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White Paper: Insight into Orphan Drug Formulations and Regulations

The US Orphan Drug Act of 1983 characterizes an orphan disease as a rare medical condition that affects a population of fewer than 200,000 people. Rare diseases have various causes— most notably, genetics. However, the National Institutes of Health (NIH) states, “many rare diseases, including infections, some rare cancers, and some autoimmune diseases, are not inherited. While researchers are learning more each year, the exact cause of many rare diseases is still unknown” (NIH, 2017).

The term “orphan disease” resulted from pharmaceutical companies not having the resources to design and scale up formulations to treat these diseases, due to the lack of financial support for such highly specialized programs. The Orphan Drug Act (ODA) allows for the necessary financial backing of pharmaceutical companies to develop treatments for rare diseases (NIH, 2017), giving special status to a drug or biological product (drug) upon request of a sponsor. This status is referred to as orphan designation or orphan status.

Topics: Resource Center Oral Solid Dose Formulations Regulatory Compliance Orphan Drugs

Case Study: Orphan Drug Product

According to the Food and Drug Administration and the Orphan Drug Designation program,  orphan status is applicable to drugs and biologics which are defined as “those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.” Due to the high costs and low demands of the drug product, it is important to efficiently use your API supply during the formulation process. This case study presents how Alcami successfully helped a client overcome an API supply shortage using Quality by Design studies to successfully formulate an orphan drug product. 

Topics: Drug Product Oral Solid Dose API Formulations Resource Center Thought Leadership Orphan Drugs

Understanding Pharmaceutical Packaging

For oral solid dose (OSD) pharmaceuticals, decisions regarding the type of packaging used are critical to the success of a product. The best formulated product in the world can fail if it is contained in an inappropriate package. So how does one decide which packaging to use for a product?

First and foremost, the package has to protect the product from damage or degradation. Different barriers can protect from moisture, light, or gases, such as oxygen or carbon dioxide. Blistered products may use cold-formed aluminum to block light completely. Vials, at times, use light protective sleeves for the same purpose. Bottled products may require the use of a coil to keep tablets from breaking during shipment. Alcami works with clients to customize the best package configuration for product protection.

Topics: Thought Leadership Serialization Packaging Pharmaceutical Packaging Oral Solid Dose

Panel Discussion: Oral Solid Dosage Manufacturing Process 

Alcami recently contributed to Pharma Horizon's first Panel Discussion of 2018, dedicated to the Oral Solid Dosage (OSD) Manufacturing Process. 

The topics discussed include main challenges that manufactures of OSD forms have to face, such as quality, safety and productivity, as well as technical advancements, production management, operational excellence aspects, tech transfer, and packaging operations.

Topics: Thought Leadership Editorials Manufacturing Oral Solid Dose

Editorial: Oral Solid Dosage Manufacturing Roundtable

Alcami recently contributed to a roundtable featured in Pharmaceutical Outsourcing magazine titled "Oral Solid Dosage Manufacturing Roundtable." The article discusses a variety of topics including current critical issues facing the industry in regards to oral solid dosage manufacturing, new manufacturing technologies/processes, the affect that globalization of the pharma industry has had on oral solid dose manufacturing, and more.

Topics: Thought Leadership Editorials Oral Solid Dose Manufacturing