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Orphaned No Longer: Big Pharma Embraces Drugs for Niche Markets

Executive Summary

Hit hard by late stage pipeline failures and the regulatory and reimbursement challenges of traditional primary care products, Big Pharmas are taking renewed interest in drugs for orphan diseases, where high unmet medical need offers the promise of premium pricing and there's potential to grow the market via approval in multiple indications.

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In Defense Of “Me-Too” Drugs: Is Primary Care Losing Out To Rare Disease R&D?

Regulatory and financial incentives have sparked major investment in orphan drug development, but now some stakeholders are questioning whether the focus on orphans is orphaning another market: primary care conditions that are still not adequately treated. Are incentives needed?

In Defense Of “Me-Too” Drugs: Is Primary Care Losing Out To Rare Disease R&D?

Regulatory and financial incentives have sparked major investment in orphan drug development. One-third of the new molecular entities and novel biologics approved by FDA in the past five years have been orphan drugs. But now some stakeholders are questioning whether the focus on orphans is orphaning another market: primary care conditions that are still not adequately treated. Are incentives needed?

The Orphan Drug Boom: Gold Rush Or Flash In The Pan?

Rare disease drug development has ballooned in just three years, powered by the success of independent biotechs like Alexion, Big Pharma’s entrée into the field, and pressure from regulators and payors that is dis-incentivizing development of traditional primary care drugs. The positive momentum has intensified dealmaking in the orphan drug space and is giving investors confidence to back rare disease-focused start-ups. Some have coined the resulting movement the “orphan drug bubble,” but interest isn’t likely to deflate soon – at least not as long as Big Pharma continues to invest in the area and industry is able to sustain a favorable reimbursement climate for ultra-premium-priced drugs.

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