Hope’s Horizon for Rare Diseases Artist Nancy J. Willis
The FDA Center for Drug Evaluation and Research (CDER) has partnered with Myositis International Health & Research Collaborative Alliance (MIHRA) Foundation and the MIHRA Clinical Trial Sites Network (CTSN) to launch a groundbreaking public-private partnership (PPP). MIHRA’s partnership follows in the pioneering footsteps of the Lupus Research Alliance’s (LRA) Lupus Accelerating Breakthroughs Consortium (Lupus ABC), making MIHRA Foundation’s FDA CDER PPP the second disease and the very first rare disease to work side-by-side with FDA lead scientists to address the challenges impacting clinical trial success in complex diseases.
The MIHRA FDA Myositis PPP brings together a broad coalition of stakeholders, including people living with myositis and their loved ones, professional societies, patient organizations, industry partners, and multi-disciplinary clinicians and scientists. These groups will work alongside the FDA, the National Institute of Environmental Health Sciences (NIEHS), other federal agencies and with the American College of Rheumatology (ACR) to overcome the critical barriers to clinical trial readiness and accelerate the development of urgently needed treatments for myositis diseases.
Myositis disorders are a group of complex, potentially life-threatening autoimmune disorders that can result in catastrophic multi-organ impairment and significantly disrupt daily life for both children and adults. Myositis diseases can involve widespread inflammation and damage to affected organs such as muscle, skin, heart, lungs, gastrointestinal tract and blood vessels. Myositis diseases affect each person differently, and its multi-organ system nature can make it one of the most challenging diseases to diagnose, treat and research.
Input from all MIHRA PPP partners will guide the prioritization of projects with the greatest potential to advance therapeutic development. Initial efforts will focus on:
- Developing and validating improved measurement tools to more accurately assess treatment efficacy. Current assessment methods have shown significant limitations in measuring a drug’s efficacy, ability to recognize patients with active disease and ensuring appropriate recruitment into clinical trials.
- Fully incorporating the patient voice into the drug development process. This includes identifying symptoms that matter most to patients, evaluating the impact of different potential types of treatments, designing patient-centred clinical trials, and developing meaningful patient-reported outcome measures.
MIHRA is committed to sharing the process of this critical PPP endeavor with other rare disease communities.
