Identifying the Benefits and Risks of Emerging Treatments for Idiopathic Pulmonary Fibrosis: A Qualitative Study

  • John F. P. Bridges
  • Victoria Federico Paly
  • Elizabeth Barker
  • Dolly Kervitsky
Original Research Article

Abstract

Objective

Idiopathic pulmonary fibrosis (IPF) is a rare, progressive, and fatal disease, with very few therapeutic options. Given a paucity of qualitative research to the perspective of patients and other stakeholders in IPF, we sought to identify issues associated with the benefits and risks of emerging treatments and other issues relevant to design of a survey for assessing patient preferences for IPF treatments.

Methods

Semi-structured key informant interviews were conducted, predominately via telephone, with a range of stakeholder perspectives identified through partnership with a national advocacy organization using a combination of purposive and snowball sampling. Stakeholders were asked guiding questions related to emerging trends impacting IPF patients, likely benefits and risks of emerging treatments, and the outcomes most relevant to patients. Detailed and de-identified field notes were analyzed using interpretive phenomenological analysis (IPA), and a taxonomy of key themes was developed.

Results

A total of 20 interviews (participation rate 63%) were conducted with patients/advocates/caregivers (n = 7), providers/researchers (n = 8), and experts associated with policy/industry (n = 5). All interviewees expressed great hope with regards to emerging treatments. Three super-ordinate themes emerged: impact of emerging therapies (spanning the benefits, risks, and unintended consequences of emerging therapies); documenting the patient experience (spanning measuring patient-reported outcomes and quality of life and understanding the burden of disease); and coping with disease progression (including symptom mitigation, lung transplantation, and end-of-life considerations).

Conclusions

In identifying issues associated with emerging IPF treatments, we demonstrate the value of qualitative research in understanding the views of diverse stakeholders and in providing a basis for future survey research. As such, qualitative methods should play an important role in understanding the benefits and risks of emerging therapies and in promoting patient-centered drug development.

