Adopting recommendations for implementing patient involvement in cancer research: a funder's approach.

BACKGROUND: The role of patients in cancer research is undergoing a significant evolution as all stakeholders seek to enhance the level of direct patient involvement in the design and development of clinical trials. However, there are significant hurdles that patients, patient advocates, laboratory researchers, clinical investigators, and funding institutions must overcome to implement relevant patient involvement in all aspects of biomedical research. By using innovative grant funding models, philanthropic organizations can lead the field in overcoming these challenges. Rising Tide Foundation for Clinical Cancer Research (RTFCCR), a private philanthropy that funds academic research, has developed a novel approach for requiring and supporting partnerships among grantees and patients in designing and conducting research projects. This paper presents a reflective case study of efforts to advance the field of patient involvement in clinical research. METHODS: The decision to focus on patient involvement stems from an expressed focus area established by the RTFCCR board of directors. In conducting this work, RTFCCR partnered with Patvocates, a patient advocacy and engagement network, to create a set of guiding documents and resources aimed at public and private health research funders within various national, international, and therapeutic settings. This effort included a landscape assessment, interviews with experts, and an iterative development process. RESULTS: To date, RTFCCR has completed and disseminated three guiding documents, one for funders, one for grant applicants, and one for patient advocates. These resources have already generated three major ongoing initiatives at RTFCCR: (1) inclusion of these recommendations in the foundation's funding guidelines; patient input to prioritization of research focus areas; and in topic selection for calls for proposals; (2) direct involvement of patient experts in the grant review process; and (3) a commitment to support high impact clinical research projects in Low- and Middle-Income Countries. Moreover, the foundation has launched a partnership with the International Cancer Research Partnership, the global alliance of cancer research organizations. CONCLUSION: By using its grantmaking function and developing standardized approaches for implementation of patient involvement, RTFCCR is seeking to advance patient-centric cancer clinical research. This approach will continue to develop as it is implemented and shared with partners throughout the world.

The Rising Tide Foundation for Clinical Cancer Research (RTFCCR), a private philanthropy that funds academic research, has developed a novel approach for requiring and supporting partnerships among grantees (scientists) and patients in designing and conducting research projects.The decision to focus on patient involvement stems from an expressed focus area established by the RTFCCR board of directors. In conducting this work, RTFCCR partnered with Patvocates, a patient advocacy and engagement network. Patvocates conducted a landscape assessment, interviews with experts, and their collective experience as patient advocates. This work generated a set of guiding documents and resources. These resources are to help public and private health research funders to better understand current challenges and support scientists and patients through their funding mechanisms. Three guiding documents, one for funders, one for grant applicants, and one for patient advocates are now available for download at the RTFCCR website: https://www.risingtide-foundation.org/clinical-cancer-research/patient-engagement#start Delivering a paradigm change involves not only the introduction of additional requirements and rules, but also enhanced education of patients and investigators. By using its grantmaking function and developing standardized approaches for implementation of patient involvement, RTFCCR is seeking to advance patient-centric cancer clinical research.Development and implementation of consistent policies and procedures for the integration of the patients' view in the design and review of research proposals is needed for funders as well as for research institutes, both public and private.

Leveraging Patient Preference Information in Medical Device Clinical Trial Design.

Use of robust, quantitative tools to measure patient perspectives within product development and regulatory review processes offers the opportunity for medical device researchers, regulators, and other stakeholders to evaluate what matters most to patients and support the development of products that can best meet patient needs. The medical device innovation consortium (MDIC) undertook a series of projects, including multiple case studies and expert consultations, to identify approaches for utilizing patient preference information (PPI) to inform clinical trial design in the US regulatory context. Based on these activities, this paper offers a cogent review of considerations and opportunities for researchers seeking to leverage PPI within their clinical trial development programs and highlights future directions to enhance this field. This paper also discusses various approaches for maximizing stakeholder engagement in the process of incorporating PPI into the study design, including identifying novel endpoints and statistical considerations, crosswalking between attributes and endpoints, and applying findings to the population under study. These strategies can help researchers ensure that clinical trials are designed to generate evidence that is useful to decision makers and captures what matters most to patients.

The promise of Immuno-oncology: implications for defining the value of cancer treatment.

The rapid development of immuno-oncology (I-O) therapies for multiple types of cancer has transformed the cancer treatment landscape and brightened the long-term outlook for many patients with advanced cancer. Responding to ongoing efforts to generate value assessments for novel therapies, multiple stakeholders have been considering the question of "What makes I-O transformative?" Evaluating the distinct features and attributes of these therapies, and better characterizing how patients experience them, will inform such assessments. This paper defines ways in which treatment with I-O is different from other therapies. It also proposes key aspects and attributes of I-O therapies that should be considered in any assessment of their value and seeks to address evidence gaps in existing value frameworks given the unique properties of patient outcomes with I-O therapy. The paper concludes with a "data needs catalogue" (DNC) predicated on the belief that multiple key, unique elements that are necessary to fully characterize the value of I-O therapies are not routinely or robustly measured in current clinical practice or reimbursement databases and are infrequently captured in existing research studies. A better characterization of the benefit of I-O treatment will allow a more thorough assessment of its benefits and provide a template for the design and prioritization of future clinical trials and a roadmap for healthcare insurers to optimize coverage for patients with cancers eligible for I-O therapy.

Developing standards for breakthrough therapy designation in oncology.

In July 2012, Congress passed the Food and Drug Administration Safety and Innovation Act (FDASIA). The Advancing Breakthrough Therapies for Patients Act was incorporated into a Title of FDASIA to expedite clinical development of new, potential "breakthrough" drugs or treatments that show dramatic responses in early-phase studies. Using this regulatory pathway, once a promising new drug candidate is designated as a "Breakthrough Therapy", the U.S. Food and Drug Administration (FDA) and sponsor would collaborate to determine the best path forward to abbreviate the traditional three-phase approach to drug development. The breakthrough legislation requires that an FDA guidance be drafted that details specific requirements of the bill to aid FDA in implementing requirements of the Act. In this article, we have proposed criteria to define a product as a Breakthrough Therapy, and discussed critical components of the development process that would require flexibility in order to enable expedited development of a Breakthrough Therapy.

Examining advocacy and comprehensive cancer control.

The effectiveness of advocacy has been well documented and its use established as a common practice in furthering sound public health policy and practices. Efforts are underway to explain how advocacy has supported, shaped and influenced public policy concerning -- and the growth and development of -- comprehensive cancer control initiatives. The objective of this paper is to assess the history, current role and future of advocacy as a means of articulating the value of, and furthering, comprehensive cancer control practices. Comprehensive cancer control is approaching a critical moment in its development, and a unified approach is necessary to achieve common goals. A call to action for supporting and contributing to the success of a comprehensive approach to cancer control is more important today than it was 11 years ago. Advocacy is an essential strategy in that call to action.