What US law requires an expanded access policy to contain and when to post it, what studies of company policies found, and what EU rules publish.
A pre-approval access policy is the public statement in which a company developing investigational medicines explains whether and how it evaluates requests for access outside clinical trials. In the United States, section 561A of the Federal Food, Drug, and Cosmetic Act, added by the 21st Century Cures Act in 2016 and amended by the FDA Reauthorization Act of 2017, requires the manufacturer or distributor of an investigational drug for a serious disease to make its expanded access policy public by the earlier of the first phase 2 or phase 3 study or 15 days after a breakthrough therapy, fast track or regenerative advanced therapy designation. The policy must give a contact, the procedure for requests, the general criteria, the anticipated time to acknowledge a request and a link to the ClinicalTrials.gov record, and it guarantees no access. The treating physician makes the request; the company decides whether to supply.
A physician whose patient has a serious condition, no satisfactory authorised treatment and no place in a clinical trial often starts in the same place: the website of the company that develops an investigational medicine, looking for the page that says whether the company considers requests and how to make one. In the United States that page is not optional: a company that develops an investigational drug for a serious disease must make its policy on evaluating and responding to expanded access requests public and readily available (21 U.S.C. § 360bbb-0). What the physician finds there, or fails to find, decides how the request starts.
This article reads these pre-approval access policies from two sides: the treating physician who must decide whether and how to ask, and the sponsor that must write one. It sets out the legal duty, the FDA and Reagan-Udall Foundation resources, what published studies found when they read company policies, and how the policies relate to compassionate use and named patient routes in Europe. For the wider context, see managed access and early access explained.
A published expanded access policy tells a physician how to ask; it does not promise that the answer will be yes.
A pre-approval access policy is a company's public statement of how it evaluates and responds to requests for an investigational medicine from patients who cannot join a clinical trial. US law calls it an expanded access policy and requires it from the manufacturer or distributor of one or more investigational drugs for the diagnosis, monitoring or treatment of a serious disease or condition (21 U.S.C. § 360bbb-0(a)). Companies use other names for the same page: reviewing 29 manufacturers' websites, the US Government Accountability Office found terms such as "pre-approval access", "compassionate use access" and "early access" (U.S. Government Accountability Office, 2019).
The policy matters because the company holds the first key: a patient can obtain an investigational drug outside a trial only if its manufacturer agrees (U.S. Government Accountability Office, 2019). Access needs the FDA's review and the active cooperation of drug companies and health care providers (Jarow et al., 2017), so the policy is the physician's first source on whether the door exists and how to knock on it.
A published policy is a commitment to answer, and answering takes people, supply and a route in every market a request can come from. Ideogen provides that capacity for partners across Europe, MENA, CIS and 94 markets worldwide: request intake, the national filings, the import and supply route and safety reporting. The decision to supply stays with the sponsor. Talk to our experts about bridging access to unmet need in orphan and rare disease, where earlier access changes patient outcomes, at [email protected].
The FDA describes expanded access, "sometimes called 'compassionate use'", as a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product outside clinical trials when no comparable or satisfactory alternative is available (U.S. Food and Drug Administration, 2026b). In the EU, compassionate use under Article 83 means making a medicine available to a group of patients with a chronically or seriously debilitating or life-threatening disease who cannot be treated satisfactorily by an authorised medicine (Regulation (EC) No 726/2004, Art. 83), while supply for one patient on a professional's order, named patient supply, rests on Article 5(1) of the Directive (Directive 2001/83/EC, Art. 5(1)). The lack of a common definition has led to access programmes being misclassified (Iudicello et al., 2016). For the programme itself, see what an expanded access programme is.
US regulations provide three categories of expanded access, and a policy may address one or all of them (21 C.F.R. part 312, subpart I):
In every category the FDA must determine that the disease is serious or immediately life-threatening, that there is no comparable or satisfactory alternative, that the potential benefit justifies the potential risks, and that providing the drug will not interfere with the clinical investigations that could support approval (21 C.F.R. § 312.305). Expanded access supplements clinical trials and does not replace them; see key differences between managed access, expanded access and clinical trials.
Information about access has not reached every physician equally: a survey of paediatric haematologists and oncologists concluded that physicians may have unequal access to information about investigational drugs (Chapman et al., 2021). Commentators noted that there was no obligation, and often little incentive, for manufacturers to offer expanded access, especially for individual patients (Hogan, 2016). As access widened, ethical issues gained prominence, including informed consent, funding, disparities in access and effects on drug development (Bunnik et al., 2017).
Patients and families turned to online petitions and social media campaigns, out of desperation and for lack of reliable information, and one analysis of these campaigns called for greater transparency (Mackey & Schoenfeld, 2016). Congress then wrote a transparency duty into the Federal Food, Drug, and Cosmetic Act: section 3032 of the 21st Century Cures Act added section 561A on 13 December 2016 (21st Century Cures Act, 2016, § 3032). The duty applies in the same terms to a small company with one asset in phase 2 and to a large one (21 U.S.C. § 360bbb-0(a)), although smaller and newer companies were later found less likely to have a public policy (Kang et al., 2021).
