Muriel Lights' Candle Designs: What is Compassionate Care?

Muriel Lights' Candle Designs: What is Compassionate Care?: As I was going through my e-mail I came across a petition for a cancer patient who had been denied experimental medication.  The patien...

What is Compassionate Care?

As I was going through my e-mail I came across a petition for a cancer patient who had been denied experimental medication.  The patient had tried every medication available and believed that the compassionate care medication available only from Drug Companies who is sponsoring drug trials was her husband only hope.   Medical professionals use the term “compassionate use” to refer to the treatment of a seriously ill patient using a new, unapproved drug when no other treatments are available in other words a cancer patient only hope.  Later that week on channel 2 news another person who had battled Uterus cancer for five years also had taken all available treatments.  Compassionate care medication was her last hope she also had a petition to help her get access to the trial medications available. Now the question for me was what is compassionate care and why was it so difficult for these cancer patients to get.

Compassionate Care                                                                                                                            Drugs that are being tested but have not yet been approved by the US Food and Drug Administration (FDA) are called investigational drugs. These drugs are generally available only to people who are taking part in a clinical trial (a research study that is testing the drug). Being able to use one of these drugs when you are not in a clinical trial has many names, but is most commonly referred to as compassionate use.  The FDA first approved investigational drugs to be use in this way for critically ill patients since 1987.  If you are not in a clinical trial there are 2 ways a drug company can allow use of their unapproved drug: through expanded access programs (EAP’s) or through single patient access.  Compassionate drug use is legal, but it’s tightly restricted to people who meet certain conditions. The FDA first approved investigational drugs to be used in this way for critically ill patients in 1987. For people who aren’t in clinical trials, there are 2 ways a drug company can allow use of their unapproved drug: through expanded access programs (EAPs) or through single patient access. 

Expanded access   
A company sponsoring a drug in the late stages of drug development, such as Phase III clinical trials, can offer expanded access programs for patients who are not able to enroll in a clinical.  The FDA generally approves these EAPs if the drug has shown that it works at least somewhat to treat cancer in the clinical trials that are being done. This can allow a lot of people access to the unapproved drug, as long as they meet the requirements of the EAP.

Single patient access    
Patients who don’t qualify for either clinical trials or an expanded access program (if one exists) may be able to get the unapproved new drug by applying for single patient access. In this case, the patient’s doctor must first ask the drug company if the drug can be used for the patient and see if the drug company will supply it. If the company agrees, the patient’s doctor works with the drug company to ask the FDA to approve the drug for use by this one patient.

The FDA requires the doctor to send information about the patient, why the request is being made, the proposed treatment plan, and a signed informed consent from the patient (see our document Informed Consent for more on this). The length of time it takes to get single patient access varies. But if it’s an emergency, the FDA can complete the paperwork in 24 hours.

Who might benefit from using unapproved drugs?      
According to guidelines from the National Cancer Institute, most compassionate drug use is for patients who meet all of these conditions:

• Have advanced disease
• Have used standard treatments and they have not worked
• Are not eligible for any clinical trial that’s in progress
• Have no other treatment options
• Have a type of cancer for which there’s reason to expect the investigational drug will help
• Are likely to have benefits that outweigh the risks involved

In a case like this, the doctor may consider trying to get a new, unapproved drug for a patient to see if it will help.

Is it difficult to get drugs for compassionate use?   
Perhaps the biggest problem with compassionate drug use is that it’s hard to get the drug. The simplest way to get an unapproved drug is through a clinical trial. But many people with life-threatening diseases can’t find suitable clinical trials, live far from cancer research centers, or are not eligible for any studies being done.

What I have seen from the patients fighting to get compassionate use they have to do a lot of advocating far beyond the process in place in a time when fighting their cancer should be their only worry. Getting the drug through expanded access programs (if one is offered by the drug company) or single-patient compassionate use is possible for some people. But going through all the steps needed to get single-patient compassionate use of an unapproved drug can be frustrating and take a lot of time. For instance, drug companies have different policies and processes. And there’s no way to force the drug company to supply the drug. There may be very limited amounts of the drug, and producing extra medicine for people who are not in clinical trials can be costly for the drug company, especially when there’s a chance the drug might never be approved.

Compassionate drug use can also be very confusing. There are several programs that regulate it. Many terms and definitions are used to describe how a patient may get access to an unapproved drug outside of a clinical trial. Drug companies, patient advocacy groups, and the FDA all may use different terms for the same things. The FDA provides definitions for the terms they use in their regulations. But most drug companies use different terms that are unique to their specific compassionate access programs.

Another big problem is cost. Some drug companies will supply the drug for free, but others charge patients. Most insurance companies will not pay for investigational drugs. There may also be other costs, such as the clinic’s cost of giving the drug and monitoring your response, which might not be covered by your health insurance. 

How common is compassionate drug use?
Before a patient or group of patients can get an unapproved new cancer drug outside of a clinical trial, 2 things MUST happen:

• The owner (sometimes referred to as the sponsor – most often a drug company) of the new, unapproved drug must agree to allow the use of their drug outside of a clinical trial.
• The FDA oncology medical officer in charge of overseeing the new drug’s development must approve the use of the drug for that person or group.

What should I ask my doctor about compassionate drug use?                                                        Here are some questions you may want to ask if your doctor is thinking about compassionate drug use at this point in your cancer treatment.

• Are there any approved treatments that I haven’t tried?
• Is there any evidence to support the use of this drug to treat my type of cancer?
• What makes you think this drug could help me?
• In what way do you think this drug is likely to work better than an approved drug?
• What are the known risks and benefits of treatment with this drug?
• Will the drug company give me the drug for free? If not, how is it to be paid for? What other costs will I have to pay to get the drug? Will my insurance cover any costs?
• What will I have to do to get access to this drug?
• How long do you think it will take for me to get access to this drug?

                                                                                                                                                                                

                                                               References

www.canceractionnow.org/living/cat_compassionate_use_and_expanded_access.phpcompassionate.php on September 15, 2013

http://www.cancer.org/treatment/treatmentsandsideeffects/clinicaltrials/compassionate-drug-use                  on September 15, 2013