Healthy Skepticism Library item: 18199

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Publication type: news

Larkin C
Drugmakers Prodded by FDA for Neglected Disease Drugs
Bloomberg Businessweek 2010 Jun 18
http://www.businessweek.com/news/2010-06-18/drugmakers-prodded-by-fda-for-neglected-disease-drugs-update3-.html

Full text:

Roche Holding AG, Johnson & Johnson and Biogen Idec Inc. are being urged by U.S. regulators to see if existing medicines may help neglected disorders, after an incentive program failed to spark research on new therapies.
The Food and Drug Administration published a list of 235 treatments today that may have benefit in rare disorders and already have marketing clearance for other uses. Identifying “low-hanging fruit” may compel large drugmakers to look beyond common ailments with guaranteed consumer demand, said Tim Cote, head of the agency’s Office of Orphan Product Development.
The list includes Roche’s hepatitis drug Pegasys, J&J’s leukemia medicine Leustatin and Biogen Idec’s multiple sclerosis drug Avonex. Regulators need new tactics since an experiment two years ago to give priority review vouchers in exchange for new drugs for tropical diseases drew only one participant.
“Large pharmaceutical companies are not as engaged as we’d like to see them be,” Cote said in a phone interview. “I myself have knocked on the doors of all the big boys.”
About 30 million Americans have one of 7,000 rare diseases, defined by the FDA as conditions affecting fewer than 200,000 people in the U.S. Medicines developed to treat these conditions are called orphan drugs, under rules that encourage their development.
2,150 Orphan Designations
More than 2,150 potential treatments have been identified since the 1983 Orphan Drug Act promised seven years of market exclusivity to any product approved for a rare disease. To date, only 358 are on the market, and most are made by small, specialized biotechnology companies.
“This is not something that companies are thinking about on a regular basis,” said Ira Loss of Washington Analysis, who has followed the FDA for more than three decades. “If things were better in terms of the general economy and the pipelines of these companies, where they had time to focus on this possibility, it might be a different story.”
The FDA’s list focuses on orphan designations that have been given to drugs whose approval in other uses suggests they’re not toxic, Cote said. Anyone can apply for orphan drug designation without the current manufacturer’s permission, according to Sandy Walsh, an FDA spokeswoman.
Roche’s Pegasys has orphan status for chronic myelogenous leukemia. Leustatin, made by New Brunswick, New Jersey-based J&J, is approved for hairy cell leukemia but has had orphan drug designation since 1990 for chronic lymphochytic leukemia and acute myeloid leukemia. Also on the list is Biogen’s top-selling MS drug Avonex, which has had orphan designation since 1991 as a treatment for cutaneous t-cell lymphoma.
Companies Respond
While Biogen initially pursued Avonex in “several indications” the Cambridge, Massachusetts-based drugmaker now has no plans to develop it for cutaneous t-cell lymphoma, said Kate Weiss, a company spokeswoman.
“While we’re certainly always open to looking at new indications for all of our drugs, we’re not pursuing that for that indication,” Weiss said today in a phone interview.
J&J decided not to further develop Leustatin in part because there are other drugs available for leukemia, Ernie Knewitz, a spokesman for the company, said today in a telephone interview. J&J is studying products for other types of neglected diseases including the areas of oncology, HIV and hepatitis C, Knewitz said.
Roche is no longer studying Pegasys in cancer, said Kristina Becker, a spokeswoman for the Basel, Switzerland-based company’s Genentech unit.
‘Long History’ of Development
“The company has pursued studies and applications for several orphan indications,” Becker said today in an e-mail. “We have a long history of developing new medicines for diseases where there are no existing therapies even if not many patients are affected.”
Companies may worry that increasing pressure to cut medical costs will limit their pricing power, making it harder to recoup the cost of their investment in research from the small number of patients taking a medicine, Cote said. Orphan drugs that have other uses may also still be victim to generic substitution, effectively limiting the promise of seven years of market exclusivity.
“The lack of research and lack of treatments is something we see every day,” said Mary Dunkle, a spokeswoman for the National Organization for Rare Disorders in Danbury, Connecticut. “It’s very frustrating for the families.”
The FDA tried a new strategy in 2008 by offering priority review vouchers for companies who win approval of drugs for one of 16 tropical diseases. The vouchers will shave four months off the regulatory review of any new product for which a company uses it. The vouchers also can be traded or sold.
Broadening Vouchers?
Novartis AG, of Basel, Switzerland, has so far won the only voucher — for a 10-year-old malaria pill — and hasn’t used it. Cote said he is considering broadening the list of conditions eligible for a voucher “within a couple months” to spark interest in the program.
Drugmakers are skeptical about vouchers, especially as an increasing number of drugs with priority review face delays, said Ramsey Baghdadi, a health-care analyst at Prevision Policy in Washington. He isn’t convinced that either initiative will be enough to encourage companies to invest in orphan-drug research they wouldn’t otherwise do.
“Nothing would hold a pharma company back from going after something if they really wanted to go after it,” Baghdadi said. “The real route is through patent extension or increased market exclusivity. People understand the value of that.”
The FDA will solicit feedback on how it approves and encourages medical products for rare diseases at a June 29-30 public hearing at its headquarters in Silver Spring, Maryland.