From gene therapy to PDPRA: Pharma trends – 2020

As we enter the second quarter of 2020, COVID-19 continues to dominate our lives not to mention, the 24-hour news cycle. We cannot lose sight that while there is a global effort in the quest for a vaccine and effective treatment, the healthcare system continues to hum along. Companies continue to innovate in the face of challenges posed by non-COVID illnesses. Price reform also remains a critical issue, particularly given the millions who have lost insurance coverage due to COVID-related layoffs.

COVID aside, the industry is on target for quite a transformation with such evidence likely to be seen in the short-term. Here we highlight a few predictions for the remainder of 2020 related to innovation and policy as noted by some key industry experts.

Growth in the gene and cell therapy spaces

By altering or replacing the faulty genes that cause debilitating disease, gene therapy is bringing hope to patients who have had few or virtually non-existent treatment options in the past. Gene therapy shows promise for treating cancer, heart disease, hemophilia, muscular dystrophy, AIDS and cystic fibrosis, among other diseases. With over 800 gene and cell therapies in the biopharma pipeline back in January of 2019 – and less than 20 on the market today – the Food and Drug Administration predicts an influx in applications starting in 2020 and growing to 10-20 therapies per year by 2025.

Companies expected to wrap up development of gene and cell therapies in 2020 include:

• Biomarin: valoctocogene roxaparvovec for hemophilia A.
• Sarepta: gene therapy for Duchenne muscular dystrophy (DMD).
• Gilead and Bristol-Myers Squibb: CAR-T cancer treatments.

However, new challenges accompany gene and cell therapy development. Up-front investments are astronomical, and securing insurance coverage for the expensive resulting therapies requires innovation and careful strategy. Additionally, gene therapies don’t fit well within typical big pharma or biotech financial and operational structures, which requires organizations to re-evaluate and sometimes re-invent their business model in order to make room.

Innovation in drug delivery methods

According to Jeremy Drummond of MedPharm, drug developers will feel pressure to identify innovative methods of drug delivery. Aside from seeking more convenient drug delivery methods for patients – such as oral and topical in place of intravenous or intramuscular – Drummond predicts that there will be increased curiosity about the direct route to the brain through the nasal cavity. This prediction correlates with the increased prevalence of and focus on neurodegenerative diseases in the pharmaceutical industry.

Continued dominance by cancer-treating drugs

Because cancer is the second leading cause of death in the United States, it should come as no surprise that oncology drugs dominated novel drug approvals in 2019, and the trend is expected to continue into 2020. While both Gilead and Bristol-Myers Squibb work to develop chimeric antigen receptor T-cell immunotherapies (CAR-T), we’re seeing a shift from chemotherapy to precision medicine that’s showing promise for a myriad of cancers. Both are expected to help the industry move away from treatments that kill healthy cells and toward treatments that work with (and empower) the body to combat tumors while preserving healthy tissue.

The Leukemia and Lymphoma Society (LLS) plans to launch a global precision medicine trial in the summer of 2020 in hopes of bringing new, personalized treatments to market for children and adolescents with acute myeloid leukemia (AML). Perhaps the greatest commonality behind cancer research in 2020 is the underlying focus on precision medicine, allowing both researchers and physicians to better predict which patients will respond to immunotherapy.

Pricing reform will continue to put pressure on the pharma industry

Notice that “pricing reform is expected” is not one of the 2020 predictions. On the contrary, prescription drug pricing reform has been in the works since 2018 with little progress made to date, which leaves many experts predicting a similar outcome in 2020. While most components of the Prescription Drug Pricing Reduction Act (PDPRA) aren’t set to go into effect until 2021 or 2022, the Medicare Part B demonstration project is slated for November 2020.

Whether pricing reform takes effect this year or not, one thing is certain: the pharmaceutical industry will continue to feel the pressure of uncertainty surrounding the proposed changes.

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