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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, 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.[1][2][3]

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 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, 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

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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 European Medicines Agency 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.[1]

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.[2][3]

United States

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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.[4][5]

These conditions were:[5]

  • 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.[5]

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.[1] The manufacturer can only charge direct costs for individual INDs; it can add some but not all indirect costs for small group or larger expanded access programs.[6] To the extent that a doctor or clinic is required for use of the drug or device, they too may require payment.[1]

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.[1]

State law

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As of February 2019, 41 states have passed right-to-try laws that permit manufacturers to provide experimental medicines to terminally ill people without US FDA authorization.[7] 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.[8][9]

Europe

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In Europe, the European Medicines Agency 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 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 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.[1] Since 2021, France has a system of early and expanded access separated in two systems: AAC and AAP.[10]

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.[1]

Philippines

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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 Food and Drug Administration for the treatment of their terminally or seriously ill patients. The issuance of CSP is stated under Department of Health Administrative Order No. 4 of 1992.[11]

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 despite cannabis in general being illegal in the Philippines.[11][12]

History

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Medicinal cannabis farmed by the University of Mississippi for the government

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 grown at the University of Mississippi, under the direction of Marijuana Research Project Director Dr. Mahmoud ElSohly. It is administered by the National Institute on Drug Abuse.[citation needed]

The program was started after Robert C. Randall brought a lawsuit (Randall v. United States)[13] against the FDA, the Drug Enforcement Administration, the National Institute on Drug Abuse, the Department of Justice, and the Department of Health, Education & Welfare. Randall, who had glaucoma, 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 (U.S. v. Randall).[13] On November 24, 1976, federal Judge James Washington ruled in his favor.[14]: 142 [15]

The settlement in Randall v. U.S. became the legal basis for the FDA's compassionate IND program.[13] 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 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.[16][14] 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 administration's policies. As of 2011, four people continued to receive cannabis from the government under the program.[17]

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.[18]

In November 2001 the Abigail Alliance for Better Access to Developmental Drugs was established by Frank Burroughs in memory of his daughter, Abigail.[19] 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, in which it was represented by the Washington Legal Foundation. 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.[20] In 2008, the Supreme Court of the United States 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".[21]

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 was declined by the drug manufacturer, Chimerix.[22] The company reversed its decision after pressure from cancer advocacy organizations, and Josh received the drug that saved his life.[23][24] Hardy later died in September 2016 due to complications related to his underlying cancer diagnosis.[25] 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.[26] Since then, FDA simplified the application process, but stressed that it cannot require a manufacturer to provide a product.[27][28] FDA receives about 1,500 expanded access requests per year and authorizes 99% of it.[29]

See also

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References

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Revisions and contributorsEdit on WikipediaRead on Wikipedia

Expanded access

View on Grokipedia
from Grokipedia
Expanded access, also known as compassionate use, is a pathway regulated by the that enables patients facing serious or immediately life-threatening conditions to obtain investigational drugs, biologics, or medical devices outside of clinical trials when no comparable or satisfactory alternative treatments exist and potential benefits justify the risks. The program operates under FDA regulations (21 CFR 312.300 et seq.), requiring a licensed physician to submit an application or protocol, secure manufacturer consent to provide the product, and often obtain institutional review board oversight to ensure ethical treatment use. The framework includes individual patient access for single cases, including emergency use without prior IRB review if immediate threats exist; intermediate-size access for small groups not feasible in trials; and treatment INDs or protocols for larger populations facing similar unmet needs during extended development periods. This mechanism prioritizes early availability of promising therapies while mandating reporting and prohibiting manufacturers from profiting beyond direct costs, aiming to balance humanitarian access with evidentiary safeguards against unproven interventions. The FDA approves nearly all qualified requests, often within days via expanded access coordinators, countering claims of undue , though it generates data that can inform future approvals or reveal risks in non-trial settings. Notable applications have included antivirals during outbreaks and agents for cancers, underscoring its role in addressing gaps in standard care, yet it has sparked debates over risks like false hope, diversion of limited drug supplies from , and incomplete safety profiles for sicker patients, prompting alternatives such as the 2018 Right to Try Act that circumvents some FDA protocols but yields fewer documented uses due to manufacturer reluctance and liability concerns. Critics argue expanded access may undermine randomized recruitment or inflate perceived without controls, while proponents highlight its empirical foundation in case-level risk-benefit assessments over blanket denials.

