Weekly Roundup 8.11.17

I’ve decided to bring back the Weekly Roundup. I have missed it and especially now with the prospect of a good deal of change in healthcare in general and FDA in particular, much more is happening than I would ever have time to write about in regular every day blog postings.  The Roundup affords an opportunity to take note of some milestones that are either interesting or should provoke some thought for stakeholders in healthcare. I strayed, but now return – back in the saddle.

And so here is a bit of what happened this week that was noteworthy:

  • New ALS Drug to Market – During the last week of July, award winning playwright, actor and director Sam Shepard died of complications from amyotrohphic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease. This week the first new drug in more than twenty years, Radicava (edaravone) became available for patients following the May 5 approval by FDA. ALS is a degenerative disease of the nerve cells that control voluntary functions of the body and is estimated to affect 12,000-15,000 people in the U.S.  Radicava is an intravenous treatment and according to the company’s multimedia news release can be administered in a number of infusion settings.
  • Expansion of FDA’s Tobacco Education Campaign – The Real Cost campaign was launched by FDA in February  2014 aimed at youth who were smoking or trying to quit smoking and has developed an array of resources to support it. Employing multiple media platforms, including social and traditional, FDA points to follow up research that demonstrates that the first two years of the campaign resulted in a significant decrease in young people taking up smoking, or quitting once they had. This week the agency announced an expansion of the campaign to now include e-cigarettes, citing estimates that the number of middle and high school students who have become users. The agency said that the expanded campaign was part of a comprehensive plan aimed at tobacco announced in late July.
  • FDA to Hold Public Meeting on Risk/Benefit – The agency announced this week a public meeting to be held on September 18, 2017 for the purpose of discussing topics related to structured assessment of benefits and risks in drug regulatory decision-making. Among other things, the meeting will focus on approaches to incorporating patient perspectives as well as methods to structured benefit risk assessment. The format will be comprised of a number of presentations and panels. Registration deadline is September 11 and the agency has set up a docket for comments at regulations.gov. For those considering attending, the meeting is to be held at the FDA’s campus in Silver Spring (which means go early and wear comfortable shoes).

That’s it for me this week. I hope you enjoy the return of the Weekly Roundup and have a good weekend.

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New FDA Directions, Part 2 – Cures Implementation

In Part 1 of FDA New Directions, we looked at the issue of pricing. Today we’re looking at implementation of the 21st Century Cures Act (the Act).

Prior to becoming the FDA Commissioner, Dr. Gottlieb was a strong supporter of the Act, signed into law by President Obama in December 2016. The legislation represents a significant bi-partisan effort and is a milestone in FDA authority and heralded by proponents as reforming the approval process to be more effective and patient-centric, among other things. As a nominee, Dr. Gottlieb expressed support for the Act’s timely implementation and has long been an advocate for some of its key provisions aimed at streamlining approval even before they were embodied in the legislation, perhaps most notably as an advocate for the use of adaptive clinical trials. Now as Commissioner Gottlieb, he is now in charge of the implementation of the Act.

It has been an oft-stated aim of this Administration to alleviate regulatory burdens on companies, especially those associated with bringing new medicines to market. The  Act has a specific legislative aim to use multiple means of liberalizing standards for drug approval consideration and to include and involve the patient experience in assessing the efficacy of new drugs. While the Act enjoyed wide support in Congress, those who did not support it were concerned that safety standards could erode in favor of facilitating approval. However, prior to the approval of his nomination and afterwards, Commissioner Gottlieb has maintained that the Act will result in drug development that is more patient-centered and efficient while at the same time maintaining the gold standard of FDA.

Implementation of the Act – which runs nearly 1,000 pages – will not be overnight, but the mechanics of implementation – and the means for following it – have begun. In a July 7 blog posting Commissioner Gottlieb announced a “detailed work plan” to outline steps to implement the Act and that an intra-agency Cures Steering Committee had been formed to coordinate implementation across FDA. Also in July , FDA published an initial list of deliverables under the Act which is designed to morph into a tracking tool by which progress can be ascertained. This provides a central place to see all of the moving parts involved in implentation, though as a tracking tool it could stand to be made a bit more user-friendly.

