Upload
cadth-symposium
View
145
Download
0
Embed Size (px)
DESCRIPTION
Citation preview
Are You Listening to Me? Patient Input on Drug Reviews
Durhane Wong-Rieger, PhDChair, Consumer Advocare
Network
1Thanks to Janssen for some of the slides and analysiis
What We Say … What You Hear
Drug slows disease progression, relieves symptoms, pain, ability to perform daily activities (including continuing or returning to work), quality of life, improved sleep patterns, and restored libido.
Fear of fractures have important impact on patients’ quality of life
Patients described eye symptoms as gritty, sore, burning, painful…with examples of quality of life affected by reduced ability to read, watch television, drive, and outside activities … adverse effects of ophthalmic corticosteroids
The majority of i symptoms identified by patient groups are included in SF-36 and HAQ-DI. No data related to restored libido or improved sleep patterns were captured.
Patient outcome fracture in only one study; pain part of QoL scales
No DB RCTs w/Moderate DED; post hoc not clinically relevant outcomes
Oct 2011Institute for Optimizing Health Outcomes 2
What We Say … What You Hear
Lupus decreases QoL and ability to work; prednisone has SAE, IV therapy tolerable
impact on QoL and family, need for alternative to onerous therapy; reduce hospitalizations
Chronic warfarin affect OoL: fear of bleeding; INR monitoring; potential drug, food, and alcohol interactions
CAPS symptoms: rashes, joint pain and stiffness, conjunctivitis; LT kidney failure, deafness, blindness, arthritis, learning disabilities; 1st therapy with CTs; off-label daily injection painful, SAE
LT effects ns; variable trial outcomes; no QoL or prednisone reduction (pat imp)
RCTs only with placebo; patient unmet need for alternatives; 25% failure; too costly
Noninferior to warfarin; No QoLstudies (not designed to measure); cheaper than dabigatran
CTs: less disease flare but QOL not statistical; lack lifelong benefits data; other than rash no patient benefits submitted
Oct 2011Institute for Optimizing Health Outcomes 3
I ASK ABOUT:
Options, what might work, be
more manageable, fewer side
effects, give more time
Range of alternatives to meet
individual patient needs, values;
QoL ≠ # or $
Individualized access based on
optimal benefit to patient and
family
Trade off quality for quantity
Is Anyone Listening?
YOU ASK FOR:
Scientific evidence, RCTs
against SOC, LT outcomes
(SAE, morbidity, mortality)
Lowest cost alternatives based
on relative benefits, projected
cost and use, $/QALY
Tiered population-based access
based on lowest acceptable
benefit
Trade off quality fo quantity
4
What Does Listening Look Like?
Decisions reflecting patient perceived needs
and wants; more options allowing for more
individualized choices
Feedback that shows understanding of
patient values and willingness to act on
patient desired outcomes
Dialogue: opportunity to be heard, to provide
feedback, and to influence decisions and
policy
5
CDR Recommendations (2007-2013)
List/Conditi
on49%
Not List51%
2007-2010
List/Conditi
on53%
Not List47%
2011-2013
CDR Recommendations Little CHANGE
Since Patient Submissions
12%
41%
47%
11%
42%
47%
List List Conditional Not List
Pre Feb 2012 (n=34) Post Feb 2012 (n=36)
7
% List Recommendations by With/No Patient Submission
10% 8%
14%
43%
49%
29%
47%43%
57%
0%
10%
20%
30%
40%
50%
60%
All (n=70) With Patient Submission (n=49)
No Patient Submission (n=21)
List List Conditional Not List
5/6/20138
%With/No Patient Submission by
List Recommendation
43%
20%
36%
30%
57%
80%
64%
70%
0% 20% 40% 60% 80% 100%
List
List Cond
Not List
All
With Patient Submission No Pat Submission
9
CDR Recommendations (2011-13)
Dec/2010-April/2013)
ALL%(n=70)
With Patient Submission
%(n=49)
No Patient Submission
%(n=21)
Overall100% (70) 70% (49/70) 30% (21/70)
List10% (7/70) 8% (4/49) 14% (3/21)
List Conditional43% (30/70) 49% (24/49) 29% (6/21)
Not List47% (33/70) 43% (21/49) 57% (12/21)
Average # Patient Submissions per
Type of Recommendation
1
2.52.3 2.3
0
0.5
1
1.5
2
2.5
3
List List Cond Not List All
2011-13 (n=49)
11
CDR (2011-13) Types of Reasons Cited in Recommendations
List(n = 7)
List Conditional(n = 33)
Not List(n = 30)
Clinical Trial Outcomes(effectiveness, sufficient, similar, certain)
100% 91% 88%
Safety (adverse effects, similar risks, certain)
