Media Doctor Canada
Follow us on Twitter








"Drug approved to treat rare but potentially deadly disease"

National Post

Source: National Post

Published: 25 Jul 2022

Category: Pharmaceutical

Rating: (2½ stars)

what they said (Hover the mouse cursor over underlined words for more info)

WASHINGTON (AP) - The first drug treatment for the rare but potentially deadly inherited disease Hunter Syndrome was approved Monday by the Food and Drug Administration.
Shire Human Genetic Therapies of Cambridge, Mass., manufacturer of Elaprase, said it plans to have the drug available in the United States within 30 days.
The FDA approved Elaprase, also known as idursulfase, as an "orphan" drug. Orphan drugs are developed to treat illnesses that affect relatively small numbers of people and the manufacturer is granted a seven-year period of exclusive marketing...

how did it rate? (more information)

Criteria Rating
Total Score 5 of 10
Availability of Treatment Satisfactory (?)
Novelty of Treatment Satisfactory (?)
Disease Mongering Satisfactory (?)
Treatment Options Not Satisfactory (?)
Costs of Treatment Satisfactory (?)
Evidence Not Satisfactory (?)
Quantification of Benefits of Treatment Not Satisfactory (?)
Harms of Treatment Satisfactory (?)
Sources of Information Not Satisfactory (?)
Relies on Press Release Not Applicable
Quantification of Harms of Treatment Not Satisfactory (?)

what we said (Hover the mouse cursor over underlined words for more info)

This is a small study of a very expensive treatment for a very rare disease. We get few details of the disease and even fewer details of the benefits of the treatment other than the patients are able to complete, on average "35-metre greater increase in the distance walked in six minutes compared to the patients on placebo." We have no idea what the placebo patients could have done. And all this for $300,000 per year?

While there is a list of adverse events, including life-threatening reactions associated with the treatment, the key quote goes to the researcher who conducted many of the trials of the product. This isn't good enough for the reader, who would most benefit from someone, outside the study, commenting on the degree to which this treatment is or isn't an advance for these patients.

public forum

There are currently no comments on this article.

voice your opinion in the forum

  • All comments and feedback submitted to Media Doctor are subject to editorial approval before being made viewable by the public. It may take up to a week for your comments to be approved. Additionally, no response will be given to questions posed in public comments. Media Doctor does not provide medical advice, or answers to medical questions posed by the public.
  • If you provide your email address it will not be displayed to the general public.
  • Comments may be edited by Media Doctor to remove defamatory or sensitive statements, and brand names.
  • Required fields are marked with an asterisk (*).
Name: *
Comments: *
Copyright © Media Doctor Canada