If your only tool is a hammer, everything looks like a nail. Such was the state of the pharmaceutical industry a decade ago. Gone are the days of pharmaceutical companies predominantly specializing in single modalities such as traditional vaccines, monoclonal antibodies, plasma-derived therapies or small molecules. Today’s Big Pharma, fueled by the acquisition of countless startup companies, are ensuring their toolboxes are flush with the trusty tools of the past decades. In addition, Big Pharma companies are leveraging the power of new tools in the form of advanced therapeutic modalities. Consequently, the pipelines of leading companies in the pharmaceutical industry are increasingly comprised of complex new modalities such as cell therapies, mRNA, non-coding RNAs (ex., oligonucleotides and siRNA) and various gene therapies.

These new therapies lead to significant advancements for patients and, in some instances, carry the promise of a cure administered in a single dose. Several of these products are now on the market, including CSL’s hemophilia B gene therapy leading the pack at $3.5 million per patient, topping Bluebird’s CALD and beta-thalassemia or Novartis’ SMA gene therapy asking prices of $3.0, $2.8 and $2.0 million respectively, making it the world’s most expensive treatment. Steep asking price – yes, but not compared to the lifetime cost of medical care for these patients, which can be anywhere from two to more than 10 times the cost over the course of the patient’s lifespan.

Despite the incredible therapeutic benefit, these drugs have yet to convert into financial successes. Most are still targeting rare genetic diseases and small patient populations. In addition, the nature of single-dose curative treatments means pharmaceutical companies are likely to set incredibly high prices as their only way to recoup the development costs that go into producing these therapies. However, insurance companies have unsurprisingly balked at the huge price tags. In April 2022, the FDA approved Kite’s Yescarta for second-line treatment of large B-cell Lymphoma, marking an important milestone for the novel therapies to move up the treatment plan, potentially benefiting a broader patient population.

Novel modalities are on the rise in the pharmaceutical industry, but their financial impact on the industry has yet to provide the needed boost to put them on the map. In 2021, Comirnaty and Spikevax (the novel mRNA vaccine technologies) exploded on the scene, taking the number 1 and number 3 sales spots. But the top 100 product sales are still dominated by small molecules, monoclonal antibodies and traditional vaccines, with novel therapies barely breaking the top 200.

The potential to transform patients’ lives is turning the corner. Science is moving faster than our healthcare system. A swift change is needed to ensure that our financial system does not become the bottleneck, impeding patients’ ability to access the best treatment available; and pharmaceutical companies’ ability to leverage financial success to fuel the development of more miracle drugs. There is no doubt this breakthrough is coming soon; there are too many more promising drugs in the pipeline, which will inevitably force the system to adapt. The future is full of promise and undoubtedly multimodal.

Noel Maestre is the vice president, life sciences at CRB. Maestre is CRB‘s biotech subject matter expert, emphasizing Advanced Therapy Medicinal Products (ATMPs). He has an extensive background in mechanical and process utilities engineering with experience in the life science industry. He specializes in the design, construction and startup of biotechnology, pharmaceutical and advanced technology facilities. Maestre is a graduate of Pennsylvania State University with a bachelor of science in mechanical engineering. 

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Filed Under: Cell & gene therapy
Tagged With: pharmaceutical industry
 

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