J.J. Doyle

Reimbursement Trends and Evidence Requirements for Ultra-Orphan Therapies Across Europe: Optimising Market Access in Increasingly Challenging Markets.

Total Morawski JH1, Paul A2, Spinner DS2, Doyle JJ3, Faulkner EC4, Ransom JF1 1 Quintiles Consulting, Hawthorne, NY, USA; 2 Quintiles Consulting, Durham, NC, USA; 3 Department of Epidemiology and the Department of Healthcare Policy & Management at the Mailman School of Public Health, Columbia University, NY, USA; 4 Institute for Pharmacogenomics and Individualized Therapy, University of North Carolina, Chapel Hill, NC, USA Reimbursement Trends and Evidence Requirements for Ultra-Orphan Disease Therapies Across Europe: Optimising Market Access in Increasingly Challenging Markets

Next Generation Sequencing Technology: Health Technology Assessment, Market Access Trends and Potential Impacts on The Future of Companion Diagnostic Testing.

PCN215 Next GeNeratioN SequeNCiNG teChNoloGy: health teChNoloGy aSSeSSmeNt, market aCCeSS treNdS aNd PoteNtial imPaCtS oN the Future oF ComPaNioN diaGNoStiC teStiNG Faulkner E.C.1, Spinner D.S.2, Ransom J.F.3, Paul A.4, Chawla A.S.5, Doyle J.J.6, Shaw W.H.2, Fitzgerald J.T.2 1Institute for Pharmacogenomics and Individualized Therapy, Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC, USA, 2Quintiles, Durham, NC, USA, 3Quintiles Global Consulting, Hawthorne, NY, USA, 4Quintiles Global Consulting, Durham, NC, USA, 5Quintiles Consulting, Durham, NC, USA, 6Quintiles, Hawthorne, NY, USA Objectives: Next Generation Sequencing (NGS) offers a potentially powerful platform for extremely sensitive, high-throughput, multiplex, quantitative detection of nucleic acid biomarkers. While NGS currently represents a small portion of global clinical molecular diagnostic testing, new funding and reimbursement initiatives promise to accelerate its clinical utilization. Given increasing numbers of predictive/ prognostic biomarkers but limited tissue and need for less invasive sample acuisition, NGS has the potential to transform personalized medicine (PM) and companion diagnostics. The current study characterized global NGS availability and reimbursement trends. Health technology assessments (HTAs) for NGS and other relevant multiplex/ gene panel tests were also studied for evolving evidence requirements. MethOds: Key health care provision, HTA agency, and payer websites in the EU, US, Australia and Canada were reviewed to identify NGS funding and reimbursement initiatives, and relevant HTAs. In addition, a limited number of stakeholder interviews were conducted to help further characterize the evolving global NGS landscape. Results: A number of NGS funding and reimbursement initiatives were identified, especially France, Germany, UK, US and Australia. Initiatives have been mainly centered on funding of pilot clinical utility demonstrations through research and clinical use. In Germany and US, specific initiatives are underway to develop specific NGS reimbursement codes and payment rates. A number of HTAs for NGS and other multiplex/ gene panel test platforms were identified, primarily for oncology, cardiovascular, infectious disease, inherited disease, and neuropsychiatry applications. Key HTA concerns include test clinical utility, cost-effectiveness, realworld reproducibility and equity of access given potential cost. cOnclusiOns: Payers and providers increasingly recognize NGS as enabling expanded adoption of PM approaches. As PM expands with increasing numbers of clinically actionable biomarkers, ensuring that test evidence development is aligned with expectations, and expectations with reality are key steps. Further, developing reimbursement/ funding mechanisms to support testing uptake will be critical in all markets.

PCN150 THE RELATIONSHIP BETWEEN DRUG UTILIZATION AND OUT OF POCKET EXPENSES FOR ORAL ANTINEOPLASTICS COMPARED TO A MARKET BASKET OF COMMONLY USED DRUGS

2) for the relationships between a drug’s average OPC and its TRx. RESULTS: For the antineoplastics, no drug-payer pairs showed significantly negative correlations (r 2 <-0.5) between OPC and TRx proportions. However, of the 64 possible drug-payer pairs in the market basket, 26 showed significantly negative correlations, demonstrating a much more price-elastic demand with respect to monthly OPC fluctuations. CONCLUSIONS: Antineoplastic utilization appears to be inelastic with respect to OPC fluctuations vis-a-vis the chosen market basket. This observation may be a function of the severity of disease, the lack of treatment options or both. Broader treatment options and specifically generic competition may contribute to this finding. Further research is warranted to track these relationships in a prospective, multi-factorial manner in order to better infer a cause-effect relationship. Furthermore, an analysis incorporating the proportion of written prescriptions that are filled as a function of monthly OPC fluctuations is an avenue of further research.