Under section 561A, a US expanded access policy must give a contact, the procedure for making requests, the general criteria for evaluating them, the anticipated time to acknowledge receipt and a link to the ClinicalTrials.gov record.
Section 561A is codified at 21 U.S.C. § 360bbb-0. Its current text, in the 2024 edition of the United States Code and unchanged since 2017, was added by section 3032 of the 21st Century Cures Act and amended by section 610(c) of the FDA Reauthorization Act of 2017 (21 U.S.C. § 360bbb-0; FDA Reauthorization Act of 2017, § 610). The policy must be "made public and readily available, such as by posting such policies on a publicly available Internet website", and it may apply generally to all of the company's investigational drugs (21 U.S.C. § 360bbb-0(b)).
The duty falls on the manufacturer or distributor of investigational drugs for serious diseases or conditions, and it concerns requests under section 561(b), the route for individual patient access (21 U.S.C. § 360bbb-0(a)). The FDA recommends that the company developing the drug for marketing, rather than a distributor, posts the policy; makers of investigational devices are not covered; and at the start of a phase 2 or phase 3 trial it is for the developer to identify whether the drug treats a serious condition, which a fast track, breakthrough therapy or regenerative advanced therapy designation indicates (U.S. Food and Drug Administration, 2026a).
The law lists five elements, and the FDA's guidance repeats that the policy must include all of them (21 U.S.C. § 360bbb-0(c); U.S. Food and Drug Administration, 2026a):
Two of these elements are often misread. The time stated is the time to acknowledge receipt, not the time to decide; the Reagan-Udall Foundation's company directory makes the same point about the acknowledgement times it lists (Reagan-Udall Foundation for the FDA, n.d.-b). And the criteria are general: the law does not require a company to list its products or to say for which of them access is available (21 U.S.C. § 360bbb-0(b)).
Since the FDA Reauthorization Act of 2017, a sponsor must post its policy by the earlier of the start of the first phase 2 or phase 3 study or 15 days after a breakthrough therapy, fast track or regenerative advanced therapy designation.
As enacted in 2016, the duty applied from the later of 60 calendar days after enactment or the first initiation of a phase 2 or phase 3 study (21st Century Cures Act, 2016, § 3032), so manufacturers had until 11 February 2017 for drugs already in those phases (Jung et al., 2018). The FDA Reauthorization Act of 2017, under the heading "Expanded Access Policy Transparency", replaced "later" with "earlier" and added a second trigger (FDA Reauthorization Act of 2017, § 610). The section now applies from the earlier of:
The FDA reads "initiation of a phase 2 or phase 3 study" as the point when enrolment begins, and states that the deadlines do not preclude posting earlier (U.S. Food and Drug Administration, 2026a). A company may therefore publish a basic statement before the legal trigger, even one that says access is not yet available.
Posting the policy on the company's own website fulfils the requirement, and the FDA names the Reagan-Udall Foundation's Expanded Access Navigator as another place to post it (U.S. Food and Drug Administration, 2026a). A company developing several investigational drugs may establish different policies for each and post them individually by name (U.S. Food and Drug Administration, 2024c); drug-specific policies should be published with reference to the products they cover (U.S. Food and Drug Administration, 2026a).
A policy that names a drug for which expanded access is available and links to ClinicalTrials.gov is not, in the FDA's stated view, promotion of an investigational drug, unless the posted policy represents in a promotional context that the drug is safe or effective for the use under investigation (U.S. Food and Drug Administration, 2026a). The wording therefore stays factual. For the European side of the same question, see what can be said in public about an unlicensed medicine.
Posting a policy "shall not serve as a guarantee of access to any specific investigational drug by any individual patient", and a company may revise its policy at any time (21 U.S.C. § 360bbb-0(d); 21 U.S.C. § 360bbb-0(e)). A sponsor that provides expanded access does so voluntarily, and the FDA cannot compel it (U.S. Food and Drug Administration, 2026a). Section 561A requires a policy to exist and be public; it does not require the company to say yes. The request itself rests on section 561(b): a person, acting through a licensed physician, may request an investigational drug, and the manufacturer may provide it if the section's conditions are met (21 U.S.C. § 360bbb).
The fifth element points to a separate duty. When a clinical trial of an unapproved drug is registered, the registration must state whether expanded access is available for those who do not qualify for the trial and how to obtain information about it (42 U.S.C. § 282(j)). The responsible party that is both manufacturer and trial sponsor submits an expanded access record with a brief summary that includes the procedure for requesting the product, eligibility criteria, the expanded access status and a contact with a toll-free telephone number and an email address (42 C.F.R. § 11.28).
The FDA notes the limits: there may be no record if the trial need not be registered, or if the responsible party is not also the manufacturer, and a physician who finds none may still contact the sponsor (U.S. Food and Drug Administration, 2026a). Registry data have their own gaps: of 398 expanded access programmes registered on ClinicalTrials.gov, only 2 per cent reported results (Miller et al., 2017), and programmes started before approval were available for a median of 10 months before it (Puthumana et al., 2018).