Definition and Principles

Core Definition

Expanded access, commonly referred to as compassionate use, constitutes a regulatory pathway enabling patients with serious or immediately life-threatening conditions to obtain investigational medical products—including drugs, biologics, or medical devices—for treatment purposes outside formal clinical trials, provided no comparable or satisfactory alternative therapies are available. This mechanism operates under the premise that the potential direct benefit to the patient justifies the risks associated with unapproved products, while mandating safeguards such as physician oversight, , and reporting to mitigate harms. In practice, expanded access applies to products in various stages of development, from early investigational phases to those nearing approval, but excludes routine commercial distribution. Eligibility hinges on the condition's severity, the investigational product's promise based on available data, and the treating physician's determination that participation in a is not feasible. Programs distinguish between individual patient access, intermediate-sized groups, and larger treatment protocols, with the latter resembling expanded treatment (IND) applications. The framework prioritizes patient autonomy and urgency in unmet needs, yet imposes manufacturer consent and regulatory review to prevent diversion from enrollment or resource strain on development pipelines. Empirical data indicate rare approvals relative to trial participants, underscoring its exceptional rather than standard role in access.

Underlying Rationale

Expanded access programs exist primarily to enable patients facing serious or immediately life-threatening conditions—who lack satisfactory alternative therapies and cannot participate in clinical trials—to obtain investigational medical products under controlled conditions. This mechanism addresses the gap between ongoing drug development and urgent patient needs, allowing treatment when the potential benefits outweigh known risks, as determined by regulatory review. The U.S. Food and Drug Administration (FDA), which oversees such access, emphasizes that it serves as a humanitarian pathway rather than a substitute for rigorous clinical evaluation, ensuring that access does not compromise broader public safety or divert resources from pivotal trials. Ethically, the rationale draws on principles of beneficence and , permitting informed patients to pursue potentially efficacious therapies absent approved options, while considerations support equitable distribution to those with no other recourse. Proponents argue that denying access equates to withholding hope from individuals with progressive diseases, where delays in approval—often spanning years—can prove fatal; for instance, programs have facilitated use of drugs like those for rare cancers when trial enrollment is infeasible due to geographic or eligibility barriers. However, ethical frameworks require robust preliminary of and to avoid undue , distinguishing expanded access from unproven experimentation. From a practical standpoint, expanded access can yield real-world data on investigational products in diverse patient populations, informing future approvals without supplanting randomized controlled trials, which remain the gold standard for establishing causal . Regulatory bodies like the FDA have approved over 10,000 individual patient expanded access requests since 2005, demonstrating its role in bridging innovation gaps for conditions such as or pediatric leukemias, where standard treatments fail. This approach aligns with causal realism by prioritizing direct intervention for verifiable unmet needs over indefinite postponement, tempered by mandatory reporting to safeguard against unanticipated risks.

Regulatory Frameworks

Open Label Extensions (OLEs) and Expanded Access Programs (EAPs, also known as Early Access or Compassionate Use Programs) in pharmaceuticals provide continued or compassionate access to investigational drugs. From a global quality perspective, both require adherence to Good Manufacturing Practice (GMP) standards for investigational products, including robust chemistry, manufacturing, and controls (CMC) data, stability testing, proper labeling, and pharmacovigilance to ensure patient safety. Regulatory expectations vary by region (e.g., FDA requires detailed CMC for EAPs; EMA emphasizes quality in compassionate use), but international harmonization efforts (e.g., ICH guidelines) promote consistent quality approaches despite non-commercial status.

United States

In the , expanded access to investigational drugs, biologics, and devices is regulated by the (FDA) under 21 CFR Part 312 Subpart I, which permits treatment use outside clinical trials for patients with serious or immediately life-threatening conditions when no comparable or satisfactory alternative therapies exist. The program requires that the potential benefits justify the risks and that access does not interfere with or delay the drug's development or marketing approval process. Physicians must submit an (IND) application or protocol to the FDA, obtain manufacturer consent to provide the product, and secure (IRB) oversight where applicable, with the FDA typically reviewing individual patient requests within 30 days. The framework originated in the as an informal compassionate use mechanism for terminal cancer patients, evolving into formalized regulations in to standardize access while ensuring safety monitoring and . By the , the FDA approved over 99% of expanded access requests, reflecting a low denial rate based on clinical need rather than stringent barriers. Three main categories exist: individual patient access (for single patients, including use without prior IRB approval if documented appropriately); intermediate-size access (for groups of up to several hundred patients when evidence suggests clinical benefit); and treatment protocols or INDs for larger populations exceeding needs. individual access bypasses prospective IRB review but requires retrospective reporting, while non- cases demand full IRB approval. The of 2016 expanded eligibility by clarifying that expanded access applies to serious conditions (not solely life-threatening ones) and mandated manufacturers of drugs in phase 2 or 3 trials to publicly post their expanded access policies on company websites. This provision aimed to enhance transparency without altering core criteria. In contrast, the federal Right to Try Act, enacted in 2018, provides an alternative pathway for patients with life-threatening diseases who have exhausted approved options and cannot enroll in trials, allowing access to investigational drugs after phase 1 testing with manufacturer agreement but without mandatory FDA or IRB review. Unlike expanded access, Right to Try prohibits the FDA from using treatment outcomes in approval decisions and has resulted in fewer than 10 documented uses annually since implementation, as manufacturers often prefer the FDA-monitored route for liability and data reasons. Sponsors must report adverse events to the FDA under both mechanisms, though expanded access facilitates broader surveillance.

