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Questions to ask your doctor

Compassionate Use & Pre-Approval Access

Compassionate Use & Pre-Approval Access

Judith Hinton Andrew, Rock Composite 22
Questions to ask your doctor

If you and your doctor are considering the use of investigational medicines that have not yet been approved by government health authorities, there are important things for you to consider. From the patient perspective, it can often help to have a list of important questions to discuss with your doctor. Based on our collective professional experience in medicine, research, and patient advocacy, we wish to share the following considerations to help guide a constructive, open, and trusting conversation with your doctor. This is not a complete list by any means, but instead represents a selection of those most pressing questions that we have personally heard most often from patients and their families.

Treatment considerations and scientific evidence

  1. Are there any approved treatments that I have not tried and if so, why not?
  2. Why do you think this investigational treatment will help treat my disease?
  3. (a) Has anyone else received this investigational treatment and if so, how did it work?
    (b) What is the current proof and rationale that this investigational treatment may help?
    (c) What are the currently known medical risks of this investigational treatment?
    (d) What are the unanswered questions about this investigational treatment?
    (e) When will we know the answers to these unanswered questions?
  4. If I take this treatment, how do you think it will impact my daily life in the short term and in the long term?
  5. If I proceed and this investigational treatment does not work, what are the potential alternative treatment options?
  6. If I take this treatment, does it make me ineligible for any other treatments/programs?
  7. If I do not proceed, what are the alternatives?
  8. What else should I know about this treatment?

Financial considerations

  1. Will I have to pay for the treatment, or will it be covered from another source such as my healthcare system, insurance plan, or the company making the investigational medicine?
  2. What will I be financially responsible to pay?
  3. If I have side effects and become hospitalized, who will pay for the treatment?

Logistical considerations

  1. Will I still be able to visit my current doctor if I take this treatment? If I cannot see my doctor, will my doctor be given information about my treatment?
  2. Will I need to travel anywhere to receive the treatment and if so, where to, how frequently, and for how long? Is support available for me to pay for this travel?


  1. What other resources are available to me if you cannot answer my questions regarding payment, treatment effects, etc.?
  2. If I were your family member, what would you want me to ask you?

Additional resources include:

From the American Cancer Society: http://www.cancer.org/treatment/understandingyourdiagnosis/afterdiagnosis/afterdiagnosis-what-should-i-ask-my-doctor

From the Agency for Healthcare Research and Quality: https://www.ahrq.gov/patients-consumers/patient-involvement/ask-your-doctor/10questions.html

From the National Institutes of Health: https://www.cancer.gov/about-cancer/treatment/drugs/access-experimental

Authored by:

Beverly Harrison, Head, Patient Support; Christine MacCracken, Director Patient Support; and Amrit Ray, M.D., Janssen R&D, LLC

© 2015 Janssen R&D, LLC