Help for the Terminally Ill


The statistics on death are impressive – one out of one die. But that’s no reason not to deny the grim reaper his day until absolutely necessary.

Recently Congress passed “Right to Try” legislation which grants terminally ill patients the right to access investigational drugs that haven’t yet proven their clinical efficacy. They are required to pass initial safety testing.

What more can be done?

Paul J. Marangos, CEO of Biomedica Partners, writes in The Wall Street Journal that he believes much more can be done. Marangos spent 13 years at the National Institutes of Health and co-founded five biotech companies in the ensuing 30 years, all focused on developing cures for terminal diseases. With this experience, he has some insights on the process of bringing new cancer cures to market.

His first complaint is the lengthy FDA regulatory process. The Phase III clinical trials, which involve many hundreds and sometimes thousands of patients, are by far the most costly and time-consuming requirement for FDA approval. This phase has been eliminated in this new legislation, but only for terminally ill patients.

His second complaint concerns the strict stipulations for obtaining patents, which require demonstrating that the treatment works in the relevant laboratory experimental models and that it is not obvious to those in the industry. Getting patents can take five years or more, representing a substantial risk for the sponsoring company. Moreover, much of the existing scientific research is unpatentable. Very often biochemical data is published first, and then after a period it falls into the public domain.

Proposed Solutions

Marangos suggests the following reforms:

  • Reduce the FDA approval requirements so that treatments for terminal disease no longer have to go through Phase III clinical trials. He believes one Phase II clinical trial of at least 100 patients is sufficient. This would reduce the time and expense of brining new drugs to market.
  • Make this change retroactive – This would allow all treatments currently in Phase III trials to be immediately approved. According to the trade group PhRMA, there are more than 200 Phase III trials currently under way just for cancer drugs. There are 26 more in Phase III for Alzheimer’s Disease.
  • Mandate a 10-year period of marketing exclusivity on treatments for terminal diseases. This would eliminate the need for patents, shortening the process and making unpatentable research commercially available.


These changes would incentivize pharmaceutical companies to focus more on terminal disease treatments, greatly enhancing our ability to fight diseases like never before. Innovative start-ups would multiply to explore treatment ideas off the beaten path, as happened before with the passage of the Orphan Drug Act of 1983, which granted exclusivity to treatments for rare disease and gave rise to many new drugs.

The usual excuse for not making such changes is the concern for safety. But Phase III trials are not about safety – they are about efficacy. When treating terminal diseases there is by definition little hope of success with currently available treatments. Let’s make it easier to try new approaches and new drugs while the terminally ill still have time. There’s little to lose when the prognosis is death.


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