Assis$ng’PaentChoices:’...

Assis$ng Pa$ent Choices: Autonomy, Paternalism, or Something In

Between? Richard S. Bedlack MD PhD MSc FAAN

Duke ALS Clinic, Durham NC Durham VAMC, Durham NC

Disclosures: Paid speaker for Pfizer, Lilly, Avanir, Athena; Paid advisor for Avanir, Athena, Biogen, UCB, Sanofi, Medacorp, Easton and Guidepoint; Research Grants from Biogen, Neuraltis, Cytokinetics, Packard Center, MNDA

Outline •  Case

– Pa$ent choices that bothered me • Why? •  How common are these choices?

•  Models for Assis$ng Pa$ent Choices – Paternalis$c, autonomous, “in between” (consumerism, shared decision-‐making)

– Define, history, example, strengths and weaknesses

•  “Best” Model and How We Get There

Case •  60 year old university professor, newly diagnosed with

clinically definite sporadic ALS. She currently has minimal bulbar weakness, moderate arm and leg weakness. No obvious cogni$ve or behavior problems

•  Following educa$on about the disease itself, she is presented

with stage-‐appropriate, evidence-‐based management op$ons including riluzole, mul$-‐disciplinary team care

•  Neurology 2009;73:1218-‐1233

•  She is also asked about par$cipa$ng in a research study

Case

•  She opts for the first two, but declines the research study in favor of pursuing off-‐label oral sodium chlorite

Why These Choices Bothered Me

•  Lack of enrollment –  She herself is being deprived of the benefits of being in trials (hope, altruism, “doing beZer” medically)

–  Journal of Clinical Epidemiology 2001;54:217-‐224 –  InternaConal Journal of Gynecological Cancer 2009;19:124-‐128 –  Mov Disord 2012;27:506-‐511

–  Trials take longer, are more expensive, may be terminated without a defini$ve conclusion

–  Clinical Trials 2010;7:312-‐321

–  Trial results may not be generalizable –  Neurology 2011;77:1432

–  Unless this can be solved, it will take longer than it should to find a cure for ALS

Why These Choices Bothered Me

•  Pursuit of alterna$ve or off-‐label therapy (AOT) •  Poten$al for harms

•  Physical •  Financial •  Psychological •  Scien$fic

PLoS Med 2009; 6(2); E-pub 2009 Feb. 17

Are These Decisions Common?

Enrollment •  Less than 10% of PALS

enroll in research studies •  Literature review shows

that ALS trial enrollment rate is 2 pa$ents per site per month and not improving over $me –  ALS 2008;9:257-‐65

Pursuit of Alterna1ve or Off-‐Label Therapy (AOT) •  Survey of 350 PALS

–  50% responded –  54% admiZed using at least 1

AOT –  J Neurol Sci 2001;191:151-‐4

•  Survey of 177 ALS Clinicians –  23% responded –  50% stated they had pa$ents

using AOTs instead of par$cipa$ng in trials

–  ALS 2008;9:257-‐65

Models for Decision-‐Making in the Doctor Pa$ent Rela$onship

Paternalism-‐Defined

•  MD role: parent or guardian •  MD obliga$on: promo$ng pa$ent health and well-‐being (as defined by MD, independent of pa$ent preferences)

•  Pa$ent autonomy: none (assent)

•  Pa$ent values: objec$ve (and defined by MD)

JAMA 1992; 267: 2221-2226

Paternalism-‐History •  Common from the beginning of

medicine un$l approximately 1960’s

•  1847 AMA Ethical Code •  “The obedience of a pa$ent to the

prescrip$on of his physician should be prompt and implicit. He should never permit his own crude opinions as to their fitness, to influence his aZen$on to them. A failure on one par$cular may render an otherwise judicious treatment dangerous and even fatal.”

•  S$ll present at $mes (may even be requested by pa$ents)

•  Canadian Family Physician 2012:58:1194-‐1195

Paternalism-‐Example

•  MD: “You should par$cipate in the research study. This will give you the benefit of contribu$ng to the greater good, and there is some evidence that those who par$cipate may do beZer medically than those who do not. Research is the only way we will find a cure for ALS. I know of no good evidence that oral sodium chlorite will even be safe for you, much less help your ALS.”

•  PT: “Yes doctor, I will do as you say.”

