By James E. Valentine* –
In recent years there has been an increasing focus on patient engagement, centering on new opportunities for FDA to incorporate the patient perspective into its regulatory decision making. These discussions have resulted in Congress and FDA establishing new policies and programs (see our previous posts on these efforts here, here, here, here, and here). However, the public dialogue has largely left out the opportunities for meaningful engagement between patients and research sponsors (academia and the medical product developers). That changed in 2014, when Clinical Trials Transformation Initiative (CTTI) initiated the Patient Groups & Clinical Trials (PGCT) Project.
CTTI is a public-private partnership established to identify and promote practices that will increase the quality and efficiency of clinical trials. Government partners included FDA, NIH, CMS, OHRP, AHRQ, CDC, and the Department of Veterans Affairs. For those of you that may not be familiar with CTTI or its work, information about its membership, project methodology, projects, and published resources can be found here.
The PGCT Project was established to find evidence-driven, actionable solutions to a number of questions: How and when should patient groups be engaged in the research and development continuum? How do patient groups and sponsors of research best assess each other’s interests, expertise, and assets to increase the chances of successful clinical trials and therapy development?
Today, CTTI is releasing the culmination of that effort at the BIO Patient and Health Advocacy Summit (view the slides presented at this session here). CTTI’s PGCT Project official recommendations identify best practices for engaging with patient groups, as well as provide case examples and tools.
The PGCT Project
Key sectors of the research community had identified a gap in the knowledge and understanding about how and when to best interact with patient groups and clinical trials. There was a dearth of empirical evidence and no guidelines or best practices existed. As a result, CTTI identified a need for actionable recommendations and metrics, which was the foundation for establishing the PGCT Project.
To work towards the goal of establishing guidelines and best practices, the PGCT Project first sought to identify gaps and barriers to effective engagement between patient groups and research sponsors. The project then took that information to experts to analyze and interpret, and subsequently inform the development recommendations. The PGCT Project consisted of a series of activities:
- Literature Review and Survey – a joint CTTI/Drug Information Association (DIA) survey elicited feedback from 244 respondents and examined current practices and perceptions among the different stakeholders about the value of, and barriers to, successful patient group engagement in clinical trials;
- Semi-structured interviews – a qualitative scientist conducted 32 semi-structured interviews with 10 leaders of patient groups, 12 industry sponsors, and 10 academic investigators to follow up on findings from the survey; and
- Expert Meeting – presentations and discussions from diverse stakeholders during a January 2015 expert meeting examined the findings of the survey and semi-structured interviews. Meeting participants also explored the diverse capabilities and assets that many patient groups are assembling, as well as examples of how and when research sponsors and patient groups are engaging to build effective partnerships. Based on these experiences, participants were asked challenging questions about overcoming barriers and developing best practices. FDA was well represented at the meeting, with presentations and/or participation by Janet Woodcock and Theresa Mullin from CDER, Richard Klein and Steve Morin from the Office of Health and Constituent Affairs, and Kathryn O’Callaghan and Annie Saha from CDRH, among others.
Through this process, CTTI was able to characterize the range of “best practices” that respondents reported are producing positive results, which are intended to serve as guideposts for patient groups and research sponsors alike.
The PGCT Recommendations
The PGCT recommendations document organizes its best practices into three categories: (1) recommendations for all stakeholders, (2) recommendations for research sponsors—industry and academia, and (3) recommendations for patient groups.
Several of the recommendations emphasize the importance of engaging early and often. CTTI developed an infographic (see below) that provides a fairly comprehensive list of opportunities for patient groups to engage during clinical trials.
At whatever point engagement begins, from the start it is recommended that the research sponsor and patient group clarify the roles of the partnership and set expectations. For example, the document emphasizes the importance of patient groups to understand that:
[W]hile [their] input may be taken into account when determining the objectives of a clinical program or development of a protocol, research sponsors must balance that input with scientific understanding as well as business and regulatory needs. These multiple influences reflect the reality of the environment that will drive the program, and PGs should understand that research sponsors reserve the right to make final decisions about study design.
In addition to establishing roles and responsibilities, the recommendations call for all stakeholders to be open, transparent, and honor the commitments they have agreed upon (including Confidentiality Agreements). CTTI recommends that agreements are documented, and such documentation can be customized to fit the needs of each partnership (e.g., may include a Memorandum of Understanding or more formal contract).
With respect to whom to engage with, CTTI recommends that patient groups should be involved with multiple research sponsors to maximize the potential pipeline of therapies in development. Likewise, it is recommended that sponsors should engage with more than one patient group in a particular disease area to ensure that a representative patient perspective is reflected in the input obtained.
Finally, the recommendations note that while there are a number of FDA-related and other legal and regulatory issues surrounding sponsor engagement with patient groups, there is no explicit prohibition against early engagement with patient groups. CTTI recommends that research sponsors clarify which kinds of interactions are permissible and which ones might violate FDA regulations or fraud, abuse, and other regulations. This includes patient groups understanding these rules and taking appropriate steps. For example, if establishing a partnership with a sponsor with trial recruitment (e.g., raising awareness, assisting with screening), patient groups should clearly characterize clinical studies as research, not misrepresent the investigational nature of the trial, and convey information about a trial as approved by the Institutional Review Board (IRB).
These recommendations discussed above cover those that apply to both research sponsors and patient groups, however, the CTTI’s document provides a number of more detailed best practices for both sets of stakeholders.
In addition to its recommendations, embedded in the document, CTTI has provided a series of tools to assist research sponsors in engaging with patient groups.
- The “Patient Group Organizational Expertise and Assets Evaluation Tool” can be used by research sponsors to analyze patient group skills and strengths and by patient groups to help define their values and document their assets.
- The “Assessment of Patient Group Internal Aspects: Focus” and “Assessment of Patient Group External Relationships: Other Patient Groups” tools provide research sponsors with a way to assess patient group expertise, interests, organizational capacity, and relationships. Using these will direct sponsors to learn about the patient group’s priorities, past and present programs, and strengths in policy, finance, and research. The hope is that these tools will make it easier for sponsors to identify patient groups whose strengths match their needs.
While the PGCT Project helped reveal several modifiable barriers to successful relationships between research sponsors and patient groups, it also identified that further work needs to be done on metrics and models to assess the value and impact of such engagement. The PGCT Project has established a Value Work Stream that is currently developing new conceptual models and methods to measure the benefit of partnerships captured in CTTI’s recommendations.
*James Valentine serves as a member of CTTI’s Patient Groups & Clinical Trials Project team. He is admitted only in Maryland, and his work supervised by the Firm while D.C. application pending.