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From Idea to IPO UCSF February 7, 2011 REIMBURSEMENT AND REGULATORY STRATEGIES. Bruce Quinn MD MBA Foley Hoag LLP [email protected] 323 839 8637. Burrill, January 11, 2010, page 138. Jain, January 25, 2010, page 16. You (In Your Mirror). You (In Your Mirror). Viewpoint of Investor.

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from idea to ipo ucsf february 7 2011 reimbursement and regulatory strategies

From Idea to IPOUCSF February 7, 2011REIMBURSEMENT AND REGULATORY STRATEGIES

Bruce Quinn MD MBA

Foley Hoag LLP

[email protected]

323 839 8637

slide6

You (In Your Mirror)

Viewpoint of Investor

who am i why am i here
Who Am I? Why Am I Here?

Originally MD PhD Stanford

Pathologist

Medical School Professor

slide8

Originally MD PhD Stanford

Neuropathologist

Medical School Professor

MBA (2000)

Physician Executive with

Global Firm (Accenture)

slide9

Originally MD PhD Stanford

Neuropathologist

Medical School Professor

MBA (2000)

Physician Executive with

Global Firm (Accenture)

Medicare Medical Director

Through 2008

slide10

Originally MD PhD Stanford

Neuropathologist

Medical School Professor

MBA (2000)

Physician Executive with

Global Firm (Accenture)

Medicare Medical Director

Through 2008

Focused Strategy Consulting:

Integrated into Law Firm

(Foley Hoag LLP)

CMS & FDA issues

Based in LA;

Frequently in DC, SF

drugs devices diagnostics
Drugs, Devices, Diagnostics

Different products raise:

  • Very different regulatory and reimbursement issues
  • Very different roles for Medicare
  • Tonight:
    • Use Case Studies regularly to “make it real”
    • Hit all the high points – parts of FDA; payment codes; etc, but:
    • Substantial “why and how” content, not just lists of facts
our product replaces surgery
Our product replaces surgery
  • Currently, Condition X is treated by surgery
  • The charges for this surgery are $30,000
  • Our product avoids the surgery for most patients, it’s a physician office procedure
  • We’re efficient, based on a disposable of $1000 and capital equipment of $25,000
  • A fair price for our service is $5000 paid to MD
  • We have more data than our competitors and should be paid more
  • Thanks for your attention
barriers to entry22
Barriers to Entry?

REGULATORY

  • Voted down by FDA advisory panel (1/19/11)
  • Likely to require additional reader accuracy studies
    • Never required for CT, MRI, PET
  • FDA treats tracer synthesis like “mfgr” as of 2012
    • F18 PET tracer: 2 hr half life; Mfgr’d in each city
barriers to entry23
Barriers to Entry?

REGULATORY

  • Voted down by FDA advisory panel (1/19/11)
  • Likely to require additional reader accuracy studies
    • Never required for CT, MRI, PET
  • FDA treats tracer synthesis like “mfgr” as of 2012

REIMBURSEMENT

  • Dementia population nearly 100% Medicare
    • Requires NEW national coverage decision
  • PET covered only for cancer, and a VERY restricted FDG PET brain indication
  • “Clinical Utility”
    • Existing Study: hospice patients + 25 year olds.
    • Technology assessment will…”point this out”
    • What would “clinical utility” study be?
    • Comparative effectiveness? Generic tracer? Blood test? MRI scan alone?
  • Acquisition $300M (+ $500M milestones)
    • Guess: Wants $2B revenue at 2k/dose = 1M patients => 200K pts/year x 5 years
    • Patent life finite
    • Current PET dementia = 3k patients/year
  • Possible Timeline: FDA Dec 2011; CMS NCD during 2012; May need more data; Studies 2013; CMS review again during 2014.
  • Will CMS allow local MD to order?
  • CMS Bundles “tracer” to cost of “PET” currently paying $1200 total to hospitals
regulatory ii25
Regulatory II
  • FDA recently (last week) approved a brand of iPAD radiology software for diagnosis
  • FDA pursues digital microscopy companies vending for digital pathology w/o FDA approval
regulatory iii
Regulatory III
  • FDA recently (last week) approved iPAD radiology software for diagnosis
  • FDA pursues digital microscopy companies vending for digital pathology w/o FDA approval
  • IF it was pathology, it would require BOTH “FDA” software clearance AND “CLIA” laboratory site (!)
slide27

