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NTM Lung Disease: The Patient’s Perspective

Amy Leitman, JD

Director of Policy & Research

NTM Info & Research

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Disclosures

No relevant financial disclosures to report

I will be discussing off-label uses of medicationsPropert

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Objectives• Review tools and resources available to providers, patients, and

caregivers to help educate patients about their illness and treatments

• Discuss patient experiences related to illness, treatments, and outcomes

• Discuss the role of new regulatory initiatives in future research, clinical practice, and therapeutic development

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The website: www.ntminfo.org

The pamphlet: A resource for patients

Co-founders:Patient & caregiver

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Online Forum for Patients

360Social

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Resources for Patients

Research Pipeline Page

Local Support Groups

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Bronchiectasis & NTM Research Registry

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Additional Resources for Patients & Providers

Or give us a call:

305-667-6461

ext. 26 or ext. 32

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Live EventsNTM & Bronchiectasis Conference

o CME-accredited Physician/Patient Conference (AMA, ANCC, ACPE, AARC, IPCE)

o May 14-15, 2020, Philadelphia, PA

o May 14: Patient sessions; May 15: clinical & scientific sessions

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NTM Research Consortium

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Diagnosis: A Life Altering Moment• What is it?• Am I contagious?• How will my life change?• Will my family help me?• Will my friends want to be around me?• Am I getting the correct treatment?• How long will I be on treatment?• What are the side effects?• Am I going to die from this?

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Improved Quality of LifeNTM is not a passive disease.

But some patients are more active decision makers/participants in their own care than others.

In addition to taking medicine, your patient must commit to:

o Airway clearanceo Nutritiono Exercise and fitnesso Proper resto Psychological support mechanisms

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Trust Yourself, Trust Your Patient

Listen to:

oYour patientoYour patient’s caregiver

oYour patient’s other healthcare providers - work as a team

Treating patients with pulmonary NTM disease is a commitment on working with them.You must ask them to commit to their health as well and make them an active participant in their own care.

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Depressive symptoms are common in patients with COPD and those with severe COPD have a 2.5 times

greater risk of developing depression.1

Limitations on activity imposed by chronic medical illness may lead to gradual withdrawal from rewarding

activities.2

Major depression is associated with increased medical utilization and costs, higher symptom burden,

increased functional impairment3 and other decrements in quality of life, and increased rates of mortality.4

Major depression is associated with 50% to 100% higher costs in medical patients even after controlling for

associated medical illness.5,6

1)Van Manen JG, et al. Risk of depression in patients with chronic obstructive pulmonary disease and its determinants. Thorax 2002;57:412-416.2)Prince M, et al. A prospective population-based cohort study of the effects of disablement and social milieu on the onset and maintenance of late-life depression. The Gospel Oak Project VII. Psychol Med 1998;28:337-350.3)Wells KB, Stewart A, Hats RD, et al. The functioning and well-being of depressed patients: Results from the Outcomes Study. JAMA. 1989; 262:914-919.4)Katon WJ. The depressed patient with comorbid illness. Program and abstracts of the 154th Annual Meeting of the American Psychiatric Association; May 5-10, 2001; New Orleans, LA. Industry Symposium, Part 2, 43B.5)Unutzer J, Katon W, Simon G, et al. Costs of medical care in an elderly primary care population. Presented at the 9th annual meeting of the American Association of Geriatric Psychiatry; February 1996; Tucson, AZ.6)Simon GE, Von Korff M, Barlow W. Health care costs of primary care patients with recognized depression. Arch Gen Psychiatry. 1995; 52:850-856.

How Depression and Chronic Disease are Linked

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Look and Listen

• Is your patient feeling OK?• DEPRESSION

• Plays a big role in chronic illness.

• How does your patient LOOK?

• Ask your patient how they’re FEELING.

• LISTEN to their answers for signs of depression.

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What To Look Foro Sadness

o Anxiety/ Fear

o Social isolation

o Anger

o Sleep disruption (too much or too little)

o Feeling of “not wanting to get up or go on”

o Suicidal thoughts or ideations

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ARS: What symptoms do patients with NTM lung disease report as experiencing most often and as most bothersome?A. Coughing without sputum, fatigue, shortness of breathB. Fatigue, coughing with sputum, weight lossC. Fatigue, coughing with sputum, shortness of breathD. Coughing with sputum, fatigue, shortness of breathProp

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Patient-Focused Drug Development

• Patients reported these three symptoms as having the most significant clinical impact on their daily lives:

o Fatigue

o Cough

o Shortness of breath

Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA). The voice of the patient: a series of reports from the U.S. Food and Drug Administration’s (FDA’s) Patient-Focused Drug Development Initiative: non-tuberculous mycobacterial (NTM) lung infection. Public Meeting: October 15, 2015. 2016 April [accessed 2016 April 30]. Available from: http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM496941.pdf

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NTM Patient Experiences

• Long delays to proper diagnosis• Long, burdensome treatment regimens• Side effects, some permanent• Symptoms including:

• Hemoptysis• Severe cough• Extreme fatigue• Shortness of breath

• Unpredictability in day-to-day health and functioning• Social isolation• Social stigma

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Survey:Patient Preferences in Clinical Trials & Treatment Outcomes

• 57 questions• Joint project with Spero Therapeutics• Reviewed by internal and external staff and researchers, NTM patients• Circulated through NTMinfo.org, social media channels, blast emails,

Social360 forum• 465 respondents• Results presented at April 8th FDA workshop on Development of

Antibacterial Drugs for the Treatment of Nontuberculous Mycobacterial Disease

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0% 10% 20% 30% 40% 50% 60% 70% 80% 90%

Anxiety

Chest pain

Lack of appetite

Coughing up blood

Weight loss

Night sweats

Coughing without sputum

Shortness of breath

Coughing with sputum

Fatigue

Top Symptoms Patients Experienced

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0% 10% 20% 30% 40% 50% 60% 70% 80% 90%

Anxiety

Chest Pain

Coughing up blood

Lack of/ decreased appetite

Weight loss

Night sweats

Coughing without sputum

Shortness of breath

Coughing up sputum

Fatigue

Most Bothersome Symptoms

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Side Effects of Antibiotic Treatment

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FDA Workshop Nov. 18-19, 2019:Exploring the Clinical Trial Enterprise for Antibacterial

Drug Development in the United States

• Co-sponsored by FDA, IDSA, and Pew Charitable Trusts

• Strategies to better support clinical trials for antibiotic development

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THANK YOU

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ntminfo.org

facebook.com/NTMinfo@NTMinfo youtube.com/ntmir

1550 Madruga Avenue, Suite 230Coral Gables, Florida

33146305.667.6461 ext. 26ntmmail@ntminfo.org

www.ntminfo.org

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