BioCentury's websites will be down for upgrades starting at 9 p.m. PDT on Monday, August 26. We expect the downtime to last no more than 6 hours, and we apologize for any inconvenience.

12:00 AM
Feb 16, 2015
 |  BioCentury  |  Regulation

Commentary: Josh Hardy: An unanswered call

Compassionate access still unaddressed a year after Josh Hardy media campaign

Almost a year has passed since an extraordinary campaign saved the life of Josh Hardy and started a public discussion about the roles and responsibilities of drug companies and government to provide compassionate access to promising, unapproved therapies.

The good news is that Josh has turned eight and is healthy.

The bad news is that the changes that are needed to help future Josh Hardys haven't been made.

The Hardy experience did succeed in creating a public discussion of important questions, notably about the equity of releasing potentially life-saving medicines to individuals whose plight catches the public imagination, while hundreds or thousands of similarly situated patients are passed over.

Academic and commercial conferences have been held to discuss expanded access, and Washington insiders have held closed-door "stakeholder" meetings to discuss policy options. White papers are being written.

Legislation that would make useful but limited improvements has been introduced, including proposals that would require companies to announce early access policies when they become eligible for one of FDA's accelerated development or review pathways, which by definition apply to the kinds of unmet needs represented by desperate patients.

And this month, FDA cut some of the red tape wrapped around applications for what it terms "expanded access."

All of these actions are laudable, but all fall short of what is needed: a system that meets the needs both of individual patients who deserve opportunities to be treated with experimental drugs, and of society as a whole that benefits from the swift approval of new drugs. Until the expanded access system is reshaped, these two needs will continue to conflict.

Two-step test

There is a very...

Read the full 1387 word article

User Sign in

Trial Subscription

Get a 4-week free trial subscription to BioCentury

Article Purchase

$150 USD
More Info >