Guest Column | September 28, 2021

3 Strategies To Engage With Patient Advocacy Organizations

By Paul Pereira, vice president of patient advocacy, Saniona

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Patient communities, especially for rare diseases, can offer extremely valuable insights to help ensure the success of your clinical development program. Beyond providing feedback to trial design and endpoints, patient advocacy groups are critical for investigator and patient recruitment and often can provide their own scientific assets, such as tissue models. To ensure you capture this input, it’s optimal to have a corporate function with skilled professionals who are well versed in engaging patient communities in meaningful and compliant ways. But how do small companies without a patient advocacy lead take that first step to introduce their company to a new patient community? I’ve found that through gathering insights about the community, developing your company’s commitment to patients, and spending time listening and learning, you can build a successful patient engagement strategy that is aligned with your mission and clinical development goals.

Stakeholder Mapping And Environmental Assessments

Given my military background, I understand the importance of intelligence gathering to ensure a mission’s success. Before cold-calling any patient organization, it is essential to conduct an environmental assessment of the patient communities in the indication in which you are working. If you have the privilege of working in rare disease, reaching out to organizations like the National Organization for Rare Disorders (NORD) or Global Genes can save you time. They have important resources, such as directories of patient advocacy organizations worldwide. Another great resource is social media. Reviewing Facebook, Twitter, YouTube, and other channels will give you a sense of the organizations and how they engage. From there, understanding their core capabilities will help prepare you for a meaningful conversation. Here are four things to take into consideration:

  • Education and Awareness: To what extent has the advocacy organization created its own patient education materials (print, digital, and/or video) addressing the patient journey, diagnosis and screening, treatment and therapies, care centers, and centers of excellence? Does it provide support services for families or mentors for new parents? You may find that, especially in rare disease, there are not many materials or resources available because the disease is not well understood. This could be an important opportunity for collaboration if your company has developed or is interested in developing educational resources. How is the advocacy group raising awareness? Is its website its primary engagement vehicle or is it more likely to use social media? Understanding a group’s strengths and unique capabilities is essential.
  • Advocacy: Advocacy can take many forms – from speaking on behalf of others to ensure adequate access, to supporting disease awareness through proclamations or resolutions through the legislature, to testifying and providing letters and comments on policy initiatives, to supporting a specific piece of legislation. Many rare disease advocates are looking to create rare disease advisory councils throughout the U.S. to give a voice to the rare disease community through state governments. Advocates may have a particular interest in a policy area, and your company may be able to support their efforts by sharing information and facilitating introductions.
  • Research:  An advocacy group’s work in research may be of particular interest to you as it may be conducting its own research in your therapeutic area. An advocacy organization may fund its own independent research efforts or fund others through venture philanthropy. Review the group’s priorities and whether it has a scientific advisory board. Also, consider if it has developed a patient registry and if there are opportunities to share information with members of the registry in a compliant way.
  • Collaborations with Industry: Find out if the organization has worked with the biopharma industry in the past. Is its website sponsored by a particular biotech company? Has it posted information about ongoing clinical trials? Past experiences with other companies can sometimes dictate what an organization will expect from you. Reviewing agendas from past meetings and conferences can be a good source of information on how a group has collaborated with other companies.

Creating Shared Objectives

Before making introductions to patient communities, ask yourself: How will we “show up” with these groups? Think about how you will communicate about your company and its commitment to patients.  

When I interviewed for my position at Saniona, our CEO shared with me that it was critical to the company’s success that we engage patient communities early – not just to support our clinical development programs, but also to help shape who we are as a company. This spoke volumes about Saniona’s commitment to patients. As employee number nine in the U.S., I took this as an important responsibility – I would be the voice and conscience of patients within Saniona. I knew this gave me license to share patient insights throughout the company, not just with clinical development but with finance, HR, manufacturing, legal, and every new hire. Engaging with patient advocates and sharing their stories and insights helps build employee engagement because it serves as a reminder that patients are counting on us. Given this, my approach to meeting with patient advocates was to communicate our commitment to listening and learning about their experiences and sharing them within Saniona to shape everything we do – from developing our medicines to building our company.

Once you have determined how you will communicate about your company and how what you are doing impacts patients, it is essential to clearly establish what patient communities can expect from you. I have found that this is best developed in collaboration with the patient advocacy groups. When I began leading patient advocacy at Saniona, my goal was to develop meaningful relationships with the Prader-Willi syndrome (PWS) and hypothalamic obesity (HO) communities. Both are serious rare diseases in which patients experience dramatic weight gain and insatiable hunger called hyperphagia.

