====== The Right to Try Act: Your Ultimate Guide to Accessing Investigational Drugs ======
**LEGAL DISCLAIMER:** This article provides general, informational content for educational purposes only. It is not a substitute for professional legal advice from a qualified attorney. Always consult with a lawyer for guidance on your specific legal situation.
===== What is the Right to Try Act? A 30-Second Summary =====
Imagine receiving the most devastating news of your life: you have a terminal illness, and all available, approved treatments have failed. You see a glimmer of hope in a new, experimental drug that is still in testing, but you don't qualify for its [[clinical_trial]]. For decades, the path to accessing such a drug was a long, complex bureaucratic maze run by the [[food_and_drug_administration_(fda)]], a process many dying patients simply didn't have the time to navigate. It was a race against the clock where the odds felt stacked against you.
The **Right to Try Act** was created to change that. It establishes a second, alternative pathway for patients with life-threatening diseases to access unapproved, investigational drugs directly from the manufacturer, without needing to go through the FDA's traditional application process. It is a law born from the passionate belief that patients who are out of options deserve the right to try and save their own lives, even with treatments that are not yet proven to be safe or effective. It's about giving hope and autonomy back to patients in their most vulnerable moments.
  *   **Key Takeaways At-a-Glance:**
    *   **A Second Path to Treatment:** The **Right to Try Act** provides a federal framework for eligible patients with life-threatening conditions to request investigational medical products that have completed at least a Phase 1 [[clinical_trial]].
    *   **Bypassing FDA Pre-Approval:** Its most significant feature is that it allows patients and their doctors to deal directly with a drug manufacturer, removing the requirement for an [[food_and_drug_administration_(fda)]] application that is mandatory under the traditional [[expanded_access_program]].
    *   **It Is a Right to Ask, Not a Right to Receive:** A critical reality of the **Right to Try Act** is that it **does not** compel a pharmaceutical company to provide its drug; the manufacturer retains the final and absolute authority to say yes or no.
===== Part 1: The Legal Foundations of the Right to Try Act =====
==== The Story of Right to Try: A Grassroots Movement for Hope ====
The journey of the Right to Try Act is a powerful story of patient advocacy fueled by desperation and hope. For years, the only way for a patient to get an experimental drug outside of a formal clinical trial was through the FDA's "compassionate use" or [[expanded_access_program]] (EAP). While well-intentioned, the EAP was often perceived by patients and doctors as slow, burdensome, and difficult to navigate, involving significant paperwork and uncertain timelines—a luxury that patients with terminal illnesses do not have.
Frustration grew as stories emerged of patients dying while waiting for approval. This frustration ignited a powerful grassroots movement, spearheaded by patient advocates and organizations like the Goldwater Institute. They argued that for those with no other options, the freedom to choose a potentially life-saving, albeit risky, treatment was a fundamental right.
This movement first took root at the state level. In 2014, Colorado became the first state to pass a "Right to Try" law. Over the next four years, a wave of similar legislation swept the country, with over 40 states enacting their own versions. However, these state laws had a major weakness: they couldn't override federal law governing interstate drug commerce or provide immunity from FDA oversight. A federal law was needed to create a uniform, national standard and, crucially, to provide robust liability protections for doctors and manufacturers.
==== The Law on the Books: The Federal Right to Try Act of 2018 ====
The culmination of this movement was the **Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017**. Signed into federal law on May 30, 2018, this act established a single, consistent pathway for patients across the United States. It is often referred to simply as the federal Right to Try Act.
The core of the law is codified in Section 561B of the Federal Food, Drug, and Cosmetic Act. One of its key passages states:
> "...an eligible patient has a right to be treated by an eligible investigational drug, and a sponsor of an eligible investigational drug or a manufacturer of an eligible investigational drug may make the eligible investigational drug available to an eligible patient..."
In plain English, this means the law creates a legal avenue for this process to happen. It removes the need for FDA permission for a specific patient's access, but it uses the permissive word "may," underscoring that the final decision rests with the drug company. It also sets forth specific criteria for the patient, the doctor, the drug, and the manufacturer to qualify for this pathway.
==== A Nation of Contrasts: Federal vs. State Right to Try Laws ====
Before the federal act, a patchwork of state laws existed. While the federal law now provides the primary framework, understanding the differences highlights why a national standard was considered so important. The federal law's key advantage is its ability to provide clear liability protections and preempt conflicting state laws related to interstate drug access.
^ **Feature** ^ **Federal Right to Try Act (2018)** ^ **Typical State Laws (Pre-2018)** ^ **What This Means for You** ^
| **FDA Oversight** | Explicitly bypasses the requirement for FDA pre-authorization for patient access. | Could not override federal FDA authority, creating legal uncertainty and risk for manufacturers. | The federal law provides a clearer, more legally secure path without needing to file an application with the FDA. |
| **Liability Protection** | Provides strong, specific legal protections for physicians, hospitals, and manufacturers from lawsuits related to adverse patient outcomes. | Protections were often weaker and varied by state, offering less comfort to drug companies. | Doctors and drug makers are more likely to consider a request under the federal law because their legal risk is significantly lower. |
| **Drug Eligibility** | Drug must have completed a Phase 1 clinical trial and be under active development. | Eligibility requirements varied, with some states having vaguer or different standards. | The federal law sets a clear, uniform baseline for what drugs can be considered, ensuring a minimum level of safety testing has been done. |
| **Geographic Scope** | National. A patient in any state can use it to request a drug from a manufacturer in any other state. | Limited to the state. Created confusion when a patient in a "Right to Try" state needed a drug from a company in a state without such a law. | Your rights and the process are the same whether you live in California, Texas, New York, or Florida. It created a single, national system. |
===== Part 2: Deconstructing the Core Provisions of the Act =====
To truly understand the Right to Try Act, we must break it down into its essential components. Think of it as a four-part checklist where the patient, the drug, the doctor, and the manufacturer must all meet specific criteria.
==== Patient Eligibility: Who Can Use Right to Try? ====
Not every patient can use this pathway. The law is very specific and is designed only for those in the most dire circumstances. To be an "eligible patient," you must meet all of the following conditions:
  * **Life-Threatening Diagnosis:** You must have been diagnosed with a "life-threatening disease or condition." This is generally understood to mean diseases like advanced cancers, ALS, or other conditions where the likelihood of death is high unless the course of the disease is interrupted.
  * **Exhausted Approved Options:** You must have tried all approved treatment options for your disease and found them to be ineffective. You cannot skip standard, FDA-approved therapies to go straight to an experimental one.
  * **Unable to Participate in a Clinical Trial:** You must be unable to enroll in a [[clinical_trial]] for the investigational drug. This could be because you don't meet the trial's strict eligibility criteria, the trial is full, or you are geographically too far from a trial site.
  * **Provided Written Informed Consent:** You must sign a document of [[informed_consent]], prepared by your physician, that clearly explains the risks of taking an unproven treatment.
==== Drug Eligibility: What Treatments Qualify? ====
Just as with patients, not every experimental drug is available under Right to Try. The "eligible investigational drug" must meet these standards:
  * **Completed Phase 1 Trial:** The drug must have successfully completed a Phase 1 [[clinical_trial]]. This is the earliest stage of human testing, which primarily evaluates the drug's safety, dosage range, and side effects, not its effectiveness. This ensures the drug has at least a preliminary human safety profile.
  * **Not Yet FDA Approved:** The drug cannot be approved or licensed by the [[food_and_drug_administration_(fda)]] for any use. Once a drug is approved for any condition, it falls outside the scope of this act.
  * **Under Active Development:** The manufacturer must be actively pursuing final FDA approval for the drug, meaning it is still in the [[clinical_trial]] process or an application is being prepared or reviewed.
==== The Gatekeepers: Roles of Physicians and Manufacturers ====
The physician and the drug manufacturer are the two key players in the Right to Try process.
  * **The Treating Physician:** Your doctor plays a crucial role. They must be in good standing and willing to certify that you meet all the patient eligibility criteria. They are responsible for overseeing your treatment, monitoring for side effects, and providing the [[informed_consent]] document. However, a doctor is under no obligation to suggest or support a Right to Try request if they believe it is not in your best interest.
