Pleading for therapeutic research

What is a fair price for the sofosbuvir?

In April of this year, the New England Journal of medicine  announced a major, long-awaited therapeutic breakthrough.  In association with another antiviral - but without interferon, a new molecule, Sofosbuvir, allowed cure rates of hepatitis C from 93 to 99% after 24 weeks of treatment in patients infected with HCV genotype 1 in failure of protease inhibitor in the fibrotic stages. The good news quickly became subject of controversy; developed by Gilead, the treatment should be proposed to 60.000 euros for twelve weeks,  600 eur par  tablet. In France, the number of patients at all stages is of about 250.000. The cost would be truly unbearable if we treated all patients – though, however, they probably could  all benefit from it.

This kind of problem can be addressed by negotiations and usually price/volume agreements- more sales, lower price. But it is worrying that for most of  public opinion and health officials, Sofosbuvir appears as an economic problem rather than a major therapeutic breakthrough- whereas, leaving apart the patient life and life quality (!!!), it is also a major economic progress if you compare to rather inefficient  interferon (70.000 euros), not to speak of  liver transplant

A more pertinent question perhaps is this: Why do France and Europe invent us less and less drug in France and Europe, why do not we have success stories like Gilead? Gilead, founded in 1987 by Michael Riordan, a 29-year-old doctor infected by the virus of dengue on a humanitarian mission and wanting to devote himself to antiviral research, has developed and put on the market within a few years  many truly innovative drugs, among other  (anti-parasitic) Ambisome, (antiviral) Tenofovir, Cayston (antibiotic), the Emptricitabine (antiviral), the Flolan (Antithrombin), the Volibris (endothelin antagonist, first treatment for pulmonary hypertension), Macugen (synthetic protein derived from VEGF, against macular degeneration due to age), the famous Tamiflu, anti-viral flu A and B and first class of inhibitors neuramidinase.

Long live  the pharmaceutical industry !

Perhaps this is because we have become accustomed to consider drug as a cost, everywhere easy victim of ministers looking for savings, and not as a progress, pharmaceutical companies as profiteers, and not as entrepreneurs who take risks (cf. the mortality rate of the biotechs!) to invent remedies ; because drugs are seen as social expenses and not as patient benefit. I have always had difficulty to understand why when the French buy fewer cars, this is a national tragedy,  to be  remedied with subsidies, whereas if they buy drugs for their health, it is anti-civic behavior to punish! (And, by the way, yes, it may be justified to prescribe an antibiotic for a viral infection - almost all of the 50 to 100 million victims of the Spanish flu died of secondary bacterial infections, not the virus!)

So, remember. In 1967 appeared beta blockers. It was the first effective treatment against this 'silent killer' –hypertension- and dozens of years of life gained for many. They were followed in 1981 by inhibitors of the enzyme conversion, by antagonists of angiotensin (1995), becoming increasingly specific and safe. By the way, this success is the illustration of the need for several drugs targeting the same pathologies and also the same mechanisms to cover the variety of individual situations. I 1977, there were the first antiulcer medicines, antihistaminics; peptic ulcer and its unbearable pain, and its dangers that have long poisoned the life of our parent or grandparents has  gradually ceased to be a surgical indication and is well and safely treated by these antihistamines, and antibiotics, and Proton pump inhibitors (1989). In 1980, the therapeutic entered the era of genetic engineering with the production of the first recombinant interferon that allowed to treat previously untreatable viral diseases, with also insulin recombinant production (1980), and then growth hormone (1985) and many others, that will treat all patients without restriction, more easily and avoiding horrendous problems with viral contamination of natural hormones. In 1983, it was the discovery of the first anti-rejection drug, cyclosporine; without this new therapeutic class, the extraordinary adventure of transplants of all kinds would have been simply impossible, despite the ingenuity and technical prowess of surgeons. In 1985 was released the first antiviral drug active against HIV/AIDS, first of a long series; never any threatening and deadly epidemic for humanity was fought as quickly. In 1988, a new class of drugs, Statins could effectively treat this second silent killer that is the 'bad cholesterol '. From 1994, triptans could relieve migraine sufferers-only migrainous can understand what it means. In 1998, a antibody obtained by genetic engineering to effectively address the pain and disability caused by rheumatoid arthritis. In 2001 appeared Gleevec, first anticancer targeted drug, which acts directly on the proteins altered in some cancers (and not killing the cells in a quasi-indifferenciate manner); This was the first of the kinase inhibitors, a class in continuous expansion ; there will be no "magic bullets" against cancer, but more and more drugs tailored to each type of cancer - it comes out almost every year. New antidiabetic agents (a disease in expansion and still badly treated), the gliptines, appeared in 2006. And progress continues ; for example, new treatments, simple small chemical molecules have appeared against the terrible cystic fibrosis...

I am rather proud to work in pharmaceutical research, in an industry that saves, heals, improves the lives of humans. And yes, there should be aggressive, wise, encouraging policy for therapeutic progress in Europe to ensure there will still be in next future a pharmaceutical industry in France and Europe ; otherwise we will have to buy new expensive drugs elsewhere, or do without.