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v Cancer, a pediatric cancer advocacy organization, launched the Compassionate Use Navigator to assist physicians and guide families about the application process. Since then, FDA simplified the application process, but stressed that it cannot require a manufacturer to provide a product. FDA receives about 1,500 expanded access requests per year and authorizes 99% of it.
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the FDA annually; this data may be helpful with regard to getting the drug or device approved, or may be harmful, should unexpected adverse events occur. The manufacturer remains legally liable as well. If the manufacturer chooses to charge for the investigational product, that price influences later discussions about the price if the product is approved for marketing.
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was declined by the drug manufacturer, Chimerix. The company reversed its decision after pressure from cancer advocacy organizations, and Josh received the drug that saved his life. Hardy later passed away in September 2016 due to complications related to his underlying cancer diagnosis. In 2016 Kids
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issued guidelines that members may follow. Each country has its own regulations, and they vary. In the UK, for example, the program is called "early access to medicine scheme" or EAMS and was established in 2014. If a company that wants to provide a drug under EAMS, it must submit its Phase I data to
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in the mid-1980s. At its peak, fifteen people received the drug. 43 people were approved for the program, but 28 of the people whose doctors completed the necessary paperwork never received any cannabis. The program stopped accepting new people in 1992 after public health authorities concluded there
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Those seeking CSP are required to provide the following information; estimated amount of the unregistered drug the patient, the "licensed drug/device establishment through which the unregistered drug may be procured", and "the names and address of the specialists qualified and authorized to use the
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In some cases, it may be in the manufacturer's commercial interest to provide access under an EA program; this is a way, for example, for a company to make money before the drug or device is approved. Companies must provide data collected from people getting the drug or device under EA programs to
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Companies sometimes make use of expanded programs in Europe even after they receive EMA approval to market a drug, because drugs also must go through regulatory processes in each member state, and in some countries this process can take nearly a year; companies can start making sales earlier under
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The sponsor (generally the company developing the investigational product for commercial use) or the clinical investigator (or the patient's physician in the case of a single patient expanded access request) submits a clinical protocol (a document that describes the treatment plan for the patient)
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In general the person and their doctor must apply for access to the investigational product, the company has to choose to cooperate, and the medicine's regulatory agency needs to agree that the risks and possible benefits of the drug or device are understood well enough to determine if putting the
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In November 2001 the Abigail Alliance for Better Access to Developmental Drugs was established by Frank Burroughs in memory of his daughter, Abigail. The Alliance seeks broader availability of investigational drugs on behalf of people with terminal illnesses. It is best known for a legal case,
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that permit manufacturers to provide experimental medicines to terminally ill people without US FDA authorization. Legal, medical, and bioethics scholars, including Jonathan Darrow and Arthur Caplan, have argued that these state laws have little practical significance because people can already
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is obligated to pay for people who fit the criteria to have access to the drug. As of 2016, governments also paid for early access to drugs in Austria, Germany, Greece, and Spain. Since 2021, France has a system of early and expanded access separated in two systems: AAC and AAP.
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In the US, actual provision of the drug depends on the manufacturer's willingness to provide it, as well as the person's ability to pay for it; it is the company's decision whether to require payment or to provide the drug or device for free. The manufacturer can only charge
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In the US, compassionate use started with the provision of investigational medicine to certain patients in the late 1970s, and a formal program was established in 1987 in response to HIV/AIDS patients requesting access to drugs in development. An important legal case was
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The closure of the program during the height of the AIDS epidemic led to the formation of the medical cannabis movement in the United States, a movement which initially sought to provide cannabis for treating anorexia and wasting syndrome in people with AIDS.
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became the legal basis for the FDA's compassionate IND program. People were only allowed to use cannabis under the program who had certain conditions, like glaucoma, known to be alleviated with cannabis. The scope was later expanded to include people with
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person at risk has sufficient potential benefit. In some countries the government will pay for the drug or device, but in many countries the person must pay for the drug or device, as well as medical services necessary to receive it.
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declined to hear their appeal. This decision left standing the appellate court decision that people who are terminal ill patients have no legal right to demand "a potentially toxic drug with no proven therapeutic benefit".
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In the Philippines, the usage of unregistered drugs may be allowed through a doctor, a specialist, or health institution or society obtaining a specific compassionate use permit (CSP) from the country's
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In March 2014, Josh Hardy, a 7-year-old boy from Virginia, made national headlines that sparked a conversation on pediatric access to investigational drugs when his family's request for
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that is consistent with FDA's statute and applicable regulations for INDs or investigational device exemption applications (IDEs), describing the use of the investigational product; and
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The FDA determines that providing the investigational product will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval;
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As of 2016, regulation of access to pharmaceuticals that were not approved for marketing was handled on a country by country basis, including in the European Union, where the
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and apply for what is called a "promising innovative medicine" (PIM) designation. If that designation is approved, the data is reviewed, if that review is positive, the
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Phillips, Lisa (4 September 2008). "Contract Law and Ethical Issues Underscore the Latest Lawsuit About Access to Experimental Drugs for Duchenne Muscular Dystrophy".
