Right To Try Or Condemned To Die

Peter F Travers
Winter Springs Florida

This is an article about people you probably know personally, cancer victims. It’s also a story of heroes. There are no bad guys. However, that makes this story of life and death no less dramatic. Right to Try laws give terminally ill patients access to promising drugs and therapies that have not yet completed full FDA approval. These special access laws have now passed in over 35 states, including Florida. Make no mistake the laws are saving lives. But they could be saving hundreds of thousands more.

Let’s talk first about a success story, Orlando’s own Mark Angelo. Mark was diagnosed with Pancreatic Neuroendocrine cancer in September 2011. For the next three years Mark fought the good fight taking every approved treatment the Doctors recommended. Nonetheless his health spiraled downward. Mark’s quality of life declined as he lost weight. At this low point Mark reached out to the Excel Diagnostic Center in Houston TX. Thanks to Texas’s Right to Try law they could offer a promising, but not yet fully approved therapy, called PRRT. So Mark Tried it. The pictures of the thinning Mark on the plane leaving for Houston for the first time in Oct of 2014 is in striking contrast to the full-faced, healthy-looking pictures of Mark today. For the previous five and a half years Mark’s cancer was so dangerous he had to come in for checkups every three months. For the first time in December of 2016 Mark was told he no longer needed to come in for checkups every three months, but only every six. This family man proudly shows a picture of he and his wife out celebrating Mother’s Day this past May. He doesn’t hesitate to say it was made possible by Right to Try laws.

There are dozens, perhaps hundreds and soon there will be thousands of such examples, but there will not be hundreds of thousands. Only a very small fraction of those who could be helped are able to take advantage of these breakthrough treatments due the way the laws are written. Because of the unintended consequences of the way the law is written, hundreds of thousands of terminally ill patients are locked out of receiving such therapies, condemned to die. Even worse, many of those therapies are near completion of the full approval process. They work, but the law actually keeps them from helping the people the law was intended to help The problem is all the heroes of Right to Try have compromised on the law to the point that it does not achieve what experts cite as aundamental liberty. For example, David Curtis Glebe is a retired attorney with a Ph.D. in analytic philosophy from The Oho State University; David was the former Chief Counsel, Chief Ethics Counsel, Director office of Disciplinary Counsel for the Delaware Supreme Court. Of equal importance is that David is a cancer survivor kept alive by the same treatment received by Mark Angelo under Right to Try laws. According to David; “The fundamental liberties of individual citizens living in a free society should be legally acknowledged and protected to the greatest extent possible, consistent with the liberties of others and the corresponding lack of harm to society at large. Obviously, the first and foremost such liberty is the individual's right to life itself... Right to Try laws directly promote these important social and political values.”

Here is the problem, the good intentioned heroes of the Right to Try or compassionate access laws were so concerned that unscrupulous companies would price gouge desperate and dying patients that they made it unlawful to make any profit providing therapy under Right to Try. Companies could only supply treatment at cost. This means companies would need to disclose their cost to the world and for all practical purpose operate the effort at a loss. There is no sustainable business model that can provide a service at cost. This is especially true of one that could reach major scale under Right to Try that could treat hundreds of thousands of patients per year. The result is that small startup companies to large Pharma are finding it too risky from a business perspective to support Right to Try on a large scale. 

I know that in today’s world, it’s hard to think of medical startups and big Pharma as heroes too. But the facts say they are indeed. My favorite case in point is a company called NovoCure the developers of Tumor Treating Fields. This company was backed by Johnson and Johnson and Pfizer as well as multiple Venture Capital firms and is now a public company. The therapy is already approved for Glioblastoma and has had stellar results in phase 2 clinical trials for Pancreatic, Ovarian, and Non-Small Cell Lung Cancer. They most recently received humanitarian approval of their therapy for Pleural Mesothelioma from the FDA.

Why are they heroes? They so far have spent 17 years proving their therapy works, getting approvals, treating patients - and have yet to make a profit! They have many investors who want a return on their investment and yet are staying the course for the long term. They have a 10 to 15-year pipeline of clinical trials to put through the FDA process to prove they can treat 20 different types of cancer, including Breast and Liver cancer. If you take the twenty-year view, you could make the case that distracting their momentum with less than break even efforts, by
losing money on Right to Try patients could cause more people to die in the long run by further delaying the development and approval of their therapies. However, there is a solution to this dilemma.

I am the president of a startup medical device company with a patented therapy. Our therapy is an improvement on the existing Tumor Treating Field therapy. It is especially designed for late stage patients with disease in multiple locations - such as patients who have cancer in their lung and liver simultaneously. What’s more, our company is set up as a social enterprise. This means our business model requires us to provide discounts to the less fortunate and even requires investors to allow a percentage of profits to be directed toward providing free therapy to those who cannot afford to pay. Once our therapy completes a phase 1 clinical we could help tens of thousands of patients under Right To Try laws, but for the reasons stated we are not incorporating Right to Try activity in our business plan. Instead we are emphasizing medical tourism guiding people to western countries with first class medical infrastructures who allow more liberty.

There are dozens, if not hundreds of companies like ours with lifesaving therapies, but not fully through the approval process. To be able to make the case to their boards that they can offer Right to Try without doing serious financial damage to the company and products in development, something must change. In this spirit, I am proposing law makers add an amendment to the upcoming national bill presently in the house. The amendment is simple:

1. Any company providing a drug or therapy under Right to Try laws could not charge more
for treatment than similar therapies already on the market.
2. Any company providing therapy under Right to Try must show evidence of selling on a
sliding scale to those with lower incomes.

The above amendment would replace the requirement to only provide therapy at cost. It would also remove the need to disclose confidential and proprietary information to the government. The result would be a viable business model that could help thousands or even hundreds of thousands get access to life saving treatments. Its impact could be felt by someone you love.

But what could the overall impact be?

In 2010, the results of a phase 2 clinical trial showed that Tumor Treating Fields doubled the survival rates of Non-Small Cell Cancer (NSCLC) patients over chemotherapy alone. With no viable business model to move forward, the results were placed toward the end of a long pipeline. Today 7 years later, there is now open a 500-person phase 3 trial based on the 2010 study. The trial is costing millions of dollars and is expected to have the consistent good results as 3 other trials. The results of the present trial will probably come out in the year 2020. Here’s the bottom line. According to the American Cancer Society - from 2010 to 2017, over 700,000 Americans died from NSCLC. Between 2018 and 2020 an estimated 360,000 Americans are expected to die from the same disease. These people are condemned to die because there is no financially viable way for NovoCure (and others) to make that therapy available for them to try. This must change, people are dying, not because a treatment doesn’t exist, but because companies can’t afford to make it available!

We need your help. You can make a difference. Please write your representative and ask them to add our proposed amendment to the pending Right to Try legislation. You may know people in the battle of their lives. You just might help save them. Or yourself.

You can Google each of your representatives and send an email form right from their web page.

District   Representative                                 Party
7th           Stephanie Murphy (D-Winter Park)         Democratic
8th           Bill Posey (R-Rockledge)                         Republican
9th           Darren Soto (D-Orlando)                        Democratic
10th         Val Demings (D-Orlando)                        Democratic

A sample of the email to your representative can be found here: