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Patient engagement in early access

Patient engagement in early access – making it real

The healthcare environment has seen a shift in recent years towards increased patient involvement in the development of therapies. Patient engagement has been operationalised in a wide range of activities, including convening expert panels of patients, industry sponsored anthropological immersive experiences, and co-development initiatives. Perhaps no company has been untouched by calls to include the patient voice in their development of medicines, and the field continues to evolve in how best to meet those demands. This evolution is critical also to early access, but how far has this been achieved when it comes to EAPs? At the moment, it would seem to be a mixed bag. Read on to discover some analysis of the current situation and some real-life experiences from those who have been part of it.

Many pharmaceutical companies are striving towards increased patient centricity and building this into processes across teams. For some companies, there might not be the structures or know-how to compliantly engage with the patient community around the topic of Early Access Programme (EAP) development, design and decision-making. ​

Geographical nuances can also play a part and create perceived or actual complications from a regulatory standpoint when it comes to engagement. Jodi Wolff, head of global patient advocacy at  Santhera Pharmaceuticals feels that:

The companies who do this well are the ones who embrace a certain level of authenticity and transparency with the patient advocacy groups in their field and incorporate a sense of partnership with patients across internal programmes. Companies who have a strong culture of developing medicines with and not for patients tend to have the motivation to work within the existing compliance structures to conduct early access engagement within the appropriate boundaries and guardrails.

Jodi Wolff, Head of Global Patient Advocacy at
​Santhera Pharmaceuticals
Jodi Wolff

Another challenge that comes up in early access is the significant number of parameters that feed into any one early access request and its assessment. For example, there are factors feeding into the question of “who can get access?” which are separate from the company’s decision on the boundaries of the access it will allow. For example, the views of the competent authority who regulate importation of medicines can have a bearing, as well as the clinical judgement of an individual treating physician. Clarity on the opinions and assessments of a wide range of stakeholders and how those impact early access is essential for patient engagement. If this, and some of the other complex nuances of early access are mutually understood, this lays the groundwork for a constructive discussion that can lead to a better outcome on all sides.

The DMD patient community has been clear that they want the option to access therapies as early in the development process as possible, as DMD is a life-threatening and progressive condition.

Jodi Wolff, Head of Global Patient Advocacy at Santhera Pharmaceuticals

​Jodi explains more about how Santhera approached the decision-making for their EAP in Duchenne muscular dystrophy (DMD), both before the EAP was live, and while it was being implemented, to work compliantly with the patient community to react to unexpected questions or situations that arose.

“The DMD patient community has been clear that they want the option to access therapies as early in the development process as possible, as DMD is a life-threatening and progressive condition. Santhera heard that call and felt compelled to offer access to its investigational product while enrolling a large Phase III clinical trial. Santhera engaged the patient community early to identify the most appropriate inclusion and exclusion criteria for the access programme, which was launched solely in the U.S. as an Expanded Access Program. Once the programme launched, patients who did not meet the inclusion criteria for either the EAP or the clinical trial sought early access to the investigational therapy. By engaging with patient advocacy leaders, Santhera felt they were able to handle such requests in the most compassionate and ethical way possible because of the established relationship and channels of communication that had been set up. These discussions also led to a protocol amendment to the EAP to include a cohort of patients who were faster progressing at a much younger age—a group of patients who weren’t initially considered when developing the program. Multiple patient advocacy groups provided guidance throughout the EAP; however, unfortunately, the company ended the programme after discontinuing the development of the drug.”

One of the advocacy groups involved in the process of developing the EAP was Parent Project Muscular Dystrophy (PPMD). Ryan Fisher, chief advocacy officer comments on the process from their perspective and the value of patient involvement.

PPMD believes the voice of the patient is essential throughout the drug development process.  Santhera’s commitment to thoughtfully engage patients established a mutual sense of trust in the desire to listen to patients, understand their needs and expectations and co-create an EAP that served both patients and company. 

Ryan Fisher, Chief Advocacy Officer, PPMD 

There is probably no such thing as a perfect EAP. Those within pharma and within patient groups who have the hard-won experience of developing an EAP where they have sought to gather input from different stakeholders are acutely aware of that fact. Going into the process with realistic optimism is needed on all sides, and over time trust can be built and progress made.


With that in mind, what is the motivation for a pharmaceutical company or a patient group to spend their time on engagement with each other? Jodi has a view on this from the industry perspective: as Santhera forged the path forwards with their EAP in DMD, the company culture was such that they had a certain humility about what they did know and what they didn’t know, and that served them well.

It’s important to note that early access is only one area in which patient advocates can be engaged and, therefore, the trust and collaboration built in these conversations can spill over into other fruitful partnerships across programmes and initiatives.

  Jodi Wolff, Head of Global Patient Advocacy at Santhera Pharmaceuticals

“As a pharmaceutical company, our motivation setting up an access program is to respond to a high unmet medical need, and to do that successfully patient engagement must be a central component. It’s important to note that early access is only one area in which patient advocates can be engaged and, therefore, the trust and collaboration built in these conversations can spill over into other fruitful partnerships across programmes and initiatives. As companies, it’s prudent to admit that we don’t always know what patients want unless we directly ask them—and we need to be learning from them and incorporating their views at every step of the development process. Even if patients don’t like the constraints that a company works within to develop an EAP, and if companies don’t like the sometimes tough feedback that can be provided, patients can feel heard and included as part of the process. If we are willing to admit what we can and can’t do with regards to early access and explain that honestly to patients, then we can work with patients to develop programmes that best meet the needs of all involved.”

On the other hand, the motivation for patient advocacy groups to engage is clearly to have an opportunity to help shape something that is going to impact upon the community in a positive way and support access, not just now but also in the future. Bettina Ryll, founder of Melanoma Patient Network Europe (MPNE) and EU Cancer Mission Board member, points out how this can be valuable when applied to one of the many balances to be struck within EAP decision-making:

Bettina Ryll

​There is always that balance that needs to be struck between early access and evidence about a treatment. Patients can get caught in the middle. But within this debate, patients can contribute to finding a fair balance when it comes to access and keep the urgency on. But this only works well if patient advocacy groups are involved and understand the different drivers within the system.

Bettina Ryll, Founder of Melanoma Patient Network Europe (MPNE) and EU Cancer Mission Board member

It’s clear that there are benefits to such engagement and getting it right will be the hard-won fruit of finding ways to give it a chance. Any resources that can help to make this a success on all sides will be welcome, and it is great to see more of them appearing as this field evolves. 


​Holly Lumgair, patient advocacy manager at Clinigen, in collaboration with Jodi Wolff, global head of patient advocacy at Santhera Pharmaceuticals, Ryan Fisher chief advocacy officer at PPMD and Bettina Ryll, founder of MPNE.

​If you are a patient group interested to learn more in this area during 2021, look out for this article about the Clinigen Patient Advocate Fellowship in early access.

Holly Lumgair

For more information about Clinigen:

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