The less frequent a medical condition is, the less likely it is to be covered and treated fully by public healthcare services. Even with a more common disease, the newest treatments and drugs are only sometimes covered or available. So, what can be done? Researcher Noa Guzner reviews some alternative methods to procure novel therapies and medications.
Orphan diseases are medical situations that affect less than 200,000 people worldwide. In addition to the complexity of treating a disease with a need for more data, developing new medications for these diseases is highly problematic. Because only a handful of people worldwide are affected by orphan diseases, conducting clinical trials to prove a new treatment’s effectiveness is challenging. Often enough, drug companies find no economic motivation to perform such trials. Even when a drug is developed, sometimes it is not included in the health services basket, thus making the medication very expensive for the patients. Some patients might encounter great difficulties with procuring the treatment they need, even when there is a treatment that has been proven effective.
It is essential to understand how to access alternative means to novel treatments. These means are relevant for patients with common and orphan diseases alike.
First, one must know if a treatment has been approved by regulatory authorities such as the American FDA and European EMA. A series of pre-clinical and clinical trials is required to approve an indication for a drug.
How to check if a drug is included in the health services basket or covered by private insurance?
The first step would be looking for the drug on the Israeli health ministry website to determine if it is included in the health services basket and for what diseases it is indicated. If you have private health insurance, you can contact your provider and check if applying the existing policy to purchase the drug is possible. Usually, there are three criteria for private insurance coverage of a drug:
- The treatment is not part of the health services basket.
- Regulatory authorities indicate the treatment for said disease.
- The treatment is approved for said diseases in one of several countries recognized by the health ministry.
How can I apply for “Expanded Access” or “Compassionate use” programs?
Even if both the options described above do not apply to you, do not give up! Your physician can contact the company manufacturing the needed drug and ask to include you in an “Expanded Access” or “compassionate use” program.
Compassionate use programs often include novel treatments yet to be approved by regulatory authorities. Receiving treatments means you will receive a drug that is not yet indicated for your disease and is intended for patients with no other treatment options or clinical trials. Different pharmaceutical companies have different policies regarding compassion programs. Some will give the drug for free, and some will require a fee.
Another option is filling out a G29 form (Tofest 29 Gimel) by your physician. This form is used for requesting a drug that is not part of the Israeli drug index or a drug included in the index but indicated for a different disease. After receiving approval for this form from the Israeli ministry of health, you must import the drug and have it delivered to Israel through a designated pharmacy specializing in drug importing.
How can I participate in a clinical trial?
The last option is to participate in a clinical trial. You can search for relevant clinical trials by using designated databases. Each trial’s inclusion and exclusion criteria include demographics, other illnesses, and previous treatments, which will determine if you can participate.
The location of the trial is also important. Clinical trials are performed worldwide, and sometimes, to participate, you must move to the country where the trial takes place.
In summary, when needing treatments and drugs not part of the health services basket, checking if you can receive the treatment using an alternative route is essential. Even if the chances are not high, it is sometimes the only way to access novel drugs and treatments, especially for patients with rare conditions.