Europe

In the , expanded access to investigational medicinal products is primarily regulated through compassionate use programs under Article 83 of Regulation (EC) No 726/2004, which allows unauthorized medicines to be made available to patients with unmet medical needs when no satisfactory authorized alternatives exist. These programs apply to products in advanced clinical development stages, typically Phase II or III, where preliminary data suggest potential efficacy against serious or life-threatening conditions. The (EMA) coordinates recommendations via its Committee for Medicinal Products for Human Use (CHMP), focusing on uniform application across member states, including protocol design, patient selection criteria, and safety monitoring requirements. National competent authorities retain primary responsibility for approvals, authorizing distribution only under strict conditions to ensure and ethical standards. Compassionate use differs from individual named-patient access, which permits one-off treatments for specific patients without a formal program, often handled at the national level without EMA involvement. Eligibility criteria emphasize patients facing chronically or seriously debilitating diseases with no comparable therapies, where the investigational product demonstrates plausible therapeutic benefits outweighing foreseeable risks, supported by non-clinical and clinical data. Programs target groups of patients rather than isolated cases, requiring manufacturers to submit detailed protocols outlining dosing, monitoring, and plans; for multi-member state programs, manufacturers notify the EMA, which may issue coordinated CHMP advice. Treatments occur outside clinical trials, with costs typically borne by patients, national health systems, or manufacturers, though varies by country. Implementation varies across EU member states due to decentralized authority. In Germany, the Paul-Ehrlich-Institut oversees compassionate use via a confirmed notification procedure under the Medicines Act, emphasizing rapid access for orphan drugs and requiring post-use reporting. France's Temporary Authorisation for Use (ATU) program, managed by the Agence Nationale de Sécurité du Médicament, allows both individual and cohort access, with over 200 ATUs granted annually as of recent data, prioritizing and rare diseases. Italy's Agenzia Italiana del Farmaco facilitates named-patient imports and compassionate use for up to 600 patients per program, mandating ethical committee review. and the employ similar frameworks, with 's compassionate use under Royal Decree 109/2010 requiring regional health service coordination, while the focuses on investigator-initiated requests through the Medicines Evaluation Board. These national processes ensure alignment with EU pharmacovigilance rules, including mandatory reporting to EudraVigilance. Manufacturer responsibilities include supplying the product free or at cost, developing plans, and collaborating with authorities on , which may inform future marketing authorizations. As of 2021, compassionate use has facilitated access to therapies like those for and rare cancers, though uptake remains limited by evidentiary thresholds and resource constraints in smaller member states. The framework prioritizes evidence-based access over expediency, reflecting causal links between rigorous preclinical data and reduced post-access risks, without compromising ongoing trial integrity.

Other Regions

In , authorizes expanded access to investigational drugs through expanded access , defined as trials providing potential therapeutic benefit to patients with serious or life-threatening conditions who cannot enroll in conventional trials due to ineligibility, disease progression, or geographic barriers. Sponsors must submit a under Part C, Division 5 of the Food and Drug Regulations, demonstrating that anticipated benefits outweigh risks based on prior data, with reviews typically completed in 30 days. These programs adhere to good clinical practices, prioritize , and avoid interfering with standard timelines. Complementing this, the Special Access Programme permits case-by-case access to unmarketed drugs for compassionate use when no authorized alternatives exist, subject to physician requests and assessment. Australia's (TGA) oversees the Special Access Scheme (SAS), which allows registered health practitioners to supply unapproved therapeutic goods, including investigational drugs, to individual patients lacking suitable options on the Australian Register of Therapeutic Goods. The scheme categorizes requests by urgency and risk: Category A for patients with serious illnesses where death is likely without treatment, requiring post-supply notification within 28 days; Category B for other cases needing prior TGA approval and clinical justification, processed in 2-3 days; and Category C for products with established safe use histories, also via notification. Sponsors bear responsibilities for legal importation, reporting within 15 days, and ensuring integrity, while practitioners must document patient suitability and monitor outcomes. In , the (PMDA) supports compassionate use via the Japanese Compassionate Use (J-CU) program, established in January 2016, enabling access to investigational drugs for patients with life-threatening diseases ineligible for clinical trials. Unlike U.S. or European systems, J-CU requests bypass PMDA approval, relying on sponsor and institutional review, yielding approval rates approaching 100% for eligible cases when safety data supports use. Prior to 2016, access occurred under physician discretion or Advanced Medical Care B, which covers basic costs via public insurance but limits broader implementation; the program emphasizes ethical conduct without formal mandates equivalent to trials. Brazil's National Health Surveillance Agency (ANVISA) regulates expanded access and compassionate use programs for unregistered drugs targeting serious unmet needs, requiring sponsor-initiated requests with commitments to pursue full marketing authorization. These mechanisms, formalized around 2013, ensure patient safety through ethics committee oversight and adverse event reporting, though supply obligations extend post-access if trials conclude without approval. In other Latin American countries, similar named-patient or import pathways exist but vary, often lacking centralized frameworks and relying on local health authorities for case-by-case approvals. Across Asia-Pacific regions beyond Japan, programs emphasize named-patient access for imported unapproved drugs, with countries like India permitting imports via Form 11 licenses for personal use under physician certification, prioritizing rapid response over extensive data collection.

