Tag: FDA

Future of Health Data –the Big Brains convene!

October 22nd, 2019 by
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Katyal, von Eschenbach, Shulkin and Siddiqui

A quiet revolution is underway in healthcare data. A decade after the HITECH Act spurred the rapid adoption of electronic medical records, we are seeing the resulting data being integrated with other sources such as insurance claims, clinical registries and social determinants of health to produce richer data sets for analysis and action.

As we’ve noted in our own consulting work, Datavant has emerged as a key player in connecting disparate data sources at the patient level while protecting privacy. The company’s methodology anonymizes patient identifiers with a unique patient key, matches records across data sets, and enables data exchange.

So I was excited to attend Datavant’s Future of Health Data Summit in Washington, DC earlier this month. As one would expect from a company that strives to organize and present data efficiently, accurately, and clearly, it was a quality event. The day featured 40-minute panels with strong moderators and 3-4 expert panelists along with keynotes from luminaries such as former FDA Commissioner Robert Califf, CDER Director Janet Woodcock, and former Senate Majority Leader Tom Daschle.

The closing panel, Healthcare Policy, Value-Based Care and Data Sharing was typical of the program. Moderator Neal Katyal (former Acting US Solicitor General) made the most of the expertise of panelists David Shulkin (Former VA Secretary), Mona Siddiqui (Chief Data Officer, HHS) and Andrew von Eschenbach (former FDA Commissioner).

They were all passionate about the ability to finally leverage healthcare data at scale. Shulkin talked about using data to prevent suicides of veterans. The highest incidence is in the first year after discharge, when they risk falling between the cracks as they transition from the DoD to VA system. Siddiqui bemoaned the difficulties of confronting the opioid crisis with 2-year old data, and von Eschenbach spoke of the potential to transform the whole healthcare system by improving care and reducing costs.

Shulkin seemed the most skeptical about where things are headed in the near term, predicting incremental change in the healthcare system as the most likely outcome of the fight between Medicare-for-All Democrats and Republicans who want to crush federal involvement. When Siddiqui gushed about the potential to use the planet’s largest healthcare data set (from CMS) in hackathons and challenges and system redesigns, Shulkin interjected that working with the government was, “not for the feint of heart,” and “,not a great strategy for a young company.”

There was an interesting back and forth about how to get the public comfortable with data sharing, how to overcome the decline of public trust in general, and how to address privacy from a policy and technological standpoint.

The panelists generally agreed that patients would be on board if they saw how use of data could help with their own care, and that even spectacular data breaches wouldn’t completely erode patients’ trust in the system. We have become accustomed to such breaches with our financial data, after all.

Shulkin thought people would get really upset if they learned that their data was for sale. I disagree -I think consumers are already coming to terms with their data being sold throughout the rest of the information economy; it’s by no means unique to healthcare.

In closing, von Eschenbach said he was “incredibly optimistic” due to the tremendous energy coming into the process right now. It’s a chaotic environment, he said, but we’ll look back in five years and be grateful for today’s chaos.

Shulkin pointed to the large number of healthcare data companies formed over the past three years, using that as an indicator of the level of optimism in the field. It wasn’t evident to me that he fully shares this optimism.

And Siddiqui presented herself as a realist but also a bit of a visionary and optimist, who is thinking about the healthcare system that we want to have. For her –and many of us– it’s one that’s more technology enabled, virtual care enabled, and homecare centric.

I’m heading to the HLTH conference in Las Vegas next week. I hope it’s as worthwhile as the Datavant event!


By healthcare business consultant David E. Williams, president of Health Business Group.

#CareTalk March 2019: Gottlieb’s out. What’s next for FDA?

March 21st, 2019 by

The latest edition of #CareTalk is out. CareCentrix CEO, John Driscoll and I explore the departure of FDA Director Scott Gottlieb and other pressing topics.

Here’s what we covered:

(0:28) Scott Gottlieb is out at FDA. Are you sad to see him go?

(2:00) Home health spending is projected to grow faster than any other category of healthcare over the coming years. Is that good or bad?

(4:00) Insulin prices are spiking and both Democrats and Republicans are up in arms. What’s happening?

(5:53) Lyft is talking about the social determinants of health. What?!

(8:47) What do you think about FDA approving ketamine nasal spray as a treatment for depression?

(9:10) What did we learn about healthcare from Michael Cohen’s Congressional testimony?

(9:33) Medicare has a new app. Have you tried it?

(9:59) Did you hear about the $48,500 bill for a cat bite?

