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“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 click here.
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) https://www.fda.gov/industry/developing-products-rare-diseases-conditions
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&
Gene therapies are developed by modifying a person’s genes and are often targeted to treat complex and chronic diseases. Due to advancements in science and research, gene therapy is positioned to transform treatment options for patients who are living with these incurable diseases. There is a growing pipeline of gene therapies globally, with 352 global trials underway at the end of 2019, up 12% from 2017.<sup>1.</sup>
Competition is increasing in the biopharmaceutical pipeline due to a growing number of new entrants, more players are bringing their drug all the way to market and many companies are utilizing accelerated development pathways, making it an even faster race to market. If you are like many other newly formed biotech companies, you’re doing your best to manage many activities critical to your success with limited resources, all while trying to accelerate your path to market.
The European medicines regulatory system is based on a network of around 50 regulatory authorities from 30 EEA (European Economic Area) countries (27 EU Member States plus Iceland, Liechtenstein and Norway), the European Commission and the EMA (European Medicines Agency). For a medicinal product to be available on the market it needs to have a marketing authorisation. The marketing authorisation application (MAA) must provide evidence of the efficacy, safety and quality of the medicine. When applying for a marketing authorisation for a medicine, the marketing authorisation application is submitted to a regulatory authority, which will assess the medicinal product’s pharmaceutical and chemical quality, efficacy and safety, as well as its risk-benefit ratio.
Biologics is a rapidly growing market, with a robust R&D pipeline bringing innovative therapies to treat patients. Today, there are 4,000 more active biologic programs in the pipeline compared to 2015, with biologics development programs growing 2.5 times faster than small molecules. New entrants into the biologics market are driving this rapid growth. As of 2018, emerging companies represented 84% of all early phase research.<sup>1</sup> Not only is this segment driving R&D growth, but they are having commercial success in bringing their drugs to market. In 2018, 65% of new drugs launched were patented by emerging companies.<sup>1</sup>
The definitions of <em>personalized medicine</em> vary. However, all of them indicate that the once popular therapy approach of “<em>one drug fits all</em>” is outdated. The Council of the European Union defines personalized medicine as <em>“[p]roviding the right treatment to the right patient, at the right dose at the right time.”</em><sup>[1]</sup>.
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