Fairness in clinical trial design - helping those in need

The ethics of placebo-controlled trials.

Is the FDA justified in demanding placebo-controlled trials nowadays when there are other FDA approved medications available.

We are seeing that nowadays our trials with placebo arms have milder patients than in the past; this is probably because a more severe patient is not going to be enrolled in a placebo-controlled trial when there are other therapeutic options available to them.

This does not only make it difficult for us to compare current ”modern” clinical trials to older ones, it also only gives us data for how these medicines work in milder patients than in more severe patients.

This means that when you face a patient with more severe disease, it is harder to know how much these medicines will really help.

Placebo-controlled trials are skewed (and unfair?) in that they select for milder patients.

So, not only do we not have medications for primary and secondary MS, we know relatively little about how these medications work in more severe relapsing-remitting MS.

As doctors, we always look out for our patients.

So, we have to ask ourselves:

1. Is this fair?

2. How can we fix this inequality?

Studies like Genzyme’s Care-MS II trial of Campath (alemtuzumab) vs. placebo, in patients who had had a relapse despite having been on a disease modifying agent for t least 6 months, may help us in studying a sicker MS population.

Many of us focus on the ethics of exposing patients to placebo, but what about the patients discriminated against who cant enter the trial for fear of worsening on placebo?

- Dr. Daniel Kantor, MD BSE
Medical Director
Neurologique

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