Understanding Named Patient Programs: How They Work, Benefits, and Challenges

One of the most common questions we get from patients, family members, caregivers, and doctors is “How are you able to provide access to medicines that aren’t approved in my home country?”

Put simply, we take an existing regulation - the Named Patient Import regulation - and make it easy for patients and doctors to use. In this article, we'll review everything you need to know about Named Patient Import and our role in it.

What is the Named Patient Import regulation?

Access to potentially life-saving medications that are not yet available through regular channels can be a daunting challenge for patients with serious illnesses. This is where Named Patient Import comes into play, offering a vital lifeline.

Named Patient Import, also known as the Named Patient Program (NPP), Named Patient Compassionate Use, Named Patient Supply, Expanded Access Program (EAP), or Individual Patient Program (IPP), is crucial in providing early access to unapproved medicines.

The regulation is in place in most countries in the world and allows a patient (the Named Patient) with a life-threatening, long-lasting or seriously debilitating illness to access, purchase, and import medicines that are not approved or available locally, but could potentially save their life or improve its quality.

What is the difference between Compassionate Use and Named Patient Programs?

The terms Compassionate Use and Named Patient Program (NPP) are often used interchangeably. However, despite their similarities, these two types of access programs are not the same.

• Similarities

Both programs help patients with serious conditions who have an urgent medical need. The programs provide access to investigational or unapproved medicines when no other treatments are available. Both programs can apply to an individual, as well as a small group of patients.

• Differences

Compassionate Use programs are overseen at a national or international level - e.g. by health authorities like the FDA or EMA. They require formal approval by the regulating body. Doctors must apply for approval and get an ethical review before the patient(s) in question can get access to the medicine.

NPPs, on the other hand, are regulated on a national basis. For this reason, their requirements may vary depending on the country. However, NPPs are overall a quicker and more flexible way to access medicines in regions where they are not yet approved. No ethical review or formal application is required. A doctor can directly obtain a medicine from its manufacturer or a relevant supplier on behalf of a specific patient (and with the support of a qualified pharmacy).

Another difference between Compassionate Use and Named Patient Import is that the latter only applies if a medicine is approved somewhere in the world. You cannot access investigational medicines via Named Patient Import, while you may be able to via Compassionate Use.

Who regulates Named Patient Programs?

NPPs operate under a variety of international regulations. In the European Union, they fall under the remit of the European Medicines Agency (EMA) and respective national authorities. The United States has a similar program known as Expanded Access, regulated by the Food and Drug Administration (FDA).

However, although officially NPPs fall within the domain of healthcare agencies, they do not require a formal application process or ethical review, unlike Compassionate Use programs.

Every country has their own specific rules and regulations surrounding the access and import of innovative medications.

Who qualifies for the Named Patient Program?

Despite some country-specific variations in Named Patient Import regulations, the eligibility criteria are the same everywhere.

An individual patient can make use of the Named Patient Program if:

• The medicine they need has market approval in another country and is not approved or available in their own country;
• There is no alternative on the local market;
• The medicine is for personal use;
• The patient has a prescription from their doctor in their home country;
• The medicine is dispensed by a pharmacy.

What documents will you need?

Some documents are essential if you want to access a medicine via Named Patient Import. For example, a prescription from your treating doctor is always required.

If you're trying to import a medicine that is approved in your country but not available (e.g. due to a supply shortage), then you may also need a letter from your doctor that states this situation.

Depending on your country, additional documents, such as an import license, may be needed.

If you're interested in the requirements regarding a specific country or medicine, get in touch with our team of Medicine Access experts. If you order a medicine through us, we'll make sure all the local requirements are met.

How do you find and import the medicine you need?

The next step after you have the documents you need is to find the medicine. You or your doctor cannot directly reach out to the medicine manufacturer to request the medicine. You will need to find a pharmacy which either has an import license or is willing to apply for a waiver. The pharmacy itself will then source the medicine on your behalf - either from the manufacturer or from a pharmaceutical wholesaler which has the medicine available.

