Historically, biopharma companies developed drugs for patients with an illness — but the illness was the primary focus. In recent years, much has changed. As drug development has become more enlightened, understanding the experience of people living with health conditions — their needs, goals, and the practicalities of daily life — has become integral to the process of developing innovative medicines.
To earn trust and deliver on the promise of their novel therapies, listening to and learning from the patient experience to help shape clinical strategies and welcoming the patient voice in commercialization planning processes is now a critical part of how today’s companies must operate.
In this month’s e-conversation, while we will be using the word “patient” to describe people living with a disease, we acknowledge the importance of seeing the whole picture — who people ARE beyond the patients with an illness.
What Has Changed?
First, today’s patients are more empowered. They have access to their own medical records and increasingly sophisticated information via the internet. They have done their homework and show up to doctor’s appointments prepared to engage as an active participant in their healthcare decisions.
Second, they are often well-connected and in regular communicationthrough both informal and formal channels with others who have the same condition. This connection can be through established local or national advocacy groups, social media, or both.
Finally, patients have become increasingly influential in advocating for themselves to ensure medicines get to the market in a timely manner. Integrating an understanding of drug development paired with their lived experience equips patients to be important stakeholders in the regulatory review process. Through its public forums, the FDA invites patients to provide testimony and share their stories about seeking effective treatment for their disease. The FDA has a standing committee — Patient Engagement Collaborative — to ensure the patient experience is part of the regulatory decision-making.
Patient Advocacy within Biopharma Companies
Consequently, the role of Patient Advocacy — representing the voice of the patient — has become both essential and highly visible in companies. It has grown in influence, not just in terms of clinical trial design but throughout the commercialization process. This team provides insights on a range of topics — from disease education efforts to advising on elements of patient support programs to helping ensure access with minimum obstacles.
Biopharma companies ensure informed decision-making by incorporating patient insights throughout the entire process. This may include understanding barriers to participation in clinical trials to appreciating the patient experience when the drug is commercially available. Patients have opinions on the number of doctor visits, the procedures they must endure, and the burdensome paperwork needed to participate in clinical trials. For example, while the difference between two and four blood draws may not look like much on paper, it could significantly limit patient willingness to participate.
Success in drug development minimally requires meeting the clinical endpoints for regulatory approval. With actionable patient insights integrated into trial design and disease education, the definition of success expands to include meeting the patients’ needs as well. This is why we recommend that biopharma companies adopt an integrated Patient Advocacy approach: developing drugs with patients.
Beyond gathering, contextualizing, and sharing insights across the organization, Patient Advocacy serves as a visible and active representative of the company to the patient community. Patient Advocacy seeks authentic and mutually beneficial opportunities to partner with professional societies and patient groups. It’s worth noting, too, that a company must understand that it is making commitments to patients regardless of whether the drug succeeds or fails. Once this trust is built with the patient community, it must be nurtured and supported.
Another responsibility that may reside within Patient Advocacy is close collaboration with Government Affairs. This alliance focuses on understanding the evolving public policy objectives of Patient Advocacy organizations. This insight will enable the biopharma company to identify shared objectives on which to partner, to increase the likelihood that government policies will be enacted to benefit patients.
Individuals within the Patient Advocacy function require exceptional communication and relationship-building skills, as well as the ability to work cross-functionally. They are a conduit to connect patients to the Company and elevate the patient voice in both internal and external forums. They must appreciate the intricacies of drug development and regulatory requirements that will determine whether the therapy makes it to the commercial market. Overall, whoever assumes this role must be able to build durable relationships, give and get respect, and cultivate trust within the community.
When Is the Right Time?
Depending on the disease and particular needs of each community, we recommend establishing the Patient Advocacy function early in clinical development. This can be done in a variety of ways. The following should be considered:
- Does it make sense to outsource to a trusted external consultant or firm that specializes in this type of capability until proof of concept is achieved?
- Are there any individuals currently within the organization that can take on this responsibility if the current scope of activities doesn’t warrant a full-time hire?
- Have the appropriate teams been established to ensure insights from the function can be applied and have an impact?
- Are the right resources in place to fund the necessary activities for the function to be successful?
- Is the organization ready to commit to establishing and partnering with external Patient Advocacy groups? The last thing a company wants to do is commit and then pull back without providing context.
Eventually, building the capability in-house and hiring full-time headcount will be necessary as the volume of work and number of activities grows. This inflection point typically occurs post-proof of concept and before embarking on a pivotal trial. To cultivate patient-centric development and commercialization planning into the culture of a Company, it is ideal that the individual is a full-time employee and is hired at a time when they can meaningfully impact commercialization.
Conclusion
For companies to achieve a truly patient-centric vision, Patient Advocacy must be an integral part of the ongoing dialogue and decision-making in a drug development program. There need to be opportunities and venues for Patient Advocacy to share insights — companies need to be prepared to incorporate this insight into their planning efforts. It’s more than just having a seat at the table.
Ultimately, success goes beyond launching a product — it’s about developing novel therapies that not only treat the illness but improve the well-being of patients for generations to come. Patient Advocacy is an integral part of enabling companies to deliver on that vision.