When I started meeting with the International Prader-Willi Syndrome Organization, the Prader-Willi Syndrome Association USA, the Foundation for Prader Willi Research, and The Raymond A. Wood Foundation, I focused on listening and learning from them about the burden of these diseases. In return, rather than sharing with them what to expect from Saniona, we created shared principles together. At Saniona our guiding principles for engaging with patient advocacy groups, in line with our core values, are as follows:

  • Putting People First: We will seek out and include the voice of patients and families in every step of the development process – from designing our trials to delivering our medicines.  And we will do so globally and equitably. 
  • Innovation with Impact: We will engage creatively and collaboratively with patient advocacy groups on initiatives that are meaningful and impactful for Saniona and for patients and families.
  • Integrity Always: We will work ethically and transparently while respecting the privacy, independence, and autonomy of organizations, individuals, and families.

Creating these shared principles has helped us set expectations while building trust with advocates – and they serve as guidelines for Saniona to live up to every day. I recommend having a candid conversation with organizations you want to work with and establishing expectations, setting the stage for future collaborations. Having engaged with these incredible communities over the last 10 months, we have found their insights to be invaluable. These advocates have helped us identify potential principal investigators, informed our clinical trial design, and are poised to assist with raising awareness of our clinical trials.

Early Engagement Opportunities

There are many examples of extraordinary patient engagement campaigns from the biopharma industry. While these can be great tools to expand disease awareness and give back to the community, early engagement should be more about listening and gathering insights that can help with clinical development. At some point, you may decide that a large awareness campaign is what the community needs, but the only way to determine that is through listening and learning. Consider these topics when having early discussions with patient advocates:

  • Ask About Their Journey With The Disease: This open-ended question can teach you a great deal. Every experience from diagnosis through treatment will be different, but through these discussions you will learn their lexicon, how they describe the disease, and how they refer to themselves. This should be how your clinical development team refers to the community. For example, people living with sickle cell disease refer to themselves as “warriors.” For people living with HO who survived a craniopharyngioma, “survivor” is an appropriate term. Keep in mind that no matter the patient community, no one likes to be referred to as a “subject.”     
  • Ask About Their Experiences With Clinical Trials: Many advocates have participated in clinical trials. Ask them what worked well and what to avoid. One mom of a child living with PWS shared with me that her child had a bad experience in a clinical trial. Part of the trial required a fasting blood draw. The nurse gave the child a breakfast menu and said the child could have anything on the menu once the draw was completed. Considering that this child was suffering from hyperphagia and had been fasting, this caused tremendous anxiety and set the expectation that there would be a large meal awaiting after every draw. Another mom reminded me that food is everywhere, and it is important to eliminate unnecessary contact with food during a clinical site visit. For example, when providing directions to the trial site, avoid sending the patient and family through the entrance near the cafeteria, or past vending machines. Patient advocacy groups are also a good source of information about which doctors are interested in clinical trials as many key opinion leaders serve on the boards of advocacy organizations. These insights and suggestions can be invaluable to the clinical development team.
  • Invite The Patient Voice In: Inviting patients, caregivers, and advocates to speak at company meetings is a great way to educate your company about the therapeutic areas you are pursuing. Even in a virtual setting, patient stories can give your clinical development team knowledge of the challenges of living with a disease and inspire them to do more.

Together, these three keys – stakeholder mapping and environmental assessments, creating shared objectives, and early engagement opportunities –  should help you successfully create a road map for how to begin conversations with patient advocacy groups. As your trust and relationships with advocates grow, you will find their insights and support to be extremely valuable. At the same time, you can offer them something meaningful as well: hope.

About The Author:

Paul Pereira is the vice president of patient advocacy at Saniona and is based in Waltham, MA. Paul’s focus is to incorporate patient perspectives to support Saniona’s clinical development programs while building relationships throughout rare disease communities worldwide. He has nearly 20 years of pharmaceutical experience in patient advocacy, public policy, and government affairs. Most recently, Paul was the senior director of patient advocacy for Vertex Pharmaceuticals, where he developed their grassroots patient advocacy program for the cystic fibrosis and sickle cell disease communities. Paul graduated from the University of Massachusetts with a B.A. in economics and J.D. in law. Paul is a licensed attorney and has been a member of the Massachusetts Bar since 2000.