Tag Archives: EMA

EMA Announces Brexit Business Continuity Plan

In a press release issued on August 1, 2017, The European Medicines Agency (EMA) announced that it “has developed and initiated a business continuity plan to deal with the uncertainty and workload implications linked to the United Kingdom’s (UK’s) withdrawal from the European Union (EU) and the Agency’s relocation”. It was indicated the plan is intended to “preserve Agency’s ability to protect public and animal health”.

The plan outlines 3 categories of EMA activities and prioritizes them “according to their impact on public health and the Agency’s ability to function”.

Category 1, the highest priority activities, includes those related to the assessment and safety monitoring of medicines such as actions taken to protect patient safety and inspections across the EU, or activities vital to maintaining the infrastructure and functionality of the EU regulatory system for medicines such as security of critical IT applications. EMA stated, “It is absolutely crucial to uphold these activities as any disruption would almost immediately have a detrimental effect on the health and well being of citizens in Europe and would also jeopardize production and distribution of medicines in the EU”.

Category 2 activities include the proactive publication of clinical data, various initiatives aimed at promoting availability of medicines, and projects such as EMA’s contribution to the fight against antimicrobial resistance or the Agency’s interactions with Health Technology Assessment (HTA) bodies. EMA indicated, “These activities will be maintained for as long as possible, workload and staffing situation permitting, in order to maintain the development of new medicines”.

However, in order to reallocate personnel for preparations for the UK’s withdrawal from the EU and EMA’s relocation, EMA has already begun to suspend activities in what it terms category 3 including:

  • the development of the European Medicines Web Portal, a new publicly-available online information intended to be a single-source for information on all medicines marketed in the EU; 
  • EMA’s contribution to the e-submission project that will allow applicants to electronically submit documents linked to authorization requests for human and veterinary medicines in a secure and efficient way; 
  • the development of a transparency roadmap for EMA that lays out future transparency measures of the Agency; and 
  • participation in the benchmarking of medicines regulatory authorities in the EU as of 2018.”

EMA said it was also reducing the number of audits, some corporate governance and support activities, EMA meetings and workshops, and participation of EMA staff in external meetings or conferences.

EMA said it will consider “how long these activities can be put on hold before they start to seriously undermine the quality of the Agency’s work and its planning, and the expectations of stakeholders”. However, the press release concludes rather ominously stating, “Further iterations of the business continuity plan will also take into account various scenarios for staff losses and how these may affect the delivery of category 1 and 2 activities. Unexpected higher, faster or more permanent loss of staff as a consequence of the Agency’s relocation may lead to a situation in which EMA’s operations can no longer be maintained.”

Source: http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2017/07/news_detail_002789.jsp&mid=WC0b01ac058004d5c1

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September is Charcot-Marie-Tooth (CMT) Awareness Month, Spread the Word!

CMT affects 1 in every 2,500 people worldwide, (a total of 2.8 million). That’s 1 too many.

What if each of those 2.8 million people ALL told five people about CMT?  Fourteen million new people would learn about CMT!  This is why the Charcot-Marie-Tooth Association (CMTA) declared September as CMT Awareness Month six years ago!

What is CMT? 

Charcot-Marie-Tooth Disease, or CMT, is a group of inherited disorders that affect the peripheral nerves, which are the nerves outside the brain and spinal cord. There are 90 kinds of CMT. Each kind is caused by a different kind of mutation, and more causes are being discovered every year.

CMT is just one kind of neuropathy (also called peripheral neuropathy), meaning simply that the peripheral nerves are damaged. There are many other causes of neuropathy, including the most common cause—diabetes.

CMT affects about 2.8 million people worldwide, of all races and ethnic groups.

Where Did the Name CMT Come From?

Charcot-Marie-Tooth is named after the three physicians who were the first to describe it in 1886: Jean-Martin Charcot, Pierre Marie and Howard Henry Tooth.

Charcot        Marie        Tooth

Inherited Disorders

CMT is inherited. It is not contagious, nor is it caused by anything in the environment. The most common forms of CMT are passed down from one generation to the next, meaning that it is dominantly inherited.

