CWR for Cancer logo

 

Monika Odermatt
Cures Within Reach for Cancer

January 9, 2019

 

Reflections on the approach and challenges of developing social impact bonds
to fund drug repurposing clinical trials - A conversation with Dr. Rick Thompson


 

Social Impact Bonds (SIBs), also known as "pay for success financing", are a variant of social policy bonds invented by Ronnie Horesh, a New Zealand economist in the late 1980. The SIB concept has been developed in an attempt to address the paradox that investing in prevention of social and health problems saves the public sector money, however, it is extremely difficult for public entities to identify the funds to do so.

A SIB is a financial instrument that raises capital, and links financial returns to the achievement of a particular socially desirable outcome. Usually, outcomes are chosen so that improvements produce cost savings as well as social good, which helps justify the outcome payments. It allows investors to put their money into projects that are designed to benefit society. These projects are delivered by third sector organizations such as charities. They use the investors’ money to set up and run their projects and receive payments from the government based upon the project’s success. Investors are repaid from these payments. This is why SIBs are known as payment by results models.

The first SIB was launched in 2010 in the United Kingdom (UK) to finance a prisoner rehabilitation program after the Prime Minister’s Council on Social Action was asked to explore alternative models to finance social actions. As of January 1, 2019, the Brookings Global Impact Bond Database tracked 127 SIBs as either completed or in implementation. SIBs are being used for homelessness, child and family welfare, education, workforce, recidivism, and health. 


 

Cures Within Reach for Cancer is developing innovative funding models and economic incentives to enable clinical testing of repurposed generic drugs for the treatment of cancer. I had the pleasure of speaking with Dr. Rick Thompson, CEO of Findacure in the UK, in October 2018. I had the opportunity to ask him about the drug repurposing social impact bond (SIB), the challenges he has encountered, and his perspective on the future of SIBs.

I started by asking Rick how he began working with SIBs. He explained that it started because of the relationship between Nick Sireau, one of the founders of Findacure, and Dr. Bruce Bloom, CEO of Cures Within Reach in the US. Both of Nick’s sons are affected by a rare genetic disease, and he was interested in drug repurposing as a way to quickly develop a treatment for them. Repurposing of drugs already approved by regulatory agencies, especially when they have a long history of safe patient use, is an efficient way to develop new therapies due to the reduced time and cost of development, greater chance of success, and lower drug costs. However, when drugs are off-patent and inexpensive, pharmaceutical companies won’t fund the necessary clinical trials since they are unlikely to generate significant profit from selling the drugs for the new indications. Bruce had been facilitating drug repurposing research for more than 10 years and was familiar with SIBs as a potential way to finance clinical trials for repurposing of generic drugs that lack commercial interest. Nick and Bruce decided to pursue a SIB in the UK, which was very attractive due to the single healthcare payer system, the National Health Service (NHS). Rick was then hired as Scientific Officer of this project, and his work with SIBs began.

Findacure secured a grant from the Big Lottery Fund Commissioning Better Outcomes Fund to deliver a proof-of-concept study on SIBs, with the support of the CEO of NHS England. The idea behind the SIB was that it would fund multiple clinical trials testing repurposed therapies for rare diseases. Findacure, working with Numbers for Good, showed that managing rare diseases that do not have an effective therapy is costly for the NHS, and effectively treating those diseases would improve patient outcomes and also enable the NHS to avoid costs. They identified three viable repurposing projects in Wolfram syndrome, Friedreich’s ataxia, and congenital hyperinsulinism and showed that these rare diseases have a high financial burden on the NHS, costing around £13 million per year in total. Next, they identified inexpensive generic drugs that could be repurposed for these diseases, and working with clinicians designed a budget impact model, which is a comparison of the cost of current treatment for those diseases and the cost of the treatment with those three therapies, assuming they are proven effective. According to their calculations, repurposing generic drugs to treat these conditions could save the NHS £2.3 million. This could be sufficient to pay back the cost of the necessary clinical trials.

There are still details to work out and NHS has not yet agreed to be the success payer for the drug repurposing SIB, so it is still in the concept phase. Findacure, Cures Within Reach, and Mission: Cure are actively exploring ways to move it forward in order to bring new treatments to patients who desperately need them.

I was really looking forward to hearing from Rick about what he sees as the next steps. According to Rick, the following ingredients are key to a successful SIB: flexibility, imagination, and funding. There is a need for flexibility in order to have an open mind and learn from the experiences of other organizations who have worked with SIBs before; imagination in order to overcome hurdles one encounters when trying to implement a SIB, and to find ways to collaborate with other countries and partners; and impact investors to fund the SIB and trials. There is also a need for broad recognition of the importance of repurposing for rare and common conditions, understanding how this different approach to funding drug development could work, and freedom to take new approaches to improving outcomes for patients.

