We just heard our chairman Tom Watkins say "We're doing big things."
Sounds good, but is that just hype, or is it for real?
Together we do really big things.
Today, I want to brag about some of the really big things, that our Biotech companies have accomplished over the past year.
But first, I want to brag about some of the big things that BIO has done on the advocacy front since we were last together.
Last year, we launched our new five-year plan to reshape the policy environment in a big way.
We took a couple of seconds to congratulate ourselves for writing this big plan - and then, we got to work putting it into action.
We got Congress to include three of our proposals in the Prescription Drug User Fee Act.
First, expand and modernize the accelerated approval pathway for serious and life-threatening conditions...We did that!
Second, improve the FDA's access to outside experts...We did that!
And, third, restore informal communications between the FDA and biotech companies...We did that too!
We got other big things done for our companies.
Make sure small venture-backed companies can once again get grants through the SBIR program…We did that!
Get a good patent reform bill passed…We did that!
And pass the Jobs Act, which makes it easier for our smaller companies to go public...We did that too!
Clearly, we're getting results for our companies.
There's a big milestone coming this summer—the world's first commercial-scale cellulosic ethanol plant is just about to start production.
And we improved the review process for biotech crops. This actually helped triple the number of new plant biotech products approved last year.
So, is this for real?
This year, I want to do something a little different with my talk. I want to celebrate the researchers who worked on some of the newly approved breakthroughs, and the patients whose lives they've changed.
Basal cell carcinoma.
It's the most common form of cancer.
When treated early, it's usually curable. But some patients have multiple recurrences, or the disease becomes locally severe. These devastating cases can result in extreme deformity, loss of hearing or vision, and even death.
In its advanced stages it often can no longer be treated with surgery or radiation.
But now the FDA has approved Genentech’s new drug, Erivedge, to treat advanced stages of this disease when radiation and surgery have failed to control it. It can shrink these disfiguring and potentially deadly lesions.
I’d like you to meet someone. His name is Don Lomas.He's 71.
This guy's my hero.Over the past 26 years,he has undergone hundreds of surgeries to remove these carcinomas.
But here's what's really incredible— it looks like these painful operations may be a thing of the past for Don.
Last week I was on the phone with Don, he told me that in January, just a few days before he was going to have an operation that would have removed a portion of his jaw, and would have kept him in the hospital for months, he began taking Erivedge, and the surgery was cancelled.
Hodgkins lymphoma, another cancer.
It strikes more than 9,000 Americans every year and kills more than 1,000.
It's a cancer of the lymph nodes. The tumors can grow very aggressively.
This type of cancer can be treated by drugs, radiation, or surgery. But these don't always work, and there are serious side effects even when they do.
Last August, the FDA approved Adcetris, developed by Seattle Genetics.It's the first new treatment for Hodgkins lymphoma since 1977.
I recently talked to one of these patients, Aimee Blaine.
She was a new mother and was working as an engineer. Then, doctors found a tumor the size of a fist in her chest cavity.
During the next seven years of treatment she couldn't work, and she told me she could never even plan a vacation with her family, because she couldn't predict if she'd be well enough. Aimee was forced to give up some of the activities she loved the most.
When I spoke with her last week, she was so excited because Adcetris has helped her avoid the side effects of traditional treatments.
Great story. This targeted drug knocks out cancer cells while limiting damage to healthy cells.
This is really a big thing.
It's a serious, potentially fatal autoimmune disease, in which the body attacks its own healthy tissues. Lupus disproportionately affects women, and usually strikes them during their child-bearing years, but it can debilitate its victims for the rest of their lives.
There is no cure yet.
One lupus patientis Rena Baldwin. Rena is a single mom who lives on Chicago's southside, with her daughter, Suzie. She was first diagnosed with lupus at eighteen, and for nearly twenty years she's been coping with this awful disease.
Human Genome Science's newly approved Benlysta is the first new treatment for lupus in over fifty years, and is the first drug specifically designed to target the problem protein.
So when Tom Watkins says we do big things, he knows what he's talking about.
This is a big thing. And it's definitely something to celebrate.
Hepatitis C now kills more Americans than AIDS—15,000 people every year. The CDC recently urged every baby boomer to be testedfor the disease. More than three million Americans are chronically infected.
Hepatitis C can cause cirrhosis of the liver and liver cancer. It's the number one reason for liver transplants, and it's why the number of these expensive surgeries is soaring. The annual cost, just to Medicare, is projected to be 85 billion dollars.
Fortunately, the FDA has now approved two new protease inhibitors to treat chronic hepatitis C infection.
Incivek, developed by Vertex, and Victrelis, from Merck. Each works in combination with standard therapy.
Building on more than twenty years of work, researchers figured out the biology of the virus, and developed drugs designed to inhibit a key protein, and prevent the virus from replicating.
Now, let's meet Judith Bartels. She's a nurse.
She was diagnosed with hepatitis C three years ago. Now, thanks to these new breakthrough drugs, she has hope.
Did you hear her say “Where there was no hope, now there is hope.”? That is what we are all about.
Atrial fibrillation, characterized by abnormal heart rhythm.
It produces blood clots and increases the risk of stroke fivefold. Many people with this condition need blood thinners, and require frequent tests to get the dosage right.
Rusty Asdourian and his doctors wrestled with this problem for decades. All that changed this year, when Janssen developed Xarelto, a once-a-day pill.
This guy's been through the mill. But now, he and his family have something pretty nice to celebrate.
These days, instead of making nearly daily visits to his doctors, Rusty enjoys his spare time. He told me he loves bird watching, spending time with his family, and woodcarving.
I was on the phone with Rusty recently when he told me he was carving a rose for his granddaughter. I asked him how he was doing. He put it very simply. He said, “Everything is fine. I feel very normal now.”
It's a deadly, inherited disease that affects about thirty thousand young people in the United States alone.
In kids with CF, a defective gene causes the body to produce unusually thick, sticky mucus that clogs the lungs and obstructs the pancreas. This leads to life-threatening lung infections, and blocks natural enzymes from helping the body break down and absorb food.
Joe and Kathy O’Donnell know all about this, in 1986 they lost their son Joey to Cystic Fibrosis.
They were devastated, but they fought back from their grief, determined to someday end this disease. The O’Donnells joined forces with the Cystic Fibrosis Foundation and with Vertex Pharmaceuticals.
This January, their first breakthrough finally came—the FDA approved Kalydeco.
It's the first drug that targets the underlying cause of CF, a specific genetic mutation that, in some patients, triggers the disease.
So now let's meet Joe O'Donnell and a couple of really great girls, Laura and Kate Cheevers.
I'd like you to welcome Joe O’Donnell and Dr. Eric Olson from the Vertex Pharmaceuticals research team.
Let's give them a big hand.
Thank you for sharing your story with us.
You know, it feels good to celebrate our successes.
After all, they don't come easily.
But we are here to do big things.
That's why we don't give up—We can't.
Because we know how many more patients and their families are counting on us.
That's why BIO will keep knocking down policy barriers. And that's why you keep knocking down the scientific barriers.
So next year, when we meet again in Chicago, we'll have even more big things to celebrate.
More cures. More treatments.
And we will celebrate them one breakthrough and one patient at a time.
The Dons, Rons, Aimees, Renas. The Judiths, Rustys, and the Lauras and Kates.
So, on behalf of the many patients and their families who are still counting on us to do big things—Thank you.
Thank you for doing big things.