HHS

Hill

FDA

CMS

Law

  • CDER
  • CBER
  • CDRH
  • Coverage
  • Policy (coding, pricing)
slide28

PCORI

MEDPAC

NIH

AHRQ

FDA

CMS

IPAB

IOM

  • CDER
  • CBER
  • CDRH
  • OIVD
  • “Regulatory Science”
  • Coverage
  • Policy (coding, pricing)
  • Center for Innovation
  • Activist Director
slide29

ACO (Accountable Care Organization)

PCORI

MEDPAC

NIH

AHRQ

FDA

CMS

IPAB

IOM

  • CDER
  • CBER
  • CDRH
  • OIVD
  • “Regulatory Science”
  • Coverage
  • Policy (coding, pricing)
  • Center for Innovation
  • Activist Director
slide30

FDA

CMS

  • CDER
  • CBER
  • CDRH
  • Coverage
  • Policy (coding, pricing)
fda approval
FDA Approval

Payor Coverage

regulatory fda
Regulatory = FDA

FDA

CDER

CBER

CDRH

drugs

biologicals

OTHER

OIVD

diagnostics

“devices”

Phase 1,2,3

(…FOBs)

generics

510K or PMA

phase 1 2 3
Phase 1,2,3

Phase 1

Phase 2

Phase 3

  • First in man
  • Animal safety shown first
  • (And would not bother w/o animal efficacy)
  • Basic kinetics
  • No big, big surprises
phase 1 2 334
Phase 1,2,3

Phase 1

Phase 2

Phase 3

  • First in man
  • Animal safety shown first
  • (Would not bother w/o animal efficacy)
  • Basic kinetics
  • No big, big surprises
  • “Pivotal” trials
  • Enrollment determined by expected size of effect
  • Entrance criteria tightly defined
  • Outcome measures tightly defined
  • Stopping rules for toxicity, clinical failure, or unexpectedly large positive results
  • Size also determined by safety concerns
  • Duration determined by future clinical setting (a bit)
  • Active vs placebo control group
phase 1 2 335
Phase 1,2,3

Phase 1

Phase 2

Phase 3

  • First in man
  • Animal safety shown first
  • (Would not bother w/o animal efficacy)
  • Basic kinetics
  • No big, big surprises
  • “Pivotal” trials
  • Enrollment determined by expected size of effect
  • Entrance criteria tightly defined
  • Outcome measures tightly defined
  • Stopping rules for toxicity, clinical failure, or unexpectedly large positive results
  • Size also determined by safety concerns
  • Duration determined by future clinical setting (a bit)
  • Active vs placebo control group
  • Serves both economic and safety purposes
  • Place before Phase III because:
  • Economic:
    • Don’t spend $200M for a flop drug
  • Ethics:
    • Don’t expose 1000 people unless drug is likely to work
  • Scientific
    • Dose ranging, biomarkers, etc.
damned if you do if you don t
Damned if you do, if you don’t…
  • Study may be heavily criticized because it used “only” a placebo control
    • Sure, anything is better than placebo
  • Study may be heavily criticized because it used “only” an active control
    • Critic may be unsure either drug in the trial was any better than placebo
    • You’ve shown you’re non-inferior to ‘zip’
  • Outcome measures: Cancer: “time to progression” versus “Survival”…?
damned if you do if you don t37
Damned if you do, if you don’t…
  • Study may be heavily criticized because it used “only” a placebo control
    • Sure, anything is better than placebo
  • Study may be heavily criticized because it used “only” an active control
    • Critic may be unsure either drug in the trial was any better than placebo
    • You’ve shown you’re non-inferior to ‘zip’
  • Outcome measures: Cancer: “time to progression” versus “Survival”…?
  • Survival usually considered “gold standard” but heavily criticized at November CMS meeting (Provenge) because patients diverge after treatment…can’t be controlled any more…
increasingly authority for rems
Increasingly authority for REMS
  • Risk Evaluation and Mitigation Strategies
  • Post-marketing requirements for education and monitoring
  • May rise as high as a formal patient registry
  • Control of drug distribution (only doctors of type X can prescribe drug)
  • http://www.fdalawblog.net/fda_law_blog_hyman_phelps/2009/03/introducing-the-fda-law-blog-rems-tracker.html
fast track and accelerated approval
Fast TrackandAccelerated Approval
  • Sound alike, but not hard to distinguish
  • Fast Track
    • Accept materials on rolling basis
    • Priority review
    • Final approval is “regular” approval, but faster
  • Accelerated Approval
    • Get on market faster
    • More requirements for stuff you still have to do
    • Somewhat like a “provisional” approval*