The FDA's expanded access page lists the conditions that must all apply: a serious or immediately life-threatening condition, no comparable or satisfactory alternative, enrolment in a clinical trial not possible, potential benefit that justifies the potential risks, and no interference with trials that could support approval (U.S. Food and Drug Administration, 2026b). Its information for industry describes the company's part: being willing to provide the product and either submitting the request, allowing the FDA to cross-reference its IND or giving the sponsor-investigator the information needed; the FDA "reviews the expanded access request and determines if the treatment may proceed" (U.S. Food and Drug Administration, 2024c).
The FDA's questions and answers guidance, revised in April 2026, devotes three answers to company policies: content and posting, timing, and where to find information on availability (U.S. Food and Drug Administration, 2026a). The FDA states that it allows over 99 per cent of single patient requests to proceed (U.S. Food and Drug Administration, 2025b); in fiscal year 2023 its drugs centre received 1,326 non-emergency individual patient expanded access INDs and allowed 1,318 (U.S. Food and Drug Administration, 2024a).
The Reagan-Udall Foundation, a non-profit organisation established by Congress to assist the FDA, launched the Expanded Access Navigator in July 2017 to help physicians and patients locate manufacturers' expanded access policies (U.S. Government Accountability Office, 2019). The Navigator offers guidance for patients, physicians and companies (Reagan-Udall Foundation for the FDA, n.d.-a). Its Company Directory can show, for each company, the expanded access web page, the ClinicalTrials.gov link, a telephone number and email, the expected time frame and single-patient or disease-specific policies (Reagan-Udall Foundation for the FDA, n.d.-b).
Two notes matter to physicians. The data come from the company or from ClinicalTrials.gov, and many companies consider requests even if they show no active listing (Reagan-Udall Foundation for the FDA, n.d.-b); participation is voluntary (U.S. Government Accountability Office, 2019). The Navigator's page for companies and sponsors suggests that a policy commit to acknowledging requests within a stated period, giving five working days as an example, address the patient's condition, benefit and risk and the adequacy of supply, and say whether the company offers single-patient access or prefers protocols for several patients (Reagan-Udall Foundation for the FDA, n.d.-c).
In a study of 423 US companies with an oncology-related investigational drug, 32 per cent had a public expanded access policy, and fewer than a third of those policies contained all the information the Cures Act describes.
After the first deadline of 11 February 2017, one team reviewed a sample of manufacturers' policies to see what information was readily available to patients online and whether the Act's information was there (Jung et al., 2018). A later study of 423 applicable US biopharmaceutical companies with an oncology-related drug found that 140 (32 per cent) had a public policy; companies with an FDA expedited designation (59 against 21 per cent) or an approved drug (57 against 28 per cent) were more likely to have one (Kang et al., 2021).
The audit Congress ordered in 2017 looked at a smaller selection (FDA Reauthorization Act of 2017, § 610). Reviewing a non-generalisable selection of 29 manufacturers' websites between 31 January and 12 March 2019, the Government Accountability Office found that 23 said whether they considered individual requests: 19 said they would and four that they would not; the other six said they were developing content. Eleven of the 19 also listed the information on the Navigator (U.S. Government Accountability Office, 2019).
Completeness is the weak point. Only 31 per cent of the public policies in the oncology study (44 of 140) contained all the information the Act describes, and the authors concluded that the Act may be having a limited impact on closing information gaps, especially for products of smaller and newer companies (Kang et al., 2021). Among the 19 manufacturers in the federal audit that considered requests, all said that physicians must submit requests on behalf of their patients and explained how, and 18 gave an estimated time frame for responding (U.S. Government Accountability Office, 2019).
Product-level information is rarer. Only five of the 19 referred to specific drugs, and only one stated how many patients had been granted access (U.S. Government Accountability Office, 2019). Physicians ask for more: in a small survey of oncologists outside the United States, most recommended that companies publish information on availability, and on how to apply, for all pipeline products (Yong & Tan, 2024). Ethicists and patient advocates writing on gene therapy argue that companies should go beyond the Cures Act and publish product-specific policies (Kearns et al., 2021).
Eligibility sections tend to follow the statutory and regulatory criteria. Eighteen of the 19 manufacturers stated that the patient must have a serious or life-threatening condition, have no comparable or satisfactory alternative and be unable to take part in a trial; 17 stated that the treating physician must judge that the risk of the drug is not greater than the anticipated benefit (U.S. Government Accountability Office, 2019). Typical criteria, mapped to their legal source, are:
The fifth criterion does much of the work. Ten manufacturers in the audit stated that a company must have enough of the drug to support its clinical development before granting access outside trials (U.S. Government Accountability Office, 2019), and the FDA notes that even a series of individual requests for a drug for a rare disease could affect a development programme (U.S. Food and Drug Administration, 2026a). A policy that explains this at the outset spares physicians and families false expectations.
The law requires only the anticipated time to acknowledge receipt (21 U.S.C. § 360bbb-0(c)). The time frames manufacturers gave varied: some meant an acknowledgement, others a decision on access (U.S. Government Accountability Office, 2019). Evidence on decision times comes mostly from Europe: in 8,934 requests from 30 European countries pooled by four manufacturers, the median internal company approval time was five days, while the time from approval to shipment varied between cohort programmes and individual patient requests (Schlatter et al., 2025).
