Pulmonary Hypertension: New Definitions, New ApproachesFinal Live Outcome Report

Prepared For Actelion Pharmaceuticals US, Inc.: Grant ID 55783073January 21, 2020

Persistent Educational Gapsv Though improvements were observed, learners demonstrated persistent gaps in the several areas including:

v Diagnosing PAH and interpreting the impact of pulmonary pressures

v Recognizing the difference between Ventilation-Perfusion scanning and CT Pulmonary Angiogram for the diagnosis of CTEPH

v Treatment strategies for PAH and how to further adjust treatment based on risk

v Management of medication related adverse events

The post-test scores, and self reported confidence regarding the management of patients with PAH, signifies a clear gap in knowledge

and an unmet need among clinicians. It continues to be an important area for future educational programs.

Executive Summary

*These numbers represent the total number of attendees, irrespective of assessment participation

917 total attendees

on site: 97 attendees

National online simulcast: 820 attendees

v This activity focused on improving the diagnosis of Pulmonary Arterial

Hypertension (PAH) with updated guidelines, using risk stratification to adjust

therapy and addressing adverse effects while improving patient adherence.

v 917 attendees in multiple professional specialties were reached in this program.

v Improvement across all learning domains was noted ranging from 29% to 65%.

v Overall, the program improved the ability of learners to recognize how to

diagnosis and manage PAH.

Learning Objectives

4

3

1 Describe new definitions of PAH and approaches to improving diagnosis

Outline an approach to rule out and appropriately manage chronic thromboembolic pulmonary hypertension (CTEPH), if present

Utilize risk stratification for selecting and escalating therapy in patients with PAH

Describe the management of adverse events with PAH therapies and strategies to improve patient adherence

2

Course DirectorFranck Rahaghi, MD, MHS, FCCPChairman, Department of Pulmonary MedicineDirector, Pulmonary Hypertension ClinicHead, Pulmonary Education and RehabilitationDepartment of Pulmonary and Critical CareCleveland Clinic FloridaWeston, FL

Gregg Sherman, MD

Michelle Frisch, MPH, CCMEP

Sandy Bihlmeyer M.Ed

Franck Rahaghi, MD, MHS, FCCP

Sheila Lucas, CWEP

Joshua F. Kilbridge

Cedric Nazareth, MBBS

Deborah Paschal, CRNP

Sandhya Khurana, MDProfessor, Pulmonary and Critical Care MedicineDirector, Mary Parkes Center for Asthma, Allergy & Pulmonary CareUniversity of Rochester School of MedicineRochester, NY

Mehdi Mirsaeidi MD, MPHDirector of UM and VA Sarcoidosis ProgramsIRB Vice-Chairman, Miami VA Healthcare SystemDivision of Pulmonary, Critical Care,Sleep and AllergyDepartment of MedicineUniversity of MiamiMiller School of MedicineMiami, FL

Franck Rahaghi, MD, MHS, FCCPChairman, Department of Pulmonary MedicineDirector, Pulmonary Hypertension ClinicHead, Pulmonary Education and RehabilitationDepartment of Pulmonary and Critical CareCleveland Clinic FloridaWeston, FL

Farbod N. Rahaghi, MD, PhD *Instructor, Harvard Medical SchoolApplied Chest Imaging LaboratoryPulmonary Vascular Disease ProgramBrigham and Women's HospitalPulmonary and Critical Care Internal MedicineBoston, MA

Gregory Cosgrove, MDAssociate ProfessorAssistant Director, Interstitial Lung Disease ProgramDepartment of MedicineDivision of Pulmonary, Critical Care & Sleep MedicineEndowed Chair in Interstitial Lung DiseaseNational Jewish HealthDenver, CO

Samuel Gurevich, MD, FCCPMedical Director, Respiratory TherapyCleveland Clinic FloridaClinical Assistant Professor of MedicineCleveland Clinic Cleveland Clinic Lerner College of Medicine of Case Western Reserve UniversityWeston, FL

