Reform Is Needed Now
We don’t expect surgeons to operate with 1950s technology. Patients would die needless deaths. Yet today, the FDA is operating with a decades-old regulatory system that is preventing patients from accessing the latest medical treatments. Reform is needed now. A friend’s life, a loved one’s life, or your life could one day depend on it.
Today, new medical treatments have to be approved by the FDA before they can be made commercially available to patients. That happens through a clinical trial, which can take years, even decades – time dying patients do not have. It also requires testing one medication on a large number of people. However, the latest kinds of treatments are tailor-made for one individual, based on their genetics. By definition, they are designed and can only work for one person and can’t go through typical FDA processes in a timely manner.
This is bureaucracy at its worst—a Catch-22 of catastrophic proportions. A major legislative reform is needed to protect patients’ right to access those treatments—the Right to Try for Individual Treatments (Right to Try 2.0). Download the model legislation here.
Patient-Centric Reforms Can Save Lives
America doesn’t have to wait for the FDA to reform itself in order to put patients first. Just a few years ago, terminally ill patients were forced to endure for the FDA’s slow-moving approval process in order to access potentially lifesaving treatments. Today, patients have new hope and more options under the first Right to Try law.
Thanks to the work of citizens and state lawmakers, 41 states and the federal government enacted Right to Try, opening a new pathway for terminally ill patients to access treatments. Today, Right to Try is working. Dying patients nationwide have been able to pursue, with their doctors’ recommendation, treatments that have passed basic FDA safety evaluation but have not yet received full approval. It has improved and saved the lives of people with illnesses like cancer, ALS, and now COVID-19. Right to Try for Individualized Treatments applies this principle to the latest in medical treatments and is now law in six states, passing with strong bipartisan support.
Safety First with Right to Try for Individual Treatments
Similar to the original Right to Try law, Right to Try for Individual Treatments will have safeguards to protect patients who are pursuing treatments. First, the law will require patients to have considered approved treatment options. Second, the treatment will have to be recommended by a doctor who is in good standing with her licensing organization and will not be compensated directly by the manufacturer for so prescribing it. Third, the patient will have to give written informed consent regarding the risks associated with taking the investigational treatment.
Frequently Asked Questions
Why is Right to Try 2.0 needed?
Rapid medical innovations have made it possible to take an individual’s genetic information and create a treatment for that individual person. More patients, especially those with rare and ultra-rare illnesses, will pursue these treatments when they have exhausted other options. Unfortunately, the FDA’s current regulatory scheme is not designed to handle these kinds of individual treatments, and that will keep life-saving medication out of the hands of patients unless reforms are adopted.
Under the FDA’s outdated system, new treatments are subject to a clinical trial, which is designed to learn something about a treatment that will be generally applicable to all the patients with the same disease. The current clinical trial evaluation is based on treatments for large population, not an individual patient which were not a reality when the system was created more than a half-century ago. The end result is that an individualized treatment is still subjected to the same clinical trial process as a single treatment that is intended for hundreds or thousands of patients. But that’s not how these new individualized treatments work.
For example, a therapeutic cancer vaccine can be designed based on an individual’s diseased cells or unique genetic mutations, allowing it to stimulate the patient’s immune response and target cancerous tumors. But that unique treatment only works for that patient. The FDA’s existing clinical trial system cannot work in cases like this because its safety and testing protocols aren’t matched to this kind of singular treatment.
By providing an additional pathway that works in tandem with the federal law and protections for patients, it is our hope that more patients today will access the right treatment at the right time.
Who qualifies for Right to Try 2.0?
To be eligible for Right to Try for Individual Treatments, a patient must meet the following conditions:
- Be diagnosed with a life-threatening or severely debilitating illness;
- Has considered approved treatment options;
- Has a recommendation for an investigative individualized treatment from their physician; and
- Gives written informed consent regarding the risks associated with taking the investigational treatment.
What drugs or treatments qualify for Right to Try 2.0?
The treatments available under the law must meet the following conditions:
- must be investigational
- must be individualized to the specific patient, and
- must be based on analysis of the patient’s genomic sequence, human chromosomes, deoxyribonucleic acid, ribonucleic acid, genes, gene products (such as enzymes and other types of proteins), or metabolites.
Is RTT 2.0 safe?
Individualized Right to Try treatments must comply with the same requirements and regulations required by federal law for the protection of human subjects in research. Facilities that hold a Federalwide Assurance (FWA) have been accepted and approved by the federal Department of Health and Human Services in meeting these standards, including:
- establishing internal laboratory procedures and review processes to ensure the highest ethical standards for protecting patients,
- using an Institutional Review Board (IRB), which is an independent committee that evaluates every proposed research project and human research to ensure that research and treatments are conducted and administered ethically, providing an independent check on these treatment decisions, and
- ensuring that the patient understands the nature of the treatment and participation through patient consent.
Most facilities that would offer individualized Right to Try treatments are already operating under the FWA requirements.
Is a manufacturer or company required to make a treatment available?
No. manufacturers and drug companies are not required to provide treatments to patients under Right to Try laws. It would not be appropriate to force companies to provide treatments that they do not think are the right fit for a patient or if they do not have sufficient supply.
Does Right to Try 2.0 force doctors to submit a request for a treatment a patient wants to try?
No. Doctors have a responsibility to ensure that patients are given treatments that they believe, in their professional opinion, could help them. A doctor who does not think a treatment will help is never obligated to make a request for the treatment.
What can manufacturers charge for treatments?
Federal law bans companies from making a profit on any drug or treatment that has not been approved by the FDA, but the law does allow companies to recover the costs that are directly related to providing an individual treatment. Existing regulations govern what can and cannot be included in the calculation for determining the direct costs that can be charged. You can read that formula here.
This means that a patient could be charged for the direct costs of providing their individual treatment, but the company cannot make a profit.
How does payment work?
Just like with Right to Try and the FDA’s existing Expanded Access program, insurance companies and taxpayer-funded healthcare programs like Medicaid or Medicare are not required to cover the cost of investigational treatments, but they may choose to do so. Some insurance companies have covered the costs of investigational treatments used by patients under state Right to Try laws, but others have not. Each patient’s cost situation will be different and determined by their individual insurance company or program and their own financial resources.
Is Right to Try for Individual Treatments a Republican or Democrat idea?
Neither. Right to Try is for patients and their loved ones. The original Right to Try had strong bipartisan support in every state and in Congress. It passed unanimously in some states and almost unanimously in most.
Providing care to those who are out of options isn’t partisan. It is about getting the right treatment, to the right patient, and at the right time.
The FDA controls drug approval. Can states legally do this?
Yes. The U.S. Constitution provides a floor of protection for individual rights, not a ceiling. States may provide additional and greater protections of individual rights—and all of them do. The right to try to save one’s own life with safe medicines is one of the most precious rights of all. And the Supreme Court has long recognized that states have great latitude in regulating health and safety, including medical standards, which are primarily and historically protected as a matter of local concern.
This was the legal reasoning behind the original state Right to Try laws, which helped patients across the country even before Right to Try became federal law.