What Does RTT Mean for Drug Companies

“The best way to predict the future

Is to create it” Abraham Lincoln

Right to Try (more formally known as S.204 the Trickett Wendler, frank Moniello, Jordan McLinn and Matthew Bellina Right to Try Act of 2017) is now the law of the land. Despite what you may have heard or read, this is not a "bad" thing for the drug development industry or patients. Indeed I believe it will be a boon to patients diagnosed with potentially terminal diseases, and an integral part of the inevitable move towards Real World Data collection required to change the broken drug discovery model of industry. RTT is the future of expanded access in the U.S.

In the heated debate leading to the laws’ passage many misconceptions have been baked in to the public’s understanding of what the law actually does and says. I was fortunate enough to be involved in the drafting of the Senate version of the law that was ultimately adopted by the House and signed into law, and proud to have been an advocate for it's passage. Through this blog, I will try to clear up the misconceptions, and even some intentionally misleading arguments that still linger; and explain how this form of expanded access will help both patients and industry.

For patients, it is important to understand that this does not open the door to snake oil salesmen. These arguments were an intentional scare tactic used to oppose the bill. Those who opposed the bill in good faith should be ashamed for not calling out the people who made these arguments. To be eligible under RTT a drug must not ONLY be through its’ phase 1 FDA safety trial, but be actively in the process of continued FDA approved trials, or having completed those trials, be in the queue for marketing authority from the FDA. With each new trial a drug goes through the level of FDA scrutiny increases, and a sponsors' obligations and costs increase. The minute a drug falls out of this queue, for any reason, including a trial being put on hold by the FDA, it is no longer an eligible drug under RTT. These drugs are only, always, by definition the exact same drugs being given to people in FDA approved studies. I promise you that if asked, the FDA will tell you that their review process does NOT approve “snake oil” for trials. This legislation was drafted specifically to rely on the FDA's expertise in areas like manufacturing and toxicology and safety in general, to insure that RTT drugs were all under FDA review on a continuing basis. NO drug can be administered under RTT that is not being administered to patients and scrutinized under an approved, ongoing FDA clinical trial process.

The new law DOES create a number of incentives to encourage drug development companies to make their experimental drugs available under RTT. This is a "good" thing. Everyone on both sides of this debate has agreed that a major hurdle for increasing expanded access is providing more incentives for industry to get involved.

For example, the law DOES put expanded access data from patients outside of the approval process, unless the sponsor chooses to ask the FDA to use it. The point here was to encourage drug originators to participate in expanded access. As a former biotech CEO developing a treatment for ALS I can tell you that we were besieged with compassionate use requests by patients outside of the inclusion/exclusion criteria for our trials. Certainly we wanted to know how the therapy would work with these patients, but we simply could not take even a possible risk that a problem with an expanded access patient would lead a halt to the ongoing clinical trials. Over and over again during this debate I heard those against the idea of RTT claim that the "real" problem limiting the FDA’s existing expanded access programs was industry participation. This law is a direct response to that legitimate critique. This provision is both necessary and at the heart of encouraging industry to participate in expanded access.

People against RTT argued that the bill should be opposed because it didn't guaranty access to experimental drugs. Of course none of the people making that argument have ever advocated FOR a law that WOULD guarantee access and mandate that companies provide access. Yet somehow the fact that RTT didn't "mandate" access was a fatal flaw. Again, the whole point of this law is to encourage more participation, to criticize it because it doesn't demand participation is simply silly.

People criticized the bill because they said it would lead to only "rich people" having access, because the bill leaves open the ability for Companies to charge (cost only) for their drug. This might have been a legitimate argument but for the Fact that the specific language of the bill incorporates the existing FDA rules and regulations around charging for experimental drugs, into the act. Though no one ever mentioned it, the FDA has rules for charging for an unapproved drug either in an FDA approved clinical trial or expanded access program, full stop. I never once heard these people criticizing RTT for this feature, criticizing the FDA's charging rules. Also on this point, let me just say that yes, the fact is that we have a serious economic divide in our Country with respect to access to all kinds of medical treatments including access to clinical trials, affordability of approved drugs and just plain basic health care. All of those issues deserve serious attention. It is certainly not however, a serious critique of this bill that it doesn't solve the societal problems our industry has in the area of income inequality and equal access to health care.

Finally, I want to talk about the future. It is clear to almost all in this industry, that the drug development model is broken. Drugs are simply too expensive to develop and take too long to get approved. The political success of RTT on first the State and now the Federal level also demonstrates that the unwritten contract between patients and the industry is fraying. We have ever increasing costs of developing and approving drugs and the inevitable push back of public and private payers with ever more limited resources. The democratization of information brought about by the internet (with the help of the scientific research industry) has created a new "demand" side to the equation. Patients want drugs sooner and cheaper and more broadly available. All of these trends, combined with the increasing inevitability of biomarker based diagnosis and treatment are converging to increase the importance of Real Word Data. Finding out sooner, in more patients not fewer, if drugs are safe and efficacious is the only way that the runaway costs of drug development can be tamed. Expanded access and big data are the future of drug development; and RTT is now the future of Expanded access in the U.S.

And yes, that may sound a bit like a prediction, but at Access Hope CRO we believe strongly that the best way to predict the future, is to help create it.

#RightToTry #PatientRights