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For patients that have exhausted all treatment options and for whom there are no clinical trials available there are two programs called “Right to Try” and “Expanded Access” that allow patients with life threatening diseases to access investigational drugs outside of clinical trials. At a glance these two programs may seem similar; however, there are some key differences to be aware of.

Right To Try Act

• Right to Try Act is a federal law passed in 2018 that allows terminally ill patients access to experimental drugs that have not yet been approved by the Food and Drug Administration (FDA).  

FDA Expanded Access

• The FDA’s Expanded Access (EA) program was formally established in 1987. This program allows patients with serious or life-threatening conditions to access experimental drugs outside of clinical trials. The Expanded Access program is also known as and referred to as “compassionate use”. You may also see Expanded Access requests referred to as “Emergency IND”, “Single-Patient IND”, or “Pre-Approval”.

Key Differences

Regulatory Oversight

• Right to Try Act
  • Bypasses FDA oversight and allows patients to request access directly from the drug manufacturer.

• Expanded Access 
  • Requires FDA approval and review by an Institutional Review Board (IRB) to gain access to an experimental drug. 
    • The FDA approves ~99% of Expanded Access (Compassionate Use) requests.
      • • Data: https://www.fda.gov/news-events/expanded-access/expanded-access-compassionate-use-submission-data  

Safety

• Right to Try 
  • Investigational drugs under this program must have a Phase 1 trial completed; however, a thorough safety review is not performed prior to giving patients access to the experimental drugs because the FDA is cut out of the process under this program.
    • Phase 1 trials tend to be small and safety data is limited even upon completion of the Phase 1 study. Further safety data is collected in larger Phase 2 studies and beyond.

• Expanded Access
  • FDA oversight and review by the IRB ensures safety issues are examined prior to a patient receiving treatment through the Expanded Access Program
    • Patients can access drugs throughout the development cycle including pre-clinical and either during or after Phase 1, 2, or 3 trials. 

Response Times

• Right to Try 
  • Companies have no legal time limit requirement in deciding whether to grant or deny access to their investigational drugs.

• Expanded Access
  • Mean decision time is ~2.2 days
    • By law cannot take longer than 30 days in regard to the FDA’s part of the process.

Cost

• Right to Try
  • Experimental drugs are not covered by insurance and costs are uncapped under this program. Patients are typically responsible for the full cost of the experimental drug, including administration fees.

• • Companies can’t make a profit and can only charge patients for the direct costs of manufacturing and distributing the drug, not for research and development costs that went into creating it. However, there is no regulatory oversight to ensure this is enforced. 
    • Companies can exploit this and make treatments astronomically expensive and take advantage of patients seeking hope in a cure with an experimental drug.
      • This publication titled “On Patient Safety: A Right to Try, not Exploit” mentions private equity backed companies exploiting the Right to Try Act and also mentions a case where a stem cell company was planning to charge $300,000 for treatment under the Right to Try program.
        • https://pmc.ncbi.nlm.nih.gov/articles/PMC8208421/#R14

• Expanded Access
  • Most insurance companies do not covered Expanded Access programs (including Medicare); however, there is regulatory oversight to ensure patients are not taken advantage of by companies in regard to out of pocket costs.
    • Expanded Access permits pharmaceutical companies to charge only the direct costs of making the drug available, but prohibits them from making a profit.
    • FDA makes sure companies are charging patients in good faith for their costs. The FDA is involved in looking at the purchase price and the documentation from the company to justify the purchase price.

Liability

• Right to Try 
  • Has an indemnification clause that shields the manufacturer, clinicians, and hospital from liability associated with treatment with these investigational drugs.
    • Since safety isn’t thoroughly evaluated under this program if something does go terribly wrong the doctor, pharmacist, hospital, or drug company can’t be sued by the patient or their heirs. 

• Expanded Access
  • Typically includes an indemnification clause for the drug manufacturer. Specific terms of the clause can vary depending on the situation and the agreement between the parties involved.
  • Expanded access program does not typically provide full indemnification for a doctor
    • However, throughout the entire history of the Expanded Access program there have been no such lawsuits.

Navigation Tools

• Right to Try
  • No tools currently exist to help patients and clinicians navigate this complex process.

• Expanded Access
  • Project Facilitate
    • Created by the Oncology Center for Excellence to provide a single point of contact call and information center to help oncology healthcare providers or regulatory professionals submit an Expanded Access Request for an individual patient with cancer through FDA’s Expanded Access Program.
      • For more information please visit: https://www.fda.gov/about-fda/oncology-center-excellence/project-facilitate

• • FDA Raegan-Udall Foundation 
    • Expanded Access Navigator online
      • Provides patients and clinicians with a directory of pharmaceutical companies that offer Expanded Access programs. The site also offers information to aid patients in making informed decisions about the risks and benefits of investigational treatments.
        • To access the website and navigator please visit: https://navigator.reaganudall.org/

Summary Point

• Without the FDA program’s protections and oversight, Right to Try could be more harmful than helpful for patients, especially those of the vulnerable cancer population.

  • “Although terminally ill patients might assume they have nothing to lose by trying an investigational treatment, this is not necessarily the case, according to Holly Fernandez Lynch, JD, MBE. ‘They could take a medication that could make them die faster or in a worse way,’ she said. ‘They might be losing valuable time with their families, or they might be missing out on palliative options.’ ” (Source: Hoag Levins)

Source

• The majority of the information in this section was from an article titled “Right to Try: A ‘well-intentioned’ but ‘misguided’ law” by Jennifer Bryne published in HemOnc Today on March 10th, 2020.
• The full article can be read here: https://www.healio.com/news/hematology-oncology/20200303/right-to-try-a-wellintentioned-but-misguided-law