CADTH_2014_D3_Sending_the_Right_Signals__How_Can_HTA_Optimally_Inform_PLAs_in_the_Context_of_pCPA__Mona Sabharwal

Sending the Right Signals: How Can HTA Optimally Inform PLAs in the context of pCPA

Mona Sabharwal, BScPhm, PharmD

Executive Director

pan-Canadian Oncology Drug Review

April 8, 2014

Building linkages to pCPA

• pCODR provides a more predictable, regularized entry point for

new drugs/new uses to be evaluated for public funding,

including non-commercially generated data

• pCODR’s commitment to transparency, need to be accountable

to patients and the public, allow for multiple perspectives on

value to be elicited and shared

• Adoption feasibility discussion systematically solicited and

incorporated into reviews

• By having singular evidence-base for recommendations,

provides clearer and more consistent direction on areas of

uncertainty which could be used for negotiation purposes

2 Copyright pan-Canadian Oncology Drug Review

National context for pCODR

• Cancer is increasingly viewed as a serious, life threatening but

chronic disease

• Number and pace of new treatments available rising fast

• National differences in structures and processes for review and

funding of cancer drugs

• Variation in use/acceptability of pharmacoeconomic information,

submission requirements, roles/expectation for manufacturers

• Variation in coverage across jurisdictions

• Frustration amongst patients and stakeholders


Key lessons from national collaborative efforts

• Ownership, involvement and support of cancer agencies is critical

• Transparency of process for all stakeholders (ministry, agency,

patients, manufacturers) is essential

• Cancer control community ready for an evidence-based review

process:

• strong culture of evidence generation (NCIC and others)

• clinical guidelines and protocol-based care well established

• systems for data capture exist or are being created

• Economic evaluation a necessary part of any HTA process

• significant need for capacity enhancement across spectrum

of generators and users of this type of information in

cancer control system

4

Copyright pan-Canadian Oncology Drug Review

About pCODR

• pCODR was created in recognition of certain unique characteristics of how

cancer care is organized and delivered in Canada

• Implemented in 2010 to:

assess cancer drugs and makes recommendations to provinces and territories to

guide their drug funding decisions by ensuring that all provinces and cancer

agencies benefit from a single, clear approach to new cancer drug evaluation

leverage best practices and expertise from across Canada to provide provinces

and territories with the best possible information on which to base their funding

decisions

bring consistency and clarity to assessment of cancer drugs by looking at clinical

evidence, cost-effectiveness and patient perspectives

• Transitioned governance under CADTH as of April 1, 2014


pCODR Review Process Updated March 31, 2011

1. Conduct

Pre-

Submission

Planning

activities

including

getting

input from

PAG and

notifying

Patient

Advocacy

Groups

2. Prepare

& submit

Request

for Drug

Review

4.2

Conduct

Economic

Review

5.

Summarize

& Review

with pERC

6. Prepare &

Publicly

Post Initial

Recomm,

Post

Reviews

8. Summarize

& Review with

pERC

3.1 Screen

Submission

and Initiate

Review

Process

End‡

Ind

us

try/

Tu

mo

ur

Gro

up

p

CO

DR

*

Variable 5 business days 70-90 business days 12 business days 10 business days 20 business days

7.1 Get

Feedback

from

Submitter

(and impacted

manufacturer)

7.3 Get

Feedback

from Patient

Advocacy

Group

7.2 Get

Feedback

from PAG

Pati

en

t A

dv

ocac

y

Gro

up

s

9. Prepare &

Publicly Post

Final

Recomm &

Post Input

12 business days

*Includes pCODR Secretariat, Clinical

Guidance Panel, Economic Guidance Panel,

pCODR Expert Review Committee (pERC)

and Provincial Advisory Group (PAG)

4.1.1/4.2.2

Clarify info

with

Submitter

during

review

4.1

Conduct

Clinical

Review

3.2 Collect

Patient

Advocacy

Group

Input

Estimated

99 – 149

business days

7.4

Eligible for

Early

Conversion?

No Yes

‡Next steps could include

Recommendation implementation,

Procedural Review or Resubmission

6 © 2013 pan-Canadian Oncology Drug Review

pERC Final Recommendation

7

pERC has issued 29 final recommendations as of

December 31, 2013

5 (17%) positive recommendation without

conditions

18 (62%) conditional recommendation*

(*subject to improve cost-effectiveness)

6 (21%) negative recommendation

17%

62% 21%

Column1 PositiveRecommendation

ConditionalRecommendation

NegativeRecommendation

Drug Name & Indication pCODR Recommendation

Pazopanib hydrochloride (Votrient) Metastatic Renal Cell Carcinoma

Recommend

Sunitinib malate (Sutent) Pancreatic Neuroendocrine Tumours

Recommend with conditions

Ipilimumab (Yervoy) Advanced Melanoma


Vemurafenib (Zelboraf) Advanced Melanoma


Eribulin mesylate (Halaven) Metastatic Breast Cancer


Everolimus (Afinitor) Pancreatic Neuroendocrine Tumours


Crizotenib (Xalkori) Advanced Non-Small Cell Lung Cancer

Do Not Recommend

Bendamustine hydrochloride (Treanda) Chronic Lymphocytic Leukemia (relapsed/refractory)

