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Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert

Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

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Page 1: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

Patient prioritization in disease specifictreatment budgets

Lauren E. Cipriano

Jeremy D. Goldhaber-Fiebert

Page 2: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

Funding, Disclosure, Acknowledgments

• Funding• Natural Sciences and Engineering Research Council of Canada

• No conflicts to disclose

• Thank you to Eldon Spackman for comments on an early draft

Page 3: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

The usual story

• ICER =∆ Cost

∆QALY

• ICER > 𝜆 Not cost-effective

• ICER ≤ 𝜆 Is cost-effective…

…now how will we find the $ to buy it…

Tota

l Hea

lth

Sys

tem

Bu

dge

t

Cost-saving

0-10K/QALY

10-20K/QALY

20-30K/QALY

30-40K/QALY

40-50K/QALY

Budget

Page 4: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

The usual story

• ICER =∆ Cost

∆QALY

• ICER > 𝜆 Not cost-effective

• ICER ≤ 𝜆 Is cost-effective…

…now how will we find the $ to buy it…

Tota

l Hea

lth

Sys

tem

Bu

dge

t

Budget

Page 5: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

The usual story

• ICER =∆ Cost

∆QALY

• ICER > 𝜆 Not cost-effective

• ICER ≤ 𝜆 Is cost-effective…

…now how will we find the $ to buy it…

Tota

l Hea

lth

Sys

tem

Bu

dge

t

Budget

Page 6: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

Tota

l Hea

lth

Sys

tem

Bu

dge

tThe usual story

• ICER =∆ Cost

∆QALY

• ICER > 𝜆 Not cost-effective

• ICER ≤ 𝜆 Is cost-effective…

…now how will we find the $ to buy it…

BudgetMay take years to divest

Budget impact may be

temporary

Page 7: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

Restricted capacity disease specific treatment budgets

• AIDS Drug Assistance Programs (ADAPs)• Established in 1987 for low-income and under-insured

• Exclusively provides funding for HIV treatment

• Discretionary US federal and state funding

• Budget shortfalls• More restrictive eligibility (income, disease severity)

• Cost sharing

• Reduce number of medications available

• Waiting lists (2008-2013)• First-come first-served or Priority based on disease severity

Treatment is cost effective for everyone: Who should go first?

Page 8: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

F0 F1 F2 F3 F440-4445-4950-5455-5960-6465-6970-7475-79

Hepatitis C treatment is cost effective

$20,000 – $25,000 per QALY gained

$25,000 – 50,000 per QALY gained$50,000 –100,000 per QALY gained

<$20,000 per QALY gained

Treating 20% of the treatment eligible population = $20-25 billion annually (25% of Medicare Part D)

Page 9: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

The research question

• Treatment is ‘cost-effective’ for everyone but budget impact is overwhelming

• At least for a transitional phase, policy is to restrict access to manage budget impact… but, who should be prioritized for treatment?

•Should patient subgroups be prioritized on ICERs?

Page 10: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

Stage 1 Stage 2

𝒑𝟏 𝒑𝟐

𝑩𝟏 𝑩𝟐 > 𝟎≫

𝒓𝟏 𝒓𝟐≥

Incr. net monetary benefit(Treat now vs. never)

Period reward, if wait

𝑵Number of people 𝑵

Dead(not trt elig.)

Two time periods (now and later) with N treatments available at each period,who should get treatment first?

TheModel

Page 11: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

Time 1 Time 2

Lower ICER goes first 𝐵1 + 𝑟2 1 − 𝑝2 𝐵2𝛿 1 − 𝑝2 𝐵2

Higher ICER goes first 𝐵2 + 𝑟1 1 − 𝑝1 𝐵1 + 𝑝1𝐵2𝛿 1 − 𝑝1 𝐵1 + 𝑝1𝐵2

Total Treatments

2𝑁 − 𝑝2𝑁

2𝑁

Lower ICER goes first

Higher ICER goes first

> ?