Paternalism

Strengths •  Physicians have many years

of specialized training, experience, tools to cri$cally evaluate treatment op$ons according to efficacy and side effects

•  Lasted a long $me so there must be something about it that sa$sfies needs of both par$es

Weaknesses •  Pa$ent may not share same

goals, values, acceptable benefits and risks

•  Pa$ents may want more or less than the physician is willing to give at par$cular $me (ac$ve seekers, selec$ve seekers, informa$on avoiders)

•  Br J Nurs 2004;13:964-‐968

•  Can be abused, at bedside and in clinical “research”

IEJHE 2001;4:41-47

Abuse of paternalism in a clinical setting

IEJHE 2001;4:41-47

Abuse of paternalism in a research setting

Autonomy-‐Defined

•  MD role: vessel to carry out pa$ent’s wishes

•  MD obliga$on: respect autonomy; advocate and help procure what pa$ent wants

•  Pa$ent autonomy: central •  Pa$ent values: objec$ve (and defined by pa$ent)

Autonomy-‐History •  Increasingly common from 1960’s through

present day •  Strengthened by:

•  Pa$ent and research subject protec$on laws •  Malprac$ce, informed consent

•  Improved access to informa$on (internet) •  1990 AMA Fundamental Elements of

Pa$ent-‐Physician Rela$onship •  “The pa$ent has the right to make decisions

regarding the health care that is recommended by his or her physician. Accordingly, pa$ents may accept or refuse any recommended medical treatment.”

Autonomy-‐History

http://www.stayonsearch.com/what-will-the-internet-be-like-in-2025

The PatientsLikeMe process is to help people share their health information in order to learn from others like them and then use that information to discuss treatment options with their health care team

Step 1: Create/update and share your health profile

Step 2: Find support from others like you and compare experiences

Step 3: Learn from aggregated community Treatment and Symptom Reports

Step 4: Take profile to your doctor to have an improved treatment conversa$on

Step 5: Play an integral part in your own health care

Slide from Paul Wicks 2012

Autonomy-‐Example

Autonomy-‐Example

PLM Research •  17 PLM members repor$ng

use of sodium chlorite, 85 matched controls

•  2.5m dura$on of observa$on

•  Graph shows parameter es$mates of effect sizes for selected ALS treatments

Waiting for p<0.05. Paul Wicks, James Heywood, Timothy Vaughan. figshare. Retrieved 23:32, Dec 01, 2012 (GMT) http://dx.doi.org/10.6084/m9.figshare.96802

Autonomy

Strengths •  Respect for pa$ent-‐defined

values and goals is and should be paramount

•  Allows pa$ents to proceed with informa$on gathering at their own pace (ac$ve seekers, selec$ve seekers, informa$on avoiders)

Weaknesses •  Underu$liza$on of

physician educa$on, training and experience

•  Pa$ent values and goals may not be well-‐defined or stable over $me

•  Informa$on pa$ent is using may be flawed or inaccurate

Reasons Given for Declining Par$cipa$on in a Research Study

•  Lack of awareness •  Concerns about burdens ($me, travel) •  Fear of unexpected costs •  Fear of “being a guinea pig” •  Perceived loss of control over decision making •  Possibility of not receiving “best possible care”

especially with placebo designs •  Confusion about research process, specifics •  Choosing an alterna$ve therapy

Annals of Oncology 2000;11:939-945 ALS 2010;11:502-507 Parkinsonism Related Disorders 2011;17:667-67

Flawed Informa$on About an AOT

Flawed Informa$on About an AOT

Details •  Inves$gators called pa$ents

and asked them to categorize themselves

Inaccurate Informa$on About an AOT

“Reversible ALS” Does Exist •  Miyoshi K, Ohyagi Y, Amano T, Inoue I, Miyoshi S, Tsuii S,

Yamada T, Kira J. A pa$ent with motor neuron syndrome clinically similar to amyotrophic lateral sclerosis, presen$ng a spontaneous recovery. Rinsho Sinkeigaku 2000;40(11):1090-‐1095.

•  Tsai CP, Ho HH, Yen DJ, Wang V, Lin KP, Liao KK, Wu Z. Reversible motor neuron disease. Eur Neurol 1993;33:387-‐389.

•  Tucker T, Layzer R, Miller R, Chad D. Subacute, reversible motor neuron disease. Neurology 1991;41:1541-‐1544.

•  Tucker T. Subacute, reversible motor neuron disease. Taehan Singyongkwa Hakhoe chi 1991;41:1541.

PLM Data-‐Issues

•  Diagnoses •  Measurements •  Compliance •  Dosage •  Combining treatments •  Controls

“In Between” Models

JAMA 1992; 267: 2221-2226

Autonomous Paternalistic


Autonomous Paternalistic

Soc Sci Med 1999;49:651-661

Consumerism Shared Decision-Making

“In Between” Models-‐History •  3 ages of medicine: paternalism,

autonomy, bureaucracy •  Age of bureaucracy

•  Bewildering array of ALS informa$on, treatment op$ons

•  Google search ALS treatment: 36 million hits

•  Unprecedented cost, regulatory framework for some of these op$ons

•  Ex. Dexpramipexole compassionate use •  Evidence for these may be absent,

flawed, inaccurate or complex •  More important than ever that

physicians and pa$ents find ways to communicate and work together

Consumerism-‐History •  ALSUntangled

•  Uses social networking to systema$cally inves$gate alterna$ve and off-‐label ALS treatment op$ons (www.alsuntangled.org)

•  Input: twiZer, email, clinics, website

•  Discussion among experts: email

•  Results: Free access publica$ons in the ALS Journal

–  ALS 2009;10:129-‐130;

