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21st Century Citizen Pharma: The FDA & Patient-Focused Product Development

Published online by Cambridge University Press:  06 January 2021

Jordan Paradise
Jordan Paradise*
Affiliation:
Beazley Institute for Health Law & Policy, Loyola University Chicago School of Law
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Abstract

Perpetual debate regarding the delicate balance between access and innovation and the protection of the public health and safety dominate discussions of the United States Food and Drug Administration (“FDA”). Established chiefly as a command and control federal administrative agency, iterative changes in legislation have shaped the FDA's activity in drug, biologic, and medical device regulation over the course of the last one hundred plus years. The most recent fundamental reframing of the agency's authority and directive presented itself in the 21st Century Cures Act, reflecting an important role for patient perspectives in the regulatory process. This Article explores recent developments in patient-focused product development efforts at the FDA and offers modest insights on the increasing role of patients, and patient advocacy groups, in agency decision-making. The Article terms this era “21st century citizen pharma.”

Type
Articles
Information
American Journal of Law & Medicine , Volume 44 , Issue 2-3: Symposium - The 21st Century Cures Act: A Cure for the 21st Century? , May 2018 , pp. 309 - 327
Copyright
Copyright © American Society of Law, Medicine and Ethics and Boston University 2018

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References

1 The use of the overarching term “pharma” here is intended to include several types of medical products regulated by the FDA that were addressed in the 21st Century Cures Act of 2016, including drugs, biologics, and medical devices. It has a better ring to it than “21st Century Citizen Medical Product Development.”

2 U.S. Food & Drug Admin., Global Engagement 2 (2012), https://wayback.archive-it.org/7993/20170722112636/https://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Reports/UCM298578.pdf.

3 U.S. Food & Drug Admin., Laws Enforced By FDA, https://www.fda.gov/RegulatoryInformation/LawsEnforcedbyFDA/default.htm [https://perma.cc/SPG9-EMHX] (last updated Jul. 7, 2017).

4 The FDA regulates food (including dietary supplements), cosmetics, human and animal drugs, biological products, medical devices, products emitting radiation, and tobacco products. 21 U.S.C. § 301-399(h).

5 Wallace F. Janssen, The Story of the Laws Behind the Labels, FDA Consumer Mag., June, 1981, at 6; see generally Upton Sinclair, The Jungle (1906). The book portrayed the conditions rampant in the meat industry in Chicago and other industrial cities at the turn of the 20th century.

6 A drug is defined as “(A) articles recognized in the official United States Pharmacopoeia, official Homoeopathic Pharmacopoeia of the United States, or official National Formulary, or any supplement to any of them; and (B) articles intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease in man or other animals; and (C) articles (other than food) intended to affect the structure or any function of the body of man or other animals; and (D) articles intended for use as a component of any article specified in clause (A), (B), or (C).” Federal Food, Drug, and Cosmetic Act § 201(g), 21 U.S.C. § 321(g) (2016). A “new drug” is one that adheres to the general definition of a drug, but is not generally recognized as safe and effective among experts that are qualified by scientific experience and training or has not been used for a material extent under particular conditions. Id. § 321(p). For new drugs, premarket approval is required through the new drug approval process. Id. § 355.

7 A medical device is defined as “an instrument, apparatus, implement, machine, contrivance, implant, in vitro reagent, or other similar or related article, including any component, part, or accessory, which is—(1) recognized in the official National Formulary, or the United States Pharmacopeia, or any supplement to them, (2) intended for use in the diagnosis of disease or other conditions, or in the cure, mitigation, treatment, or prevention of disease, in man or other animals, or (3) intended to affect the structure or any function of the body of man or other animals, and which does not achieve its primary intended purposes through chemical action within or on the body of man or other animals and which is not dependent upon being metabolized for the achievement of its primary intended purposes.” Id. § 321(h).

8 The FDA is tasked with regulation of biological products in the Public Health Service Act. New drugs are chemically synthesized, while biological products are naturally-derived and defined as a “virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, protein (except any chemically synthesized polypeptide), or analogous product, or arsphenamine or derivative of arsphenamine (or any other trivalent organic arsenic compound), applicable to the prevention, treatment, or cure of a disease or condition of human beings.” Public Health Service Act § 351(i), 42 U.S.C. § 262(i) (2018).