Also in his July 7 blog posting, Commissioner Gottlieb outlines activities for implementation under FDA’s various centers – CBER, CDER, and CDRH.

And speaking of CDRH, in a blog posting in June, Commissioner Gottlieb called out the importance of the work FDA is doing in relation to the digital health provisions of the Act and stated that FDA would be providing additional clarification related to what falls outside the scope of FDA regulation and to make clear the agency’s position on products containing multiple software functions – components of which may be under FDA regulation and containing some that may not. Perhaps indicating a more flexible approach to the regulation of digital health, he stated that the agency will be devoting resources to “higher risk priorities” and discussed the creation of a possible third party certification program for low risk products to be marketed without FDA review.

As Dr. Gottlieb, he made clear that he has favored streamlined approaches to regulation – both in terms of less regulation and in focusing resources on areas where risk of harm is higher – as FDA Commissioner Gottlieb it seems equally clear that he meant what he said.

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New FDA Directions, Part 1 – Pricing

With a change in leadership at FDA there naturally a great deal of anticipation with regard to the potential for new direction for the agency and the impact it will have on the agency’s diverse stakeholders.

That is especially true right now. Not only has there been the serial drama of Congressional actions around healthcare reform, the new commissioner has taken over FDA at a time when the agency itself is experiencing vast amounts of change while facing ever increasingly complex new challenges. Prior to the confirmation of the new FDA commissioner, many of those challenges were deliberated by members of Congress during the nomination approval process. These ranged from the implementation of the wide-ranging 21st Century Cures Act to facing down the threats posed by opioid abuse. Now that we have a new commissioner, it is a good time to look back at those issues and how they are actually playing out. In Part 1 of examining the new directions for FDA, we’re going to look at the issue of the agency’s role in addressing issues related to pharmaceutical pricing, actions which have garnered national media attention.

FDA does not have a direct role in directly regulating the price of prescription products. In fact, if the subject of price comes up during an advisory committee deliberation, the chair will usually step in to remind the participants that the subject is not one they need discuss. It did come up during Dr. Gottlieb’s confirmation hearings, however.

In fact, while not directly involved in pricing, there are nevertheless actions that FDA takes that can have an indirect impact on price. One of the more obvious has to do with bringing more generics to market. The more generic versions of a drug that come to market, the greater the downward pressure on brand pricing. Therefore the faster FDA approves generic versions the greater the pressure brings down price.

During his confirmation hearing on April 5, Endpoints reported that in response to a question about FDA’s role in lowering the cost of prescription drugs, Dr. Gottlieb talked about enhancing the entry of generics into the market. He said this would be accomplished by (1) reforming the regulatory pathway especially for complex generics and (2) improving the ANDA process.

That was then, this is now. What has happened since confirmation? On June 21, Commissioner Gottlieb published a blog posting on FDA Voice “FDA Working to Lift Barriers to Generic Competition” in which he announced a Drug Competition Action Plan, the first manifestation of which was a public meeting held July 18 to solicit input on where and how FDA might remove obstacles to generic competition. One of the stated goals was also to make sure that FDA regulations were not being “gamed” to delay generic entry to the market. He also said that FDA would be looking to work with the Federal Trade Commission (FTC) to identify and publicize anti-competitive practices by industry – another point he had raised in his Senate hearing. His opening comments to the meeting are available here.

On the FDA Meetings site, there is no link for the meeting on the “FDA Conferences, Meetings and Workshops” page. However, the Federal Register Notice for the meeting presents 6 questions for which the agency is seeking input on a range of subjects related to the generic pathway. As of this writing, 14 comments have been posted in the docket, including one from former Congressman Waxman which is well worth noting for the breadth and scope of the suggestions put forth.

