Bringing Clear Thinking to Evidence Based Medicine and Evidence Based Policy

Two very recent articles, each discussed in a separate blog, discuss how to bring more lucid and objective insights to two common policy problems.

The question of whether the FDA is "too strict" or "too lax" is perennial for decades.  In a new open access paper, MIT authors Montazaerhodjat and Lo frame this as a "type I" and "type II" error problem, and show an analytical approach to decision-making.  The paper is here - "Is the FDA Too Conservative or Too Aggressive?  A Bayesian Decision Analysis of Clinical Trial Design."  For a summary you can read on your smartphone, see the blog at marginalrevolution.com by Alex Tabarrok (here).  [*]  Tabarrok's blog includes an active discussion chain.

Another very important and perennial question is:  "How Do You Know Which Health Care Effectiveness Research You Can Trust?"  Soumerai, Starr, and Majumdar (from Harvard, Boston University, and University of Alberta) discuss this at the CDC journal Preventing Chronic Disease, here (open access).  Soumerai and Koppel overview the study at Health Affairs Blogs, here.

[*]
Footnote.
Companion piece to Montazerhojdat and Lo.  In July 2015, BIO released a white paper called, "Life Cycle Perspective for Structured Risk Benefit Design."  Here.

CMS Posts Youtube Video of Lab Advisory Expert Panel (August 26, 2015)

At the beginning of August, CMS announced the membership of its new Congressionally required expert panel on laboratory issues, including payment and coverage (article here).  The panel held its first meeting on site at CMS in a public hearing on August 26, 2015.  

Note:  CMS also convened the panel AFTER its preliminary price postings; that second panel meeting was October 19, 2015.

Added 10/2017:  Unofficial uncorrected transcript of the August 26 meeting, here.

Highmark Announces "Coverage with Evidence Development-Like" Program

Highmark announces a program called VITAL for selected new technologies.  VITAL is described as, "a program that aims to accelerate the adoption of new technologies that have received regulatory approval but are not yet covered by most commercial insurance companies."

According to the industry website Healthcare Dive:

     “Technologies that have received regulatory approval from the FDA often lack sufficient scientific data to convince commercial insurers to pay for them,” the company says. “Without support from commercial payers, it is difficult for new innovations to influence the practice of medicine.”
     Highmark Health aims to influence this process through its position as the country’s third-largest integrated healthcare delivery and financing system. It describes the VITAL program as “the missing link between FDA approval of an innovative technology and its full reimbursement.”

For more on Highmark and this program, see Healthcare Dive, here.

Healthcare Dive's home page is here.  See also their BioPharma Dive, here.  Both are good industry news sources.

CMTP Issues Guidance for Payer Coverage of Next Gen Sequencing

CMTP Press Release:

Collaborative Issues Initial Guidelines for Health Insurance Coverage of Next Generation Sequencing (NGS) Testing in Oncology

Multi-stakeholder group recommends coverage for NGS panels of 5-50 genes (including all standard of care genes); 23 pages; online here.

Press release after the break.

IQWIG TWEETS....

Germany has a powerful and very active health technology assessment group, IQWiG.   The official English translation is:  Institute for Quality and Efficiency in Healthcare; the German original is, Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen.

Since they are viewed as powerful and ominous by some stakeholders, it's interesting to see their Twitter feed.  After the break.

Two excellent discussions of biopharma/medtech innovation, pricing, and strategy

Two excellent articles this week on biopharma/medtech innovation, pricing, industry challenges, and strategy.

At the Health Affairs blog, Jane Horvath has a broad and content-rich review of why pharma prices land where they are, giving the reader a deep view of pharma economic strategies.  Here. (2000 words.)   Horvath is an experienced industry consultant and former HHS policymaker.

At Harvard Business School's thought capital site, Working Knowledge, Michael Blanding writes: "Why Medical Devices Get to Patients Too Slowly."   Here.   Discusses and paired with a 59-page PDF white paper by Ariel Dora Stern ("Innovation under Regulatory Uncertainty: Evidence from Medical Technology.")  Here.

Stern is Assistant Professor at Harvard Business School, faculty webpage here; her impressive CV here.  See also her "Limits on the use of health economic assessments for rare diseases," Quarterly Journal of Medicine, 2014, here.

Structured Risk Benefit and Flibanserin

One of the most-visited articles on this blog is a 2014 article on structured risk-benefit, which makes some comparisons with how regulatory bodies are trying to cope with uncertainty and risk/benefit and how this might give insights to payer coverage decision-making (here).  The FDA's approval of Addyi (flibanserin) raises an opportunity to look at structured risk-benefit theory in the context of a drug approval that was a rapid media sensation.

