Ethical implications of scarcity of patients for HTA

Prof. dr. G. J. van der WiltRadboud University Medical CentreDepartment of Epidemiology, Biostatistics & HTA

Case-study: discontinuation of reimbursement of Mexiletine for patients with Non-myotonic Muscular Dystrophy (NMD) for lack of evidence of clinical effectiveness

How can we identify and resolve the ethical issues in a systematic and transparent way?

The problem of moral inquiry

We can not deductively infer what follows from our commitment to abstract, general moral principles in concrete cases (‘open-texture’ of moral principles)

The balancing or weighting of conflicting moral principles is arbitrary

Possible solution: Specifying norms (Richardson, 1990). Qualifying our moral commitments in concrete cases by adding clauses indicating what, how, by what means, by whom or to whom the action is to be, or may not not be done.

Identifying candidate norms

[1] All patients should be equally protected from harmful interventions.

[2] Communities should be protected from health care interventions that produce outcomes that do not outweigh their costs and risks.

[3] Access to health care should be equal for all patients.

Should the costs of Mexiletine for patients with NMD be reimbursed? No (1 & 2) / Yes (3)

Can these general norms be specified in such a way, that the conflict is resolved and yet the reasonable motivation behind the initial, unqualified norms is captured by what one ends up doing?

[1] All patients should be equally protected from harmful interventions and this may be achieved ex ante (regulatory approval), ex post (notably through conducting and reporting N-of-1 trials and careful follow up), or both, provided that the individual patient knows and understands the associated risks and has consented to treatment.

[2] Communities should be protected from health care interventions that produce outcomes that do not outweigh their costs and risks and the level of evidence of safety and (cost)-effectiveness that is required (= the level of uncertainty that is deemed acceptable) should be proportional to the associated public health risk

[3] Access to health care should be equal for all patients.

The costs of Mexiletine should be covered

Discussion

It is important to be aware of the possibilities and limitations of moral inquiry.

Systematic approaches such as specifying norms may be helpful in identifying the relevant ethical norms and in resolving conflicts in a transparent and discursive way.

Scarcity of patients poses ethical dilemmas that deserve to be examined in such a way.

The analysis suggests that the discontinuation of the reimbursement of Mexiteline for patients with NMD is ethically unfounded.