Few of the policies reviewed say who decides: one manufacturer in the audit stated that it uses an external advisory committee, including bioethical experts, physicians and patient representatives, to evaluate certain requests (U.S. Government Accountability Office, 2019), and a published pilot describes such a committee, formed by a company and an academic ethics department to allocate a scarce investigational agent among single patients (Caplan et al., 2018). Thirteen manufacturers required the relevant regulatory authority to review requests after their own approval, and five an ethics committee or institutional review board (U.S. Government Accountability Office, 2019).
Of the four manufacturers not considering requests, two gave reasons: both cited safety, and one also cited limited resources and its focus on clinical trials; both said they would periodically re-evaluate (U.S. Government Accountability Office, 2019). Although an expanded access programme is regulated, the decision to set one up is the sponsor's responsibility, and it should rest on ethical and practical considerations as well as regulatory criteria (Klopfenstein et al., 2015). Sponsors facing that decision can read planning expanded access programmes for biotech sponsors.
The physician initiates the request: section 561(b) lets a person request an investigational drug acting through a licensed physician (21 U.S.C. § 360bbb), and every manufacturer in the federal audit that considered requests required physicians to submit them (U.S. Government Accountability Office, 2019). Before writing, check:
Many physicians have tried this route: in a survey of 238 community oncologists and haematologists, 46 per cent had attempted expanded access (Zettler et al., 2021). In another survey, most oncologists had learned of existing programmes by contacting a sponsor or another provider (Yong & Tan, 2024), and pharmacists can help with identification, preparation and regulatory compliance (Smith & Redic, 2022). The steps from request to delivery are set out in the managed access workflow from physician request to delivery.
A company's agreement is not the end of the process. In the US, the physician usually obtains the company's permission for the FDA to refer to its IND and submits the patient-specific information (21 C.F.R. § 312.310). The FDA developed Form FDA 3926 for these requests and estimates the burden at 45 minutes per response (U.S. Food and Drug Administration, 2024b; U.S. Food and Drug Administration, 2025a). Institutional review board approval and informed consent must be obtained before treatment begins (U.S. Food and Drug Administration, 2025b), although published IRB policies on expanded access vary considerably and are often difficult to find and interpret (Folkers & Bateman-House, 2018).
At the end of treatment, the physician or sponsor sends the FDA a written summary of the results (21 C.F.R. § 312.310). In one survey, 43 of 44 paediatric haematologists and oncologists who had sought access reported that the company granted it (Chapman et al., 2021). For the content of a protocol for several patients, see what belongs in an expanded access protocol.
The federal Right to Try Act, signed into law on 30 May 2018 (U.S. Food and Drug Administration, 2018), created a second route for patients with a life-threatening disease who have exhausted approved options and cannot join a trial, for drugs that have completed a phase 1 trial and remain in active development (21 U.S.C. § 360bbb-0a). The FDA does not review or approve requests under it, the Act does not require a sponsor to provide a drug, and sponsors submit an annual summary of use (U.S. Food and Drug Administration, 2024d). Unlike expanded access, it involves neither the FDA nor an institutional review board in individual requests (Chapman et al., 2020). In one interview study, only 4 of 21 oncologists experienced with expanded access had discussed it, and none had formally tried to use it (Smith et al., 2021). Section 561A does not mention it, so a policy that addresses Right to Try does so by the company's choice.
Deciding what to publish is a strategic call as well as a legal one. Say too little and the policy fails the physician; promise more than the company can deliver and it creates expectations the company cannot meet. Beyond the five legal elements (21 U.S.C. § 360bbb-0(c)), ethicists argue for systems in which "criteria are clearly described, responsibilities are assigned and arrangements are made, so that patients will know what (not) to expect" (Bunnik et al., 2018). Practical points follow:
Plain wording that still sets firm limits serves a frightened family better than defensive legal language. Before publishing, confirm that the people, budget and supply behind the policy exist, because the decision to provide access carries consequences for current patients, development and future patients (Klopfenstein et al., 2015). The division of tasks is set out in who does what in a managed access programme.
A published policy is often the first thing a family reads after a hard diagnosis, and it should be honest about limits without being cold. The most useful policies state that a request does not guarantee access, as the law itself says (21 U.S.C. § 360bbb-0(d)), and explain why a company might decline, such as limited supply, safety monitoring or the need to complete the trials (U.S. Government Accountability Office, 2019). A short section for patients can explain that a treating physician must make the request, encourage them to ask their doctor about clinical trials first, and point to the registry entry (U.S. Government Accountability Office, 2019; 21 U.S.C. § 360bbb-0(c)).
Costs and continuity are the questions families ask next. Under US rules a sponsor may charge for an investigational drug only with the FDA's prior written authorisation, and may recover only the direct costs of making it available, with monitoring and administrative costs added for larger programmes (21 C.F.R. § 312.8). A patient treated outside a trial also needs to know whether treatment will stop when the programme ends, and a policy that answers this, even in general terms, prevents painful surprises. EU law addresses it for cohort programmes: the applicant must ensure that patients in a compassionate use programme have access between authorisation and placing on the market (Regulation (EC) No 726/2004, Art. 83(8)). See transitioning a managed access programme to registered supply.