*They wouldn’t say that.

See: PhD thesis, Univ. Edinborough, D. Messner, “Fast Track”, 2008

clinical trial end points
Clinical Trial End Points
  • FDA has high standards
    • Payors may have even higher standards
    • You are at risk if next competitor uses “better” end points in their trial
  • Biomarkers as end points
    • Must be highly validated: T-count in HIV
  • Biomarkers for patient selection
    • Only patients with Tumor Gene X enter the trial
    • Reduce trial size & improve relative effectiveness
  • Recent topics like Bayesian/Adaptive trials
    • Reduce net trial size
  • Especially hot controversy over oncology endpoints
    • PFS versus OS:
    • Progression Free Survival versus Overall Survival
    • Tiny PFS without OS annoys FDA, public, payors
    • Yet PFS could be important – if it’s substantial – so it depends
      • Did the study FAIL to show OS, or is it a question of study duration & power (OS is noisy)
devices
Devices
  • 510k
    • Device has a predicate device before 1976
    • There are very artful and legalistic ways to argue this
    • See an FDA lawyer
      • Example labeling: MRI, PET; Colonoscopy, Pillcam
      • 510k Reform: 2010, 2011, 2012
  • PMA (pre-market approval)
    • Device is higher risk
    • Requires clinical trial AND other rules
  • Note the hurdle: Payors will look to the ACTUAL clinical data
fda oivd
FDA: OIVD
  • Office of In Vitro Devices
  • Genomics revolution: Much larger companies, with much more specialized tests
    • Genomic Health, Redwood City, 21-gene breast cancer test, $500M market cap
    • FDA announces intent to regulate some LDTs in 2007
    • Holds public meetings on plans in 2010
    • Announces initial plan to appear in by March 31, 2011

Traditionally Did Not Regulate LDT:

Laboratory Developed Tests;

These were “services” and “practice of medicine”

Traditionally Regulated:

Clinical Chemistry Lab Equipment

(Kits, Cartridges)

slide44

Clinical Policy Bulletin:

  • Colorectal Cancer Screening
  • Aetna considers any of the following colorectal cancer screening tests medically necessary …Colonoscopy…..
  • No current guidelines of leading medical professional organizations or Federal public health agencies recommend the following.
  • Aetna considers colorectal cancer screening of stool using molecular genetic techniques experimental and investigational.
  • Aetna considers colorectal cancer screening using methylated Septin 9 (ColoVantage) as experimental and investigational.
why don t we see cost saving innovation in health care
Why don’t we see cost-saving innovation in health care?

Five years

Same size

Same price

1000X better

2002

2007

why don t we see cost saving innovation in health care47
Why don’t we see cost-saving innovation in health care?

Five years

Same size

Same price

1000X better

2002

2007

Fifty years

10,000X price

Not sure it’s better

here are a few of the innovation problems we are stuck with in healthcare technology
Here are a few of the innovation problems we are STUCK WITH in healthcare technology
  • Human subjects research
  • Locked-in timelines for outcomes are longer than consumer goods – 1,2,3 year trials; the cycle of iteration is long
  • Regulatory hurdles
  • Incentives for quality + cost-savings are lacking
    • Indirect payment
    • Assymetric information (K. Arrow)
    • Very complex information

But let’s think further about what the payer system does to us.

slide49

A Regular Industry

$$

Book

Venture

Capital

Amazon.com

Consumer

$$

$$

$$

Book

Publisher

slide50

A Regular Industry Gets Simpler

$$

Info

Venture

Capital

Amazon.com

Consumer

$$

$$

$$

Info

Publisher

slide51

Govt

Payor

Private

Payor

The Healthcare Industry

Physician

Patient

Self Pay

Info

$$

Venture

Capital

Pers. Med.

