Orphan drugs – small batches that demand attention

Nova set

“There is no disease so rare that it does not deserve attention.” This statement from  Orphanet, a portal for rare diseases and orphan drugs, makes that point very clearly. For patients suffering from a rare disease, orphan drugs and the special attention that they receive during the drug review and approval process can be both life changing and saving.

Thanks to Orphanet, we can learn much about these rare diseases that are serious, progressive and often chronic. If a disease affects a small number of people compared to the general population then it is defined as a rare disease. In Europe, a disease is considered to be rare when it affects one in 2,000 people. In the U.S., the ratio is closer to one in 1,500 and in Japan it’s approximately 1 in 2,500. About 7,000 rare diseases are known so far, and researchers are continually identifying new diseases that could be considered orphan diseases.

Among orphan diseases there are genetic and infectious forms, including autoimmune diseases and rare cancers. For many rare diseases, the specific cause has not yet been identified, which may be a reason why cures are still so hard to find. Encouragingly though, an appropriate treatment and medical care can improve the quality of life of those affected and can extend their life expectancy1.

People affected by rare diseases take on various challenges including a search for the correct diagnosis, receiving appropriate and adequate medical treatment, as well as striving for improved social integration and independence. Development of medical treatments for rare diseases are covered by dedicated regulations in many markets such as the EU, US, Japan, Australian and Singapore. In the U.S.2 and Europe3, an orphan drug designation must be obtained before a marketing authorization is filed.

The right packaging options for orphan drugs are crucial to help the pharmaceutical industry meet the specific requirements of this type of drug and to be by the side of the patients living with various health challenges. Batch sizes in the manufacturing of these medicines are certainly smaller compared to most other drug formulations. As such, Fill-Finish processes can benefit from tailored solutions that offer high quality primary packaging components in smaller packaging sizes.

West’s Ready Pack® component system is a one source solution for product development drug packaging and is suitable for small quantity filling of high value drugs. These components are available in a variety of sizes that are compatible with one another and proven to ensure container closure integrity. Delivered in a sterile, ready-to-use format, West Ready Pack components are available quickly and conveniently.

West Ready Pack provides a solution for risk mitigation and change control, so the drug product and its packaging maintain a consistent profile from clinical stages through commercial development. For more information, please view the West website.

Kofler Heike 354

Author
Heike Kofler, Ph.D.
Manager, Technical Customer Support
Heike.Kofler@westpharma.com

 

 

References:

1: Orphanet
http://www.orpha.net/consor/cgi-bin/index.php

2: Developing Products for Rare Diseases & Conditions by the U.S. Food and Drug Administration (FDA)http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ucm2005525.htm

3: Orphan designation by the European Medicines Agency (EMA) http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000029.jsp&

Small-scale injectables packaging: a new option from Daikyo and West

Daikyo

Small is the new big. Growth in specialty treatments that target smaller patient populations means less emphasis on economies of scale and more focus on adaptable and modular manufacturing. High-value drugs may be manufactured in hundreds or even tens of thousands of units a year instead of in millions. Increasingly, products are piloted and manufactured using the same equipment. This changing backdrop throws into sharp relief the constant pressure that manufacturers are under to prepare and package drugs for trial as quickly as possible: today’s clinical or early-stage injectables packaging formats are more likely than ever to become tomorrow’s commercial presentations.

In this context, it is critical for biotechnology developers and other pharmaceutical operations engaged in clinical trials and scale-up to have at their disposal a range of closure choices to meet different small-scale needs. West has now added Daikyo’s new Ready to Use Validated® (RUV) to its existing offerings for small-scale manufacturing environments, extending customer options further in this area.

Sterile, ready to use Daikyo RUV stoppers are now available directly from West locations in North America and Europe with low minimum order quantities and accelerated lead-times. Daikyo RUV stoppers incorporate full vision inspection, subvisible particle specifications, and Drug Master File (DMF) support in the U.S. and Canada. They are based on a modern elastomer formulation that meets USP, Ph. Eur, and JP compendia requirements and lends itself to a range of applications, including those requiring multiple punctures.

Daikyo RUV small packs join a select group of premium West and Daikyo closure products in small scale formats that can be ordered directly from West locations in North America and Europe. These include West’s Ready Pack®, an offering that integrates ready to use, sterile Daikyo Crystal Zenith® vials, glass vials, seals and stoppers, as well as West NovaPure® components available in modern bromobutyl and chlorobutyl formulations. Small-scale manufacture has a range of needs and preferences to be met, and West provides a rich portfolio from which to select the best match for a specific project.

Our customers tell us that the advantages of high-quality pharmaceutical closures supplied on a small scale are clear. Lighter cartons containing fewer units can be shipped quickly and easily to where they are needed and are attractive in terms of lab and pilot-scale ergonomics. They can be stored and managed more readily than large packages and help avoid waste. Ready to use, sterile components furthermore eliminate the extra step of sterilization, which may be cumbersome, time-consuming, risky, or even beyond the scope of some sites.

To discuss your small-scale packaging needs and find out more about Daikyo RUV, contact your West Sales Accounts Manager.

