6:38 AM
Apr 12, 2019
 |  BioCentury  |  Politics, Policy & Law

Compassionate use of gene therapies raises ethical, commercial quandaries

Why compassionate use of gene therapies raises commercial and ethical challenges

No matter how quickly gene therapies are developed, there will always be a gap between the news that a product may radically alter the course of a disease and the drug’s approval. Like other investigational drugs for unmet serious conditions, that gap fuels requests for pre-approval access. The ethical and commercial quandaries associated with compassionate use of gene therapies are more challenging than those posed by most medicines.

The first cohort of gene therapies is targeted at rare diseases, and many are destined for patients with no alternatives who face irreversible loss of function or disease progression that will not wait for even an accelerated drug development timeline.

And given that many of these are being developed as one-time treatments, manufacturers are expected to set unprecedented prices.

Together, these factors complicate decisions by gene therapy developers to grant compassionate use requests.

The possibility of obtaining access via compassionate use could make patients reluctant to participate in clinical trials.

“One big thing companies will have to think carefully about is the eligibility criteria for their trials.”

Carolyn Chapman, NYU

In most cases developers will not charge for preapproval access. For a curative therapy, this can mean that every individual who is given a curative therapy prior...

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