Obama’s Precision Medicine Initiative: Paying For Precision Drugs Is The Challenge

Earlier this afternoon, I had read that in tonight’s State of the Union address, President Obama was to discuss the fruits of the Human Genome Project in the context of what’s become known as precision medicine.

Precision medicine has evolved from the concept of personalized medicine in that our focus in disease treatment and prevention is not semantically correct, i.e., focused solely on the individual. Instead, medicines are emerging that are targeted precisely to a subset of molecular problems shared by small groups within the larger population with a given disease.

Precision medicines are those directed toward a precise defect in the primary cause of a disease to either inhibit a malfunction or, even better, restore a normal function.

The goal of precision medicine is to give the treatment to the patients most likely to benefit because we now know the precise cause, at the genetic level, for this disease in a group of people.

Inherent in this definition is that the drug is not given to people with the disease who do not share the same precise defect, thereby avoiding waste of a drug on someone not only won’t benefit, but would only experience side effects.

The precision comes from advances in genetics and protein biochemistry that allow diseases to be identified and then broken down into subsets based on precise defects that cause the disease. But the other side of precision medicine is knowing how to develop drugs that work in these cases of precise defects.

Let’s take cystic fibrosis, for example. Sitting tonight with the First Lady was Stanford University graduate and Wright State University medical student, Bill Elder. The 27-year-old doctor-to-be was born with a disease we call cystic fibrosis. The disease results when a protein pump controlling chloride in organs like the lungs, pancreas, and intestines, doesn’t perform up to snuff.

In the lungs especially, the malfunctioning chloride pump causes thin secretions that protect the tissues to become thick and difficult to expel, allowing persistent bacterial infections and tissue damage where gas-exchanging lung tissue is replaced by non-functional, non-pliable fibrous tissue.

While cystic fibrosis is considered one disease, many molecular paths exist to get there. Bill Elder has one of those paths shared by 4% or 5% of the 70,000 cystic fibrosis patients worldwide. Scientists were able to identify the molecular change in chloride pump that causes it to malfunction, then start working on drugs that could restore its normal function.

Paying for the miracle of precision medicines

Vertex Pharmaceuticals developed one drug that dramatically improved the function of the chloride pump in this small percentage of patients with this group that inherited these molecular changes. The drug, Kalydeco (ivacaftor), was deservedly called by Forbes Senior Editor Matthew Herper, “The Most Important Drug of 2012.”

Herper pointed out that not only was this drug a triumph of genomic medicine, but it was targeting the gene identified by the current NIH director, Francis Collins, 25 years ago.

While the challenges of developing precision medicines are monumental on their own, the real-life challenges come down to economics. Kalydeco is priced at around $300,000 per patient per year. As medicine becomes more precise in classifying diseases into their discrete, genetic subtypes, the patient populations benefitting from a given drug are reduced.

The result is that we are developing more effective drugs with dramatically greater effectiveness, but for a market of a few thousand or even hundreds of patients.

And even with this cost, Herper pointed out that Kalydeco probably wouldn’t have been developed at all if not for the investment in Vertex by the Cystic Fibrosis Foundation. Developing drugs for rare diseases or for populations with small subsets of common diseases is a truly risky business. Even with the tax breaks provided by orphan drug status (drugs intended for diseases suffered by 200,000 Americans or less) and a seven-year period of marketing exclusivity, stockholders don’t give many companies the luxury of bringing such drugs to market.

As a result, patient advocacy groups have been increasingly taking some responsibility for drug development through venture philanthropy and forming substantive, financial partnerships to share the risk with researchers and companies willing to bring a precision drug to market.

On one hand, the Cystic Fibrosis Foundation has been applauded for their partnership with Vertex in getting a truly transformational drug to market. But they received some criticism and endured conflict-of-interest assertions resulting from their receipt of a $3 billion royalty deal on sales of the drug. The Foundation is obviously committed to reinvestment of those funds to expand discovery and development partnerships with other companies with precision drugs for larger groups of patients with cystic fibrosis.

Precision Medicine: An old idea for smaller patient populations

The truth is that we already have some precision medicines that were developed with tools available before the Human Genome Project. Tamoxifen, the estrogen receptor blocking drug, transformed the treatment of breast cancer when it was recognized that subsets of the disease were stimulated by the presence of the hormone and in turn, inhibited by tamoxifen.

But we now have many more tools, with greater precision, to identify druggable molecular defects at the heart of human disease. President Obama’s proposal for a federal precision medicine effort has yet to be fully described but the time is ripe to make this a national priority. My scientific career has taught me to be somewhat suspicious of grant new names placed on federal initiatives where small research teams are making good progress. But the conversion of basic and translational science into manufactured drugs is where I hope this leads.