  * **The Drug Manufacturer (Sponsor):** The manufacturer holds the ultimate power. They have the sole discretion to provide the drug. They can say no for any reason—such as limited supply, concerns about the drug's suitability for the patient, potential disruption to their ongoing [[clinical_trial]]s, or financial cost—and they are not required to provide an explanation.
==== The Shield: Liability Protections Explained ====
Perhaps the most important provision of the act is the robust "safe harbor" it creates. This was designed to remove a major hesitation for companies and doctors. The liability protections include:
  * **For Manufacturers and Physicians:** They are shielded from legal liability related to a patient's death or injury, as long as there was no [[gross_negligence]] or willful misconduct.
  * **For FDA Approval Process:** The [[food_and_drug_administration_(fda)]] cannot use negative clinical outcomes from a Right to Try case to delay or deny the final approval of a drug, unless the agency determines that the data is critical to assessing the drug's overall safety. This was a huge concern for companies who feared that a single negative result in a very sick patient could jeopardize a multi-billion dollar drug's future.
===== Part 3: Your Practical Playbook =====
If you or a loved one are facing a terminal illness and believe Right to Try might be an option, navigating the process can feel overwhelming. This step-by-step guide breaks it down into a clear, manageable plan.
==== === Step 1: Have an Open Conversation with Your Doctor === ====
The first and most important step is to talk to your treating physician. This is not a path you can walk alone.
  - **Discuss Your Condition:** Confirm with your doctor that you have exhausted all approved treatment options and that your condition is considered life-threatening.
  - **Explore Clinical Trials First:** Ask your doctor to help you exhaustively search for any available [[clinical_trial]]s you might be eligible for. The clinical trial pathway is almost always preferable because it involves rigorous safety monitoring and the treatment is usually provided at no cost.
  - **Raise Right to Try:** If no trials are viable, ask your doctor if they are familiar with the Right to Try Act and if they would be willing to support a request on your behalf. Be prepared that some doctors may be hesitant due to the risks involved.
==== === Step 2: Identify Potential Investigational Drugs === ====
If your doctor is supportive, the next step is to research potential treatments.
  - **Consult with Your Doctor:** Your physician is the best resource for identifying drugs in development for your specific condition.
  - **Online Resources:** Websites like ClinicalTrials.gov can be used to find drugs currently in testing, even if you don't qualify for the trial itself. Patient advocacy groups for your specific disease are also excellent sources of information.
  - **Focus on Phase 2/3 Drugs:** While a drug only needs to have completed Phase 1, companies are often more willing to provide drugs that are further along in development (Phase 2 or 3) and have shown some evidence of effectiveness.
==== === Step 3: The Doctor Initiates Contact with the Manufacturer === ====
The request must come from your physician.
  - **Formal Request:** Your doctor will need to formally contact the drug manufacturer. Most large pharmaceutical companies now have a specific department or contact person for "compassionate use" or "Right to Try" requests.
  - **Providing Medical Records:** The company will require detailed medical records to verify your diagnosis, treatment history, and to determine if their drug might be suitable for you.
==== === Step 4: Understanding the Manufacturer's Decision === ====
This is often the most difficult part of the process: waiting for a decision.
  - **Patience is Key:** It may take weeks or even months for a company to respond.
  - **Prepare for a "No":** It is essential to understand that the answer is often no. Companies turn down the vast majority of requests due to limited supply, safety concerns, or other business reasons.
  - **The Decision is Final:** A manufacturer is not required to justify its decision. The Right to Try Act gives you the right to ask, not the right to receive.
==== === Step 5: If Approved - Treatment and Informed Consent === ====
If the company agrees to provide the drug, you will move forward with treatment.
  - **Informed Consent:** You will need to review and sign the [[informed_consent]] document. Read it carefully. It will detail all the known and unknown risks, the fact that the drug may not work, and that you are waiving certain rights to sue if something goes wrong.
  - **Treatment Plan:** Your doctor will work with the manufacturer to develop a treatment protocol.
  - **Cost:** The law allows manufacturers to charge for the drug, but they cannot profit from it. They can only charge for the direct costs of manufacturing. This can still be extremely expensive, and insurance rarely covers it.
