehension on the part of the viewer. Here, among other things, the agency took issue with important risk information being conveyed visually while competing material is being conveyed orally.

The issuance of a letter involving a DTC advertisement is another indication that OPDP is still more focused on traditional communications vehicles than it is emergent digital platforms.

Two questions unrelated to the contents of the letter may naturally arise in the wake of this letter.

First – does the issuance of a letter after a five-month silence indicate a renewed effort at enforcement following the change in leadership at FDA?  Since these letters take a good deal of time to generate and the change in hands has occurred only recently, that is doubtful. Also the flurry of activity in December did not indicate more letters forthcoming in their wake.

And secondly – why has OPDP had such a downturn in enforcement? The agency has stated that there are other forms of enforcement activity besides the issuance of letters, but provided no clue as to what that might be. Lacking transparency in that regard, the only quantifiable means we are left with is the issuance of letters. Stay tuned.

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