In addition to these actions, on June 27, the agency issued a press release “FDA Tackles Drug Competition to Improve Patient Access” that announced that it was (1) publishing a list of off-patent, off-exclusivity branded drugs without approved generics and in addition was (2) issuing a new policy to expedite review of generic drugs and were positioned as the first actions by the agency under the Drug Competition Action Plan. Of course, actions on generic drugs can impact price, but that addresses the price of older drugs, not new ones coming onto the market.

FDA does not move on a dime and there is usually a good deal of process (collecting information, studies, e.g.) that is put into place before any real action is taken, but clearly the change in leadership is affording the opportunity to demonstrate momentum towards specific goals and putting pre-nomination approval words into post-approval action. Time will tell whether that momentum continues from process to action to impact. In the meantime, FDA has taken steps and through a fairly aggressive communications strategy has made sure that people knew about them.

In the next installments, we’ll look at some of the other issues talked about before the change in leadership and where there has been action.

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FDA and Patient Involvement

“I am a cancer survivor, I was treated for cancer during my last tour at FDA, so I k now the importance of what American medicine does — and what the FDA does — for every one of us.”

Scott Gottlieb, M.D., April 5, 2017 – Senate Confirmation Hearing

Three decades ago patient involvement in the development and approval process for new medicines was virtually non-existent, except of course for being the study subjects in clinical trials. The AIDS epidemic drove a wedge in the system that changed that. Many HIV/AIDS activists, while not scientists, were extremely familiar and aware of the clinical aspects of the approval process and were highly conversant in science.

This week marks a new milestone in the arc of progress that has been patient involvement since that time. FDA announced in a blog posting on FDA Voice, and in a Federal Register notice, that the inaugural meeting of the Patient Engagement Advisory Committee (PEAC) will be held in October. The first meeting will take place on October 11 and 12 and the agenda is focused on getting input into getting patient input into medical device trials.

The current roster of the committee has 9 members, two of whom are from disease-specific organizations – the American Association of Kidney Patients and the Arthritis Foundation.  Other member slots represent individuals who work in the area of patient engagement. At least one person appears to be experienced in minority outreach, but unlike other FDA Advisory Committees, the CV’s of the members are not yet linked to their names on the current roster listing.

The launch of the committee is an important step, but it is important to note that it is one step in a much longer journey. It is important for FDA to connect the dots so that all the stakeholders – patient advocates, researchers and companies involved in the development of new medicines – understand how the input from patients is going to be integrated into practice. The Regulatory Affairs Professional Society reported in April of this year that Dr. Gottlieb has been an advocate of the use of Patient Reported Outcomes as endpoints in clinical trials – is there a committee role in making that happen and how does it fit into a larger process for achieving that goal?

Also, as FDA is well aware, the inauguration of such a committee is only part of the bigger picture to gain patient involvement. One committee of 9 cannot represent the entire spectrum of input from the vast array of conditions and diseases that need attention. The committee will have to turn its attention to issues related to rare diseases and to extremely important matter of the role of real life patient experience into the assessment of new drugs. The complexity and range of issues means that FDA will have to have a broader system in place beyond the development of the committee as well as broader input into the workings of the committee to extend it beyond the current membership. This committee meets a few times a year and has a two-year remit. The scope of work is bigger than that.

The agency has been making strides, particularly in devices, in getting patient input. Since 2013 FDA has been involved in disease/condition-specific meetings to facilitate Patient-Focused Drug Development. The launch of the committee is an important milestone, but there will need to be others and a perhaps most importantly a holistic approach to patient input needs to be articulated to understand how the efforts at increasing the conversation with patients results in solid outcomes. The dots need to be connected. Discussion about enhanced patient involvement and patient reported outcomes have been under discussion for years – it is time to move to greater implementation. It is good that the committee is established. Now, less talk, more action.

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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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