A critical drug that has experienced shortages is Genzyme’s, Fabrazyme (agalsidase beta), the only enzyme replacement therapy approved in the US for Fabry disease. The drug was originally produced at the company’s Allston, Massachusetts plant. The Allston plant was plagued with quality problems, which resulted in a consent decree, temporary closure of the plant in 2010, and rejection of some lots of Fabrazyme for quality reasons. In January of 2012, FDA granted approval for Genzyme to start producing Fabrazyme at its Framingham, Massachusetts plant, bringing to an end a shortage that left patients without an adequate supply of medication for nearly two years. Read more »

This blog post was written by Ana Nicholls, Healthcare Analyst at the Economist Intelligence Unit.

Is it a mark of desperation? UK-based pharma company AstraZeneca has resorted to suing the US Food and Drug Administration in a bid to stop it from approving generic competition to Seroquel, the company’s blockbuster anti-depressant, before December 2012. The core patent for Seroquel IR expired in September last year, and its paediatric patent runs out this month, although Seroquel XR (the later version) still has protection till 2017. Read more »

This week, the Drug, Chemicals, and Associated Technologies Association (DCAT) is holding its annual week-long meeting in New York City. The meeting covers topics including the business and regulatory environment, manufacturing practices, and supply chain logistics of the pharmaceutical industry. Wednesday Mar. 14, 2012 featured a session devoted to biologics, in which the first two speakers spoke at length on the topic of biosimilars, and highlighted important differences between follow-on biologics and generic small molecules. Read more »

Medicine regulatory agencies in the EU, US and Australia have been successfully collaborating for some time now in the area of GMP inspections of API manufacturers, but now the partners are seeking to further expand the international effort by looking to include additional partners. Read more »

A short time ago, I wrote about the Transforming the Regulatory Environment to Accelerate Access to Treatments Act, (TREAT), legislation proposing to modify the regulatory environment to speed the approval of drugs intended to treat life-threatening conditions for which there are no effective therapies. This week brings us the Faster Access to Specialized Treatments (FAST) Act, introduced by Rep. Clifford Stearns (R-FL) and Rep. Ed Towns (D-NY) to the House Committee on Energy and Commerce on Mar. 5, 2012. FAST proposes to amend section 506 of the Federal Food, Drug, and Cosmetic Act to expedite approval of drugs for serious or life-threatening diseases or conditions. Rep. Stearns explains in a press release, “This measure codifies much of the Accelerated Approval regulations into statute and modernizes the program to reflect the amount of medical and scientific innovation that has occurred in the past 20 years.” The aim of the legislation is to encourage FDA to make full use of the accelerated approval pathway to speed the approval of life-saving drugs.

FAST enjoys the support of The National Organization for Rare Disorders (NORD) and of the biotech industry group, BIO. Jim Greenwood, president of BIO, said in a release, “Like the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act, S. 2113, which was introduced by Senator Kay Hagan (D-NC), the FAST Act will update the Accelerated Approval process at the FDA so that it works in a transparent, predictable way for all therapies intended to treat debilitating conditions for which there is an unmet medical need. Making this change will better enable biotechnology companies to bring new, safe and effective treatments and cures to patients in need at the earliest point in time.”

Time is of the essence for those with rare diseases or with other conditions for which there are no effective therapies. Certainly, FDA should do its part to get therapies to market in the shortest time possible, and the introduction of two similar bills in such a short time span underscores the public’s impatience with the drug development process. However, human physiology is complex, drug development is hard, and FDA always has to balance speed with safety. Will urging FDA to go faster bring the desired results?

Gartner has developed a body of research around the convergence of IT and Operational Technology (OT) and the implications for organizations (1). Much of the research speaks to the opportunities and challenges this convergence presents to organizations. By way of definition operational technology, according to Gartner, refers to “physical-equipment-oriented technology” (e.g., manufacturing equipment) that is shifting from proprietary, hardware-based to open, software-based technology. Thus the lines are becoming blurred as to who “owns” and supports the infrastructure, integration, upgrades, security and analyses of OT software systems. Read more »

This week an interesting report was released about the use of gelatine in medicines. Most of us are comfortable with the use of animal products as ingredients, but for vegetarians or other people that follow restrictive diets it can be an issue.  Anybody who falls into this situation should be able to choose whether they will or won’t take a particular product, but the report highlights the fact that many people do not realise that their medicines contain gelatine. In particularly, many vegetarians may unknowingly be prescribed medicines that contain animal derivatives.

As such, the authors of the report, published in the Postgraduate Medical Journal are calling for clearer drug labelling for such medicines. Read more »

Rare disease day is an annual awareness-raising event coordinated at the international level by EURORDIS, a non-governmental patient-driven alliance of patient organizations representing more than 502 rare diseases patient organizations in over 46 countries.  This year marks the fifth that EURORDIS has coordinated the event, which was marked by outreach activities in more than 40 countries that centered on the theme of solidarity.   EURORDIS organized a summit in Brussels, Belgium on Feb. 29, 2011, featuring speakers from European governments, rare disease research groups, and patient advocacy groups who highlighted both the progress and the remaining challenges around developing medicines for rare diseases and bringing them to market in Europe. Read more »

Last week, counterfeits of the anti-cancer drug Avastin were discovered in the US and this week fake pharmaceuticals continue to occupy headlines. Read more »

Senator Kay R. Hagan (NC) introduced a piece of legislation called the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act. The bill is intended to accelerate the approval process for medicines targeted to conditions for which there are no effective treatments. The bill will be referred to the Senate Health, Education, Labor and Pensions (HELP) Committee that oversees healthcare issues. Read more »