The role of patient organisations in research and development: Evidence from rare diseases.

Patient organisations play an increasingly crucial role in the pharmaceutical sector, yet their impact on innovation remains unexplored. We estimate the impact of patient organisations on R&D activity in the context of rare diseases in Europe using a proprietary dataset that maps clinical trials from discovery to phase III across 29 countries, 1893 indications, and 30 years (1990-2019). By applying difference-in-differences and event study methodologies to a panel of 1,646,910 unique R&D observations, we find that country-indication pairs with at least one operating patient organisation have a higher rate of R&D activity compared to those without, with stronger effect in more prevalent rare diseases compared to ultra-rare conditions. We observe a lag in effects from patient organisation introduction, suggesting it takes approximately five years for these organisations to affect R&D activity. Overall, our work suggests that patient organisations play an important role in steering R&D efforts in rare diseases. Further research is needed to better understand mechanisms driving this effect and the potential impact of patient organisations on existing health inequities.

Algorithms and heuristics of health technology assessments: A retrospective analysis of factors associated with HTA outcomes for new drugs across seven OECD countries.

CONTEXT: Positive health technology assessment (HTA) outcomes can have important implications for equity, efficiency and timely patient access to novel therapies. Several outcomes and dimensions of benefit beyond utility feed into HTA processes. OBJECTIVE: We analyse a proprietary dataset of HTA outcomes in 7 countries, to (a) test whether HTA decision-making is grounded in welfarist or extra-welfarist approaches; and (b) empirically determine the factors associated with positive HTA outcomes, the time to achieve these and establish the magnitude of inter-country differences in assessment processes. METHODS: Data were extracted from publicly available HTA reports on drugs that received marketing authorisation between 2009 and 2018 (N = 1415). The outcomes of interest were the probability of positive HTA outcomes and the time-to-HTA outcome; these were examined with respect to clinical, regulatory, product- and disease-related, evidence uncertainty and contextual variables. Econometric models utilising survival analysis and multinomial logistic regression were specified. FINDINGS: Positive HTA outcomes accounted for 87.3% of the sample (n = 1235), of which 71% (n = 1004) were restricted. Drugs with positive HTA outcomes were subject to clinical restrictions (n = 652, 46%), financial risk-sharing (n = 439, 31%) or had been rejected at least once (n = 282, 20%). Significant predictors of positive HTA outcomes were orphan drugs with cancer indications, high quality of evidence linked to clinical and economic evidence uncertainties which had been overcome, and contextual considerations, particularly innovativeness and unmet need. Comparative analyses revealed systematic differences between countries in their propensity to accept the same drugs, particularly oncology and orphan drugs. CONCLUSIONS: Our results are contextual and reinforce arguments in favour of explicitly accounting for social value judgements, establishing separate assessment frameworks for highly uncertain products, adopting risk mitigation strategies for novel therapies with early phase evidence, and sharing of HTA practices across settings. Lastly, HTA agencies have adopted an extra-welfarist approach to value assessment and resource allocation.

Industry funding of patient organisations in the UK: a retrospective study of commercial determinants, funding concentration and disease prevalence.

OBJECTIVES: To assess the relationship between UK-based patient organisation funding and companies' commercial interests in rare and non-rare diseases in 2020. DESIGN: Retrospective analysis of the value and volume of payments from pharmaceutical companies to patient organisations in the UK matched with data on the conditions supported by patient organisations and drugs in companies' approved portfolios and research and development pipelines. SETTING: UK. PARTICIPANTS: 74 pharmaceutical companies making payments to 341 UK-based patient organisations. MAIN OUTCOME MEASURES: Alignment between the commercial interests of pharmaceutical companies and the disease area focus of patient organisations; difference in the volume and value of payments to patient organisations broken down by prevalence of conditions; industry funding concentration, measured as the number of companies funding each patient organisation, the share of overall industry funding coming from each contributing company and the share of industry funding of each organisation comprised by the single highest payments. RESULTS: 1422 payments were made by 74 companies to 341 patient organisations. Almost all funds (90%) from pharmaceutical companies were directed to patient organisations that are aligned with companies' approved drug portfolios and research and development pipelines. Despite rare diseases affecting less than 5% of the UK population, more than 20% of all payments were directed to patient organisations which target such conditions. Patient organisations focusing on rare diseases relied on payments from fewer companies (p value=0.0031) compared to organisations focusing on non-rare diseases. CONCLUSIONS: Companies predominantly funded patient organisations operating in therapeutic areas relevant to companies' portfolio or drug development pipeline. Patient organisations focusing on rare diseases received more funding relative to the number of patients affected by these conditions and relied more heavily on payments from fewer companies compared to organisations targeting non-rare diseases. Increased independence of patient organisations could help avoid conflicts of interest.