References
- Holbein ME, Berglund JP, Weatherwax K, et al. Access to investigational drugs: FDA expanded access programs or “Right-to-Try” legislation? Clin Transl Sci. 2015;8(5):526–532.
- Saiyed MM, Ong PS, Chew L. Off-label drug use in oncology: a systematic review of literature. J Clin Pharm Ther. 2017;42(3):251–258.
- Code of federal regulations title 21. SERVICES DOHAH, editor; 2017. Available from: https://www.ecfr.gov/cgi-bin/text-idx?SID=3ee286332416f26a91d9e6d786a604ab&mc=true&tpl=/ecfrbrowse/Title21/21tab_02.tpl
- The history of Food and Drug Administration Agency. Available from: https://www.fda.gov/AboutFDA/WhatWeDo/History/default.htm
- 21 U.S.C. § 355. PLN-, 76 Stat. 780. [Safety and efficacy amendments to the Federal Food, Drug and Cosmetic Act]; 1962.
- 21 U.S.C. ch. 9 § 301 et seq. [Kefauver-Harris Amendment].
- Richey EA, Lyons EA, Nebeker JR, et al. Accelerated approval of cancer drugs: improved access to therapeutic breakthroughs or early release of unsafe and ineffective drugs? J Clin Oncol. 2009;27(26):4398–4405.
- Kaitin KI, DiMasi JA. Pharmaceutical innovation in the 21st century: new drug approvals in the first decade, 2000–2009. Clin Pharmacol Ther. 2011;89(2):183–188.
- Lanthier ML, Sridhara R, Johnson JR, et al. Accelerated approval and oncology drug development timelines. J Clin Oncol. 2010;28(14):e226–e227. author reply e8.
- Downing NS, Aminawung JA, Shah ND, et al. Regulatory review of novel therapeutics–comparison of three regulatory agencies. N Engl J Med. 2012;366(24):2284–2293.
- Ns D, Ad Z, Ross JS. Regulatory review of new therapeutic agents - FDA versus EMA, 2011–2015. N Engl J Med. 2017;376(14):1386–1387.
- FDA. Investigational new drug, antibiotic, and biologic drug product regulations: treatment use and sale. Fed Regist. 1987;52:19466–19467.
- Food and Drug Administration, HHS. Expanded access to investigational drugs for treatment use: final rule. Fed Regist. 2009;74(155):40900–40945.
- Trotta F, Leufkens HG, Schellens JH, et al. Evaluation of oncology drugs at the European Medicines Agency and US Food and Drug Administration: when differences have an impact on clinical practice. J Clin Oncol. 2011;29(16):2266–2272.
- FDA. Available from: https://blogs.fda.gov/fdavoice/index.php/2017/10/expanded-access-fda-describes-efforts-to-ease-application-process/
- DiMasi JA. Risks in new drug development: approval success rates for investigational drugs. Clin Pharmacol Ther. 2001;69:297–307.
- Martell RE, Sermer D, Getz K, et al. Oncology drug development and approval of systemic anticancer therapy by the U.S. Food and Drug Administration. Oncologist. 2013;18(1):104–111.
- Jarow JP, Lemery S, Bugin K, et al. Access of investigational drugs: the experience of the center of drug evaluation and research over a 10-year period. Ther Innov Regul Sci. 2016;50(6):705–709.
- Planchard D, Smit EF, Groen HJM, et al. Dabrafenib plus trametinib in patients with previously untreated BRAFV600E-mutant metastatic non-small-cell lung cancer: an open-label, phase 2 trial. Lancet Oncol. 2017;18(10):1307–1316.
- Bang YJ, Van Cutsem E, Feyereislova A, et al. Trastuzumab in combination with chemotherapy versus chemotherapy alone for treatment of HER2-positive advanced gastric or gastro-oesophageal junction cancer (ToGA): a phase 3, open-label, randomised controlled trial. Lancet. 2010;376(9742):687–697.
