Wednesday, January 25, 2017

Gulfo and Soon-Shiong Continue to Circulate as Trump Allies

Additional news stories this week on a fourth candidate for head of FDA, Dr. James Gulfo.

Prior potential nominees for FDA have included Republican speechwriter and investor Jim O'Neill, young biotech founder Balaji Srinivasan PhD, and well-respected physician-policy expert Dr. Scott Gottlieb.

In addition, in a speech on January 31, President Trump focused attention not only on pharma prices, but on (A) speeding approval "instead of waiting for many many years" and (B) access for the terminally ill, usually viewed as "right to try" laws (here).  Trump also stated that "We have a fantastic person that I think I'll be naming fairly soon who's gonna streamline the FDA and you're gonna get your products either approved or not approved, but it's gonna be a quick process."  See here.

Dr. Joseph Gulfo

The week of January 23, new attention has focused on Dr. Joseph Gulfo.  For a full listing of his credentials, see his impressive Linked-In page, here.

Gulfo trained in oncology, worked in CROs and biotech startups, and had his longest career segment as CEO of Mela Sciences, which developed the Melafind device for augmented accuracy in selecting atypical moles for biopsy.  Melafind had a difficult multi-year course at the FDA, which Gulfo detailed in his book, Innovation Breakdown (2014).  At Amazon, here.  Book website, here.  A 2011 6-page white paper at the Progressive Policy Institute (here) gives a summary of the FDA's delays and policy issues on Gulfo's product Melafind.

Gulfo, an MD/MBA, has also written a general book on leadership and management (here).

Gulfo is a strong proponent of approvals based on intermediate endpoints, including biomarkers.  More about Gulfo and his book, after the break.

New News on Dr. Soon-Shiong

On January 24, the subscription health news website STAT ran an article that Dr. Patrick Soon-Shiong, who's been shortlisted by NIH watchers as a  potential head of NIH, could also appear in the Trump administration in a novel role.  The role could be closer to health innovation czar, rather than an existing title like Surgeon General, Head of CMS, Head of NIH, etc.  The subscription article is here with a follow-on the next day here.  Aggregated news sources suggest that Soon-Shiong has met at least three times with Trump, one shortly after the election and twice during January.

Soon-Shiong is a very, very strong proponent of molecular medicine, precision medicine, and Cancer Moonshot.  For additional speculation on the health czar role, Managed Care, here; LA Biz, here.

Update: On February 5, a New York Times article by Katie Thomas surveyed the FDA candidate field, dealing at most length with Jim O'Neill, suggesting the Times viewed him as a leading candidate worthy of comment.   O'Neill and Scott Gottlieb declined to comment to the Times.  Gulfo commented at some length.  Times article here.  More on Gulfo after the break.

For a representative trade journal news article on Gulfo and the ongoing FDA leadership "rumor mill," see Fierce Biotech, here.

Gulfo was interviewed January 24 by RAPS, here.

STAT found some negative re-tweets by Gulfo about Trump (e.g. lack of use of traditional word order for sensemaking) which Gulfo has disavowed, here.

A Bloomberg article argued that the FDA nominee should, like Gulfo, have clinical experience (here), a viewpoint that narrows the current field to Gulfo and Gottlieb.

Gulfo Blogs and Op-Eds

Gulfo had a blog at The Hill on December 27, proposing a new Trump administration to FDA approvals (it would also require legislation) with several categories of drug approvals (by biomarker, by disease modification, by long-term outcome).  Here.  

He also had a WSJ op ed in November, here.  He urges readers to review his full 13-page white paper on his FDA plan before criticizing it (white paper here).

Becker's Hospital Review provides "Six Quotes from Joseph Gulfo," here.

Gulfo Book

Gulfo's book, Innovation Breakdown -- I had a chance to read cover to cover (or the ebook equivalent) on a cross country flight yesterday, as I returned to California from an all-day health policy conference at Harvard.

I found the book very well-written, even surprisingly so for a first-time book author.  It's so full of detail and nuance on the events, it couldn't be ghostwritten (nor is there any implication it is.)

Gulfo  describes the travails of leadership of a small, sometimes financially struggling medtech company, and he found that the FDA sharply moved the goalposts on his product, ran a biased advisory board, and had a near-vendetta against his product.   He was highly dissatisfied with decisions he viewed as made from Dr. Shuren's office downward, and feels his product problems were around the time of Dr. Shuren's move to head of CDRH.  (For the FDA's concerns in a nutshell, which do have their own logic, see the Progressive Policy Institute's PDF, here).