Subscribe to the #CareTalk Podcast
iTunes: https://apple.co/2DIDTcr
Google Play: https://bit.ly/2RobqMB

Would an FDA radical make any real difference?

December 9th, 2016 by
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Totally radical, dude!

Trump considers naming FDA chief who would radically overhaul the agency, blares the headline from STAT. The candidate, Jim O’Neill is indeed a radical: he wants to eliminate the requirement for drugs to demonstrate efficacy in order to gain approval, favors payments for organ donors, and is part of a group that wants to create sea-based libertarian communities.  He also doesn’t seem to know much about the FDA and its people –he said “one thing that surprised me is that the actual human beings at the Food and Drug Administration like science; they like curing disease and they actually like approving drugs and devices and biologics.”

As scary as the guy sounds, in practice he probably would not have much of an impact. Here’s why:

  • Just because a drug or device is FDA approved does not mean payers will pay for it. Pharma and device companies have to demonstrate that their products work and that they are cost effective before they will be reimbursed
  • Since the biggest payers are Medicare and Medicaid, even if you take FDA out of the business of judging efficacy the government is still heavily involved. (However, some Republicans including the likely HHS Secretary look favorably on eliminating Medicare and Medicaid, which I guess would negate my point)
  • O’Neill still thinks products should be shown to be safe before they gain approval, and that’s arguably more important than efficacy in terms of saving lives. But safety can’t be fully evaluated without considering efficacy. A really effective drug that’s active against a serious illness can be considered “safe” even if there are occasionally serious side effects and even deaths. But we wouldn’t accept the same safety profile as “safe” for more benign conditions
  • O’Neill says FDA is under too much pressure from Congress in its current structure. Whenever there is an unpopular decision the commissioner has to testify before Congress, where he’s raked over the coals. I think the same thing will still happen –the public will rise up, perhaps even more, if FDA backs off its traditional role

Ted Cruz actually had an even more radical approach.

So, rest easy for now.

By healthcare business consultant David E. Williams, president of Health Business Group.

Is Generally Regarded as Safe (GRAS) safe enough?

January 25th, 2016 by
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What did I eat?

Unlike most people exposed to the Frontline investigation of supplements and vitamins, I really didn’t find it shocking that a lot of supplements are dangerous and that the bottles don’t contain what they say they do. I feel sorry for people who take these products and are not helped –and are even harmed– but frankly the customers should know better.

The story got me thinking about a related topic: the safety (or lack thereof) of food additives. An NPR story from last year (Why the FDA has never looked at some of the additives in our food) sums it up well:

Companies have added thousands of ingredients to foods with little to no government oversight. That’s thanks to a loophole in a decades-old law that allows them to deem an additive to be “generally recognized as safe” — or GRAS — without the U.S. Food and Drug Administration’s blessing, or even its knowledge.

The loophole was originally intended to allow manufacturers of common ingredients like vinegar and table salt — when added to processed foods — to bypass the FDA’s lengthy safety-review process. But over time, companies have found that it’s far more efficient to take advantage of the exemption to get their products on shelves quickly. Some of these products contain additives that the FDA has found to pose dangers. And even ingredients the agency has agreed are GRAS are now drawing scrutiny from scientists and consumer groups that dispute their safety.

Basically companies have been declaring their own products as GRAS. Sometimes they let the FDA know and sometimes not. Since the GRAS concept was introduced during the Eisenhower administration the number of additives in food has gone from around 800 to over 10,000. Chances are you eat GRAS substances every day.

The FDA review process for food is cumbersome, which is a deterrent to undertaking it. But some safety testing is becoming radically less expensive and more reliable. One area I know about is cardiac safety, thanks to my role on the iCardiac Technologies board. Until recently, drugs in development were assessed for cardiac safety by a highly manual and expensive process of measuring QT prolongation. Now iCardiac’s Early Precision QT approach can provide definitive results with a much smaller and more affordable study and it is becoming the standard that is endorsed by FDA and international bodies.

So with the new approach, maybe it’s time to reconsider the cost/benefit of cardiac safety testing at least for certain food additives.

FDA’s “threshold of concern” is reached when a drug extends the QT interval by 10 milliseconds (ms). As it turns out, some food ingredients can reach or exceed this level. For example:

  • An American Heart Association study found that energy drinks could boost QT by 10 ms.
  • Licorice (also used as a sweetener in some products) can lead to long QT when “abused”
  • Ginseng can increase QT by 15 ms.