29% 10% 18%
Cost-effectiveness(more, less, certain)
100% 90% 72%
Quality of Life (studied, better, specific)
14% 47% 61%
Patient submissions (reference, substantiated
29% 60% 48%
Reasons Cited in LIST Recommendations (2011-13)
N=7* N=7* N=7*
Clinical Trial Outcomes(effectiveness, sufficient,similar, certain)
Same100%
Better0%
Other0%
Safety (adverse effects, similar risks, certain)
Same29%
Better0%
Other0%
Cost-effectiveness (more, less, certain)
Same43%
Less costly57%
Other0%
Quality of Life (studied, better, specific)
Not confirmed0%
Not addressed14%
Other0%
Patient submissions (reference, substantiated
Not substantiated29%
Not addressed14%
None43%
* % May not add to 100% since not all recommendations cited all reasons
Reasons Cited in “LIST with Conditions” (2011-13)
N=30* N=30* N=30*
Clinical Trial Outcomes(effectiveness, sufficient,similar, certain)
Same33%
Better60%
Other6%
Safety (adverse effects, similar risks, certain)
Same3%
Better7%
Other0%
Cost-effectiveness (more, less, certain)
Same20%
Less costly7%
More costly63%
Quality of Life (studied, better, specific)
Same17%
Better30%
Other0%
Patient submissions (reference, substantiated
Included60%
Not addressed20%
None20%
* % May not add to 100% since not all recommendations cited all reasons
Reasons Cited in “Do Not LIST” Recommendations (2011-13)
N=33* N=33* N=33*
Clinical Trial Outcomes(effectiveness, sufficient,similar, certain)
Same15%
Insufficient/Uncertain/No
Comparator72%
Failed/Wrong
12%
Safety (adverse effects, similar risks, certain)
Uncertain3%
Fewer6%
Worse9%
Cost-effectiveness (more, less, certain)
Uncertain33%
Less costly0%
More costly39%
Quality of Life (studied, better, specific)
Not confirmed21%
Not addressed18%
Included12%
Patient submissions (reference, substantiated
Included27%
Not confirmed/Not
studied51%
None33%
* % May not add to 100% since not all recommendations cited all reasons
Patient Groups Are Making
Submissions
Patient group submissions = 70%
Average # submissions = 2.3 (range 1 to 9)
Number of submissions not as important as quality
All drugs for rare diseases have patient submissions
Conditions least likely to have submissions: diabetes, cardiovascular, pain, general (allergy, overactive bladder, skin rash)
What Did Patients Cite?
Impact of Condition
Quality of Life: 63%
Work or finances: 16%
Family or caregivers: 14%
Morbidity: 6%
Reasons for New Treatment
Adverse effects of current: 49%
Better symptom management: 39%
Need for alternatives: 35%
Improve Quality of Life: 33%
Improve convenience or management: 33%
Improve Adherence: 16%
Willingness to accept AE: 10%
Reduce disease progression: 8%
Patient Submissions => “List
Conditional” vs. “Do Not List”
Patient submissions related to “List Conditional”
recommendation
Little difference in “list” and patient submissions (8-
14%)
More “list with conditions” with patient submissions
(49% vs. 29%)
Fewer “do not list” with patient submissions (43%
vs. 57%)
List conditional vs. Not list
Not List: 64% with patient submission
List Conditional: 80% with patient submission
Patient Submissions: Possible Role in “List/Do Not List”
Patient submission NOT key factor
List: 100% cite CT outcomes and cost-effectiveness
Do Not List: Based on CT outcomes, cost-effectiveness, QoL
(lack data)
List Conditional: Reference CT outcomes, cost-effectiveness,
patient submission
Patient submissions influence when confirmed by
data
List Conditional: 60% confirm patient submission
Do Not List: 51% do not study or confirm patient submission
How to Improve Listening…
Clarify “What do you want to hear?” How will patient
submissions be integrated into HTA evaluation?
Open the dialogue: Not just information in and decisions out
but collaboration to get best common outcome.
Engage patients in CT design to ensure patient values are
included in measures.
Demystify HTA by training potential HTA patient-public
members on technical processes of HTA and decision making
Provide means for patient-public members of HTA
committees to dialogue with patient representatives
Train all HTA committee members on methods for integrating
qualitative information
Promote transparent decision-making (records of deliberation
as well as outcomes); open meetings.
20
Contact:
Durhane Wong-Rieger
Consumer Advocare Network
www.consumeradvocare.org
416-969-7435