Robert K. Coughlin
President and CEO, Massachusetts Biotechnology Council
It’s an incredible time for innovation in the biopharma space. Science that’s been tested for decades is finally a reality and is creating unbelievable opportunities to transform patient care and address unmet medical needs around the world.
New treatments
We’re now using the word “cure” to describe some of these new therapies – a word I was told offered false hope a decade ago. In fact, the FDA recently approved a one-time cure for spinal muscular atrophy (SMA), a rare and fatal genetic condition that impacts motor neuron development for infants, affecting their breathing and swallowing. Babies born with SMA almost always die before their second birthday, and those that are alive require breathing assistance and constant care. A once-fatal disease is now a treatable condition. Babies with SMA and their families now have hope that they’ll be able to live long and healthy lives.
This is the true value prescription drugs bring to patients and the health care system. These therapies improve the quality of life for patients with debilitating disease, keep them alive and healthy, and prevent frequent hospitalizations and other unforeseen costs, saving money across the health care system.
Challenges
But discovering and developing these breakthroughs is no easy feat. It can take up to $2 billion and 12 years to bring a new drug to market. There are countless failures along the way and countless mutations of a single disease to consider. Take my son, for example. He was born with a rare and fatal genetic condition called cystic fibrosis (CF). We’ve been lucky in this regard since CF is one of the rare disease success stories, but 95 percent of rare diseases do not have a single approved treatment. There are several treatments out there that now address the underlying cause of CF, not just masking its symptoms.
But my son has a complex mutation, and the current therapies do not work for him. We’ve been hopeful over the last decade that some day there will be one that works for him – and we’re getting closer to that reality every day, as these biopharma companies continue to pour the money from their successful therapies into future research and development.
More than hope
Therapies offer more than hope for patients across the world. They promise to address the most complicated and devastating medical issues, from HIV to Alzheimer’s, and are critical to our collective health and economic wellbeing. Alzheimer’s alone affects about 5.7 million Americans, with that number predicted to reach 7.1 million by 2025, according to the Alzheimer’s Association. This same group estimates that it costs $277 billion annually to care for patients with Alzheimer’s in the United States, and this could top $1.1 trillion by 2050. These are staggering numbers, but they show the dire need for us to continue to incentivize the biopharma industry to solve the toughest medical challenges on the planet.
I’m proud to be from Massachusetts, the No. 1 life sciences cluster in the world, where we have an incredible concentration of biopharma companies who are leading the country in developing first-in-class therapies for once-untreatable diseases. It’s our partnership with academia, industry, and government that has earned us a spot at the top, and it’s up to us to educate the public about the value we bring to patients and the healthcare system. As former FDA Commissioner Scott Gottlieb wrote in a recent op-ed, “A therapy that can cure disease in a single treatment isn’t a unit of drug. It’s a public health solution.” That’s the critical role the biopharma industry plays for our future, and I hope you’ll all join me in supporting its essential mission.
Robert K. Coughlin, President and CEO, Massachusetts Biotechnology Council, [email protected]