Application and Implementation

Eligibility and Criteria

Eligibility for expanded access, also known as compassionate use, typically requires patients to have a serious, life-threatening, or debilitating condition without satisfactory approved alternatives, and where enrollment in clinical trials is not feasible. Physicians must assess that the investigational product's potential benefits outweigh its risks based on available data, and provision must not compromise the product's development or ongoing trials. In the United States, the (FDA) specifies three core criteria under 21 CFR 312.305 for treatment use expanded access: the patient must have a serious or immediately life-threatening disease or condition with no comparable or satisfactory alternative therapy; the prospective benefit justifies the potential risks, which are reasonable given the condition; and access will not interfere with clinical investigations supporting marketing approval or the drug's development. These apply to individual patient, intermediate-size patient populations (typically fewer than 100 subjects), and larger treatment protocols. For single-patient access, the requesting physician submits Form FDA 3926, certifying the criteria are met and that adequate preclinical and clinical data exist on the drug's and risks. (IRB) approval or concurrence is required for non-emergency individual access, while emergency use bypasses prior IRB review but mandates retrospective notification. In the , compassionate use eligibility is determined at the member state level, with the (EMA) offering optional scientific opinions via its Committee for Medicinal Products for Human Use (CHMP) on quality, safety, and efficacy for specific programs. Patients generally qualify if they have life-threatening, chronically or seriously debilitating diseases without authorized therapies and cannot participate in clinical trials; the investigational medicine must be in active clinical trials (typically phase III) or the marketing authorization process, with sufficient early evidence of safety and potential benefit. National authorities set distribution, patient targeting, and monitoring conditions, prioritizing trial-eligible patients first. Internationally, criteria align closely with U.S. and standards but vary by jurisdiction; for instance, programs in countries like or emphasize unmet need and ethical review, often requiring sponsor agreement and regulatory notification without unified global protocols.

Process and Procedures

The process for expanded access typically begins with the treating physician evaluating the patient's eligibility, confirming a serious or immediately life-threatening condition with no comparable alternative therapy available, and determining that the potential benefits of the investigational product justify its risks. The physician must then obtain the manufacturer's agreement to provide the product, as sponsors are not obligated but often cooperate under FDA encouragement. For individual patient access, which constitutes the majority of requests, the physician submits Form FDA 3926 to the FDA's Center for Drug Evaluation and Research or Center for Biologics Evaluation and Research; in emergencies, verbal or electronic communication suffices initially, followed by the form within 15 working days. FDA review occurs within 30 days for non-emergency individual requests, though approvals are frequent—over 99% since —due to the program's low denial threshold focused on risk-benefit imbalance rather than proof. Prior to administration, the physician secures from the patient or guardian, detailing the experimental nature, uncertainties, and s. For single-patient emergency use, institutional review board (IRB) oversight may be retrospective or waived if immediate treatment is needed, but non-emergency cases require prospective IRB review or a of minimal . Intermediate-size expanded access, for groups of patients up to several hundred, follows similar steps but involves an expanded access IND or protocol submission, with FDA review ensuring no interference with pivotal trials. Treatment protocols or INDs for larger populations demand manufacturer initiation, including safety monitoring plans and annual reports to FDA. Throughout, physicians report serious adverse events to FDA and the sponsor within 15 days, while manufacturers handle , labeling, and distribution compliant with current good manufacturing practices. Costs may be recovered by the sponsor for direct production and distribution but not for research or profit.

