A Patient-Centric Approach to Sickle Cell Disease Clinical Trials: Integrating Patient Perspectives in the RISE | |
UP Phase 2/3 Trial of Mitapivat for Informed Protocol Design and Associated Patient Community Benefit | 2376 |
Charles Jonassaint, PhD1, Blaze Armon Eppinger2, Dominique Friend3, Golie Lorenzo Green4, Mia Robinson5, Teonna Woolford6, DeMitrious Wyant7, Janie Davis8, Abdulafeez Oluyadi8, PharmD, Ahmar U Zaidi, MD8, Holly John8, Wally R Smith, MD9
1University of Pittsburgh Medical Center, Pittsburgh, PA; 2Independent Contributor, Eatonton, GA; 3Independent Contributor, Orlando, FL; 4Independent Contributor, Houston, TX; 5Sickle Cell Awareness 365, Atlanta, GA; 6Sickle Cell Reproductive Health Education Directive, Owings Mills, MD; 7Independent Contributor, Washington, DC; 8Agios Pharmaceuticals, Cambridge, MA; 9Virginia Commonwealth University, Richmond, VA
- The campaign launched a YouTube advertisement during World Sickle Cell Day 2022 (June 19) and monitored site traffic to the video, as well as to clinicaltrials.gov, Twitter, and the RISE UP clinical trial website
- Media metrics, including views, clicks, and webpage visits, as well as other key performance indicators were used to measure campaign success as well as infer insights for future trial design or awareness campaigns
RESULTS
- Patient contributions were considered at appropriate points throughout the drug development process (Figure 1)
Figure 1. Patient engagement process
Phase 2/3 Clinical Trial | Enrollment Campaign | Continued Development |
Design | ||
Drug Development
Journey
Tracking enrollment | Patients notified of trial | ||||||||||||
Patients notified of | |||||||||||||
regulatory feedback | uptake and online | milestones and consulted | |||||||||||
and final trial | design | engagement | on next steps | ||||||||||
Proposal made | Patients participation in | ||||||||||||
to regulators, | promotional materials | ||||||||||||
feedback | |||||||||||||
received | |||||||||||||
Patient recommendations | |||||||||||||
Patient selection of | |||||||||||||
incorporated in initial trial | |||||||||||||
campaign idea | |||||||||||||
design | |||||||||||||
Patient advisory | Patient opinions on trial | ||||||||||||
board formed for | messaging collected | ||||||||||||
collection of | |||||||||||||
perspectives |
Protocol Design
- Patient contributions to the protocol design included modified inclusion/exclusion criteria and the addition of pain (beyond pain crises) and fatigue as study outcomes; an overview is shown in Table 1
- As per patient input, the trial was adjusted to include a recommendation for tailored management of SCD pain crises using a daily diary; the trial also approved reimbursement for study-related travel, lodging, and specific non- study assessments
Table 1. RISE UP trial design, summary of community feedback on different aspects of the study design
Study Aspects | Community Feedback | Protocol Design Elements | ||
Study | • | 12-month study duration may | • | Approximately monthly study visits |
Duration | hamper participation and/or | for 7 months, followed by visits | ||
compromise compliance | every 3 months | |||
Post-study | • | Important to provide patients | • | An open label extension period |
access | access to treatment after | was also added with visits on the | ||
completion of the clinical trial | 2nd, 4th, 8th, and 12th weeks of the | |||
extension period, followed by | ||||
every 3 months up to 1.5 years | ||||
and every 6 months thereafter |
Campaign
- An example of a campaign that was co-created with the sickle cell warriors can be seen in Figure 2
Figure 2. Campaign co-created with sickle cell warriors
- The RISE UP campaign heightened interest within the community for clinical trial participation (Figure 3)
- Strong social media engagement and digital efforts resulted in increased trial website views
- Community education and awareness efforts resulted in increased clinicaltrials.gov views to an average of 2-3 clicks per day
Figure 3. Media metrics following RISE Up campaign launch during
World Sickle Cell Day
Table 2. Key learnings and recommendations for patient involvement in trial design and recruitment
Action | Learnings | Recommendations | |
Trial design | • Prioritizing patient input | • | Create patient advisory board to guide |
and involvement can | decision-making processes | ||
enhance the clinical trial | • | Take action on recommendations and keep | |
design process and help | patients informed of continued | ||
address patient concerns | developments | ||
• | Ensure trial protocol meets both regulatory | ||
requirements and patient needs | |||
Patient relevant | • Including patient- | • | Prioritize the development of the most |
endpoints | centered outcomes and | convenient protocol possible for patients | |
