Advanced therapy medicinal products (ATMPs) including gene therapy medicinal products, somatic cell therapy medicinal products and tissue-engineered products, are at the cutting edge of innovation and offer a major hope for various diseases for which there are limited or no therapeutic options and therefore, have been a subject of considerable interest. Given the increase in knowledge surrounding ATMPs and to make sure that the data being generated for these products with complex characteristics, it is important for robust clinical trials to be conducted in order to determine their risk-bene ..read more

Medsafe oversees pharmaceutical product regulation in New Zealand and operates under the Medicines Act 1981 which mandates sponsors to report substantial untoward effects of their medicines. The requirement for regulations are highlighted in the Guidelines on the Regulation of Therapeutic Products in New Zealand (GRTPNZ) which also has a Pharmacovigilance (PV) guidance document. This provides PV related obligations for sponsors that have their products on the market in New Zealand. In March 2024, updates to the guideline were published under “Guideline on the Regulation of Therapeutic Produc ..read more

Early-phase clinical trials for cell and gene therapy (CGT) products differ from those for other pharmaceuticals due to unique product features and previous clinical experiences, which have revealed substantial risks, including multi-organ failure, leukemia, and tumor development. Factors such as prolonged biological activity, immunogenicity, and invasive administration procedures contribute to these risks. The design of CGT trials requires careful consideration of safety, preclinical, and chemistry, manufacturing & control (CMC) complexities that are not typically seen with other pharma ..read more

Join DDReg at the Biotech Beach of the world! Meet the team at the BIO International Convention and DIA Global Annual Meeting to discuss clinical, regulatory, and safety requirements.  San Diego, often known as the “Biotech Beach,” has been a thriving center for biotech for several reasons. Its coastal location and the presence of numerous biotech companies and research institutes create an ecosystem that fosters innovation. This environment attracts both industry leaders and startups, leading to a vibrant community. San Diego’s combination of infrastructure, business expertise, and sci ..read more

Brazil is emerging as an attractive location for clinical research as it offers access to a range of patient demographics and medical facilities due to its diverse population and robust healthcare infrastructure. Conducting clinical trials for investigational products is a meticulous and highly regulated process, requiring sponsors to adhere to stringent guidelines. This ensures the safety, efficacy, and the ethical conduct of clinical trials. However, a robust and thorough knowledge base on regulations is needed to navigate the regulatory landscape for clinical trials. This includes protoco ..read more

Regulatory agencies across the globe are committed to ensuring that patients and the public have timely access to safe, effective, and high-quality medicines. They make significant developments, be it revision of guidelines to provide industry with robust support & guidance or expediting review and response times. Recently, the US FDA issued a guidance document for controlled correspondence incorporating new enhancements with the aim to improve the utility and efficiency of abbreviated new drug applications (ANDA) assessment cycles. The US FDA aims to decrease the number of ANDA assessme ..read more

Human gene therapy represents a groundbreaking frontier in medicinal products for treating complex diseases. This therapy aims to transform gene expressions or modify cellular properties for therapeutic purposes. Regulatory agencies support this innovation by ensuring the required guidelines are in place. For example, the US FDA stands as a guiding beacon for sponsors that wish to venture into Investigational New Drug Applications (INDs) of human gene therapies. With a focus on CMC information, the US FDA’s guidance document on “Chemistry, Manufacturing, and Control (CMC) Information for Hum ..read more

Advanced rare disease drug developers are companies or organizations that specialize in the research and development of treatments for rare diseases. Developing drugs for rare diseases poses unique challenges due to small patient populations, genetic variations, and the need for novel endpoint selection. In response, the FDA has allocated resources through initiatives like Prescription Drug User Fee Act (PDUFA VII) and the Accelerating Rare disease Cures (ARC) Program, launched in May 2022. Under the ARC Program, Center for Drug Evaluation and Research (CDER) established the Rare Diseases Te ..read more

The Association of Southeast Asian Nations (ASEAN) region presents an attractive opportunity for global pharmaceutical organizations that are looking to obtain market access in the respective ASEAN markets. This is due to its rapidly expanding healthcare market and harmonized regulatory processes. DDReg is thrilled to announce a significant stride in its commitment to supporting its customers for the ASEAN market by establishing its office in Singapore. This strategic expansion is tailored in providing global pharmaceutical companies with the support they need for seamless market access withi ..read more

In the emerging boom of biopharmaceuticals, the remarkable advancements in cell and gene therapy (CGT) have ushered in a new era of medical possibilities. These groundbreaking treatments have the potential to revolutionize medicine, offering hope to patients with previously untreatable conditions. However, with innovation comes the need for strong regulatory frameworks to ensure safety, efficacy, and accessibility. The US FDA and Health Canada sponsored an international meeting, organized by the ICH, in which Health Canada officials and industry key representatives met to develop harmonized ..read more