Friday, January 30, 2015

White House Details $215M Initiative in Precision Medicine

In the President's State of the Union Speech, January 20, 2015, one sentence referred to a precision medicine initiative:
21st century businesses will rely on American science, technology, research and development. I want the country that eliminated polio and mapped the human genome to lead a new era of medicine -- one that delivers the right treatment at the right time. In some patients with cystic fibrosis, this approach has reversed a disease once thought unstoppable. Tonight, I'm launching a new Precision Medicine Initiative to bring us closer to curing diseases like cancer and diabetes -- and to give all of us access to the personalized information we need to keep ourselves and our families healthier.
On January 30, the White House describes the initiatives tied to this proposal (here).  The President's 20 minute press conference streams on CNN (here).  Some talk highlights here.  Details after the break.

    • $130M to NIH for a "voluntary national research cohort of 1M or more volunteers" with open data sharing
    • $70M to the National Cancer Institute "to identify genomic drivers in cancer"
    • $10M for new hires at the FDA and "high quality, curated databases"
    • $5M to the Office of the National Coordinator (ONC) [for health technology] for secure interoperability standards.
New York Times summary, here.  See also trade press at Modern Healthcare, here.  At Xconomy, here.  Some commenters have noted the President's remarked that we need new FDA approaches for next generation sequencing; that gene-sequencing technology is not a pacemaker.  I've included my notes on the President's 21-minute presentation at the bottom of this article.  As the Wall Street Journal points out, Obama is describing plan to include a few lines in his next massive, annual presidential budget proposal - but actual budgets come from Congress.

The New York Times had an article on January 25 that preceded the January 30 information release (here).   A few days later, a NYT Op Ed from a Mayo physician, Dr. Joyner, raised questions whether this was, or wasn't, a realistic "moon shot" - here.  For a discussion of these pieces, see here.


The President's speech and White House press releases suggest a win for the term "precision medicine" over "personalized medicine" - see an essay on that topic, here.

For the White House Blog on precision medicine, here.   For an NIH op ed on the Precision Medicine Initiative in the New England Journal, here.  For a blog on the PMI and the Digital Health industry, here.  For Forbes' review of the President's speech, here.  The trade journal Genomeweb interviews experts for their responses to PMI, here (subscription).  For the trade journal Dark Daily, here.  For the CDC's take on it - precision medicine and "precision public health," see their March 2, 2015 blog here.

The President's January 30 Presentation

These are my detailed running notes, but are not a transcript:

The President was preceded by Ms. Ilana Simon, a 19 year old Harvard student who survived a rare liver cancer at age 12.  She met Obama at last year's White House Science Fair, and introduces him today.  
[At age 18, Ms. Simon - whose father is a cancer biologist - authored a Science paper on the causative gene for fibrolamellar liver cancer (here) and (here).]
The President joked that to say the least, he was not sequencing genes at age 19.  However, Ilana was able to explain basics of genetics with styrofoam to the President.  ("That was helpful to me.  I know what swim noodles are.")

Obama then introduced leaders in the room; Sylvia Burwell (Secretary of Health), Vivek Murthy (Surgeon General), Harold Varmus (NCI), Francis Collins (NIH), and White House Science Advisor John Holder.  Added Obama, "We have a doctor in the house."  Lamar Alexander (TN) was introduced, Senator Patty Murray couldn't come, but Congr. Diana DeGette was present.

In the SOTU, Obama emphasized, we must ensure the middle class, build a competitive economy, upgrade skills.   Today is about the competitive economy.  We have scientists, privacy advocates, patients.  We'll harness America's dreams, taking of risks, spirit of innovation.  That improves our economy and our lives.

We have the possibility of an entirely new era of medicine, with new industries, new life saving treatments, right here in the US, and we must invest, nourish, encourage this.

When American researchers developed a polio vaccine - Congress distributed it.  Federal studies and grants have supported understanding heart disease, the MRI...By definition, not all basic research pays off.  But it also tells what we don't know, and we can figure out new pathways.

When things do pay off, they create unimagined economic opportunity.  Dr. Collins led the Human Genome Product, others here were involved.  Every dollar spent there has returned $140 to the economy.  It's a huge economic stake to tap into that.  We can clap for that.

But we've seen a loved one battle with illness - everyone has.  The most important impact is not measured in dollars, but preventing hurt and heartbreak.  having longer healthier lives.  Ilana was stricken too young - but she's OK and cured, and making incredible contributions.   Let's seize this.

We're hear today because something called Precision Medicine - also Personalized Medicine - gives us one of the greatest opportunities for breakthroughs.  Doctors always knew patients were unique and tailored treatments - we have always matched transfusions.  What if figuring the right dose of medicine was as simple as taking a temperature?  That is the promise: The right treatment, the right time, every time, to the right person. For a small number of patients, that future is here.

8/10 people with a type of leukemic returned to normal with a targeted drug.  HIV targeted testing helps target new anti viral drugs.  With advances in technology, these may be just the beginning.   Because the first genome cost $100M, today $2000. Wearable electronics can record blood sugar.  EHR's let doctors collaborate across the country, and powerful computers analyze the data.  It's applying medicine more effectively, higher impact, less waste - the possibilities are boundless.  

This is the new era, precision medicine, just like we launched new genetics 25 years ago.  And there's bipartisan support !!  (APPLAUSE).  In 2005, I worked with Richard Burr on a precision medicine bill, with Bill Cassidy as well.  He's called this an incredible area of promise.  So my budget will include a new precision medicine initiative, and all of us can have access to the personalized medicine information we'll need.

First, we'll work with NCI to find genetic factors for cancer, then, develop more effective approaches.

Second, we'll work with FDA for new approaches to evaluate next generation genetic tests.  The way we approve a new sequencing technology is different than a pacemaker, and our approach will reflect that.

We'll work with NIH making a research group of "one million volunteers" - analyzing this data will teach us more about the connections than ever known.  The big data set, varied people, allows us to map out not only the genome of one, but the connections among many.

Finally, privacy will be built in from day one, and patients rights advocates will be on board from day one.

The initiative we launch today will lead to a new generation of discoveries.  Hospitals, privacy experts, nonprofits, entrepreneurs, will all be involved.

We want not just new cures, but a health care system - not a disease system.  We'll each understand ...our make better life decisions, and that's one of the most promising aspects.  Focus on prevention and keeping healthy.

Medical breakthroughs take time.  This area is no different.  But the patients here today are living proof of a new era.  We'll start today, seize the moment, no telling how many lives we can change.  Every life matters.

Bill Elder was at the SOTU with Michelle Obama.  20 years ago, Bill was diagnosed with cystic fibrosis.  He's one of 4% of patients who disease is caused by one genetic mutation, and a few years ago, FDA fast tracked a new drug for that mutation.  Bill tried it in 2012 - a few years later, he knew something was different.   He could breath out of his nose.   He is 27.  27 used to be the median age of survival.  He's in the 3rd year of medical school.  And he truly believes he may become a grandfather now.

He'll one day tell the story to his grandchildren - not only a doctor, but an inspiration to others.  

In Washington, we don't always agree, but we want a strong nation, where everyone has opportunity, where accident of birth doesn't determine fate - including being born with a particular disease or genetic makeup.   It's not your destiny, we can remake it.

It's the power of scientific discovery.  We want Bill's generation, and those after, to inherit the gift, not just of a long life, but continuously remake this world.

Thanks, god bless.  Let's get to work.