How Sanofi Created A Patient-Informed R&D Organization
By Ed Miseta, Chief Editor, Clinical Leader
Getting the patient voice embedded in drug discovery is a goal of many pharma companies. When Sanofi opted to tackle this challenge, the company knew it wanted the voice of patients to extend across the entire organization. That effort would mean embedding the patient experience, perspectives, and priorities in every step of drug development, from discovery through to regulatory approval.
Speaking on an online seminar presented by Patients As Partners, Victoria DiBiaso, global head of patient informed development & health value translation for Sanofi, shared the company’s Patient Informed Research and Development model and how the company worked with patients and patient communities to develop the model.
“We use the word meaningful because the outcomes we are able to elicit in our research address the true unmet needs of individuals living with those diseases,” says DiBiaso. “Our solutions will ultimately improve their health-related quality of life.”
Get Early Involvement
Today, patients have many different touch points within Sanofi. Individuals from the patient community sit and work with research and development teams within the company to inform decisions, shape strategy, and help identify the right tactics, avenues, and methods of developing new medicines. The journey starts at the benchtops in the discovery research area. When thinking about a new molecule, Sanofi scientists want to know if the new mechanism of action, upon reaching a post-approval setting, will have a side-effect profile that is acceptable to individuals. They also want to know if the method of administration will make it easy for patients to be compliant.
“That partnering transcends right through to clinical trials,” states DiBiaso. “We want to know how we can make our clinical trials more relevant, easier, and less burdensome for patients. Representatives from the patient community can also help us understand how to let people know that a trial exists, what support they need to be able to participate, and if the endpoints we selected are relevant. The outcome measures must truly differentiate our product for patients.”
DiBiaso stresses the importance of engaging with the patient community early enough to understand how to intelligently design the research. Thought must be put into the digital solutions that will be used to understand how they will fit into the study and not overburden patients and investigator sites. Sponsor companies will also need to understand how to take studies to people in their homes more easily while also being conscious of the digital divide that exists in some patients.
“We like to think of this as a co-development model of new medicines,” says DiBiaso. “We started this journey over 10 years ago, and our development programs are now 100% informed by individuals from the patient communities. Our priority preclinical assets also fall under that model and last year alone, that translated into 57 unique programs. Since some programs may have more than one indication, that equated to 67 indications and 156 new clinical trials. We are thankful to the more than 100 patient advocacy groups across 33 countries that collaborated with us and helped inform our decision making.”
DiBiaso notes the company’s co-development model revolves around listening to people who understand what it is like to live with a disease. Those insights are then incorporated into the work the company performs. The insights also help Sanofi prioritize what it believes to be the most patient relevant research programs.
Understand The Risks And Benefits
Sanofi hopes the insights it receives from patients will also help the company better understand the benefits and risks of trials. For example, DiBiaso cites a study Sanofi is running where the treatment presents a temporary side-effect profile that arises during a specific part of the treatment process. In the past, the company may have assumed that patients would not be willing to take the medication because of that side-effect profile. Rather than make that assumption, the company is now collaborating with patients and patient communities to discuss what they think about the side effect.
“We listened to patients and heard that the side effect was not a big concern,” she says. “Moving forward we will continue to inform patients of the benefit/risk of treatments rather than make assumptions around how we think they may interpret it. We will obtain feedback from patients as well as surrogates such as physicians. We are also looking at how we can differentiate our label, our publications, and the data we put out for review. We know individuals want a better understanding of the medications they take, and we are working to ensure the information we put out will answer their questions.”
A decentralized approach to trials is also part of the Sanofi effort to focus on patients. According to DiBiaso, 100% of the company’s Phase 2 and Phase 3 studies will have a remote capability built into them, stressing the importance of taking the research to the patients. Additionally, 100% of Sanofi trials will also have a diversity and inclusion plan in place. Both of those efforts will be incorporated into Sanofi’s KPIs to ensure they are a priority for all employees.
Have A Clear Vision
Moving forward, Sanofi will continue to integrate patient priorities into every trial. The focus now is on making sure these patient efforts become the company’s long-term vision and that the effort involves co-development opportunities with select organizations around the world.
“We have a vision of where we want to go,” adds DiBiaso. “For example, we know that in many indications, the outcome measures available do not adequately address the true burden of the disease. In those diseases we are collaborating with patient organizations to help us identify what the true burden of disease looks like. We then work to determine how to best measure it, validate it, and get it into our research programs. This can take a lot of time and effort, and is why we are committed to building collaborations beyond traditional interactions within R&D.”
As trials move into proof-of-concept, each one will have a validated patient-focused disease-area strategy. That strategy looks at the multiple journeys individuals may take within their healthcare systems. Sanofi hopes to learn about their needs, their expectations, and how to better address considerations around diversity and inclusion. That is followed by a gap analysis that gets built into the evidence generation function and clinical development plans.
A Commitment To Transparency
By the time a Sanofi trial reaches Phase 2, patient input has already been gathered and is helping to establish the development plan. Sanofi understands what the patient communities want and need to know from a scientific perspective, and a scientific exchange takes place with individuals within those advocacy organizations where information about the drug, the study, and the results are shared.
While that sharing of information is not allowed in some countries, Sanofi maintains a commitment to transparency and the sharing of results at the end of a study. KPIs ensure 100% of clinical trials have lay language summaries where legally allowed. The information is available on the Sanofi.com website for anybody who is interested in learning about the study results.
“Individuals who participated in a trial deserve to know what came from the research that they helped generate,” adds DiBiaso. “We want to deliver healthcare solutions that address the most pressing needs of the patient community and their caregivers with a model that involves co-collaboration and co-development. Data generation will go beyond traditional safety and efficacy and will involve tolerability and issues important to quality of life. We need to create clinical trials that are diverse and representative, with innovations that make it easier for people to participate and data collection that involves no additional burden. That is our goal for every trial we conduct.”