Allan Grill, MD, CCFP, MPH Family Physician Member, pERC

Mona Sabharwal, BScPhm, Pharm. D., R.Ph.,

Executive Director, pCODR

Nianda Penner, BSc(Pharm), R.Ph., Knowledge Management, pCODR

CADTH Symposium, Concurrent Session B1 Monday, April 13, 2015

Disclaimer

•  I have no financial conflicts of interest to declare •  I receive a per diem remuneration for work

associated with CED and pERC membership •  The opinions expressed in this presentation

reflect the presenters own personal experiences with Health Technology Assessment and public drug policy, and do not represent the opinions of other pERC members, pCODR, CADTH, the Ontario Public Drug Programs, Cancer Care Ontario, or the Ontario Ministry of Health and Long-Term Care

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•  To review the discordance rates associated with pERC recommendations and provincial cancer drug funding decisions

•  To outline the potential reasons for discordance in the context of Health Technology Assessment (HTA)

•  To present the results of a national survey among drug funding decision makers outlining the challenges and potential solutions towards enhancing alignment for Canada’s national cancer drug review process

OBJECTIVES

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•  pCODR/CADTH Overview: §  Health Technology Assessment §  National, evidence-based cancer drug review process §  Consistency & clarity via Deliberative Framework à legitimacy §  Process should guide provinces in drug-funding decision making

Background

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Background

•  pERC recommendations are meant to: §  Achieve best possible health outcomes §  Contribute to health system sustainability §  Promote equity across provinces

•  Is there discordance between recommendations and decisions?

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•  A state of disagreement and disharmony

What is Discordance?

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As of December 31, 2014, pERC had issued 39 final recommendations:

•  7 (18%) positive recommendations •  24 (61%) conditional recommendations •  8 (21%) negative recommendations

pERC Final Recommendations

18%  

61%   21%  

Positive Recommendation

Conditional Recommendation

Negative Recommendation

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Of the funding decisions made, percentage of those funding decisions that are in concordance with the pERC recommendations:

Rate of Concordance

75  

80  

85  

90  

95  

100  

BC   AB   SK   MB   ON   NB   NS   PEI   NL  

%  

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pERC Recommendations to Fund

1.  Pazopanib (Votrient) for metastatic renal cell carcinoma [Jan 20, 2012]

2.  Bendamustine hydrochloride (Treanda) for NHL [Dec 14, 2012]

3.  Axitinib (Inlyta) for metastatic renal cell carcinoma [Apr 11, 2013]

4.  Bortezomib (Velcade) for multiple myeloma, pre-ASCT [Apr 11, 2013]

5.  Pazopanib (Votrient) resubmissions for metastatic renal cell carcinoma [Sep 16, 2013]

6.  Afatinib (Giotrif) for non-small cell lung cancer [May 20, 2014]

7.  Arsenic trioxide (Trisenox) for Acute Promyelocytic Leukemia [Mar 5, 2014]

0%

10%

20%

30%

40%

50%

60%

70%

80%

90%

100%

1 2 3 4 5 6 7

Provincial Funding as of December 31, 2014

Funded Under Negotiation with Manufacturer

Under Provincial Consideration Not Funded

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pERC Recommendations to Not Fund 1.  Pazopanib (Votrient) for soft

tissue sarcoma [Dec 14, 2012]

2.  Bendamustine hydrochloride (Treanda) for CLL [Dec 14, 2012]

3.  Bortezomib (Velcade) for multiple myeloma, post-ASCT [Apr 11, 2013]

4.  Lapatinib (Tykerb) for breast cancer [Jul 22, 2013

5.  Regorafenib (stivarga) for metastatic colorectal cancer [Dec 2, 2013]

6.  Cetuximab (Erbitux0 for metastatic colorectal cancer [Jan 27, 2014]

7.  Aflibercept (Zaltrap) for metastatic colorectal cancer [Sep 22, 2014]

0%

10%

20%

30%

40%

50%

60%

70%

80%

90%

100%

1 2 3 4 5 6 7

Provincial Funding as of December 31, 2014

Funded Under Negotiation with Manufacturer

Under Provincial Consideration Not Funded

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pERC Recommendations to Fund on Conditions

0  

10  

20  

30  

40  

50  

60  

70  

80  

90  

100  

1   2   3   4   5   6   7   8   9   10   11   12   13   14   15   16   17   18   19   20   21   22   23   24  

Provincial  Funding  as  of  December  31,  2014  

Funded   Under  NegoMaMon  with  Manufacturer   Under  Provincial  ConsideraMon   Not  Funded  

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•  So we know there is some discordance.

•  What are the potential reasons for discordance between pERC and the provinces ?