References

  1. 1.
    Olson AL, Swigris JJ. Idiopathic pulmonary fibrosis: diagnosis and epidemiology. Clin Chest Med. 2012;33(1):41–50.PubMedCrossRefGoogle Scholar
  2. 2.
    Raghu G, Collard HR, Egan JJ, Martinez FJ, Behr J, Brown KK, et al. An official ATS/ERS/JRS/ALAT statement: idiopathic pulmonary fibrosis: evidence-based guidelines for diagnosis and management. Am J Respir Crit Care Med. 2011;183(6):788–824.PubMedCrossRefGoogle Scholar
  3. 3.
    Raghu G, Weycker D, Edelsberg J, Bradford WZ, Oster G. Incidence and prevalence of idiopathic pulmonary fibrosis. Am J Respir Crit Care Med. 2006;174(7):810–6.PubMedCrossRefGoogle Scholar
  4. 4.
    Collard HR, Tino G, Noble PW, Shreve MA, Michaels M, Carlson B, et al. Patient experiences with pulmonary fibrosis. Respir Med. 2007;101(6):1350–4.PubMedCrossRefGoogle Scholar
  5. 5.
    Ley B, Collard HR, King TE Jr. Clinical course and prediction of survival in idiopathic pulmonary fibrosis. Am J Respir Crit Care Med. 2011;183(4):431–40.PubMedCrossRefGoogle Scholar
  6. 6.
    Schoenheit G, Becattelli I, Cohen AH. Living with idiopathic pulmonary fibrosis: an in-depth qualitative survey of European patients. Chron Respir Dis. 2011;8(4):225–31.PubMedCrossRefGoogle Scholar
  7. 7.
    Cottin V. Changing the idiopathic pulmonary fibrosis treatment approach and improving patient outcomes. Eur Respir Rev. 2012;21(124):161–7.PubMedCrossRefGoogle Scholar
  8. 8.
    Jones MG, Fletcher S, Richeldi L. Idiopathic pulmonary fibrosis: recent trials and current drug therapy. Respiration. 2013;86(5):353–63.PubMedGoogle Scholar
  9. 9.
    Noble PW, Albera C, Bradford WZ, Costabel U, Glassberg MK, Kardatzke D, et al. Pirfenidone in patients with idiopathic pulmonary fibrosis (CAPACITY): two randomised trials. Lancet. 2011;377(9779):1760–9.PubMedCrossRefGoogle Scholar
  10. 10.
    Richeldi L, Costabel U, Selman M, Kim DS, Hansell DM, Nicholson AG, et al. Efficacy of a tyrosine kinase inhibitor in idiopathic pulmonary fibrosis. N Engl J Med. 2011;365(12):1079–87.PubMedCrossRefGoogle Scholar
  11. 11.
    Horton MR, Santopietro V, Mathew L, Horton KM, Polito AJ, Liu MC, et al. Thalidomide for the treatment of cough in idiopathic pulmonary fibrosis: a randomized trial. Ann Intern Med. 2012;157(6):398–406.PubMedCrossRefGoogle Scholar
  12. 12.
    King TE, Bradford WZ, Castro-Bernardini S, Fagan EA, Glaspole I, Glassberg MK, et al. A phase 3 trial of pirfenidone in patients with idiopathic pulmonary fibrosis. N Engl J Med. 2014;370(22):2083–92.PubMedCrossRefGoogle Scholar
  13. 13.
    Richeldi L, du Bois RM, Raghu G, Azuma A, Brown KK, Costabel U, et al. Efficacy and safety of nintedanib in idiopathic pulmonary fibrosis. N Engl J Med. 2014;370(22):2071–82.PubMedCrossRefGoogle Scholar
  14. 14.
    Swigris JJ, Kuschner WG, Jacobs SS, Wilson SR, Gould MK. Health-related quality of life in patients with idiopathic pulmonary fibrosis: a systematic review. Thorax. 2005;60(7):588–94.PubMedCentralPubMedCrossRefGoogle Scholar
  15. 15.
    De Vries J, Kessels BL, Drent M. Quality of life of idiopathic pulmonary fibrosis patients. Eur Respir J. 2001;17(5):954–61.PubMedCrossRefGoogle Scholar
  16. 16.
    Belkin A, Swigris JJ. Patient expectations and experiences in idiopathic pulmonary fibrosis: implications of patient surveys for improved care. Expert Rev Respir Med. 2014;8(2):173–8.Google Scholar
  17. 17.
    Gooberman-Hill R. Qualitative approaches to understanding patient preferences. Patient. 2012;5(4):215–23.PubMedGoogle Scholar
  18. 18.
    O’Haire C, McPheeters M, Nakamoto E, LaBrant L, Most C, Lee K, et al. Methods for engaging stakeholders to identify and prioritize future research needs. Methods Future Research Needs Report No. 4. June 2011, AHRQ Publication No. 11-EHC044-EF.Google Scholar
  19. 19.
    Fleurence R, Selby JV, Odom-Walker K, Hunt G, Meltzer D, Slutsky JR, et al. How the Patient-Centered Outcomes Research Institute is engaging patients and others in shaping its research agenda. Health Aff (Millwood). 2013;32(2):393–400.CrossRefGoogle Scholar
