Pharmaceutical companies moved fast and furious in response to Covid-19. So, why hasn’t the same initiative and effort gone into developing transformative treatments for even deadlier diseases?
The pandemic has claimed 550,000 American lives and counting. That’s an astounding and truly tragic figure, to be sure, but it’s still lower than the annual U.S. death toll from heart disease.
The fundamental problem is that too many drug companies focus on incremental improvements to existing medicines. As we have all witnessed during the past year, a better way is possible. One that could save millions of lives. If only researchers approached the development of new drugs with the same focus, determination, urgency, and fortitude.
Sure, a conservative approach is a more prudent business strategy. Of the novel experimental drugs that enter clinical trials, fewer than 10% ultimately reach pharmacy shelves, according to a Harvard Business School review. By contrast, over 20% of experimental “me-too” drugs—based on existing medicines—secure regulatory approval.
But a more ruthless and ambitious approach to biopharmaceutical innovation could deliver greater benefits to more patients—and ultimately generate bigger profits for investors.
First, pharmaceutical companies need to concentrate more on patients’ high unmet clinical needs. Americans can live without another erectile dysfunction drug or an additional statin to lower cholesterol. Instead, we need new medicines for high-prevalence, high-mortality diseases with no good treatment alternatives, such as heart disease, chronic obstructive pulmonary disease, and Alzheimer’s.
To ensure these needs are front of mind, patients and their advocates should be involved at all stages of drug discovery and development, not just postmarketing. But there is also a role for government on both sides of the equation. As was done for Covid-19, political leaders need to issue a call to action and focus public and press attention on our major medical problems. They also need to eliminate bureaucratic burden and accelerate the process for promising new drugs.
Second, drug companies and their investors should concentrate on creating long-term, durable impact, instead of fixating on momentum and short-term, transient profits. Capital markets, influential investment funds, and equity research analysts should return to properly valuing growth and sustainable upside instead of accentuating volatility. More emphasis should again be directed to the number of lives impacted and relief of societal health and economic burden. Ultimately, that should be the barometer we all use to assess the vitality of biotech companies and our economy.
Finally, drug companies should restore discipline and adhere to their areas of expertise, instead of following the latest trends. Last spring, many biopharmaceutical companies tried to pivot to Covid-19 research. They had good intentions — but without a plausible scientific hypothesis or validated drug mechanism, many of them wasted their scientists’ time and investors’ money, not to mention fueling false expectations and undermining public trust.
They, and patients, would have been better off had they stuck to their authentic research.
I know these challenges and pressures all too well. For two decades, our company has persevered in the battle against heart failure, the leading cause of hospitalizations in patients over the age of 65 and the No. 1 killer of Americans. We knew from the outset that it would require an uncommon endurance for our industry. Developing a novel cardiovascular medicine takes time and resources. Well over $1 billion has been invested in our cardiac muscle activator program, which spans over 30 clinical trials over 15 years.
The main challenge that we in our industry face when we seek to pursue such lofty endeavors is what I call “financing for fortitude.” Finding long-view value investors and unconventional sources of capital to ensure our research could continue despite inevitable bumps in the road.
In our case, we were fortunately able to build a longer runway. It required creative deal-making and diversifying sources of capital investment. We were able to take the time required to do research right. Most of all, we could set bolder goals, knowing we would have the time and other resources to deliver on the promise of our ingenuity in biology and pharmacology.
This more ambitious model should be more the norm for medical research. It requires drug companies to aim higher and reach farther, both in objectives and funding. Those of us on the frontiers and frontlines of this work have to seize this moment and not settle for modest gains and small steps forward. Together, we can set new standards and compel investors and other stakeholders to look beyond the benefits that can accrue over the next quarter to instead pursue the potential of that which can redefine the next quarter century.
A new age of medical innovation is possible after Covid-19. But it will require longer range optics, patience, and endurance. If we can sustain science on the frontiers of discovery, I am certain that we can develop new drugs for some of our most elusive and deadliest diseases.
Robert Blum is the president and CEO of Cytokinetics and an Aspen Institute Henry Crown fellow.