Some forms of CMT are recessively inherited—a person may be affected even though his or her parents do not have CMT. In this case, each of the parents harbors a mutation in one of their two copies of a CMT gene. If a child inherits one mutated CMT gene from each of their parents (the chance of this happening is one out of four), the child will develop CMT.

Sometimes the mutation that causes CMT happens spontaneously during the process that produces the eggs or sperm. In these rare cases, a child will have CMT even though neither parent has CMT. If a child has such a spontaneous mutation, he/she may pass that mutation down to his/her offspring.

Symptoms

Some types of CMT cause damage to the covering (myelin sheaths) that surrounds nerve fibers. Other kinds of CMT directly damage the nerves fibers themselves. In both cases, the damaged nerve fibers result in neuropathy. The nerves in the legs and arms, which are the longest, are affected first. Nerve fibers that create movement (called motor fibers) and nerve fibers that transmit sensations (called sensory fibers) are both affected. CMT causes weakness and numbness, usually starting in the feet.

In the most common kinds of CMT, symptoms usually begin before the age of 20 years. They may include:

  • Foot deformity (very high arched feet)
  • Foot drop (inability to hold foot horizontal)
  • “Slapping” gait (feet slap on the floor when walking because of foot drop)
  • Loss of muscle in the lower legs, leading to skinny calves
  • Numbness in the feet
  • Difficulty with balance
  • Later, similar symptoms also may appear in the arms and hands
  • CMT almost never affects brain function

foot

The foot of a person with CMT. The lack of muscle, a high arch, and claw toes are signs of this genetic disease. 

Diagnosis

A diagnosis of CMT is established through a thorough neurological evaluation by an expert in neuropathy, including a complete family history, physical exam, and nerve conduction tests, and appropriate genetic testing.

A physical exam may show:

  • Difficulty lifting up the foot while walking
  • Difficulty with dorsiflexion of the toes and ankles (upward movement, away from the ground) and other foot movements
  • Reduced or absent deep tendon reflexes (like the knee-jerk reflex)
  • Loss of muscle control and atrophy (shrinking of the muscles) in the feet and lower legs (and later the hands)

Genetic testing can provide the exact cause for most people who have CMT.

Prognosis (Expectations)

CMT usually gets worse, slowly, with age; rapid progression is rare, and should motivate a prompt re-evaluation. The problems with weakness, numbness, difficulty with balance, and orthopedic problems can progress to the point of causing disability. Pain can be an issue, either as a direct result of the neuropathy (neuropathic pain) or as consequence of orthopedic problems. Other potential complications include the following:

  • Progressive inability to walk from weakness, balance problems, and/or orthopedic problems
  • Progressive inability to use hands effectively
  • Injury to areas of the body that have decreased sensation

Treatments

There are no known treatments that will stop or slow down the progression of CMT, but the CMTA is funding research to find these treatments.

Physical therapy, occupational therapy, and physical activity may help maintain muscle strength and improve independent functioning.

Orthopedic equipment (such as braces, inserts, AFOs or orthopedic shoes) may make it easier to walk.

Orthopedic surgery on the feet can often maintain or even restore function to enable walking.

Clinical Trials

Although there is no cure for CMT, there are many clinical trials both completed and ongoing currently.  As many as 199 according to the website clinicaltrials.gov.

Participating in a clinical trial is an invaluable way to get involved in helping the research community cure CMT and related inherited neuropathies (INs). There are various types of clinical trials. Clinical trials are sometimes referred to as clinical studies or clinical research involving human volunteers that have been Food and Drug Administration (FDA) and European Medicines Agency (EMA) approved, the regulatory agencies for the U.S. and Europe. These agencies require that a potential therapy be extensively tested in large groups of human volunteers before it can receive approval for commercialization.

Clinical trials are a vital part of the scientific research process and essential for developing therapies to prevent, treat, reverse and cure CMT. 

Strict adherence to clinical trial material management protocols, in combination with proven GMP storage, clinical trial labeling, secondary packaging and global drug distribution allows Sentry to provide clinical trial outsourcing clients with a variety of flexible services.

For more information about how Sentry BioPharma Services can integrate your requirements into a scalable, secure, value-added clinical trial logistics solution, contact Sentry via email or by phone at 1-866-757-7400.

For more information please visit the following websites:

http://www.cmtausa.org/

https://clinicaltrials.gov/