Regulatory Focus™ > News Articles > 2013 to Bring Regulatory Opportunities, Difficulties: Report

2013 to Bring Regulatory Opportunities, Difficulties: Report

Posted 09 January 2013 | By

Manufacturers of pharmaceutical products may be experiencing their best approval rates in years thanks to a more receptive US Food and Drug Administration (FDA), but that doesn't mean the industry is out of the woods yet, writes the Tufts Center for the Study of Drug Development (CSDD).

In a study released on 8 January, the center said the changes in approval numbers still aren't addressing the problem now endemic to product development pipelines: scarcity.

"Many companies are taking steps to improve clinical success rates and reduce the cost of new product development, including utilizing enhanced clinical trial designs, making greater use of biomarkers, and adopting sophisticated statistical analyses," wrote CSDD Director Kenneth Kaitin. "It's a good start, but in a world shaped by increased patent expirations, diminished cash flow, and fewer promising breakthrough products, companies will need to hone their efforts to streamline development."

As more companies find themselves having plunged off the so-called "patent cliff"-the date after which their largest-grossing branded products go off-patent and are subject to generic competition-they need to be cognizant of the inefficiencies of their early- and mid-stage research and development pipelines, Kaitin added.

These can be improved by placing an emphasis on innovative development models, including partnerships with other companies and organizations, "open innovation," adaptive clinical trial designs, the use of new biomarkers in clinical trials and novel formulation techniques.

So how, exactly, are companies coping with these patent expiries, and what is 2013 likely to bring? Like the rest of us, Tufts doesn't exactly know, but it did identify some trends likely to occur in the coming year.

Biosimilars: The Interchangeability Problem

Of note: "The biosimilar market is poised for significant growth following the creation of an abbreviated approval pathway for biological products that are demonstrated to be "interchangeable" with an FDA-approval biologic, as required by the Patient Protection and Affordable Care Act."

That is, if that pathway comes to fruition in 2013. FDA has been deluged by comments from both the branded and generics biologics industries, all of which trying to carve out an edge. One of the main points of contention, as identified by CSDD, is that of "interchangeability"-the ability of two products two be substituted for one another with no clinically meaningful differences.

Branded biologics company have been pressing for a high bar for interchangeability, arguing that such products should be subject to rigorous testing in the interests of protecting patients from experiencing uncertain benefits or potential harms. Still other groups have argued that biosimilar products should be required to have non-generic, unique names as part of a push to ensure that consumers and physicians understand that a biosimilar product is not necessarily substitutable for the original biologic.

[For more information, please see Regulatory Focus' 28 September 2012 feature, "Biosimilar Interchangeability Problems Pose Complex Challenge for Regulators."]

Little Drugs, Big Profit Potential

Tufts also identified a growing trend that it says is likely to continue into, and beyond, 2013: big pharmaceutical companies' collective focus on "little" diseases.

"Prescription drug policy will be shaped by global concerns to a greater degree than ever before, with international coordination growing in relation to the development of personalized medicines as well as drugs for neglected diseases," the organization wrote.

They have good reason to think the trend will continue, as companies find that they can make considerable amounts of money using a strategy that charges tremendous amounts of money-hundreds of thousands of dollars-for otherwise neglected diseases. Insurers are most often stuck paying the majority of the bill for the treatment, while many companies opt to pick up the patient's co-pay charges in a bid to ensure that they maintain adherence to the treatment regimen.

Profits in the sector are beginning to attract increasing amounts of attention from investors. Some orphan drugs marketed by some companies, including Sanofi-owned Genzyme Corporation, bring in billions in revenue each year, making them "blockbuster" drugs despite their extremely small markets (by definition, fewer than 200,000 patients affected in the US).

With R&D pipelines drying up and new blockbuster drugs for more broad-based conditions becoming harder to come by, some companies are looking at the orphan drug space as a lifeline. Perhaps, even as a treatment for what ails them.


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