Gene therapies raise particular questions. Their effects in most cases cannot be reversed, and a vector-induced immune response may preclude repeat dosing, so a patient treated outside a trial may lose access to other therapies using the same vector; the authors argue for product-specific policies, reasons when a company declines and consultation with patients and advocates (Kearns et al., 2021).
Expanded access can generate data, and a policy that mentions research use of that information raises consent and data-use questions it should address openly. Efforts to facilitate expanded access have made it easier for companies to gather outcome data, with ethical issues to anticipate (Bunnik et al., 2018). A review found 1,231 publications from expanded access programmes describing 507,481 patients, most from Europe and the Americas (Polak et al., 2023). In Europe, the heterogeneous national approach leaves uncertainty about how far data can be collected and analysed (Polak et al., 2022). For evidence questions, see real-world evidence.
In the EU, a Member State that uses Article 83 notifies the EMA, and the EMA publishes an up-to-date list of the CHMP's compassionate use opinions on its website.
The EU rules read for this article set duties for authorities and for those who run programmes. Under Article 83 of Regulation (EC) No 726/2004, a Member State that makes a medicine available for compassionate use notifies the European Medicines Agency; the CHMP may adopt opinions on the conditions for use and distribution and the patients targeted, after consulting the manufacturer; and the Agency publishes an up-to-date list of those opinions (Regulation (EC) No 726/2004, Art. 83). Compassionate use programmes are coordinated and implemented by Member States, which set their own rules (European Medicines Agency, n.d.). The individual route rests on a bona fide unsolicited order by an authorised health-care professional for an individual patient (Directive 2001/83/EC, Art. 5(1)).
National rules decide what is notified and published. In Germany, the person responsible for a hardship programme notifies it to the competent federal authority with the patient selection criteria and expected patient numbers (AMHV, § 3), and the Federal Institute for Drugs and Medical Devices lists confirmed and active programmes; named patient use is outside that procedure (Federal Institute for Drugs and Medical Devices, n.d.). In Italy, companies inform the Italian Medicines Agency before opening a programme and at least 30 days before closing it (Decreto 7 settembre 2017, Art. 5); the physician makes the request, the company confirms free supply, and the agency publishes a list of programmes (Agenzia Italiana del Farmaco, n.d.).
Public rules matter. Of one manufacturer's 31,711 compassionate use requests from 110 countries, 94 per cent came from countries whose regulations were publicly available online, and public availability was independently associated with request activity (Aliu et al., 2022). A policy analysis of seven European countries found variable national regulations, inconsistent reimbursement and 36 programmes, each available in up to four countries (Rosenberg et al., 2025). Companies weigh regulatory, medical, operational and financial factors before applying, including supply and the faster approval of named patient use (Timmerman et al., 2026), and differing national requirements create operational challenges (Schlatter et al., 2025).
Patient organisations ask for the transparency the US law pursues: EURORDIS has called for equal access, transparency of the programme for patients and the public, and a clear definition of who will benefit (EURORDIS-Rare Diseases Europe, 2017). For the comparison of the two systems, read expanded access and Article 83 compared; for the regulation, compassionate use under Article 83; and country by country, how named patient programmes work in Europe.
Several features of the system support more detailed policies: the law treats them as living documents (21 U.S.C. § 360bbb-0(e)), the FDA allows product-specific policies (U.S. Food and Drug Administration, 2026a), and the Navigator gathers them in one directory (Reagan-Udall Foundation for the FDA, n.d.-b). The gaps are largest for products of smaller and newer companies (Kang et al., 2021). Across the sources, the policies that work best are specific, honest and written for their readers: they say what they cover, set clear eligibility rules, tell physicians how to apply and how long acknowledgement takes, and explain their limits in plain language (U.S. Government Accountability Office, 2019; Bunnik et al., 2018).
In the US, yes, if it manufactures or distributes an investigational drug for a serious disease, by the earlier of the first phase 2 or phase 3 study or 15 days after an expedited designation (21 U.S.C. § 360bbb-0(f)).
No. A policy is not a guarantee of access, and the FDA cannot compel a sponsor to provide it (21 U.S.C. § 360bbb-0(d); U.S. Food and Drug Administration, 2026a).
The treating physician; in the federal audit, every manufacturer that considered requests required physicians to submit them (U.S. Government Accountability Office, 2019).
The policy states the anticipated time to acknowledge receipt, which is not the time to decide (21 U.S.C. § 360bbb-0(c); Reagan-Udall Foundation for the FDA, n.d.-b).
On the company's website, in the Navigator's Company Directory and through the ClinicalTrials.gov expanded access record where one exists (U.S. Food and Drug Administration, 2026a).
Only with the FDA's prior written authorisation, and only for direct costs, plus some monitoring and administrative costs for larger programmes (21 C.F.R. § 312.8).
The EMA publishes the list of CHMP compassionate use opinions, and national authorities such as BfArM and AIFA publish lists of programmes (Regulation (EC) No 726/2004, Art. 83(6); Federal Institute for Drugs and Medical Devices, n.d.; Agenzia Italiana del Farmaco, n.d.).