- Tsimberidou AM. Initiative for molecular profiling and advanced cancer therapy and challenges in the implementation of precision medicine. Curr Probl Cancer. 2017;41(3):176–181.
- Tsimberidou AM, Iskander NG, Hong DS, et al. Personalized medicine in a phase I clinical trials program: the MD Anderson Cancer Center initiative. Clin Cancer Res. 2012;18(22):6373–6383.
- Tsimberidou AM, Wen S, Hong DS, et al. Personalized medicine for patients with advanced cancer in the phase I program at MD Anderson: validation and landmark analyses. Clin Cancer Res. 2014;20(18):4827–4836.
- FDA expanded access. Available from: https://www.fda.gov/downloads/ForIndustry/DevelopingProductsforRareDiseasesConditions/OOPDNewsArchive/UCM294794.pdf.
- Black L. Experimental breast cancer treatments and health insurance coverage. The Virtual Mentor. 2007;9(1):34–37.
- Falit BP, Gross CP. Access to experimental drugs for terminally ill patients. Jama. 2008;300(23):2793–2795.
- Mack GS. Expanded access rules pose quandary for drug developers. Nat Biotechnol. 2009;27(10):871–872.
- Kroll D 2014 March 12. Available from: https://www.forbes.com/sites/davidkroll/2014/03/12/why-everyone-has-a-stake-in-the-chimerix-drug-offered-to-josh-hardy-cmx001-brincidofovir/#6d2a1c7a40da
- Electronic Code of Federal Regulations, Part 314.510. US Food and Drug administration silver spring. MD US Food and Drug Administration; 2008. Available from: https://www.ecfr.gov/cgi-bin/text-idx?SID=3ee286332416f26a91d9e6d786a604ab&mc=true&tpl=/ecfrbrowse/Title21/21tab_02.tpl
- Jc D. Connecting patients with experimental drugs. Wall Street J. 2012, October22. Available from: https://www.wsj.com/articles/should-the-fda-relax-rules-on-compassionate-access-to-new-drugs-1491962521
- United States v. Rutherford, 442 U.S. 544; 1979.
- Abigail Alliance for Better Access to Developmental Drugs v. von Eschenbach, 495 F.3d 695 (D.C. Cir. 2007).
- Dahl v. HEM Pharms, 7 F.3d 1399 (9th Cir. 1993).
- Mello MM, Joffe S. Compact versus contract–industry sponsors’ obligations to their research subjects. N Engl J Med. 2007;356(26):2737–2743.
- Horstmann E, McCabe MS, Grochow L, et al. Risks and benefits of phase 1 oncology trials, 1991 through 2002. N Engl J Med. 2005;352(9):895–904.
- Freireich EJ, Kurzrock R. The role of investigational therapy in management of patients with advanced metastatic malignancy. J Clin Oncol. 2009;27(2):304–306.
- Woloshin S, Schwartz LM, Welch HG. Patients and medical statistics. Interest, confidence, ability. J General Internal Med. 2005;20(11):996–1000.
- Kolva E, Rosenfeld B, Brescia R, et al. Assessing decision-making capacity at end of life. Gen Hosp Psychiatry. 2014;36(4):392–397.
- Leonard EW. Right to experimental treatment: FDA new drug approval, constitutional rights, and the public’s health. J Law, Med Ethics. 2009;37(2):269–279.
- Right to try model legislation. Available from: https://goldwater-media.s3.amazonaws.com/cms_page_media/2016/1/5/GoldwaterInstituteRighttoTryModel.pdf
- Dennis B, Cha AE “Right to try” laws spur debate over dying patients’ access to experimental drugs. Washington Post. May 16, 2014
- H.R. 891, Gen. Assemb., Reg. Sess. (La. 2014) [“right-to-try” laws, manufactures can provide experimental medicines to terminally ill patients without FDA authorization].
- H.R. 1685, Gen. Assemb., Reg. Sess. (Mo. 2014) [“right-to-try” laws, manufactures can provide experimental medicines to terminally ill patients without FDA authorization].
