Right to Try: 
Questions and Answers

Today, new medical treatments must be approved by the FDA before they can be made commercially available to patients. In brief, Phase 1 trials are undertaken to ensure the new medication or treatment is safe. Once passing those safety trials, drugs are evaluated for their efficacy in treatment. Unfortunately, the entirety of the FDA’s clinical trial process, can take years, even decades to complete.

Too often, this is time that patients afflicted with debilitating or life-threatening rare diseases do not have.

Further, because the FDA’s clinical trial process was developed over 60 years ago, it is focused on drugs that are targeted at diseases impacting a larger portion of the population, not rare or ultrarare diseases. 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. Therefore, they cannot be evaluated using the current FDA processes.

This is bureaucracy at its worst—a Catch-22 with potential catastrophic impact for those with rare and ultrarare diseases.

Meaningfully significant legislative reform is needed to protect patients’ right to access this class of treatments—the Right to Try for Individual Treatments (Right to Try 2.0).

View the model legislation here.

Right to Try (Original)

The original Right to Try laws, enacted in 41 states and federally in 2018, provide a pathway for terminally ill patients to access investigational drugs, biological products, and devices that have completed a Phase 1 clinical trial. These laws aim to provide hope and a potential last resort for patients facing terminal illnesses, bypassing the full extent of an otherwise lengthy FDA approval process.

Key Characteristics of Original Right to Try:

• The original Right to Try permits patients to gain access to drugs that have successfully passed the FDA’s Phase 1 Safety Trials but are still undergoing the Phase 2 Efficacy Trials.

• The original Right to Try generally applies to drugs developed for conditions that impact a significant number of patients.

• The original Right to Try does not apply to drugs or substances restricted by the Drug Enforcement Administration, nor does it apply to drugs that have not passed the FDA’s Phase One Safety Trials.

Right to Try for Individualized Treatments (Right to Try 2.0)

Building on the success of the original law, the Goldwater Institute is now advancing Right to Try for Individualized Treatments, specifically focusing on individualized, investigational treatments that are highly personalized and on the cutting-edge of therapy for patients with rare and ultrarare diseases. Rapid advancements in medical science have enabled the development of personalized treatments tailored to an individual's unique genetic makeup. These treatments hold immense promise for patients with life-threatening or severely debilitating illnesses, particularly those with rare and ultra-rare conditions. However, the current FDA regulatory framework, designed for drugs mass produced for large populations, presents significant hurdles for the timely access of these individualized therapies.

16 states and counting have already enacted this next generation of the Right to Try movement.

Congress and the FDA have been agonizingly slow to update the process by which new drugs and treatments are made available. But, patients need not wait for FDA reform to put patients first.

Just 10 years ago, terminally ill patients were at the mercy of the FDA’s slow-moving approval process in order to access potentially lifesaving treatments. Today, patients have new hope and more options under the original Right to Try law.

The Right to Try for Individualized Treatments builds on the success of the original Right to Try law by allowing patients with life-threatening or severely debilitating diseases to access individualized investigational treatments that would almost certainly never become available under the original Right to Try pathway.

Rapid medical innovations have made it possible to take an individual’s genetic information and create a treatment specific to that person. As medical innovation continues to advance at a more rapid pace, more patients, especially those with rare and ultra-rare illnesses, will seek to pursue these treatments when all other treatment options have been exhausted.

The Right to Try for Individualized Treatments creates a pathway for those patients to safely access promising innovative treatments.

Similar to the original Right to Try law, Right to Try for Individual Treatments has safeguards in place designed to protect patients who are pursuing individualized, investigational treatments: 

1. The law requires patients to consider all currently approved treatment options. 
   
   
2. The individualized, investigational treatment must be recommended by a physician caring for the patient who is in good standing  with the applicable state licensing authority and who will not be compensated by the manufacturer for prescribing the treatment. 
   
   
3. The patient will be required to provide written informed consent acknowledging their knowledge and acceptance of the risks, benefits and alternatives to the investigational, individualized treatment. 
   
   
4. Treatments can only be administered in an eligible facility that is in compliance with federal ethical and safety mandates meant to protect human subjects in medical research, e.g. federal wide assurance guidelines. 

The rapid pace of advancements in medical technology have made it possible to create an individualized treatment for patients with certain types of rare and ultra-rare diseases that are based in that person’s DNA. When patients with these conditions have exhausted all other approved treatment options, they should have the right to pursue an individualized, investigational treatment. However, the FDA’s current regulatory scheme is simply not designed to accommodate these types of individualized treatments.

Under the current drug approval process, new medications and treatments are subject to FDA-prescribed clinical trials. This clinical trials process is designed for treatments that will be applicable to a large population of patients with the same disease. The current clinical trials process is NOT designed with individual patients in mind. Individualized treatment were simply not a reality when the system was mandated over 60 years ago.

Thus, as a result, individualized treatments remain subject to the same clinical trial process designed for medications intended for thousands of patients. For example, therapeutic cancer vaccines can now be designed based on an individual’s diseased cells or unique genetic mutations. This allows the vaccine to stimulate the patient’s immune response, targeting cancerous tumors. But this unique treatment only works for that specific individual patient.

The FDA’s existing clinical trial system cannot work in cases like this. The current safety and efficacy clinical trials process simply was not designed to accommodate this type of therapeutic intervention.

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.

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;
• Have considered approved treatment options;
• Have a recommendation for an investigative individualized treatment from their physician; and
• Gives written informed consent regarding the risks associated with taking the investigational treatment.

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.

Individualized treatments made available by Right to Try 2.0 must comply with the requirements and regulations mandated by federal law for the protection of human subjects research. Facilities that hold a Federalwide Assurance (FWA) have been accepted and approved by the United States Department of Health and Human Services in meeting the following standards: 

• Established internal laboratory procedures and review processes that ensure the highest ethical standards for protecting patients,
• Use of an Institutional Review Board (IRB), an independent committee that evaluates every proposed research project and human research. IRBs are designed to ensure that research and treatments are conducted and administered ethically, providing an independent review and monitoring of treatment decisions, and
• Comprehensive informed consent standards designed to ensure that the patient understands the nature of the treatment including inherent potential risks, benefits and alternatives.

Most facilities that would offer individualized Right to Try treatments are already operating under the FWA requirements. 

No. Manufacturers are not required to provide treatments to patients under Right to Try.

No. Physicians have a responsibility to act on their professional opinion to ensure that patients are only given treatments they believe are appropriate and warranted. A physician who does not believe a treatment will help is never obligated or mandated by Right to Try 2.0 to make a request for the treatment.

Federal law prohibits manufacturers and companies from making a profit on any drug or treatment that has not been approved by the FDA. However, RTT 2.0 specifies that companies may recover 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.

As with the original Right to Try and the FDA’s existing Expanded Access program, insurance companies and taxpayer-funded healthcare programs (Medicaid or Medicare) are not required to cover the cost of investigational treatments. However, they may choose to do so. In fact, some insurance companies have covered the costs of investigational treatments under state Right to Try laws. Each patient’s circumstances will be different and ultimately be determined by their individual insurance coverage and their financial resources.

No. In each of the sixteen states in which Right to Try for Individualized Treatments has become law, the votes in the legislature have been unanimous or nearly so. RTT 2.0 has been signed into law by governors of both political parties.

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 the most fundamental of rights.

Additionally, the Supreme Court has long recognized that states have great latitude in regulating public health and safety, including the prescription of standards for medical care. The practice of medicine is almost exclusively a matter of concern regulated by state standards. This principle of legal doctrine in inherent to the original state Right to Try laws, which helped patients across the country even before Right to Try became federal law.