Subscribe Now

By entering these details you are signing up to receive our newsletter.

Patient engagement driving success in Early Access Programmes

Patient group interview with Dr Nicole Gusset, Acting President of SMA Europe

Patient expert Dr Nicole Gusset spoke to us about the value of patient engagement in Early Access Programmes to generate the best outcomes and success for all stakeholders involved in the process.

When Nicole’s daughter, Victoria, was diagnosed with spinal muscular atrophy (SMA) 10 years ago this was her first exposure to rare disease and, like many parents in her situation, Nicole immersed herself in researching the condition. Finding support was also important, however unable to find an organisation in Switzerland, Nicole founded SMA Schweiz, a patient organisation committed to the development of therapies for people with SMA.  From the outset Nicole felt that it is vital for rare disease groups to join forces and collaborate, with SMA Schweiz, Nicole joined the European umbrella organisation for SMA, SMA Europe, who are a patient and research organisation that campaigns to improve the quality of life of people who live with SMA and to bring effective therapies. 

Nicole and Victoria Gusset

Spinal muscular atrophy (SMA is a rare inherited neuromuscular condition affecting the motor nerve cells in the spinal cord causing progressive muscle weakness and loss of movement. For more on SMA please visit SMA Europe

From her daughter’s diagnosis to her role in advocacy and Early Access Programmes, Nicole’s journey and her determination have made her a powerful advocate. Building these experiences into a unique combination of skills has enabled her to both understand and successfully work with pharma and other stakeholders in Early Access Programmes. 

Working with something that is so personal gives Nicole the energy and passion needed to advocate for the SMA community however, Nicole is clear, that evidence-based advocacy is the foundation to bring patient relevant advances to the community. For both Nicole and SMA Europe improving the long-term outcomes for all those living with SMA is the goal.

We need evidence as the basis for our advocacy tools and arguments, and emotions as our engine.

Dr Nicole Gusset

In 2016 the first ever treatment targeting the underlying genetic cause for SMA, was approved and with further treatments now “popping up,” it is an exciting and pivotal time for research – providing much hope for the community. Over the last 10 years Nicole’s transition from a parent advocating for her child, to advocating for the European SMA community has been a natural progression.


Nicole describes herself as a “patient expert”, besides her experience in the disease she has undertaken special training that organisations such as EUPATI and Eurordis provide patients and patients representatives to understand and meaningfully contribute to medicines research and development. Nicole strongly believes the value that patient experts add to Early Access Programmes is vital to their success. Patient experts bring invaluable insights and working together with pharma and other stakeholders this experience can ensure Early Access Programmes are designed with the patient needs at the fore and in turn improve success and outcomes minimising failures.

Nicole’s experience of Early Access Programmes has been diverse. Each programme came with its own set of challenges. Nicole describes her first role in an Early Access Programme as “passive and reactive but an incredible learning experience” where she was able to educate herself on the whole process of Early Access Programmes. 

The next Early Access Programmes, Nicole and her organisation played more active roles, they were gradually able to have more input and to shape the programmes. 

​Reflecting on these experiences, Nicole stressed the importance of involving patient organisations at the earliest stage possible in medicines research and development and building strong working relationships with all parties involved. This can take time but is vital to the partnership. Whilst Nicole understood how important it was from the patient group’s perspective, understanding that pharma also need to be able to entrust patient organisations with confidential and sensitive information helped them to understand how they could work together.

“It is important that there is a continuous and meaningful dialogue between stakeholders.” SMA Europe educated themselves about the process and understood the constraints and legal framework that pharma work within, this really helped them to understand why certain decisions were made and earned the respect of the pharma groups they worked with.

Dr Nicole Gusset

They also challenged pharma groups and disagreed with some decisions. A key for success has been maintaining a continued dialogue, with explanations being provided for why certain decisions had been made and why in certain instances, patient group suggestions were not able to be adopted.

Explaining the rationale behind decisions will help to establish a fruitful working- relationships.

Dr Nicole Gusset

Patient organisations and patient experts are a vital component to pharma in designing these Early Access Programmes as they bring to the table a unique understanding and knowledge not only of life with the disease but of the disease environment. They can advise where the unmet needs are within the patient community and then they can “work together to really assess and translate it into a meaningful and powerful programme.” 

Understanding the impact an Early Access Programme may have on the family will help to ensure programmes are designed with the patient in mind, leading to increased engagement. For example, families already juggling weekly physiotherapy, occupational training and speech therapy can be overwhelmed if an Early Access Programme requires lots of hospital visits on top, particularly if balancing caring and work. 

Likewise, the right engagement can help with maintaining a balance with Early Access Programmes to ensure that these programmes do not hamper clinical development and consequently, broad access. 

Dr Nicole Gusset

The emotional impact of these programmes can be significant on patients and their families, when a family wants to be a part of a programme but are not eligible due to the programme’s criteria, this can cause significant distress. An example Nicole highlights, “from a global perspective is the extreme emotional distress it can cause families when a programme runs in say Europe and not in Asia”. Patient organisations can help to manage expectations in the community. They can also help with other stakeholders to define factors that lead to the inclusion criteria. 

“It is important the patient organisation has a good internal structure in place”. Gaining knowledge of early access and understanding the role you can play in advance means you can hit the ground running. However, if you do not have experience or understand the technical language and processes don’t be afraid to ask questions and if you need to take a step back, then do. 

I think there is a gap in training and educating patient organisations to navigate complex Early Access Programmes. We can really support those communities who have not yet been exposed to the situation. 

​Dr Nicole Gusset

Organisations like Eurordis and EUPATI or government bodies provide training opportunities, but it can be a daunting and complex space and a big commitment. Nicole thinks it would be great to have “more places to go to for information, training and to begin the early access journey.” Creating a network of rare disease patient organisations working in early access would be incredibly beneficial and empowering, allowing shared knowledge and experience on a larger scale benefitting not only patient organisations but all stakeholders. 


For more information about SMA Europe

For more information about Clinigen:

Skip to content