513
Views
11
CrossRef citations to date
0
Altmetric
Review

The evolution of patient-focused drug development and Duchenne muscular dystrophy

ORCID Icon, , , &
Pages 57-68 | Received 10 Dec 2019, Accepted 21 Feb 2020, Published online: 06 Mar 2020

References

  • FDA mission. [cited 2020 Mar 2]. Available from: https://www.fda.gov/aboutfda/whatwedo/default.htm-mission.
  • Van Norman GA. Drugs, devices, and the FDA: part 1: an overview of approval processes for drugs. JACC. 2016;1(3):170–179.
  • Carpenter DP. The political economy of FDA drug review: processing, politics, and lessons for policy. Health Affairs. 2004;23(1):52–63.
  • Califf RM. Benefit-risk assessments at the US food and drug administration: finding the balance. Jama. 2017;317(7):693–694.
  • Anderson M, McCleary KK. From passengers to co-pilots: patient roles expand. Sci Transl Med. 2015;7(291):291fs25–291fs25.
  • Mendell JR, Shilling C, Leslie ND, et al. Evidence-based path to newborn screening for duchenne muscular dystrophy. Ann Neurol. 2012;71(3):304–313.
  • Emery AE. Population frequencies of inherited neuromuscular diseases–a world survey. Neuromuscul Disord. 1991;1(1):19–29.
  • Mah JK, Korngut L, Dykeman J, et al. A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy. Neuromuscul Disord. 2014;24(6):482–491.
  • Furlong P, Bridges JFP, Charnas L, et al. How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the US food and drug administration. Orphanet J Rare Dis. 2015;10(1):82.
  • Dunkle M, Pines W, Saltonstall PL. Advocacy groups and their role in rare diseases research. Adv Exp Med Biol. 2010;686:515–525.
  • Patient-focused drug development: collecting comprehensive and representative input, in draft guidance for industry, food and drug administration staff, and other stakeholders.  Silver Spring, MD: US Department of Health and Human Services; 2018.
  • Medical Device Innovation Consortium (MDIC). Patient centered benefit-risk project report: a framework for incorporating information on patient preferences regarding benefit and risk into regulatory assessments of new medical technology. Minneapolis, MN: Medical Device Innovation Consortium; 2015.
  • Hunter NL, O’Callaghan KM, Califf RM. Engaging patients across the spectrum of medical product development: view from the US food and drug administration. Jama. 2015;314(23):2499–2500.
  • Hellstadius Y, Lagergren J, Zylstra J, et al. Prevalence and predictors of anxiety and depression among esophageal cancer patients prior to surgery. Dis Esophagus; 2016;29(8):1128-1134. doi: 10.1111/dote.12437. Epub 2015 Nov 6.
  • Kondo T. “Rational Medicine” Initiative. Pharmaceuticals and Medical Devices Agency of Japan; 2017. [cited 2020 Mar 2]. Available from: https://www.pmda.go.jp/files/000227456.pdf.
  • PDUFA Reauthorization performance goals and procedures fiscal years 2013 trough 2017. Silver Spring, MD: Dept. of Health and Human Services, US Food and Drug Administration; 2012.
  • Green BN, Johnson CD, Adams A. Writing narrative literature reviews for peer-reviewed journals: secrets of the trade. J Chiropr Med. 2006;5(3):101–117.
  • Collins JA, Fauser BC. Balancing the strengths of systematic and narrative reviews. Oxford, UK: Oxford University Press; 2005.
  • Aslakson RA, Schuster ALR, Miller J, et al. An environmental scan of advance care planning decision AIDS for patients undergoing major surgery: a study protocol. Patient - Patient-Centered Outcomes Res. 2014;7(2):207–217.
  • Smith JA, Flowers P, Osborn M. Interpretative phenomenological analysis and the psychology of health and illness. In: L. Yardley, editor. Material discourses of health and illness. London, Uk: Taylor & Frances/Routledge. 1997;68–91.
  • Larkin M, Watts S, Clifton E. Giving voice and making sense in interpretative phenomenological analysis. Qual Res Psychol. 2006;3(2):102–120.
  • Framework for FDA’s real-world evidence program. Silver Spring, MD: US Food and Drug Administration; 2018.
  • Guidance for industry patient-reported outcome measures: use in medical product development to support labeling claims. Silver Spring, MD: US Food and Drug Administration; 2009.
