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The promise of new therapies is tempered by the need for affordability, safety, and ethics.
Researchers continue to report exciting new discoveries in science and medicine that have the potential to improve life and address challenges facing society. The intersection of scientific opportunity and business profitability can, however, lead to ethical conflicts.
Emerging scientific methods such as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) genome editing technology, which can be used to modify genes in living cells and organisms, offer the promise of correcting genetic mutations to treat genetic causes of disease. Researchers and ethicists generally agree that gene editing methods are suitable for research applications, but not for reproductive purposes.
Concerns about the misuse of gene-editing processes were realized when a scientist in China announced in November 2018 that he used CRISPR techniques to edit the genes in human embryos of twin girls with the intention of protecting them against the human immunodeficiency virus. This controversial work was denounced by scientists worldwide; in December 2019, a court in China sentenced the scientist to three years in prison for working outside the boundaries of scientific and medical ethics.
This experiment illustrates the ethical, safety, and legal issues associated with promising new scientific discoveries, and many unanswered questions associated with their long-term use. FDA has pursued enforcement action against dozens of unapproved therapies and treatments based on emerging sciences. In announcing a permanent injunction against a Florida-based facility for selling adulterated and misbranded cellular products, FDA noted many offenders selling unproved stem-cell products are taking advantage of vulnerable patients “by leveraging the widespread belief in the eventual promise of these products, flouting the statutes and our regulations” (1). FDA and industry experts also recognize the challenges with developing emerging therapies. FDA also noted that while stem cell products have potential to improve human health, “… that potential will never be fully realized if careful scientific work and thoughtful clinical investigation supporting the safety and efficacy of these products are not conducted.”
In the cover story in this issue, experts from bioprocessing equipment manufacturers and contract development and manufacturing organizations emphasized that standardized commercial manufacturing platforms and processes are needed to ensure that these therapies can be delivered in a safe, cost-effective manner.
Emerging therapies are expected to contribute to the growth in global sales of prescription drugs from an estimated $839 billion in 2019 to $1.18 trillion in 2024, a compound annual growth rate of 6.9%, according to Evaluate Pharma. For the top 100 products by sales, Evaluate Pharma analysts expect biotech product sales to overtake conventional product sales for the first time. In addition, biotech products are forecast to represent 32% of prescription drug sales by 2024, up from 28% in 2018 (2).
Emerging therapies represent a growing share of the prescription drug market; however, policy decisions about drug affordability lag rapid scientific developments. “The advances in cutting edge science are, for now, outpacing the traditional pricing and reimbursement systems the industry has been built on,” Evaluate Pharma reported. “This disconnect is leaving both patients and payers wondering how accessible these life-altering products will be.”
1. N. Sharpless and P. Marks, “Statement on Stem Cell Clinic Permanent Injunction and FDA’s Ongoing Efforts to Protect Patients from Risks Of Unapproved Products,” Press Release, www.FDA.gov (June 25, 2019).
2. Evaluate Pharma, World View 2019, Outlook to 2024 (12th edition, June 2019).
Vol. 33, No. 1
When referring to this article, please cite it as: R. Peters, “Can Policy Keep Pace with Science and Discoveries?” BioPharm International, 33 (1) 2020.