Rare Disease Clinical Trials: Facts and figures

In Clinical Development by Cameron2 Comments

This month Total Orphan Drugs is taking a look at clinical trials within the orphan drug space. We will be looking at the current state of trial design, challenges to implementing rare disease trials, as well as a glance towards the future of clinical trials for orphan drugs.

This week we have put together an infographic providing information about the clinical trial process, as well as a few facts and figures unique to rare disease clinical trials.

What are you’re thoughts on clinical trials within the orphan drug sector? Let us now by commenting below, or chatting with us via email.

rare disease clinical trial facts total orphan drugs


  1. Christopher McCabe

    The first figure (average cost per patient in trials) is unhelpful. The average cost is going to be a lot higher – its the total cost, which the companies seek return on, that helps judge reasonable returns. Pity, rest of info graphic is excellent; but first part looks like marketing to support price of orphan drugs.

  2. Michele Longabaugh

    Since I have had my rare disease, anal cancer (5000) diagnosed per year, it costs 0 dollars to run the clinical trial that doesn’t exist. We are left, like the other rare cancers, to depend on discoveries in the larger studied cancers (breast, prostate) to perhaps be made eligible for compassionate use without proven benefit. When the time comes, you and your doctor can beg big pharma for treatment and you’ll get it if you can convince your insurance to pay for it. It doesn’t make me sad or mad…just tired.

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