Finding a novel medicine can be a tedious and time-consuming process. At Everyone.org, we're specialized in making it easy for you and your doctor. If you order the medicine you need via our platform, we will source the medicine on your behalf and arrange to have it delivered safely to one of our partner pharmacies (for pick up) or a pharmacy in your area.

How does Everyone.org support you with the Named Patient Program?

We understand that the process of importing a medicine you urgently need can be difficult and overwhelming on your own. That's why we've specialized in supporting you and your doctor with the process of Named Patient Import.

If you request the medicine you need via our platform, our team of Medicine Access experts will guide you through the necessary documentation. We'll find the supplier with the best price and arrange for the safe and secure shipment of your medicine to a pharmacy, where you can pick it up. In some cases, the pharmacy may be able to ship it to your address or a hospital's address directly. If that's the case, we'll inform you.

In short, Everyone.org oversees the entire process. We manage every aspect -- from sourcing to shipping, customs and delivery - so you and your doctor can focus on your treatment. From beginning to end, we keep in close touch with you, so you know what to expect .

Find out more about how we work.

Benefits of Named Patient Programs

Named Patient Programs are an important tool for both patients and healthcare providers. They provide an opportunity to bridge the gap between development and approval. As well as the gap between approval and availability. For time-sensitive conditions, this gap of months or years can be critical. Here's a list of the main benefits of NPPs:

• Access to unapproved treatments: Named Patient Compassionate Use allows patients with serious conditions early access to potentially life-saving medicines not yet approved in their country.
• Flexibility in treatment options: NPPs allow healthcare providers to explore additional treatment options for patients who have exhausted all other approved therapies.
• Faster treatment: Through NPPs, patients and doctors can start a treatment faster than they would be able to if waiting for formal approval or local availability. For some conditions, this opportunity can be life-saving.
• Data collection for pharma companies: Individual Patient Programs enable pharmaceutical companies to gather real-world data on medicine efficacy and safety, aiding future regulatory submissions.

Challenges and considerations related to Named Patient Import

While there are a lot of benefits associated with NPPs, there are also some challenges, including:

• Regulatory complexity: Navigating different regulatory requirements across countries can be challenging and time-consuming. Finding the right information may be more challenging in some countries than others.
• Limited data on safety and efficacy: Since NPPs involve locally unapproved medicines, there may be limited information on their long-term safety and efficacy, posing potential risks to patients. That's why the decision to access a medicine via a Named Patient Program always has to be taken in close cooperation with the treating doctor.
• High costs: The cost of buying and importing medicines from abroad can be high, especially if the medicine is only approved in the USA, where medicine costs are the highest in the world. This can make access to specific medicines unaffordable for some patients. To do our part in alleviating the problem, we created the Everyone.org Foundation. 
• Access inequality: Availability of NPPs may be inconsistent, leading to disparities in access to treatments based on geographic location and healthcare infrastructure.

Your health is the end goal

As with anything else, there are pros and cons of Named Patient Programs. However, at the end of the day, they exist to support you and your doctor in arriving at the best health outcome. In many cases, a locally available treatment alternative may also be applicable. But for the patients who have run out of local options, or for those with a rare disease stuck in the medicine approval "waiting room", it's good to know there's more you can do.

If you and your doctor decide to explore NPPs, our team at Everyone.org is here to help you. We aim to offer customised support to each patient and treating doctor. This way, you don’t have to worry about how you will receive the medicine you need. At the end of the day, our goal is for everybody, no matter who they are or where they live, to have equal, fast and fair access to the latest and best healthcare.

Ready to start? Search for the medicine you need on our platform.

Resources:

1. Questions and answers on the compassionate use of medicines in the European Union [PDF], European Medical Agency, January 2010.
2. Iudicello A., Alberghini L., Benini G. and Mosconi, P. Expanded Access Programme: looking for a common definition. Trials, 2016;17:21, 2016/01/12.
3. Glossary of Pre-Approval Access Terminology [PDF]. Pre-approval Access Patient Advocacy Summit, Janssen, October 2018.