Sajive Aleyas, MDInterventional PulmonaryCleveland Clinic FloridaWeston, FL

Faculty

Activity Planning Committee

*Presented PAH lecture

Commercial Support

The Challenges in Pulmonary and Critical Care: 2019 CME activity was supported through educational grants or donations from the following companies:

vActelion Pharmaceuticals US, Inc.

vGenentech

vNovartis Pharmaceuticals Corporation

vCSL Behring, LLC.

vGrifols

vMallinckrodt, LLC

vShire

Levels of EvaluationConsistent with the policies of the ACCME, NACE evaluates the effectiveness of all CME activities using a systematic process based on Moore’s model. This outcome study reaches Level 5.

Level 1: Participation

Level 2: Satisfaction

Level 3: Declarative and Procedural Knowledge

Level 4: Competence

Level 5: Performance

Level 6: Patient Health

Level 7: Community Health

Moore DE Jr, Green JS, Gallis HA. Achievingdesired results and improved outcomes: integratingplanning and assessment throughout learningactivities. J Contin. Educ. Health Prof. 2009 Winter;29(1):1-15

Level 1:Participation and Demographics

917 total attendees

On site: 97 attendees

90%Provide direct patient care

National online simulcast : 820 attendees

Level 1:Participation

November 23, 2019 Coral Springs, FL

Profession Years in Practice

Patient Care Focus: 90%

Level 1: Demographics and Patient Reach

Patients seen each week, in any clinical setting:Specialty

4%

56%

6% 3% 4%

26%

Pulmonology InternalMedicne/Primary

Care

Cardiology Hospitalist EmergencyMedicine

Other

17%

2%

59%

12%7% 3%

MD DO NP PA RN Other

30%

27%

20%

23%

0% 20% 40% 60% 80% 100%

<25

25-50

51-75

>75

26%17% 21%

36%

<5 5-10 11-20 >20

Level 2-5:Outcomes Metrics

88% rated the activity as excellent

89% indicated the activity improved their knowledge

88% stated that they learned new and useful strategies for patient care

91% said they would implement new strategies that they learned

98% said the program was fair-balanced and unbiased

Level 2: Satisfaction

Please rate your confidence in your ability to manage patients with PAH:(Learning Objectives 2, 3, 4)

Confidence Assessment

Pre: 342 PCA: 161N=

54%

33%

8%3% 2%

12%

41%

32%

12%

3%

0%

10%

20%

30%

40%

50%

60%

70%

80%

90%

100%

Not at all confident Slightly confident Moderately confident Pretty much confident Very confident

246 249 161

Which of the following is NOT a critical reason for the necessity of right heart catheterization in the diagnosis of PH?(Learning Objective 1)

Knowledge Assessment

Pre: Post: PCA:N=

8%

18%

28%

14%

31%

8%14%

37%

18%

48%

14%

22%

31%

21%

13%

0%

10%

20%

30%

40%

50%

60%

70%

80%

90%

100%

Obtaining accurate measuresof mean pulmonary pressure

for the purpose ofestablishing the diagnosis of

PH

Distinguishing precapillaryand post-capillary PH

Pulmonary vasodilatorytesting

Obtaining accurate measuresof pulmonary pressures for

prognostication/riskassessment

Measuring Cardiac Index toevaluate degree of heart

failure

P<=.05

Pre to Post Change 29%Pre to PCA Change 49%

257 259 161

25 y/o female with history of contraceptive use and obesity who was admitted for acute shortness of breath and diagnosed with acute pulmonary embolism comes in for follow up 6 month after the diagnosis. She has remained on anticoagulation and has been adherent to therapy. She says that although she feels better she still feels short of breath. Her chest x-ray remains clear. Which of the following tests would best rule out chronic thromboembolic disease. (Learning Objective 2)