Consumerism-‐Example

•  Pa$ent asks physician, or sends tweet or email asking ALSUntangled for an opinion on oral sodium chlorite

•  Opinion: – Ac$ve ingredient similar to IV WF10 and IV NP001, macrophage modulators

–  IV NP001 has shown safety and some efficacy in small Phase 2 study in human PALS

–  hPp://www.neuraltus.com/pages/news_rel10_30_12.html


•  Opinion: – According to emails with one of the inventors of WF10, Dr. Friedrich-‐Wilhelm Kuehne “WF10 can act on these mechanisms only intravenously, not orally. I have studied oral WF10 intensively more then 30 years ago, aqer 2 pa$ents collapsed and one nearly died I terminated the development of oral WF10. I could show that oral chlorite generates Chordioxid under acidic condi$ons which was unwanted.”


•  Opinion: –  According to PLM experiences with oral sodium chlorite in ALS, costs are $25-‐50 per month. Analysis of 17 PLM members ALSFRS-‐R scores compared to historical controls shows no benefit.

•  Pa$ent takes informa$on away and deliberates with self or family to make final decision

Shared Decision-‐Making History •  ALS Clinical Research Learning Ins$tute

–  Modeled aqer successful programs in PD, cancer

–  Lectures on research including ethics, informed consent, trial designs, sta$s$cs

–  Lectures on advocacy including $ps on public speaking, wri$ng leZers to the editor, wri$ng leZers to congress, internet tools, mee$ng with the press and congress

–  Discussion and debate between faculty and aZendees regarding research barriers and how to address them; both leave with new apprecia$on for the others’ issues

-Nat Rev Cancer 2005;5:73-78

Shared Decision-‐Making Example •  Pa$ent frustrated by limited number of trial op$ons,

restric$ons on entry criteria, use of placebo, overall slow pace of research

•  AZends ALSCRLI •  Hears lectures on research, discusses and debates with

physicians and scien$sts •  Pa$ent learns of new research op$ons she did not know

about and comes to understand research beZer •  Clinicians and scien$sts mo$vated to design beZer trial

search engines, and to consider more inclusive and pa$ent friendly study designs


Strengths •  U$lize physician talents and

skills while s$ll allowing pa$ent to ul$mately define values, goals, acceptable risks and benefits

Weaknesses •  In consumerism, the

physician is reduced to a detached informa$on source; pa$ent values may be uncertain and may change

•  Fine line between shared decision-‐making and paternalism

•  Make take more $me

Which Model Is “Best?”

Pa$ent Preferences •  USA telephone survey (n=3,000; 72% comple$on rate) •  Shared-‐decision making favored by 62%, consumerism 28%,

paternalism 9% •  Older age, income <25K, educa$on level< high school,

African-‐American ethnicity independently associated with shiq toward paternalism (though all groups picked shared decision-‐making first)

•  Having a regular doctor, especially one that encouraged informa$on-‐seeking, associated with shiq toward shared decision making

Patient Education and Counseling 2007;65:189-196

Physician Preferences

•  USA mail survey, physicians who spent more than 20h per week in pa$ent care, mul$ple special$es, iden$fied via Medical Marke$ng Service (n=2,000, 53% response rate)

•  Shared-‐decision making favored by 75%, consumerism 11%, paternalism 14%

•  Older age and training “overseas” were associated with shiq toward paternalism (though all groups picked shared decision-‐making first)

BMC Family Practice 2007:8:10.

Compliance and Outcomes

•  Several studies have shown that shared decision-‐making results in improved pa$ent sa$sfac$on, compliance and health outcomes versus paternalism or consumerism

– Med Care 1989;33:s110-‐127 – Med Care 1995;1176-‐1187 –  Control Clin Trials 2000;21:233s-‐240s –  BMJ 2001;323:908-‐911 –  J Gen Intern Med 2002;17:243-‐252 –  J Gen Intern Med 2002;17:857-‐866 –  Int J Nurse Pract 2006;12:166-‐173

How Do We Get There?

BMJ. 1999 September 18; 319(7212): 766–771

Conclusions •  In the age of bureaucracy, there are many mainstream and

alterna$ve ALS treatment op$ons •  Cost, regulatory burden high •  Informa$on complex

•  There are 4 models by which ALS treatment decisions may get made in the doctor pa$ent rela$onship •  Paternalism, shared decision-‐making, consumerism and autonomy

•  Most pa$ents and physicians prefer shared decision-‐making; this is associated with improved compliance and health outcomes, but takes more $me than other models

The goal in (the doctor pa1ent rela1onship) is not to think alike, but to think together.

Robert C. Dodds

Thanks •  Motor Neurone Disease Associa$on •  Packard Center •  ALSRG, NEALS, ALSA •  Pa$ents Like Me •  ALS TDI •  AAN Palatucci Advocacy Leadership

Forum •  ALS Journal •  PALS and CALS

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