𝐵1 + 𝑟2 + 𝛿 1 − 𝑝2 𝐵2 > 𝐵2 + 𝑟1 + 𝛿 1 − 𝑝1 𝐵1 + 𝑝1𝐵2

Page 12: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

Lower ICER goes first

Higher ICER goes first

>

(𝐵1−𝐵2)(1 − 𝛿) − (𝑟1 − 𝑟2) + 𝛿 𝑝1 𝐵1 − 𝐵2 − 𝑝2𝐵2 >0

+ve, small𝛿 ≈ 1

Benefit lost when Stage 1 Stage 2 (because of delay)

-ve, smallBenefit lost when Stage 2 Dead(because of delay)

Page 13: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

𝐵1 − 𝐵2𝐵2

>𝑝2𝑝1

Ratio of the progression probabilities

Relative gain in INMB

from treating at Stage 1

Priority to lower ICER• Large difference in ICERs

• Low rate of out of Stage 2• People are unlikely to become

treatment ineligible

• High rate of out of Stage 1• Fleeting opportunity for early

high-impact intervention

>

Page 14: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

𝐵1 − 𝐵2𝐵2

<𝑝2𝑝1

Priority to higher ICER!• Small difference in ICERs

• High rate of out of Stage 2• People are going to become

ineligible for cost-effective treatment!

• Low rate of out of Stage 1• Slowly progressing disease

• People can wait for treatment with little health consequence

Ratio of the progression probabilities

Relative gain in INMB

from treating at Stage 1

<

Page 15: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

What if you had (much) less than N treatments available each period?

• Prioritize lower ICER when

(𝐵1−𝐵2)(1 − 𝛿) − (𝑟1 − 𝑟2) + 𝛿 𝑝1 𝐵1 − 𝐵2 − 𝑝2𝐵2 >0

If the de-prioritized group will have long treatment delay,Large difference in ICER Prioritize lower ICER

Page 16: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

Does it make a difference in real life?Priority on ICER

Optimize on NMB

F0 F1 F2 F3 F4

40-44 6 5 3 0 0

45-49 6 4 2 0 0

50-54 6 5 3 0 0

55-59 7 6 3 0 0

60-64 7 5 4 1 0

65-69 7 6 4 1 2

70-74 7 6 7 5 8

75-79 7 7 7 6 8

F0 F1 F2 F3 F4

40-44 5 0 0 0 0

45-49 5 0 0 0 0

50-54 5 0 0 0 1

55-59 7 0 1 1 3

60-64 8 2 3 2 4

65-69 9 5 5 5 7

70-74 9 7 7 6 8

75-79 9 8 8 8 8

ICER Opt.

Within 5 years

Within 10 years

ESLD and cancer

ICER Opt.

QALYs

Page 17: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

Another way to think about it

• The mutually exclusive collectively exhaustive set of decision alternatives is a complete set of times until each subgroup will be prioritized

F0 F1 F2 F3 F440-44 6 5 3 0 0

45-49 6 4 2 0 0

50-54 6 5 3 0 0

55-59 7 6 3 0 0

60-64 7 5 4 1 0

65-69 7 6 4 1 2

70-74 7 6 7 5 8

75-79 7 7 7 6 8

Page 18: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

All immediately

Severityonly

F0 F1 F2 F3 F4

40-44 5 3 3 1 0

45-49 5 3 3 1 0

50-54 5 3 3 1 0

55-59 5 3 3 1 0

60-64 5 3 3 1 0

65-69 5 3 3 1 0

70-74 5 3 3 1 0

75-79 5 3 3 1 0

Page 19: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

Severityonly

All immediately

Page 20: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

Severityonly

All immediately

Priority on ICER

Maximizes NMB s.t. Annual Treatment Budget

ICER

Optimize on NMB

F0 F1 F2 F3 F4

40-44 3 3 0 0 0

45-49 3 0 0 0 0

50-54 4 0 0 0 0

55-59 4 0 0 0 0

60-64 4 2 0 0 0

65-69 4 3 2 1 1

70-74 4 3 2 3 2

75-79 4 3 3 3 3

F0 F1 F2 F3 F4

40-44 0 0 0 0 0

45-49 1 0 0 0 0

50-54 2 0 0 0 0

55-59 2 0 0 0 0

60-64 4 0 0 0 1

65-69 5 2 2 2 3

70-74 5 3 3 3 4

75-79 5 4 4 4 4

Page 21: Patient prioritization in disease specific treatment budgets · Patient prioritization in disease specific treatment budgets Lauren E. Cipriano Jeremy D. Goldhaber-Fiebert. Funding,

Conclusion

• Health care budgets should not always be allocated in order of ICER

• How long will the wait be? Consequences of waiting?

• Maximizing NMB for all individuals s.t. the annual budget constraint incorporates • relative benefit of treatment

• consequences of waiting

• time until resources available to expand treatment access