Innovation Breakdown closes with a 18-point plan, which I rephrase and summarize here:
  1. Encourage leadership at FDA, including vision and direction of FDA.  (See his book on leadership, Care Quotient).
  2. Clarify priorities of FDA.  Includes concerns about FDA/Payer formal interactions (a recent priority of FDA).
  3. Define FDA review guidelines much more clearly.
  4. Have an Ombudsman report to the Commissioner, not parochially to each division head.
  5. Reform the advisory panel process, including veto power for companies (aka "voir dire" in jury selection).
  6. Expand and enforce the need for binding agreements on requirements for approval (on a per product basis).  Avoid shifting goalposts.
  7. Hold FDA accountable for missed deadlines.
  8. Redefine Major and Minor deficiencies (so the FDA can't arbitrarily and indefinitely stop the review clock).
  9. End the "Transparency Initiative."  (It should be an expectation, not a gift.)
  10. End the "Interactive Review Initiative."  (Same reasoning.)
  11. End the Innovation Pathway.    (It's become a confusing parade of window-dressing verbiage).
  12. Remove certain requirements at SEC (he was head of a small struggling but public company.)
  13. Eliminate baseless class action shareholder lawsuits.  (Today law firms spend $500,000 to manage and file $500M suits, and then settle for their $500,000 - he calls this a law firm scam.)
  14. "Eliminate Policies That Demand Evidence Based Medicine and Comparative Effectiveness Analysis Before Payment/Reimbursement."   (Aka, payers don't have to pay for everything, only things, that doctors order or use.)  AHRQ, PCORI = bad.
  15. Expand patients' ability to self-pay.  (He cites limited applications now, such as in a super-obscure Medicare policy for patient-pay intraocular lenses (here); it's a version of balance billing.  HHS nominee Price also favors balance billing, here, here).
  16. Establish an Innovation Index for CMS decisions.  This is an interesting idea.
  17. Replace redundant and copycat PDUFA/MDUFA "initiatives" with a sensible continuous improvement program.   (Again, a plea for less verbal window-dressing.)
  18. Encourage public feedback to FDA. Right now, sponsors don't want to risk burocratic retaliation by complaining about FDA in press or books.
Gulfo Article List

See an extensive hot-linked listing of Gulfo's recent articles at his Linked In, here.   For a six-minute, video interview on Youtube, from 2014, see here.

Gulfo On The Hill

For 20 minutes of Hill testimony by Gulfo, from February 2016, Senate webpage here, and at Youtube here.  For a transcript in the cloud, available only here: click here.

The panel focused on Right-to-Try drug law, and Gulfo spoke alongside Darcy Olsen, CEO of the Goldwater Institute, which has a policy initiative in this area (here).

The Initiative for Patient Centered Innovation (iPCI) at Fairleigh Dickinson University

See the website and further links for iPCI, here and here.  See also the 38-page white paper, "The Proper Role of the FDA for the 21st Century," by Gulfo, Briggeman, and Roberts (here).

Towards a Medical Innovation Impact Index (MI3)

One project of the iPCI institute under Gulfo is to produce and promote a Medical Innovation Impact Index, or MI3 (here, here).


Minor Footnotes

Footnote: Mela Sciences Recent News

The New York Times discussed the history of Melafind in July 2013, here.  I'm not an expert on the company's history, but Google shows it acquired two additional businesses in  June 2015, had a recall of Melafind in May 2015, and changed its name to Strata Skin Sciences (ticker SSKN) in January 2016.  According to the five-year view on Yahoo Finance, the stock price has been as high as $45, but is currently $0.55 with a market cap of $6M.   A transcript of the November 2016 earnings call is here.   SSKN still has Melafind (which also has its own branded website here) but the company also has devices with narrow-bandwidth light for treating psoriasis and vitiligo. (XTRAK, VTRAK devices).

For some SEC filing text about Melafind and reimbursement, here.

Footnote: Sleep and CEO Role in a Small Firm

Gulfo asserts he slept no more than 2-3 hours per night, for three months, leading up to his presentation at the FDA advisory board on his product.

Footnote: Some Voices Extol Need for Less Restrictive FDA

For a blog on the need for a more liberal FDA, at Marginal Revolution, here.  Alex Tabarrok writes that any current candidate - Srinivasan, O'Neill, Gulfo, Gottlieb - would be a favorable sea change.

For another conservative thinker on FDA reform, see the Hoover Institute's Henry Miller MD, here.

Footnote:  Califf's Outgoing Interview with Forbes

Just before resigning, Califf gave an exit interview with Forbes, here.  Discussing the Exondys decision, Forbes writes that Califf decided that, in his role as a "political appointee," Woodcock's decision as a senior career agency decisionmaker should stand, "unless she had mental illness or acted in a manner that was fundamentally unfair."

Footnote: Stanford Efforts at Digital Melanoma Diagnosis

In the footsteps of the long path of the Melafind device - On January 26, see press releases and a Nature article on a Stanford-based approach to digital melanoma diagnosis using neural networks.  Here and here.  The system was trained on 129,000 clinical images and could run on smartphones.