==== Essential Paperwork: The Written Informed Consent Document ====
The single most critical piece of paperwork in the Right to Try process is the [[informed_consent]] document. This is more than just a signature; it's a legal and ethical cornerstone of the entire process.
  * **Purpose:** Its purpose is to ensure that you, the patient, fully understand what you are agreeing to. You are acknowledging that you are stepping into the unknown.
  * **What it Must Include:**
    *   A clear explanation of the known risks and potential benefits of the investigational drug, based on the data from the Phase 1 trial.
    *   A statement that the drug's safety and effectiveness are not yet fully known.
    *   A description of any alternative treatments available, including palliative care.
    *   A clear notice that you have the right to withdraw your consent and stop treatment at any time without penalty.
    *   An explanation of any costs you may be expected to cover.
===== Part 4: The Right to Try in Action: Controversies and Impact =====
Since its passage, the Right to Try Act has been a subject of intense debate. It's not a traditional law with landmark court cases; rather, its impact is measured in patient stories, ethical discussions, and its relationship with the FDA's existing program.
==== The Great Debate: Right to Try vs. FDA's Expanded Access (EAP) ====
The Right to Try Act did not replace the FDA's [[expanded_access_program]] (EAP); it created an alternative to it. Proponents of the FDA's program argue it provides a crucial layer of safety oversight that Right to Try lacks. Supporters of Right to Try argue that this oversight is an unnecessary bureaucratic hurdle for patients who have already accepted the risks.
^ **Aspect** ^ **Federal Right to Try Act** ^ **FDA Expanded Access Program (EAP)** ^
| **Primary Goal** | Patient **autonomy** and speed. | Patient **safety** and oversight. |
| **FDA Role** | No role in approving a specific patient's access. The FDA is simply notified after the fact. | The physician must submit an application to the FDA, which reviews the case and must approve it. |
| **Process Speed** | Potentially faster, as it removes the FDA application step. The timeline depends entirely on the manufacturer's response time. | Can be slower due to FDA paperwork and review, though the FDA approves over 99% of requests, often in days for emergencies. |
| **Oversight Body** | None. The decision is between the patient, doctor, and manufacturer. | An Institutional Review Board (IRB), typically at a hospital, must often review and approve the treatment plan to ensure it is ethical. |
| **Data Collection** | The law requires annual summaries from manufacturers, but public data on usage and outcomes is very limited. | The FDA tracks every application and can monitor outcomes, contributing to the overall safety knowledge about a drug. |
==== Early Outcomes and Data: Has It Worked? ====
One of the biggest criticisms of the Right to Try Act is the lack of transparent data. While manufacturers must report the number of doses supplied each year to the FDA, this information is not always made public in a clear or timely manner. This makes it very difficult to answer the question: "How many people have been helped?"
Proponents argue that success isn't just measured in cures, but in providing hope and a sense of control to patients. They point to anecdotal stories of patients who believe their lives were extended or their quality of life improved.
Critics, however, raise serious concerns. They argue that without structured data collection, we learn nothing from these experiences to help future patients. They also worry that the law can promote "false hope" and expose vulnerable patients to potentially harmful and ineffective compounds with no safety net.
==== A Patient's Story: The Human Face of the Law ====
Consider the hypothetical case of "Jane," a 45-year-old with a rare and aggressive form of cancer. She has been through multiple rounds of chemotherapy and radiation, but the cancer continues to spread. Her oncologist tells her there are no more approved options.
Jane and her family discover a new immunotherapy drug in a Phase 2 trial. She doesn't qualify for the trial because her cancer has spread to her brain. Desperate, her family raises the Right to Try Act with her oncologist. After weeks of research and discussion, the doctor agrees to make the request. The family endures an agonizing two-month wait before the drug company agrees to provide the drug on a compassionate basis.
For six months, Jane's tumors shrink. She is able to attend her son's graduation, a milestone she thought she would never see. Ultimately, the cancer adapts and she passes away. Was the Right to Try a success for Jane? For her family, who got six precious months with her, the answer is an unequivocal yes. For a scientist, the lack of data from her case makes it an unproven anecdote. This dichotomy is at the very heart of the Right to Try debate.