•  Do challenges exist that undermine pERC’s impact on policy-makers leading to discordance?

•  Can these challenges be overcome?

More Questions

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•  Hypothesized reasons for discordance §  pERC member/HTA perspective

•  Designed an on-line survey •  Survey issued to participating provincial ministries of health and

cancer agencies •  Survey period March 4, 2015 to April 1, 2015 •  4 questions: Multiple choice + Free text

§  Choose answers based on our developed hypotheses §  List challenges that limit the effectiveness of HTA processes §  List solutions that enhance alignment between pERC

recommendations and provincial drug funding decisions •  Anonymity respected •  100% response rate

Methods

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1. If your province has made or is to make a decision to fund a drug where the recommendation from the HTA review is to not fund, what are the reasons for funding?

Survey Responses

54.5  

36.4  

63.6  

27.3  

Comparators  in  trial  not  funded  by  province  

Comparators  in  trial  not  relevant  to  pracMce  

PoliMcal  pressure  

High  tumour  group  priority   %

%

%

%

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Other Reasons for decision to fund where the recommendation from the HTA review is not to fund: •  Rarity of cancer

§  Low probability of future clinical trials

•  Exceptional access – patient sub-groups (e.g. no other treatment options)

•  Risk-sharing agreement/pay for performance agreement §  If no patient benefit, then no cost to the province

•  “Under consideration” may mean compassionate case-by-case funding

Survey Responses

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2. If your province has made or is to make a decision to not fund a drug where the recommendation from the HTA review is to fund (with or without conditions), what are the reasons for not funding?

Survey Responses

9.1%  

18.2%  

63.6%  

27.3%  

90.9%  

Disagreement  with  the  economic  review  

Disagreement  with  the  clinical  review  

Drug  was  not  a  priority  for  local  tumour  group  

PaMent  populaMon/disease  not  treated  in  province  

Budget  constraint    

%  

%

%

%

%

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Other Reasons for decision not to fund where the recommendation from the HTA review is to fund: •  Multiple choices/lines of therapy already exist (no therapeutic gap) •  Budget impact analysis (not always clear for every province in pERC

review) •  Provincial budgets do not align with the HTA process, particularly in

a fiscally constrained year •  Unsuccessful negotiation with manufacturers •  Uncertainty – may require additional information

Survey Responses

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3. If your province makes a funding decision to fund a drug prior to completion of the HTA review, what would be the reason(s)?

Survey Responses

%

%

%

%

42.9%  

57.1%  

28.6%  

Local  tumour  group  priority  

PoliMcal  pressure  

High  disease  burden  in  the  province   %  

%  

%  

%  

%  

%  

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Other reasons for funding prior to HTA recommendation: •  Evidence shows survival advantage

§  Ethical challenge not to fund

•  Pressure re: patient access to clinical trials •  Overwhelming clinical need •  Expanded eligibilities not mentioned in pERC review

§  other lines of therapy, indication creep

Survey Responses

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Challenges and barriers that may limit evidence-based recommendations: •  Quality of clinical trails (design, outcomes measures) •  Standard of care in other jurisdictions (e.g. comparators not available in

Canada) •  Re-interpretation of evidence not being studied in a clinical trial

(tumour groups) •  Trial population not generalizable (e.g. too wide vs. too narrow) •  Technology constraints (f/u trial protocols may be easier to follow in

some jurisdictions) •  Too much dependence on manufacturer for submissions

•  Would prefer more tumour group submissions but resources scarce •  pERC’s adherence to evidence-based patient eligibility criteria

•  Provinces feel pressure to expand eligibility due to demands

Survey Responses

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Potential solutions to enhance alignment of funding decisions with HTA recommendations: •  Pan Canadian Pricing Alliance •  Quicker response from pCODR •  More tumour group input on priorities •  pERC to be more clear on prioritization with each recommendation •  National Drug Program •  PAG should be more aligned due to their involvement in the pERC

process •  Funding decisions may suggest otherwise •  Wording of same decisions varies province to province

•  Strengthen national consensus on treatment pathways and add to pERC’s clinical review

Survey Responses

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•  Some discordance should be expected regarding HTA recommendations §  Majority associated with conditional recommendations

•  Overlap exists between HTA challenges and solutions §  Trade-off between being too strict and too lenient (e.g. eligibility

criteria) §  Trade-off between evidence-based framework vs. local interests

•  Discordance may lead to a lack of consistency in decision making •  Discordance may lead to a lack of transparency •  Uncertainty requires more data, and priorities could be shifted to

achieve this •  Consider HTA committee performance review to enhance

accountability

Take Home Messages

Thank you

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