Manufacturer Responsibilities

Manufacturers, also referred to as sponsors in regulatory contexts, are not obligated to provide investigational drugs through expanded access programs, as participation remains voluntary; the U.S. (FDA) lacks authority to compel supply to patients or physicians. If a manufacturer elects to participate, it must supply the investigational product—typically a or biologic—while ensuring compliance with Good Manufacturing Practice (GMP) standards for investigational products, including robust chemistry, manufacturing, and controls (CMC) data, stability testing, proper labeling, and pharmacovigilance to ensure patient safety and product quality during distribution outside clinical trials. From a global quality perspective, Open Label Extensions (OLEs) and Expanded Access Programs (EAPs, also known as compassionate use programs) require adherence to these standards, with regulatory expectations varying by region (e.g., FDA requires detailed CMC for EAPs; EMA emphasizes quality in compassionate use), but international harmonization efforts (e.g., ICH guidelines) promote consistent quality approaches despite non-commercial status. This decision often weighs potential impacts on ongoing clinical development, such as resource diversion or data integrity risks, against patient needs, with policies required to be publicly disclosed under the . Upon agreement to provide access, manufacturers hold primary responsibility for submitting or facilitating an (IND) application under 21 CFR Part 312 Subpart I, which may amend an existing IND or initiate a new one, including protocols for individual patient, intermediate-size, or treatment IND categories. Safety monitoring constitutes a core obligation, requiring expedited reporting of serious unexpected suspected adverse reactions per 21 CFR 312.32, along with annual IND reports if access extends beyond one year, and summaries of treatment outcomes including adverse effects at protocol conclusion. Manufacturers must also designate qualified investigators, such as physicians, to oversee administration and ensure patients receive risk-benefit information, while prohibiting any promotional claims of or for the unapproved product under 21 CFR 312. Charging patients or third parties for the drug is permissible only to recover —such as manufacturing, packaging, and shipping—provided the manufacturer demonstrates to the FDA that such recovery does not interfere with enrollment and that sufficient supply exists for development needs, as outlined in 21 CFR 312.8 and related guidance. In intermediate or treatment use scenarios involving larger patient groups, manufacturers bear additional burdens to track usage and report aggregated safety data to inform potential labeling updates or risk assessments. Internationally, analogous responsibilities apply under frameworks like the European Medicines Agency's (EMA) compassionate use procedures, where manufacturers must supply unauthorized medicines to national competent authorities upon request, ensure ongoing safety monitoring in line with good practices, GMP adherence, CMC documentation, and stability, and collaborate on reporting without direct EMA authorization but with notification to member states. Variations exist by jurisdiction, such as in or other regions, emphasizing voluntary provision, GMP adherence, and pharmacovigilance to mitigate risks while facilitating access.

Benefits and Empirical Outcomes

Successful Case Studies

In 2014, during the West African outbreak, two American healthcare workers, Kent Brantly and Nancy Writebol, received the investigational cocktail ZMapp under FDA expanded access protocols after contracting the virus in . Both patients, treated at in starting in August 2014, showed clinical recovery, with Brantly discharged on August 21 and Writebol on August 19; their survival was attributed in part to the experimental therapy alongside supportive care, though causality remains observational given limited supplies and concurrent cases without ZMapp who succumbed. Another prominent case involved Josh Hardy, a seven-year-old boy with a history of recurrent who developed disseminated following transplantation in early 2014. After initial denial by manufacturer Chimerix, public advocacy led to FDA-facilitated access to via an on March 12, 2014; Hardy's became undetectable within weeks, he was removed from ICU shortly thereafter, and discharged home from in July 2014, marking a reversal from near-fatal progression. A scoping review of compassionate use for rare diseases, covering 46 studies and 2,079 patients from 1991 to 2022, documented favorable in 84.8% of cases, including symptom in 76.1% and disease resolution in 8.7%. One specific instance involved administered to an infant with hepatic veno-occlusive disease, resulting in complete cure where standard therapies failed. Such outcomes underscore expanded access's role in addressing unmet needs, though individual successes do not imply generalizability without controlled data.

Data on Patient Access and Survival

In the United States, the (FDA) receives thousands of expanded access submissions annually, primarily for single-patient use, enabling with serious or life-threatening conditions to access investigational drugs outside clinical trials. From 2019 to 2023, the FDA processed 17,349 single-patient (IND) submissions (5,614 non- and 11,735 ), approving 17,271 (99.7% approval rate), corresponding roughly to the number of individual treated under these protocols. Intermediate-size and treatment INDs, which serve small groups or larger populations, accounted for fewer submissions (154 and 37 received, respectively), with approval rates of 87% and 78%. These figures reflect a surge during the , where expanded access facilitated broad distribution of investigational therapies, but non-pandemic access remains in the low thousands of yearly, limited by eligibility for those exhausted of approved options. Empirical data on survival outcomes from expanded access remains sparse and heterogeneous, as programs target terminal or patients without randomized controls, complicating causal attribution of benefits to the investigational agent. A 2019 analysis of 266 patients receiving single-patient expanded access to targeted therapies reported median of 4.0 months, with 38.9% surviving progression-free at 6 months and 24.5% at 1 year; overall response rates were approximately 20% across similar compassionate use cohorts for various cancers, though overall survival data were not uniformly tracked. Broader reviews of peer-reviewed expanded access publications from to 2022 indicate variable efficacy by disease—e.g., positive signals in rare genetic disorders or subsets—but highlight toward advanced cases, where placebo-like outcomes predominate absent the drug. Serious adverse events, including fatalities possibly linked to treatment, occurred in under 0.2% of cases across 1,033 drugs in one FDA-reviewed dataset, underscoring safety but not efficacy. Limitations in outcome reporting stem from the program's nature; FDA mandates safety reporting but not systematic survival tracking, leading to reliance on voluntary publications that may overrepresent successful cases from academic centers. from expanded access has informed post-approval labeling in some instances, but aggregate survival gains are not quantifiable due to factors like concurrent standard care and patient frailty. Critics note that while access expands hope for the ~1% of pediatric cancer patients or similar cohorts annually utilizing the program, survival extensions are often modest or unproven, prioritizing individual access over population-level trial data generation.