9 Org. for Econ. Co-operation & Dev., Glossary of Statistical Terms 115 (2007). See also Claeys, Eric R., The Food and Drug Administration and the Command and Control Model of Regulation, 45 St. Louis U. L.J. 105, 127 (2004)Google Scholar (discussing the FDA's command and control approach as arising out of the Great Society and New Deal era); McCubbins, Mathew D., The Legislative Design of Regulatory Structure, 29 Am. J. Pol. Sci. 721, 726 (1985)CrossRefGoogle Scholar (describing the Environmental Protection Agency's command and control structure).

10 For a description of the legislative provisions creating PCORI, see Ass'n of Am. Med. C., Patient-Centered Outcome Provisions Summary (March 2010).

11 About the All of Us Research Program, Nat'l Insts. of Health, https://allofus.nih.gov/about/about-all-us-research-program [https://perma.cc/E2R2-ARDK].

12 About the FDA Patient Education Network, Food & Drug Admin., https://www.fda.gov/forpatients/about/default.htm (last updated Jan. 8, 2018) [https://perma.cc/QN5RV9PV].

13 For Patients, Food & Drug Admin., https://www.fda.gov/ForPatients/UCM20041944 (last updated Dec. 18, 2017) [https://perma.cc/7DM6-SAYE].

14 Sue Sutter, FDA's 21 st Century Cures Plan Gives Patient-Focused Drug Development a Boost, Pink Sheet, 16, 16 (July 17, 2017), https://pink.pharmaintelligence.informa.com/PS121056/FDAs-21supstsup-Century-Cures-Plan-Gives-PatientFocused-Drug-Development-A-Boost [https://perma.cc/227B-76KC]. For a list of those meetings conducted in 2016-2017, see Announcement of Disease Areas for Meetings Conducted in Fiscal Years 2016-2017, 80 Fed. Reg. 38,216 (July 2, 2015); Patient-Focused Drug Development: Disease Area Meetings Planned for Fiscal Years 2013-2017, Food & Drug Admin., https://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm347317.htm (last updated Feb. 21, 2018) [https://perma.cc/J2CX-W37V].

15 Reporting Serious Problems to FDA, Food & Drug Admin., https://www.fda.gov/Safety/MedWatch/HowToReport/default.htm (last updated Aug. 11, 2016) [https://perma.cc/94T3-9EXE].

16 CDRH Patient Engagement, Food & Drug Admin., https://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDRH/CDRHPatientEngagement/default.htm (last updated Dec. 12, 2017) [https://perma.cc/G99M-RC34].

17 Patient Preference Initiative, Food & Drug Admin., https://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDRH/CDRHPatientEngagement/ucm462830.htm [https://perma.cc/3NLP-2AS4].

18 The PEAC held its first meeting in October 2017. See Section IV.B, infra.

19 U.S. Food & Drug Admin., Guidance: 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 (2016), https://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM446680.pdf.

20 Id., at 6.

21 Id. For an informative blog post detailing the final guidance on PPI and patient-centered activities at FDA, see McKenzie E. Cato & Allyson B. Mullen, FDA Finalizes Guidance Regarding Patient Preferences Information for Medical Device Submissions, Hyman, Phelps & McNamara: FDA L. Blog (Sept. 27, 2016), http://www.fdalawblog.net/2016/09/fda-finalizes-guidance-regarding-patient-preference-information-for-medical-device-submissions/ [https://perma.cc/SV36-NSL6].

22 Establishment of the Patient and Care-Partner Connection; Establishment of a Public Docket; Request for Comments, 81 Fed. Reg. 78169 (Nov. 7, 2016).

23 U.S. Food & Drug Admin., 2016-2017 Strategic Priorities, Center for Devices and Radiological Health 7-9 (2016), https://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDRH/CDRHVisionandMission/UCM481588.pdf.

24 Id.

25 Id. at 7.

26 Food & Drug Admin., Value & Use of Patient-Reported Outcomes in Assessing Effects of Medical Devices, https://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDRH/CDRHVisionandMission/UCM588576.pdf.

