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Orphan Drugs: A Discussion With Dr. Timothy R. Coté

July 22, 2014

Coté Orphan Consulting, LLC (COC), a regulatory affairs advisory firm in Silver Spring, Maryland, provides consulting services to companies that are developing or looking to develop orphan drugs. The company is founded in 2012 and led by Dr. Timothy R. Coté, former FDA Director of the Office of Orphan Products Development (OOPD). The COC team has provided regulatory consulting services including FDA orphan designation applications, breakthrough therapy designations, pediatric priority review vouchers, and regulatory strategic planning to over 150 major biotech and pharmaceutical companies. COC recently signed a $7 million contract resulting in the establishment of Coté Orphan Clinical Trials, a full-service clinical research organization.

The Orphan Druganaut Blog has the opportunity to interview Dr. Timothy R. Coté from COC about the services they provide and the state of the global orphan drug regulatory landscape.

1)   Can you please tell us a little about the Coté Orphan Consulting firm and what services you provide for companies interested in developing or are currently developing orphan drugs? Do you offer services globally or for specific geographic areas (EU, US, etc.)?

Coté Orphan Consulting provides valuable regulatory strategic planning to biotech and pharmaceutical companies seeking orphan status designation and marketing authorization for their orphan products. Services provided by the COC team include FDA orphan designation applications, regulatory strategic planning, breakthrough therapy designations, pediatric priority review vouchers, and clinical trials design and execution. COC caters to all major established regulatory markets.

2)   What role and impact do you see advocacy organizations and patients having on global regulatory agencies for orphan drugs ?

Patients are what this is all about. Previously, as chief medical officer for NORD I saw first-hand the critical role that patients play in orphan drug clinical trials, regulatory approvals, and eventually, marketing efforts. This is as it should be. Patients are the reason this endeavor exists.

3)   What changes would you like to see with global regulatory agencies to improve the speed of bringing orphan drugs to market and for providing faster access to patients ?

I was honored to serve as the director of FDA’s Office of Orphan Products Development and can state from first-hand knowledge that the OOPD as well as the many review divisions are committed to expedient review and licensing of orphan drugs. Sadly, the lapse is not theirs; they are a beleaguered, underfunded, bureaucratically constrained organization of dedicated public servants who, were it within their power, would indeed accelerate the licensure of therapies we all desire. To make this happen, Congress must appropriate the funds requisite to intelligent regulation of 25% of the US Economy. And the American people need to support the strivings of these selfless servants.

4)   Do you advise companies on the pricing of orphan drugs ? What do you see as the challenges for how insurance/government agencies will handle the high prices of orphan drugs ?

We do not advise on price points, but we recognize that orphan drugs merit higher price points for the 7 or 10 years of market exclusivity to which they are entitled. While some of the numbers may be eye-popping (e.g., $700,000 per patient per year) the knowledge is eternal and the exclusivity time-limited. For 32 years, the Orphan Drug Act has remarkably advanced capacity of the entire biotechnology industry. Therefore, it was no surprise when the Affordable Care Act (ACA)-the most radical revision of healthcare financing since 1776-left our system for incentivizing orphan drugs completely untouched. This was rational, given that orphan drugs represent less than 1% of the total healthcare budget. It is rare to see such rationality expressed by Congress, but this time we were lucky.

5)   Do you think collaborations/partnerships between different international regulatory agencies for orphan drugs will be the direction for the future ?

No, but I think they should be. For example, while I instituted regular monthly meetings between FDA and EMA offices responsible for orphan designation, and lobbied hard for a mutual recognition of such designation, I doubt it will occur in my lifetime. Drug regulation is the extension of the formal authority of States, and States are loathe to share authority.

6)   What do you see for the future of orphan drugs globally ?

Orphans are hot. They represent the great frontier in healthcare. We have 7,000 rare diseases but only about 400 licensed orphan products which together treat about only 200 rare diseases. That leaves 6,800 rare diseases without any form of treatment. We have work to do.

7)   Does COC have any upcoming meetings or special announcements to share ?

COC and the ERA Consulting Group (ERA) are pleased to announce the establishment of a strategic transatlantic alliance to provide comprehensive services to clients developing products for orphan indications.

The ERA Consulting Group, with offices in the UK, Germany, Washington, DC, and Brisbane, Australia, is a consulting organization in Europe and Australasia for biologics, including gene and cell therapy products, which are often developed for orphan indications. With more than 27 years of experience, the ERA team has successfully achieved orphan medicinal product status in the EU for many clients. Also, ERA has full electronic publishing capabilities to process electronic submissions both to FDA and EU authorities.

Together, COC and ERA can provide advice and practical assistance at all stages of development of orphan drugs, including concept, orphan designation, clinical trial applications, and BLAs/MAAs.

Thank you for your time.

COC Logo courtesy of COC.

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