Mayo Clinic College of MedicineMayo Clinic Comprehensive Cancer Center

Affordable Strategies for the Treatment of Myeloma

S. Vincent RajkumarProfessor of Medicine

Mayo Clinic

Scottsdale, Arizona Rochester, Minnesota Jacksonville, Florida

S. Vincent Rajkumar, and Jean Luc Harousseau Blood 2016;128:2757-2764

S. Vincent Rajkumar, and Jean Luc Harousseau Blood 2016;128:2757-2764

©2016 by American Society of Hematology

Number of new myeloma patients in the US in 2017: 30,000

Excludes spending on hospital, infusion, laboratory, imaging, physician, nursing, and 

ancillary costs

Total lifetime costs to treat all patients diagnosed in 2017 : $22.4 billion

Mailankody S. ASCO 2016

What’s going on?

Rajkumar SV. ASCO Ed Book. 2018

Rajkumar SV. ASCO Ed Book. 2018

1 week of VRD

Active Drugs in Multiple Myeloma

Alkylators Steroids Interferon Anthracyclines

Old Drugs

Carfilzomib Pomalidomide Panobinostat

Ixazomib Daratumumab Elotuzumab

Recently ApprovedDrugs (2013-2015)

Bortezomib Thalidomide Lenalidomide Liposomal

doxorubicin

Older Drugs(2003-2007)

Rajkumar SV. 2018

Oprozomib Marizomib

Isatuximab

Filanesib LGH 447 Venetoclax Dinaciclib CAR-T Selinexor Anti-BCMA ab

Future Drugs

FACTS

All new cancer drugs approved by FDA in 2014: >$120,000/year

Average annual household gross income in USA:

$52,000

1 in 3 get cancer

• Compliance

• Bankruptcy

Its not just new drugs….

http://www.nytimes.com/2015/09/21/business/

Daraprim $4477 at Costco with coupon! (from @goodrx)

Life in the last 20 years

Courtesy: Twitter user @lollydaggle

Courtesy: Twitter user @lollydaggle

Insulin

Why are prices so high?

#1

Why are prices so high?

• Blatant and Occult Patent Evergreening• Cost of drug development• Seriousness of Cancer

Why are prices so insanely high?

Unique to USA• Medicare cannot negotiate• Ban on personal importation• Reimbursement System encourages more

expensive option• Liberal Off-Label use • No equivalent of Health Authorities

Lack of Allies

• Physicians• Professional Organizations/ Societies• Patients • Patient care organizations• Insurance companies

What can we do?

What can we do?

• Establish Value-based pricing • Medicare allowed to negotiate• Allow importation for personal use• Facilitate easy entry of generics and bio-similars

Unique to USA

But what can we do?

1. Choose best possible option

Alternative lower cost approach to the treatment of multiple myeloma.

S. Vincent Rajkumar, and Jean Luc Harousseau. Blood 2016;128:2757-2764

©2016 by American Society of Hematology

VCD or VTD instead or VRD

Limit maintenance to 1-2 years

Consolidation instead of maintenance

Attal M et al. N Engl J Med 2017;376:1311-1320.

VRD

Early versus Delayed ASCT

2. Recognize how US drug prices matter worldwide

• Global cancer drug spending

• >US$ 100 billion each year

• United States is nearly half (46%) of this market

Modified from: Prasad V, Dejesus K, and Mailankody S. Nature Reviews Clinical Oncology, 2017.

$ 100 billion

Why?

? France

3. Generate Careful, Powerful Guidelines

Why what we say matters

Drug Bortezomib Carfilzomib 27/m2 Carfilzomib 56/m2

ASP 50,000 130,000 260,000

6% (Doctors office) 3000 7800 15,600

Why what we say matters

Drug VCd VRd Dara‐VRD

ASP 60,000 220,000 340,000

6% (Doctors office) 3600 13,200 20,400

National Guidelines combined with the US reimbursement system makes it easy to justify more expensive option

Ease of Off-Label Use facilitates this

What can we do

•Strict guidelines – true metric of benefit•Regulatory Trials ≠ Practice Changing

•Careful with words like “Standard of Care”

4. Conduct Strategic Trials

Strategic Trials

• Response Directed Therapy• Curative Strategies (CESAR, ASCENT)

• Limited Duration Therapy• Targeted Trials

6-12 months and STOP should be our long-term Goal

MAF MMt(14;16) (C-MAF) t(14;20) (MAF-B)

TrisomicMM

t(4;14) MM

Kumar S, Rajkumar SV. Nature Rev Clin Oncol 2018

t(11;14) MM

Tefferi, Kantarjian, Rajkumar et al. Mayo Clinic Proceedings 90: 996; Jul 2015

5. Advocacy

1. Access2. Doctor vs Expert Role

CHALLENGES

For more info: @VincentRK on Twitter