27 2016-2017 Strategic Priorities, supra note 23, at 7.

28 See Med. Device Innovation Consortium, http://mdic.org/ [https://perma.cc/675D-7JY4].

29 Med. Device Innovation Consortium, 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, http://mdic.org/wp-content/uploads/2015/05/MDIC_PCBR_Framework_Web1.pdf.

30 Patient Affairs Staff, U.S. Food & Drug Admin., https://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/ucm589472.htm [https://perma.cc/7FV8-CWCD].

31 U.S. Food & Drug Admin., Guidance for Industry and Food and Drug Administration Staff: Factors to Consider Regarding Benefit-Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions (2016), https://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM506679.pdf.

32 Id.

33 Citizen Science, Nat'l Geographic, https://www.nationalgeographic.org/encyclopedia/citizen-science/ [https://perma.cc/D9L2-27LL].

34 See About, CitizenScience.gov, https://www.citizenscience.gov/about/ [https://perma.cc/6JKK-QUKV]. The website emphasizes crowdsourcing as one key aspect of citizen science efforts.

35 Citizen Science Definition, Citizen Sci. Ctr., http://www.citizensciencecenter.com/citizen-science-definition/ [https://perma.cc/KMW3-YUW5] (quoting the 2014 Oxford Dictionary).

36 2016-2017 Strategic Priorities, supra note 23.

37 Rebecca Skloot, The Immortal Life of Henrietta Lacks (2010).

38 Susan M. Reverby, Examining Tuskegee: The Infamous Syphilis Study and Its Legacy (2009).

39 Moore v. Regents of the Univ. of Cal., 51 Cal. 3d 120 (Cal. 1990), cert. denied, 499 U.S. 936 (1991).

40 Id. at 125.

41 Id. at 126.

42 Id. at 127.

43 Id. at 128-29.

44 Id. at 148-49.

45 Id. at 131-32.

46 Dennis McLellan, Obituaries: John Moore, 56; Sued to Share Profits from His Cells, L.A. Times, Oct. 13, 2001.

47 Moore, 51 Cal. 3d at 168-70.

48 Greenberg et al. v. Miami Children's Hosp. Research Inst., 264 F. Supp. 2d 1064 (S.D. Fla. 2003).

49 Id. at 1067-68.

50 Id. at 1068.

51 Press Release, Canavan Found., Nat'l Tay-Sachs & Allied Diseases Ass'n (Sept. 23, 2003) (on file with author).

52 Alessandra Colaianni et al., Impact of Gene Patents and Licensing Practices on Access to Genetic Testing and Carrier Screening for Tay-Sachs and Canavan Disease, 12 Genetic Med. S5-14 (2010) (citing an August 2003 Canavan Foundation Press Release that is no longer available at the link provided in the article).

53 Wash. Univ. v. Catalona, 437 F. Supp. 2d 985 (E.D. Mo. 2006).

54 Id. at 993.

55 Id. at 997.

56 Wash. Univ. v. Catalona, 490 F. 3d 667 (8th Cir. 2007), cert denied, 552 U.S. 1166 (2008).

57 For an excellent account of the history and the procedural posture of the cases, see Katherine Drabiak-Syed, Lessons from Havasupai Tribe v. Arizona State University Board of Regents: Recognizing Group, Cultural, and Dignitary Harms as Legitimate Risks Warranting Integration into Research Practice, 6 J. Health & Biomed. L. 175-226 (2010).