Deutsch, Michelle 002 LR

 

Michelle Deutsch

Product Manager, Vial Containment

Michelle.Deutsch@westpharma.com

 

West Gives Back—One Employee’s Hands on Experience

In May of 2015, Exton announced their West Without Borders focus on the Year of the Veteran, with The Travis Manion Foundation named as one of the recipient charities. Along with this charity came a life changing opportunity for one of our employees. Laura Pitt, Manager, Community Affairs, was offered a chance to travel to Guatemala with the Travis Manion Foundation and 15 family members of veterans who have lost loved ones in military conflicts. Their mission was to help a family in extreme poverty, so Travis Manion partnered with Casos por Cristos—an organization dedicated to providing support in impoverish areas—to build this family a home in three days.10007068_10153991203670704_718768065_n

The house they were building was on the side of a mountain in the outskirts of San Raymundo.  “It was amazing how the community came together. There was no jealousy from those who wouldn’t directly benefit, everyone was so excited for the family that did,” said Laura. In order to construct a foundation for the home, members of the community had to work together to carry all the materials required to make concrete up the mountain to the site. The women in the community carried pails of water on their heads to fill three 55 gallon buckets. The children brought the stone from the bottom of the hill to the site in small containers they found. The men carried the big bags of the cement mixture to the site.

The first day of work focused on getting the concrete foundation in place and building the lumber walls. “It was a challenge to build the walls, because trucks were constantly driving on the roadway and the crew would have to move the walls each time so they could pass through,” said Laura. Laura was part of the lumber team and helped to build and place the siding on the walls and then ultimately placed the roof on the house. On Day 3, the crew went to the local market to buy food, clothing, toys and supplies for the family and the community.  They worked on the interior of the home, hooked up electricity, hung windows and a front door as well as built shelving in the 2 bedrooms.

The last day was Laura’s favorite because the community came together to present the house to the family. The crew also presented the family with photos of their loved ones who had lost their lives.  These framed pictures were placed on a shelf with the message that these veterans would watch over the house, the family and their community.

The crew also had the opportunity to visit the local school where they donated more supplies and West donated new instruments for the school’s band. “If children are even lucky enough to attend school in Guatemala, it is only government funded until 3rd grade, Laura said.  “After that, the family must pay  and in most cases they can’t afford to.”

West without Borders, West’s employee-led fundraising program, supports charities and communities around the world in which our employees live and work.  The campaign kicked off this week at the global headquarters in Exton. This year’s selected charity is the Leukemia and Lymphoma Society (LLS). In addition to individual employee support, West will sponsor both the Philadelphia and Chester County “Light the Night” walks in October 2016.  Stay tuned to the West blog to learn more!

Iris Bavaj named West’s Healthcare Businesswomen’s Association (HBA) Luminary

Iris Bavaj Luminary

We are pleased to announce that Iris Bavaj, Senior Director, Quality Operations, Europe, has been recognized by the Healthcare Businesswomen’s Association (HBA) as West’s 2016 Luminary. Iris is a key leader at West, responsible for the Quality Operations teams in West Europe, and for leading the Quality team for the critical Global Network Optimization Transfer Project.

“We are celebrating Iris and her contributions to West because of her outstanding performance as a leader, manager and mentor within our Quality organization. She leads by example, working with customers to ensure their needs are met , while providing counsel and coaching to her team so they are able to grow and develop, “said Andy Polywacz, VP, Quality and Regulatory Affairs, “Iris exemplifies West’s ‘by your side’ spirit, and we are proud to honor her as this year’s HBA Luminary from West.”

HBA Luminaries are professionals in the healthcare industry that demonstrate leadership, act as mentors to advance other women’s careers, contribute significantly to their organization, exhibit dedication to the healthcare industry and are a true example of top talent.

“I am truly honored to be recognized by West and to celebrate with other female leaders from across our industry at the HBA Woman of the Year event,” said Iris. “As one of the founding members of our WIN group in Germany, I am encouraged to see that West is working to support and develop women in our organization, as there is so much benefit to having diversity at every level within our company.”

Iris will be formally recognized at the HBA 27th Woman of the Year event that takes place in New York City on Thursday, May 12, 2016, and will celebrate with the Exton-based WIN team on May 10th.

West’s engagement with the HBA is part of the broader West Women Investing and Networking (WIN) Group initiative, which is dedicated to fostering the development of the women of West. If you are interested in joining an existing WIN Team (Exton, Germany or South America), please contact Karsyn Kraft.

Congratulations to Iris on this well-deserved recognition!

Extractables and Leachables – Assessing Risk in a Complex Landscape

At West, we often get asked “What extractable compounds are present in your products?”, “What are the current requirements for Extractable and Leachable testing?” and “How do I make sense of all of this data and get the regulatory authorities what they want?”

Labs 143_Lattice-0031

It is not surprising that there is still confusion on the topic. Guidances, where available, do not give step by step instructions. In addition, drug products and other drug product contact materials are getting more and more complex. So, where should you start?

