Policy Changes Could Cut Short a Golden Age of Drug Development
By Rachel King
2022 was an incredibly productive year for American scientists. Biopharmaceutical researchers conducted over 7,000 clinical programs, including more than 3,000 for cancer, 182 for cardiovascular diseases, 111 for Alzheimer's, and 57 for Type II diabetes. Importantly, 77% of these therapeutics originated at small biotech companies working toward their first breakthrough.
And there's just as much to look forward to in 2023, at least on the science side of the biopharmaceutical world. Companies are making major strides in addressing obesity, late-stage breast cancer and Alzheimer's, and are even seeing promise in using mRNA (the technology which made many of the COVID vaccines possible) to develop new vaccines for RSV, HIV and the flu.
Unfortunately, 2022 also saw several policy missteps that could moot all the excellent work taking place in labs nationwide. The good news is that it's still possible to reverse these adverse shifts.
Several blockbuster FDA approvals highlight last year's pharmaceutical achievements. In May, the FDA approved a once-a-week injection that improves blood sugar control in adults with type 2 diabetes.
In September, the FDA approved a new treatment for amyotrophic lateral sclerosis, better known as ALS. The oral medicine has been shown to extend the lives of those living with ALS by slowing the progression of the inevitably fatal disease.
2022 also brought many developments in CRISPR and gene therapy treatments, including clinical trials aimed at treating sickle cell disease. In addition, the FDA approved the first gene therapy for adults with Hemophilia B, and the first cell-based gene therapy for beta-thalassemia.
Still, the potential of these cutting-edge tools has hardly begun to be tapped. In the coming years, these transformational technologies could help combat everything from genetic diseases to solid tumors, blood cancers and HIV.
The prognosis for 2023, however, isn't uniformly favorable. Discovering and developing effective new drugs requires not only scientific know-how, but also a policy environment that supports innovation and patient access to new medicines.
Unfortunately, the current investment climate for drug development remains challenging. From the World Trade Organization's decision to waive intellectual property protections on Covid-19 vaccines in June to Congress imposing price controls on medicines in an August bill, policymakers at home and abroad aren't giving scientists the support they need.
Certain policy improvements, such as President Biden's Cancer Moonshot initiative, which dedicates more federal grants to oncology research, are welcome, but not nearly enough to counteract these threats to progress for patients. Without a supportive policy environment, scientists will struggle to make future breakthroughs like the Covid-19 vaccines.
The Economist once noted that "creating new drugs through biotechnology is at the risky end of a business in which superhuman strength and bottomless pockets are minimum requirements." Biotech companies are willing to accept this herculean task because they want to improve the lives of patients around the globe -- but they can only do so if the right incentives exist.
That's why it's essential that we foster a strong policy environment that allows drug development to keep up with its potential.
Rachel King is the interim CEO of the Biotechnology Innovation Organization. The co-founder and former CEO of GlycoMimetics, she also serves on the board of Novavax.
