Right to Try - Yes on Proposition 303

rvival, such as a child unable to get a lung transplant because federal regulations require a 10 year-old to be on a different list that a 12 year-old. We hear stories of families that have abandoned their lives in the U.S. and spent every penny they have to relocate abroad, where their loved one can be treated with a non-FDA approved medication. Often, those drugs were invented in the U.S. and have been successfully administered in other countries for years. World renowned cancer treatment and research centers create individualized medicines designed to treat individual patients but are unable to administer them because the treatments don’t fit the traditional FDA approval model. These are the stories and concerns that motivated the development of the Right to Try Act. Can anyone access investigational medications under Right to Try? No, only patients who have received a terminal diagnosis by their treating physician who has made the determination that the risk posed by trying an investigational medication is not greater than the risk posed by the underlying disease. Are these investigational medications safe? The investigational medications available under Right to Try have all successfully completed Phase 1 of an FDA approved clinical trial. This requirement ensures the medications are not inherently dangerous. Because there may be unknown side-effects, the patient must provide informed consent, noting their understanding of the investigational nature of the medication. Further, Right to Try requires that the medication remain under active investigation in an FDA approved clinical trial. Thus, if the FDA has serious safety concerns and pulls the plug on the trial, the medication would no longer be available under Right to Try. If there is an on-going trial, why don’t these patients wait for completion of the trial? Clinical trials often take 10 years or longer to complete. Terminal patients do not have the luxury of waiting years for medications. How many patients are able to obtain investigational medications in a clinical trial? Most terminal patients are unable to join a clinical trial. Each trial has a unique and stringent set of guidelines that define which patients are eligible to join. Patients that have an advanced stage of a disease, who have participated in another clinical trial, or who have any other complicating medical factor are routinely excluded. Other patients, who meet the guidelines for the clinical trial, are unable to join because the location of the trial makes it physically or financially impossible for them to participate. The end result is that only 3% of patients are able to join a clinical trial. How many patients are able to obtain investigational medications through an existing expanded access program? Very few. There are two types of expanded access programs – the treatment IND and the single patient IND. A treatment IND may be sought by a company for a specific investigational medication, usually late in the clinical trial process. If approved, the treatment IND would make the medication available to a defined population outside the clinical trial. While they can provide a single medication to a sizeable group of patients, treatment INDS are incredibly rare. Over the past three years, an average of 9 treatment INDs were approved by the FDA each year. The single patient IND occurs when an individual patient petitions the FDA for access to a specific medication. The application process is time-consuming and complex, and ultimately only a small number of patients succeed in obtaining access. For the last year in which data is available, the FDA granted only 904 patients access through a single patient application. Will Right to Try hurt or slow down clinical trials? If a company is concerned that access outside the clinical trial could slow down final FDA approval, companies have options under Right to Try. For instance, if a company is worried about enrollment in a clinical trial, it would be able to say that access will only be able available, 1) to individuals who are otherwise ineligible for the clinical trial, or 2) after the trial has been fully enrolled. What do doctors say about allowing patients access to investigational medications?

: In a recent poll, doctors from several medical specialties were asked if they would support “a proposal to change FDA law so that unapproved drugs or medical devices could be made available to physicians as long as they carried a warning label about their unapproved status.” Such a proposal was supported by 73% of neurologists, 70% of orthopedic surgeons, 69% of emergency room doctors and 68% of oncologists. Right to Try is a much more limited proposal that would only make such medications and devices available to terminal patients. Under Right to Try, who determines if a patient may use an experimental drug? This is determined jointly by the patient, the doctor and the drug manufacturer. The doctor first makes the determination that a patient is terminal and may recommend an investigational medication. The patient then needs to decide if he or she is willing to accept the risks involved in trying an investigational medication. Finally, the manufacturer decides whether it is willing and capable of fulfilling the request. Will companies supply the medicines? This is completely up to the individual companies. Companies will consider issues such as the costs of production and whether they will be reimbursed when making this determination. Under Right to Try, investigational medications could be available at any time after the successful completion of the FDA’s Phase 1 testing. While some companies will provide an investigational medication during Phase 2, others might not be comfortable providing access until Phase 3. Will companies be able to charge for the medicine? Companies should be able to recover the cost of additional doses of the investigational medication made available outside the trial. While large pharmaceutical companies often provide the medications pro bono in such instances, smaller and start-up firms may lack the resources to do so. For that reason, Right to Try allows a company to recoup the costs associated with producing and providing the investigational medications outside the clinical trial. Do individual states have the authority to pass Right to Try laws? It is well-established that the U.S. Constitution was designed to provide a floor of protection for individual rights, not a ceiling. States may provide additional and greater protections to individuals--and all of them do. For instance many states protect speech and privacy rights to a greater extent than the U.S. Constitution. The Right to Try Act is designed to provide the individual right to life by ensuring a right to medical self-preservation. Do FDA regulations supersede the Right to Try Act? Regulations such as those promulgated by the FDA can never preempt state laws that preserve constitutionally-protected rights, such as a person’s right to try to save their own life. Is this measure just giving people false hope? Right to Try is not simply about giving people hope – it’s about saving lives. While not all investigational medications will prove effective, many lives will unquestionably improve and be saved as a result of having access to them. Paid for by Your Right to Try Committee with major funding from the Goldwater Institute