'Right to try' efforts have been timid and poorly conceived

'Right to try' efforts have been timid and poorly conceived
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Great societies are judged by how they protect their citizens and we are failing at that when it comes to terminal disease patients. Cancer, Alzheimer’s and cardiovascular disease still prematurely end millions of  lives each year while the rest of us wonder if we are next. How long must we tolerate this?

Current failed efforts to address this greatest of all assaults on humanity — “Right to Try” (RTT) — initiative have been timid, poorly conceived and will likely do more harm than good.

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Terminal patients must have immediate access to credible treatment options and the medical regulatory arena must be reformed to make drug companies focus on curing terminal diseases. This has worked before with the Orphan Drug Act (ODA) of 1983,  which transformed therapeutics for rare diseases and Congress must pass similar legislation to do the same for terminal patients. For the sake of discussion let’s call this legislation the “Terminal Disease Act” or TDA.

 

To provide immediate assistance to terminal patients, the TDA would remove Phase III clinical trials as an approval requirement for terminal disease drugs. It would mandate that only a phase I safety trial and one positive phase II efficacy trial of at least 100 patients be required for FDA approval. This provision would be retroactive and apply to all the terminal disease drugs currently in phase III trials which will now become FDA approved drugs and be immediately available to terminal patients.  

A 12-year period of commercial exclusivity for any approved terminal disease treatment should be the second provision of the TDA. This will obviate the need for patents and would make vast amounts of currently un-patentable science and technology available to be developed. The ODA did this for rare diseases and it gave rise to numerous companies and new drugs for rare diseases.

These two reforms will provide terminal patients with immediate access to scores of new drugs with demonstrated efficacy and insure that cures will come faster than currently possible. Eliminating the need for phase III trials and patents for terminal disease drugs will make them much easier to develop because of the reduced time and capital required.

Phase III trials constitute the major expense of drug development and often take three to four years to complete. Eliminating phase III trials and patents would have an especially dramatic on venture capital funded startup companies by enabling them to reach the FDA approval stage without the need for excessive equity offerings or corporate partners. Start-ups are where the innovative approaches to drug discovery often occur.

This legislation will speed both terminal disease cures and the spiraling health-care budget which is on its way to bankrupting our economy. End of life care constitutes a major component of the health-care budget and it is largely due to terminal diseases.

The TDA will immediately change the lives of terminal patients by giving them access to scores of clinically tested drugs (all drugs finishing a phase II clinical trial and those currently in phase III) rather than the untested investigational pre-clinical drugs that the RTT initiative proposes.

This is one of the reasons that RTT has failed to gain Congressional approval; it pushes the efficacy issue out of the process since these drugs have not demonstrated any efficacy in clinical trials. Patients should not be subjected to such risk.

An additional major flaw in RTT is that it does nothing to incentivize and accelerate cures for terminal disease while the TDA will supercharge the entire pharmaceutical and biotech industry to focus on this greatest of all human tragedies. A great society must provide this.

Paul J. Marangos is the author of, “A Roadmap for Curing Cancer, Alzheimer’s and Cardiovascular Disease” He has published over 250 research papers, is inventor on 14 patents and has co-founded four biotech companies.