Abstract
Get full access to this article
View all access options for this article.
References
1.U.S. Food and Drug Administration News Release , “FDA News Release: FDA Approval Brings First Gene Therapy to the United States,” FDA , August 30 , 2017 , available at <https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm> (last visited Nov. 30, 2017 ).
2.Novartis , “Novartis CAR-T Cell Therapy CTL019 Receives FDA Breakthrough Therapy Designation for Treatment of Adult Patients with r/r DLBCL,” April 18 , 2017 , available at <https://www.novartis.com/news/media-releases/novartis-cart-cell-therapy-ctl019-receives-fda-breakthrough-therapy-designation> (last visited Nov. 30, 2017 ); U.S. Food and Drug Administration , Transcript for the July 12, 2017 Meeting of the Oncologic Drugs Advisory Committee (ODAC) (PM Session) , July 12 , 2017 , at 136 -140 , available at <https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/OncologicDrugsAdvisoryCommittee/UCM573721.pdf> (last visited Nov. 30, 2017 ).
3.
R. Stein
, “‘Living Drug’ That Fights Cancer By Harnessing Immune System Clears Key Hurdle,” National Public Radio , July 12 , 2017 , available at <http://www.npr.org/sections/health-shots/2017/07/12/536812206/living-drug-that-fights-cancer-by-harnessing-the-immune-system-clears-key-hurdle> (last visited Nov. 30, 2017 ); See U.S. Food and Drug Administration, supra note 2, at 137.
4.See U.S. Food and Drug Administration News Release, supra note 1 .
5.See U.S. Food and Drug Administration News Release, supra note 1 .
6.ClinicalTrials.gov , “Determine Efficacy and Safety of CTL019 in Pediatric Patients With Relapsed and Refractory B-cell ALL (ELIANA),” available at <https://clinicaltrials.gov/ct2/show/NCT02435849> (last visited Nov. 30, 2017 ).
7.U.S. Food and Drug Administration , FDA Briefing Document: Oncologic Drugs Advisory Committee Meeting, BLA 125646:Tisagenlecleucel, Novartis Pharmaceuticals Corporation , at 43 , available at <https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/OncologicDrugsAdvisoryCommittee/UCM566166.pdf> (last visited Nov. 30, 2017 ).
8.See U.S. Food and Drug Administration, supra note 7, at 16 .
9.
D.W. Lee
, et al. “Current Concepts in the Diagnosis and Management of Cytokine Release Syndrome,” Blood 124 , no. 2 (2014 ): 188 -195 , at 191.
10.
M. O’Leary
, U.S. Food & Drug Administration Clinical Presentation: BLA125646, Tisagenlecleucel, Novartis Pharmaceuticals Corporation , July 12 , 2017 , at 23 , available at <https://www.fda.gov/downloads/Advisory-Committees/CommitteesMeetingMaterials/Drugs/OncologicDrugsAdvisoryCommittee/UCM567383.pdf> (last visited Nov. 30, 2017 ).
11.See O’Leary, supra note 10, at 23 .
12.See O’Leary, supra note 10, at 24 .
13.See U.S. Food and Drug Administration, supra note 1 .
14.
A. Lash
, “After Trial Deaths, Juno Pivots and Scraps Lead CAR-T Therapy,” Exome , March 1 , 2017 , available at <http://www.xconomy.com/seattle/2017/03/01/after-trial-deaths-juno-pivots-and-scraps-lead-car-t-therapy/#> (last visited Nov. 30, 2017 ).
15.
S. Brozak
, “Gilead-Kite: A New Transformative Deal for Biotech,” Forbes , August 30 , 2017 , available at <https://www.forbes.com/sites/stephenbrozak/2017/08/30/gilead-kite-a-new-transformative-deal-and-maybe-the-new-future-of-healthcare-deals/#3269e0165d49> (last visited Nov. 30, 2017 ); Business Wire , “Kite Presents Ongoing Response Rate in Plenary Session from its Pivotal CAR-T Trial of Axicabtagene Ciloleucel in Patients with Aggressive Non-Hodgkin Lymphoma at the 2017 American Association of Cancer Research Annual Meeting,” Business Wire , April 2 , 2017 , available at <http://www.businesswire.com/news/home/20170402005096/en/Kite-Presents-Ongoing-Response-Rate-Plenary-Session> (last visited Nov. 30, 2017 ).
16.
Novartis Press Release, “Novartis Receives First Ever FDA Approval for a CAR-T Cell Therapy, Kymriah(TM) (CTL019), for Children and Young Adults with B-cell ALL that is Refractory or has Relapsed at Least Twice,” August 30, 2017, available at <https://www.novartis.com/news/media-releases/novartis-receives-first-ever-fda-approval-car-t-cell-therapy-kymriahtm-ctl019> (last visited Nov. 30, 2017 );
J.M. Broderick
, “FDA OKs Tisagenlecleucel in ALL as First Approved CAR T-Cell Therapy,” OncLive , August 30 , 2017 , available at <http://www.onclive.com/web-exclusives/fda-oks-tisagenlecleucel-in-allas-first-approved-car-tcell-therapy> (last visited No. 30, 2017 ).
17.