The sponsor decides whether to supply and keeps its regulatory responsibilities. The operational work can be run by a partner: request intake and tracking, the national filings, the import and supply route, and safety reporting in each market. Ideogen does this for partners across Europe, MENA, CIS and 94 markets worldwide, and does not decide access to any medicine. Talk to our experts about bridging access to unmet need in orphan and rare disease, where earlier access changes patient outcomes, at [email protected].
For physicians, a policy maps the first step: whom to contact, what to send, what the company looks at, how long acknowledgement takes and where the registry record is (21 U.S.C. § 360bbb-0(c)). The decision to supply is the company's, and the decision to let treatment proceed is the regulator's (U.S. Food and Drug Administration, 2024c).
For sponsors, the legal minimum is a starting point, not the goal: treat the published policy as a promise and make sure the team can keep it. Ideogen does not decide access to any medicine: the treating physician makes the request, and the sponsor decides whether to supply under the law. For partners planning this work, see how Ideogen supports partners with end-to-end commercialisation.
21st Century Cures Act, Pub. L. No. 114-255, § 3032, 130 Stat. 1033, 1100-1101 (2016). https://www.govinfo.gov/content/pkg/PLAW-114publ255/html/PLAW-114publ255.htm
Agenzia Italiana del Farmaco. (n.d.). Farmaci a uso compassionevole [Compassionate use programmes]. Retrieved October 19, 2026, from https://www.aifa.gov.it/en/farmaci-a-uso-compassionevole
Aliu, P., Sarp, S., Reichenbach, R., Behr, S., Fitzsimmons, P., Shamlajee, M., Kola, S. P., Nunes Radimerski, S., & Scosyrev, E. (2022). International country-level trends, factors, and disparities in compassionate use access to unlicensed products for patients with serious medical conditions. JAMA Health Forum, 3(4), Article e220475. https://doi.org/10.1001/jamahealthforum.2022.0475
Bunnik, E. M., Aarts, N., & van de Vathorst, S. (2017). The changing landscape of expanded access to investigational drugs for patients with unmet medical needs: Ethical implications. Journal of Pharmaceutical Policy and Practice, 10, Article 10. https://doi.org/10.1186/s40545-017-0100-3
Bunnik, E. M., Aarts, N., & van de Vathorst, S. (2018). Little to lose and no other options: Ethical issues in efforts to facilitate expanded access to investigational drugs. Health Policy, 122(9), 977-983. https://doi.org/10.1016/j.healthpol.2018.06.005
Caplan, A. L., Teagarden, J. R., Kearns, L., Bateman-House, A. S., Mitchell, E., Arawi, T., Upshur, R., Singh, I., Rozynska, J., Cwik, V., & Gardner, S. L. (2018). Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials. Journal of Medical Ethics, 44(11), 761-767. https://doi.org/10.1136/medethics-2016-103917
Chapman, C. R., Belli, H. M., Leach, D., Shah, L. D., & Bateman-House, A. (2021). A survey of pediatric hematologists/oncologists' perspectives on single patient Expanded Access and Right to Try. Medicine Access @ Point of Care, 5, Article 23992026211005991. https://doi.org/10.1177/23992026211005991
Chapman, C. R., Eckman, J., & Bateman-House, A. S. (2020). Oversight of right-to-try and expanded access requests for off-trial access to investigational drugs. Ethics & Human Research, 42(1), 2-13. https://doi.org/10.1002/eahr.500038
Charging for investigational drugs under an IND, 21 C.F.R. § 312.8 (2026). https://www.ecfr.gov/current/title-21/chapter-I/subchapter-D/part-312/subpart-A/section-312.8
Decreto 7 settembre 2017 del Ministero della Salute. Disciplina dell'uso terapeutico di medicinale sottoposto a sperimentazione clinica [Decree of the Ministry of Health of 7 September 2017 governing the therapeutic use of medicinal products under clinical trial]. (2017). Gazzetta Ufficiale della Repubblica Italiana, Serie Generale n. 256 (2 November 2017). https://www.gazzettaufficiale.it/eli/id/2017/11/02/17A07305/sg
Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating to medicinal products for human use (consolidated version of 1 January 2025). (2025). EUR-Lex. https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX:02001L0083-20250101
European Medicines Agency. (n.d.). Compassionate use. Retrieved October 19, 2026, from https://www.ema.europa.eu/en/human-regulatory-overview/research-development/compassionate-use
EURORDIS-Rare Diseases Europe. (2017, May). Early access to medicines in Europe: Compassionate use to become a reality [Position paper]. https://www.eurordis.org/publications/early-access-to-medicines-in-europe-compassionate-use-to-become-a-reality/
Expanded access policy required for investigational drugs, 21 U.S.C. § 360bbb-0 (2024). https://www.govinfo.gov/content/pkg/USCODE-2024-title21/html/USCODE-2024-title21-chap9-subchapV-partE-sec360bbb-0.htm
Expanded Access to Investigational Drugs for Treatment Use, 21 C.F.R. pt. 312, subpt. I (2026). https://www.ecfr.gov/current/title-21/chapter-I/subchapter-D/part-312/subpart-I
