Annals of the New York Academy of Sciences is the premier publication of the Academy, offering review articles in topical areas and proceedings of conferences. Learn more and order copies at www.nyas.org/annals.
Folate Status in Women and Neural Tube Defect Prevention
Neural tube defects (NTD) are one of the most common types of congenital malformations characterized by incomplete closure of the embryonic neural tube. Anencephaly and spina bifida are the two most common types of NTD that affect the brain and spinal cord, respectively. These defects occur between 21 and 28 days after conception when most women are unaware of their pregnancies. NTD contribute to stillbirths, as well as neonatal, infant, and under-five mortality, along with life-long disability and substantial health care costs among those who survive.
The March of Dimes Global Report on Birth Defects, using methods developed by Modell and colleagues, published a global estimate of the number of live births with NTD. According to this report, an estimated 324,000 births (2.4 per 1000 live births) were affected by NTD globally during the year 2001, which contributed to over 2.3 million disability-adjusted life years.
This special Ann NY Acad Sci issue was developed in support of the technical consultation “Folate Status in Women and Neural Tube Defect Prevention,” convened by the Micronutrient Forum and supported through Nutrition International by a grant provided by the Bill & Melinda Gates Foundation. The Bill & Melinda Gates Foundation also provided support for open access of the 10 papers comprising the special issue.
In the special issue, Blencowe et al. extended the Modell methodology and estimated 260,100 NTD-affected pregnancies worldwide in 2015, of which approximately 50 percent were electively terminated. Over 75 percent of NTD-affected births resulted in death by 5 years of age. The global prevalence of NTD was estimated by Blencowe and colleagues to be 1.86 per 1000 live births in 2015, while varying in different regions of the world between 0.75 and 3.12 per 1000 live births.
The other individual papers cover many areas related to folate status and prevention of NTD, including a public health approach for preventing neural tube defects; the safety of folic acid; investment case for folic acid fortification in developing countries; and a proposal for an integrated strategy to support and accelerate birth defect prevention.
Folate Status in Women and Neural Tube Defect Risk Reduction
Ann NY Acad Sci 1414: 1–136. February 2018.
Getting Complex Drugs to Market: the Regulatory and Scientific Hurdles
The rise of biotechnology and nanotechnology has accelerated the development of complex drug products and their copy versions, the growth of which is expected to increase significantly. While regulatory guidance for authorization of generic versions of small molecule drugs is well established, guidance for complex drug products is still evolving. Experts from various backgrounds and parts of the world gathered at the New York Academy of Sciences to discuss the current regulatory frameworks, share experiences and identify open issues. The outcome of these discussions and the identified outstanding challenges are reported in the Ann NY Acad Sci special issue “Equivalence of Complex Drug Products: Scientific and Regulatory Challenges.”
The complex drug landscape classifies products based on the challenge of assessing pharmaceutical equivalence (PE) and bioequivalence (BE) of two drug products. According to this classification, complex drug products can be divided into two categories: (1) products with complex active ingredients and/or complex formulations for which both PE and BE are difficult — if not impossible — to demonstrate, and (2) products with complex routes of delivery, dosage forms or complex drug–device combinations, where either BE or PE is difficult to establish. The first category includes the majority of nonbiological complex drugs (NBCDs).
NBCDs are fully synthetic materials: They are medicinal products but not biological medicines, where the active substance is not a homogeneous molecular structure but consists of different closely related and often nanoparticulate structures that cannot be isolated and fully quantitated, characterized and/or described by physicochemical analytical means. The composition, quality, and in vivo performance of NBCDs are highly dependent on the manufacturing processes of the active ingredient, as well as (in most cases) the formulation. Although the term NBCDs is not officially recognized by the European Medicines Agency (EMA) or the U.S. Food and Drug Administration (FDA), it is gaining traction in the scientific community.
This special issue presents seven papers discussing the challenges of evaluating and regulating complex biological and non-biological drugs in a number of contexts, including the example of the World Health Organization standards for biotherapeutics and biosimilars, and specific examples of complex drugs such as iron sucrose, used in the treatment of iron-deficiency anemia, and glatiramer acetate, a complex nonbiological drug for treatment of multiple sclerosis.
Equivalence of Complex Drug Products: Scientific and Regulatory Challenges
Ann NY Acad Sci 1407: 1–89. November 2017.
Additional Recent Annals Volumes
Musculoskeletal Repair and Regeneration I & II
The Year in Diabetes and Obesity
Ann NY Acad Sci 1411: 1–183. January 2018.
Myasthenia Gravis and Related Disorders I & II
Ann NY Acad Sci 1412: 1–177 and Ann NY Acad Sci 1413: 1–169. January and February 2018.