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Expanded access or compassionate use is the use of an unapproved drug or medical device under special forms of investigational new drug applications (IND) or IDE application for devices, outside of a
clinical trial Clinical trials are prospective biomedical or behavioral research studies on human participants designed to answer specific questions about biomedical or behavioral interventions, including new treatments (such as novel vaccines, drugs, dietar ...
, by people with serious or life-threatening conditions who do not meet the enrollment criteria for the clinical trial in progress. These programs go under various names, including early access, special access, or managed access program, compassionate use, compassionate access, named-patient access, temporary authorization for use, cohort access, and pre-approval access. In general the person and their doctor must apply for access to the investigational product, the company has to choose to cooperate, and the medicines 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 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. 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 ''
Abigail Alliance v. von Eschenbach ''Abigail Alliance for Better Access to Developmental Drugs v. von Eschenbach'', 495 F.3d 695 (D.C. Cir. 2007), ''cert denied'', 552 U.S. 1159 (2008) was resolved in early 2008 when the Supreme Court of the United States declined to hear the appea ...
'', in which the Abigail Alliance, a group that advocates for access to investigational drugs for people who are terminally ill, tried to establish such access as a legal right. The Supreme Court declined to hear the case, effectively upholding previous cases that have maintained that there is not a constitutional right to unapproved medical products.


Programs

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
European Medicines Agency The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of medicinal products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products or Euro ...
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 developing. 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.


United States

In the US as of 2018, people could try obtain unapproved drugs or medical devices that were in development under specific conditions. These conditions were: * The person wanting the drug or device and a licensed physician are both willing to participate. * 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. * That the probable risk to the person from the investigational product is not greater than the probable risk from the disease or condition. * 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; * The FDA determines that providing the investigational product will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval; * 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) 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 * The person is unable to obtain the investigational drug or device under another IND application (for drugs), IDE application (for devices), or to participate in a clinical trial. Drugs can be made available to individuals, small groups, or large groups. 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
direct costs Direct costs are costs which are directly accountable to a cost object (such as a particular project, facility, function or product). Direct cost is the nomenclature used in accounting. The equivalent nomenclature in economics is specific cost. By ...
for individual INDs; it can add some but not all
indirect costs Indirect costs are costs that are not directly accountable to a cost object (such as a particular project, facility, function or product). Like direct costs, indirect costs may be either fixed or variable. Indirect costs include administration, pers ...
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. 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 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.


State law

, 41 states have passed
right-to-try laws Right-to-try laws are U.S. state laws and a federal law that were created with the intent of allowing terminally ill patients access to experimental therapies (drugs, biologics, devices) that have completed Phase I testing but have not been appro ...
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 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.


Europe

In Europe, the
European Medicines Agency The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of medicinal products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products or Euro ...
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 the
Medicines and Healthcare products Regulatory Agency The Medicines and Healthcare products Regulatory Agency (MHRA) is an executive agency of the Department of Health and Social Care in the United Kingdom which is responsible for ensuring that medicines and medical devices work and are acceptably ...
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
National Health Service The National Health Service (NHS) is the umbrella term for the publicly funded healthcare systems of the United Kingdom (UK). Since 1948, they have been funded out of general taxation. There are three systems which are referred to using the " ...
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. 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 these programs.


Philippines

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
Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respon ...
for the treatment of their terminally or seriously ill patients. The issuance of CSP is stated under
Department of Health A health department or health ministry is a part of government which focuses on issues related to the general health of the citizenry. Subnational entities, such as states, counties and cities, often also operate a health department of their ow ...
Administrative Order No. 4 of 1992. 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 product." A CSP may also be obtained for processed
medical cannabis Medical cannabis, or medical marijuana (MMJ), is cannabis and cannabinoids that are prescribed by physicians for their patients. The use of cannabis as medicine has not been rigorously tested due to production and governmental restrictions ...
despite cannabis in general being illegal in the Philippines.