D. Grady
, “F.D.A. Panel Recommends Approval for Gene-Altering Leukemia Treatment,” New York Times , July 12 , 2017 , available at <https://www.nytimes.com/2017/07/12/health/fda-novartis-leukemia-gene-medicine.html?_r=0> (last visited Nov. 30, 2017 );
S. Mukherjee
, “FDA Panel Backs Groundbreaking Novartis Treatment That Turns Your Cells Into Cancer Killers,” Fortune , July 12 , 2017 , available at < http://fortune.com/2017/07/12/fda-novartis-car-t-cancer-treatment/> (last visited Nov. 30, 2017 ).
18.
D. Roland
and
P. Loftus
“FDA Approves Pioneering Cancer Treatment With $475,000 Price Tag,” Wall Street Journal , August 30 , 2017 , available at <https://www.wsj.com/articles/fda-approves-first-gene-therapy-in-us-1504108512> (last visited September 7, 2017 ).
19.
Patients for Affordable Drugs Press Release, “Statement From Patients For Affordable Drugs On Novartis’ Drug Price,” Patients For Affordable Drugs, August 30, 2017, available at <http://www.patientsforaffordable-drugs.org/2017/08/30/statement-from-patients-for-affordable-drugs-on-novartis-drug-price/> (last visited Nov. 30, 2017 );
A. Weintraub
, “Novartis CEO Opens Door to Cancer Patient Demanding ‘Fair’ CAR-T Pricing,” FiercePharma , August 21 , 2017 , available at <http://www.fiercepharma.com/financials/patient-advocacy-group-demands-fair-car-t-pricing-from-novartis> (last visited Nov. 30, 2017 ).
20.See Novartis Press Release, supra note 16 .
21.See Novartis Press Release, supra note 16 .
22.
A. Kaltenboeck
and
P.B. Bach
“Outcomes-Based Drug Contracts Do Not Move Us Closer to Value,” Morning Consult , June 21 , 2017 , available at <https://morningconsult.com/opinions/outcomes-based-drug-contracts-not-move-us-closer-value/> (last visited Nov. 30, 2017 ).
23.
B. Berkrot
, “Novartis Gene Therapy Approval Signals New Cancer Treatment Era,” Reuters , August 30 , 2017 , available at <https://www.reuters.com/article/us-novartis-fda/novartis-gene-therapy-approval-signals-new-cancer-treatment-era-idUSKCN1BA-1ZY> (last visited Nov. 30, 2017 ).
24.Novartis Press Release , “Novartis CAR-T Cell Therapy CTL019 Receives FDA Breakthrough Therapy Designation for Treatment of Adult Patients with r/r DLBCL,” Novartis , April 18 , 2017 , available at <https://www.novartis.com/news/media-releases/novartis-car-t-cell-therapy-ctl019-receives-fda-breakthrough-therapy-designation> (last visited Nov. 30, 2017 ).
25.Experts in Chronic Myeloid Leukemia , “The Price of Drugs for Chronic Myeloid Leukemia (CML) is a Reflection of the Unsustainable Prices of Cancer Drugs: From the Perspective of a Larger Group of CML Experts,” Blood 121 , no. 22 (2013 ): 4439 -4442 , at 4440;
R. Pazdur
, Letter to Robert A. Miranda, Associate Director Drug Regulatory Affairs at Novartis Pharmaceuticals Corporation , 2001 , available at <https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2002/21335s001ltr.pdf> (last visited Nov. 30, 2017 ).
26.
A. Tversky
and
D. Kahneman
“Judgement under Uncertainty: Heuristics and Biases,” Science 185 , no. 4157 (1974 ): 1124 -1131 , at 1129.
27.21 U.S.C. § 360ff (2016) .
28.
A. Gaffney
,
RAC, M. Mezher
,
Z. Brennan
“Regulatory Explainer: Everything You Need to Know About FDA’s Priority Review Vouchers,” Regulatory Affairs Professionals Society , August 30 , 2017 , available at <http://www.raps.org/Regulatory-Focus/News/2015/07/02/21722/Regulatory-Explainer-Everything-You-Need-to-Know-About-FDA%E2%80%99s-Priority-Review-Vouchers/> (last visited Nov. 30, 2017 ).
29.National Cancer Institute , NCI Guidelines For Investigators: Adverse Event Reporting Requirements For DCTD (CTEP and CIP) and DCP INDs A IDEs , March 28 , 2011 , at 22 -23 , available at <https://ctep.cancer.gov/protocolDevelopment/electronic_applications/docs/aeguidelines3-29-11.pdf> (last visited Nov. 30, 2017 ).
30.
W.W. Bryan
, “Letter to Dr. Patel on STN: BL 125646/0,” August 30 , 2017 , available at <https://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM574106.pdf> (last visited Nov. 30, 2017 ).
31.
Z. Brennan
, “‘Historic Action’: FDA Approves First CAR-T Therapy,” Regulatory Affairs Professionals Society , August 30 , 2017 , available at <http://raps.org/Regulatory-Focus/News/2017/08/30/28367/Historic-Action-FDA-Approves-First-CAR-T-Therapy/> (last visited Nov. 30, 2017 )
32.
X. Lin, Statistical Review, Biologics License Application 125646/0, U.S. Food and Drug Administration, at 24, available at <https://www.fda.gov/BiologicsBloodVaccines/CellularGene-TherapyProducts/ApprovedProducts/ucm573706.htm>; see ClinicalTrials.gov, supra note 5.
33.See U.S. Food and Drug Administration, supra note 7, at 40; see Clinical-Trials.gov, supra note 6 .