October From the Editor

Often I am dismayed to hear local news reporting of early stage clinical milestones for therapies that may still be years in development. I assume that those reporters and news editors receive pretty much the same press releases that we do here at BPI. But I worry that a more general readership hears only the hype, missing details of the process and time required for approval. More than one letter to the editor has opined that “delays” in bringing new therapies to market result more from bureaucratic top-heaviness than necessary controls placed on a drug-development program. Such delays can drive desperate people to treatments in less-regulated regions or to counterfeit therapies altogether — which have for many years included “stem-cell injections” — further complicating relationships between patients and the industry as well as attitudes toward regulation as a concept.

When our local paper (the Register-Guard) published news of a breakthrough approved therapy, I was relieved to see that its Associated Press author Laren Neergaard described how CAR-T cells work in a way that seemed clear and accessible for a general audience. The statement that “there will be no charge for patients who [don’t] show a response within a month” should emphasize to readers that this is an entirely different kind of treatment.

Even in that context, I am surprised at how few articles related to the Novartis approval cover the need for different approaches to pricing and reimbursement for advanced therapies. We see such discussions within the industry, but elsewhere I still see more complaints about a high one-time cost — $475,000 for Kymriah (tisagenlecleucel) makes the $93,000 for three administrations of Provenge (sipuleucel-T) pale by comparison — than productive, rational discussions comparing it with the equal or greater costs spread out over the lifetime of traditional long-term treatments. With more regenerative medicines approaching approval stages, this may very well be the tipping point the industry has needed to move such reimbursement discussions forward specific to an emerging category of drugs rather than exceptional one-off approvals.

At our annual BPI Conference in Boston (26–28 September), BPI publisher Brian Caine and I moderated a short session on “Leveraging Key Lessons Learned from Protein Production to Drive Cell Therapy Innovation.” Panelists looked at key lessons learned at similar points in the development of monoclonal antibodies (MAbs) as a therapeutic class. Selected case studies provided context through comparisons with immunotherapies. Assessing points of divergence among the different therapeutic classes, we identified key activities to be further assessed for commercial success of advanced therapies: defining metrics, their goals, and how they can accelerate technology; innovation for proving efficacy and reducing production costs; and how risk relates to cost and can be reduced.

In the early 1990s, we had similar discussions about the high costs of MAb therapies and the need to convince patients and physicians to try something besides pills. Although MAb recipients may not understand mechanisms of action, the way is paved by the public’s increasing familiarity with the effectiveness of such treatments. As new approvals expand the vocabulary of cell and gene therapies for a wider public, one company’s breakthrough therapy no longer needs to be viewed in isolation.