Putting patients at the center of our clinical trials

Engaging with patients and caregivers across the life cycle of our medicines is critically important to ensure we develop treatments that not only are safe and effective, but also truly address unmet medical needs.

The Novartis Commitment to Patients and Caregivers, launched in 2018, guides our teams across functions and divisions in ensuring patients have a voice in the research, development and commercialization of our medicines. We continue to roll out a systematic approach to patient engagement that covers the full development life cycle – from early research to post-launch.

We continue to roll out a systematic approach to patient engagement that covers the full development life cycle – from early research to post-launch

In 2021, 57, or around 30%, of our clinical development programs had a patient engagement component, up from 44 in 2020. Patients provided input on key aspects such as clinical study endpoints that matter to their disease, how to facilitate easier access to clinical trial sites, and how to enable remote trial participation. We also integrated patient insights into early-stage research: In 2021, 46 NIBR programs had a patient engagement component.

57

Clinical programs

with a patient engagement component, up from 44 in 2020

Patient engagement also enables us to streamline our clinical trials and bring our medicines to market more quickly. For example, early input from patients in trial design can help us reduce the need for costly and time-consuming protocol changes later in the development process while ensuring that the trial design is aligned with study participants’ needs and preferences. In addition, we continue to help patients access medicines through our patient support and managed access programs. Please see the section “Build trust with society” for more details.

In 2021, we engaged with 162 patient organizations across 48 disease areas to inform our decision-making.

During the COVID-19 pandemic, the need for patient support grew while the income of patient organizations dropped, primarily due to the cancellation of educational events and fundraising activities. We provided funding to the community that was – among other purposes – used to strengthen digital communications tools and channels to help bridge these gaps. In 2020, the latest year for which data is available, Novartis supported 1 248 patient organizations in 79 countries. Please see the Novartis corporate website for more information.

Diversity in clinical trials

We strive to include diverse patient populations in our clinical trials – both to understand how patients who are most likely to be treated for a disease will respond to a medicine, and because it is the right thing to do.

We are working to address barriers to clinical trial participation, such as identifying sites where patients with a particular disease or condition may be located, identifying healthcare providers that treat underserved or underrepresented populations, and collaborating with researchers to enroll diverse populations in clinical trials.

In 2021, we published a Commitment to Diversity in Clinical Trials. In the short term, we committed to evaluate diversity and inclusion principles for all our Phase III studies with US country participation. In the longer term, we aim to expand this to all our global trials while leveraging data science and digital technology to track diversity data across our drug development programs.

In the US, health disparities affecting minority groups are endemic – an issue that was highlighted and exacerbated by the COVID-19 pandemic. Compared with non-Hispanic whites, the Black and African American community has a lower life expectancy, a higher mortality rate from cancer, a greater likelihood of diseases such as asthma, and significantly increased rates of maternal and infant mortality. Health inequity is also a particular challenge for the Black and African American community in medical schools and among physicians and clinical trial investigators; in all three groups, Black and African Americans are proportionally underrepresented.

In 2021, Novartis and the Novartis US Foundation announced plans to invest approximately USD 13.7 million to establish three research centers at Morehouse School of Medicine in Georgia, including a clinical trial center of excellence that could be a model for possible expansion to other historically Black colleges, universities and medical schools.

The investment is part of a 10-year commitment with Morehouse; 26 historically Black colleges, universities and medical schools; and other organizations. The objective is to co-create programs that address the root causes of systemic disparities in health outcomes and foster greater diversity, equity and inclusion across the R&D ecosystem.

In addition, we are collaborating with GlaxoSmithKline (GSK) to support research investigating the link between genetic diversity across different regions in Africa and its potential impact on response to drug therapeutics. With a combined funding commitment of GBP 2.8 million (USD 3.6 million) over five years, the project calls on African researchers to submit research proposals on the relevance of African genetic diversity to the treatment of malaria and tuberculosis. Proposals selected for funding are expected to be announced in the first half of 2022.

For more information on the Novartis R&D pipeline, please see www.novartis.com/researchdevelopment/novartis-pipeline