Do Not Recommend

Bendamustine hydrochloride (Treanda) Non-Hodgkin Lymphoma and Mantle Cell Lymphoma

Recommend

Pazopanib hydrochloride (Votrient) Soft Tissue Sarcoma (STS)

Do Not Recommend

Ruxolitinib (Jakavi) Myelofibrosis


Bendamustine hydrochloride (Treanda) Chronic Lymphocytic Leukemia (first-line)


Axitinib (Inlyta) Metastatic Renal Cell Carcinoma

Recommend

Everolimus (Afinitor) Advanced Breast Cancer


Bortezomib (Velcade) Multiple myeloma pre ASCT

Recommend

Bortezomib (Velcade) Multiple myeloma post ASCT as monotherapy

Do Not Recommend

Crizotinib (Xalkori) - Resub Advanced Non-Small Cell Lung Cancer


Lapatinib (Tykerb) in combo with letrozole Do Not Recommend

Enzalutamide (Xtandi) Recommend with conditions

Pertuzumab (Perjeta Herceptin) Recommend with conditions

Pazopanib (Votrient) Resubmission Recommend

Brentuximab (Adcetris) - HL Recommend with conditions

Regorafenib (Stivarga) - mCRC Do Not Recommend

Abiraterone (Zytiga) - mCRP Recommend with conditions

Lenalidomide (Revlimid) – Multiple Myeloma Recommend with conditions

Trametinib (Mekinist) – Metastatic Melanoma Recommend with conditions

Dabrafenib (Tafinlar) – Metastatic Melanoma Recommend with conditions

Pemetrexed disodium (Alimta) – Advanced or Metastatic Non-Squamous Non-Small Cell Lung Cancer Recommend with conditions

Brentuximab (Adcetris) - SALCL Recommend with conditions

Copyright pan-Canadian Oncology Drug Review

pERC Recommendations

• pERC recommendations help guide funding decisions; final

funding decisions remain responsibility of each participating

jurisdiction

• Funding recommendations are not static - they are context

specific, such as:

• evidence available at that point in time

• existing programs and policies – who is covered, what is/is

not covered

• basket of currently available and/or funded treatment

options

• current pricing arrangements


pERC Recommendations

Recommend

• A drug with a clear clinical benefit and economic benefit

Consider with Conditions

• Provides context and describes conditions under which a specific

jurisdiction may or may not want to fund the drug

• These conditions would relate to issues that directly change the

efficacy or cost-effectiveness of the drug

• Factors or conditions to consider could include utilization

patterns, funding of comparators, availability/accessibility of

other options

Do Not Recommend

• No reason to recommend identified during pERC deliberations


Building linkages to pCPA

• pCODR provides a more predictable, regularized entry point for

new drugs/new uses to be evaluated for public funding,

including non-commercially generated data

• pCODR’s commitment to transparency, need to be accountable

to patients and the public, allow for multiple perspectives on

value to be elicited and shared

• Adoption feasibility discussion systematically solicited and

incorporated into reviews

• By having singular evidence-base for recommendations,

provides clearer and more consistent direction on areas of

uncertainty which could be used for negotiation purposes


Common Sources of Uncertainty in pCODR reviews

• Clinical uncertainty

need, burden of illness, practice patterns, magnitude of benefit

• Statistical uncertainty

cross over, non-inferiority

• Uncertainty in patient values

QoL, preferences and priorities

• Economic uncertainty

assumptions due to lack of clinical, epidemiological and

utilization data

• Structural/systems uncertainty

ability to deliver and integrate into systems, affordability


Time of launch evaluation (current HTA focus)

• Characterize uncertainty

which parameters are most uncertain e.g., surrogate marker,

magnitude of benefit

what are the drivers for that uncertainty e.g., clinical use

assumptions

what could help reduce uncertainty e.g., companion testing

for managing eligibility, marker for response

what will mitigate some risks e.g., controlled distribution

and delivery

• Challenge – what to actually do about uncertainty and

when


Post-launch evaluation (future HTA focus?)

• Coverage with evidence development

help define questions of relevance for specific drug and

disease instance

• Performance / Real world effectiveness

advice on what to measure and when

• Disinvestment opportunities

identify potentially obsolete therapies in favour of newer

option

• Challenges: simplicity, transferability, measurability


Health & Medicine

CADTH_2014_D3_Sending_the_Right_Signals__How_Can_HTA_Optimally_Inform_PLAs_in_the_Context_of_pCPA__Mona Sabharwal