58 Tilousi v. Ariz. State Univ. Bd. of Regents, No. CV2005-013190, 2006 WL 4642922 (Ariz. Super. Feb. 7, 2006).

59 Havasupai Tribe v. Ariz. State Univ. Bd. of Regents, 204 P.3d 1063 (Ariz. Ct. App. 2008).

60 Id. at 1066-67.

61 Drabiak-Syed, supra note 57, at 195.

62 See, e.g., Bloom, Diana et al., The Rules of Engagement: CTTI Recommendations for Successful Collaborations Between Sponsors and Patient Groups Around Clinical Trials, 52 Therapeutic Innovation & Reg. Sci. 1, 1-8 (2017)Google ScholarPubMed; Evans, Barbara J., Barbarians at the Gate: Consumer-Driven Health Data Commons and the Transformation of Citizen Science, 42 Am. J. L. & Med. 651, 651-85 (2016)CrossRefGoogle ScholarPubMed; Hoffman, Sharona, Citizen Science: The Law and Ethics of Public Access to Medical Big Data, 30 Berkeley Tech. L. J. 1741 (2015)Google Scholar; Stodden, Victoria, Open Science: Policy Implications for the Evolving Phenomenon of User-Led Scientific Innovation, 9 J. Sci. Commission 1 (2010)Google Scholar; Paradise, Jordan, Patient Advocacy Group Collaboration in Genetic Research and the Scope of Joint Inventorship under US Patent Law, 3 Int'l J. Intell. Prop. Mgmt. 97 (2009)Google Scholar; Barbara Prainsack, The Powers of Participatory Medicine, 12 PLoS Biology e1001837 (2014).

63 See, e.g., Hall, Judith G., The Role of Patient Advocacy/Parent Support Groups, 103 S. African Med. J. 1020, 1020-1022 (2013)CrossRefGoogle ScholarPubMed; Landy, David C., How Disease Advocacy Organizations Participate in Clinical Research: A Survey of Genetic Organizations, 14 Genetics Med. 223, 223-228 (2012)CrossRefGoogle ScholarPubMed; Terry, Sharon F., Genetic Alliance Registry and Biobank: A Novel Disease-Advocacy Research-Driven Solution, 8 Personalized Med. 207, 207-213 (2011)CrossRefGoogle Scholar; Terry, Sharon et al., Advocacy Groups as Research Organisations: the PXE International Example, 8 Nat'l Rev. Genetics 157, 160 (2007)Google Scholar. See also generally Mitchell, Derick et al., Biobanking from the Patient Perspective, 1 Res. Involvement & Engagement 4 (2015)CrossRefGoogle ScholarPubMed; Simeon-Dubach, Daniel & Henderson, Marianne K., Sustainability in Biobanking, 12 Biopreservation & Biobanking 1 (2014)CrossRefGoogle ScholarPubMed.

64 Corrigan, Oonagh & Tutton, Richard, What's in a Name? Subjects, Volunteers, Participants, and Activists in Clinical Research, 1 Clinical Ethics 101, 101-104 (2006)CrossRefGoogle Scholar.

65 Sharon F. Terry et al., Advocacy Groups, supra note 63, at 160.

66 Marshall, Eliot, Patient Advocate Names Co-Inventor on Patent for the PXE Disease Gene, 305 Sci. 1226, 1226 (2008)CrossRefGoogle Scholar.

67 See Paradise, supra note 62 (providing an analysis of the patent law regarding inventorship).

68 Marshall, supra note 66.

69 H.R. 6, 114th Cong. (1st Sess. 2015).

70 Jeannie Baumann, House Energy/Commerce Unanimously OKs Bill That Would Amend HIPAA Privacy Rule, Bloomberg News (June 1, 2015), https://www.bna.com/house-energy-commerce-n17179927227/.

71 H.R. 6.

72 Jennifer Steinhauer & Robert Pear, Sweeping Health Measure, Backed by Obama, Passes Senate, N.Y. Times, Dec. 8, 2016, at A17. Several Senators cited objections to the influence of the pharmaceutical industry. Id.

73 21st Century Cures Act, Pub. L. No. 114-255, 130 Stat. 1033 (2016).

74 Michael McCaughan, FDARA, 21 st Century Cures and the Era of Perpetual FDA Reform, 79 Pink Sheet 5 (Sept. 11, 2017).

75 See, e.g., Sy Mukherjee, Everything You Need to Know About the Massive Health Reform Law that Just Passed Congress, Fortune, Dec. 7, 2016.

76 21st Century Cures Act § 3032.

77 Right to Try in Your State, Goldwater Inst., http://righttotry.org/in-your-state/ [https://perma.cc/DV3S-DV9N].

78 Id.

79 Jordan Paradise, FDA Guidance to Industry Streamlines Physicians' Compassionate Use Requests, Health Reform Watch (Feb. 10, 2015), http://www.healthreformwatch.com/2015/02/10/fda-guidance-to-industry-streamlines-physician-compassionate-use-requests/ [https://perma.cc/3WU6-A2H7].

