Pink Sheet is part of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By



Executive Summary

ORPHAN DRUG BRAFT BILL WOULD REDUCE MARKER EXCLUSIVITY under the Orphan Drug Act to four years but allow for a three-year extension for products whose "net cumulative sales" do not exceed $200 mil. during the first four years of marketing. The draft by Sens. Metzenbaum (D-Ohio) and Kassebaum (R-Kan.) defines "net cumulative sales" as total U.S. sales "minus discounts, allowances and returns." Market exclusivity currently lasts seven years under the orphan law. The Metzenbaum-Kassebaum draft would allow competing brands of an approved orphan product to be approved during the first product's exclusivity period if the initial and subsequent brands were "developed simultaneously." Products are simultaneously developed, the draft explains, "only if" the sponsors of subsequent brands request orphan designation within six months after designation of the initial product, begin clinical trials within 12 months after trials began on the initial product, and submit an NDA within 12 months after the initial orphan product NDA was submitted. The draft also would end an exclusivity period of an orphan product if the patient population for the product grows beyond the 200,000-patient threshold. The draft further stipulates that the threshold for orphan drug designation is based not only on a measurement at the date of designation, as provided under current law, but also on projections of the size of the population three years after designation. The Biotechnology Industry Organization is expected to issue a formal position on the draft after its Dec. 15 board meeting. A proposal developed and floated by BIO last spring would have amended the act by limiting exclusivity to five years and allowing for two-year extensions. A more striking difference in the industry proposal is its lack of a sales-based threshold. The BIO proposal would have allowed the extension only for products for patient populations of less than 100,000 ("The Pink Sheet" June 21, T&G-7). In addition, the industry proposal sought to eliminate the reliance of biotechnology products on the Orphan Drug Act for market exclusivity. The proposal would have provided four-year exclusivity periods for biotech products. The Metzenbaum-Kassebaum draft contains no such provision. Metzenbaum and Kassebaum had planned to introduce their bill before Congress recessed but instead withheld introduction until after they receive industry's reaction. Their decision suggests that the drafting process remains open and that they will consider requests for changes. The senators reportedly considered including their proposal as a provision of the health care reform bill but now plan to move it as free-standing legislation.

You may also be interested in...

Part D Discount Liability Coming Into Focus: CMS Releases Drug Cost Data

Newly released Medicare Part D data sheds light on the sales hit that branded pharmaceutical manufacturers will face when the coverage gap discount program gets under way in 2011

FDA Skin Infections Guidance Spurs Debate On Endpoint Relevance

FDA appears headed for a showdown with clinicians and the pharmaceutical industry over the proposed new clinical trial endpoints for acute bacterial skin and skin structure infections, the guidance's approach for justifying a non-inferiority margin and proposed changes in the types of patients that should be enrolled in trials

Shire Hopes To Sow Future Deals With $50M Venture Fund

Specialty drug maker Shire has quietly begun scouting deals with a brand-new $50 million venture fund, the latest of several in-house investment arms to launch with their parent company's pipelines, not profits, as the measure of their worth



Ask The Analyst

Please Note: You can also Click below Link for Ask the Analyst
Ask The Analyst

Your question has been successfully sent to the email address below and we will get back as soon as possible. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts