Weekly Roundup 5.29.15

In spite of the fact that summer does not officially begin until the Summer Solstice which will occur on Sunday, June 21, if you are like me and many others the unofficial beginning of summer happens on Memorial Day. And Memorial Day was last weekend and it was early this year. Labor Day on the other hand will be late. That means that it is going to be a much longer summer than we usually get to experience wearing linen, white bucks, Hawaiian shirts. If that is your cup of tea, you are in for a treat.

And speaking of treats, here are a few for you this week.

  • FDA Approves New Treatment for LAM – The agency is continuing its long streak of approvals for rare conditions with the announcement that Rapamune was being approved for lymphangioleiomyomatosis – LAM – which is a rare, progressive disease of the lungs that primarily affects women of child bearing age. With LAM, there is apparently an abnormal growth of cells that invade lung tissues that can cause destruction that affects air flow. According to the agency’s release, the condition is extremely rare, though in looking around on the Internet, estimates seem to vary. Per the LAM Foundation, the cause of the condition is not known, but you can learn more about the condition there, including an insightful breakdown into each element of the long name – lymphangioleiomyomatosis.  Rapamune was originally approved in 1999 to prevent organ suppression for patients (over 13) receiving kidney transplants and was given breakthrough therapy designation, priority review, and orphan drug status.
  • Irritable Bowel Syndrome Patients Have Two New Drugs - Treatment options for patients with IBS have been narrow and the category has been the subject of a great deal of scrutiny by FDA Advisory Committees. This week FDA announced approval of two new treatments from two different companies for those patients who have IBS-D – the type associated with diarrhea. The new treatments are Viberzi (eluxadoline) and Xifaxan (rifaximin) and both are taken orally.
  • NIH Funded Study Likely to Result in HIV Treatment Changes Globally – From the time that long-awaited effective treatments for HIV emerged, there existed the accompanying question of when it is best to introduce therapy to an infected individual. A new study – Strategic Timing of AntiRetroviral Treatment (START) – is the first large-scale randomized clinical trial that establishes that earlier antiretroviral treatment benefits all HIV infected individuals. The study found that if individual who are infected begin treatment earlier after infection rather than later, then they are less likely to develop serious illnesses as a consequence of their infection and less likely to develop AIDS. In addition, as noted by Dr. Anthony Fauci, NIAID Director, in the NIH press release, earlier treatment has the double benefit of not only preventing illness, but in reducing the risk of transmission as well. The study will conclude at the end of 2016, but interim results were released early due to their likely impact.

That is all for me this week folks. Have a wonderful weekend – begin to enjoy the summer. Wear straw hats. Go on picnics. Play in the garden. Be well.

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Advisory Committee Track Records

Needless to say, every time a product goes before an FDA Advisory Committee (AdComm), there are circumstances that make the deliberations that occur during that meeting a unique experience. A specific investigative compound being studied for a specific indication with its own clinical trial track record for safety and efficacy to consider by a large group of people of varied backgrounds to pass on a recommendation for its use, or not, in a therapeutic are that may be serious or not and where there may already be many existing choices or none. And those are just some of the variables.

I have worked many times over the years to help teams prepare for such meetings and the issues that will likely come up. It has involved everything from helping to message out the presentations that teams make in an advisory committee to speaker prep to researching the panel to preparing for various communications issues facing the compound or the sponsor.

Some of the prep involves looking at the environment very narrowly – examining a few issues close up – and some of the prep may be macro – how has this committee dealt with similar issues in the past? Do some committees present unique patterns? Who meets most often? Who approves more of what they see?

So I have put together yet another database – the FDA AdComm database to look at just a few of those questions and more. I have gone back through 2009 and compiled an overview of the meetings of all of the advisory committees related to drugs since then. While it is a work in progress, I thought I would share some of the preliminary topline numbers.

  • How Many AdComms have Occurred? By my count there have been 298 advisory committee meetings since the beginning of 2009;
  • How Many were Joint Meetings? Of those 298 meetings, 69 of the meetings have been joint meetings where a meeting occurs with members of another committee, often the Drug Safety Risk Management Committee – only 22 of those meetings have involved consideration of a product approval and of those 22, only 8 (36 percent) have been recommendations for approval;
  • How Many Meetings Involved Product Approvals? 170 AdComm meetings have been in regard to product approvals and of those, the product was recommended for approval 111 times – or 65 percent of the time;
  • Which Committee Had the Most Meetings? The Committee with the most meetings was the Oncologic Drugs Advisory Committee which met 42 times, only 23 of which were product approval meetings with 56.5 percent of those being recommended for approval;
  • Which Committee Had the Most Products Approval Recommendations? The Committee with the most product approval meetings was the Endocrinologic Drugs Advisory Committee which met a total of 33 times, but 27 of which were product approval meetings and 23 (85 percent) of those products were approval recommendations;
  • What Committee Met the Fewest Number of Times? The Pharmacologic Drugs Advisory Committee had only 4 meetings, 3 of which regarded product approvals and 2 of which were recommended for approval.
  • What Committee Had the Highest Percentage of Approvals? The Dermatologic and Ophthalmologic Drugs Advisory Committee had 5 meetings that involved product considerations and recommended approval for all 5 of them and so got 100 percent.

One final question being compiled – how many times did FDA go against the recommendation of the advisory committee? The answer to that question is still being researched and the data entered.

AdComm prep is very complicated, and I go about it from many angles. But this is one more way – a kind of fun way – to get a big picture and just one more little piece of data to throw on the pile. Besides, I always wanted to do it.

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Weekly Roundup 5.15.15

Today is one of those days where if you took the date and read it backwards – 5.15.15, it would still be the same date and it has pretty much been that way all week. 5.14.15, 5.13.15 and so on. So there’s that. And it was a busy one for me – I could barely hang on. And I didn’t get to my AdComm posting that I had intended, but promise to next week.

And it is Spring. So there.

In the meantime, a bit of what happened this week:

  • House E&C Sets Another Opioid Hearing – The House Energy and Commerce Committee, Subcommittee on Oversight and Investigations has scheduled  another hearing regarding opioid abuse. The subcommittee has already held several meetings and brought numerous people forward under various subject headings, including academia, federal officials and local communities. This Thursday, the category under examination is “What are State Governments Doing to Combat the Opioid Abuse Epidemic”. The materials for the hearing will be found here.
  • AdComm Recommends Approval on Cystic Fibrosis Compound - It is always interesting to watch media coverage around an advisory committee meeting called to consider an application for approval for a new compound. As the FDA documents are posted to the committee docket prior to the meeting, there is a tendency for headlines to express the questions raised by FDA staff as they pick through the clinical history of an investigative compound, resulting in headlines that dwell on the negative side. But then the actual deliberations occur and a different outcome emerges, which was the case this week when the Pulmonary-Allergy Drugs AdComm voted 12-1 to recommend approval for a new cystic fibrosis treatment. The reported PDUFA date is July 5, however that falls on a Sunday, so it would likely be sooner.

That’s it for me this week and my apologies for the late posting. Time is not what it used to be. Hope you all had a good weekend.

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The Guidance that Dare Not Speak Its Name

In December 2014, Commissioner Hamburg announced a proposed change in the FDA’s long-standing policy regarding blood donations from gay men – See “FDA Commissioner Margaret A. Hamburg’s statement on FDA’s Blood Donor Deferral Policy for Men Who Have Sex With Men“. The term “men who have sex with men” was coined as a means to be inclusive of those men who have sex with other men who do not identify themselves as being gay – perhaps with good intentions so that the message reach the target audience.

For many years – since 1985, the policy on the subject of blood donation was draconian, deferring donations from any men who have had sex with men since the year 1977 – even if the sexual episode occurred only once – despite the increasing ability over time to screen and detect the presence of a virus. This was a policy opposed by many groups for many reasons – from the medicine and science and from a human rights point of view. The American Medical Association had stated that it supported scientifically based blood donation deferral and opposed the lifetime ban.

