Framework for Experimental Drug…

Outside Editorials

Ms. Nelson’s recent article starts by framing the debate this way:

A terminally ill cancer patient has the right to refuse treatment, but does that same patient have the right to demand access to investigational therapies? That question has spurred emotional debate from both sides of the issue, ranging from advocates who believe that experimental drugs should be made readily available, to those who argue for restrictions. An article in the December 17 issue of JAMA attempts to provide a framework for a workable and effective balance of access and oversight.

She follows with a summary of the JAMA piece, quotes from its authors, and some quotes from others working to change (or defend) the system. 

This quote from our Executive Director follows the table of potential issues and solutions from the JAMA piece:

Scott Riccio, executive director of Accelerate Progress, a public charity created to increase the speed and efficiency of the systems that most directly affect patients fighting cancer and other life-threatening diseases, believes that the authors have raised important issues and proposed reasonable solutions.

“It is well worth further discussion,” he commented to Medscape Oncology. “I think that the current system should be revamped to speed access to the most promising new therapies by recognizing that our learning about these therapies is not a ‘binary’ event, as it’s essentially now treated. In other words, right now the system essentially says the drug is unsafe and ineffective right up until the point at which it’s approved, when we ‘suddenly’ know it to be safe and effective.”

In reality, information is continuously accumulated over time, he added. “When the benefit-to-risk profile of the therapy can be said to have a reasonable likelihood of outweighing the risk to the patient from their disease, the system ought to begin to expand access to the therapy to those patients that fit the analysis, and begin to expand or restrict access as further data emerge.”

To be clear, when we speak of “expanding access to the therapy to those patients that fit the analysis...” we mean that at this point, the system should be approving these options for use by those patients and their doctors.  We believe that meaningful access would be provided by and should be measured in new therapeutic approvals (be they conditional/provisional, accelerated, or other similarly named constructs).

The full article should be read here (opens in new window).

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