Compassionate Use of Investigational Drugs: an Analysis of Ethical &Amp; Legal Challenges and Comparison of International Regulations

Do terminally ill patients who have exhausted all other available, government-approved treatment options have a right to experimental treatment that may prolong their lives? In April of 2016, Taiwan Department of Health revised Regulations on Human Trials to introduce a new mechanism under which patients that do not meet the inclusion criteria of clinical trials are able to access unapproved human cell treatment after sufficient clinical evidence of safety and effectiveness of the products are confirmed. This move symbolizes recognition of the so-called compassionate use of or expanded access to investigational drugs. Allowing patients to try unproven treatments, outside of controlled clinical trials, risks both the validity of the scientific study and the health of current and future patients who stand to benefit from the deliberate, careful new drug approval process as it currently operates under the regulatory framework. A range of constituents, including terminally ill desperate patients, pharmaceutical companies, government regulators, physicians and the public all have important but conflict interests in ethical issues in expanded access to experimental drugs. The regulations also face challenges in stipulating eligibility, review processes, and oversight mechanisms to reconcile the protection of vulnerable patients from the use of products that may be useless or worsen their conditions with the provision of opportunities to access to treatments that may be beneficial. The compassionate use under Article 3-1 of Regulations on Human Trials only allows access to human cell treatment (not all unapproved products). It also permits medical institutions to charge patients for obtaining experimental drugs (patients shall not be charged for investigational drugs in a clinical trial), raising concerns about equity and justice. Moreover, the above mentioned regulations is unlikely to withstand a constitutionality challenge since it lacks legislative authorization and contradicts with the Pharmaceutical Affairs Act. How safeguards adopted by current rules, including limiting eligibility and number of patients and pre-approval by the ethical review committee, may eliminate potential harms as well as how the system would be implemented need to be evaluated against numerous regulatory considerations. This project aims at exploring expanded access related ethical issues and comparing regulatory mechanisms of the US, the EU and Japan by adopting systematic literature review and comparative law research methods. Research results will be helpful in guiding the discussion and clarifying the ambiguities of the current regulations.

Project ID:PF10607-0658
External Project ID:MOST106-2410-H182-010