Thursday, April 18, 2024

Re-Thinking Drug Development Roadmap In United States

March 21, 2008 by  
Filed under Health Care Policy, Virtual BioTechs


No Insurance. No Money. No Drugs. No Hope.

We Need A New RoadMap For The Future

Of 7,000+ diseases that affect humankind, treatments for only 200 of these diseases are being actively pursued by pharmaceutical and biotechnology companies. With the cost of bringing a new drug to market now exceeding $1 billion dollars and taking up to 10+ years to test and develop, global pharmaceutical companies are focused on return on investment.

Meanwhile, millions of people suffering from all types of diseases will never see new drugs reach them or their loved ones in their lifetime. As a nation, we must unite to think about the drug development process in a different way and push for change.

Today, one family is faced with paying $180,000 a year for a highly experimental drug treatment for their two children afflicted with Niemann Pick Type C. Who can afford a single experimental drug at this price?  Like millions of others, many Niemann Pick Type C children have no health insurance.  No insurance. No money.  No drugs.  No hope.

The dire situation we are faced with our drug development process is happening across disease states — this is not a unique problem to Niemann Pick Type C. The FDA is in a major crisis and we can no longer continue on this path when millions of lives are at stake. The Addi and Cassi Fund, named in honor of our five year old identical twin girls, plans to bring attention to the drug development crisis and to push for change.

We are building one of the first “Virtual Pharmaceutical” models in the United States with the help of Silicon Valley heathcare company CollabRx. Our Virtual Pharmaceutical is named "SOAR-NPC" (Support Of Accelerated Reserach for Niemann Pick Type C) and we are focused on creating a drug therapy pipeline for NPC afflicted children around the world. If global pharmaceutical companies and the FDA won’t help us save our children by rapidly bringing new drugs to market, we must find solutions ourselves in order to attack and treat a rare and fatal disease like NPC.

One of the greatest resources we have as a nation is our National Chemical Genomics Center. The robotic technology at the NCGC which is pictured above should continue to be invested in by our government — NCGCs technology can find existing drugs sitting on shelves that can be repurposed and used to treat diseases they were not originally intended for. Currently, we are funding the NGCG to test thousands of approved drugs as well as natural supplements against multiple Niemann Pick Type C cell lines in the hopes of finding immediate targets to treat afflicted children.

Every dollar donated to The Addi and Cassi Fund goes directly to cutting edge research projects to rapidly accelerating finding immediate treatments for brain diseases. Translational research – studies that bridge the gap between the discoveries in the laboratory with the need to find ways to intervene in the disease process and treat patients who are living with illnesses – is the roadmap for the future. With your help, we can build this roadmap and prove there are alternative approaches to the current drug development process.

We must seek treatments today and cures tomorrow!

The “Lucky 200” Health Care and Drug Club

January 27, 2008 by  
Filed under Health Care Policy


Are you lucky? Did you know that of the 7,000+ diseases that affect humankind, treatments for only about 200 are being actively sought by pharmaceutical and biotechnology companies?  Crazy, isn’t it?  As it turns out, it costs about $1 billion for a pharmaceutical company to bring a new drug to market so they are entirely focused on return on investment. It also can take from 10+ years to get them to market.

This is why we see drugs like Viagra coming on the market and not medicine that can actually cure people who are sick. Which bucket do you think you might end up in someday?  The 200 Club or 7,000 Club?   Why are the political candidates not talking about this topic? You can have all the healthcare insurance you want, but if there is not a drug for you when you become sick, what will you do?

Nearly 47 million Americans, or 16 percent of the population, are currently without health insurance according to the National Coalition on Health Care.   Even if we were able to provide all these uninsured people with health care, the concept of universal health care will never work in a broken system that is full of insane regulations and bloat.

If politicians like Barack Obama or Hillary Clinton want to know what is wrong in the medical system, they should ask people like us who are in the midst of a medical battlefield and trying to win a war.  You can’t understand what is going on until you are fully immersed in this nightmare like we are.  The first thing I would tell them is that $160,000 dollars per year for an experimental drug for two children is a little excessive, don’t you think?

We have only been navigating the medical system for three months — here are some other major problems.

  • Lawyers: everyone in the medical system is afraid of lawyers and paralyzed as a result. The system can’t move and doctors won’t take risks because lawsuits is all they can see.  They are afraid to dispense an aspirin to kids who are dying.  Seriously.
  • Insane Federal Regulations put the fear into Institutional Review Boards (IRBs): we need tort reform on a national level. I had someone smart tell me that what is happening in our system is “criminal.” Doctors and scientists are sick of IRBs and goverment regulations because they can’t get anything done
  • The IRBs Themselves – They are insane: The red tape and paperwork are standing in the way of getting to cures and treatments.  Everyone in the system is complaining about the IRBs – they were designed to help people and protect people and now they are killing people. By the time researchers get IRB approval, their grants have run out. I have been in an IRB nightmare for 3 months with the NIH and our local hospital to get simple samples of urine and blood from healthy kids to compare to Addi and Cassi.  There are plenty of other insane examples that I will post in future blogs.
  • Wrong Incentives – Publish or Perish: Research labs are competitive — all they can think about is their next grant and putting food on the table. Many are running the same experiments and do not talk or collaborate well. There is tons of waste in the system because of lack of data sharing.  Scientists are afraid to put ideas out for fear of ridicule by others scientists and before numerous tests are done 50 times to confirm they are not crazy. Getting information out to the medical community and public is a major bottleneck – the incentives for science needs to change! Future Nobel Prizes should not be given out to scientists or researchers who are not collaborators
  • Technology – many of the people in medicine are still folks who “dictate” and don’t know what a Blog is. Old timers are resistant to change but have the most experience, new scientists are technology savvy but need exceptional tools to make their lives easier. The right kinds of technology need to exist to make it easy for the medical industry to do their jobs
  • Collaboration:  it’s practically nonexistent across the entire medical field
  • Cross Sharing Among Disease States: It’s not simply collaboration between scientists in a particular disease state, it’s creating collaboration between scientists working in different disease states that have connection points
  • Therapies Now: how do you translate what is in petri dishes and mice into potential therapies for people with diseases?  How do you take existing drugs and test them against disease to see if they work or could be used “off label.” Studies in labs with mice do not translate well for people who are dying
  • Case Manager: I am working full time to move the needle for Addi and Cassi. What happens if you have cancer and don’t have an advocate to help organize all of this or can’t get out of bed? There could be a whole business spun out of this idea and people would pay for Case Managers
  • Money: If you want to live, start saving now. You better be prepared to shell out as much money as you can in a broken system.  Not only do you need insurance, you need your own money ….. and lots of luck!

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