  20. 20.
    Mullard A. Patient-focused drug development programme takes first steps. Nat Rev Drug Discov. 2013;12(9):651–2.PubMedCrossRefGoogle Scholar
  21. 21.
    Coast J, Al-Janabi H, Sutton EJ, Horrocks SA, Vosper AJ, Swancutt DR, et al. Using qualitative methods for attribute development for discrete choice experiments: issues and recommendations. Health Econ. 2012;21(6):730–41.PubMedCrossRefGoogle Scholar
  22. 22.
    Bridges JF, Gallego G, Blauvelt BM. Controlling liver cancer internationally: A qualitative study of clinicians’ perceptions of current public policy needs. Health Res Policy Syst. 2011;9:32–4505.PubMedCentralPubMedCrossRefGoogle Scholar
  23. 23.
    Bridges JF, Anderson BO, Buzaid AC, Jazieh AR, Niessen LW, Blauvelt BM, et al. Identifying important breast cancer control strategies in Asia, Latin America and the Middle East/North Africa. BMC Health Serv Res. 2011;11:227–6963.PubMedCentralPubMedCrossRefGoogle Scholar
  24. 24.
    Smith JA, Osborn M. Qualitative psychology: a practical guide to research methods. London: Sage Publications; 2008.Google Scholar
  25. 25.
    Smith JA, Flowers P, Larkin M. Interpretive phenomenological analysis: theory, method, and research. London: Sage Publications; 2009.Google Scholar
  26. 26.
    Malterud K. Qualitative research: standards, challenges, and guidelines. Lancet. 2001;358(9280):483–8.PubMedCrossRefGoogle Scholar
  27. 27.
    Larkin M, Watts S, Clifton E. Giving voice and making sense in interpretative phenomenological analysis. Qual Res Psychol. 2006;3(2):102–20.CrossRefGoogle Scholar
  28. 28.
    BioMed Central. Qualitative research review guidelines - RATS. Available at: http://www.biomedcentral.com/authors/rats. Accessed 3 Mar 2014.
  29. 29.
    Food and Drug Administration. Enhancing benefit-risk assessment in regulatory decision-making. 2014; Available at: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm. Accessed 01 Feb 2014.
  30. 30.
    Flaherty KR, King TE Jr, Raghu G, Lynch JP 3rd, Colby TV, Travis WD, et al. Idiopathic interstitial pneumonia: what is the effect of a multidisciplinary approach to diagnosis? Am J Respir Crit Care Med. 2004;170(8):904–10.PubMedCrossRefGoogle Scholar
  31. 31.
    Swigris JJ, Stewart AL, Gould MK, Wilson SR. Patients’ perspectives on how idiopathic pulmonary fibrosis affects the quality of their lives. Health Qual Life Outcomes. 2005;7(3):61.CrossRefGoogle Scholar
  32. 32.
    Hauber BA, Fairchild AO, Johnson RF. Quantifying benefit-risk preferences for medical interventions: an overview of a growing empirical literature. Appl Health Econ Health Policy. 2013;11(4):319–29.CrossRefGoogle Scholar
  33. 33.
    Bridges JF, Kinter ET, Kidane L, Heinzen RR, McCormick C. Things are looking up since we started listening to patients: trends in the application of conjoint analysis in health 1982–2007. Patient. 2008;1(4):273–82.PubMedCrossRefGoogle Scholar
  34. 34.
    Facey K, Boivin A, Gracia J, Hansen HP, Lo Scalzo A, Mossman J, et al. Patients’ perspectives in health technology assessment: a route to robust evidence and fair deliberation. Int J Technol Assess Health Care. 2010;26(3):334–40.PubMedCrossRefGoogle Scholar
  35. 35.
    Peay H, Hollin I, Fischer R, Bridges J. A community-engaged approach to quantifying caregiver preferences for the benefits and risks of emerging therapies for Duchenne muscular dystrophy. Clin Ther. 2014;36(5):624–37.PubMedCrossRefGoogle Scholar

Copyright information

© Springer International Publishing Switzerland 2014

Authors and Affiliations

  • John F. P. Bridges
    • 1
    • 2
  • Victoria Federico Paly
    • 1
  • Elizabeth Barker
    • 3
  • Dolly Kervitsky
    • 3
  1. 1.Department of Health Policy and ManagementJohns Hopkins Bloomberg School of Public HealthBaltimoreUSA
  2. 2.Center for Medicine in the Public InterestNew YorkUSA
  3. 3.Pulmonary Fibrosis FoundationChicagoUSA

Personalised recommendations