The principles guiding decisions about compassionate access should be the same regardless of the details of the individual request, and no matter who makes the decision. Criteria should be clear, transparent and designed to meet the immediate needs of as many individuals as possible without jeopardizing the health of future patients who must rely on approved products. Compassionate access should be granted only to patients with serious or life-threatening conditions for whom there are no acceptable approved therapies or clinical trials. Additional criteria should seek to minimize disruption to development and approval of therapies, ensure equitable access for patients and avoid undue financial burden on companies. What constitutes equitable access may depend on the setting; it could be random or based on factors like age or likelihood of cure.

These principles could in theory be implemented under the system in place today, which leaves final choices about compassionate access in the hands of drug companies. It would be better if decisions were made based on the advice of an independent third party. Determinations about drug supply, threats to approval and financial burdens on companies would be far more credible if they were made by an entity that had no financial interest in the outcome of compassionate use decisions. To ensure credibility, an appeals process also is necessary to validate that the criteria were objectively applied.