  • Kolata G. Prescription drug ads put FDA on the spot. Science. 1983;220(4595):387–388.
  • House JS, Landis KR, Umberson D. Social relationships and health. Science. 1988;241(4865):540–545.
  • Federal Food, Drug, and Cosmetic Act, in 21 U.S.C. ch. 9 §301 et seq, t.U.S. Congress, Editor. 1938.
  • Drug Ammendments Act, in 21 U.S.C. ch. 9 §301 et seq, t.U.S. Congress, Editor. 1962.
  • Wardell WM, Hassar M, Anavekar SN, et al. The rate of development of new drugs in the United States, 1963 through 1975. Clin Pharmacol Ther. 1978;24(2):133–145.
  • Wardell WM. The drug lag revisited: comparison by therapeutic area of patterns of drugs marketed in the United States and Great Britain from 1972 through 1976. Clin Pharmacol Ther. 1978;24(5):499–524.
  • Kaitin KI, Mattison N, Northington FK, et al. The drug lag: an update of new drug introductions in the United States and in the United Kingdom, 1977 through 1987. Clin Pharmacol Ther. 1989;46(2):121–138.
  • Gieringer DH. The safety and efficacy of new drug approval. Cato J. 1985;5:177.
  • Holt-Lunstad J, Smith TB, Layton JB. Social relationships and mortality risk: a meta-analytic review. PLoS Med. 2010;7(7):e1000316.
  • The Orphan Drug Act, in 21 U.S.C. ch. 9, subch. V § 360aa-360ee. 1983.
  • Novas C. Orphan drugs, patient activism and contemporary healthcare. Quaderni Commun Technol pouvoir. 2009;68:13–23.
  • Gammie T, Lu CY, Babar ZU. Access to orphan drugs: a comprehensive review of legislations, regulations and policies in 35 countries. PLoS One. 2015;10(10):e0140002.
  • Agency for Healthcare Research and Quality. National healthcare quality report. Rockville, MD: Agency for Healthcare Research and Quality; 2006.
  • FDA Backgrounder on FDAMA. 1997 [cited 2019 Mar 25]. Available from: https://www.fda.gov/RegulatoryInformation/LawsEnforcedbyFDA/SignificantAmendmentstotheFDCAct/FDAMA/ucm089179.htm.
  • Institute of Medicine. Institute of Medicine committee on quality of health care in America, in Crossing the quality chasm: a new health system for the 21st century. Washington (DC): National Academies Press (US); 2001.
  • Selby JV, Beal AC, Frank L. The patient-centered outcomes research institute (PCORI) national priorities for research and initial research agenda. Jama. 2012;307(15):1583–1584.
  • Acquadro C, Berzon R, Dubois D, et al. Incorporating the patient’s perspective into drug development and communication: an ad hoc task force report of the patient‐reported outcomes (PRO) harmonization group meeting at the food and drug administration, February 16, 2001. Value Health. 2003;6(5):522–531.
  • Burke L Acceptable evidence for pharmaceutical advertising and labeling. DIA Workshop on Pharmacoeconomic and Quality of Life Labeling and Marketing Claims, New Orleans, LA. 2000.
  • Colapinto J, Mother Courage, in The New Yorker. 2010.
  • Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010;9(1):77–93.
  • Berke R, Kaplan S. Former FDA chief margaret hamburg speaks out about califf, cruz, and congress. STAT; 2016. Available from: https://www.statnews.com/2016/03/16/margaret-hamburg-fda/
  • Food and Drug Safety and Innovation Act, t.U. Congress, Editor. 2012.
  • Millar SL, Chambers M, Giles M. Service user involvement in mental health care: an evolutionary concept analysis. Health Expect. 2016;19(2):209–221.
  • PDUFA V: medical innovation, jobs, and patients, in committee on energy and commerce. Available from: https://www.govinfo.gov/content/pkg/CHRG-112hhrg72917/html/CHRG-112hhrg72917.htm
  • Pitts PJ. FDA and the critical path to twenty-first-century medicine. J Med Philos. 2008;33(5):515–523.
  • Food, U. and D. Administration. Structured approach to benefit-risk assessment in drug regulatory decision-making: draft PDUFA V implementation plan—February 2013: fiscal years 2013–2017. Silver Spring, MD: US Food and Drug Administration; 2013.