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The FDA determines that there is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance;
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for small group or larger expanded access programs. To the extent that a doctor or clinic is required for use of the drug or device, they too may require payment.
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issued guidelines for national regulatory agencies to follow. In the US, Europe, and the EU, no company could be compelled to provide a drug or device that it was
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Balasubramanian, G.; Morampudi, S.; Chhabra, P.; Gowda, A.; Zomorodi, B. (2016), "An overview of Compassionate Use Programs in the European Union member states",
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In the US, one of the earliest expanded access programs was a compassionate use IND that was established in 1978, which allowed a limited number of people to use
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The person's physician determines that there is no comparable or satisfactory therapy available to diagnose, monitor, or treat the patient's disease or condition.
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obtain pre-approval access through the FDA's expanded access program, and because the FDA is generally not the limiting factor in obtaining pre-approval access.
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Companies sometimes provide drugs under these programs to people who were in clinical trials and who responded to the drug, after the clinical trial ends.
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That the probable risk to the person from the investigational product is not greater than the probable risk from the disease or condition.
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to argue against criminal charges of marijuana cultivation that had been brought against him, because his use of cannabis was deemed a
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In the US as of 2018, people could try obtain unapproved drugs or medical devices that were in development under specific conditions.
38:, by people with serious or life-threatening conditions who do not meet the enrollment criteria for the clinical trial in progress. 1314: 1388: 595: 244: 332:
administration's policies. As of 2011, four people continued to receive cannabis from the government under the program.
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Gerasimov, Elena; Zhang, Lindy; Moerdler, Scott; Roth, Michael; Goodman, Nancy; Weiser, Daniel (2016-07-15).
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for the treatment of their terminally or seriously ill patients. The issuance of CSP is stated under
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The person wanting the drug or device and a licensed physician are both willing to participate.
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The Criminal Law Reporter. 20. Bureau of National Affairs. Arlington, Va. 1976. p. 2300.
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Ben Amar M (2006). "Cannabinoids in medicine: a review of their therapeutic potential".
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Smoke Signals: A Social History of Marijuana - Medical, Recreational and Scientific
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Darrow, Jonathan J.; Sarpatwari, Ameet; Avorn, Jerry; Kesselheim, Aaron S. (2015).
671: 630: 548: 538: 495: 487: 447: 437: 381: 257: 233: 104: 701:"Practical, Legal, and Ethical Issues in Expanded Access to Investigational Drugs" 660:"Practical, legal, and ethical issues in expanded access to investigational drugs" 619:"Practical, legal, and ethical issues in expanded access to investigational drugs" 265: 1258:"Expanded Access: FDA Describes Efforts to Ease Application Process | FDA Voice" 144:
The person is unable to obtain the investigational drug or device under another
165: 35: 1282: 883: 543: 1412: 1243: 726: 442: 363: 750:"Medical Ethicist Arthur Caplan Explains Why He Opposes 'Right-to-Try' Laws" 491: 315:). On November 24, 1976, federal Judge James Washington ruled in his favor. 1040: 891: 765: 734: 717: 700: 685: 676: 659: 644: 635: 618: 562: 509: 461: 161: 156:
Drugs can be made available to individuals, small groups, or large groups.
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is the use of an unapproved drug or medical device under special forms of
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Werner, Clinton A. (June 2001). "Medical Marijuana and the AIDS Crisis".
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Darrow, JJ; Sarpatwari, A; Avorn, J; Kesselheim, AS (15 January 2015).
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reversed an earlier ruling in favor of the Alliance. In 2008, the
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Program providing access to unapproved drugs or medical devices
1190:"Josh Hardy, Va. Boy Who Inspired Social Media Campaign, Dies" 1166:"Drug company: Ailing 7-year-old Josh Hardy will get medicine" 236:
despite cannabis in general being illegal in the Philippines.
1283:"FDA Simplifies Compassionate Use Application and Drug Costs" 324: 1217: 352:
U.S. Court of Appeals for the District of Columbia Circuit
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Hyry, HI; Manuel, J; Cox, TM; Roos, JC (21 August 2015).
