“Compassionate drugs“, also called “named patient drugs” or “early access drugs”.
Compassionate drugs can be one of three cases:
Compassionate drugs (1) –
Can be an experimental, investigational drug that is at the stage of evaluating its efficacy and side effects in humans in an orderly setting of clinical studies.
Compassionate drugs (2) –
Can be a drug that is not available to people in clinical studies because all the studies that have tested it in humans have ended and their results are submitted to the health authorities for approval of the use of the drug in humans.
Compassionate drugs (3) –
May be a drug available through a “Compassionate Program” which is a program initiated by the drug company for a drug that has been approved for use by the health authorities, like the FDA, but is not covered under the patient’s insurance.
In cases (1) and (2) –
The drug supply should be approved by the pharmaceutical company. There is no law that requires pharmaceutical companies to agree to supply the drug and each company has its own considerations as to whether or not to approve the drug supply. Therefore, the likelihood that a drug company approves is 50%. In such a case where a pharmaceutical company approves the drug, it does so in its discretion to show compassion to the patient and hence the name “compassionate drug”. The origin of the name does not in any way imply a poor condition of the patient. Absolutely not
In cases (1) and (2), it is about administering drugs that have not yet been approved. You have to remember that. On the other hand, these are drugs that are at the forefront of science, some of which will become the standard drugs in a few years after they are approved and entered into international guidelines. In addition, there is also the matter of the patient’s right to try … and we will get to this elementary right later.
In case (3) –
It is an approved drug but is not covered and basically anyone who does not have private health insurance will usually have difficulty purchasing it. The cost of cancer drugs, for example, is thousands of dollars per month for one treatment. In this case, the pharmaceutical company can initiate a “compassionate program” in which it delivers the drug at its expense until it enters coverage by insurance. The pharmaceutical company can limit its compassionate program to a number of patients and not beyond and also instruct the doctors that patients who have private health insurance, it is better to send them to purchase the drug at the expense of the insurance company and utilize the drug quota for those who do not have private insurance coverage.
Some examples of compassionate drugs for each of the three cases
Cases (1) and (2) –
We have obtained a drug called Molibresib for a patient with an aggressive type of cancer called midline carcinoma. Molibersib is still in clinical trials and is not approved in any country in the world. On the other hand, based on the literature and data published from the studies, it emerged that this drug may make a huge contribution to the patient. We contacted the pharmaceutical company and they agreed to deliver the drug and the patient actually received it.
Case (3) –
A drug called Alpelisib, is not reimbursed in the national health basket in Israel but is approved for breast cancer patients found to have a mutation in the PIK3CA biological component. The pharmaceutical company has initiated a compassionate program to deliver this drug to the same patients as required by the treating oncologist and after verifying that the patient does meet the conditions for receiving the drug.
Who compassionate drugs are for?
Here we will focus the discussion on cases (1) and (2) because in case (3) these are approved drugs but not covered.
So compassionate drugs are for:
- Patients with life-threatening illness, such as cancer, nowadays corona and more
- Patients who have exhausted all the standard treatments and have nothing more to offer them
- Patients who do not have available clinical studies because, for example, are in a distant country and the patient has difficulty in flying or as stated, there are no open studies that patients can participate in
Notice that all of these conditions do not relate to the patient’s preference and the question is what about the patient’s right to try any drug even if it has not yet been approved? After all, these are patients with life-threatening illness. Very recently, the United States has promoted a set of laws on the subject, entitled “Right-to-try laws“.
Phase I trial is a study with a drug that is first given to a specific patient population. Phase I trial can be with an approved drug for example for lung cancer and in the trial setting it is given for the first time to pancreatic cancer patients. In this case, the side effects of the drug and their effect on patients are already known so that both doctors and patients feel more comfortable with them. In addition, phase I trial could be with a drug that has not been approved at all to treat any disease and the FDA is referring exactly to this case and hence the Right-to-try laws.
Compassionate drug is an advanced and innovative drug that allows patients to raise their chances beyond any other treatment or drug.