  • Mullard A. Patient-focused drug development programme takes first steps. Nature Publishing Group. 2013;12:651–652.
  • Louviere JJ, Islam T. A comparison of importance weights and willingness-to-pay measures derived from choice-based conjoint, constant sum scales and best–worst scaling. J Bus Res. 2008;61(9):903–911.
  • Guidance for Industry: Factors to consider regarding benefitrisk in medical device product availability, compliance, and enforcement decisions. Silver Spring, MD: US Food and Drug Administration. 2017.
  • Prescription Drug User Fee Act reauthorization (PDUFA VI, Medical Device User Fee Act reauthorization (MDUFA IV), Generic Drug User Fee Act reauthorization (GDUFA II), and Biosimilar User Fee Act reauthorization (BsUFA II), in Senate Committee on Health, Education, Labor, and Pensions.
  • Roter D. The enduring and evolving nature of the patient–physician relationship. Patient Educ Couns. 2000;39(1):5–15.
  • 21st Century Cures Act in H.R. 34, 114th Congress. 2016: United States.
  • Guidance for industry and food and drug administration staff: policy for device software functions and mobile medical applications. Silver Spring, MD: US Food and Drug Administration; 2019.
  • Patient-Focused Drug Development: Methods to Identify What Is Important to Patients Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders. Draft Guidance.Silver Spring, MD: U.S. Food and Drug Administration; 2018.
  • Patient-focused drug development guidance public workshop: methods to identify what is important to patients. Silver Spring, MD: US Food and Drug Administration. 2018.
  • Patient-focused drug development discussion document: incorporating clinical outcome assessments into endpoints for regulatory decision-making. Silver Spring, MD: US Food and Drug Administration; 2019.
  • Benefit-Risk Methodology Project. London, UK: European Medicines Agency; 2009.
  • Patel MX, Doku V, Tennakoon L. Challenges in recruitment of research participants. Adv Psychiatric Treat. 2003;9(3):229–238.
  • Outcome report on pilot to involve patients in benefit/risk discussions at CHMP meetings. London, UK: European Medicines Agency; 2017.
  • Epstein RM, Franks P, Fiscella K, et al. Measuring patient-centered communication in patient-physician consultations: theoretical and practical issues. Soc Sci Med. 2005;61(7):1516–1528.
  • Garrison LP, Towse A, Bresnahan BW. Assessing a structured, quantitative health outcomes approach to drug risk-benefit analysis. Health Affairs. 2007;26(3):684–695.
  • Berwick DM. What ‘patient-centered’should mean: confessions of an extremist. Health Affairs. 2009;28(4):w555–w565.
  • Roter D, Hall JA. Doctors talking with patients/patients talking with doctors: improving communication in medical visits. Westport, CT: Greenwood Publishing Group; 2006.
  • Stakeholder Engagement Report. European Medicines Agency; 2017.
  • Putting Patients First. Parent Project Muscular Dystrophy; 2013.
  • Patients are Waiting. Parent Project Muscular Dystrophy; 2014.
  • Peay H, Hollin I, Fischer R, et al. 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):624–637.
  • Hollin I, Peay H, Bridges J. Caregiver preferences for emerging duchenne muscular dystrophy treatments: a comparison of best-worst scaling and conjoint analysis. Patient-Patient-Centered Outcomes Res. 2015;8(1):19–27.
  • Hollin I, Young C, Hanson C, et al. Developing a PATIENT-CENTERED BENEFIT-RISK SURVEY: A COMMUNITY-ENGAGED PROCEss. Value Health. 2016;19(6):751–757.
  • Hollin I, Peay H, Fischer R, et al. . Engaging patients and caregivers in prioritizing symptoms impacting quality of life for Duchenne and Becker muscular dystrophy. Quality of Life Research. 2018;27(9):2261-2273.
  • US Department of Health and Human Services, F.D.A.. Duchenne muscular dystrophy and related dystrophinopathies: developing drugs for treatment guidance for industry. Silver Spring, MD: US Food and Drug Administration. 2015.
  • Duchenne muscular dystrophy and related dystrophinopathies: developing drugs for treatment guidance for industry. Silver Spring, MD: US Food and Drug Administration; 2018.
  • Caron-Flinterman JF. A new voice in science: patient participation in decision-making on biomedical research. Amsterdam: Vrije Universiteit; 2005.
