PHARMACEUTICALS
Figure 2 - Differences between GMP & cGMP
In the US , new patents have a 20- year term from the filing date except when other factors affect patent duration , such as orphan drug and new chemical exclusivity . Since the United States Patent & Trademark Office ( USPTO ) takes about two years on average to approve applications , that truncates the market life of the patent . 7
Market research firm Statista reported that in 2022 the pharmaceutical industry is expected to have almost $ 50 billion prescription drug sales worldwide at risk due to patent expiration . 8
As more patents expire and market penetration of generics continues , putting pressure on pharma profit margins , pharmaceutical company executives have to assess the level of risk tolerance for the commitment , investment and time necessary to develop new pharmaceuticals
in-house . Some Big Pharma companies are looking to outside biopharmaceutical drug pipelines for possible future blockbusters .
According to McKinsey & Co ., biopharmaceuticals generate $ 163 billion / year , about 20 % of the total pharmaceutical market . This is the fastest growing part of the industry . Biopharma ’ s current annual growth rate is more than 8 %, double that of conventional pharma and this is expected to continue for the foreseeable future . 9
Orphan drugs upsurge
In 2022 , 54 % of FDA approvals for new medicines were for drugs to treat rare diseases , as compared to 34 % for orphan drugs in 2018 . This trend is expected to continue in 2023 . GlobalData predicts that at least 35 US regulatory decisions on drugs for rare diseases are on the horizon this year . 10
Big Pharma is actively pursuing orphan drug development , in part because the pool of patents for rare disease medicines is much smaller and the pathway to FDA approval can be expedited . Since these drugs may launch faster , patient safety and risk have to be considered at the early discovery / clinical development stages , not just during Phase II and III clinical trials .
Traditionally , therapies for rare diseases have been small molecules or biologics . Moving into the future therapies will include tissue and cellbased therapies , gene therapy and other innovative routes , resulting in additional safety risks . Going forward , even greater collaboration between clinical pharmacovigilance and manufacturing will be necessary to ensure the safety of medicines . 11
Patient safety risks
Ensuring patient safety is the highest priority and responsibility during the development and clinical testing of new drug candidates . Even the smallest amount of impurities and side products can compromise safety . New pharmaceutical agents must pass a battery of preclinical tests , as well as numerous safety hurdles .
Patient safety risk and protection outcomes need to be built in from the early decisions in R & D . Early clinical trials monitor patients to start proving the safety , efficacy and tolerability of new pharmaceuticals . This patient health monitoring continues throughout the later clinical testing phases and beyond . 12
Testing for impurities and other contaminants is crucial to drug development , not only to meet regulatory requirements but also to minimise the risk of development failures and adverse events . Designing comprehensive testing plans for all process phases is critical to success , as is selecting the highest-purity raw materials , reagents and excipients . An experienced industry supplier that offers customised testing and
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