The Human Genome Project has helped us identify the molecular causes of disease; we now need to marry these efforts with chemists, pharmacologists, biophysicists, and physicians who can use this information to develop treatments, and even cures, for patients harboring these molecular defects.


We as a society must come to terms with the fact that prices in the tens of thousands of dollars per year will become the norm, with certain drugs commanding six figures per year. These prices, while seemingly out of step with the $4 generic drugs you can get at a big box pharmacy, are in many cases quite reasonable when one considers the value that they provide to patients and the health care system. The debate over the prices of $84,000 drugs for hepatitis C often overlooks the value provided in simply abrogating the need for a liver transplant that runs upwards of $500,000 (not to mention the suffering that’s alleviated).

If precision medicine is now becoming an initiative supported by the leader of the federal government, the costs of converting the intellectual capital to therapeutic modalities must also be taken into account and built into the health care system.

For precision medicine to have its greatest impact, federal and private health insurance companies have no option but to become comfortable with value-based drug pricing.

27,281 thoughts on “Obama’s Precision Medicine Initiative: Paying For Precision Drugs Is The Challenge

  1. My spouse and I stumbled over here different page and thought I
    should check things out. I like what I see so
    now i’m following you. Look forward to exploring your web page repeatedly.

  2. Hi there, You have done a fantastic job. I’ll certainly
    digg it and personally suggest to my friends. I am confident they will be benefited from this website (Sal).

  3. The Pitcairn Islands ( /ptkrn/; Pitkern: Pitkern Ailen), officially named the Pitcairn, Henderson, Ducie and Oeno Islands, form a group of four volcanic islands in the southern Pacific Ocean. The islands are a British Overseas Territory. The four islandsnamed Pitcairn, Henderson, Ducie, and Oenoare spread over hundreds of miles of ocean and have a total land area of about 18 square miles (47 km2). Only Pitcairn, the second biggest and measuring about 2 miles (3.2 km) across, is inhabited
    http://rarebook.ncity.net/xe/?document_srl=546890 http://rarebook.ncity.net/xe/?document_srl=546890

  4. The Home Secretary said an atmosphere should be created where women and weaker sections of the society get the capability to register a complaint without facing difficulty. He said in cases like molestation, the very thought of going through the ordeal to brief the policemen concerning the incident dissuade many to go to a police station and that has to be changed.
    http://mansplay.net/?document_srl=350774 http://mansplay.net/?document_srl=350774

  5. FOR Dwell HQ Insurance AND SUPERCOACH SCORES CLICK ON THE Credit score CENTRE ABOVE OR [url=http://www.airmaxnike.us.com/]cheap nike air max[/url]
    He said Twenty three pilgrims were killed due to the taking pictures and the attackers also blew up the buses. [url=http://www.onlinecoachoutlets.us.com/]Coach Outlet Store Online[/url]
    Possessing previously performed in Encore ohydrates production of Phantom in the Opera within Launceston, Garwood said he previously had struggled to show up at ease in the media. [url=http://www.oakleysunglasses.nom.co/]http://www.oakleysunglasses.nom.co[/url]
    Housing Proper care has worked with all the at-risk the younger generation within the Crouse Street position for around four to 5 years and features about 29 clientele, claimed Hollie Ashworth, manager connected with advancement regarding Pound Care, who had been matching the particular cleansing projects in the house intended for Mitzvah Day. [url=http://www.moncleroutlet.net.co/]http://www.moncleroutlet.net.co[/url]
    In 2011, his or her group positioned No. One inch yards (Three or more,667) and touchdowns (Thirty eight). The Thirty-eight touchdowns rank second all-time in NFL history. Many five wideouts got 25 receptions for the first time inside Packers background. [url=http://www.ugg-bootscheap.us.com/]Cheap Uggs[/url]
    and i also explained so what can we all do, to check it out, what [url=http://www.coachfactoryoutlet.click/]Coach Outlet[/url]
    The particular friends were fired up and also every little thing looked like there was running smoothly. They established a vehicle to select them upward in their desired destination. [url=http://www.moncleroutlet.net.co/]moncler parka[/url]
    Cruz isn’t calm concerning the service along with opportunities he’s got by simply other people inside instruction along with the area and also stated this individual won’t throw away all of them.

