precision medicine

Obama’s Precision Medicine Initiative: What It Means for Orphan Drugs

In Regulation & Government by Chris Hackett

In last night’s State of the Union (SOTU) address, President Barack Obama brought to the forefront of the nation something that we in the Orphan Drug industry have known for a while: medicine is becoming increasingly precise. What he didn’t mention was that this means medicine is becoming increasingly more expensive, and people’s mindset hasn’t quite caught up with the realities.

Precision vs. Personalized

[pullquote type=”right”]”Precision” medicine was the President’s new buzzword[/pullquote] This year’s World Orphan Drug Congress USA, the largest rare disease event on the globe, heavily features the notion of “personalized medicine”. Personalized medicine is the direction many people believe the industry is heading – medications specifically tailored to the individual based on their genome. This is important in the rare disease space. Many, if not most, rare diseases are due to one or more genetic mutation(s), as opposed to an infectious agent like a virus or bacteria. To be able to alter treatment types and doses based on the specific gene makeup of the patient will reduce off-target effects and increase efficacy.

But this isn’t what Obama discussed last night. “Precision” medicine was the President’s new buzzword, but what exactly does that mean?

Personalized medicine is exactly what it sounds like – it is about the person, and tailoring specific drugs to specific people. Precision medicine, on the other hand, is less about the individual and more about pathways. If a disease is due to a mutation in a specific gene on a specific chromosome, the medicine will target that specific gene. Or at the very least, if we can understand the pathway in that gene, we can target the most important part of that pathway. It allows for that same specificity people seek in personalized medicine, but without the added constraint of requiring and individual genome to make it happen.

So, the difference is slightly more than just semantics, but not by much. They both have similar means but slightly different goals. In a personalized medicine utopia, everyone would get their genome scanned and get the perfect medicine based on your genome to treat your specific disease based on your specific genetic make-up, curing you without side effects. For the precision medicine utopia, gene scanning would be a necessity, but only to figure out your exact mutation: we know the exact gene and pathway of the disease you have, and the medicine you are being given will hit that precise target, and only that target, curing you without side effects.

Rare Genetic Disorders

At the SOTU, Michelle Obama invited as a guest the 27-year-old Cystic Fibrosis patient, a disease treated with the Orphan Drug Kalydeco produced by Vertex Pharmaceuticals. Kalydeco was recently embroiled in a lawsuit, but not for what you may think: CF patients were suing Medicaid for denying them use of the drug due to its high price tag. Not every day you have people suing to use a drug.

Kalydeco was unique in its approach when it was first released; it didn’t just attack the symptoms of CF. It targeted a specific gene mutation that affects 4-5% of CF patients. Considering an Orphan Drug has to have less that 200,000 people treated in the US, and CF patients account for only about 70,000 worldwide, that 4-5% of patients equates to only around 3,500 globally patients best case scenario. But this drug didn’t just treat CF; this drug restored function to the disabled pump that caused the disease in the first place. That isn’t just a treatment; for all intents and purposes, Kalydeco is a cure.

Kalydeco is the perfect example of a precision medication. Rather than treating the broad symptoms of the disease, this drug took into account the science behind what caused the disease to treat much further upstream in the process. It significantly reduced side effects, because the people receiving this treatment already got tested and we knew that they had the target necessary.

“Orphanization” or “Precision”?

A big debate, part of which will be taking place at this year’s WODC 2015, is regarding the “orphanization” of diseases. Increasingly, companies are focusing on rare disorders instead of blockbuster drugs, and this has led to even some “larger” diseases, such as cancer, being broken down into its component parts, each of which can then fall below the 200,000 threshold and become a rare disease. It’s no longer just “lung cancer”, it’s “large cell squamous carcinoma” with its own patient subgroup. Eventually, that can be broken down even further if necessary or required to reduce the patient population. Part of industry’s propensity is due to the Orphan Drug Act’s (ODA) regulatory incentives; part of it has to do with recent industry trends.

Orphan drugs come with hefty price tags. Kalydeco, for example, is $300,000 per patient per year because it only treats 3,500 patients. This has payers, such as Aetna, Kaiser Permanente, and Medicaid, balking at the costs. Industry, they say, is “orphanizing” diseases to claim large premiums for their drugs while older, cheaper drugs can do the same thing for less.

In this case, it may not be all about the money. These precision (or “orphanized”) treatments aren’t just more expensive, they are often better. They help restore many of these patients to a way of life they may have never had before by being exactly that: precise. This isn’t just industry making money, it is science making inroads. We are diving deeper into the causes of these diseases than ever before, and its showing in our medical innovations.

Obama’s Push

In Obama’s speech, he mentioned that he would be pushing for a new Precision Medicine Initiative. As seen, these medicines were around, and being researched, since long before Obama’s speech. In fact, they were being researched full steam ahead. So what could he mean?

What it has to mean is a sincere look into the current pricing structure. There has long been a debate as to whether or not orphan drug pricing, particularly as the numbers of orphan drugs increase, is sustainable. With payers and industry at each other’s throats, and with these drugs not going anywhere, something has to be done.

What will Obama’s initiative mean for the industry, now that the leader of our Federal Government has asserted tacitly that “precision medicines” and as such “orphanization” is the direction we are headed?

It means the the country, as a whole, has to prepare for better medicine at higher prices. The science has been ahead of the mindset for years, but if the President is serious, then the challenge that will be faced is the inherent necessity of the average person to balk at an $80,000 drug considering it is more than a good chunk of our nation’s average salary. The cost to make these drugs will be the same, but each one individually will be reaching millions less people. Pharma has to recoup there investment somewhere, and as such, as we approach the era of remarkably effective medicines, we need to get ready for remarkably high prices in the long term.

So what does it mean? It means the Precision Medicine push may finally give Orphan Drug companies what they’ve been looking for – acceptance of their high drug prices. How long it will last is an entire different discussion. After all, if every drug is an orphan drug, none of them are.

Unlike gas prices, at least these higher prices will get us farther. The problem will come if that ceases to be the case.

Chris Hackett is the Producer of the largest rare disease event globally, the World Orphan Drug Congress USA, taking place in Washington D.C. on April 23/24, 2015. For more orphan drug news and opinions, follow me on twitter @chrishackettcp and find me on Linkedin!