B4 - Are you listening to me? - Wong-Rieger - Salon D

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%


Same29%

Better0%

Other0%

Cost-effectiveness (more, less, certain)

Same43%

Less costly57%

Other0%


Not confirmed0%

Not addressed14%

Other0%


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*


Same33%

Better60%

Other6%


Same3%

Better7%

Other0%


Same20%

Less costly7%

More costly63%


Same17%

Better30%

Other0%


Included60%

Not addressed20%

None20%


Reasons Cited in “Do Not LIST” Recommendations (2011-13)

N=33* N=33* N=33*


Same15%

Insufficient/Uncertain/No

Comparator72%

Failed/Wrong

12%


Uncertain3%

Fewer6%

Worse9%


Uncertain33%

Less costly0%

More costly39%


Not confirmed21%

Not addressed18%

Included12%


Included27%

Not confirmed/Not

studied51%

None33%


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

[email protected]

http://www.consumeradvocare.org

Health & Medicine

B4 - Are you listening to me? - Wong-Rieger - Salon D