Defining Value in Cancer Care

US Supreme Court Passes the Affordable Care Act (Obama-care)

• 2014- all in US will either have insurance or pay a fine

• Will hopefully improve screening for cancer• Does not solve rising cancer care costs

Healthcare Reform: Time of Sacrifice or Opportunity?

• Successes to date have required significant investment coupled with global collaboration between governments, scientists, industry and patients

• The money we are investing in research is paying off.

• Our current healthcare system is unsustainable

Changes in Cancer Care Perspective

1971• Desperate for anything• All cancers the same• Dose intensity for all• Curing cancer• Minor cost impact• Safety and efficacy

2012• Treatments for all cancers• All cancers are different• Right drug, dose, patient• Cancer as a chronic disease• Major cost impact• Safety and efficacy

Copyright © American Society of Clinical Oncology

Meropol, N. J. et al. J Clin Oncol; 25:180-186 2007

The US spends twice as much as any other country on cancerbut has the same survival.

Cancer Costs- USA

• Hospitals• Drugs• Scans/ Surveillance• Doctors• Labs• Reduced patient productivity

The more we do to patients, the more money we make

Cancer care cost are rising exponentially in the US- $158 billion due to more of us- $173 billion at 2% growth rate

020406080

100120140160180

1990 1995 2000 2005 2010 2015 2020

Year

Cancer CareCosts (Billions)

Mariotto AB, et al. Projections of the cost of cancer care in the United States: 2010-2020. J Natl Cancer Inst. 2011 Jan 19;103(2):117-28.

Drug costs are increasing at more than double-digit rates.

• Worldwide, cancer drugs ~$40 billion per year. • U.S. - cancer drugs second biggest category of

overall sales.• 70% of these sales come from products

introduced in the last 10 years.• Currently, 200+ new molecules in Phase III trials

– Homeruns (imatinib CML, GIST)– doubles (vandetanib in med thyroid ca)– singles (sorafenib in HCC).– Most of them will be desired-

• How could we say no?

Hillner BE, Smith TJ. J Clin Oncol. 2009;27:2111-3

Inconvenient Truths

• The majority of U. S. cancer patients are treated with “practice guideline, evidence-based therapies.”

• Fewer than 5 percent of cancer patients participate in clinical trials

• Evidence-based medicine ≠ highly effective medicine

• We both curse and cling to our health insurance

U S Oncology pathways preserve survival, reduce cost by 34% in metastatic colon cancer.

Hoverman R, et al. Am J Manag Care. 2011 May;17 Suppl 5 Developing:SP45-52.

Table 1: Impact of pathways in colon cancer

Overall survival (mos)

Chemo Cost ($)

Total Cost

($)

Pathway(limited types)

26.9 22,564 103,379

Non-pathway(no limits)

20.1 60,787 156,020

P value 0.03 <0.001 <0.001

Crowded Closets

Inconvenient Truths

• Cancer care is a luxury item for wealthier countries

• Doctors are rationing treatment now• The current return on investment may not

support continued investment in cancer research• FDA approves drugs based on safety and efficacy-

ignores cost• CMS (US Medicare) provides coverage for new

therapies- ignoring magnitude of benefit

Regulatory Approval vs Payer Approval

FDA CMSSafety and Efficacy Pay for it somehow

A war should be fought together

• Our healthcare system is fragmented, with each component making individual and discrete decisions and with its own lobby

• What we need is an interconnected system in which the various components are complementary

Regulatory Approval vs Payer ApprovalGreat Britain

Regulatory Approval

NICEValue Metric

PHS Approved NO

Cash

Access

Poorer Countries

Their patients do our trials

Limited access to the drugs they

helped test

Emerging Markets and Cancer Care

ASCO 5 things• Don’t use cancer-directed therapy for solid tumor patients with the following

characteristics: low performance status (3 or 4), no benefit from prior evidence-based interventions, not eligible for a clinical trial, and no strong evidence supporting the clinical value of further anticancer treatment.

• Don’t perform PET, CT, and radionuclide bone scans in the staging of early prostate cancer at low risk for metastasis.

• Don’t perform PET, CT, and radionuclide bone scans in the staging of early breast cancer at low risk for metastasis.

• Don’t perform surveillance testing (biomarkers) or imaging (PET, CT, and radionuclide bone scans) for asymptomatic individuals who have been treated for breast cancer with curative intent.

• Don’t use white cell stimulating factors for primary prevention of febrile neutropenia for patients with less than 20 percent risk for this complication.

–Schnipper et al JCO 2012

Different perspective?

“We do not need to bend the cost curveWe need to bend the efficacy curve….

And we need to do it now….If we continue with the paradigm of the past

decade we will continue to sow the seeds of mediocrity”

Tito Fojo, MD NCI 2011

Benefits of Precision & Personalized Medicine:Rothenberg 2011

Bigger Treatment Effect Months

# Patients

400

800

18 30

Smaller Clinical Trials

+Less Costly, Faster Trial Completion

Benefit to Clinical Development

Benefit to PatientsEarlier Regulatory

Submission+

Earlier Launch

More Dramatic Effect in Treated Patients Value

of Treatment Easier to

Demonstrate

Months on Treatment

Unselected Patients

Selected Patients

Patients Treated More Likely to Benefit Longer Time on Treatment

• They require a lot of patients• They cost enormous amounts of money• They require a long time to complete accrual• During the time it takes to conduct the trial, new biological insights

– and new therapies – may emerge that could render the outcome of the trial irrelevant

• Pressure from payers, regulators, patients, and the public is increasing to develop effective treatments more quickly and cost-effectively

• Why would we ever do this?– We make it worth the gamble

Impact on Pivotal, Phase III Clinical Trial DesignRothenberg 2011

ValueSafety and

Efficacy

Agent HR $ Cost/Month(÷100)

Toxicity(G1+2) * (G3+4) # Patients

QOL/Utility ScorePass/Fail

Imatinib vs IFNCML 0.17 55.90 0.67

Nilotinib vs ImatCML 0.8 76.40 0.17

ImatinibGIST 0.4 55.90 1.22

Erlotinib vs ChemoMut NSCL

0.75 52.80 0.71

ErlotinibPancreas 0.82 52.80 11.9

Bevacizumab 2nd line CRC 0.74 22.90 0.8

Aflibercept2nd line CRC 0.79 ????- 3.0

Value Metric

Finding Value

• Come together• Listen to each other• Respect what we hear• Find the common threads• Weave a new fabric

- provide global healthcare with value

Global Drug Approval?

• We should strive for regulatory systems that permit new cancer therapeutics to be reviewed – Rapidly– Based on criteria that are flexible and relevant to the disease and

disease setting. – Replace “safety and efficacy” with a “value score”

• magnitude of benefit (eg hazard ratio)• drug costs• quality of life measured by patient recorded measures

– Price therapies and reimbursement in recognition of their: • value to the patient• the risk taken by the sponsor(s) in the development of the therapy• the degree of innovation it represents in the treatment of the disease.

Next Steps- US

• Re-examine intellectual property laws • Redesign the roles of the NCI, FDA and CMS • Incentivize the collection and sharing of

appropriately protected patient health information. • Incentivize patient partnerships