- H.R. 1281, Gen. Assemb., Reg. Sess. (Colo. 2014) [“right-to-try” laws, manufactures can provide experimental medicines to terminally ill patients without FDA authorization].
- 21 U.S.C. § 823 [United States Code (USC) Controlled Substances Act].
- 21 C.F.R. § 1301.36 [Code of Federal Regulations, suspension or revocation of registration].
- Walker MJ, Rogers WA, Entwistle V. Ethical justifications for access to unapproved medical interventions: an argument for (limited) patient obligations. Am J Bioethics. 2014;14(11):3–15.
- Chapman A. Proposal for patient obligations for access to unapproved medical interventions: both too much and not enough. Am J Bioethics. 2014;14(11):25–26.
- Darrow JJ, Sarpatwari A, Avorn J, et al. Practical, legal, and ethical issues in expanded access to investigational drugs. N Engl J Med. 2015;372(3):279–286.
- Magnus D. Compassion and research in compassionate use. Am J Bioethics. 2014;14(11):1–2.
- Mackey TK, Schoenfeld VJ. Going “social” to access experimental and potentially life-saving treatment: an assessment of the policy and online patient advocacy environment for expanded access. BMC Med. 2016;14:17.
- Enhanced Clinical Trial Design Act of 2017. S. 1048, H.R. 2430.
- Kim ES, Bruinooge SS, Roberts S, et al. Broadening eligibility criteria to make clinical trials more representative: American Society of Clinical Oncology and friends of cancer research joint research statement. J Clin Oncol. 2017;35(33):3737–3744.
- Jin S, Pazdur R, Sridhara R. Re-evaluating eligibility criteria for oncology clinical trials: analysis of investigational new drug applications in 2015. J Clin Oncol. 2017;35(33):3745–3752.
- Scoggins JF, Ramsey SD. A national cancer clinical trials system for the 21st century: reinvigorating the NCI Cooperative Group Program. J Natl Cancer Inst. 2010;102(17):1371.
- Korphaisarn K, Kopetz S. BRAF-directed therapy in metastatic colorectal cancer. Cancer J. 2016;22(3):175–178.
- Tsimberidou AM, Eggermont AM, Schilsky RL. Precision cancer medicine: the future is now, only better. Am Soc Clin Oncol Educ Book. 2014;61–69.
- Tsimberidou AM, Hong DS, Ye Y, et al. Initiative for Molecular Profiling and Advanced Cancer Therapy (IMPACT): an MD Anderson Precision Medicine Study. JCO Precis Oncol. 2017;2017. doi: 10.1200/PO.17.00002.
- IMPACT 2. Available from: https://www.clinicaltrials.gov/ct2/show/NCT02152254
- TAPUR Study. Available from: https://clinicaltrials.gov/ct2/show/NCT02693535
- The Drug Rediscovery Protocol (DRUP Trial) (DRUP). Available from: https://clinicaltrials.gov/show/NCT02925234
- Buzyn A, Blay JY, Hoog-Labouret N, et al. Equal access to innovative therapies and precision cancer care. Nat Rev Clin Oncol. 2016;13(6):385–393.
- https://clinicaltrials.gov. Available from: https://clinicaltrials.gov/ct2/show/NCT02034981?term=crizotinib&draw=1&rank=1
- Mullard A. PRIME time at the EMA. Nat Rev Drug Discov. 2017;16(4):226–228.
- PRIME. Available from: http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000660.jsp
- Em A. Cluster activities. Available from: http://www.ema.europa.eu/ema/index.jsp?curl=pages/partners_and_networks/general/general_content_000655.jsp&mid=WC0b01ac0580953d98.
- Dal Pan GJ, Arlett PR. The US Food and Drug Administration-European Medicines Agency collaboration in pharmacovigilance: common objectives and common challenges. Drug Saf. 2015;38(1):13–15.