Pharmaceutical Technology interviewed some of the speakers for the upcoming INTERPHEX 2013 conference to get a “sneak peek” of their presentations. The first of these, an interview with Par Almhem, president of ModWave and Modular Partners, discusses advances in modularization. Check out the media player on the PharmTech website or click here to listen to the podcast, and look for more to come soon. If you’re at the show, be sure to stop by PharmTech’s booth (Booth #4139).
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INTERPHEX 2013 begins later this month and Pharmaceutical Technology will be previewing the action in the weeks to come.
Look for posts from our Manufacturing Editor Jennifer Markarian, as well as PharmTech’s Editorial Director Rita Peters and Executive Editor Patricia Van Arnum.
And be sure to stop by PharmTech’s booth (Booth #4139) during the show!
The US Orphan Drug Act of 1983 defines a rare disease as one that affects fewer than 200,000. There are nearly 7000 rare diseases affecting nearly 30 million—or nearly one in 10—Americans, according to the National Organization for Rare Disorders (NORD).
The National Institutes of Health (NIH) reports that approximately 80% of rare diseases are genetic in origin; about half affect children. The challenges of facing a chronic, disabling, severe, or even life-threatening disease is compounded by difficulties in receiving an accurate diagnosis, and the lack of therapies to address the condition.
For the past six years, the last day in February has been designated Rare Disease Day. First observed in Europe in 2008 by the European Rare Disease Organization, the initiative expanded to the US in 2009. In 2012, more than 60 countries participated.
This year, events will be held worldwide on Feb. 28 to bring the cause of rare diseases to the attention of patients, the public, the medical profession, and policy makers. For example, the NIH is sponsoring a two-day conference for researchers and policymakers, including FDA representatives, to raise awareness of rare diseases, bring stakeholders closer together, coordinate policy actions between nations, and emphasize rare disease research and the search for new therapeutics.
The Orphan Drug Act provides tax credits and marketing incentives for sponsors to develop products for rare diseases. Since 1983, the number of applications submitted and products designated as orphan drugs has continued to grow. More than 400 drugs and biologic products for rare diseases have been approved. In contrast, fewer than 10 such products supported by industry came to market between 1973 and 1983.
Drug makers have financial incentives to develop orphan drugs. A 2012 report from Thomson Reuters, The Economic Power of Orphan Drugs, estimated the orphan-drug market at the end of 2011 at $50 billion globally, or 6% of total pharmaceutical sales. However, a compound annual growth rate (CAGR) of 25.8% for 2001 to 2010, compared to a 20.1% CAGR for a matched group of non-orphan control drugs, and an increasing number of orphan-drug approvals, suggests that the growth rate of orphan drugs will exceed that of non-orphan control drugs over the next 30 years, according to the report. Because many orphan drugs are biologics, they are less susceptible to generic competition than small-molecule drugs.
The impact of the smaller population needing the orphan drug is offset by the high cost of the drugs. In fact, the Thomson Reuters study found that 29% of orphan drugs have annual sales greater than $1 billion, about the same percentage as non-orphan drugs.
While the Orphan Drug Act has spurred the development of life-saving and life-improving therapies for individuals with rare diseases, it also has provided financial incentives for drug companies to invest in these narrow specialties. However, as the sponsors of Rare Disease Day point out, there is still much work to be done.
PharmTech’s March issue will feature an update on process analytical technologies that will include a focus on validation. As part of my research for the March feature, I would like to know your answer to the following question.
How has the FDA process validation guidance, Guidance for Industry: Process Validation: General Principles and Practices, changed validation in pharmaceutical manufacturing?
Please send your answers to email@example.com or post them directly here in the comments section.
The second half of 2012 has not been kind to FDA. Last month, Secretary Hamburg was grilled by the House of Representatives for failing to follow-up on warning letters to the New England Compounding Center (NECC), now comes word that a U.S. District Court has ruled that a sales representative’s off-label promotion of a drug was protected speech. The Food, Drug and Cosmetic Act (FDCA) appears to be coming apart at the seams.
In US v. Caronia, the Court ruled that Alfred Caronia, a sales rep promoting Xyrem for Orphan Medical (now Jazz Pharmaceuticals), could make statements about off-label uses of the drug, even though FDCA specifically prohibits “introducing a misbranded drug into interstate commerce.” The Court did not consider whether the statements were true.
Given the Supreme Court’s general affection for sales rep’s free speech rights, it is not out of the question to presume a difficult fight ahead for FDA. While it is too early to suggest that a wholesale rewrite of the FDCA is in order, clearly some significant revisions are in order.
“FDA and industry will need to come to an understanding on what exactly is needed to substantiate a product claim,” notes Pharm Exec Senior Editor Ben Comer.
It’s not just about free speech and drug marketing, though. With more than five hundred patients stricken and 36 deaths from tainted compounded steroid injections, there is an obvious need to clarify FDA authority over compounding pharmacies as well. Given the range of issues surrounding FDA authority a rethinking of the FDCA might just be in order.
Student Innovation Across the Pharma Sciences
Last week, Merck agreed to settle a Missouri consumer class-action suit, which claimed the company violated the Missouri Merchandising Practices Act when it promoted and sold its pain reliever Vioxx. Merck removed Vioxx from shelves in 2004 due to evidence that it increased the risk of heart attacks. According to the Justice Department, Merck began marketing Vioxx as a treatment for rheumatoid arthritis shortly after being approved by FDA as a painkiller in May 1999. However, FDA had not approved it for the treatment of rheumatoid arthritis until 2002. The settlement is reportedly for $220 million and Merck agreed to pay validated claims as well as approved attorneys’ fees, and settlement notice costs.
Merck’s press release on the settlement didn’t mention any admission of wrongdoing. In a company press release, Bruce N. Kuhlik, Merck’s executive vice-president and general counsel, said “This agreement is in the best interest of the company and its shareholders. It reduces the uncertainty of litigation and ongoing defense costs, and helps us to remain focused on bringing forward innovative products and services for our customers.” This is one of several recent examples of off-label drug promotion. Are the millions pharma companies end up paying in lawsuit payouts worth sidestepping FDA rules?
FDA has pushed back goals in relation to the Prescription Drug User Fee Act (PDUFA), the Biosimilar User Fee Act (BsUFA), and Medical Device User Fee Act (MDUFA) as a result of the closing of agency offices during Hurricane Sandy. FDA says it will assess the goals that were due October 31 and extend them as needed. The extensions will be no more than the number of business days the agency was closed.