Expanded access to unapproved therapies and diagnostics, 21 U.S.C. § 360bbb (2024). https://www.law.cornell.edu/uscode/text/21/360bbb
FDA Reauthorization Act of 2017, Pub. L. No. 115-52, § 610, 131 Stat. 1005, 1051-1053 (2017). https://www.govinfo.gov/content/pkg/PLAW-115publ52/html/PLAW-115publ52.htm
Federal Institute for Drugs and Medical Devices. (n.d.). Compassionate use. Retrieved October 19, 2026, from https://www.bfarm.de/EN/Medicinal-products/Clinical-trials/Compassionate-Use/_node.html
Folkers, K. M., & Bateman-House, A. (2018). Improving expanded access in the United States: The role of the institutional review board. Therapeutic Innovation & Regulatory Science, 52(3), 285-293. https://doi.org/10.1177/2168479018759661
Hogan, M. (2016). (R)evolution: Toward a new paradigm of policy and patient advocacy for expanded access to experimental treatments. BMC Medicine, 14, Article 39. https://doi.org/10.1186/s12916-016-0586-6
Individual patients, including for emergency use, 21 C.F.R. § 312.310 (2026). https://www.ecfr.gov/current/title-21/chapter-I/subchapter-D/part-312/subpart-I/section-312.310
Investigational drugs for use by eligible patients, 21 U.S.C. § 360bbb-0a (2024). https://www.law.cornell.edu/uscode/text/21/360bbb-0a
Iudicello, A., Alberghini, L., Benini, G., & Mosconi, P. (2016). Expanded access programme: Looking for a common definition. Trials, 17(1), Article 21. https://doi.org/10.1186/s13063-015-1108-0
Jarow, J. P., Lurie, P., Ikenberry, S. C., & Lemery, S. (2017). Overview of FDA's Expanded Access Program for investigational drugs. Therapeutic Innovation & Regulatory Science, 51(2), 177-179. https://doi.org/10.1177/2168479017694850
Jung, E., Zettler, P. J., & Kesselheim, A. S. (2018). Prevalence of publicly available expanded access policies. Clinical Pharmacology & Therapeutics, 104(5), 1016-1021. https://doi.org/10.1002/cpt.996
Kang, S., Chang, S., Ross, J. S., & Miller, J. E. (2021). Implementation of 21st Century Cures Act expanded access policies requirements. Clinical Pharmacology & Therapeutics, 110(6), 1579-1584. https://doi.org/10.1002/cpt.2401
Kearns, L., Chapman, C. R., Moch, K. I., Caplan, A. L., Watson, T., McFadyen, A., Furlong, P., & Bateman-House, A. (2021). Gene therapy companies have an ethical obligation to develop expanded access policies. Molecular Therapy, 29(4), 1367-1369. https://doi.org/10.1016/j.ymthe.2021.03.008
Klopfenstein, M., Van Campen, L. E., & Garnett, T. (2015). Expanded access programs: Ethical and practical considerations for biopharmaceutical sponsors. Therapeutic Innovation & Regulatory Science, 49(3), 352-358. https://doi.org/10.1177/2168479015578154
Mackey, T. K., & Schoenfeld, V. J. (2016). Going "social" to access experimental and potentially life-saving treatment: An assessment of the policy and online patient advocacy environment for expanded access. BMC Medicine, 14, Article 17. https://doi.org/10.1186/s12916-016-0568-8
Miller, J. E., Ross, J. S., Moch, K. I., & Caplan, A. L. (2017). Characterizing expanded access and compassionate use programs for experimental drugs. BMC Research Notes, 10(1), Article 350. https://doi.org/10.1186/s13104-017-2687-5
National Institutes of Health; clinical trial registry data bank, 42 U.S.C. § 282(j) (2024). https://www.law.cornell.edu/uscode/text/42/282
Polak, T. B., Cucchi, D. G. J., Schelhaas, J., Ahmed, S. S., Khoshnaw, N., van Rosmalen, J., & Uyl-de Groot, C. A. (2023). Results from expanded access programs: A review of academic literature. Drugs, 83(9), 795-805. https://doi.org/10.1007/s40265-023-01879-4
Polak, T. B., Cucchi, D. G. J., van Rosmalen, J., Uyl-de Groot, C. A., & Darrow, J. J. (2022). Generating evidence from expanded access use of rare disease medicines: Challenges and recommendations. Frontiers in Pharmacology, 13, Article 913567. https://doi.org/10.3389/fphar.2022.913567
Puthumana, J., Miller, J. E., Kim, J., & Ross, J. S. (2018). Availability of investigational medicines through the US Food and Drug Administration's expanded access and compassionate use programs. JAMA Network Open, 1(2), Article e180283. https://doi.org/10.1001/jamanetworkopen.2018.0283
Reagan-Udall Foundation for the FDA. (n.d.-a). Expanded Access Navigator. Retrieved October 19, 2026, from https://navigator.reaganudall.org/
Reagan-Udall Foundation for the FDA. (n.d.-b). Company directory. Expanded Access Navigator. Retrieved October 19, 2026, from https://navigator.reaganudall.org/company-directory
Reagan-Udall Foundation for the FDA. (n.d.-c). For companies and sponsors. Expanded Access Navigator. Retrieved October 19, 2026, from https://navigator.reaganudall.org/companies-and-sponsors
Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Union procedures for the authorisation and supervision of medicinal products for human use and establishing a European Medicines Agency (consolidated version of 28 January 2022). (2022). EUR-Lex. https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX:02004R0726-20220128