Knowledge Assessment

8% 9%

32%

4%

47%

9% 8%

49%

3%

31%

8%13%

40%

6%

34%

0%

10%

20%

30%

40%

50%

60%

70%

80%

90%

100%

Planar PulmonaryAngiography

Echocardiogram Ventilation-Perfusion lungscanning

Cardiopulmonary ExerciseTesting

CT pulmonary angiography

Pre to Post Change 53%Pre to PCA Change 8%

P<=.05

Pre: Post: PCA:N=

Which of the following is true of therapies in patients with pulmonary arterial hypertension?(Learning Objective 3)

Pre: Post: PCA:

Knowledge Assessment

N= 249 251 161

22% 21%17%

40%

18%14%

28%

40%

24% 26%

19%

31%

0%

10%

20%

30%

40%

50%

60%

70%

80%

90%

100%

In high risk patients, monotherapy withIV Prostacyclin is recommended, as

additional agents add very little to thistherapy

Transition from high risk to intermediaterisk after therapy suggests that no

further escalation of therapy isindicated

Oxygen requirements are not a majorcomponent of the decision to escalate

treatment in PAH

Upfront combination therapy is notrecommended in a patient with a new

diagnosis of PAH who is deemed to below-risk

P<=.05

Pre to Post Change 65%Pre to PCA Change 12%

Which of the following is true about adverse event management in treatments of PAH:(Learning Objective 4)

Pre: Post: PCA:

Knowledge Assessment

N= 175 162 161

22%

13%

21%

9%

35%

16%

9%14%

5%

56%

18% 17% 19%

10%

36%

0%

10%

20%

30%

40%

50%

60%

70%

80%

90%

100%

Switching medications withina class rarely effects tolerance

of specific adverse events

Constipation is a commonside-effect of IV Prostacyclin

therapy

Headache associate withprostacyclin therapy shouldnot be treated with over the

counter medications

Edema is not a side-effect ofpulmonary vasodilators

Upfront combination therapycan increase the incidence of

certain adverse events

Pre to Post Change 60%Pre to PCA Change 3%

P<=.05

Timely referralPatient education Patient engagement

Disease state awareness Non-pharmacotherapy

Please select the specific areas of skills, or practice behaviors, you have improved regarding the screening, diagnosis and treatment of Pulmonary Arterial Hypertension since this CME activity. (Select all that apply.)N=117

(4-week Post Assessment)

37% 34% 20%

55% 16%

Insurance/Financial issues

Medication costs Time constraints

Patient adherence/compliance

Lack of knowledge

What specific barriers have you encountered that may have prevented you from successfully implementing screening, diagnosis and treatment of Pulmonary Arterial Hypertension since this CME activity? (Select all that apply) N=117

(4-week Post Assessment)

36%

42%33%

13%36%

Persistent Educational Gaps After 4 Weeks

Diagnosing PAH and interpreting the impact of pulmonary pressures

Recognizing the difference between Ventilation-Perfusion scanning and CT Pulmonary Angiogram for the diagnosis of CTEPH

Treatment strategies for PAH and how to further adjust treatment based on risk

Management of medication related adverse events

Greater recognition of the benefits of right heart catheterization in the diagnosis of PAH but that it is not required for prognostication/risk stratification

More aware that Ventilation-Perfusion lung scanning is most appropriate to rule out chronic thromboembolic disease

Greater awareness that oxygen requirements are not a major component of the decision to escalate therapy in PAH

More aware of adverse events associated with PAH therapy and how to manage it

Participant Educational Gains

Key Take-home Points

Significantly improved learner confidence in the ability to manage patients with PAH

90% of learners are engaged in direct patient care and 91% reported that they will implement new strategies they learned

After 4 weeks, participants reported the following barriers regarding the screening, diagnosis and treatment of PAH: 42% lack of knowledge, 33% patient adherence/compliance, 36% insurance/financial issues

After 4 weeks, participants reported the following improved skills regarding the screening, diagnosis and treatment of PAH: 55% disease state awareness,37% timely referral, and 34% patient education