We know from ICH Q9 that, “The evaluation of the risk should be based on scientific knowledge and ultimately link to the protection of the patient.” Therefore, we should view all work done to characterize extractables and leachables in terms of patient risk.

Starting with component selection we can ask questions such as:

  • What information am I able to obtain from my vendor as to material characteristics and safety?
  • Does this material have inherent properties that are hazardous? If so, what are the main concerns and does data exist to confirm or deny presence?
  • What is the drug product contact (time and temperature) and how can that increase or decrease patient risk?

With this information, you have a starting point in order to select the packaging materials that best fit your needs.

Now that you have selected the appropriate components, you can assess extractable information gaps and design fit for purpose analytical evaluations. The point here is not to do less testing, but rather, more focused testing in order to answer any outstanding questions.

Once all the pertinent extractable data has been obtained to understand your materials, you can further organize the information based on risk in order to decide which compounds should be monitored over the shelf life of the drug product and which should not (Leachables phase). Some questions to ask at this stage are:

  • What is the toxicity of the analytes?
  • What are the relative amounts, volatility and solubility of the compounds? How might that impact migration into your drug product?

After the organization of compounds is completed, you can use those justifications to define a leachables testing plan. Methods used to quantitate leachables should be validated and detection/quantitation levels should be meaningful in terms of patient safety. USP <1664> tells us that, “The completeness of an extractables assessment can only be judged against the overall goals of the assessment.” By defining your risks up front and developing thoughtful test plans, you can ensure that your decisions are both data-driven and patient-focused.

Orme Lauren 2 _D7A0120_v2

Lauren Orme, PMP
Senior Technical Account Specialist
Lauren.Orme@westpharma.com

 

 

 

References:

  1. USP-NF <1664> – Assessment of Drug Product Leachables Associated with Pharmaceutical Packaging/Delivery Systems
  2. ICH Harmonized Tripartite Guideline – Quality Risk Management Q9

Employees at West in Germany Kick-off West without Borders 2016

IMG_2089

On May 4, 2016, team members in West’s Eschweiler, Germany site launched their West without Borders (WWB) campaign for the year. Stephan Selke, VP Finance, EU, hosted the exciting event and welcomed board members from recipient organizations, “Zwischen Uns“ and “FortSchritt” as well as two young representatives from the organizations. Both Zwischen Uns and FortSchritt are no strangers to West. Employees in Germany have supported FortSchritt since 2012 and have witnessed the organization evolve and establish itself in the community. The organization offers conductive therapy, which integrates medical knowledge with educational methods to enable children and young adults to learn how to gain control over their movements. Individuals with mobility disorders, neurological conditions, physical and mental impairments strongly benefit from conductive therapy as they are taught to see themselves as active and self-reliant participants in the world. Zwischen Uns, who also received WWB support last year, accommodates a group of young adults with various disabilities or mobility impairments who have set out on a path to independence and autonomy. The organization operates a handicraft shop and painting studio, as well as a kitchen and housekeeping area–offering a workplace to individuals with physical challenges.

Money raised throughout the month of May will finance the following projects:

  • Fortschritt has rented a new therapy room that requires major renovations. Once completed the organization will be able to offer additional conductive therapy sessions. The long term plan is to also include adult patients with Parkinson’s disease or those who have suffered a stroke.
  • Zwischen Uns has rented an apartment within the same building to accommodate its painting group. To allow for optimal care and supervision of the young adults with special needs, the number of full-time employees and volunteers will increase to six.

West Germany looks forward to a new, exciting West without Borders campaign and thanks all employees who will participate and show their generosity.

Nadine Gerstler
Communications Specialist
Nadine.Gerstler@westpharma.com

 

 

Mike Treadaway Completes London Marathon–Raising Funds and Awareness at the Same Time

Mike_marathon

A huge congratulations to our own Mike Treadaway, Vice President & General Manager, Contract Manufacturing, who finished the 35th annual London Marathon on April 18, 2016! Mike joined over 39,000 athletes who participated in this prestigious event—completing the race in 4 hours, 31 minutes. Mike combined his passion for running and goodwill by raising more than $3,000 for JDRF. JDRF (formerly known as the Juvenile Diabetes Research Foundation) is a major non-profit organization dedicated to funding type 1 diabetes research (T1D), with the vision of “a world without type 1 diabetes.”

With the completion of the London Marathon, Mike has now completed 5 of the 6 Abbott World Marathon Majors and is well on his way to achieving his goal of completing all 6 by running in the Tokyo Marathon in 2017. The Abbott World Marathon Majors consists of six of the largest and most renowned marathons in the world: Boston Marathon, Virgin Money London Marathon, BMW Berlin-Marathon, Bank of America Chicago Marathon, Tokyo Marathon, and TCS New York City Marathon. The organizers of these events are united in their effort to advance the sport, raise awareness of its elite athletes, and increase of the level of interest in elite racing among running enthusiasts.

The Tech Group, West’s Contract Manufacturing group, is a preferred supplier to several market leaders for continuous glucose monitoring systems and insulin pens that improve quality of life for patients living with diabetes. Mike’s commitment to giving back is a great example of West’s philanthropic culture.