Risks and Criticisms

Safety and Adverse Events

Expanded access programs incorporate monitoring protocols comparable to those in clinical trials, requiring sponsors to serious adverse events (SAEs) to the FDA within 15 calendar days of awareness, including any unexpected events potentially related to the investigational drug. For single-patient expanded access, immediate reporting of SAEs is mandatory, followed by annual summaries if treatment exceeds one year and a final detailing outcomes, ensuring continuous evaluation without compromising access. Empirical reveal that adverse events arising from expanded access infrequently alter trajectories or contribute to restrictive labeling. A review of FDA regulatory actions from 2005 to 2015 identified only one case where expanded access safety alone prompted adverse product labeling—a drug-drug interaction—amid thousands of requests, underscoring the rarity of such impacts. Similarly, serious adverse events sufficient to affect overall program progression occur exceedingly rarely, with FDA analyses confirming negligible influence on investigational drug approvals or holds. In compassionate use contexts, particularly for rare diseases, have been documented in approximately 67% of reviewed studies spanning 1991–2022, though causality attribution is complicated by patients' advanced disease states and comorbidities; no events were reported in 28% of cases, and outcomes often aligned with expected risks in uncontrolled settings. The FDA integrates expanded access data into broader safety assessments via systems like the Adverse Event Reporting System (FAERS), but isolated incidents from these programs seldom trigger clinical hold or withdrawal decisions. Critics of less-regulated variants, such as Right to Try provisions bypassing certain FDA pre-approvals, highlight risks including unmonitored toxicities or interactions, potentially exacerbating harm in terminal patients without trial safeguards. Nonetheless, post-enactment utilization data through 2024 shows minimal reported SAEs leading to program-wide changes, attributed to manufacturer discretion and voluntary reporting, though comprehensive outcomes remain understudied due to limited mandatory disclosures. Overall, while adverse events occur at rates reflective of high-risk populations, structured reporting mitigates systemic threats to drug safety profiles.

Effects on Drug Development and Trials

Expanded access programs are regulated to minimize interference with clinical trials and overall , with the U.S. (FDA) requiring under 21 CFR 312.305(a)(3) that such access does not compromise the initiation, conduct, or completion of trials needed for marketing approval. This determination involves assessing patient eligibility—typically limited to those unable to enroll in ongoing trials due to factors like or stage—and evaluating larger-scale programs (e.g., treatment INDs) for higher risks of recruitment overlap. FDA may impose clinical holds if interference is evident and often requires sponsors to detail accrual plans to ensure trial viability. Critics argue that expanded access can divert limited resources, including drug supply and personnel, from pivotal trials, potentially delaying approvals and broader patient access to validated therapies. It may also deter trial enrollment by offering patients an alternative pathway to investigational without randomization or placebo risks, prolonging the time to accrue sufficient participants for robust efficacy data. Sponsors face additional burdens, as are typically provided free, imposing financial and logistical costs that compete with development priorities and may discourage participation in such programs. Empirical analyses, however, indicate limited adverse effects on regulatory outcomes. A of 321 FDA decisions from 2010–2016 found drugs with prior expanded access had an 84% approval rate versus 76% without (P=0.001), with no refusals or complete responses attributed to expanded access safety data; in one case, approval relied solely on such data. Labeling changes from expanded access events were rare (two instances of warnings for interactions or liver issues), often tied to comorbidities rather than inherent risks, suggesting concerns over undermining or approval are overstated. Real-world data from these programs can supplement , potentially accelerating development for rare diseases where trials are challenging.

Ethical and Philosophical Debates

Individual Rights vs. Collective Safety

The ethical debate surrounding expanded access programs centers on the tension between a patient's to pursue potentially beneficial investigational therapies and the broader imperative to safeguard through rigorous evidence generation. Proponents of prioritizing individual rights argue that terminally ill or severely afflicted patients, facing imminent death or irreversible harm, possess a moral claim to access unapproved drugs when clinical trials are unavailable, grounded in principles of and the alleviation of extreme suffering. This perspective gained legislative traction with the federal Right to Try Act of 2017, which enables eligible patients with life-threatening conditions to obtain investigational drugs directly from manufacturers after phase 1 trials, bypassing certain FDA oversight to affirm personal liberty over bureaucratic delays. Critics contend that such access undermines collective by exposing vulnerable individuals to therapies lacking robust and , potentially yielding harms without verifiable benefits due to the absence of controlled comparisons. Adverse events in expanded access cohorts, often comprising sicker patients than trial participants, can complicate regulatory approval and erode confidence in the pipeline, as unmonitored outcomes may obscure risks that would otherwise inform randomized controlled trials (RCTs)—the gold standard for establishing causal and minimizing population-level harms. The Right to Try Act exacerbates these concerns by prohibiting the FDA from accessing generated under its provisions, insulating manufacturers from liability while depriving regulators of critical insights that protect future patients. From a utilitarian standpoint, the collective benefits of mandatory RCTs—evidenced by their role in averting widespread adoption of ineffective or dangerous treatments, as seen in historical rejections of agents like laetrile—outweigh isolated individual gains, particularly given the low probability of benefit in settings where inflates anecdotal successes. Manufacturers frequently decline expanded access requests to avoid jeopardizing trial enrollment or generating noisy that could derail approval, highlighting how individual pursuits can inadvertently stall innovations benefiting millions. Empirical from FDA programs show high approval rates for requests (over 99% since 2009), yet sparse long-term tracking reveals persistent risks of overpromising without evidence, underscoring the need for that candidly weighs personal hope against societal evidentiary standards.