Some of the more responsible companies like Cargill are undertaking safety studies for new products. Cargill has been evaluating a new sweetener (3000x as sweet as fructose!) derived from root bark. After some initial concerns were unearthed, the company conducted a QT study to determine the ingredient’s safety. As it turned out, this ingredient was found to cause a 20+ ms prolongation of the QT interval, which will certainly give Cargill pause before continuing development. Results of the study (Detection of ECG effects of (2R,4R)-monatin, a sweet flavored isomer of a component first identified in the root bark of the Sclerochitin ilicifolius planthave been published in Food and Chemical Toxicology.

With new techniques, you don’t have to be Cargill to be able to afford to test food ingredients for safety. As consumers become more discerning it will become a good business decision to test more thorougly.

Image courtesy of David Castillo Dominici at FreeDigitalPhotos.ne

By healthcare business consultant David E. Williams, president of Health Business Group.

 

How crazy is Ted Cruz's FDA reform proposal?

January 4th, 2016 by

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I don’t think very highly of Senator and Republican Presidential candidate Ted Cruz, but his proposal to loosen the drug approval process is at least worth discussing. If you haven’t heard, Cruz’s RESULT Act proposal is as follows:

  • FDA to grant reciprocal approval of “life-saving” drugs and devices from regulatory agencies in developed countries including EU, Israel, Australia, Canada and Japan. FDA would have only 30 days to review and approve
  • Congress can override FDA decisions with a majority vote

Basically he is trying to make it easier to get drugs and devices on the market. There are a number of problems with this approach, which are pointed out by my most of the respectable analysts. Objections include:

  • FDA may just reject the applications anyway since 30 days is not a long enough time to review and they may object to procedures used elsewhere
  • Insertinbg Congress directly into the process undermines the scientific basis of the decision
  • Trial sponsors may seek the jurisdiction with the lowest standards or fastest review times, imperiling safety for Americans
  • It would hurt US economic development by shifting development resources overseas
  • We would be ceding our sovereignty to foreigners
  • FDA is already pretty fast and responsive and has programs for compassionate use and acceleration of the approval process when warranted

These are all reasonable, and yet I was struck by the fact that almost all the commentators use  the Thalidomide debacle as their one and only example.  See for example STAT, The FDA Group Blog, and Harvard Law Blog, which are the top articles that come up under a Google search for “Ted Cruz FDA proposal.”

Thalidomide was approved in Europe as a sleeping pill and for morning sickness but rejected in the US. It caused serious birth defects, with thousands of people affected in Europe but not the US. Great example but we’re talking 1956. Remember 1956? I don’t. But to give you some perspective it was just two years after food rationing was lifted in the UK and nine years before Medicare was established. Soviet troops rolled into Hungary to crush the uprising there.

I don’t hear people citing 1956 examples about anything else in healthcare.

A more serious discussion could be held on the balance between safety and efficacy in FDA approvals. Cruz’s impulses might be better directed to that debate. Maybe it’s ok for FDA to approve any product that’s safe, and not force sponsors to prove efficacy. Sponsors would still want to demonstrate efficacy, but they’d need to do so for healthcare payers, not the FDA.

That’s not as simple as it sounds either. Here are some of the challenges:

  • The biggest payers are Medicare and Medicaid, so even if you take FDA out of the business of judging efficacy the government is still involved
  • In many situations, safety is relative, not absolute. I might accept a modestly effective cancer drug that kills 1 percent of those who take it, while I wouldn’t accept a cure for toenail fungus with the same death rate. But it’s also important to protect cancer patients from dangerous drugs. A recent NYT article about the death from cancer of the wife of the FDA’s head of oncology included this important and under-appreciated point: “Cancer medicines not only often fail to save patients but can accelerate their deaths and make their last weeks far more painful”
  • Safety and efficacy are individualized. (Cruz seems to recognize this.) Some drugs will be effective for only a small population; similarly safety issues don’t always apply across the board. I would want access to a drug that could cure me even if it could kill someone else, especially if there were a companion diagnostic to sort out who will be helped and hurt

Overall I think the FDA is doing a good job of managing the conflicting pressures it faces. Some parts of FDA (such as the groups with responsibility for HIV and oncology) do a better job than others.

I really would like to see Cruz’s proposal spur a more productive debate and not simply be dismissed out of hand.

Image courtesy of vectorolie at FreeDigitalPhotos.net

By healthcare business consultant David E. Williams, president of Health Business Group.