After less than a year on the job, the head of FDA’s Office of Generic Drugs (OGD) has announced his departure, a sign that all is not well with plans for major organizational changes at the Center for Drug Evaluation and Research (CDER). Read more »
Archive for the 'Regulation' Category
The greatly feared federal budget sequestration mandate went into effect Mar. 1, 2013, and, initially, the impact was fairly muted. The stock market soared, employment rose and government workers continued on their jobs. Federal agencies, including the Food and Drug Administration (FDA), launched initiatives to comply with the mandated 5% across-the-board cut in spending (in reality a 9% cut that exceeds $200 million) to minimize the impact on basic operations. That means curbs on training and staff travel, no new hires and a delay in launching new programs. Read more »
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.
The growing dangers from substandard and falsified medicines around the world has prompted a blue-ribbon panel formed by the Institute of Medicine (IOM) to call for clear international standards for higher quality medical products, plus an electronic tracking system in the US to uncover bogus products in the supply chain. Read more »
I’ve written frequently about the problem of counterfeit medicines in the United Kingdom and how the country’s Medicines and Healthcare products Regulatory Agency (MHRA) is working to prevent these, but counterfeits are not the only potentially dangerous medicines that are becoming increasingly prevalent. In a recent news release, the MHRA explained it is receiving a growing number of reports of unlicensed medicines being sold across the UK. Read more »
Revisions to the United States Pharmacopeia (USP) General Chapter<231>Heavy Metals have been much discussed over the past decade. USP is in the process of replacing this chapter with General Chapters <232> Elemental Impurities—Limits and <233> Elemental Impurities—Methods, which were published in USP 35-NF 30, Second Supplement. The official date for the new chapters—originally Dec. 1, 2012—was briefly postponed due to an appeals process but was recently re-established as Feb. 1, 2013, as described in today’s PharmTech news article.
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With the release of a draft guidance on the evaluation and labeling of abuse-deterrent opioid formulations, FDA is one step closer to clarifying its thinking on acceptable formulations for this product class. Read more »
PharmTech’s February issue will feature a guest editorial by Aaron Davidson, a partner in the life sciences practice at Baker Botts, on social media and the pharmaceutical industry. In his editorial, Mr. Davidson points to FDA and a lack of regulatory guidance as the reasons that social media has not caught on in the pharma and bio/pharma industries. FDA’s requirement to provide consumers with FDA-approved promotional materials that contain risk information may limit a pharmaceutical company’s ability to use mediums such as Twitter and Facebook effectively.
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.
On the second day of the PDA/EMA joint conference, we had a choice of three different topics, all running simultaneously: GMP inspection practice, process and validation, and challenges for EU harmonisation. I somehow found myself listening to Lina Ertle from the French national agency for medicines and health products (ANSM), who gave an update on process validation guidelines. Read more »