Prelude 12 steps collectively aim to ensure that the potential advantages outweigh the potential side effects. Naturally, the risk/reward balance varies per disease, and changes over time as more evidence becomes available. Types and timing of expanded access Expanded access programs are usually split into individual access programs (named-patient use) for single patients, and group programs that simultaneously allow multiple patients to access unregistered medicine.3 There is no rigid, formal timeline dictating when expanded access can be initiated. In theory, it is possible to request access for any unapproved drug at any stage of development. However, as an investigational product advances through clinical development, evidence accumulates, interest from physicians and patients increases, and singlepatient programs can evolve into group access programs.27 Furthermore, regulators and drug manufacturers are more prone to grant expanded access requests as safety and efficacy become increasingly established. Additionally, expanded access serves as a means of bridging the time between the completion of regulatory phase III trials and marketing approval. Frequently, drugs are launched in the United States first, making FDA approval the initial endorsement. This approval would spark interest in, for example, Europe, prior to the approval of the European Medicines Agency (EMA).28 Adding to the confusion, expanded access is not limited to pre-approval access, as it can also be used to access unregistered medicine after they have been withdrawn from the market. In certain regions, alternate forms of expanded access may be employed if a product has received approval from a regulatory authority outside their jurisdiction, as the associated risks are potentially reduced when another regulator has endorsed the product. Moreover, although not strictly considered ‘expanded access’, various regulators allow these programs to continue to bridge the time needed to obtain local reimbursement, particularly in countries with a national health system. If a drug is withdrawn from the market for any reason, the product reverts to the ‘unapproved’ status and, therefore, may strictly qualify for expanded access once more. Figure 1 depicts the relative interest in expanded access over the course of drug development.
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