A leading pharmaceutical company with a novel disease-modifying product was seeking Government subsidy and wanted to better communicate the broader value of the drug, beyond the clinical measures captured through the clinical trial.
Advances in medical science have led to the development of new treatments which enhance patient outcomes in combination with broader societal impacts, such as productivity and family relationships. Traditional approaches to assess the value of new healthcare technologies are often limited to direct financial implications, such as reductions in hospital resource use or changes in medication use. Typical economic analyses, including cost-effectiveness analyses (CEA) or cost-benefit analyses (CBA) fail to capture the broader societal impacts of transformative treatments.
Recently, there has been a call for a fundamental shift in the Government health technology assessment process to capture the entirety of benefits provided by new and novel technologies, including the social, clinical, and economic impacts on patients, their families, and other groups or organisations in society. Achieving subsidised access to new medical technology is a time-consuming and complex process. For pharmaceuticals, the time between submission to the therapeutic goods administration (TGA) for regulatory approval, which makes a drug available to Australian patients, and PBS listing, which grants subsidy via the public healthcare system, is at least 14 months if successful upon first application, but some medicines may take several attempts to achieve a successful listing. At times, medicines that received a positive recommendation from the Pharmaceutical Benefits Advisory Committee (PBAC) are still not freely available for patients because of unsuccessful negotiations between pharmaceutical companies and the Government in reaching a mutually agreeable price.
HTANALYSTS chose SROI as the most rigorous methodology to assess and communicate the broader value of PBS funding of this innovative disease-modifying high impact pharmaceutical.
An SROI approach was chosen to capture those broader impacts, relying heavily on the input of key stakeholders to identify and understand the importance of observed changes, consider how to estimate change, and how to value the outcomes. Given the vast quality of life and productivity implications of improved organ function of the treated patients, and the prevention of steep organ function decline over time, an SROI analysis was selected to reveal the full picture of value created.
In head-to-head randomised trials, the assessed drug showed significant improvement compared to what is currently publicly funded in Australia. Also, since the drug has been available in the United States for a number of months, patients have since then posted testimonials online describing drastic improvements in their organ function and most importantly their quality of life. For the purpose of the SROI, once outcomes were defined, the next step entailed finding evidence to confirm that each outcome would occur through these sources and to measure how much change would be created. Published literature and patient research provided evidence for the baseline experience of stakeholders in a world without this medicine. Assessments of the impact of the new drug were made based on stakeholder consultations and secondary research including news articles and patient testimonials. All of the assumptions were developed in parallel with a traditional CEA for Government assessment via the PBAC, in order to provide the Government with a traditional and alternate valuation.
These powerful outcomes were used by HTANALYSTS together with stakeholder consultations to develop a theory of change, mapping the sequence of events resulting in impact for a stakeholder group.
High impact pharmaceutical products often struggle to demonstrate their true value using traditional valuations or methodologies, particularly in the rare diseases space. The novel SROI analysis showed that a substantial proportion of the value of the new medicine was generated through patients living with the disease and their family members, which is often ignored in traditional decision-making processes. In particular, patients not only improved their health, but also had increased hope for the future and increased fulfilment as well as improved time with their family. Families were able to reduce their uncertainty about the future, increase their income and savings and take better care of their own health. Value also extended beyond the family unit to healthcare providers, schools, employers as well as Government. Overall, the analysis showed that every dollar invested in the drug would be returned over a modest timeframe.