VIVEK VIKRAM SINGH, SUBJECT MATTER EXPERT, RESEARCH & ANALYTICS CAN COMPETITIVE INTELLIGENCE STRATEGIES HELP IN ORPHAN DRUG DEVELOPMENT?

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1 VIVEK VIKRAM SINGH, SUBJECT MATTER EXPERT, RESEARCH & ANALYTICS CAN COMPETITIVE INTELLIGENCE STRATEGIES HELP IN ORPHAN DRUG DEVELOPMENT? wns

2 wns CAN COMPETITIVE INTELLIGENCE STRATEGIES HELP IN ORPHAN DRUG DEVELOPMENT? VIVEK VIKRAM SINGH, SUBJECT MATTER EXPERT, RESEARCH & ANALYTICS Introduction They are serious, chronically and progressively disabling, and can be life-limiting and life-threatening. According to the U.S. National Organization for Rare Disorders 1 (NORD), there are close to 7,000 2 notified rare diseases. Although there is no consensus on the definition of rare disease, many geographies define it predominantly on the basis of their prevalence. Others classify them based on the severity of the disease and the availability of alternate options for treatment. Pharmaceutical products aimed at treating rare diseases or disorders are called orphan drugs. The Orphan Drug Act of 1983 was the first U.S. initiative to facilitate and incentivize the development of 3 drugs to treat rare diseases. The success of the Orphan Drug Act of 1983 in the U.S. led to its adoption in other key markets. Notable among them were Japan (Article 77-2 of the Pharmaceutical 4 Affairs Law-1993) and the European Union (the EU Regulation on Orphan Medicinal Products ). The key features of the orphan drug regulatory framework in the USA, EU and Japan are summarized as shown in Table COPYRIGHT 2017 WNS GLOBAL SERVICES WNS.COM

3 INSIGHTS Table 1: Orphan Drug Regulatory Framework and Incentives U.S. (ODA 1983) EU (Regulation [EC] No 141/2000) Japan (Article 77-2 of PAL 1993) Authorities Involved Food and Drug Administration (FDA) European Medicines Agency (EMA) Ministry of Health, Labor and Welfare (MHLW) Panels Involved in Designation FDA OOPD nema-comp nec nmhlw npafsc npmda Prevalence Criteria <200,000 patients <5 in 10,000 patients <50,000 patients Review Period Typically 90 days Maximum 90 days procedure None specified Incentives Grants Orphan Products Grant Program and NIH s Rare Diseases Grants European Commission (EC) through its Framework Program (FP6) National Institute of Biomedical Innovation (NIBIO) Financial Incentives ntax credit up to 50% of qualified clinical development costs (U.S. studies) nwaiver of FDA fees for application review nno general tax credit nno specific subsidy for clinical trials nregulatory fee reductions - specifically for Subject Matter Experts (SMEs) nfinancial subsidies for up to 50% of expenses for clinical and non-clinical research nuser fee waivers n15% tax credits nup to 20% reduction in corporate tax n30% reduction in marketing application fees Market Exclusivity 7 years 10 years Re-examination period extended to 10 years at marketing authorization Scientific Advice (Protocol Assistance) Access to free scientific guidance at the FDA Access to free protocol assistance at the EMA for SMEs 30% fee reduction for protocol assistance Regulatory Tools for Accelerating Approval nfast-track approval nbreakthrough designation naccelerated approval pathway npriority review designation npriority medicines (PRIME) designation nconditional approval or under exceptional circumstances naccelerated assessment npriority review nfast-track approval COPYRIGHT 2017 WNS GLOBAL SERVICES WNS.COM 02

4 Till date the U.S. Food and Drug Administration (FDA) has approved 628 orphan drugs and biological products for rare diseases. When compared to fewer than 10 products that were approved in the 6 decade before the Act was passed, the efficacy and success of the Orphan Drug Act (1983) becomes evident. The European Commission has authorized 137 orphan medicines to benefit patients suffering from rare diseases since 7 the inception of their regulation. The increase in the number of Orphan Drug Designation (ODD) granted in the last five years, both in the U.S. and EU indicates the increasing interest of the pharma industry in developing orphan drugs (Figure 1). Figure 1: U.S. and EU Orphan Designations Granted Every Year ( ) Orphan Designations (per year) Strong increase since last 5 years USA EU Sources: FDA Application: Orphanet (European portal for rare diseases and orphan drugs): Pharma Interest in Orphan Drugs The development of orphan drugs opens the door for potential commercial opportunities for pharma companies. A sluggish prescription drug market in both U.S. and Europe regions makes orphan drug development an attractive proposition. Driven by increasing regulatory challenges and high costs of getting a massmarket drug approved, the pharma industry has been moving from blockbuster models to nichebuster opportunities. Besides financial incentives (that include tax incentive for certain Research and Development, and clinical development costs), waiver of regulatory application fees and extended market exclusivity through monopoly protection, there are a few more factors that add to COPYRIGHT 2017 WNS GLOBAL SERVICES WNS.COM

