COLUMNS

'Right to Try' is way better than just wait to die

Joan Lappin

The U.S Congress finally took steps to end one of the most perplexing ethical dilemmas facing people with terminal diseases: How to gain access to an experimental drug that might be your last chance at life if you are terminally ill?

The Right to Try Act, written by Sens. Ron Johnson, R-Wisconsin, and Joe Donnelley, D-Indiana, was passed unanimously by the Senate in August 2017. Even though President Donald J. Trump mentioned the bill in his State of the Union address in January, it languished in the House until this week. The final vote was 250-169, with 22 Democrats crossing over. Trump is expected to sign the bill.

Old memories die hard. Again this week, the specter of patent medicines and snake-oil salesmen was injected into the House debate. Yes, there are many products that claim to erase wrinkles, improve your brain or lengthen your life that have no more therapeutic benefit than a placebo.

Getting a drug through the Food and Drug Administration approval process is one of the more daunting challenges to companies trying to bring new drugs or medical devices to market. It is fairly typical for drug trials to take more than a decade to complete and cost hundreds of millions of dollars. That makes the whole process nigh-on impossible for small companies with limited financial resources. It puts them in a position that allows stock manipulators to force down their price by spreading false rumors and then endlessly shorting the shares. This can result in excessive dilution if the company must sell hundreds of millions of shares for just pennies just to stay afloat.

This becomes especially true if the company has an exceptional product or revolutionary approach that is truly threatening to the big pharmaceutical companies with the most to lose. These giant corporations have the financial wherewithal to support drug tests for years. They use these expensive trials to justify the tens of thousands of dollar they charge for the drug once approved by the FDA. In some cases, the drugs gain approval even if they only extend life by a few weeks or months beyond the standard and help only a tiny percentage of people, often with terrible side effects.

While it has been possible for a determined person to try to gain access to drug trials, the trials are often constructed so that truly terminally ill people are excluded to improve the end results. For example, if you have already been in another trial and failed to respond, you are less likely to be approved for a new trial. If you have too many other ailments than the one being treated or are over a certain age, you are also likely to be disqualified.

Initially, drugs must undergo preclinical testing in animals before they may proceed to testing in humans. Phase 1 trials usually involve 20 to 80 patients to ascertain safety, the common side effects and how the drug is metabolized and excreted, and to establish toxicities. Phase 2 is to establish the effectiveness of the drug. Phase 3 is an expanded study of more patients, (300 to 3,000) to determine the most effective dosage. It can include possible uses of the drug in combination with other approved drugs.

The new legislation states that a drug must have successfully passed Phase 1 before you may try it. Drug companies must report deaths, but only once a year.

The change in the law immediately takes the foot off the neck of some very vital small companies struggling to bring great new drugs to market right now. I’ll be writing about one such company left for dead that should rise from the ashes in the weeks ahead after the American Society of Clinical Oncologists meetings I’ll be attending in Chicago scheduled for June 1-4.

Joan Lappin CFA has been called an “investment guru” by Business Week and a “top manager” by the Wall Street Journal. The Sarasota resident founded Gramercy Capital Management, a registered investment advisor, in 1986. Email her at jlappinCFA@gmail.com. Follow her on twitter: @joanlappin. Her past columns appear at heraldtribune.com/topics/joan-lappin. Neither she, Gramercy Capital, nor its clients own any security mentioned in this column.