Firm

Info

Individual

$$

Patient

$$

Employer

Blood

$$

Hospital

Govt

$$

Info

$$

Taxpayer

$$

The square boxes, where the money originates, have weak linkages to anything else or to each other.

One pathway is mapped, e.g. a blood test for cancer for a Medicare patient.

four horsemen of the apocalypse
Four Horsemen of the Apocalypse

We just saw this one.

slide53

Industry structure → fundamental economics problem

  • FIRST LAW:
    • INNOVATION requires prices above marginal cost, to repay development

Innovation and marginal costs: McKenzie & Lee (2008) In defense of monopoly. Univ Mich Press.

Margin & overutilization: see 8/29/2008, http://medpac.gov/documents/Physician%20fee%20schedule%202009%20NPRM%20comment%20letter%20to%20CMS.pdf

slide54

Industry structure → fundamental economics problem

  • FIRST LAW:
    • INNOVATION requires prices above marginal cost, to repay development
  • SECOND LAW:
    • Prices above marginal cost drive “over utilization” in health care

Innovation and marginal costs: McKenzie & Lee (2008) In defense of monopoly. Univ Mich Press.

Margin & overutilization: see 8/29/2008, http://medpac.gov/documents/Physician%20fee%20schedule%202009%20NPRM%20comment%20letter%20to%20CMS.pdf

slide55

Industry structure → fundamental economics problem

  • FIRST LAW:
    • INNOVATION requires prices above marginal cost, to repay development
  • SECOND LAW:
    • Prices above marginal cost drive “over utilization” in health care
  • RESULT ONE:
    • Government tries to drive prices to marginal cost as fast as possible
      • Particularly acute in laboratory tests
      • Payor tries to pay nothing (non coverage decision)

Innovation and marginal costs: McKenzie & Lee (2008) In defense of monopoly. Univ Mich Press.

Margin & overutilization: see 8/29/2008, http://medpac.gov/documents/Physician%20fee%20schedule%202009%20NPRM%20comment%20letter%20to%20CMS.pdf

slide56

Industry structure → fundamental economics problem

  • FIRST LAW:
    • INNOVATION requires prices above marginal cost, to repay development
  • SECOND LAW:
    • Prices above marginal cost drive “over utilization” in health care
  • RESULT ONE:
    • Government tries to drive prices to marginal cost as fast as possible
      • Particularly acute in laboratory tests
      • Payor tries to pay nothing (non coverage decision)
  • RESULT TWO:
    • Innovators complain that system is blind to value-based reimbursement

Innovation and marginal costs: McKenzie & Lee (2008) In defense of monopoly. Univ Mich Press.

Margin & overutilization: see 8/29/2008, http://medpac.gov/documents/Physician%20fee%20schedule%202009%20NPRM%20comment%20letter%20to%20CMS.pdf

slide57
An industry-based workshop participant asserted,”There is a substantial market failure on the payor side.”

UW Seattle

Nov 2007

New Technology Stakeholders

Venture

capital

Wall Street

Pharma

NIH

FDA

Marketing

Payor

Big Labs

Start Ups

His assertion was, Payors are in a pivotal position in the entire system but drastically under-resourced and/or dealing with fuzzy tools, vague guidelines, and creating uncertainty across all the other (earlier) stakeholders in achieving improved patient care.

Univ Washington workshop on personalized medicine, comment of participant, 10/31/2007.

slide58

New Yorker’s View of the World

March 29, 1976

Saul Steinberg

slide59

Payor’s View of Health Care (Apologies to Steinberg)

Net health outcomes might seem to be the most important aspect of the system!

