Access to experimental medicines for TB: ethical and human rights considerations.

ABSTRACT

The revised edition of the WHO's Ethics Guidance for the Implementation of the TB Strategy has added a new chapter on compassionate use (CU) and expanded access (EA) to TB drugs. CU and EA programmes authorise access to drugs that have not yet received marketing approval outside of clinical trials. They are aimed at allowing researchers access to investigational drugs in the absence of complete evidence of efficacy and safety to patients with multidrug-resistant (MDR) or rifampicin-resistant TB (RR-TB) when no other treatment options are available. In doing so, the guidance acknowledged the urgent necessity to offer these patients all possible treatments in respect of considerations of justice, human rights, human dignity, autonomy of the individual and protection of the community. Regulators are in general willing to accept a higher level of uncertainty in the risk-benefit assessment of medicines for life-threatening diseases when there is an unmet medical need. This attests to a paradigm change, which this article argues should also apply to allow for effective access to experimental TB medicines. Furthermore, in this article, we analyse the challenges connected to the establishment of a secure and effective regime of access to experimental drugs in the context of MDR/RR-TB as well as the ethical principles and human rights arguments in favour of the development of such programmes.