N. Horowicz-Mehler

The Case For Early Payer Engagement.

Global healthcare expenditure is increasing [1], creating budget pressures and challenging market access for an increasing number of drugs [2]. For instance, from 1998-2008, NICE granted restricted or no access to ~60% of drugs from the top 10 Pharma and since 2004, IQWiG classified 70% of reviewed drugs as “benefit not proven” [3]. Payers are often bewildered by decisions made by pharmaceutical manufacturers when designing their clinical trials, choosing their comparators, or making certain safety and efficacy claims with mismatched evidence. This misalignment may be the result of clinical development plans focused on regulatory compliance that do not fully consider the real-world patient populations that reflect Payer’s chief concerns. Not engaging Payers as early as possible during the asset development process could result in delay of approval and/or rejection of reimbursement. Knowledge of payer priorities and the level of evidence they seek are crucial to characterization of real value of assets and competitive differentiation. The objective of this study was to illustrate the case for early payer engagement to ensure optimal patient access to differentiated assets. We here present the relative merits of various approaches to engage with Payers as well as case studies exemplifying the positive impact of early engagement.

FRI0443 Impact of etanercept therapy on disease activity and health-related quality of life of a moderate rheumatoid arthritis patient population from a national british observational cohort

Background Prior work on real-world burden of moderate RA patients highlighted low health-related quality of life (HRQOL) and high disease burden at time of biologic therapy initiation [1]. The impact of biologics on clinical and patient-reported measures in moderate patients remains unclear. Objectives To assess change over time in disease status, HRQOL, disease-modifying antirheumatic drug (DMARD) treatment and adverse events (AEs) in etanercept (ETN)-treated patients. Methods A subset of patients aged ≥18 years with moderate RA (3.2<DAS28≤5.1) recruited to a UK longitudinal cohort study 2001-2009 comparing a non-biologic treated group (nBG) exposed to at least one traditional DMARD to a biologic group (BG) treated with ETN, were analyzed. Anonymous data were provided by the British Society for Rheumatology Biologics Register. HRQOL was assessed by the Health Assessment Questionnaire (HAQ) disability index score and SF-36 [physical component score (PCS), mental component score (MCS)]. Groups were matched on factors predisposing to progression (i.e. age, gender, disease duration, baseline HAQ and DAS28, previous DMARDs). Change from baseline to 6 months, and disease remission and progression were assessed by multivariate regression. Results 1754 patients were assessed (211 BG/1543 nBG). BG compared to nBG had 1) a greater reduction in DAS28 and HAQ, 2) disease remission occurring more often (OR=2.7, CI:1.3-5.5, p=0.006) and 3) disease progression occurring in fewer patients (OR=0.3, CI:0.2-0.7, p=0.002). BG were more likely to be “good responders” (OR=2.4, CI:1.4-4.1, p=002). There were no significant differences between groups in PCS/MCS, DMARD therapy changes and time to DMARD discontinuation. BG had a higher overall incidence of “other serious infection” (not bone/joint infection, septicaemia) and “other CNS-related events” (not optic neuritis and peripheral neuropathy); with no significant differences in associated hospitalisation rates or deaths. Table 1 Parameters nBG (n***/N%) BG (n/N%) (N=1543) (N=211) Disease remission (DAS28 <2.6) 84 (609/13.8%) 41 (171/24%)* Disease progression (DAS28 >5.1) 112 (609/18.4%) 20 (171/11.7%)* Change in DAS28 total score from baseline 0.08±0.39 (39.47%) –0.63±0.41 (81.04%)** Change in HAQ disability index score from baseline 0.08±0.09 (63.90%) –0.14±0.10 (64.45%)** EULAR: Good Responders 142 (609/23.3%) 63 (171/36.8%)** Overall Incidence of AEs 101 (16.9%) 22 (11.5%) Occurrence of other CNS related AEs 4 (0.3%) 3 (1.6%)* Occurrence of other serious infection 13 (0.9%) 6 (3.1%)* *p<0.01, **p<0.001 comparing nBG to BG, ***reflective of available baseline and 6 months data. Conclusions BG had significantly reduced disease activity and better HRQOL after 6 months compared to matched nBG (the possibility of unmeasured confounding remains). BG were more likely to be “good responders” and have disease remission, but less likely to progress. The ETN group reported higher incidences of “other serious infection” and “other CNS-related events”, neither leading to higher hospitalisation rates. References Hyrich K, Deighton C. et al. Benefit of anti-TNF therapy in RA patients with moderate disease activity. Rheumatology 2009;48:1323–1327. Disclosure of Interest S. Kotak Shareholder of: Pfizer, Employee of: Pfizer, J. Mardekian Shareholder of: Pfizer, Employee of: Pfizer, N. Horowicz-Mehler Consultant for: Pfizer, A. Shah Consultant for: Pfizer, A. Burgess Consultant for: Pfizer, J. Kim Consultant for: Pfizer, E. Gemmen Consultant for: Pfizer, H. Boyd Employee of: Pfizer, A. Koenig Shareholder of: Pfizer, Employee of: Pfizer

How the Biopharmaceutical Industry Can Navigate a Healthcare Ecosystem Increasingly Driven by Demands for Comparative Effectiveness Research

While the approval of biopharmaceuticals hinges on a demonstration of safety and efficacy through randomized clinical trials, other stakeholders have historically evaluated the approved product based on repackaged clinical trial data with limited ‘real-world’ validity. The US healthcare system is now evolving into a more complex ‘ecosystem’ where regulatory approval is just a first step toward successful market access. Indeed, the adoption and financial success of a product relies on stakeholder preference modulated by environmental and competitive factors, such as the clinical burden of a disease or the availability of cheaper alternative therapies. One market challenge faced by the biopharmaceutical industry is that of measuring product performance under the imperfect research conditions of the real world, replete with its confounders and effectmodifiers. This has led to discordance in the healthcare ecosystem; regulators have the data they need but other stakeholders are left with blind spots when making decisions. Comparative effectiveness research (CER) has the potential to fill these data gaps, helping to ensure optimalmarket access for innovative products. This article examines how companies can meet increasing demands for CER using a three-step planning approach involving CER diagnosis, CER prognosis and CER prescription. The approach assesses (i) the likelihood of a drug or device being included in a CERstudy, the parameters of such a study (e.g. comparators, endpoints, patient population) and the groups likely to conduct and fund the research; (ii) the potential for the drug or device to receive a positive evaluation as well as the conditions that can optimize the evaluation; and (iii) the activities to be undertaken throughout the clinical development process to best position a drug or device for CER. If initiated prior to approval, these steps will ensure that the data developed for each intervention is relevant to the web of healthcare stakeholders that will determine its success in the marketplace.