Carve Outs

(Vision, etc)

Net Health

Outcomes

Customer

Service

Lines

Technology

Assessments

Claims

Processing

Fraud

Enrollment

Risk

(Loss)

Appeals

Competitors

Enrollment

Processes

(Pt, MD)

Regulatory

Environment

slide60

CONTENTOF HEALTH CARE

Patient’s Illness

Therapeutic Choices

Patient’s Illness

Follow up

Diagnostic Tests

Evidence for each

Diagnostic Tests

Risks/Benefits

Concurrent Factors

Judgment

Comparative Effectiveness

Judgment

Patient Perception

Availability/Costs/Margin

Judgment

slide61

CONTENTOF HEALTH CARE

Patient’s Illness

Therapeutic Choices

Patient’s Illness

Follow up

Diagnostic Tests

Evidence for each

Diagnostic Tests

Risks/Benefits

Concurrent Factors

Judgment

Comparative Effectiveness

Judgment

Patient Perception

Availability/Costs/Margin

Judgment

PROCESSOF HEALTH CARE REIMBURSEMENT

Policy

“Bene”

Pay

Procedure Code

Claim

Form

Edits

Provider

Diagnosis Code

Deny

who will be getting paid for your product
Who will be getting paid for your product?

B = Bundled. ASP = Drug Average Sales Price. POC = Point of care.

payors in the us
Payors in the US

Government

Non Government

Medicare

Medicaid

Employer

Insurance

Individual

Insurance

  • Also:
  • Veteran’s Health system
  • Public/City Hospitals
hochkomplex elsewhere too
Hochkomplex Elsewhere Too
  • Das deutsche Gesundheitswesen is hochkomplex and für Aussenstehender nur schwer durchschaubar.
  • Selbst Experten haben Schwierigkeiten, die Struktur und Funktionsweise der vershiedenen Teilsysteme und Versorgungsbereiche insgesamt zu Überblicken.
  • Das Gesundheitssystem in Deutschland: Eine Einführung in Struktur und Functionsweise, M Simon (Verlag Hans Huber, 2005)
medicare
Medicare
  • Medicare is the largest single payer
  • Sets many of the rules for coding and payment system
  • Odd mix of defined and undefined rules and processes
  • National system and subordinate local systems are highly confusing
four different sides of medicare
Four Different Sides of Medicare

Organizations

Regulatory

Cascade

Operations

Contractors

organizations for medicare
Organizations for Medicare

Congress creates Health & Human Services as part of the Executive Branch

Centers for Medicare & Medicaid (CMS; Baltimore)

OIG

Federal Employees

Medicare Central Office (CO)

CMS Regional Offices

ALJ system

MEDICARE PART B

(NHIC)

Fee For Service

(80% of Part B)

Contractors

MEDICARE PART A

Hospitals & Institutions; Renal Benefits

MEDICARE DME

Durable Medical Equipm.

MEDICARE PART D

Drug Benefit

CAP (Competitive Acquisition Program; Delivery of office drugs)

RHHI

MEDICARE Managed Care (PART C)

Medicare Advantage (Formerly Medical +Choice)

QIO Quality Improvement Organization

(CA = Lumetra)

PSC “Program Safeguard Contractors” (Fraud)

QIC (Quality Contractors – Appeals)

RAC (Recovery Audit Contractors – Demo Project CA FL NY)

regulatory cascade
Regulatory Cascade

Congress

US Code (Social Security Act, SSA Title XVIII, Health)

CMS

Code of Fed Reg CFR

National Coverage Decisions (NCD)

Medicare Program Manuals

(Program Manual Guidance)

  • Local Coverage Decisions (LCD)
  • Local public comment
  • Local advisory board
  • The SSA stands, unless ruled unconstitutional.
  • The CFR stands, unless ruled outside the agency’s enabling act, the SSA
  • The program manual binds carriers, but can be overruled by an ALJ.
  • The publicly available documents are voluminous.
  • Also secondary sources: OIG, GAO, RWJ Fdn, MEDPAC, Congress

Carrier

Policies (LCD)

