By John Lagus, founder of Bluestem Pharma Consulting, LLC

In 1979, the actor Steve Martin starred in a movie called “The Jerk”. A scene from the movie had Martin’s character running down the road yelling “The new phone book is here! The new phone book is here!” because he was so excited to see his name in print. 

My “new phone book is here!” moment is when the European Federation of Pharmaceutical Industries and Associations (EFPIA) releases its annual Patients W.A.I.T. Indicator Survey where W.A.I.T. stands for Waiting to Access Innovative Therapies. EFPIA has published these data since 2004, and it is the largest European study of medicine availability and patient access wait time. 

The implication of these data is important for stakeholders including pharma companies, physicians, patients and patient advocates to understand. The data are presented visually helping to quickly give a big-picture understanding of the access challenge faced by rare disease patients in Europe. 

Intro to John Lagus

Greetings to rare disease advocates all over the world. My name is John Lagus and Rare Revolution has asked me to write a regular Industry Insights column. During my career in the pharmaceutical industry, I have been involved in the development and commercialisation of medicines. However, my unique expertise is in early access to medicines, which is sometimes referred to as expanded access, managed access, compassionate use or named patient supply. 

I’ve seen firsthand what rare disease patients and their families endure trying to get a diagnosis, let alone a treatment. It is particularly emotional to engage with a parent who knows there is an approved medicine available somewhere in the world that could treat their child, but that medicine is not available in the country where the patient lives.

It’s become a life mission for me to connect physicians and patients with pharmaceutical companies who can supply their medicines to treat the patient’s rare diseases. This and future editions of my column will focus on various features of early access.

2023 EFPIA Patient W.A.I.T Indicator

Information about serious or life-threatening diseases and treatments available, whether already commercialised somewhere or going through the clinical trial process, has never been more available than today. People may be desperately seeking information and access to medicine for themselves or for a loved one.

For countries in the European Union, all orphan medicines being developed for rare diseases must go through the centralised process managed by the European Medicines Agency (EMA). This means there is one regulatory approval for all the countries in the EU. Other European countries such as the UK go through separate reviews by their local regulatory body (for example, the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK).

The EFPIA Patient W.A.I.T. Indicator 2023 Survey (published in June 2024) considers the availability of medicines approved by European regulators over a four-year period (2019 to 2022). For the purpose of this article, I am going to focus on orphan medicines (those being developed to treat rare diseases) which begins on slide 25 of the current report. The analysis calculates how many of the medicines approved in that four-year period are available in each European country as of 5 January 2024, as well as the number of days it took for those medicines to become available. 

Figure 1 below highlights the rate of availability measured by the number of orphan medicines available to patients in each European country. For most countries, this means the medicine has been added to the public reimbursement list so the cost of the medicine will be covered if prescribed by the patient’s physician. 

Figure 1 

Orphan rate of availability (2019-2022)

From 2019 to 2022, 63 orphan medicines were approved by the EMA and other European regulators. Each European country is listed across the bottom of the graph from left to right. The green bars show for each country the number of orphan medicines available during those four (4) years. The data show the country a rare disease patient lives in significantly impacts their ability to access new medicines.

Rare disease patients in Germany had the best access among all European countries with 56 of the 63 orphan medicines (89%) available for patient treatment. In Spain 32 of the 63 medicines (51%) were available. Patients in England had access to 28 of the 63 medicines (44%) over the same period. On the right side of the figure, 7 of the 63 medicines (11%) approved by EMA were available in smaller EU countries like Estonia and Latvia. 

Figure 2 below presents the average time to availability of orphan medicines for patients in European countries. For most countries the time is the number of days after approval when the medicine is added to the public reimbursement list. 

Figure 2

Orphan time to availability (2019-2022)

As in Figure 1, each European country is listed across the bottom of the graph from left to right. For the 56 orphan medicines available in Germany, it took on average 96 days from the time of approval for the medicine to be added to the public reimbursement list. Similarly for the 32 orphan medicines available in Spain, it took on average 704 days. For the 28 orphan medicines available in England, it took on average 394 days. For Estonia and Latvia, the time to availability was 622 and 700 days respectively. The data show the time for a new orphan medicine to be available following regulatory approval in most countries in Europe is a year to two years. 

Despite a centralised EMA approval (this means on a single date the medicine is approved across all the countries in the EU), a relatively small proportion of the medicines are available via public reimbursement, and it takes a long time before the medicine can be accessed by patients in the country they live.

Early access programmes fill the access gap

Luckily, most countries in the world have laws in place allowing patients with serious or life-threatening conditions to access medicines even if they are not available officially in that country. For instance, the patient may not be able to join a clinical trial, or they simply live in a country where this new medicine will not be launched. 

Within Europe and the UK, the legislation and process to get access to an “unlicensed medicine” varies from country to country. One consistent requirement is that a physician must initiate a request on behalf of their patient. The request often needs to be approved by the pharmaceutical company, as well as by the health authority in the country where the patient lives.

In France, “Early Access Authorisation” or “Compassionate Use” describes two different available access paths. In Germany, a medicine can be made available via a “Compassionate Use Program” if supplied before the first global approval of the medicine. 

In the UK, medicines were historically accessed on a patient-by-patient basis using named patient supply. This means a physician seeks to import a medicine not available in the UK for a specific “named patient”. If a medicine is needed for a group of patients, the UK also has a mechanism called the Early Access to Medicines Scheme (EAMS). This is a formal programme set up by the pharmaceutical company with the MHRA prior to regulatory approval. 

Understanding that they are developing new medicines that can change the lives of patients, pharmaceutical companies often set up early access programmes to help as many patients as possible. Because an early access programme may have inclusion and exclusion criteria similar to a clinical trial, all patients with a specific disease may not qualify. 

Final thoughts

It is heartbreaking when patients cannot get access to new medicines for their serious or life-threatening disease. Especially when help is available in other parts of the world, but not in theirs. Today, many medicines developed and commercialised in Europe have limited and delayed availability through normal commercial pathways depending on the country in which the patient lives. 

Given this reality, it is important that all stakeholders know about early access programmes and their complexities because in many parts of the world these access programmes may be the only way for patients to get access to new medicines. In addition to the patient, the key stakeholders needed to successfully facilitate this type of access include the country’s health authority, the pharmaceutical company, and ultimately the patient’s physician. All must be involved to ensure that the patient has the best chance of successful treatment with the new medication.

About John Lagus

John is the founder of Bluestem Pharma Consulting, LLC, helping patients around the world access medicines to treat serious or life-threatening diseases. John works with pharma and biotech companies to better understand and implement best practices for global early access and expanded access programmes that meet a patient’s unique medical needs.

Early in his career, John was introduced to the orphan drug and rare disease space, when he joined Orphan Medical. Early access strategies were utilised to supply several of the company’s FDA-approved medicines to patients outside the US.

Over the years, John has had extensive experience with the design, set-up and implementation of more than 200 global early access programmes. John also provides help to patient groups and those they serve. Supporting the rare disease community continues to be his passion.

Contact John at john@bluestempc.com, visit his website bluestempc.com, or connect with him on LinkedIn.

---

Editor’s note: Sponsored Industry Insights columns are available. Please contact hello@rarerevolutionmagazine.com