1. The Cost of PH- How do we
Manage this?
Terry Fortin MD
Duke Pulmonary Vascular Disease Center
Laura Nowicki RN
UNC Pulmonary Hypertension
2. Case TS Flashback to 1/2003
• Presented with syncope age 29 and had full work up
• Cath RA 15, Mean PAP 60, Index of 1.6L/min/m2 PVR 14
• Echo severely enlarged RV, Severe TR and hypokinesis
with pericardial effusion
• Walk 400 meters but only 50% predicted (actually post
med) due to syncope need for pressor
• pBNP did not exist.
• Admitted and started on IV prostacyclin. Later addition of
PDE 5 inhibitor and endothelin antagonist
• Had WU for transplant to accrue time then listing changed
and stopped seeing
• No hospitalizations since, On thyroid medication
3. Case TS Updated
• Repeat cath in 2016 with plans to convert IV to oral
prostacyclin PVR 4.3 WU and CI 2.7
• Most recent cath 2021 on triple oral therapy PVR 3.8 but still
with moderately enlarged RV only mild dysfunction
• Walk 560 meters 91% predicted Now age 50
• Normal pBNP since about 2005
• Although good, Not perfect and does he need more
• Has worked full time since mid 2003. Changed jobs 2 times.
Once because had used up lifetime insurance allotment of
policy. Once as Promotion. Now manager
• Married , 2 kids in their 20s PRICELESS
4. Cost Effects Many
• Cost to Society
• Cost to Health Care System ( Local/State/National)
• Effect on Pulmonary Hypertension Community
• Cost to the Individual PAH Center/ Health Center
– Takes a village and not necessarily reimbursable
• Cost to the Individual Patient and Family
• Not All COST Monetary
5. What Drives/Controls Cost
• Pharmaceutical or Biologic Companies Developing
Compounds
• Research Enterprise (Private or Governmental)
• Pharmaceutical Companies Manufacturing, Marketing
Products
• Health Systems, Hospitals and MD, RN, CMA,
• Insurance Companies
• Pharmacies/ Specialty Pharmacies
• Ancillary Services- Oxygen/ Sleep Companies
• Medicare/ Medicaid (Government/State)
6. Costs: Direct Health Care and Indirect
• Direct
• Cost of Diagnostic
Testing
• Walks, echos, caths,
PFDs, MRI, CT scans
• CXRs, ECGs
• Clinic visits/ED visits
• Hospitalizations
• Medications/Supplies/
• Indirect
• Disability
• Days Missing Work
• Family members
missing work
• Loss of productivity
• Death
• End of employment
• Other
7. Health Care Terms
• Quality Adjusted Life Years (QALY)
– One value to combine quality of Life (QoL) and length of Life due to
some treatment/procedure
• Disability adjusted Life years (DALY) ( 1- QUALY)
– Loss of healthy life, premature mortality plus years of healthy life lost
secondary to disability
Health Care Resource Utilization (HCRU)
• HRQoL Health Related Quality of Life
– Includes disease and physical symptoms
– Function status (ability to do ADLs
– Psychological and emotional statu
– Social factors
8. Managing Cost
• In PAH have Data to Support
• Getting earlier Diagnosis
– Early Initiation of Appropriate Therapy
– Education Important
• Referral to Specialized Treatment Centers
• Using Guidelines, Risk Sores to adjust therapy
• Combination therapy early in Treatment
• Escalation to Higher levels of therapy when Risks increase
• More is not always Better (Triton Study)
• Risk Scores may reflect illness not related to PAH
9. What is the Burden Of PAH Exposto, F
• Retrospective study in England 2012 to 2018 Using
National Health Services Data. 2500 pts
• Mean annual Hospitalization 2.9 to 3.2 (25% had 5
admissions)
• 9-10 yearly OP visits and ED visits 0.8 to 0.9
• Incident Patients Highest cost first Year with most
admissions for PAH related issues
• 79% cost inpt admissions, PH admissions more
costly(3x)
• Cost of medications was not addressed
• 20% of pts accounted for 55% of costI
• Data captured mixed etiology as top 20% far more likely to
have CV disease or Heart failure (not clear if right or left)
10. Economic Burden of PAH
• Cost and resource utilization in US Managed Care Group
• 2004 to 2010 Had to have >2 PAH claims or > 2 claims
with PAH diagnosis and1 + claims for prescribed med
• Followed once med prescribed and look at annualized
• 500 pts. Costs lower once treatment period started
compared to prior 98,000$ compared to 116,000$
• Much higher than other disease states
• Meds cost more in post treatment 38,000 vs 6400
• Other medical costs went down 60,000 post and 110 pre
• This included less clinic or OP visits and hospitalizations
Sikirico, M. Economic burden of PAH in the US on payers and patients.