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United States Department of Health and Human Services
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was no scientific value to it, and due to President
1088:Correspondent, By Elizabeth Cohen, Senior Medical. 798: 232:product." A CSP may also be obtained for processed 200:
Medicines and Healthcare products Regulatory Agency
468: 524: 164:for individual INDs; it can add some but not all 41:These programs go under various names, including 1410: 903: 901: 180:As of February 2019, 41 states have passed 937: 865: 863: 861: 943: 898: 297:Department of Health, Education & Welfare 808:Viray, Patricia Lourdes (18 December 2018). 967:"4 Americans get medical pot from the feds" 858: 590: 588: 586: 419: 417: 415: 413: 411: 409: 407: 350:. On August 7, 2007, in an 8–2 ruling, the 1090:"Company denies drug to dying child - CNN" 869: 520: 518: 1125:CS1 maint: multiple names: authors list ( 1087: 716: 675: 657: 634: 610: 552: 542: 499: 451: 441: 1280: 1164:Catherine E. Shoichet; Elizabeth Cohen. 1041:"Your Weekly Update of News and Reviews" 1010: 583: 480:Intractable & Rare Diseases Research 404: 243: 1051: 515: 1411: 983: 747: 833: 807: 778: 596:"Expanded Access (Compassionate Use)" 423: 346:, in which it was represented by the 28:investigational new drug applications 228:Administrative Order No. 4 of 1992. 1390:Expanded Access (Compassionate Use) 914:. Simon and Schuster. p. 234. 907: 658:Darrow JJ; et al. (Jan 2015). 623:The New England Journal of Medicine 527:"Compassionate use of orphan drugs" 13: 1360:as a named patient import Provider 964: 569: 356:Supreme Court of the United States 343:Abigail Alliance v. von Eschenbach 88:Abigail Alliance v. von Eschenbach 14: 1435: 1332: 1311:U.S. Food and Drug Administration 1281:Frellick, Marcia (June 3, 2016). 1065:. 14 January 2008. Archived from 1027:10.1097/01.nt.0000337676.20893.50 692: 531:Orphanet Journal of Rare Diseases 430:Perspectives in Clinical Research 248:Medicinal cannabis farmed by the 1382: This article incorporates 1377: 1344:Food and Drug Administration U.S 1230:(14 Supplement): LB–146–LB–146. 1200:from the original on 22 May 2018 986:Journal of Cannabis Therapeutics 944:Rupnow, Chuck (August 9, 1997). 305:Common Law doctrine of necessity 289:National Institute on Drug Abuse 270:National Institute on Drug Abuse 113: 1295: 1274: 1250: 1236:10.1158/1538-7445.AM2016-LB-146 1211: 1182: 1157: 1133: 1081: 1033: 1004: 977: 928: 834:Cabal, Ruth (16 October 2017). 827: 772: 754:Oncology (Williston Park, N.Y.) 705:New England Journal of Medicine 285:Drug Enforcement Administration 67:temporary authorization for use 1366:as an Expanded Access Provider 908:Lee, Martin A. (August 2012). 741: 651: 275:The program was started after 214: 1: 392: 268:. It is administered by the 872:Journal of Ethnopharmacology 779:BOREL, CĂ©line (2023-03-13). 397: 303:, had successfully used the 222:Food and Drug Administration 175: 7: 1364:myTomorrows Expanded Access 387:Emergency Use Authorization 370: 348:Washington Legal Foundation 94: 10: 1440: 1370: 1339:Europeans Medicines Agency 951:Eau Claire Leader-Telegram 239: 34:for devices, outside of a 965:AP (September 27, 2011). 884:10.1016/j.jep.2006.02.001 544:10.1186/s13023-015-0306-x 262:University of Mississippi 250:University of Mississippi 195:European Medicines Agency 188: 101:European Medicines Agency 1353:EMA Named Patient Import 1349:FDA Personal Importation 1047:(#07–30). 7 August 2007. 748:Caplan, A (2016-01-16). 443:10.4103/2229-3485.173779 492:10.5582/irdr.2016.01054 283:) against the FDA, the 204:National Health Service 121:These conditions were: 1384:public domain material 1045:Consumer Health Digest 718:10.1056/NEJMhle1409465 677:10.1056/NEJMhle1409465 636:10.1056/NEJMhle1409465 253: 51:managed access program 1419:Clinical pharmacology 998:10.1300/J175v01n03_03 971:Associated Press News 293:Department of Justice 247: 1063:Agence France-Presse 785:OMEDIT Ile de France 598:. FDA. 27 April 2018 299:. Randall, who had 226:Department of Health 63:named-patient access 59:compassionate access 1196:. 22 October 2016. 1145:www.kidsvcancer.org 846:on October 19, 2017 814:The Philippine Star 279:brought a lawsuit ( 75:pre-approval access 1118:has generic name ( 318:The settlement in 254: 252:for the government 1424:Clinical research 1358:The Socialmedwork 424:Patil, S (2016). 330:George H. W. Bush 309:medical necessity 277:Robert C. Randall 182:right-to-try laws 55:compassionate use 24:compassionate use 1431: 1404: 1402: 1401: 1381: 1380: 1326: 1325: 1323: 1322: 1313:. 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Index

investigational new drug applications
IDE application
clinical trial
Abigail Alliance v. von Eschenbach
European Medicines Agency
developing
IND application
IDE application
direct costs
indirect costs
right-to-try laws
European Medicines Agency
Medicines and Healthcare products Regulatory Agency
National Health Service
Food and Drug Administration
Department of Health
medical cannabis

University of Mississippi
medical cannabis
University of Mississippi
Mahmoud ElSohly
National Institute on Drug Abuse
Robert C. Randall
Drug Enforcement Administration
National Institute on Drug Abuse
Department of Justice
Department of Health, Education & Welfare
glaucoma
medical necessity

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