A Resource for Accessing Rare Disease Therapies Globally

As featured on Patient Worthy: Rare Patient News

Access Opportunities for Rare Disease Patients

At present, there are around 7,000 identified rare diseases, with only 5% having treatments. That leaves more than 95% of rare disease patients with little to no options, especially those in low-resource countries where access can prove even more challenging. Often, even if a commercial therapy is available, patients may not have access due to commercialization constraints in a patient’s home country, differences in reimbursement and pricing systems causing complex reimbursement challenges, and a number of other factors. In fact, there are many countries in the world that new medicines for rare diseases will never be registered, leaving rare disease patients with limited opportunities.

Early Access, Compassionate Use and Named Patient Supply

In the event that a rare disease patient encounters one of these challenges, there are other channels available that can help provide access to the medicine needed. Most countries have pathways that allow patients to access unapproved medications when no other therapies are available on the market. Some of these interventions fall under the umbrella of “early access” and can help address the needs of rare disease patients who suffer from life-threatening conditions. “Named patient supply” and “compassionate use” are two access pathways that fall under this umbrella.

Compassionate use is a pathway that allows a patient with an immediate life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. Named patient supply is another way to provide patients and physicians outside the US access to medicines that are approved somewhere in the world, but not available to them in their own country. Similar to compassionate use, the physician must make the request to their local competent authority to treat an individual patient under their direct responsibility.

Differences Between Clinical Trials and Early Access Programs

Both clinical trials and early access programs (EAPs) provide patient access to investigational medicines, with noteworthy differences.

When a biopharmaceutical company wants to get a new medicine approved, they must conduct clinical trials to prove the safety and efficacy of the treatment prior to widespread use. In turn, select patients are able to obtain the medicine in a controlled setting, and the biopharmaceutical companies are able to collect the corresponding data. To take part in a clinical trial, patients must meet certain eligibility criteria that might include age, gender, type and stage of a disease, previous therapies and other medical conditions. Often patients who are too sick do not qualify based on the strict requirements.
With early access programs the main purpose is to meet the unmet medical needs of patients. While safety and effectiveness data are sometimes captured, these programs are not directly aligned with a medicine’s future commercialization plans.

Process and Requirements for Access Through an EAP

First, the patient’s country of residence must allow for the supply of treatments on a compassionate use or named patient basis with established programs in place. Specifications include:

1. Country-specific legislation that allows for named patient supply
2. Legislation that allows companies to respond to unsolicited medication requests that are not commercially available in that country
3. Legislation that requires doctors to get permission from their local health authority to treat a “named” patient if no alternative therapy is available
4. Importation of the medicine by the doctor on behalf of the named patient

Once the in-country pathway is confirmed, typically a patient must meet the following criteria to ensure eligibility:

1. The patient must have a life-threatening, chronic, or severely debilitating condition
2. No approved therapies can be available to effectively treat the disease or condition
3. The patient cannot be currently enrolled in an ongoing clinical trial
4. The therapy must be going through clinical trials or have started the application process for marketing authorization

If the patient meets the eligibility criteria, the residing physician can submit a request to the patient’s local health authority. For medicines that are not yet licensed anywhere in the world, the product is only available through the biopharmaceutical company. Medicines that are currently licensed in one or more countries can be obtained through an international medicine wholesaler. Once purchased, the physician can supply the medicine to the patient in compliance with local regulations. Unlicensed medications provided by the pharmaceutical company may be supplied either free of charge or at the cost of the medicine.

If you are a patient who needs access to an unlicensed medicine, speak with your doctor to see if early access is a viable option for you.

What We Do at Tanner Pharma Group

For over two decades, Tanner Pharma Group has developed a portfolio of service offerings dedicated to improving global access to medicines. Part of our focus is advocating for the rare disease community and offering solutions that can cross borders. We operate as a liaison between pharmaceutical companies, physicians, patients, and health authorities to expedite access and deliver therapies to those with unmet medical needs.

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