5 INSIGHTS the commercial appeal for orphan drug development. Legal frameworks provide a fast-tracked regulatory review and approval process for orphan drugs. Low manufacturing costs, scientific breakthroughs and stronger advocacy through increased coordination between doctors and patient groups have also helped. The orphan drug market has thus become a profitable opportunity for companies who are willing to invest in the rare disease space. According to a recent report by EvaluatePharma, the worldwide sales of orphan drugs is expected to touch USD 209 Billion by This is a doubling of sales between 2016 and 2022, with a CAGR of percent for the period Notably, the report says orphan drugs are expected to touch 21.4 percent of worldwide prescription sales by 2022 (excluding generics). Unique Challenges in Orphan Drug Development Increased licensing for the development of orphan drugs is opening the gates to greater competition and changing the market dynamics. The orphan drug market is now beginning to show trends seen in traditional pharma markets. Additionally, the development of orphan drugs is faced with the following challenges that are unique to rare diseases: n Huge heterogeneity in disease pathophysiology, and poorly understood natural histories of disease progression n Geographic dispersion of patients that lead to their limited availability for clinical trials n Lack of previous clinical studies to establish a template for study execution (this leads to uncertainties in applying appropriate end points and duration for treatment) n Heterogeneity in treatment effects, and complexity of statistical analysis associated with limited sample sizes 8 COPYRIGHT 2017 WNS GLOBAL SERVICES WNS.COM 04

6 n Lack of knowledge in appropriate biomarkers to predict outcomes n Limited number of experienced clinical investigators worldwide n Absence of regulatory precedents and harmonization of the approval process between different national agencies n Unclear post-market commitments that impact longterm risk-benefit studies n Changing payer demands in rare disease cases where multiple treatment options are now available any internal bias, effectively aggregate them to connect the dots and have a global view of the competitive landscape to identify opportunities and threats. CI can play a significant role in objectively providing better drug development strategies. By analyzing the past and modeling the future it can: n Provide trends, and anticipated responses of regulatory authorities and payers to outcomes of new initiatives n Identify the changing standards of care that need to be adapted n Analyze the market response to new agents n Predict 'most likely' competitor scenarios While no two rare disease areas can be tackled with a set template of CI strategy, an arsenal of previously acquired knowledge base can be leveraged for efficient analysis of complex scenarios. This can enable the successful development of orphan drugs. Despite the challenges, creative Competitive Intelligence (CI) strategies can provide a focused and optimized approach for the successful development of orphan drugs. Effective CI Strategies for Orphan Drug Development CI can provide actionable corporate intelligence and global competitive landscape analysis to enable superior pharma strategies. When properly executed, CI unravels invaluable assumptions and insights on pharmaceutical competitors' capabilities, current strategy and future goals. CI strategies help gather primary and secondary intelligence without Conclusion Rare diseases provide significant opportunities to the pharmaceutical industry. However, clinical and regulatory challenges, and unique market dynamics for reimbursements have a significant impact on the development and commercialization of orphan drugs. CI strategies and tools can be valuable assets in assisting the development of orphan drugs. However, they will need to be tailored to specific rare disease environments and deployed with ingenuity to provide meaningful outcomes. 05 COPYRIGHT 2017 WNS GLOBAL SERVICES WNS.COM

7 INSIGHTS COPYRIGHT 2017 WNS GLOBAL SERVICES WNS.COM 06

8 About WNS WNS (Holdings) Limited (NYSE: WNS) is a leading global Business Process Management (BPM) company. WNS offers business value to 200+ global clients by combining operational excellence with deep domain expertise in key industry verticals, including banking and financial services, consulting and professional services, healthcare, insurance, manufacturing, media and entertainment, retail & consumer packaged goods, telecom and diversified businesses, shipping and logistics, travel and leisure, and utilities. WNS delivers an entire spectrum of business process management services such as customer care, finance and accounting, human resource solutions, research and analytics, technology solutions, and industry-specific back-office and front-office processes. WNS has delivery centers world-wide, including China, Costa Rica, India, the Philippines, Poland, Romania, South Africa, Sri Lanka, Turkey, UK and US. To know more, write to us at marketing@wns.com or visit us at wns COPYRIGHT 2017 WNS GLOBAL SERVICES