endpoints improves data | based on their lived experience | ||
collection methods | |||
Trial awareness | • Patient involvement can | • Build relationships and trust with patients, | |
create a uniquely | families, and patient advocacy groups | ||
impactful and | • Engage local and national decisionmakers | ||
differentiated awareness | and influencers | ||
approach | • | Provide information to patients in a | |
comprehensive manner, tailored to | |||
common questions and potential | |||
concerns | |||
Communications | • Meeting patients where | • Leverage social media platforms for | |
channels | they are, using familiar | campaign reach and engagement | |
language and engaging | • Allow patients to guide the process to | ||
with their priorities drives | generate authenticity and trust among | ||
engagement and action | peers |
• Monitor traffic to trial websites and clinical trial registration platforms to assess impact and course correct, as needed
CONCLUSIONS
- By partnering with patients with SCD to gather their contribution to protocol design, Agios integrated feedback from patients into the design and implementation of the study
- The campaign succeeded in building awareness and engagement with patients, families, and advocacy groups, as well as local and national decision-makers and influencers
- The Phase 2 portion of the RISE UP clinical trial was launched on time and fully enrolled, a notable result given that 80% of clinical trials globally are delayed due to missed enrollment targets5
- Learnings and recommendations from this innovative patient-forward approach may be applied further throughout the drug development process
- Reaching/engaging individuals and local community groups, fostering relationships, and building trust to gain support
- Connecting with decision-makers and influential individuals who could advance the campaign's objectives
Study | • | Evaluate effect as assessed by | • | Key secondary and other |
endpoints | high quality patient-reported | secondary endpoints in the study | ||
outcomes (PROs) | include Health-Related Quality of | |||
• | Evaluate pain and fatigue | Life and Performance Outcome | ||
• | Provide flexibility with | Assessments | ||
questionnaire and minimize | ||||
questionnaire burden | ||||
Eligibility | • | Assessing effect in patients with | • | Subgroup analyses specified in the |
criteria | Hb <8 g/dL could be beneficial | protocol based on baseline Hb (<8 | ||
• | Prohibiting concomitant therapies | g/dL, >=8 g/dL) | ||
(e.g., hydroxyurea or exchange | ||||
transfusion) may preclude patient | ||||
participation | ||||
Hb, hemoglobin; PRO, patient reported outcome.
Key Learnings and Recommendations for Future Patient Engagement
- Patient engagement for clinical trial design for the RISE UP study yielded important insights relating to trial design, endpoint selection, trial awareness, and effective use of communication channels (Table 2)
By engaging with patient communities, the RISE UP campaign was able
to build trust, engage with local and national decisionmakers, and
provide timely information to patients, allowing them to make informed
decisions about trial participation
Disclosures
- This study was funded by Agios Pharmaceuticals, Inc.
- Writing support was provided by FleishmanHillard, funded by Agios Pharmaceuticals, Inc.
- Abdulafeez Oluyadi, Ahmar U. Zaidi, Holly John, and Janie Davis are active employees at Agios Pharmaceuticals
- Received honoraria from Agios: Blaze Eppinger, DeMitrious Wyant, Dominique Friend, Golie L. Green, Mia Robinson, and Teonna Woolford
- Charles Jonassaint: Honoraria received from Agios; employed by and current shareholder in Expressive Painimation
- Wally Smith: Honoraria received from: Agios, Novartis, bluebird bio, and Pfizer
Acknowledgments
- Thank you to the Sickle Cell Warriors who participated and their families, our collaborators, the study investigators, and our advisors in the patient and clinical communities
- We would like to acknowledge Agios Pharmaceuticals, Inc. for supporting and funding this campaign and study
References: 1. Brandow AM, Liem RI. J Hematol Oncol. 2022;15(1):20; 2. Geißler et al. Commun Med 2022;2,94; 3. Lebensburger JD et al. Pediatr Blood Cancer. 2013;60(8):1333-1337;4. ClincalTrials.gov. A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP). https://classic.clinicaltrials.gov/ct2/show/NCT05031780. Accessed Oct 27, 2023; 5. Desai M. Perspect Clin Res. 2020;11(2):51-53.
This study was funded by Agios Pharmaceuticals, Inc.
Presented at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition, December 9-12, 2023, San Diego, CA, USA, and Virtual
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Agios Pharmaceuticals Inc. published this content on 09 December 2023 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 19 December 2023 18:03:30 UTC.