BMC Health Serv Res 2014 Dec
24;14
11. Real World Treatment Patters HRU and Cost among
adults with PAH in US
• Oct 2015 to Nov 2020
• 21% initial combination therapy
• 54% Combination therapy
• 58% Hospitalized and 41% others ED visits
• Hospitalization costs pre PAH diagnosis vs post therapy
$14,200 down to 6350 per person per month
• Costs of medications 909 to 7800
12. Hospitalization Burden France
• 384 Incident PH patients Hospitalized
• Next 12 months 1270 Hospital stays
• Hospitalization to start meds much less costly than
Hospitalization for worsening disease
• Bergot, E Hospital burden of PAH in France Plos One
:2019 Sep 19(9) year studied 2013
13. Economic Burden 2022 Spain
• Direct and indirect cost to society for PAH
• Divided into functional class (incident and prevalent)
• Total costs Functional class I-II 65,000 Euros per pt
• FC III 61 % of patients. Cost was 103,000 Euros
• FC IV 208,000 Small % total pts only 7% but 14% ot
total cost
• Direct Health care costs 64% Indirect 24% and non health
care costs 12%
• Zozoya, N Economic burden of PAH in Spain, BMC Pulm
Med 2022 Mar 26: 22(1)
14. Hospitalization Increased in High Risk Patients
• What Happens when we Hospitalize June 2014 to 2019
• Look at meds 30 Days prior to admission and 90 days Post
• 43 monotherapy on admission only 17% went to double
therapy
• 3%
• Joszt, L PAH Treatment Patterns Cost Related to
Hospitalization. AJMC Clinformatic Data
15. Multiple studies confirm Hospitalization drives
largest component of cost
• Studies above show Hospitalization Majority of cost
• REVEAL data base that one Hospitalization increases risk
of further hospitalization
• Hospitalization is a negative prognostic factor
• In Griphon hospitalization for worsening PH portended
increased mortality
• Reveal Risk score also includes extra point for all cause
hospitalization within 6 months
• One study despite this cost and risk there was no change
in therapy in most cases post hospitalization
16. PAH in scleroderma
• Screening Australian Scleroderma interest group
• Algorithm using pBNP rather than an echo for yearly
screening of Scleroderma patients
Using new algorithm would save Australia between
367 and 725,000 $ annually
Quinlivan, A, Cost savings with a novel algorithm for early detection of
systemic sclerosis-related PAH: alternative scenario analysis. Intern Med
J 2019Jun 49(6) 781-85
Combination therapy is Cost Effective from Simulated Costs comparing
real pts on mono Cost higher is combo or dual therapy 20,000 vs 16
Mean life years 7.1 for mono and 9.2 for dual. QALY increased form
3 to 3.9 from mono to dual ( Tran Duy) 2021
17. CTEPH Cost = Mortality
• Cost of Refusal of Pulmonary thromboendarterectomy
• Worse long term survival
• Late Diagnosis, Lack of surgery facility, Referrals
Inoperable
• Registry 3 year survival 89% vs 70% ( Europe and Canada
• Operated majority had class 1-2 symptoms at one year
• Those eligible for surgery and refused 5 yr survival 53% vs
83% for those that opted for surgery
• Kim, N ERJ 2018 52Pulmonary endarterectomy and the
cost of patient refusal
18. Worldwide Practice
• Challenges in Middle and Low Income Regions
• Late presentation and more severe disease at diagnosis
and other untreated comorbidities.
• PAH/PH Etiology is Different
• Unrepaired congenital heart disease, More HIV,
Schistosomiasis, Group 2 related to valvular heart disease,
High Altitude, Smoke inhalation/COPD
• Less Access to therapy, and testing
• Barbar, H. Challenges and Special Aspects of PH in Middle to Low
income regions. JACC state of the art review. JACC 2020 May
19:75(19)
19. Make Research more Cost effective
• Enriched groups for increased risk in event driven trials
using risk scores /receiver operating curves
• Pooled data from Ambition (ambrisentan plus tadalafil),
Seraphin (macitentan morbidity /mortality) and Griphon
(Selexipag)
• can use lower sample size and and treatment time if use
pts with higher risk
• Easier or less waste in screening.
• Current study with sotatercept using similar theory
• Scott, JV Enrichmeny Benefits of Risk Algorithms for PAH
Clinical trials Am J Respir Crit Care med 2021 Mar 15: 203
20. Non Monetary Costs
Psychosocial Burdens
• Patient, Family, caregiver
• Impact of Physical Limitations
• Emotional Strain ( all parties)
• Loss of Confidence or Purpose
• Financial Strain
• Social Isolation
• Change in Relationship, Loss of intimacy
Doyle-Cox, C. Psychosocial burdens of PAHDiscussion paper Can J
Cardiovasc nursing 2016 Winter 26(1)
21. SF-36 Score Higher is Better
• Literature is dated
• What do we do with triple therapy
• Quadruple Therapy Who needs this.
• How to assess short term vs long term costs
• As we add new medications where do they fit in.