  • Key Considerations in Developing & Integrating Patient Perspectives in Drug Development. Parent Project Muscular Dystrophy & Biotechnology Innovation Organization; 2016.
  • Bridges JF, Tsai JH, Janssen E, et al. How do members of the Duchenne and becker muscular dystrophy community perceive a discrete-choice experiment incorporating uncertain treatment benefit? An application of research as an event. Patient-Patient-Centered Outcomes Res. 2018;12(2):247 - 257.
  • FDA turns down santhera pharmaceuticals’ request for accelerated approval for idebenone to treat DMD. Muscular Dystrophy Association; 2016. Available from:   https://www.mda.org/press-releases/fda-turns-down-santhera-pharmaceuticals%E2%80%99-request-accelerated-approval-idebenone-treat
  • Califf R. Scientific dispute regarding accelerated approval of Sarepta therapeutics’ eteplirsen - commissioner’s decision. Silver Spring, MD: US Food and Drug Administration. 2016. [cited 2020 Mar 2]. Available from:https://www.accessdata.fda.gov/drugsatfda_docs/nda/2016/206488_summary%20review_Redacted.pdf
  • A look at deflazacourt and exon-skipping therapies for Duchenne muscular dystrophy. Institute for Clinical and Economic Review; 2019. [cited 2020 Mar 2]. Available from: https://icer-review.org/wp-content/uploads/2018/12/DMD-RAAG-081519-004.pdf
  • DMD: Public Comment on ICER Draft Report. 2019.
  • Burkell J. What are the chances? Evaluating risk and benefit information in consumer health materials. J Med Libr Assoc. 2004;92(2):200.
  • Benefit-risk assessment in drug regulatory decision making; draft PDUFA VI implementation plan. Silver Spring, MD: US Food and Drug Administration; 2018.
  • Memorandum of Understanding Between the U.S. Department of Health and Human Services, Food and Drug Administration, and the National Organization For Rare Disorders. 2018.
  • FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. 2019.
  • Paquin RS, Fischer R, Mansfield C, et al. Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients. Orphanet J Rare Dis. 2019;14(1):102.
  • Landrum Peay H, Fischer R, Tzeng JP, et al. Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents. PLoS One. 2019;14(5):e0213649.
  • Benefit-risk assessment in rare disorders. Hackensack, NJ: Parent Project Muscular Dystrophy; 2013.
  • DeGette D, Upton F. Cures 2.0. 2019. [cited 2020 Mar 2]. Available from: https://degette.house.gov/sites/degette.house.gov/files/Cures%202.0%20Call%20to%20Action%20Document.pdf
  • BENEFIT Act S.1052. 2017, 115th Congress of the United States.
  • Ishikawa H, Hashimoto H, Kiuchi T. The evolving concept of “patient-centeredness” in patient-physician communication research. Soc Sci Med. 2013;96:147–153.
  • Rare diseases: natural history studies for drug development guidance for industry. Silver Spring, MD: U.S. Food and Drug Administration; 2019.
  • Califf RM, Ostroff S. FDA as a catalyst for translation. Sci Transl Med. 2015;7(296):296ed9.
  • Ferrari R. Writing narrative style literature reviews. Med Writing. 2015;24(4):230–235.
  • PDUFA REAUTHORIZATION PERFORMANCE GOALS AND PROCEDURES FISCAL YEARS 2018 THROUGH 2022. 2016., U.S.F.D. Administration, Editor. 2016.
  • Crossnohere NL,  Richardson DR, Reinhart C, et al. Side effects from acute myeloid leukemia treatment: results from a national survey. Curr Med Res Opin. 2019;35(11):1965-1970.
  • CDRH F. Patient preference information–voluntary submission, review in premarket approval applications, humanitarian device exemption applications, and de novo requests, and inclusion in decision summaries and device labeling. Silver Spring, MD: Food and Drug Administration Staff, and Other Stakeholders; 2016.
  • Health spending. Organisation for Economic Co-operation and Development; 2018. [cited 2020 Mar 2]. Available from: https://data.oecd.org/healthres/health-spending.htm
  • Hughes-Wilson W, Palma A, Schuurman A, et al. Paying for the orphan drug system: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments? Orphanet J Rare Dis. 2012;7(1):74.
  • Quality-adjusted life years and the devaluation of life with disability. Washington, DC: National Council on Disability; 2019.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.