Compassionate use
Compassionate Use Treatment
Many new drugs remain under development and have not yet received marketing authorization. Patients may therefore have access to these drugs only by participating in a clinical trial or through an application under a compassionate use program.
- What Is Compassionate Use?
Taiwan’s Ministry of Health and Welfare has promulgated the Regulations Governing the Special Approval of the Manufacture or Importation of Specific Medicinal Products. Where an unapproved drug is required to prevent or treat a life-threatening or severely debilitating disease for which no appropriate drug or alternative therapy is currently available in Taiwan, or to address an urgent public health need, the Pharmaceutical Affairs Act allows a designated hospital to apply to the central health authority for special approval to manufacture or import the drug for a specific patient. This mechanism is commonly referred to as the compassionate use provision.
- Scope of Application:
- Compassionate use: This applies to patients with critical or serious medical conditions for whom no alternative approved treatment is available in Taiwan, or who have failed to respond to all available treatments, experienced disease recurrence, or have contraindications to available therapies. An application may be submitted for access to an investigational drug that has undergone scientific evaluation but has not yet been approved for marketing anywhere in the world.
- Special importation for an individual patient: This applies to a medicinal product that has been approved for marketing in another country but has not received marketing authorization in Taiwan. Its efficacy and safety have therefore not yet been reviewed and confirmed by Taiwan’s regulatory authority.