He also emphasized that it is important to focus on the patient impact possible through the SIB model, instead of solely the cost-savings element. This is because the NHS’s main goal is to improve health outcomes with its approved budget. The agency does not act based on savings alone; interventions should produce overall savings within the entire agency budget. Spending less money to treat patients with a specific condition would serve to increase the efficiency of NHS spending because they could use those funds for expenses that would not otherwise be covered, but this would not necessarily generate excess cash with which to make success payments.

I asked Rick what he thinks about multiple countries working together, for example, to create a global SIB, as opposed to SIBs being developed within one country. He emphasized that whichever approach is used, collaboration is always key. A joint effort across countries would result in an increase in market size in terms of more patients to enroll in the clinical trials if they are run in multiple countries, and also increased returns when the treatments are incorporated into medical practice and improve patient outcomes. He also mentioned that collaborations between multiple countries could lead to engaging more generic drug manufacturers in SIBs and clinical trials. Generic drug manufacturers could potentially provide initial investment in the SIBs or support the clinical trials, such as by supplying the drugs.

Crucial decisions when developing a SIB are deciding which drugs, diseases, and outcomes to include. Findacure and Cures Within Reach developed a series of metrics to assess the existing evidence and economic impact for repurposing projects. Their approach was to identify promising projects currently in development for specific diseases. Careful consideration must be used when choosing the relevant outcomes to measure in the SIB, since they should reflect direct patient impact.

According to Rick, near-term outcomes such as decreased hospitalizations within the first year after starting a new treatment might be easiest to implement since they are easier to track and measure. This would simplify the outcome tracking process for healthcare systems that work with annual cycles. However, if one is pursuing a cause-based route rather than a savings-based route, this could open the door to leveraging long-term savings and a more collaborative approach, where one can also look at savings in terms of government payments to people who are ill, unemployed, impoverished, or who have children.

Finally, I asked Rick if he thought it would be better for SIBs to focus on treatments for rare or common diseases, and if a SIB should include multiple trials for one disease or trials for multiple diseases. He explained that if you are looking at it from a scale perspective, there is a larger pool of patients for trials and SIB return payments for common diseases. However, common diseases may be more complicated since there are existing models for delivering treatments. In his experience, the best approach is to focus on a condition that is struggling for attention, where current models are failing, and where there is a lack of drug development.

Rick mentioned that Findacure’s original approach was to include in the SIB clinical trials testing therapies for multiple diseases. With Cures Within Reach and Mission: Cure, they are now considering a focus on multiple trials/therapies for a single disease. There are pros and cons for both approaches. A SIB including trials for different diseases is technically more challenging because one needs both deep health economic expertise and scientific understanding. Different patient populations and outcomes will need to be tracked in order to assess the results of the trials in terms of outcomes and cost savings. On the other hand, if one is pursuing different treatments for a single disease, it is easier to determine and track the outcomes.

I would like to thank Rick for sharing his knowledge and experiences. It is very important that we continue to work in a collaborative environment to overcome the challenges and realize the potential of SIBs to improve outcomes for patients. In Rick’s own words - collaboration is key - and I couldn't agree more.


 

About Findacure
Findacure is a UK-based charity building the rare disease community to drive research and develop treatments. Findacure works to empower patient groups to meet the challenges of their conditions and to promote collaboration between rare disease stakeholders to facilitate treatment development for all. The charity is setting up a social impact bond for rare disease drug repurposing to provide a platform to fund promising projects identified by patient groups, clinicians, and researchers. More information at https://www.findacure.org.uk/

 

About Cures Within Reach
Cures Within Reach, based in Chicago in the US, works to catalyze repurposing research for all diseases to quickly and affordably improve patient lives. This is accomplished through collaborations that connect researchers and funding, by facilitating crowdsourcing and community, and by identifying alternative financing models and incentives. More information at http://www.cureswithinreach.org

 

About Mission: Cure
Mission: Cure, an organization fiscally sponsored by Cures Within Reach, aims to demonstrate a new model for curing disease using financing based on patient outcomes. The first disease it is tackling is pancreatitis, a costly and debilitating disease with no effective treatment. More information at https://mission-cure.org/

 

About Cures Within Reach for Cancer
Cures Within Reach for Cancer, a Boston-based initiative sponsored by Cures Within Reach, is developing new cancer treatments using inexpensive drugs that are already FDA-approved for other indications and have a long history of safe patient use. Their main objectives are to identify the therapies with the strongest evidence of effectiveness for cancer, facilitate definitive clinical trials, and create sustainable funding models. More information at http://www.cureswithinreach.org/cancer

Click here to sign up for our eNewsletter today!

Please include your Full Name* and Email Address*

*required

If the link above doesn't work, please email susan@cureswithinreach.org with your Full Name and Email Address to sign up!

 

Cures Within Reach

134 N. LaSalle, #1130
Chicago, IL 60602

info@cureswithinreach.org

All funds donated to Cures Within Reach may be tax deductible for federal income tax purposes. 

Partnership for Cures DBA Cures Within Reach is recognized by the IRS as a 501(c)3 tax-exempt not-for-profit organization, tax ID 20-3620169.

 

Tuesday the 23rd. Copyright 2018 Cures Within Reach.