FasterCures: Patient-centric R&D
Despite decades of innovation, there are growing appeals for medical research to be more patient-centric. This calls for purpose-led organizations with resilient leaders who are able to challenge the status quo
When randomised trials entered the medical research system in the 1940s, they revolutionised the industry. For the first time, outcomes could be confidently ascribed to the drug or therapy itself, rather than other variables.
Today, drug development faces new challenges. High prices for some therapies, and a sense that patients are passive consumers rather than active participants, are key issues facing the industry. Could patients play a bigger part in research, product development and marketing? Such searching questions are being asked by FasterCures, a Washington DC-based not-for-profit group.
According to its executive director, Margaret Anderson, FasterCures is driven by a singular goal: to save lives by speeding up and improving the medical research system. People with multiple sclerosis might value mobility over reduced pain, for instance. Those with Crohn’s disease can share insights into their experience and diagnosis, which can inform drug development pathways. Patients also can provide feedback about the logistics of clinical trial participation such as timing and childcare.
“The whole healthcare system is poised for disruption,” Ms Anderson says. “I wouldn’t have said that 10 years ago.” Resilient leadership is needed to push for change in a complex industry. In the early days, the team faced scepticism from many who were not ready for a close-up examination of the system. That is part of the natural progression for any disruptive organisation, says Lisa Simms Booth, director of external affairs.
A clinical trial
Drug development is expensive and risky. Billions are invested to develop new medicines and therapies. But the process should be transparent and consultative, and the final products more patient-friendly, in terms of price, mode and accessibility.
In the past, the pharmaceutical industry faced criticism from patients’ associations, doctors’ groups and governments for being too insular. Failure to share clinical trial data, for example, has been a common critique. So has a lack of transparency in drug pricing. Over the last decade, this has started to change. Though far from perfect, there is greater transparency over clinical trial data today than ever before, for instance.
FasterCures’ own history, dating back 13 years (it was founded by a think tank) runs parallel to this shift in perspective. Ensuring that patient feedback is more deeply integrated into drug development requires strong leadership and new ideas, and Ms Anderson’s organisation is striving to provide it. The organisation finds that its leaders must forcefully promote change in a highly complex and regulated industry while appealing to the varied needs of diverse stakeholders, including policymakers, not-for-profit healthcare groups, insurance firms, biotech firms and big pharma companies.
Their approach to achieving this is to hold partners accountable for their promises, and ask tough questions. “We have a reputation for saying what needs to be said without shying away from thorny areas,” says Ms Anderson. This includes calling out companies and regulators who fail to incorporate patient feedback in their decision-making, and challenging them to do better. “That’s what collaboration is all about.”
But not-for-profit groups must be selective about how they deploy their resources, which means leaders should be thoughtful about where to focus. “One of the most important ingredients in our leadership approach is being purposeful in the projects we pursue, then following through on our promises so that we don’t squander the goodwill of our partners,” says Kim McCleary, managing director of FasterCures.
That means learning from failures. Several years ago, FasterCures launched a programme called “Patients Helping Doctors,” which advocated for patients to work with their medical providers to get involved in research. But its leaders learned that patients having a voice with doctors wasn’t enough, Ms Anderson says. “We realised that patients need to be partners throughout the whole research and development process. Treatments should be developed in concert with patients.”
As a result, the group created a “Benefit Risk Bootcamp” in 2014 to enhance patient-centred decision-making about medical products in development, under regulatory review and on the market. The output helped build the “Patients Count: The Science of Patient Input” programme the following year, which provides tools and training to industry professionals on how to engage patients in research; and a Benefit-Risk Advisory Council of industry experts from academia, government, not-for-profit groups and pharmaceuticals, who offer advice on how to shape product development and influence regulatory decisions. The council’s feedback has since been adopted by various stakeholders, including the US Food and Drug Administration, industry groups and patient organisations, and its advice was reflected in the ‘21st Century Cures’ legislation introduced in the US Congress in 2015.
The value of credibility
The group’s not-for-profit status may have given it an advantage as a disrupter in its business. “What we were saying started to resonate with people,” Ms Booth says. “People trust us and it has helped us create alignment between stakeholder groups.”
This gave FasterCures the credibility to create its own framework, in partnership with healthcare consulting firm Avalere, to assess the value of new medical treatments using patient perspectives. Such ‘value frameworks’ calculate the health benefit and cost-effectiveness of a new therapy or drug, and are designed to better inform pricing and policy decisions, as well as patient choices.
“The recent proliferation of value frameworks has taken the conversation about value to a new level, but the perspective of patients is still largely absent,” Ms Anderson says. She believes a collaborative effort will help bring that patient voice to the forefront. “We want to see things move faster and more efficiently, and that requires more conversations between all stakeholders, including patients.”
FasterCures is now seeking to use that framework with new partners. Ms Anderson says that a patient focus has grown to be more universally accepted: “After 12 years in this role, I feel like I have the credibility to have a candid dialogue about what needs to change. It takes focus, and candour, and data, and ideas.”
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