Recognizing Rare Disease Day and the Importance of Pharmaceutical Good Manufacturing Practices in Orphan Drug Compliance
Every year, on the last day of February, the world comes together to recognize Rare Disease Day, raising awareness for the more than 300 million people living with rare diseases worldwide.
For these patients, treatment options are often limited, making every medication critical to their well-being. In this high-stakes environment, Good Manufacturing Practices (GMP) play a vital role in ensuring that life-changing treatments are produced with the highest standards of quality, safety, and consistency.
Why GMP Matters for Rare Disease Pharmaceuticals
Unlike mass-market drugs, orphan drugs—medications developed specifically for rare diseases—represent a niche but extremely critical segment in the global pharmaceutical industry. Orphan drugs are produced in small batches and are capable of changing the quality of life for patients with conditions that are rare and sometimes overlooked.
This limited production scale presents unique challenges in manufacturing. Although compliance to Pharmaceutical Good Manufacturing Practices (GMP) is important in all drug manufacture, adherence to Pharmaceutica Good Manufacturing Practices for orphan drugs is essential. Here’s why:
Regulatory Compliance
Pharmaceutical and drug manufacture, including orphan drugs is subject to stringent regulations, including US Code of Federal Regulations, (21 CFR Parts 210 & 211), European Medicines Agency (EMA) , and Health Canada’s Food and Drug Act Regulations. Any deviation from these regulations can result in serious consequences that may result in unsafe products being placed in the market.
Risk Mitigation in Small-Batch Production
Because rare disease treatments often involve complex formulations that require limited production runs and experimental therapies, the importance of conformance to specifications is significantly higher. Pharmaceutical Good Manufacturing Practices ensure every batch, regardless of size meets specifications required for safe, unadulterated drugs that have known treatment outcomes.
Patient Safety & Efficacy
Patients with rare diseases rely on precisely formulated and highly controlled medications. Pharmaceutical Good Manufacturing Practices reduce the chance of errors in manufacture, and enforce quality control or quality assurance at every stage of production—from raw materials to final packaging—thus maintaining consistency and reliability.
Challenges in Pharmaceutical Good Manufacturing Practice Compliance for Orphan Drug Manufacture
When manufacturing orphan drugs, compliance to Pharmaceutical Good Manufacturing Practices comes with specific challenges:
Production Methods
Orphan drugs may require changing known production methods to adapt an existing therapy to a new or novel use.
Modality
“Rare Disease” implies a small number of patients, so drug manufacture is more likely to be carried out on a smaller scale than larger, more common, proven therapies.
High Manufacturing Costs
Small-scale production often leads to higher costs, requiring companies to balance efficiency with quality assurance.
Complex Supply Chain Management
Sourcing high-quality APIs and raw materials for orphan drugs can be challenging, requiring strict supplier qualifications and audits.
Regulatory Hurdles
Adherence to Good Manufacturing Practices means the drug development company has followed regulatory to allow for Fast Track, Breakthrough Therapy, Accelerated Approval and Priority Review of rare disease drugs to expedite their review and final regulatory approval.
How GMP Certification Supports Pharma Companies Developing Rare Disease Orphan Drugs
For pharmaceutical manufacturers, adhering to GMP is not just about compliance—it’s about ensuring patient safety and treatment efficacy.
Pharmaceutical Good Manufacturing Practice compliance provides:
Regulatory Readiness
Meeting US Code of Federal Regulations, European Medicines Agency, and Health Canada regulations ensures smooth approval processes and minimizes compliance risks.
Market Access & Credibility
Retailers, healthcare providers, and government agencies require compliance to Good Manufacturing Practices to ensure product safety.
Quality Assurance Across the Supply Chain
Implementing Pharmaceutical Good Manufacturing Practices safeguards drug production, from sourcing of raw materials, through final packaging and distribution.
Prioritizing Pharmaceutical Good Manufacturing Practices to Support the Rare Disease Community
On Rare Disease Day, we recognize not just the patients and families impacted by these conditions but also the scientists, manufacturers, and quality teams working tirelessly to develop safe and effective treatments. By complying with Good Manufacturing Practices, pharmaceutical companies help ensure that every medication for rare diseases is manufactured with safety and reliability that patients deserve.
Intertek supports pharmaceutical companies in meeting Good Manufacturing Practice (GMP) compliance by providing expert auditing, certification, and quality assurance solutions. Our global network of GMP specialists helps ensure adherence to regulatory requirements, risk mitigation in small-batch production, and quality control at every stage of manufacturing. By partnering with Intertek, pharmaceutical companies can enhance regulatory readiness, improve supply chain integrity, and maintain the highest standards of safety and efficacy for orphan drug production. Learn more about our GMP services: Intertek GMP Compliance.
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