History

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
medical cannabis Medical cannabis, or medical marijuana (MMJ), is cannabis and cannabinoids that are prescribed by physicians for their patients. The use of cannabis as medicine has not been rigorously tested due to production and governmental restrictions ...
grown at the
University of Mississippi The University of Mississippi (byname Ole Miss) is a public research university that is located adjacent to Oxford, Mississippi, and has a medical center in Jackson. It is Mississippi's oldest public university and its largest by enrollment. ...
, under the direction of Marijuana Research Project Director Dr. Mahmoud ElSohly. It is administered by the
National Institute on Drug Abuse The National Institute on Drug Abuse (NIDA) is a United States federal government research institute whose mission is to "advance science on the causes and consequences of drug use and addiction and to apply that knowledge to improve individual a ...
. The program was started after Robert C. Randall brought a lawsuit (''Randall v. U.S'') against the FDA, the
Drug Enforcement Administration The Drug Enforcement Administration (DEA; ) is a Federal law enforcement in the United States, United States federal law enforcement agency under the U.S. Department of Justice tasked with combating drug trafficking and distribution within th ...
, the
National Institute on Drug Abuse The National Institute on Drug Abuse (NIDA) is a United States federal government research institute whose mission is to "advance science on the causes and consequences of drug use and addiction and to apply that knowledge to improve individual a ...
, the
Department of Justice A justice ministry, ministry of justice, or department of justice is a ministry or other government agency in charge of the administration of justice. The ministry or department is often headed by a minister of justice (minister for justice in a v ...
, and the Department of Health, Education & Welfare. Randall, who had
glaucoma Glaucoma is a group of eye diseases that result in damage to the optic nerve (or retina) and cause vision loss. The most common type is open-angle (wide angle, chronic simple) glaucoma, in which the drainage angle for fluid within the eye rem ...
, had successfully used the ''Common Law doctrine of necessity'' to argue against criminal charges of marijuana cultivation that had been brought against him, because his use of cannabis was deemed a
medical necessity Medical necessity is a legal doctrine in the United States related to activities that may be justified as reasonable, necessary, and/or appropriate based on evidence-based clinical standards of care. In contrast, unnecessary health care lacks such ...
(''U.S. v. Randall''). On November 24, 1976, federal Judge James Washington ruled in his favor. The settlement in ''Randall v. U.S.'' 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
AIDS Human immunodeficiency virus infection and acquired immunodeficiency syndrome (HIV/AIDS) is a spectrum of conditions caused by infection with the human immunodeficiency virus (HIV), a retrovirus. Following initial infection an individual m ...
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 was no scientific value to it, and due to President
George H. W. Bush George Herbert Walker BushSince around 2000, he has been usually called George H. W. Bush, Bush Senior, Bush 41 or Bush the Elder to distinguish him from his eldest son, George W. Bush, who served as the 43rd president from 2001 to 2009; pr ...
administration's policies. As of 2011, four people continued to receive cannabis from the government under the program. 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. 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, which it lost, ''
Abigail Alliance v. von Eschenbach ''Abigail Alliance for Better Access to Developmental Drugs v. von Eschenbach'', 495 F.3d 695 (D.C. Cir. 2007), ''cert denied'', 552 U.S. 1159 (2008) was resolved in early 2008 when the Supreme Court of the United States declined to hear the appea ...
'', in which it was represented by the
Washington Legal Foundation The Washington Legal Foundation (WLF) is a non-profit legal organization located at 2007-2009 Massachusetts Avenue NW, on Embassy Row in Washington, D.C. Founded in 1977, the Foundation's stated goal is "to defend and promote the principles of f ...
. On August 7, 2007, in an 8–2 ruling, the U.S. Court of Appeals for the District of Columbia Circuit reversed an earlier ruling in favor of the Alliance. In 2008, the
Supreme Court of the United States The Supreme Court of the United States (SCOTUS) is the highest court in the federal judiciary of the United States. It has ultimate appellate jurisdiction over all U.S. federal court cases, and over state court cases that involve a point o ...
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". 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
brincidofovir Brincidofovir, sold under the brand name Tembexa, is an antiviral drug used to treat smallpox. Brincidofovir is a prodrug of cidofovir. Conjugated to a lipid, the compound is designed to release cidofovir intracellularly, allowing for higher int ...
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. In 2016 Kids 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.


See also

*
Orphan drug An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of ...
*
Right-to-try law Right-to-try laws are U.S. state laws and a federal law that were created with the intent of allowing terminally ill patients access to experimental therapies (drugs, biologics, devices) that have completed Phase I testing but have not been appro ...
*
Emergency Use Authorization An Emergency Use Authorization (EUA) in the United States is an authorization granted to the Food and Drug Administration (FDA) under sections of the Federal Food, Drug, and Cosmetic Act as added to and amended by various Act of Congress, Acts of ...


References


Citations


External links

* Europeans Medicines Agency * Food and Drug Administration U.S
FDA Personal Importation
an
EMA Named Patient Import

The Socialmedwork
as a named patient import Provider * myTomorrows Expanded Access as an Expanded Access Provider


Sources

* {{DEFAULTSORT:Expanded Access Clinical pharmacology Clinical research