Tag: Pharmaceutical industry

  • Entrepreneurial scientist…

    Entrepreneurial scientist…

    HiResI regularly hear leading scientists say that patients can make a difference by being closely involved in projects that could ultimately lead to breakthroughs for their diseases. On the other hand, I also hear researchers say “leave the science to the scientists” and that we shouldn’t get involved because of our biased opinions. Contradictio in terminis or a rusty scientific paradigm?

    You can’t deny that a certain subjectivity exists when it comes to patients being involved in research projects related to their own diseases. With the amount of knowledge that’s now only a quick Google search away, I don’t blame researchers for their hands-off opinions when it comes to dealing with patients. However, this type of knowledge has become such an abundant, primary source of hope and inspiration for patients with rare diseases, that even when restricted by paywalls, these patients are often more up to date with the latest breakthroughs and information than their doctors.

    Certainly, entrepreneurial patients like myself have strong opinions. We don’t take no for an answer. We can be instrumental in the process to develop new drugs for our diseases. We can accelerate development times because of our devoted focus. We are the ones who can help translate basic research into real products. Why? Because we have a vested interest in how this all turns out. Our intimate involvement with our diseases gives us the passion and the power to combine our business skills with the scientists’ practical knowledge in a robust collaboration. Biased or not, it doesn’t really matter, the ultimate goal to find a cure is what binds us to our cause.

    In the biotech and pharma industry, translational research is more common in certain parts of the world, like the USA, where companies collaborate at an early stage with academic institutions. With rare diseases like ALS, it is essential to do so. However, there is still much to learn. Academics should share their discoveries with companies and patient groups at a much earlier stage, instead of caring about publication dates. Some breakthroughs take almost a year before becoming public knowledge. That’s another year lost translating them into real products. When the papers are finally published, accompanied by a positive press release, the scientists continue researching in another direction, leaving their discovery for the business world to pursue.

    Innovation, and ultimately curing patients, is halted by this scientific paradigm. This really should change. Scientists should actively engage with fledgling biotech companies and offer them their “off paper” insights in return for a small licensing fee when the efforts prove successful. At the end of the day, it’s all about shared value, shared success and curing patients. At least it should be… Researchers should transform themselves into entrepreneurial scientists, while the business men and women should become more like scientific entrepreneurs.

  • So far… but so good!

    So far… but so good!

    One of the conclusions that came out of a report on the financial crisis was the complexity and lack of transparency that led to the ultimate meltdown of a system that was put in place over 40 years ago. Forced by public opinion, the excessive bonus culture of the financial industry came under intense scrutiny and the banking world was forced to reinvent itself, ultimately returning to a more sensible definition of home base.

    Many high-profile economists agree that our medical and health care system is about to collapse, hence prompting the Harvard Business Review to open @Hbrhealth and inspiring countless blogs with just as many opinions. Can we really fix the health care system from a business angle, like a failing bank, or might there be a different approach?

    Healthcare systems differ per country, but basically the concept is the same. Workers pay taxes and/or insurance premiums, while insurance companies and national health institutions provide the insured with care, medication, and more. From an economic point of view, the two sides of this equation should balance out. So far, so good…

    Let’s move on to pharmaceutical companies, many of whom made billions with blockbuster drugs which they invented in the sixties and seventies when development costs were mere peanuts compared to now. Some scandals at that time forced them to cooperate with government agencies to ensure that safety comes first in clinical trials. This has evolved into one of the most complex governance models there is on the globe. These companies now spend a lot of their earnings in research and development on trying to develop drugs for lifetime use. So far, so good…

    So where’s the catch? Well, as we say in the business world, “cash is king” and this is what is completely different from the financial crisis. Big pharmaceutical companies are sitting on huge piles of cash, scanning the market for small, innovative biotech companies to buy instead of using their capital to develop drugs themselves. The small biotech companies are doing their utmost to garner attention from the major players in the pharmaceutical industry with smartly designed trials which show efficacy to their bigger brothers.

    Is the system in an equilibrium? Hell no! Health care costs are skyrocketing and have doubled in most developed countries in the last 10 years. The pharmaceutical industry is struggling with longer development times and huge governance costs, not to mention the pressure to find cures…

    I sincerely think we face a chronic autoimmune disease in the health care system, where the system is attacked by its own antibodies, placed in the system many years ago, and is now suffering from a chronic illness. Eerily similar to what it was meant to prevent, isn’t it?

    But what if we apply some of the lessons learned from the global economic crisis in order to cure the current illness plaguing the health care system?

    Remember what it’s all about…

    The people at the top of the health care food chain are cognitively disassociated from the people it was designed to serve…the patients! It’s just like how the executives in the financial sector forgot about their customers.

    My idea would be for pharmaceutical/biotech companies to team up with insurance companies, health care institutions, and patients to create dedicated task forces per disease with a shared cost/revenue model. Pharmaceutical companies with great views and expertise will be in more task forces and thus make more money. Innovative biotech pioneers can look for neglected task forces for rare diseases. This is what I call a shared, innovative health care system, where everyone benefits and gets what they want. Drug companies make money for their shareholders, governments reduce health care spending, and last but not least, patients get their cures.

    Simplicity rather than complexity…

    Bigger doesn’t always means better. With the global population doubling in just over half a century, in parallel we created the most complex system on earth to provide for our health care. An average blockbuster drug costs over 1 billion dollars and between 12 to 15 years to make it available to the public. A clinical trial culture aimed to design and meet pre-defined outcomes, with many patients failing to meet the inclusion criteria, is thus left to its own devices. In a recent blog, Dan Ollendorf, Chief Review Officer at the Institute for Clinical & Economic Review, pleads to implement the so called “adaptive licensing” model, whereby a drug is admitted earlier to the market, with open access to all patients. This should be paired with a monitoring system that prevents us from creating a “learning” system, but rather a “curing” system. If we could implement platforms that make this model more easily and readily available for patients, with less knowledge being hoarded by the system, I sincerely hope that we would be able to remove the one and only obstacle to true change… the existing mindset.

    Transparent as glass…

    The data that comes out of drug trials and the research undertaken by task forces should be made public at all times. In a system that basically has been paid for by the people, it should not be allowed to put information behind paywalls. Trial data should not be protected by intellectual property rights and I am not the only one with that opinion. 4 senior scientists from the European Medicines Agency write in the leading New England Journal of Medicine that putting clinical trial data in the public domain will make it more cost-effective to develop new medicines.

    The system itself has a responsibility to serve the people it is meant to protect, inform, and heal. Companies are rewarded for the results they achieve in the task forces, and publishers should develop a different business model aimed at elucidating rather than educating.

    Reinvent the governance model…

    We all have to rely on science, otherwise we would be forced to jump out of an airplane without a parachute and learn to fly (or wait for God to save us). Undoubtedly science should be at the core of all the above. Companies that claim over-the-counter-drug effects from fuzzy trial data can be part of the sketched model and scientifically prove their concept. It is, at last, a shared model, but also with shared responsibilities. No tricky business, just a clear, open, and honest approach between task force members, supervised by independent bodies with a clear mandate and the authority to protect the system from becoming ill again.

    Without ignoring the current costs of health care, I think we should start at the root of the problem. This will automatically lead to a significant reduction in the rapidly emerging problems of a global health care system on the verge of collapse.

    So far…but so good!

    One Man

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