The ALPHA Project

• Identify knowledge gaps both in clinical care and research
• Create consensus around the fundamental pillars that are missing in the field and stalling drug development and progress
• Pave a path forward for more accurate and timelier diagnosis, greater access to care, and improved treatment options for people with lupus
• Result in an actionable, documented, and published roadmap that is meaningful and useful to everyone in the field, no matter where they are in the world

We know that the level and quality of data and research addressing issues related to lupus care, treatment, and healthcare access are not consistent across the global field. For example, there are differences in opinion when it comes to disease definition, treatment regiments, diagnostic criteria, and clinical trial design. The forum and opportunity to develop consensus around these issues had not existed.

This lack of consensus has led to a poor understanding about lupus, the inability to diagnose lupus in a timely fashion, especially by physicians with less experience in lupus, and a string of clinical trials of potential new treatments that failed to reach their primary endpoints and preventing them from being approved for use in treating lupus.

• Established a geographically and professionally diverse Global Advisory Committee (GAC) of lupus thought leaders to guide and oversee the study
• Conducted in-depth interviews to obtain further opinions and detailed rationale for the most urgent barriers in the field and access the level of existing agreement across the lupus expert community
• Conducted a global online survey of lupus experts focusing on the attitudes and perceptions about lupus and prioritization of the most pressing challenges to improve diagnosis, care, treatment and research
• Analyzed the survey results to identify and prioritize the barriers as ranked by the survey participants
• Global Advisory Committee has met and established next steps for addressing the identified barriers
• Implementation working groups were established for the Clinical Care, Access to Care and Drug Development Pillars
• A global meeting on clinical trial outcome measures was convened to discuss new and newly modified clinical trial outcome measures with a major focus on patient preference and needs for pediatric inclusivity
• A series of meetings were convened to determine challenges related to time to diagnosis and steps required to define the lupus spectrum

Lupus heterogeneity (diversity of the disease and the many manifestations or ways that it can impact the body) was identified as the primary barrier impeding the development of clinically meaningful treatments, the advancement of clinical care, and improvements to access and value. The study validated known challenges in lupus, identifying the five top barriers to improving outcomes in lupus:

• Lack of diagnostic, predictive and prognostic biomarkers for lupus and deficiency of biomarkers to predict drug response in clinical trials;
• Flawed clinical trial design;
• Lack of access to clinicians familiar with lupus/limited awareness of lupus among non-expert medical professionals;
• Barriers to the effective management of lupus due to social determinants of care in predominantly lower socioeconomic status areas;
• And, lack of treatment adherence.

• Drug development: Update current lupus outcome measures and issuing a declarative consensus statement on steroid-sparing drugs.
• Clinical care: Lead the development of a consensus effort to define lupus. The new definition will build on current research definitions to include closely related immune-mediated inflammatory disorders, such as cutaneous variants. After establishing a consensus definition, it will be disseminated through publication and broader discussion.
• Access to care: Spearhead an effort to gain a better understanding of social determinants of health and contributors to health disparities, and investigate the unique access to care issues faced by children with lupus.
• Additional steps: Conduct an assessment of current biomarker development and data-sharing activities and identify gaps. The assessment will lead to the creation of standardized global data-sharing efforts and support international collective research collaborations.

The ALPHA team will continue to engage stakeholders and support further research endeavors to implement solutions focused on accelerating drug development and improving access to high quality care. The Global Advisory Committee (GAC) clinical care and access to care teams will come together to focus on access to care barriers related to patient, provider and policy. The drug development team will continue to implement solutions focused on clinical trial outcome measures that are simplified but also elevate the patient voice. 

• Kathleen Arnsten | Lupus and Allied Diseases Association, U.S. and Person with Lupus
• Sang-Cheol Bae, MD, PhD, MPH | Hanyang University, South Korea
• Ian Bruce, MD, FRCP | University of Manchester, U.K.
• Denesh Chitkara, MD | EMD Serono Research & Development Institute, Inc. 
• Karen Costenbader, MD, MPH | Brigham and Women’s Hospital, U.S.
• Brad Dickerson | Lupus and Allied Disease Association, U.S.
• Thomas Dörner, MD | Charité University, Germany
• Monique Gore-Massy | Person with Lupus
• Ken Kalunian, MD | UC San Diego Health
• Roger Abramino Levy, MD | GSK
• Hetlena Johnson, M.ED | Person with Lupus
• Sue Manzi, MD, MPH | Allegheny Health Network, U.S.
• Eric Morand, MBBS (Hons), FRACP, PhD | Monash University, Australia
• Sarah Peddicord | AstraZeneca
• Sandra Raymond | Lupus Foundation of America, U.S.
• Brad Rovin, MD, FACP, FASN | Ohio State University, U.S.
• Tamar Rubinstein, MD, MS | Children's Hospital at Montefiore, U.S.
• Laura Schanberg, MD | Kuke University, U.S.
• Joan Von Feldt, MD, MSEd | Professor Emeritus, University of Pennsylvania
• Victoria Werth, MD | University of Pennsylvania and Philadelphia VAMC, U.S.

The ALPHA Project was launched in partnership with founding partner EMD Serono Research & Development, Inc. (a business of Merck KGaA, Darmstadt, Germany) and through additional support by GlaxoSmithKline.

The continued support and additional contributions of AstraZeneca and Lupus and Allied Diseases Association have made current Phase II efforts possible.