80 21 C.F.R. § 312 (2017); Paradise, supra note 79.

81 See, e.g., Michael Cipriano, Right-To-Try Legislation Must Change Language to Narrow Spectrum of Patients, Gottlieb Says, Pink Sheet 25 (Oct. 9, 2017).

82 The Act defines “regenerative medicine therapy” as including “cell therapy, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies or products, except for those regulated solely under section 361 of the Public Health Service Act and part 1271 of title 21, Code of Federal Regulations.” 21st Century Cures Act, Pub. L. No. 114-255, § 3033, 130 Stat. 1033, 1103 (2016).

83 Id. § 3035.

84 Id. § 3036.

85 Id. § 3033.

86 Id. § 3034.

87 FDA Announces Comprehensive Regenerative Medicine Policy Framework, U.S. Food & Drug Admin. (Nov. 16, 2017), https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm585345.htm [https://perma.cc/TKY2-6Q3H].

88 U.S. Food & Drug Admin., Draft Guidance: Evaluation of Devices Used With Regenerative Medicine Advanced Therapies (Nov. 16, 2017), https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM585417.pdf.

89 Michael Cipriano, US FDA's Drug Seizures Seen as Most Significant Aspect of Stem Cell Crackdown, Pink Sheet 16 (Sept. 6, 2017).

90 See Paradise, Jordan, Cultivating Innovation in Precision Medicine through Regulatory Flexibility at the FDA, 11 N.Y.U. J.L. & Liberty 426 (2017)Google Scholar.

91 For FDA's Biomarker Qualification Program and the list of qualified biomarkers, see Biomarkers at CDER, U.S. Food & Drug Admin., https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/BiomarkerQualificationProgram/ucm535927.htm [https://perma.cc/T4XM-EYCE] (last updated Oct. 11, 2017).

92 U.S. Food & Drug Admin., IRB Waiver or Alteration of Informed Consent for Clinical Investigations Involving No More than Minimal Risk to Human Subjects: Guidance for Sponsors, Investigators, and Institutional Review Boards (July 2017).

93 The FDA has issued breakthrough devices draft guidance, implementing § 515B of FDCA created by § 3051 of the Cures Act and § 901 of FDA Reauthorization Act of 2017, Pub. L. 115-52. Breakthrough Devices Program; Draft Guidance for Industry and Food and Drug Administration Staff; Availability, 82 Fed. Reg. 49,373 (Oct. 25, 2017); U.S. Food & Drug Admin., Breakthrough Devices Program; Draft Guidance for Industry and Food and Drug Administration Staff (Oct. 25, 2017), https://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM581664.pdf.

94 The FDA has announced the publication of a guidance document framing these principles. The Least Burdensome Provisions: Concept and Principles, 82 Fed. Reg. 59,623 (Dec. 15, 2017); U.S. Food & Drug Admin., Draft Guidance: The Least Burdensome Provisions: Concept and Principles (Dec. 15, 2017), https://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM588914.pdf.

95 Patient experience data is defined as data that “are collected by any persons (including patients, family members and caregivers of patients, patient advocacy organizations, disease research foundations, researchers, and drug manufacturers)” and “are intended to provide information about patients' experience with a disease or condition, including – (A) the impact of such disease or condition, or a related therapy, on patients' lives; and (B) patient preferences with respect to treatment of such disease or condition.” 21st Century Cures Act, Pub. L. No. 114-255, § 3001, 130 Stat. 1033, 1084 (2016).

96 Id.

97 Id.

98 Id. § 3002(a)-(b).

99 Id. § 3004.

100 Sue Sutter, Patient Experience Data May Require Separate Label, Genentech Suggests, Pink Sheet 10 (Sept. 25, 2017).

101 Drug Development Tools Qualification Program, U.S. Food & Drug Admin. (June 22, 2017), https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/ [https://perma.cc/48Y6-LGZE].

102 21st Century Cures Act § 3011.

