Expanded Access | Information for Patients

Emerging Therapies

• How might I access experimental treatments legitimately?

  !Qualifies this

  “On a legitimate access route, costs can still fall to you. Under the United States programme the product and the medical services around it are often not covered by insurance or Medicare, and the ethics review may be charged to the patient. What is covered varies by country.”

  Says all the costs of the investigational product and the medical services around it have to be considered, and that these are often not met by private insurance or Medicare. It describes United States cover; other countries fund this differently.

  These are often not covered by third-party payers such as private insurance or Medicare.

  What costs may be involved?

• How might I access experimental treatments legitimately?

  !Qualifies this

  “On a legitimate access route, costs can still fall to you. Under the United States programme the product and the medical services around it are often not covered by insurance or Medicare, and the ethics review may be charged to the patient. What is covered varies by country.”

  Adds that the ethics review can itself be billed to the patient, depending on which board reviews the request.

  Depending on the IRB, you may have to pay for the review.

  Treatment can begin once everyone agrees to expanded access to the medical product.

• How might I access experimental treatments legitimately?

  ✓Supports this

  “Meeting the criteria for an access programme does not mean you will get the treatment, because several other parties each have to agree.”

  Says meeting the criteria is not enough. The doctor, the ethics committee and the company each have to agree, and a company is under no obligation to supply a product outside its own trials and may not have enough of it.

  Even if you meet the criteria under the law and FDA regulations, the licensed physician, the IRB, or the company may not agree that expanded access is appropriate for you.

  If I meet the criteria, will I qualify?

• How might I access experimental treatments legitimately?

  ✓Supports this

  “Access outside a trial is generally reserved for a serious condition with no satisfactory alternative, where joining a trial is not possible and the likely benefit justifies the risk. These are the United States criteria; other countries set their own.”

  The US regulator's own account of expanded access, also called compassionate use. It applies when the condition is serious or life-threatening, there is no comparable or satisfactory therapy, taking part in a trial is not possible, and the possible benefit justifies the possible risk. Other countries run their own schemes on their own rules.

  Whenever possible, an investigational medical product should be used as part of a clinical trial.

  What is expanded access?

• How might I access experimental treatments legitimately?

  ✓Supports this

  “A treatment obtained outside a trial is still unproven: it may turn out not to work, and it may cause side effects nobody has seen yet.”

  States the same uncertainty about anything supplied under expanded access. These products have not been found safe and effective for the use in question, may or may not work, and may cause side effects that were not expected.

  Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause serious side effects that were not expected.

  What is expanded access?

• How might I access experimental treatments legitimately?

  !Qualifies this

  “Legitimate access is not a sale at a profit. A company may cover the cost itself, but where it charges, the charge must be justified and limited to recovering what the product costs to make, research, develop and handle. This is the United States rule; other countries set their own.”

  Adds a limit worth knowing. A company may cover the cost itself, but it is allowed to charge, and where it does the charge must be justified and no more than what it costs to make, research, develop and handle the product.

  However, if a company decides to charge for the investigational medical product, the costs must be justified and limited to those necessary to recover costs of manufacture, research, development and handling.

  What costs may be involved?