The fight for global access to elsewhere-approved medicines

The current state of global health access

Support for an accessible healthcare agenda is shown by a statement by the World Health Organisation (WHO) for the right to the highest attainable standard of health for all people, and the United Nations (UN) pledging to “Ensure healthy lives and promote well-being at all ages” and “Reduce Inequalities”.

However, it is estimated that at least two billion people in the world still do not have access to essential medicines.

Despite an adamant belief in the right to global healthcare by the WHO and the UN, a clear tightening of global donor government budgets towards medicine development and reimbursement plans still exists.

Why now?

In the particular case of life-debilitating illnesses, the process for approving new medicine and its market availability is extremely complex. With a delay of up to five years in some cases, patients with conditions such as cancer, ALS (Amyotrophic lateral sclerosis) and TTR-FAP (Transthyretin-Related Familial Amyloid Polyneuropathy) often find themselves in difficult and hopeless situations.

Manufacturers will often choose one country to launch a new medicine in, but not all. There are even countries where the manufacturers will never try to get approval. While some patients may see an elsewhere-approved medicine become approved within one or two years in their home countries, others might never have access to the same medicine.

This lack of harmonisation makes it difficult for patients and their doctors to research and find the best course of treatment. Furthermore, there has been no single source of publicly available data that tracks what medicines are approved in which country and the time it took from its initial approval into another country.

However, by collecting and comparing qualitative data in a series of data pillars, the Futureproofing Healthcare Sustainability Index gives a useful overview on the staggering discrepancies that exist between countries within the EU when it comes to access and treatment options. This information is vital if we are to address the shortcomings of EU countries in healthcare provision.

At TheSocialMedwork, we were able to collect the data ourselves by monitoring the latest medicines approvals in various parts of the world. Here are two examples showcasing the inconsistency of the global medicine approval processes:

Ibudilast

In 1989, patients in the United States did not have access to an ALS treatment approved in Japan by the PMDA (Pharmaceuticals and Medical Devices Agency). This treatment, Ibudilast, was granted orphan drug status in 2016 for the treatment of patients affected by ALS. However, to access this treatment, US patients had to make use of early access programmes.

Vyndaqel

Vyndaqel (tafamidis) is used for the treatment of transthyretin amyloidosis in adults with early-stage symptomatic polyneuropathy. It was approved by the EMA in 2011 and gained FDA (US Food and Drug Administration) approval nine years later. However, it is far from being approved in regions of the world where patients are awaiting access this much needed-treatment.

A silver lining

Thanks to an existing regulation, patients with life-debilitating illnesses can access elsewhere-approved medicines on a personal import basis. In Europe, this is known as The Named Patient Regulation.

Each country has its own specific rules surrounding the access and import of innovative medications. There are different programmes and regulations set up for this purpose, including the Named Patient Import, Compassionate Use Programme, and Early Access Programme, to name a few.

These exceptions ensure that everyone, in theory, can benefit from all medicine innovations.

At TheSocialMedwork, we provide access to medicines specifically on the Named Patient Import basis. This means that we work on behalf of the patient in cooperation with their treating doctor.

In order for us to support patients in accessing approved medicines using the Named Patient Import basis, these requirements must be met:

1. The medicine has market approval in another country and is not approved or available in their own country
2. There is no alternative on the market
3. The medicine is for personal use
4. The patient has a prescription from their doctor in their home country
5. The medicine must be dispensed from a pharmacy

Depending on the national legislation of the specific country, additional requirements may apply, such as approval by the Ministry of Health and a limit on the import amount.

What happens next?

It's critical that pharmaceutical companies understand the importance of making life-saving medicines available and accessible to everyone in the world. But the burden needs to be shared by governments and healthcare partners by broadening pricing schemes, growing donations programmes, and working on joint procurement plans. In addition, patients are hindered by the sometimes exorbitant costs of new medicines.

It is important to note that the problem of accessibility and affordability is not only limited to developing countries. The high price of treatments and long delays is also a burden for citizens in the United States and Europe.

In Europe, the soaring prices of medicines caused the European Council to establish conclusions on how to address issues in pharma systems in 2016. Aiming to address the issues of affordability and accessibility, the council proposed several activities such as exchange of information, horizon scanning and joint price negotiations. It adopted accessibility, effectiveness and sustainability as its core values with a long-term goal of facilitating and optimising healthcare access to patients.

In order to achieve global health access, we need to create a new health ecosystem where everyone works together. Patients, carers, healthcare professionals, pharma, health advocates, health technologists and policy makers all have a responsibility to work together to enable good health for all. Data aggregating Initiatives like the Futureproofing Healthcare index are crucial to help us understand what is needed in the access-to-healthcare industry and how we can each contribute to a more fair and just world.