  6. My hunch is the fact that Snape turned to DD because he’d heard the prophecy and knew that Voldemort would get hurt (or even killed) if he attempted to kill Harry. I don’t think he was noble, I simply think that he saw the benefits of being on DD’s team by the time LV fell. I believe his willingness to swear to that, as well as giving a nice show of remorse for what he’d done, would have led DD to give him a chance.
    http://syslab.jnu.ac.kr/?document_srl=109022 http://syslab.jnu.ac.kr/?document_srl=109022

  7. “Yes, this is the potential in the event that, aah, say in this case Labor and also the Greens, umm, it’s the power to vote down legislation in the suitable circumstances,In . he said. [url=http://www.airmax-90nike.us.com/]nike factory store[/url]
    Filipino assaulted: An unidentified Filipino man provides filed any complaint with all the police passing the buck to a compatriot associated with beating him, reports Al-Anba each day. The automobile accident happened while you’re watching Philippines Embassy. In the mean time, an mysterious Kuwaiti has been busted by law enforcement for careless driving along with hitting a pair of Egyptian people on the streets – mommy and girl, reports Al-Anba every day. The episode happened before the Al-Amayer Investment Corporation. [url=http://www.michaelkorsbags.me/]http://www.michaelkorsbags.me[/url]
    take control of: to be in control – มีอิทธิพลเหนือ ครอบคลุม ควบคุม [url=http://www.moncleroutlet.net.co/]moncler parka[/url]
    This is just what took place, and sadly with regard to Campbell, this umpires have it correct. [url=http://www.uggbootsblackfriday.us.com/]Cheap Uggs[/url]
    Iowa: Down Two,093, pointless granted [url=http://www.michaelkors-bags.us.com/]michael kors outlet online[/url]
    Struggles [url=http://www.oakleysunglasses.nom.co/]oakley sunglasses[/url]
    with his fantastic company will be merging together with the UW College of [url=http://www.moncleroutlet.nom.co/]moncler outlet online[/url]
    This author can be a student in the School connected with Sussex. She’ll be located @MiriamCsTravels about twitting. [url=http://www.oakleysunglasses.in.net/]oakley sunglasses[/url]
    POLSON – The Idaho person has been killed early Fun as soon as their ultralight-style planes crashed in an RV holiday resort near to the Polson International airport.

  8. Use EDGAR. The Electronic Data Gathering, Analysis and Retrieval System is a data processing system through the Securities and Exchange Commission. If you want to look up a to make money business, you can look it up using EDGAR. There are any of three forms that companies are required to fill out through EDGAR: 8K; 10K or the 10Q form.Major businesses and companies are often difficult entities with which to communicate. Fortunately, such search engines literally bring you right to the source. When you want to buy a product from a small business, you have the added benefit of receiving personalized attention. You may see something on the site that you like but have an idea as to how it can be customized to suit your specific needs. You need not wonder for long. The sites given to you on this search engine generally provide much ease for that customer to business communications.
    http://nhsale.co.kr/xe/?document_srl=663277 http://nhsale.co.kr/xe/?document_srl=663277

  9. How do we continue the soul of service in our time? That question was the attention of a summit at Gettysburg, California., this week, subsidized by the Franklin Venture at The Aspen Initiate. Retired normal Stan McChrystal convened the bipartisan group of frontrunners and activists around a big, much-needed concept: to provide A million civilian national service options for 18- to be able to 28-year-olds every year, comparable to the more compared to 1 million whom serve on active duty in our nation’s armed forces. [url=http://www.glassesoakley.us.com/]oakley sunglasses[/url]
    Lucy Hale [url=http://www.coachoutletstores.net.co/]Coach Outlet[/url]
    The investigation had been released within an on the net complement on the record Snooze. [url=http://www.rosherunshoes.com.co/]Nike Roshe Run Shoes[/url]
    Brodie Grundy pulled Sydney ohydrates protection pertaining to height using about three scars within Fifty inside the minute time period nevertheless after the first two collection images drifted vast and a break sought out on the whole this individual performed on from your third draw somewhat reluctant to consider one more shot. [url=http://www.ArizonaCardinalsJersey.us.com/]Cheap NCAA Jerseys[/url]
    As you move the moms of Designer Fantastic and also Kelli Bullard say not only a time passes many people do not think about their own little girls.

  10. I paid for this particular looking for a great gifts of my personal mama. Their headphone came within a perfect purple tied container. This was ideal looking gift less than on christmas tree!! The actual headphone furthermore seemed stunning, conversely our mother is actually huge boned as well as the headphone is actually somewhat tight, although it really is outstanding gifts! I appreciate things.

Leave a Reply

Your email address will not be published.