Repurposing Drug Research for Improved Patient Outcomes

Repurposing Drug Research for Improved Patient Outcomes

By David McKie, VP of Marketing, Medidata Solutions

In late 2017, Medidata’s Leadership & Organizational Development team, in conjunction with our Corporate Social Responsibility team, rolled out a new program called the ‘Social Innovation Lab’. A group of five employees were recruited to work with two UK non-profit companies – Cures Within Reach and Mission: Cure – to support them in one of their projects.

Cures Within Reach is an organisation focused on encouraging drug repurposing research as an effective approach to helping patients, and, Mission: Cure is dedicated to outcome based financing for curing disease. The goal of the project was to build a proposal for funding drug repurposing research around a Social Impact Bond (SIB) – and to find and engage a funder willing to participate in the effort.

Wanting to better understand the Life Sciences industry and to get closer to clinicians and researchers, I raised my hand to join the team. Over the next year, the project underwent two phases, with a second set of Medidata employees joining for phase two. I continued on as the project lead, bridging the work already done early in 2018, with the new group of project members.

A Two-Fold Challenge: Optimising Patient Outcomes & Finding Financial Support

Throughout the project, two issues arose as challenges within our current industry. Firstly the number of diseases without effective treatments and the patients that continue to suffer because of this. Secondly the many worthwhile ideas and drug repurposing projects that fail to advance because of a lack of financial support.

The pharmaceutical industry is built around the idea of patenting drugs, proving their safety and efficacy in treating diseases (preferably those affecting large numbers of patients), and providing returns to shareholders by maximizing revenues. The model clearly isn’t as effective for roughly 7,000 rare diseases that affect the smaller numbers of patients.

One of our site visits during the project involved meeting with medical researchers, Dr. S Berish Rubin and Sylvia Anderson, at Fordham University in the Bronx, NY. Visiting their offices and lab, we learned about the impact of drug repurposing on the lives of patients affected by familial dysautonomia (FD), a rare life threatening genetic disorder. Through their research they discovered the cause of FD and more importantly, identified beneficial diet restrictions and natural compounds—including tocotrienols, a form of Vitamin E—that allowed people with FD to quickly and dramatically improve. Because of this, today, a progressively fatal disease affecting relatively small numbers of patients has an effective treatment. And, because the treatment is focused around diet and nutraceuticals, it is also affordable for most patients.

Project Focus: Repurposing Drug Development for Chronic Pancreatitis

Having seen the impact that repurposing can have, we turned back to evaluating the list of drug repurposing projects that Cures Within Reach had collected through an open call. There were many worthwhile projects on this list, however, we quickly realized there were too many diseases and potential drugs for us to provide an effective recommendation.

Together with Cures Within Reach and Mission: Cure, we opted to focus our efforts on a single disease namely chronic pancreatitis. With Mission: Cure’s support we were able to speak with doctors and medical researchers focused on chronic pancreatitis, reviewing relevant medical literature, and meeting with patients suffering from this disease.

One patient we met, was a five year old boy named Lucas, who bravely dealt with a condition characterized by recurrent bouts of debilitating pain and progressive destruction of the pancreas. Without effective treatments, he will continue to experience episodes of chronic pancreatitis that will likely result in hospitalization lasting days or weeks, missing school and not being able to venture far from home in case of another episode. A majority of chronic pancreatitis sufferers ultimately develop type 3c diabetes as their pancreatic function degrades.

The costs of chronic pancreatitis are significant, with one research article estimating the total direct and indirect costs to be almost £90k per year. These costs cover areas as varied as pain management, absenteeism, depression, endocrine and exocrine insufficiency, hospitalization, and medical intervention (e.g., ERCP, Whipple Procedure). However the costs affect a relatively small number of UK patients (estimated 33,000) and are spread across many different parties (e.g., employers, Clinical Commissioning Groups).

A New Funding Model

Seeing the potential from both the patient and economic perspective, we began to build out a proposal for a healthcare provider to fund the repurposing research directly. If funding drug repurposing research for rare diseases is not an economically viable proposition for pharma companies, then we believe that funding this research from cost savings just might be the right approach. And, with social impact investors providing the upfront cash, healthcare payers only pay if the research is successful and results in improvements for patients.

To build a proposal we first had to identify potential research projects. Without a central organizing body to help bring researchers together and catalogue research projects, it is a daunting task to understand the different research paths and treatment options under evaluation. This is particularly challenging for rare disease where there are few patients and limited focus from the medical community.

Fortunately for us, Mission: Cure had already invested considerable time and effort in networking with chronic pancreatitis researchers and cataloging their different repurposing projects. Even still, there was much effort needed to standardize these projects down to the level of disease target, potential benefits / endpoints, pre-clinical or other research indicating efficacy, countries where drug was approved, and cost of drug etc. Furthermore, much work still needs to be done to select the most promising projects for funding. It can be difficult to determine success potential for each drug, especially when each drug may target different pathways or receptors.

We also needed to lay out the criteria for success measures for the project. While some of the desired outcomes may be relatively obvious (for example reducing pain), they are not always easily or objectively evaluated. Fortunately in our discussions with medical researchers, we uncovered a validated PANQOLI (PANcreatic Quality Of Life Instrument) that incorporated pain measures. Other relevant outcomes include the number of ER visits and hospital admissions, and the number of surgical interventions, as well as measures of digestive function (secretin stimulation test). Selecting outcome measures for research projects included in the SIB, and setting desired outcome levels for payout requires involvement of all parties involved in the SIB (impact investors, outcome payers, lead investigator). Putting forward potential measures and current state for those measures gives them a heads start.

There is a lot of work that  still needs to be done to launch a SIB for drug repurposing. However, with the groundwork laid for the chronic pancreatitis cases, we’ve made a contribution to changing industry dynamics. I’m looking forward to seeing a future where patient outcomes and cost savings, rather than simply increased revenues, drive more of the drug research agenda. And that we see greater focus on repurposing existing drugs and treatments to address new diseases.


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Leah Jones


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