2017 has been an eventful year for the life sciences industry, with new breakthrough therapies coming to market, more M&A activity and on-going debate at the state and federal governments about drug pricing legislation. As we all gear up for the new year, I wanted to take a moment to reflect on the major trends we’ve seen come to fruition or accelerate in Massachusetts and beyond in the last 12 months – and to think about what’s coming in 2018.
Gene and cell therapies are finally a reality – increasing potential for cures.
This year, the FDA approved the first two gene therapies for use in the U.S. for two different kinds of cancer. Some are even using the word “cure” to describe these therapies, most of which are one-time treatments. Although researchers have been experimenting with gene and cell therapies for decades, 2017 is a major turning point for what is sure to be a wave of new gene and cell therapies that will hit the market in the next several years. They represent the absolute cutting edge of medical science and the practical application for them is nearly endless – from treating rare forms of cancer to reversing hereditary blindness.
More emphasis on value-based care and partnerships.
The healthcare system has been trying to emphasize value over volume for some time. More advanced treatments are becoming available to patients (think cell and gene therapies I mentioned above), but some of these come with a price tag of close to half a million dollars due to the complexity of both developing the treatments and the small patient populations. This has created a renewed sense of urgency to change the payer system so it can absorb the costs of these drugs, and many have been testing value-based partnerships to do so. These agreements reward biopharma companies based on the value the drug brings to the patient. If a patient doesn’t respond as expected, the insurer gets partially or fully refunded. More companies and insurers adopted value-based partnerships in 2017, including many industry leaders in Massachusetts, and I expect these will continue to gain momentum in 2018.
More companies going ‘virtual’ – turning to CROs/CMOs to fill research and manufacturing needs.
The move to outsource more business processes and functions is certainly not limited to the life sciences industry – companies across the board are realizing its potential to increase their competitive advantage and minimize operating costs. But within the life sciences industry, there’s the added pressure to accelerate drug development, which is why so many are turning to CROs/CMOs to handle some or all of their research and manufacturing needs. Although this trend began to take off a few years ago, it’s seen continued growth in 2017 – and we’re not likely to see it slow down anytime soon. In fact, Grand View Research just released a report that says the global preclinical CRO market is predicted to reach $6.6 billion by 2025, up from $3.25 billion in 2016.
Companies increasingly look to real-world evidence to support new drug applications.
New drugs cannot be approved by the FDA without clinical trials that measure their safety and efficacy, but real-world evidence is also becoming an important part of the drug development and approval process. For example, real-world evidence can help supplement clinical trial data in instances where trial participants may not be fully representative of the overall patient population. In the past year, more companies are also using real-world data to find new indications for an approved drug, identify new side effects and refine dosing. In August, the FDA issued guidance for medical device companies on how to evaluate real-world data, and is currently working on similar guidelines for drug development. According to a recent Tufts study, this comes at a critical time, as it found 97% of life sciences companies plan to increase their use of real-world data for more accurate decision-making.
More focus on the patient voice throughout drug discovery pipeline.
Prioritizing the patient experience during clinical trials has been industry practice for some time, but more and more life sciences companies are realizing the value of incorporating the patient voice earlier on in the drug development pipeline. As a result, the patient advocacy function at these companies is increasing in importance. Instead of being a siloed department, patient advocacy efforts are more often aligned across the entire organization, ensuring that every single step in the R&D process incorporates patient feedback in some way. Patients are the reason for what we do – and we see this trend as a very positive one that will undoubtedly continue in 2018.
Don’t miss MassBio’s Annual Meeting on March 21-22, 2018 to hear directly from industry professionals on how they’re addressing these new developments and what they’re planning for next year. We hope to see you there!
– Bob Coughlin, President & CEO of MassBio