Incentives and Market Realities

Pharmaceutical manufacturers often weigh participation in expanded access programs against substantial disincentives, including heightened liability exposure from adverse events occurring outside controlled settings, where causality attribution to the investigational drug may be scrutinized in litigation. Such risks are amplified for early-stage drugs, as uncontrolled use could generate data conflicting with outcomes, potentially jeopardizing regulatory approval or product labeling. and distributing limited supplies at cost—or for free—imposes financial burdens without offsetting revenue, particularly straining smaller firms with limited resources. Market dynamics further discourage broad expanded access, as it may divert eligible patients from enrolling in pivotal trials essential for demonstrating and under randomized conditions, thereby prolonging development timelines and delaying market entry. Clinical trials offer manufacturers proprietary data yielding protections and exclusivity periods, whereas expanded access yields anecdotal, non-randomized outcomes of limited regulatory value, though occasionally supportive for post-approval expansions. U.S. data indicate that while 99% of expanded access requests receive agency approval—often within days—manufacturer consent remains discretionary and frequently withheld, especially for single-patient cases in non-orphan indications, reflecting a prioritization of integrity over ad hoc access. Positive incentives, such as reputational benefits or aiding label negotiations, exist but are insufficient to override core commercial imperatives for most sponsors, who structure policies favoring trial participation over compassionate use. Proposals for compensatory mechanisms, including extended market exclusivity or expedited reviews to offset expanded access costs, have been advocated but not enacted, underscoring a regulatory framework misaligned with industry incentives. In practice, participation rates skew toward later-stage drugs nearing approval, where risks are lower and access aligns with strategies, rather than foundational phases critical to recouping development investments averaging $1-2 billion per approved .

Historical Development

Origins and Early Precedents

The origins of expanded access trace to the 1938 Federal Food, Drug, and Cosmetic Act, which included Section 505(i) permitting the shipment of unapproved drugs labeled "for investigational use only" to qualified experts, enabling limited access for patients lacking alternatives. This mechanism operated informally, often through physician requests or telephone approvals from the FDA, without formalized regulations for broader compassionate distribution. The 1962 Kefauver-Harris Drug Amendments, enacted after the tragedy, imposed requirements for adequate safety and efficacy data, , and institutional review, which constrained but did not eliminate informal investigational access via individual investigator INDs or provisions. By the , amid growing demand for experimental cancer therapies, the FDA established the Group C Investigational New Drug program in 1976, authorizing distribution of promising anticancer agents to multiple physicians outside clinical trials for patients with refractory diseases. In 1979, the FDA issued its first explicit policy on expanded access, formalizing pathways for terminal cancer patients to receive unproven drugs under treatment protocols. Early precedents highlighted both potential benefits and risks. In the early , the FDA approved an open-label IND for 5-iododeoxyuridine (IDUR) to treat herpes encephalitis, a condition with no alternatives, allowing over 70 patients to receive the drug; subsequent placebo-controlled studies revealed severe toxicities, including linked to 3-5 deaths and persistent viral presence in autopsied cases. These cases underscored the need for safeguards, yet set the stage for expanded mechanisms amid the 1980s AIDS crisis, where patient activism pressured regulators; by 1987, FDA regulations codified treatment INDs, enabling access to investigational drugs for larger groups with serious conditions when trials were unavailable.