103 Id.

104 Id.

105 Id.

106 Paul, David E. & Clements, Catherine, Getting by with a Little Help from Their Friends: FDA Using External Experts to Enhance Biomarker Qualification and Enhance Precision Medicine, 72 Food & Drug L.J. 660 (2017)Google Scholar.

107 21st Century Cures Act § 3022.

108 Id.

109 Id.

110 Id.

111 Id.

112 Id. § 3051.

113 Food and Drug Administration Safety and Innovation Act of 2012, Pub. L. No. 112-144, § 902, 126 Stat. 993, 1086-1087 (2012). Breakthrough therapy status signals that the drug is progressing through clinical trials subject to the statutory provisions provided for breakthrough therapies, not that the drug has been approved by the FDA. The FDA defines a breakthrough therapy as a drug “intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.” Fact Sheet: Breakthrough Therapies, U.S. Food & Drug Admin. (Dec. 10, 2014), https://www.fda.gov/RegulatoryInformation/LawsEnforcedbyFDA/SignificantAmendmentstotheFDCAct/FDASIA/ucm329491.htm [https://perma.cc/JD2G-3UUU]. This language tracks that used in the statute.

114 U.S. Food & Drug Admin., Guidance for Industry: Expedited Programs for Serious Conditions – Drugs and Biologics (May 2014), http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf.

115 A table within the FDA's breakthrough therapy guidance document compares the four expedited mechanisms. Id. at 11.

116 Congress directs the FDA to focus breakthrough status on medical devices “that provide for more effective treatment or diagnosis of life-threatening or irreversibly debilitating human disease or conditions … that represent breakthrough technologies … for which no approved or cleared alternatives exist … that offer significant advantages over existing approved or cleared alternatives, including the potential, compared to existing approved alternatives, to reduce or eliminate the need for hospitalization, improve patient quality of life, facilitate patients' ability to manage their own care (such as through self-directed personal assistance), or establish longterm clinical efficiencies; or … the availability of which is in the best interest of patients.” 21st Century Cures Act § 3051.

117 Breakthrough Devices Program, supra note 93, at 1.

118 Enhancing Patient Engagement Efforts Across the Food and Drug Administration; Establishment of a Public Docket; Request for Comments, 82 Fed. Reg. 13,632 (Mar. 14, 2017).

119 Id. at 13,633.

120 Id. at 13,633-34.

121 Derrick Gingery, Patient Advocates Continue to Push US FDA for Central Office, 79 Pink Sheet 1, 3-4 (Sept. 11, 2017).

122 Brenda Sandburg, Novartis CAR-T Therapy's Swift Approval Aided by REMS and New US FDA Review Model, 79 Pink Sheet 1, 4 (Sept. 4, 2017).

123 Id. (quoting Commissioner Scott Gottlieb, M.D.).

124 FDA Therapy Brings First Gene Therapy to the United States, U.S. Food & Drug Admin. (Aug. 30, 2017), https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm [https://perma.cc/L2RG-J9U2]; see Michael Cipriano, FDA's Gottlieb Pushing ‘Seamless’ Clinical Trials for Faster Development, Pink Sheet 9 (Sept. 18, 2017).

125 U.S. Food & Drug Admin., Plan for Issuance of Patient-Focused Drug Development Guidance Under 21st Century Cures Act Title III Section 3002 (May 2017), https://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm563618.pdf.

126 21st Century Cures Act, Pub. L. No. 114-255, § 3032, 130 Stat. 1033, 1084 (2016).

127 Derrick Gingery, FDA Ends Regularly Scheduled Patient-Focused Drug Development Meetings, Pink Sheet 14 (Oct. 2, 2017).

128 U.S. Food & Drug Admin., supra note 125, at 4.

129 Id. at 3.

130 Sutter, supra note 14, at 17.

131 U.S. Food & Drug Admin., Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices: Guidance for Industry and Food and Drug Administration Staff (Aug. 31, 2017), https://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm513027.pdf.

132 For a discussion of agency considerations leading up to issuance of the guidance, see Cole Werble, Real-World Evidence: Advice, Principles and Examples Emerge from FDA, Pink Sheet 7 (Oct. 16, 2017).

