Written by Lori Lawter, MPH

We’ve heard a lot about bringing drugs to market lately, especially in light of the COVID-19 vaccine.  You might wonder, “Is there a ‘secret sauce’ or most effective way to bring innovative drugs to patients of rare disease quickly and safely?” I’m glad you asked. While every new therapy and condition is different, there is one stand-out practice that has proven over and over to be vitally important.  Maybe the most important.  Drum roll please… Involve patients. Yes, you heard that correctly.  Involve affected patients in all stages of research and development. Seems like a no-brainer, right? Who knows more about a disease or condition than the person living with it? You may be surprised to learn that this doesn’t always happen.

Recognizing the need for patient involvement is the first step. There are compelling economic reasons to engage patients in trial design. For a pre–Phase 2 project, the cumulative impact of a patient engagement activity that avoids one protocol amendment and improves enrollment, adherence, and retention is an increase in net present value (NPV) of $62 million ($65 million for pre–Phase 3) and an increase in estimated net present value (ENPV) of $35 million ($75 million for pre–Phase 3).¹ In addition, the involvement of patients at early stages can build important bridges between patient organizations and industry, increasing trust. Most importantly, it’s just the right thing to do. It demonstrates a respect for the knowledge and lived experiences of those closest to the problem we’re attempting to address.

The Patient-Centric Trial Development Toolkit was designed to provide sponsors and champions of patient centricity with tools they can use to operationalize patient engagement at the level of a specific clinical program. This is a free, open-source, toolkit developed by the ICON’s Rare Disease Advocacy Committee² members in collaboration with the Center for Rare Diseases team at ICON, plc.  These tools were designed to be easy-to-use and provide value whether patient centricity is new or well established within a company’s team.

Components of the Patient-Centric Trial Development Toolkit

• Patient Involvement Value Dossier-This tool outlines the evidence of ROI/benefit involvement in the trial development process from extant literature and provides illustrative case studies.
• Rapid Participation Burden Survey Tool-An easy-to-use questionnaire development guide that helps sponsors and patient advocates develop a rapid survey for patients and caregivers tailored to their clinical trial’s specific context. The aim of this tool is to help sponsors quantify risk to the clinical program through direct patient engagement.
• Patient-centric Protocol Risk Assessment Tool-Interactive, spreadsheet-based tool that enables sponsors to rapidly identify potential risks, track evolution of risk assessment through stages of the development process, and identify potential mitigation strategies.
• “What to Ask When You’re Interested in a Clinical Trial: A Guide for Rare Disease Patients and Caregivers”- This guide helps prospective participants identify the barriers to participation they may encounter and request the support they need to enroll and stay in the trial.

Please feel free to request the Patient-Centric Trial Development Toolkit at no charge here: [email protected]  Or download it on your own here.

 References

  ¹Levitan, B et al. (2019) Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI’s Patient Groups and Clinical Trials Project.Therapeutic Innovation & Regulatory Science, 52(20), pp. 220-229.

² The Rare Disease Advisory Committee is an independent, voluntary, standing committee of rare disease patients, caregivers and advocacy leaders.