Can’t give it a label?

The pharma landscape is constantly shifting.

Regulations vary significantly, depending on each country’s economic and medical standards, and each regulatory body has a different process for approving a medicine.

As a result, medicines approved in some countries are not approved in others. However, there is a cross-border solution, and unlicensed medicines (ULMs) could go some way to bridging the healthcare gap for millions of patients around the world.

According to NHS England, an off-label or unlicensed medicine is one that is not approved for treatment of a patient’s condition.

So why might a doctor recommend a ULM? The medical community understand there is a long list of reasons why a medicine could be unlicensed and yet still be a good care option for some patients.

They treat with ULMs because of a critical unmet medical need in their patients and they assess that potential benefits of the treatment are greater than any risks. But there is still more to do to make the practice more accepted and commonplace.

Because ULMs lack regulatory approval, the first assumption is that they’re illegal and the two terms are often conflated.

Although the promotion of ULMs themselves is not permitted, a general lack of awareness results in the public consensus that ‘unlicensed’ equates to off-limits.

Appropriate and common usage of ULMs includes when the medicine is already available and on the market in one country, and a doctor accesses it for the patient in another, even though it’s unlicensed in the patient’s jurisdiction.

ULMs play an important role in the global landscape. Clinical trials can sometimes take ten to 15 years to complete but existing ULMs can provide an alternative option for an unmet need while a patient is waiting for a medicine to become approved in the patient’s market or to be selected for a clinical trial.

Equally, sometimes a patient group is too small for pharmaceutical companies to justify a clinical trial. Clinical trials can cost millions to execute, and pharmaceutical companies have to balance investment against the need to continue to supply existing therapies.

Even beyond the clinical trial expense, full launch of a product is a significant investment.

There are more than 7,000 rare diseases and over 3.5 million people in the UK live with one of these rare conditions.

While these conditions only affect less than one in 2,000 people, one in 17 people will be affected by a rare disease at some point in their lives and there is an alarming awareness gap between what treatments could be used and what patients think is available to them.

We surveyed a group of members of the rare disease community – including patients and patient advocates – and two-thirds of respondents expressed a willingness to receive medicines through their country’s healthcare systems prior to regulatory approval.

A third of the same patient group, however, was not aware of the potential to do so through managed access programmes (MAPs). This points to a significant knowledge gap between the care patients want and need and the care they’re aware of.

This knowledge gap isn’t the fault of any of the interested parties. The doctors are bound by the restrictions of their market and don’t necessarily have awareness of products that could be made available.

At the same time, pharmaceutical companies and insurance providers have limited direct access to patients, and try to support fair access while not compromising their ability to get products to market to ultimately reach the largest group of patients who can benefit.

And regulators can only regulate within the confines of their knowledge base and what information is available.

All of this results in a disconnect between patients with rare diseases and the medicines that may be most suitable to them.

Increased patient access to ULMs and MAPs will help more patients facing an unmet need, not only in the UK, but across the globe.

For ULMs to reach the deserving patient population, it’s critical to have easy to access to this information around ULMs and MAPs.

This means from diagnosis to treatment, patients have access to information from experts around regulations, and are provided with the access to ULMs and MAPs that are available and suited to them.

Equal support must be applied to all patient populations, irrespective of size. Pharmaceutical companies also need a way to reach patients in cases where ULMs could be life-saving and provide patients access to clinical trials their medical provider may not otherwise know about.

Providing access to ULMs also helps to reduce the impact of the ‘postcode lottery’ that many patients experience.

Some institutes and communities are able to provide doctors with the budget and resource to make medication available within their local Trust’s patient community. The ownership cannot fall on the doctors alone, as there should be a stronger link between researchers and those developing new therapies, and patient groups.

Licensing medicines is critically important to ensure that the broadest group of patients can access the medicines that they need.

Regulatory standards are essential controls to ensure patient safety. That said, ULMs can be vital for patient populations that don’t often see the spotlight.

They can offer support across the spectrum from regulating a patient’s condition all the way to accessing an organ transplant.

The key to unlocking these underused resources is to remove the taboo around these medicines, raise awareness about the sensible, appropriate use of them and give providers a viable route to alternative patient-centric care when it’s needed most.

Suzanne Aitken is SVP of Managed Access at Clinigen.
Go to clinigengroup.com