Emmanuelle Voisin, Founder, and CEO of Voisin Consulting Life Sciences is an expert in regulatory sciences and market access. In this exclusive interview, she shares with Biotech Finances her view of the rapid changes in the requirements of regulatory agencies and payers, and their impact on the entire Biotech and Medtech ecosystem.
Can you describe in a few words, the activity and the mission of VCLS?
Basically, our role at VCLS is to build the clinical trial application and marketing authorization application for health tech products developed by our clients and represent them in front of the health authorities in Europe and the United States. We translate science into regulatory terms, a benefit/risk ratio, which is the sine qua non-condition for this science to be valued. We work with some major pharmaceutical companies and medical devices manufacturers, on very specific topics, where they do not have all the expertise. Our main clients are biotechs and Medtechs, who sometimes have neither regulatory expertise nor internal resources.
You created Voisin Consulting Life Sciences in 1997, What was the genesis of this company?
Initially, my business plan was very basic, to collaborate with people who share common values, participate in very innovative projects and leave the world of large structures and work on my own account. The leaders of biotechs are brilliant, their products are very promising hence working with them is exhilarating. Since health product development plans and the construction of regulatory dossiers are not their specialties or their core business, I had something to offer them and the challenge was to move forward with them as pioneers. In some innovative therapeutic areas, regulatory pathways did not yet exist. Our mission was, therefore, to support our partners as much as the health authorities in new territories. This was the case with my first client, the Paris biotech IDM Pharma. It was specialized in cell therapy at a time when there was no regulation, neither in France, nor in Europe, and just in the United States, for this type of product. In the evening, after the working day, a director of the French Medicines Agency came to talk with the CEO of IDM. These exchanges were at the origin of the very first regulation specific to cellular therapy products in France.
At the end of 1999, the regulations for orphan drugs were introduced in Europe, we had in mind the philosophy of the orphan as it already existed in the United States. We were able to anticipate the agencies’ expectations and be the first to submit a dossier, with Trisenox. In the years that followed, we also played a leading role in European regulatory authorities in the development of regulation for gene and cell therapy products (ATMPs), and later for pediatric products.
In 21 years, VCLS has grown a lot. In which directions did you advance?
I started my career at the FDA and then at Quintiles thus the American DNA of VCLS. My first clients were mostly based in the United States for which we accompanied to the European agencies and worked hand-in-hand with Dr. Peter Hoyle, my former FDA supervisor, who became a consultant who referred some of his clients for the European approach. Today, we have many more European clients and at the same time, we also handle 100% US cases, representing local biotechs with the FDA which explains our internationalization. During the five last years, we developed strong market access expertise, which today represents a strategic business at VCLS.
Why has this topic become key to you?
Over the years, science has been asking questions about regulations and moving them forward. The latter has in turn allowed science to spark innovation and a virtuous circle has taken place. At the same time, prices have long been negotiated informally, almost relatively, between industry and the public authorities. But on this front, things change.
Today, payers are demanding ever-increasing amounts of data, extensive studies and ultimately, a real scientific demonstration of the cost-effectiveness of products and their societal impact. This is of course due to the soaring prices of certain medicines linked to the advent of precision medicine and the development of ultra-targeted therapies for pathologies with limited populations, or by breakthrough innovations. In therapeutic areas, such as oncology or orphan diseases, where the price of standard therapies is already high. Payers are under pressure and logically harden their level of requirements.
Today, we can say that it is neither the scientific innovation nor the regulatory constraint that represents a potential obstacle to the growth of the sector, but the economy of health. In other words, we anticipate that the prerequisites of the paying agencies will supplant those of the regulatory agencies in the future, since obtaining a favorable price will require more data and more evidence from the laboratories than obtaining the marketing authorization. It is already clear that a marketing authorization is no longer a guarantee of commercial success and we make our customers, biotechs and medtechs in particular, very aware of the fact that the pricing strategy of a product must be started in phase II of the clinical development.
Do you fear a blockage on these price issues?
If the question is "how to make gene therapy products, now under development, tomorrow reimbursed around a million dollars?” Then I do not have the answer. But we begin to imagine new patterns, with more post-approval commitments, more post-market data and more detailed information on the natural history of diseases. The principle is that no product will be left on the market without close monitoring and constant reassessment beyond basic pharmacovigilance. In short, the development of drugs will no longer stop at the marketing authorization as we also see things change on the payer’s side.
While regulatory agencies FDA and EMA collaborate closely under confidentiality agreements, payers’ particularly in Europe and national organizations are quite isolated lacking and demanding information. But a cultural change is on its way, there are more and more forums where we can meet and see them exchange and collaborate with others. There is a beginning of reconciliation which is very positive and needed by the industry. At this stage, they are not asked for a consensus, but at least a common language on the major topics of innovation.
What are the strong trends at the regulatory level?
One of the most exciting is probably in the field of digital with the processing of the algorithm as a medical device. We now see software capable of generating treatment recommendations whose effectiveness is clinically proven. With one of our partners, we proposed to the FDA a totally innovative development plan, starting from a clinical trial conceived from in silico data and whose results are used to model on the basis of prediction, a second test, and pivot. In other words, a development plan containing a cocktail of real data from patients and data in silico. The FDA did not tell us no! Which is a real revolution in our profession when we know how much the American agency has long been suspicious of the "predictive.”
Do you consider that the gap is widening between the FDA and the EMA?
Undeniably, the US agency under the leadership of its Commissioner Scott Gottlieb and driven by his holistic vision goes far and works well which was illustrated in the example I gave you prior. We are trying to steer the Europeans and encourage emulation. The EMA is populated by excellent experts, but they are overwhelmed and things would not work out right now nevertheless, there is nothing ideological. The FDA and EMA are open to innovation, and this is essential for the long term.
You work with a large number of French biotechs, what is your view of the French ecosystem?
Our science is excellent, but we have very few good leaders. The mentality that still prevails in public research is archaic and too many beautiful discoveries mold in the drawers of academic laboratories. As for public money, it is sprinkled rather than focused on the best biotechs.
Finally, too many biotech leaders want to do everything alone whereas even with several hundred collaborators, you cannot go on a clinical development program or go to the market alone. Not to mention the exchanges with the FDA, which are complex and codified. This work requires a wide range of expertise and skills and the establishment of internal and external virtual teams with consultants and CROs working in silo is very dangerous.