Pharma Advances recently published “Expert opinion on Real-World Evidence (RWE) in drug development and usage” co-authored by Dr. Luca Pani, VP Regulatory Strategy and Market Access Innovation at VeraSci. This article presents the Italian Society of Pharmacology’s expert opinion on RWE as a technical-scientific reference for institutions, the scientific community, health care professionals, and pharmaceutical companies.

Randomized clinical trials are the gold standard for assessing the safety and efficacy of treatments. Still, they come with limitations that make their results difficult to generalize to the entire treatment population. RWE, information collected through routine clinical practice, offers opportunities in both the pre-marketing and post-marketing phases to provide valuable information. In the pre-marketing phase, RWE can provide insight into disease burden and unmet medical needs. In the post marketing phase, RWE can contribute to comparative effectiveness research, drug safety information, and more. RWE also has the potential to play an important role in innovative pricing and reimbursement models. Read the full article here.

Learn more about VeraSci’s HEOR capabilities

The European Review for Medical and Pharmacological Sciences recently published “Impact of reimbursement limits on patient access to direct-acting antivirals in Italy: analysis of data from national registries” co-authored by Dr. Luca Pani, VP Regulatory Strategy and Market Access Innovation at VeraSci. The study looked at patients in Italy with Hepatitis C treated with Direct Antiviral Agents (DAA) when Dr. Pani was the Director General of the Italian Medicines Agency (2011-2016) and after that (2017-2018).

The World Health Organization has called to eliminate Hepatitis C based on the efficacy and safety of DAA therapy. As a result, Italy adjusted its reimbursement practice in 2017. From 2015 until the change in 2017, DAA therapy was reimbursed only for patients with advanced Hepatitis C. This changed in 2017 when universal access to DAA therapy was granted. The study examined patient recruitment trends with and without limitations to access to DAA therapy. The study found that removing limits on reimbursement impacted the types of patients treated but did not significantly change progress towards disease elimination highiligting the importance of including the ability of modern healthcare systems to deploy innovative medications to patients in need. Read the full article here.

Learn more about VeraSci’s HEOR capabilities

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.

Abstract
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.

Article is no longer available. Please contact VeraSci for more information.

Computer-based virtual reality assessments of functional capacity have shown promise as a reliable and valid way to assess individuals with multi-episode schizophrenia. However, there has been little research utilizing this innovative approach with young patients who are in the early phase of schizophrenia.

VeraSci’s proprietary Virtual Reality Functional Capacity Assessment Tool (VRFCAT) integrates virtual reality into the assessment of functioning in clinical trials. The VRFCAT is administered on a computer or our tablet-based platform, Pathway, and simulates key instrumental activities of daily living (iADLS) in a realistic and interactive virtual environment. With demonstrated sensitivity to basic functional capacity deficits, the VRFCAT was developed to improve clinical trials by detecting functionally meaningful improvements in patients’ everyday lives. The VRFCAT has numerous advantages over conventional assessments, and meets the highest psychometric and regulatory standards, with strong support from industry sponsors, NIH and FDA as a functional co-primary outcome measure.

A recently published manuscript by Ventura et al., including as a co-author VeraSci’s CEO and Co-founder Dr. Richard Keefe, extended previous findings to patients with first-episode schizophrenia. Virtual-reality–based performance was correlated with a standard test of functional capacity, indicating VRFCAT validity. Furthermore, correlations with cognitive functioning and occupational/school and social functioning indicate promise as a coprimary measure to track changes in response to treatment.

CLICK HERE to read the full manuscript: Virtual reality assessment of functional capacity in the early course of schizophrenia: Associations with cognitive performance and daily functioning by
Joseph Ventura, Tamara Welikson, Arielle Ered, Kenneth L. Subotnik, Richard S. E. Keefe, Gerhard S. Hellemann, Keith H. Nuechterlein. Early Intervention in Psychiatry. 2019;1–9.