Requirements for all expanded access uses, 21 C.F.R. § 312.305 (2026). https://www.ecfr.gov/current/title-21/chapter-I/subchapter-D/part-312/subpart-I/section-312.305
Rosenberg, N., Post, H. C., Schutte, T., de Visser, S. J., Bartelink, I., Pasmooij, A. M. G., van Laarhoven, H. W. M., & Hollak, C. E. M. (2025). Access to anticancer and orphan medicines through compassionate use programs and named patient basis in seven European countries. ESMO Open, 10(11), Article 105855. https://doi.org/10.1016/j.esmoop.2025.105855
Schlatter, P., Heiss, N., Franco, P., Martin, A. O., Robson, S., & Reichenbach, R. (2025). An industry perspective on compassionate use in Europe: A call for change. Therapeutic Innovation & Regulatory Science, 60(2), 455-465. https://doi.org/10.1007/s43441-025-00902-y
Smith, A. J. F., & Redic, K. A. (2022). Single-patient expanded access: A primer for pharmacists. American Journal of Health-System Pharmacy, 79(23), 2118-2127. https://doi.org/10.1093/ajhp/zxac242
Smith, C., Stout, J., Adjei, A. A., Buckner, J., Wentworth, M., Tilburt, J., & Master, Z. (2021). “I think it's been met with a shrug:” Oncologists' views toward and experiences with right-to-try. Journal of the National Cancer Institute, 113(6), 735-741. https://doi.org/10.1093/jnci/djaa137
Timmerman, A., Rodenhuis, N., Crane-van Opstal, L. M. A., de Boer, A., Bongers, L., & Pasmooij, A. M. G. (2026). Factors influencing pharmaceutical companies' decisions to pursue compassionate use programs in the EU: A qualitative study in The Netherlands. Journal of Pharmaceutical Policy and Practice, 19(1), Article 2605391. https://doi.org/10.1080/20523211.2025.2605391
U.S. Food and Drug Administration. (2018, January 4). Learn about expanded access and other treatment options. https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options
U.S. Food and Drug Administration. (2024a, May 2). Expanded access (compassionate use) submission data. https://www.fda.gov/news-events/expanded-access/expanded-access-compassionate-use-submission-data
U.S. Food and Drug Administration. (2024b, October 16). Expanded access: How to submit a request (forms). https://www.fda.gov/news-events/expanded-access/expanded-access-how-submit-request-forms
U.S. Food and Drug Administration. (2024c, October 16). Expanded access: Information for industry. https://www.fda.gov/news-events/expanded-access/expanded-access-information-industry
U.S. Food and Drug Administration. (2024d, December 12). Right to Try. https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try
U.S. Food and Drug Administration. (2025a). Form FDA 3926: Individual patient expanded access investigational new drug application (IND) [Form FDA 3926 (01/25); OMB No. 0910-0814]. https://www.fda.gov/media/98616/download
U.S. Food and Drug Administration. (2025b, September 16). For physicians: How to request single patient expanded access ("compassionate use"). https://www.fda.gov/drugs/investigational-new-drug-application-ind/physicians-how-request-single-patient-expanded-access-compassionate-use
U.S. Food and Drug Administration. (2026a, April). Expanded access to investigational drugs for treatment use: Questions and answers [Guidance for industry, Procedural, Revision 2]. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expanded-access-investigational-drugs-treatment-use-questions-and-answers
U.S. Food and Drug Administration. (2026b, October 1). Expanded access. https://www.fda.gov/news-events/public-health-focus/expanded-access
U.S. Government Accountability Office. (2019). Investigational drugs: FDA and drug manufacturers have ongoing efforts to facilitate access for some patients (GAO-19-630). https://www.gao.gov/products/gao-19-630
Verordnung über das Inverkehrbringen von Arzneimitteln ohne Genehmigung oder ohne Zulassung in Härtefällen (Arzneimittel-Härtefall-Verordnung, AMHV) vom 14. Juli 2010 (BGBl. I S. 935), zuletzt geändert durch Artikel 8 der Verordnung vom 6. Juli 2022 (BGBl. I S. 1102) [Ordinance on placing medicinal products on the market without approval or authorisation in cases of hardship]. (2010). Gesetze im Internet. https://www.gesetze-im-internet.de/amhv/BJNR093500010.html
What constitutes clinical trial registration information?, 42 C.F.R. § 11.28 (2026). https://www.ecfr.gov/current/title-42/chapter-I/subchapter-A/part-11/subpart-B/section-11.28
Yong, F. L., & Tan, W. C. (2024). The current state of understanding of oncology expanded access programs in Malaysia. Medical Journal of Malaysia, 79(2), 191-195. https://pubmed.ncbi.nlm.nih.gov/38553925/
Zettler, M. E., Jeune-Smith, Y., Feinberg, B. A., Phillips, E. G., & Gajra, A. (2021). Expanded access and Right To Try requests: The community oncologist's experience. JCO Oncology Practice, 17(11), e1719-e1727. https://doi.org/10.1200/OP.20.00569