===== Part 5: The Future of the Right to Try Act =====
The legal and ethical landscape of medicine is constantly evolving. The Right to Try Act sits at the intersection of medical innovation, patient rights, and regulatory science, making its future a topic of ongoing discussion.
==== Today's Battlegrounds: The Ethical Tightrope ====
The core controversy surrounding Right to Try is unlikely to be settled soon. It pits two deeply held values against each other:
  * **Patient Autonomy:** The belief that individuals have the right to make their own decisions about their bodies and their lives, especially when facing death.
  * **Patient Protection:** The principle of "first, do no harm" ([[primum_non_nocere]]) and the societal obligation, carried out by the FDA, to protect the public from unsafe and ineffective drugs.
Debates continue to rage: Does Right to Try undermine the integrity of the [[clinical_trial]] system by siphoning off potential participants? Does it exploit desperate patients who may not fully grasp the long odds and severe risks? Or is it a vital last resort that rightfully empowers patients?
==== On the Horizon: How Technology and Society are Changing the Law ====
The future of Right to Try will be shaped by the very science it seeks to accelerate.
  * **Personalized Medicine:** As treatments like gene therapy and CAR-T cell therapy become more common, the traditional three-phase clinical trial model may not always apply. These are often treatments designed for a single individual. Right to Try could potentially become a more common pathway for accessing these bespoke therapies.
  * **Data and AI:** In the future, there may be pushes to amend the law to require better data reporting. AI and real-world evidence analysis could one day allow us to learn from the experiences of Right to Try patients without requiring them to be in a formal trial, bridging the gap between autonomy and data collection.
  * **The Role of the FDA:** The ongoing debate will continue to influence the [[food_and_drug_administration_(fda)]]. In response to Right to Try, the FDA has worked to streamline its own [[expanded_access_program]], simplifying the application form and improving its turnaround times. This "competition" may ultimately benefit patients by making all pathways more efficient.
===== Glossary of Related Terms =====
  * **[[clinical_trial]]:** A research study in human volunteers to answer specific health questions; the primary way to determine if a new treatment is safe and effective.
  * **[[compassionate_use]]:** A common term for the FDA's Expanded Access Program.
  * **[[expanded_access_program]]:** The formal FDA pathway for patients to gain access to investigational medical products for treatment outside of clinical trials.
  * **[[food_and_drug_administration_(fda)]]:** The U.S. federal agency responsible for protecting public health by ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, and medical devices.
  * **[[gross_negligence]]:** A conscious and voluntary disregard of the need to use reasonable care, likely to cause foreseeable grave injury or harm to persons, property, or both.
  * **[[informed_consent]]:** A process in which a patient is given important information, including possible risks and benefits, about a medical procedure or treatment to help them decide if they want to be treated.
  * **[[investigational_drug]]:** A drug that has been tested in a laboratory and has been approved by the FDA for testing in people, but has not yet been approved for sale.
  * **[[liability]]:** Legal responsibility for one's acts or omissions.
  * **[[palliative_care]]:** Specialized medical care for people with serious illness, focused on providing relief from the symptoms and stress of the illness.
  * **[[phase_1_clinical_trial]]:** The first stage of human testing, which evaluates a drug's safety, dosage, and side effects in a small group of people.
  * **[[phase_2_clinical_trial]]:** The second stage of human testing, which assesses a drug's effectiveness and further evaluates its safety.
  * **[[phase_3_clinical_trial]]:** The third and typically final stage before approval, which confirms a drug's effectiveness, monitors side effects, and compares it to commonly used treatments.
  * **[[primum_non_nocere]]:** A Latin phrase that means "first, do no harm," a fundamental principle in bioethics.
  * **[[statute]]:** A written law passed by a legislative body.
===== See Also =====
  * **[[clinical_trial]]**
  * **[[informed_consent]]**
  * **[[food_and_drug_administration_(fda)]]**
  * **[[expanded_access_program]]**
  * **[[medical_malpractice]]**
  * **[[wrongful_death]]**
  * **[[products_liability]]**