Team:Evry/Practices/State

   

Why develop medicine is expensive and classical funding way?




First of all, a historical point of view is necessary to appreciate the current context. Throughout history, some medicinal molecule has been abandoned due to a lack of money to start the clinical trials, unpatented possibility or not enough profitability for the company. To cite an example the dichloroacetic acid is a cheap molecule derived of acetic acid unpatented, which could be used in the cancer therapies E.D. Michelakis et al. (Publication available at http://stm.sciencemag.org/content/2/31/31ra34).

To go further a focus on the company behavior highlighted different origins and regulatory constraints of it. The first reason is pratical. When the company found new interesting molecule, it has to file a patent to protect their discovery. The maximum duration of a patent is twenty years (INPI institution: http://www.inpi.fr/fr/brevets.html), and twelve years are necessary to realize the diverse tests and clinical trials, consequently 8 years stay available to exploit the molecule. In the case of orphan or rare diseases not enough people are concerned to be economically profitable for companies. In continuity, the development of certain medicine is just too expensive to justify the risk takes by the companies, or the production cost is too high. Finally, the pediatric clinical trials are complicated with a special law for children. In overview, companies still companies, and have to keep a financial health, in regard to their investor’s responsibilities. Actually, a public-private European Innovative Medicines Initiative (IMI) aiming to speed up the development of better and safer medicines for patients. The collaboration is facilitated. The budget is €3.276 billions. The other funding is the investment by shares, and tender. An interview of J.J Garraud Co-founder and CEO of Inotrem illustrates the collaboration between pharmaceutical and medicinal research.

In the case of cancer the overall cost in 2010 amount to $263.8 billion: $102.8 billion for direct medical costs whose $20.9 billion for indirect morbidity costs (e.g., cost of lost productivity due to illness); and $140.1 billion for indirect mortality costs (cost of lost productivity due to premature death).  The immunotherapies still also expensive, an illustration of the cost following. Nowadays 17 targeted therapies can be offered.

The clinical trial is a long and expensive phase separate in three. The first include between 20 and 80 healthy adult volunteers, the aim is to evaluate initial safety and tolerability profile, and determine a safe dosage range.

In phase II, in the volunteer patients usually between 100 and 300 in this case the idea is to determine an optimal dose. Finally, in phase III is a large patient population: 1,000 and 3,000 patients to generate statistically significant evidence to confirm the results obtained.




Collaborations between pharmaceutical and medicinal research

Our team interviewed Jean-Jacques Garaud, non-executive Director of the Board of Directors at MedDay, co-Founder and CEO at Inotrem to learn more about the interection between these two entities.

Dr. Jean-Jacques Garaud is a senior executive with a broad international experience in Research and Development in the pharmaceutical industry. Dr. Garaud was most recently Head of Pharma Research and Early Development at Roche (2010 to 2012) and prior to that Head of Development and Chief Medical Officer at Roche (2007 to 2010). He previously held other senior positions with Schering-Plough in the USA (1991 to 2001) and Novartis in the USA and Switzerland (2001 to 2007). He is known for his leading role in the development of Interferon alpha, ribavirin and PEG-interferon in hepatitis C in the 90s and his contributions in building and implementing Personalized Health Care at Roche.He is also a board member of Circassia, Polyphor, MedDay and Inatherys.

Interview

- Could you present yourself and describe your professional career?

I am an MD trained in Paris in the field of critical care medicine and infectious diseases. After a few years of medical practice I moved into drug development to have a stronger imp[act on public health. Over the last 30 years I have had positions of growing R&D responsibilities with “big pharma”. My last position form 2007 were with Roche in Basel where I was responsible for global drug development and Chief Medical officer, and then head of research and early development. I left Roche 3 years ago to move in smaller and more focused areas to help early academic projects to mature and to get funded via interaction with technology transfert offices and seeding funds and VCs. I am non-executive director on four biotech in France, UK and Switzerland and the CEO of a startup called Inotrem which develops a new entity in septic shock.

- In your opinion, what are the loopholes in the actual medical system, with regard to the new therapies and medicine developed in medical and biology research?

The main deficiency of the system to bring new promising molecules to the system is the lack of resources upstream  at the very early stage of research and development when an academic team of a startup has a biological proof of concept in an animal model with a lead compound but lacks resources to progress the program in an effective way. Here lack of resources means both financial resources but also and even more importantly human resources because one needs professionals who are experienced with the transition to early academic status to the pre-industrial status in R&D.

- What do you think about the idea that the “big pharma” stop deliberately the development of new molecule or buy patent, and this for economic reasons?

This is a bit ridiculous for two reasons, big pharma is busy enough protecting their own intellectual property  to spend time doing something totally illegal and anti-competitive. To a large extend it is just an illegal practice. In 30 years I have not seen such a behaviour. The truth is that the portfolio of large pharmaceutical companies in made for more than half by products coming from biotech’s and startups, and not from their own R&D research therefore is a large company buys a licence for a product or a product this is because they see potential in this drug and they do everything they can to develop it and bring it to the market. You have to realise that the attrition rate is very high in drug development. Only about 5% of the products that are in phase 1 will reach the market successfully. Some people who are on the paranoid side of the world may see this high attrition rate evidence supporting the statement you are quoting. But again this quite ridiculous, the process of drug development is pretty complex and leads to a lot of failures, unfortunately.

- How the associations can develop the abandoned therapies by “big pharma” for economic reasons?

A lot of progress has been made over the last two decades regarding this matter. Today this happens via large charity organisation such as the Gates foundation or the alliance to fight neglected diseases in the third world. Also Big pharma companies have been opening their huge library of chemical molecules for free to those organisations so they can test the molecules in diseases such a malaria or tuberculosis for instance. These can lead to the discovery of new drugs for those diseases. Another example is Novartis which created in the 90’s a research center in Singapore exclusively devoted to Tropical infectious diseases.

- What is your vision about the “My Pharma Company” iniative or initiative similars, which consist to apply the web system for the medicinal research domains? On an ethical point of view, do you think this new funding include risks (financial explotation of patient distress, scam ecc…)?

Any new way of funding the early program so that they can mature if good to take and although it is still experimental, I am convinced that it will be a way to support projects at their very early stage. I am not sure this will be possible to take projects to clinical development where the costs are very high counting in millions or tens of millions euros.  But I am convinced this may be quite helpful in the maturation of new fascinating science that would not have been able to progress and mature otherwise. I do not have much more to say about the risks associated to this new way of funding, any practice that involves money and human nature do expose to some risks such as fraud and embezzlement, but this should not distract any one from the main purpose of this type of funding that is supporting young scientists to mature their programs and ideas.

This interview was conducted by Lucie Raymond, on September the 10th, 2015.

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