Understanding RTT Parameters for Doctors & Hospitals
In addition to having a life threatening disease, eligible patients must be “trial challenged” in some way, either by inclusion/exclusion criteria, or as is often the case geographically. For Institutions and investigators conducting clinical trials Right to Try offers a unique opportunity to treat “screen fails” and both deliver access to patients, and create meaningful data for sponsors. For doctors treating patients without treatment options Right to Try offers a path to secure experimental treatments for their patients outside of clinical trials.
Treatment protocols under RTT require patient information and informed consent forms similar to FDA approved clinical trials. IRB approval is not required, but can be used at an institution’s discretion. The point of the law is to make the process as friction free as possible, not to impose any particular structure on health care providers.
Physicians and other service providers can be paid under Right to Try. Under the Act patients can only be charged the “cost” for the actual drug if they are charged for it at all. In most instances it is assumed the drug will be provided at the sponsor’s expense as in a traditional clinical trial. At Access Hope CRO we will also be administering programs that are “patient pay” for services and possibly even for the drug as well as programs that look more like traditional sponsor funded studies.
There are no limits to the number of patients that can be treated in a Right to Try program. There is no approval needed to begin treatment. There is no renewal required after a year as with the FDA’s expanded access programs and there is no reporting requirement for the treating physicians or institutions, unless you are also the sponsor or manufacturer of the drug.
The Federal, and almost all of the State Right to Try laws also provide a heretofore unavailable immunity from lawsuits throughout the entire ecosystem. This was an important inducement written into the law to improve access under Right to Try. Like the Federal law, almost all of the State laws have exceptions to this immunity for gross negligence and outright fraud.
Advantages of Right to Try & Real World Data For Drug Developers
It allows for the creation of data from a broader population than randomized clinical trials; often this is a population that is intended to be treated by the drug.
The process is considerably less expensive and more timely than costly post approval phase 4 trials, where often this very data will be required for label expansion.
Payers are increasingly demanding RWD for broader label reimbursement.
A NEW FDA MANDATE
The 21st Century Cures Act mandates the FDA find a way to incorporate RWD into the approval process. The time and money required to recruit and execute traditional randomized clinical trials is simply overwhelming the system, everywhere. While the final guidance for the use of RWD are not yet worked out, no one doubts that reliance on RWD is going to become an ever increasing part of drug development.
Finally, data collection, while limited in some ways compared to traditional clinical data collection will be easier, less expensive and less burdensome on physicians using electronic case report forms and electronic health care records systems to provide data.
"It’s not that we have so little but lose so much"