Articles

Internal Guides

the odd power of medicare contractors
The odd power of Medicare contractors
  • Highly variable individuals
  • Some are excellent
  • Some would not survive ten minutes in the business world
  • Much delegation to “local medical directors”
    • May be highly overcommitted and limited management skills
    • Virtually none have ever worked in a business environment
  • Weak controls
    • Factors include “Administrative Procedures Act” – Agency is often unwilling to “tell” contractors what to do without formal rulemaking
coding systems
Coding Systems
  • HIPPA law says that HHS must specify: specific code sets shall be used between providers and payers
  • CMS sets this in regulation and reviews every ten years
  • There are several very different coding sets
  • ICD-9 for diagnoses
  • Codes for procedures
  • Codes for “stuff”
the 20 000 foot view
The 20,000 foot view

ICD international classification of disease; MD physicians; ASC ambulatory surgicenter; CPT AMA Common Procedural Terminology ®;

NCD National Drug Code; HCPCS Healthcare common procedure coding system

the rvu process
The RVU process
  • Survey of physician time, other clinical time, disposables, capital equipment
  • Must be approved by committee

Breakdowns down CPT office services to:

  • Minutes of physician, nurse, staff time
  • Disposable inputs (from rubber gloves to surgical disposables)
  • Capital equipment (e.g 5 year life, price allocated at 20 cents per minute of use)
  • Indirect cost (varies somewhat by specialty)
  • Various budget neutrality factors
  • RVU = relative value unit = physician time and supply inputs are valued in the same unit
rvu prognostication
RVU Prognostication
  • With a little experience, you can usually predict the result of the AMA RVU pricing within 20%
  • NOT value based
  • NOT value based
clinical chemistry is handled differently
Clinical Chemistry is handled differently
  • Clinical chemistry lab codes fall in 80,000’s of the CPT system
  • Most prices were set by freezing 1984 prices
    • CLFS Clinical Laboratory Fee Schedule
  • There are no copayments (in CMS system at least)
  • Prices slightly updated for inflation, when Congress feels like it
  • MOST payors contract using Medicare CLFS as a reference
    • 90%, 100%, 120%, etc.
  • MOST new chemistry tests are cross walked to existing test pricings
    • Eg Hep A is $15, so Hep B is $15, and if you invent Hep E, it will be $15….
    • New CPT codes for the coming January are priced by CMS during summer and fall
    • If CMS can’t price, it lets its regional contractors price for 6 months, then freezes the median price (“gap fill” pricing)
molecular tests have component prices
Molecular Tests have Component Prices
  • Code stacking can be used to price molecular tests
  • Code for DNA extraction $20
  • Code for DNA amplification $20
  • Code for capillary electropheresis $20
  • This may work very well for some tests
    • E.g. 10 steps x $20 = $200 and you may be able to make a kit for $30. The provider still gets $200.
incentives in lab tests
Incentives in Lab Tests

The fixed price system creates several important incentives to innovation:

  • Do service faster & cheaper (time is money; labor is money)
  • Lower price of your kit vs competitors
  • Fixed price for provider laboratory
      • Lab kit industry (IVD industry) faces commodity pricing in some situations
  • Provide unique tests with strong IP
    • Pricing power, no competitors
  • Difficult to fund substantive clinical research
    • Between rock and a hard place
    • FDA standards were lighter in 1970s, the era on which 1984 prices are based
  • Advamed has set up special division for IVD
some producers have stretched the lab fee system
Some producers have stretched the lab fee system
  • Classic example: Genomic Health
  • High research investment for single test
  • “Pharma” model pricing
    • CMS has public rules that can allow “median price of other insurers” to be CMS price
  • High burden in negotiating 1:1 for coverage and payment for every possible insurer
  • CMS unsure what to do next
    • AMA CPT is actively producing codes for specific genomic tests; lobbies to have them placed on “physician fee schedule”;
    • RVU pricing = time and motion pricing?
evidence based medicine comparative effectiveness
Evidence Based MedicineComparative Effectiveness
  • Really nothing new relative to last decade
  • A lot more attention to how decisions are made
  • Medicare coverage decisions at National level are more thoughtful and less from-the-hip than in 2000 (!)