This week, FDA issued a draft guidance awaited since the December 2014 statement by Dr. Hamburg “Revised Recommendations for Reducing the Risk of Human Immunodeficiency Virus Transmission by Blood and Blood Products” which is, let’s face it, about blood donation by gay men, though you might not get that from the title. There was no press release and there was no blog posting on FDA’s Voices blog, but the revised recommendations are a significant departure from the old ones. Under the new recommendation, blood donations will be deferred from a specific list of characteristics that include men who have had sex with men within a period of one year.

The change is well past time. When the AIDS epidemic first emerged, individuals and institutions – both public and private – often took extreme measures in response and these extreme responses were often not based on science, but on unfounded concerns or fears. And too often these extreme responses were formalized into policies – policies that had the opposite impact of what was intended. Rather then act to protect, they harmed. People with HIV were not allowed to travel or immigrate to the United States. Needle exchanges which could save lives were not funded. Correcting these mistakes took decades.

As bad policies go, the lifelong ban on gay blood donation may not be the worst – it was a waste of a potential resource. But it did represent a policy not premised on science but on fear – and further stigmatized gay men. That may seem to some unimportant, but be clear on this – stigma kills. Especially in the dark early days of the epidemic, children thought to have HIV were kicked out of schools, people lost their jobs, patients were denied services. Stigma has a real cost to those who feel the brunt of it.

And in the case of FDA, stigma can backfire. The development of policies based not on fact, but on the weak premise of theoretical conjecture not only wastes valuable resources and contributed to the stigma of a group of people, it erodes the credibility of the policy maker. And for an institution that is charged with protecting the public health – credibility is a commodity that cannot be squandered.

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Weekly Roundup 5.8.15

It is one of my three favorite times of year – the days are so much longer – the evenings cool and pleasant for sleeping near open windows – the mornings crisp and the afternoons languidly warm. Flowers abound. There is a lot of birdsong. It is a good time to be living in the moment and not worrying about the future or the past.

Though speaking of the past, we will cast our eyes backward just a bit to see what the week brought us of interest. And then look onward to the weekend and next week, when I promise an interesting posting about Advisory Committees.

In fact, the folks at FDA must be enjoying Spring too. There were no press releases from this agency, which normally has a lot to say, but which can suddenly from time to time go all quiet. But other than press releases, there were some items of note:

  • FDA Withdraws 47 Draft Guidance Documents – As the entire community of people who waited for 5 years for FDA to come forward with draft guidance documents related to the social media and internet will attest, the agency is not very speedy when it comes to drafting, developing and issuing these important documents. And once the draft guidance documents come out, and the agency collects input through open dockets, a final version may take months, years, and even more than a decade to come into being. This week the agency decided apparently to clean house by announcing the withdrawal of 47 draft guidances that were developed before December 31, 2013 and never finalized. The complete list can be found here, and includes some documents going back to 1991, during the first Bush presidency. It is good to remove the clutter, but even better to improve the process.
  • Senate HELP Committee Questions FDA on Biosimilars – And speaking of guidance, Republicans on the Senate HELP Committee has sent Acting FDA Commissioner Stephen Ostroff a letter to express concern over FDA’s implementation of regulatory pathway for biosimilars. Specifically, they noted that the agency has not provided guidance on a number of fundamental issues, even as biosimilars are coming before AdComms for consideration for approval. The letter stated that the agency has not provided guidance on issues relating to the review and approval of applications for biosimilar products such as naming, interchangeability and production of patent information. They also expressed concern that guidance remains in draft form at this stage and set forth a list of questions that sough answers from the agency by May 22.
  • AdComm Set to Consider NDA for HSDD Treatment – A joint meeting of the Bone, Reproductive and Urologic Drugs Advisory Committee with the Drug Safety and Risk Management Advisory Committee has been set for June 4 to consider an investigational treatment for hypoactive sexual desire disorder (HSDD). This is significant on a number of fronts. First and foremost, while there are many treatments available for male sexual disorder – erectile dysfunction, there are no approved treatments available for women. Secondly, the meeting comes after a 2-day Public Workshop on the subject held by FDA in the Fall of 2014 – the materials from which can be found here. And third, many therapies have been explored, none have been approved. This is one to watch.

That’s all from me this week folks. Have a wonderful weekend. And next week, look forward to some observations on AdComms.

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