Key Legislative and Regulatory Milestones

The U.S. (FDA) initiated informal provisions for compassionate use of investigational drugs in the late , primarily to provide terminal cancer patients access to unapproved therapies outside clinical trials when no alternatives existed. This practice evolved in response to advocacy from patient groups and physicians, marking the practical origins of expanded access without formal regulatory codification at the time. In 1987, amid the AIDS crisis, the FDA established the Treatment Investigational New Drug (IND) application under 21 CFR 312.34, allowing broader access to investigational therapies for patients with serious diseases after sufficient development to support safety data, but before marketing approval. This regulation formalized "treatment use" for groups of patients unable to participate in trials, reflecting pressure from activists demanding faster access to experimental antiretrovirals. By 2009, the FDA consolidated and expanded these pathways through a final rule amending IND regulations to create Subpart I of 21 CFR Part 312, which explicitly governed individual patient, intermediate-size, and emergency expanded access, emphasizing criteria like the absence of comparable therapies and minimal risk to drug development. The , enacted on December 13, 2016, introduced Section 3033 requiring applicable drug and device manufacturers and distributors to develop and publicly post policies on evaluating and responding to expanded access requests, aiming to enhance transparency without mandating approval of requests. The FDA Reauthorization Act of 2017 further refined access by authorizing expanded intermediate-size populations and streamlining reporting for single-patient INDs. On May 30, 2018, President signed the Trickett Wendler, Omar Burleson, and Frank M. Buckles Right to Try Act into law, creating a federal pathway for terminally ill patients to access eligible investigational drugs directly from manufacturers after Phase 1 trials, bypassing FDA pre-approval while still requiring sponsor consent and liability protections under certain conditions. This legislation supplemented rather than replaced FDA expanded access, addressing concerns over bureaucratic delays but drawing criticism for lacking oversight on safety data collection compared to traditional IND processes.

Post-2020 Innovations and Challenges

The spurred significant scale-up in expanded access programs, culminating in the largest such initiative in U.S. history with over participants accessing investigational convalescent plasma under FDA oversight by late 2020. This expansion highlighted the potential for expanded access to serve as a bridge during emergencies, enabling rapid distribution of unapproved therapies like for severely ill patients when clinical trials could not accommodate demand. Manufacturers reported heightened managed access activity for indications, with programs adapting to facilitate broader compassionate use while collecting real-world safety data to inform future regulatory decisions. Post-pandemic innovations included FDA's emphasis on master protocols to streamline expanded access in , discussed in a February 2020 Critical Path Innovation Meeting, which aimed to integrate treatment access with efficient generation across multiple investigational agents. Project Facilitate, an FDA service launched prior but intensified post-2020, provided oncology providers with expedited regulatory consultations for expanded access requests, processing thousands of inquiries to reduce administrative barriers. In October 2025, FDA issued updated guidance incorporating post-2017 queries on eligibility, sponsor duties, and intermediate-size access, clarifying pathways without altering core regulations but enhancing practical implementation for rare diseases and serious conditions. These steps reflected a shift toward leveraging expanded access as to support approvals, as seen in cases where compassionate use outcomes contributed to post-approval labeling expansions. Challenges emerged from the pandemic's volume surge, requiring clinical and translational science award programs to rapidly adapt protocols amid evolving FDA guidance on COVID-19 investigational uses, often under compressed timelines that risked incomplete safety monitoring. Ethical tensions intensified, as expedited access for desperate patients clashed with imperatives to prioritize trial enrollment and rigorous evidence generation, potentially diluting incentives for controlled studies. Resource constraints strained sponsors and regulators, with manufacturing scale-up for compassionate distribution proving logistically demanding, while gaps between expanded access and emergency use authorizations led to inconsistent patient outcomes and data silos. By 2023, implementation hurdles persisted in oncology and advanced therapies, including case-by-case variability and the need for better harmonization to avoid diverting drugs from pivotal trials.

Emerging Variations in Low-Resource Settings

In low-resource settings, expanded access programs face significant barriers, including absent or underdeveloped regulatory frameworks, limited , and low clinical trial activity, resulting in only 3% of global compassionate use requests originating from low- and lower-middle-income countries between 2018 and 2020. These disparities persist due to economic constraints and insufficient public awareness, often forcing reliance on import provisions rather than structured programs. For instance, in , compassionate use remains nascent as of 2020, with no formal terminology but allowances for physicians to import unapproved drugs on a named-patient basis under Central Drugs Standard Control Organization guidelines. Emerging variations adapt to these constraints through international pharmaceutical managed access extensions and public-private partnerships, particularly for rare diseases and . Novartis's Zolgensma gene therapy program, launched globally around 2019, provided free access in select low-resource contexts despite regulatory gaps, highlighting pharma-led models that bridge deficiencies. Similarly, Roche's risdiplam compassionate use for , expanded post-2020, enrolled patients from low- and middle-income countries lacking dedicated regulations by leveraging sponsor oversight and simplified eligibility. In , Brazil's ANVISA formalized compassionate use under RDC 608/2022 for medical devices and drugs, enabling structured access amid regional variations where has robust rules but and lack explicit definitions. NGO advocacy drives further adaptations, such as MSF's 2021 push for AbbVie's compassionate use in low- and middle-income countries for hepatitis C treatment failures, emphasizing named-patient programs where standard regimens fail. For anticancer therapies, strategies include compulsory licensing and patent pooling to lower costs, alongside expanded access pilots integrated with financing reforms, as proposed in 2024 analyses of resource-constrained care. These variations prioritize equity by combining global pharma commitments with local policy evolution, though implementation remains uneven due to persistent resource limitations.

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