Orphan Drugs to Create Paradigm Shift in the Pharmaceutical Industry

The economic recovery process has proved difficult for the pharmaceutical industry. Factors like patent expiry, dry pipeline, and strict approval guidelines, have slowed down the attractive drug discovery and industry growth. Coupled with an investment risk in new therapy or molecules, this creates a challenge for the industry to overcome. However, in the midst of the current status, there seems to be a bend in the road - marked by the evolution of a paradigm shift in the industry: Orphan drugs.

"While the pharmaceutical industries have been focusing on 'blockbuster' small molecules (chemical drugs) for high revenue generation in the past, it is expected that in 5 years, around $90.0 billion worth of branded drugs will lose their exclusivity," finds Frost & Sullivan Healthcare consultant Shabeer Hussain. “The current economic situation plus the huge generic competition shifted the focus of pharmaceutical companies and they are moving to a new business model - 'Niche busters', also called Orphan drugs."

This new business model will provide an approach to an integrated healthcare solution, thereby enabling pharmaceutical companies to develop newer areas of therapeutics, diagnosis, treatment, monitoring and patient support. Orphan drugs provide attractive opportunities to reduce the impact of revenue loss due to the patent expiry for blockbuster drugs. Clinical trials for orphan drugs are run efficiently with smaller patient groups, thereby reducing costs significantly. Incentives for drug development provided by governments, the FDA and the EU commission support in special protocols are a further boost for companies developing orphan drugs.

Pharmaceutical and biopharmaceutical companies are also joining in partnerships by licensing products to maintain revenue. Such collaborations reduce the cost of developing and marketing orphan drugs. In numerous ways, a balanced, strong alliance will achieve mutual benefit to both pharma and biotech companies.

While the focus on "niche busters" grows, the safety regulations and approval processes will become stricter, putting larger companies in a better position to cope with the increasing demands. However, though smaller pharma-biotech companies will struggle to compete with the more powerful competitors in the industry, Hussain believes there will still be assurance for niche players with specialist therapies, technologies, unique capabilities and expertise. Acquisitions at a global level, aimed at specific niche capabilities with technologies, are likely to be the most effective way of achieving a better partnership and collaboration, as well as a more diverse client space. This will add the necessary stability smaller collaborations need to survive and thrive.

If you are interested in more information on Frost & Sullivan's research on Orphan Drugs, please send an e-mail to Katja Feick, Corporate Communications, at This email address is being protected from spambots. You need JavaScript enabled to view it., with your full name, company name, title, telephone number, company e-mail address, company website, city, state and country.

About Frost & Sullivan
Frost & Sullivan, the Growth Partnership Company, enables clients to accelerate growth and achieve best-in-class positions in growth, innovation and leadership. The company's Growth Partnership Service provides the CEO and the CEO's Growth Team with disciplined research and best-practice models to drive the generation, evaluation, and implementation of powerful growth strategies. Frost & Sullivan leverages over 45 years of experience in partnering with Global 1000 companies, emerging businesses and the investment community from 40 offices on six continents. To join our Growth Partnership, please visit http://www.frost.com.

Most Popular Now

Aspirin green light for brain bleed stroke patient…

People who suffer a stroke caused by bleeding in the brain - known as brain haemorrhage - can take common medicines without raising their risk of another stroke, a major ...

Cancer cells are quick-change artists adapting to …

Until now, researchers have assumed that the growth of solid tumors originates from cancer stem cells characterized by specific surface markers, which develop in a fixed...

APRINOIA Therapeutics awarded grant from The Micha…

APRINOIA Therapeutics, a clinical-stage biotechnology company with a pipeline of therapeutics and imaging diagnostics for neurodegenerative diseases, announces today that...

Benralizumab not effective reducing exacerbations …

More than 15.3 million people in the U.S. suffer from chronic obstructive pulmonary disease (COPD), which is the third leading cause of death in this country, according t...

Pfizer announces top-line results from Phase 3 Tri…

Pfizer Inc. (NYSE: PFE) announced today that a Phase 3 study to assess the use of LYRICA® (pregabalin) as adjunctive therapy for epilepsy patients 5 to 65 years of age wi...

Novartis phase II data for new inhaled combination…

Novartis announced today that new phase II data for IND/GLY/MF (QVM149), an investigational, once-daily, fixed dose combination asthma treatment containing indacaterol ac...

Cannabis use among older adults rising rapidly

Cannabis use among older adults is growing faster than any other age group but many report barriers to getting medical marijuana, a lack of communication with their docto...

New data show Symbicort reduces attacks in mild as…

New data from Novel START, an open-label trial designed to reflect real-world practice, has demonstrated the effectiveness of Symbicort Turbuhaler (budesonide/formoterol)...

Merck pioneers new effort to see MS from the insid…

Merck, a leading science and technology company, joins the global multiple sclerosis (MS) community in recognition of World MS Day, an initiative created by the Multiple ...

Amgen joins with community oncology networks for n…

Amgen (NASDAQ: AMGN), along with leading community oncology networks, today announced the launch of Amgen Community Oncology Research Collaborators (ACORC), a new initiat...

Bayer receives U.S. FDA breakthrough therapy desig…

Bayer announced today that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for copanlisib (Aliqopa™) for the treatment of adult patie...

Novartis Kisqali significantly extends life in wom…

Novartis announced statistically significant overall survival (OS) results for Kisqali in combination with endocrine therapy[1]. The Phase 3 MONALEESA-7 trial evaluated K...