"Addyi is a serotonin 1A receptor agonist and a serotonin 2A receptor antagonist, but the mechanism by which the drug improves sexual desire and related distress is not known."     [FDA press release, 8/18/2015]

More after the break.

Clinical Sequencing in Breadth and Scope - Conference 8/2015

We're frequently confronted by the scale of the next generation sequencing (NGS) industry, but I was uncommonly struck by the scope and scale of a conference this week in Washington DC, where I'm chairing a panel on federal payment reform in diagnostics.

We take for granted that NGS is  moving incredibly fast - see, for example, a September 2014 market report that states, "The global NGS market will be worth $2.5B in 2014 and is poised to reach $8.7B by 2020" (here).

The clinical literature is growing at a hockey-stick rate as well, as shown in the graphic below that culls PubMed for the term "clinical exome."  There were 2000 publications using this search term in the last several years, up from a near standstill in 2011.

More after the break.

MolDX Finalizes Coverage of Nanostring Prosigna Test

On May 14, 2015, the MolDX program published a draft LCD proposing to cover the Nanostring Prosigna test for breast cancer (here).   On August 13, 2015, MolDX published a final coverage LCD which becomes effective on 10/1/2015, coincident with the new ICD-10 diagnosis codes.

The final Prosigna LCD (essentially the same as the draft) is available here - L36125.   MolDX also published a concise response to comments, primarily acknowledging letters of support of the draft policy (A54565, here).

The LCD also exists in draft form in KY/OH under the CGS MAC.  That draft LCD, L36127, does not yet state it is released-to-final (here).

The company's press release is here.

MolDX Updates its Frequently Asked Questions (August 5, 2015: Ver 5)

On August 5, 2015, MolDX updated its "Frequently Asked Questions," which is now in Version 5.
The new version is available on its own MolDX webpage, here.
For those interested in updates between the February 2015 version and the August 2015 version, a redline copied is here.

Noridian Announces Expansion of MOLDX to Jurisdiction F (And J E "Pt A")

On August 4, 2015, Noridian announced expansion of the MolDX program from its current Noridian footprint - Jurisdiction E "Part B" only - to Jurisdiction E "Part A" and Parts A/B in Jurisdiction F.  Their website article provided after the break.

Association for Molecular Pathologists Releases Lab Regulation Plan

As released on its website, on August 4, 2015, the Association for Molecular Pathologists (AMP) met with Senate Finance Committee staff to discuss the AMP's framework for some upgraded controls or regulation of laboratory developed tests.  The project is titled, "Modernization of CLIA Regulations
for Laboratory Developed Testing Procedures (LDPs)."

More after the break.

Federal Court Finds DNA Fingerprint for Specimen I.D. is not a Medicare Benefit

For a couple of years, it's been possible to track the progress of a molecular pathology case through the Medicare appeals system, which works through independent review contractors, a federal administrative law judge, a Medicare board of administrative law judges, and eventual the federal courts.

The lab in question can use DNA fingerprinting to confirm that the tissue in a paraffin block is a perfect match to the patient's DNA (based on blood or saliva).   Some local MAC decisions had found that this was not a Medicare benefit but a lab quality operations procedure.

This summer, a federal judge agreed with the CMS agency that this type of DNA fingerprinting was not, in itself, a payable Medicare benefit as a laboratory service.  The case is online here.

Early parts of the Medicare case, from February 2013, online here, and September 2012, here.  A discussion of the case is also available here (page 4 forward) by Wachler, who represented the lab.

2015 White Paper: Building the Genomics Infrastructure

In late July, the Center for Data Innovation and the Health IT NOW coalition released an insightful ten page white paper, "From Evolution to Revolution: Building the 21st Century Genomic Infrastructure."

In the last few days I've heard a half-dozen different individuals or organizations raise thoughts along these lines, so the white paper must be capturing the Zeitgeist.

Links to the press release, here - and to the actual white paper, here.

Press release quoted after the break.

CMS Announces Members of Lab Advisory Panel; First Meeting 8/26/2015

CMS has announced the experts it has selected for a fifteen-member expert advisory panel on laboratory policy.   The panel will hold its first public meeting on August 26, 2015.

The panel will specifically give CMS an opportunity to reopen and revisit issues about molecular test crosswalking and issues on drugs of abuse testing that were discussed at a prior July 26, 2015 public meeting (here).  Details after the break.  Updated August 20, 2015 with additional information about each panelist.