The Secret Sauce for Bringing Innovative Therapies to Patients with Rare Diseases Faster

Nov 02, 2021

Guest Blog by Lori Lawter, MPH, Senior Patient Advocacy Liaison, Center for Rare Diseases

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?” 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 a survey with Sponsor companies, one company noted that working with patient groups had saved the company millions of dollars that otherwise would have been spent on patient recruitment and retention. 2

In addition, the involvement of patients at early stages can build important bridges between patient organizations and industry, increasing trust. The importance of the patient advocacy organization cannot be overstated. In rare disease specifically, patients and caregivers seek support, education, and guidance from patient advocacy organizations. These important organizations provide disease state information, clinical trial awareness, and policy updates to their constituents. Building transparent, authentic relationships with patient advocacy organizations in the space you’re working in is essential, not just for trial efficiency, but throughout the lifecycle of your drug candidate. Muriel Finkel, Founder and President of Amyloidosis Support Groups states, “It is vital that pharma and patient advocacy organizations connect and maintain a close trusting relationship. Pharma needs to learn and understand the patient’s perspective on all aspects of research, clinical trials, and treatments. Everything they do will eventually trickle down to the patients. In turn the patients need to learn about the research, clinical trials and treatments that will affect their very lives. The Patient Advocacy groups are the perfect liaison for this exchange.” If your trial experiences challenges or setbacks, your relationships with patient advocacy organizations may make the difference in maintaining goodwill in the patient community. Most importantly, involving patients in trial design is just the right thing to do. It demonstrates a respect for the knowledge and lived experiences of those closest to the problem we’ve resourced so diligently. To address the need for increased patient input in clinical trials, ICON developed a free, open-source Patient-Centric Trial Development Toolkit 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.

Learn more here.


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 Stergiopoulos, S. et al. (2019) Measuring the Impact of Patient Engagement and Patient Centricity in Clinical Research & Development. Therapeutic Innovation & Regulatory Science, DOI: 10.1177/2168479018817517


Lori Lawter, MPH
Senior Patient Advocacy Liaison, Center for Rare Diseases

Lori Lawter is an expert in Patient Advocacy with over 13 years in the field, working for both patient advocacy organizations as well as biotech.  Specializing in rare hereditary diseases and hematology/oncology, Ms Lawter has experience in hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), acute hepatic porphyria, primary hyperoxaluria type 1, as well as blood cancers including leukemia, lymphoma, and myeloma, in addition to rarer hematologic conditions. She has experience across the continuum of drug discovery and commercialization with an emphasis on developing lasting, mutually beneficial relationships with patient advocacy organizations.  She has experience leading disease awareness and education initiatives as well as product launch experience.  She holds a master’s degree in public health from Benedictine University and sits on the board of The Amyloidosis Foundation.

See all MassBio News