BUT:

  • We are much better at “reviewing the literature” than making decisions
  • A forty-page dossier format is NOT a “decision”

My slide January 2010

evidence based medicine comparative effectiveness87
Evidence Based MedicineComparative Effectiveness
  • Really nothing new relative to last decade
  • A lot more attention to how decisions are made
  • Medicare coverage decisions at National level are more thoughtful and less from-the-hip than in 2000 (!)

BUT:

  • We are much better at “reviewing the literature” than making decisions
  • A forty-page dossier format is NOT a “decision”

WE HAVE GOTTEN WORSE

AT REVIEWING THE LITERATURE

slide88

PCORI

MEDPAC

NIH

AHRQ

FDA

CMS

IPAB

IOM

  • CDER
  • CBER
  • CDRH
  • OIVD
  • “Regulatory Science”
  • Coverage
  • Policy (coding, pricing)
  • Center for Innovation
  • Activist Director
hierarchy of evidence
Hierarchy of Evidence
  • Meta-analysis of RCT’s
  • Multiple RCTs
  • RCT
  • Cohort study
  • Observational study
  • Retrospective study
  • Expert opinion
hierarchy of evidence91
Hierarchy of Evidence
  • Meta-analysis of RCT’s
  • Multiple RCTs
  • RCT
  • Cohort study
  • Observational study
  • Retrospective study
  • Expert opinion

The gene that

causes

Cystic Fibrosis

hierarchy of evidence92
Hierarchy of Evidence
  • Meta-analysis of RCT’s
  • Multiple RCTs
  • RCT
  • Cohort study
  • Observational study
  • Retrospective study
  • Expert opinion

1-year survival of

Heart Transplant patients

At Harvard…

hierarchy of evidence93
Hierarchy of Evidence
  • Meta-analysis of RCT’s
  • Multiple RCTs
  • RCT
  • Cohort study
  • Observational study
  • Retrospective study
  • Expert opinion

Henry Kaplan and

radiotherapy of

Hodgkin’s disease

Mid 1960s

idiocy really happens
Idiocy really happens
  • AHRQ/USPSTF rated the evidence that BRCA is associated with familial breast cancer as: “fair”
  • ! ! ! ! ! ! ! ! ! ! !
bad technology assessments
Bad Technology Assessments
  • Provenge Medcac (November 2010)
    • Data rated only “fair” (only 3 RCTs)
    • Questioned use of “overall survival” endpoints
    • Raised “stupid” concerns
  • Renal transfusion/transplant Medcac (January 2011)
    • Tranfusions cause antibodies to others; those antibodies make transplants difficult
    • TA didn’t find this convincing: written by non-professionals, confused older and newer data; mixed different types of transfusions; LEFT OUT patients who were transfused into an “untransplantable” condition
    • Ridiculous statements made at Medcac
    • EBM is Reverse of “emperor has no clothes”: Trying to say a fully dressed emperor is really naked!
  • Badly framed questions lead to ridiculous TA’s
    • Imagine asking: What is the data that American Indian women respond to Herceptin?
    • …There is no data!
rhetorical question
Rhetorical Question
  • We remember the great medical scientists of the 60’s and 70’s
  • We may remember the most prominent med school deans
  • Who were the great insurance medical directors of the 60’s and 70’s
    • Question is nonsensical!
real world requires judgment
Real world requires “judgment”

Lancet 2008 372:2152, also:

http://www.rcplondon.ac.uk/pubs/brochure.aspx?e=262

http://www.cmtpnet.org/documents/egd_ge.pdf

psa rcts 2009
PSA RCTs 2009

Outdated PSA cutoff

40% of men had neg PSA prior

50% of “controls” had PSA’s

Often no action for 1 year

Prematurely published

Flawed at the starting gate

Will never be informative…

[NONE of these relate to P values, etc]

possible case studies
Possible Case Studies
  • Least Costly Alternative Pricing
  • Focal Prostate Cancer Radiation Therapy
  • Gene panel tests for Tumor of Unknown Origin
  • Provenge (prostate cancer immunotherapy)
  • FDA orphan drugs; Colchicine drug
  • Politics of CPT codes for top-end genomics
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