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Zambon Pharma

The Importance of Patient Advocacy

EBR sits down with Jennifer McGrain, Coordinator of Patient Advocacy at Zambon, to discuss the critical role of the patient advocate in understanding and addressing rare diseases


EBR: What is the patient advocacy function within a pharmaceutical company and why is it important?

Jennifer McGrain: The role of a patient advocate is to serve as a liaison between the patient community and a pharmaceutical company – it is an essential part of incorporating the patient voice into company programmes as both parties look to secure optimal outcomes for the end-users. The role addresses three key goals:
• Build relationships with the patient community to uncover their needs and concerns, better understand the unmet needs for their condition, and determine how we can help improve their experience
• Raise awareness of the disease and treatments in development with policymakers in an effort to improve patient access to therapies upon approval
• Educate the patient community about new research opportunities for their disease to ensure that they are informed

Why is patient advocacy of particularly high importance in rare diseases compared to more common diseases?

An estimated 300 million people worldwide have one or more of the 6,000 identified rare diseases (1). The unmet need in this patient population is high and extremely urgent as most rare diseases have no approved treatments.
As rare diseases often affect only a handful of people located in a multitude of locations worldwide, many patients have no organised group they can rely upon for resources, emotional support, and advice. While social media has, to some extent, been helpful in connecting patients and their caregivers to others with the same rare conditions, a patient advocate can help unite these populations, giving them both a voice and direction.

Is it common for biotechnology and pharma companies to have dedicated patient advocacy personnel?

Among participants surveyed by BioNJ, 76% of biopharmaceutical companies had patient advocacy programmes, or planned to implement them, yet only 19% had personnel dedicated to patient advocacy. These numbers suggest that although companies place a value on patient advocacy, the resources to staff this function may be limited (2).

What situations might prompt a company in the rare disease space to incorporate the patient advocacy area?

As previously discussed, patient advocacy in rare disease communities is crucial. Working with patients and patient organisations helps the company improve disease understanding, proactively answer relevant clinical questions, and furthers an understanding of important patientcentric outcomes for a rare disease population (3). A key situation that may encourage the enhancement of patient advocacy is in clinical trials. With rare disease patients being a group limited in size, geographically disparate and heterogeneous amongst themselves, identifying patients for participation in clinical trials can be challenging (3). The support of patient advocacy to facilitate engagement can be essential in raising awareness of trials within the patient population, which may lead to faster enrolment.

What would a strong collaboration process with rare disease patient advocacy organisations look like?

A strong collaboration between a patient advocacy organisation and a company should be of mutual benefit. The collaborations should start as early in the drug development process as possible and be consistent.
The relationship should also be meaningful in nature and reflect a true partnership (4). That means that the pharma company should demonstrate a genuinely patientorientated approach and that patient advocates should ensure regular and frank communication with the most important stakeholders – the patients.

In what areas should a company directly engage with patients and caregivers? Why are these important?

Companies should work with patients and caregivers in the areas of clinical trial design, patient-reported outcomes for trials, reviews of patient-facing resource materials, product packaging designs, comprehension of the patient journey and patient support programmes. One approach may be to involve the patient and caregivers, as well as other stakeholders involved in disease management, in an advisory capacity. An advisory board can provide meaningful input throughout all the stages of drug development and through regulatory approvals. These activities are important because they provide us with first-hand insight into what really matters to those affected by the disease.

Are there regulatory and compliance standards to be adhered to?

There are organisations that set regulatory and compliance standards for patient engagement. In Europe, the organisation is called the European Federation of Pharmaceutical Industries. A similar group in the US is called Pharmaceutical Research and Manufacturers of America. As Zambon is Italy-based, we also adhere to the standards set by Farmindustria.
These organisations provide our industry with guidance around ethical standards and require total transparency in partnerships with patient organisations.

What background is needed for this role?

Ideally, an individual that is working in a patient advocacy role should have experience working with patients.



1. Nguengang Wakap S et al, Estimating cumulative point prevalence of rare diseases: Analysis of the Orphanet database, Eur J Hum Genet 28(2): pp165-73, 2020

2. Visit:

3. Forsythe LP et al, A systematic review of approaches for engaging patients for research on rare diseases, J Gen Intern Med 29 Suppl
3(Suppl 3): s788-800, 2014

4. Bloom D et al, The rules of engagement: CTTI recommendations for successful collaborations between sponsors and patient groups around clinical trials, Ther Innov Regul Sci 52(2): pp206-13, 2018


This article is taken from European Biopharmaceutical Review October 2020, pages 50-52. © Samedan Ltd


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