Eleven potential blockbuster drugs offer hope, threaten budgets

Crowned by a potential cure for severe hemophilia A, which could become the most expensive drug ever, a new list of 11 medicines expected to generate $1 billion-plus in annual sales by the end of 2024 or earlier throws into stark relief the growing tension between medical innovation and society’s ability to pay for it. The 2020 Cortellis Drugs to Watch list, including medicines both approved and likely to be, points to a future of ongoing conflict between payers and industry spurred by fundamental disagreements.

The list and corresponding analyses focus on the treatment and possible cure for chronic, progressive and often debilitating diseases and conditions, including drugs that target the indications of breast cancer, multiple sclerosis (MS), migraine and type 2 diabetes.

The drugs listed have been almost universally accelerated in their development by orphan drug status or other designation intended to speed their path to market. There is a high level of expectation of commercial success for each of these drugs – despite the fact that most medicines on the list are entering or are poised to enter markets already crowded with competitors, meaning they’ll face substantial pressures to differentiate themselves. Many are expected to tout improved safety vs. alternative therapies, especially regarding cardiovascular risk, while others will seek to highlight novel mechanisms of action or even curative potential.

More than half of the medicines on the list are biologics, a fast-growing and increasingly expensive segment of prescription medicines for which regulators and payers alike have sought to rein in costs during recent years. Gene and cell therapies – represented on this year’s list by hemophilia A therapy Valrox (Biomarin Pharmaceutical, Inc.) and lisocabtagene maraleucel (Bristol-Myers Squibb Co) for large B-cell lymphoma, respectively – have drawn particular attention for their high costs. If approved, Valrox will be crowned as the first potential curative approach for severe hemophilia A. The one-and-done therapy will be transformative as it corrects the genetic defect underlying the condition and eliminates the need for blood transfusions and FVIII replacement therapy. If, as expected, it takes a leadership position it could become the most expensive drug ever to reach the market with a cost of $2.5 million – $3 million per treatment.

Despite the ongoing tension among scientific innovation, commercial motive and public good, as medical research and the pursuit of innovative medicines continues to accelerate, the success of these drugs will bring hope to many – if they can afford them – and seek to provide the benefits of novel mechanisms of action alongside curative potential.

The full list and analysis are freely available at: www.bioworld.com/drugstowatch2020


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Methodology for the Cortellis Drugs to Watch 2020

Data for this list were compiled from Cortellis, the suite of life sciences intelligence solutions from Clarivate Analytics. Cortellis includes the broadest and deepest range of sources of intelligence across the R&D lifecycle, including annual filings, drug pipelines, clinical trials, patents, chemistry, deals, conferences and company announcements. Drugs in phase 2 or phase 3 trials, at pre-registration or registration stage, or already launched early in 2020 were selected for analysis; drugs launched prior to 2020 were excluded. The dataset was then filtered for drugs that had total forecast sales of $1 billion or more in 2024. This filtering process produced a list of drugs which was then manually reviewed by our in-house experts and consultants to determine whether these products were likely to go to market in 2020, based on factors such as the company’s expected approval or launch dates. Following this manual review, 11 drugs to watch for 2020 were determined. Each drug was subsequently researched and evaluated in its individual context, including clinical trial results, regulatory status, the market for each drug (including analysis of competitor drugs) and regulatory designations (e.g., Orphan Drug, Priority Review). Sources used included SWOT analyses compiled by Cortellis editors, biopharma company press releases and other publications (e.g., annual filings), peer-reviewed publications and Cortellis sales data (sourced from Refinitiv I/B/E/S). Data extraction – January 20, 2020.