Expanded Access, Named Patient and Emergency Use Programs for Bristol-Myers Squibb Medicines
In general, a drug may not be sold or otherwise distributed (outside of clinical trials) in a country before marketing authorization is obtained. However, in most countries, there are regulatory mechanisms that allow pharmaceutical companies to make an investigational drug available for the treatment of certain patients.
This Policy describes the standards that must be applied to ensure that requests for such access by patients to Bristol-Myers Squibb medicines are considered and processed in accordance with applicable legal/regulatory requirements and principles consistent with the Bristol-Myers Squibb Mission and Pledge. It is applicable globally and across all therapeutic areas.
Such programs are considered Expanded Access and essentially are government sanctioned mechanisms that enable a patient access to promising new therapies. Although the product is not approved for marketing in the country in the particular indication from which the request originates; Bristol-Myers Squibb must have the intent to file a marketing authorization application in that country for such programs.
Expanded Access Programs (EAP) are generally:
- Limited to patients with a serious/life-threatening condition
- For patients for whom there is no real therapeutic alternative
- Outside of a clinical trial (i.e., the patient is ineligible to participate in a clinical trial or there is no ongoing clinical trial in the patient’s country of residence.)
EAPs will only be approved in countries where there is an intent to submit a marketing application. If Bristol-Myers Squibb decides not to proceed to commercialization, no new requests are to be granted for that country.
Also, in no case will such programs be implemented in a way that could be construed as marketing or promoting the use of the product prior to regulatory approval. Accordingly, commercial involvement is expressly prohibited unless specifically authorized.
In order to approve an EAP, all of the following criteria must be met:
- Sufficient evidence of efficacy that a clinically meaningful benefit may be expected (i.e., available scientific evidence, taken as a whole, provides a reasonable basis to conclude that the drug may be effective.)
- Sufficient evidence of safety indicating that patients would not be exposed to unreasonable risk if prescribed the product.
- The patient has the serious or life-threatening disease for which the expanded access program is authorized.
- There is no comparable or satisfactory alternative therapy to diagnose, monitor or treat the disease or condition.
- The patient is ineligible for, or otherwise unable to participate in a clinical trial for the product (e.g., due to geographical constraints).
- There is nothing unique about a particular patient that suggests that a clinically meaningful benefit would not be expected or that there would be an unreasonable risk posed by prescribing the product drug to that patient.
In general, the product is provided at no charge; under no circumstances will the patient be charged for the product. Bristol-Myers Squibb may seek reimbursement in countries that specifically authorize government, insurer or other party reimbursement for products provided as part of an EAP. Any questions about whether reimbursement is permitted, and the procedure for obtaining reimbursement, will be referred to R&D legal counsel.
Expanded Access Programs include Named Patient and Emergency Use programs.
Named Patient Programs refer to the government-sanctioned provision of an investigational drug to a specifically identified patient who has no other treatment options.
Emergency Use is only applied in a limited number of countries and is utilized in rare situations in which loss of life is imminent without treatment. In these situations there is insufficient time to submit a regulatory application (e.g., Investigational New Drug [IND]) in accordance with regulations that govern non-Emergency Use or there is insufficient time to properly file an amendment to an existing regulatory application prior to administering treatment. In such circumstances, reporting mechanisms must still be established related to any adverse events that may occur during the course of treatment and all necessary internal and health authority approvals in the local country must be obtained.