133 Id. at 10.

134 Id. at 12.

135 Statement by FDA Commissioner Scott Gottlieb, M.D. on New Steps by FDA to Advance Patient Engagement in the Agency's Regulatory Work, U.S. Food & Drug Admin (Oct. 11, 2017), https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm579842.htm [https://perma.cc/E7RH-5J6S].

136 Id.; U.S. Food & Drug Admin., Summary of the Patient Engagement Advisory Committee Meeting (2017), https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/PatientEngagementAdvisoryCommittee/UCM580449.pdf.

137 Statement by FDA Commissioner Scott Gottlieb, M.D., supra note 135; Patient Engagement Advisory Committee, U.S. Food & Drug Admin., https://www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/PatientEngagementAdvisoryCommittee/default.htm [https://perma.cc/U46U-JRE2] (last updated July 25, 2017) (giving advice to the Commissioner or designee, on complex issues relating to medical devices, the regulation of devices, and their use by patients).

138 Advisory Committee; Patient Engagement Advisory Committee, 82 Fed. Reg. 50,882 (Nov. 2, 2017).

139 U.S. Food & Drug Admin., supra note 93; Breakthrough Devices Program; Draft Guidance for Industry and Food and Drug Administration Staff; Availability, 82 Fed. Reg. 49,373.

140 FDA Reauthorization Act of 2017, Pub. L. No. 115-52, § 901, 131 Stat. 1005, 1077-78 (2017).

141 U.S. Food & Drug Admin., supra note 93.

142 See, e.g., Thomas M. Burton, FDA Plans New Medical Device Approval Process, Wall St. J. (Dec. 13, 2017), https://www.wsj.com/articles/fda-plans-new-medical-device-approval-processes-1513000850?elq_cid=2418807&x_id=.

143 Patient Affairs Staff, supra note 30.

144 Patient Engagement Collaborative, U.S. Food & Drug Admin., https://www.fda.gov/ForPatients/PatientEngagement/ucm506248.htm [https://perma.cc/C8WX-4QPD] (last updated Dec. 22, 2017).

145 Patient Affairs Staff, supra note 30.

146 Id.

147 Strategic Plan, Clinical Trials Transformation Initiative (May 2016), https://www.ctticlinicaltrials.org/who-we-are/strategic-plan [https://perma.cc/ZCX8-2U87].

148 U.S. Food & Drug Admin., supra note 92. This guidance implements the 21st Century Cures Act, Pub. L. No. 114-255, § 3024, 130 Stat. 1033, 1099 (amending §§ 520(g)(3) & 505(i)(4) of the FDCA).

149 Sy Mukherjee, The FDA Just Made Its Most Controversial Drug Approval of the Year, Fortune (Sept. 19, 2016), http://fortune.com/2016/09/19/fda-drug-approval-exondys-51/ [https://perma.cc/SU4GRS4H].

150 Biggar, W. Douglas, Duchenne Muscular Dystrophy, 27 Pediatrics Rev. 83, 84 (2006)CrossRefGoogle ScholarPubMed.

151 Jeff Buchanan, Bio Execs Talk Patient Advocacy, Duchenne Approval at BioForward Panel, Exome (Sept. 28, 2016), https://www.xconomy.com/wisconsin/2016/09/28/bio-execs-talk-patient-advocacy-duchenne-approval-at-bioforward-panel/ [https://perma.cc/B6FK-8BQC].

152 Robert Weisman, FDA Approves Sarepta's Disputed Drug, Overruling Staffers and Advisors, Bos. Globe (Sept. 19, 2016), https://www.bostonglobe.com/business/2016/09/19/fda-approves-duchenne-drug-overturning-staff-and-advisers/mKVY1HQ0Fn1Mcs2WJQWS0J/story.html.

153 Mukherjee, supra note 149.

154 Weisman, supra note 152.

155 Id.

156 Robert Weisman, Muscular Dystrophy Drug Advocates Plan to Pack Crucial Meeting, Bos. Globe (Apr. 18, 2016), https://www.bostonglobe.com/business/2016/04/18/drug-advocates-plan-pack-crucial-meeting/OMb3xupTONfQm61CmDptTM/story.html.

157 Weisman, supra note 152.

158 Id.

159 Buchanan, supra note 151.