Drug development for major chronic health conditions-aligning with growing public health needs: Proceedings from a multistakeholder think tank.

The global pharmaceutical industry portfolio is skewed towards cancer and rare diseases due to more predictable development pathways and financial incentives. In contrast, drug development for major chronic health conditions that are responsible for a large part of mortality and disability worldwide is stalled. To examine the processes of novel drug development for common chronic health conditions, a multistakeholder Think Tank meeting, including thought leaders from academia, clinical practice, non-profit healthcare organizations, the pharmaceutical industry, the Food and Drug Administration (FDA), payors as well as investors, was convened in July 2022. Herein, we summarize the proceedings of this meeting, including an overview of the current state of drug development for chronic health conditions and key barriers that were identified. Six major action items were formulated to accelerate drug development for chronic diseases, with a focus on improving the efficiency of clinical trials and rapid implementation of evidence into clinical practice.

Creating Equitable and Inclusive Clinical Trials for Multiple Myeloma.

Black and Latino/Hispanic populations are disproportionately impacted by multiple myeloma (MM) in the United States and are underrepresented in many clinical trials. The Multiple Myeloma Research Foundation sponsored a 1-day workshop of 46 experts spanning the ecosystem of MM research and care, including government, academia, nonprofits, pharma/biotech, community partners, and retail pharmacy. Specific, tangible steps to overcome the well-documented barriers to improving the diversity and inclusivity of clinical trials were discussed, including broadening inclusion/exclusion criteria, reducing the financial and other burdens of trial participants, selecting diverse study sites, including implicit bias training, and taking steps to empower patients.

Closing Knowledge Gaps to Optimize Patient Outcomes and Advance Precision Medicine.

Realizing the promise of precision medicine requires patient engagement at the key decision points throughout the cancer journey. Previous research has shown that patients who make the "right" decisions, such as being treated at a high-volume academic medical center, for example, have better outcomes. An online survey was conducted to understand awareness of and barriers to these decision points among patients with multiple myeloma and pancreatic, lung, prostate, and metastatic breast cancers. Survey respondents were identified by 5 participating foundations (multiple myeloma: n = 86, pancreatic: n = 108, lung: n = 56, prostate: n = 50, metastatic breast: n = 86) and recruited by an e-mail or social media invitation. Descriptive analyses were calculated, and the proportion of patients from each of the 5 groups was compared for each response category for each survey item. Consistent gaps in knowledge and actions were identified across all cancers evaluated in terms of finding the right doctors/team at the right center; getting the right diagnostic testing done before beginning treatment; engaging in the right course of treatment, including clinical trials; and in sharing data. Improving awareness of and changing behavior around these 4 decision points will allow patients to receive better care and contribute to the advancement of precision medicine.

One foundation's strategy to accelerate drug discovery through genomics.

The Multiple Myeloma Research Foundation (MMRF) has the principal goal of accelerating development of next-generation drugs for treating multiple myeloma. By making targeted investments in key research areas such as genomics and epigenetics, the MMRF is helping to elucidate the basic biology of multiple myeloma, to drive promising new treatments into clinical development, and ultimately to link the right treatment to the right patient.