• Shuld we get early . Only after optimized
• How will insurance companies see this
• If many people getting fourth medication and aid pool is
same then who misses out
22. PAH Impact on quality of life
• Adverse effect of severe incurable disease on physical ,
emotional and social factors
• More PAH specific HR QoL Instruments such as Emphasis
10 and PAH-SYMPACT
• Medical therapies do improve HR QoL
• Also benefit to HR QoL with social support
– emotional support
– Physical therapy / or Rehab
Delcroix, M PAH:the burden of disease and impact on quality
of life Eur Respir Rev 2015 Dec 24(138)
23. Impact of PAH on Patients and caregivers
• Physical limitations
• Limited ability to carry out normal daily activity ADLs thus
need help from caregivers
• Social isolation of patient and caregivers
• Financial impact to patient and caregivers who may lose
time at work as well
• Physical , emotional, social, Practical Needs
• informational needs
• Mental health including depression, anxiety, stress.
Guillevin, L Understanding the impact of pulmonary arterial hypertension on
patients’ and carers’ lives. Eur Respir Rev. 2013 Dec 22(13)
• Gu, S. Systematic Review of health-related quality of life in patiens with PAH.
Phamacoeconomics. 2016 Aug:34(8)
24.
25. Optimize Cost Effective Care
• PAH High economic cost but also devastating outcomes
• Managed care providers must balance optimal care with
efficient use of healthcare resources
• Later diagnosis means more severe disease , poor prognosis
and more costs and more burden on system.
• Facilitate care through excellence centers to stream line and
use evidence based care to help lower costs
Studer, S. Considerations for optimal management of patients with PAH:
a multi-stakeholder roundtable discussion. Am J Manag Care 2017 May
23(6 Supp
26. Comparing one drug to another
• Effficacy/Side effects
• Means of Administration
• Safety
• Economic Aspects
• Is Combination Safe and Effective
• Direct Comparison Difficult as studies/ Data/Guidelines
have changed over time. Length of Follow up.
• Costs change with generic forms/Insurance coverage
• Efficacy within Functional Class must be considered
• Prostacyclins had greatest Life Years Gained and QALY
but may or may not be cost effective
27. PH Community Needing AID for Health COSTs
• Group1 PAH
• Group 2 Left Heart . Some Pre and Post Capillary patients
being enrolled in studies
• Group 3 ILD Patients now getting inhaled prostacyclin
– Prevalence of ILD 120-130/ 100,000
– 46% had PA pressures > 25
• Group 4 CTEPH Surgery/BPA/ Meds incidence 2.3% at 2
years post PE
• Group 5 Anemias, ESRD, Sarcoid…
• More potential patients for the same AID resources
29. Addition of Other Classes of Medications
• Hopeful Additions to Therpy
• Where will they fit
– Order of Therapy Early / Late
– Combinations
– For all comers or FC?
• What will they cost
Prior Authorization
Insurance Coverage
Assistance and AID
How do we add more choices and greater number of meds
per patient into already limited Resource Funds
30. How Can We Manage Cost
• Diagnose Early
• Treat with Combination Therapy
• Careful Use of Risk Scores to Guide additional therapy
• Aggressive treatment may prevent Hospitalization where
Cost is greatest.
• Treat Appropriate patients
• Participate in Research to Find better therapy, CURE
• Personalized /Targeted Therapy
• Fiscally Responsible Prescribing Meds and Assisting
Patients in Obtaining Them
31. Assistance Funds > 50% get AID
• Manufacturers Patient Assistance Programs (PAPs)
• Specialty Pharmacy Assistance
• Accredo, Alliance Rx , Briova now Optum, Cigna,
CVS/Caremark, Humana
• Assistance Fund
• Good Days
• Healthwell Foundation
• PAN Foundation
• Patient Advocate Foundation
Other Strategies, Good Rx, Cost Plus Pharmacy
32. Resources
• Ogbomo, A et al. Direct and Indirect Health Care Costs
Associated with PAH in commercially insured Patients in
US. J Managed Care Spec Pharm 2022 June:28(6)
• Roman, A. et al. Cost effectiveness of prostacyclins in
pulmonary arterial hypertension. Applied Health Econ
Health Policy 2012 May1:1-(3) 175-88
• Valerio, L. CTEPH and impairment after PE: the FOCUS
study. Eur Heart J. 2022 Sep 21:43
• Dong, W Cost effectiveness analysis of selexipag for he
combined treatment of PAH.Front Pharm 2023 Aug 11:14
• Bruger, C. Early Intervention in the management of PAH
:clinical and economic outcomes
33. Resources
• Scott, J.Enrichment benefits risk algorithms for PAH clinical
trials. Am J Respir Crit Care 2021 Mar15:203(6)
• Tran-Duy, A. Cost Effectivenes of Combination therapy for
patients with systemic sclerosis related PAH. J Am Heart
Assoc 2021 Apr 6:10(7)
• Exposto, F. Burden of PAH in Respir Dis 2021 Jan-Dec:15
• Meng-Chien, W. Potential application and promising role of
targeted therapy in PAH. Biomedicines 2022 Jun 15:10(6)