VeraSci’s VP of Regulatory Strategy and Market Access Innovation and former AIFA Director, Dr. Luca Pani, participated in the Boston Consulting Group (BCG) Market Access Roundtable and recently published a paper discussing the variables and challenges involved in the assessment of drug value.
After decades of decline, the industry pipeline of approved products has rebounded1 over the last five years and in 2018, 59 new drugs were approved by FDA, the highest number in history. This includes 19 (32%) novel drugs approved as first in class. More than half of the new drugs (34 out of 59) were approved to treat rare or orphan disease.2 These innovative drugs enter into constrained healthcare systems that struggle to cope with demographic change and complex incentive and payment systems. While patients demand fast access to these new treatment options, payers are forced to make trade-offs in allocating their constrained budgets.
There is broad consensus that a focus on “value” is the appropriate approach for optimizing spend of constrained healthcare resources. By focusing on value, payers aim to optimally and equitably allocate constrained resources to areas of greatest need and to treatments with greatest impact on patients and healthcare systems. However, value assessments are not only about the equitable allocation of resources and funding, but also need to cover the creation of appropriate rewards to spur innovation.
Virtual Reality (VR) approaches have had considerable success in measurement of functional capacity. However, it is not clear if factors other than cognitive impairment influence performance on VR measures. Many people with schizophrenia have significant negative symptoms and they could reduce engagement in assessment. 158 patients with schizophrenia performed the VRFCAT, were tested with the MCCB, were rated with the PANSS, and were rated on everyday functioning. Scores for reduced emotional experience and reduced expression were derived. Reduced emotional experience, but not reduced expression, was correlated with socially relevant VRFCAT subtasks and real-world social functioning. Performance on the socially relevant subtasks, but not the solitary subtasks, shared variance with work outcomes. MCCB performance was associated with both subdomains, but socially relevant subtasks shared more variance. Patients with higher reduced emotional experience validly engaged in socially relevant VR simulations, as indexed by correlations with outcome measures. These patients had poorer performance on socially relevant tasks than on solitary tasks. The differential validity of solitary vs. socially relevant simulations was supported by differences in correlates, suggesting that assessments with a focus on performance of simulated socially relevant tasks could be developed.
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Classical analyses of pharmaceutical pipelines from 1990 to 2010 reported increasing attrition rates and duration of clinical trials, leading to a perception of a “productivity crisis” in this sector. However, in contrast, a more recent analysis of over 45,000 projects in the last decade completed by VeraSci’s Dr. Luca Pani and colleagues showed an upsurge in R&D Productivity. This result is largely driven by two factors: company efficiency – in particular aborting studies in early phases – and concentration on high yield projects. The number of new advanced therapy projects are at an all-time high and time to market from initial IND is at an all-time low. This combination will put additional pressure on regulators and payers world-wide. It is therefore essential that sponsors are fully prepared to interact with regulators and payers efficiently and productively. VeraSci’s Regulatory Strategy and Market Access Innovation team led by Dr. Pani can greatly facilitate those interactions.
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