by Yannis Natsis (17/03/2015)
I recently attended a seminar in Brussels on medical innovation beautifully hosted by an important pharmaceutical company. As always, the setting was ideal. Fancy 5 star hotel, delicious buffet lunch and lots of people working in public relations. The composition of the panel followed the usual recipe successfully tried in corporate events: An esteemed member of the academia, a president of a European patient federation, a classy pharmaceutical executive, an influential certainly pro-business EU public official and an expert. Last but not least, the discussion was moderated by an excellent American facilitator.
As the presentations kicked off, the University professor emphasized the incredible economic significance of the pharmaceutical sector for jobs and growth Europe and reminded everyone that the patent system needs to be defended by all means as this is one of the very few guarantees for innovation. Innovation was underlined by the patient representative too along with repeated and rather emotional calls for patients’ timely access to new treatments in order to meet “unmet” medical needs. The pharmaceutical representative stressed that her sector invests considerable amounts of money in research and development of life-saving treatments. She added that in spite of the very high failure rates, pharma companies do not give up because “human lives are at stake”. The public official agreed and reassured her that her business’ aspirations are taken on board by EU policy-making institutions. Last but not least, the independent expert reaffirmed the need for higher patent protection and stronger enforcement of intellectual property rights as the way forward.
During the Q&A session, the high cost of medicines was briefly touched upon only to be overshadowed by voices claiming that non-treatment costs society more and that patient outcomes should be the guiding criterion in cost-effectiveness assessments. As the icing on the cake, the public official noted that the absence of streamlining of regulatory and reimbursement requirements leads to higher costs for drug development and consequently to higher end prices. The patient representative along with people from the audience reiterated the necessity to institutionalize the early dialogue between all stakeholders, increase public funding and expedite access to new treatments. “Accessibility” of medicines was another highlight of the day seen under the prism of “innovative” methods of bringing drugs to European citizens through adaptive licensing, differentiated pricing, prolonged payment of curative treatments and a more active role of patients in the authorization procedure of new medicinal products.
The meeting came to a close and I felt puzzled. “Early dialogue”, “speedy access to new treatments”, “patient outcomes”, “innovation saves lives”, “innovative payment methods”, “streamlining of regulatory requirements” were all familiar themes and at the end of the day, they make sense and do sound nice. It struck me however that new battlegrounds are appearing as pro-industry lobbies work proactively to guide future European legislation. On the EU level, there is a consistent effort to review and eventually redesign the drug development pathway from discovery to product launch and post authorization monitoring to make industry’s life easier to the detriment of public interest. To this end, the pharmaceutical industry is shaping the agenda by choosing its facts and promoting a language that is easily owned by everyone. Let’s examine some of this rhetoric but from a different perspective, that of thinking of health in terms of a public good and not merely a business.
Early dialogue of all stakeholders (regulators, payers, European Medicines Agency-EMA, Health Technology Assesment-HTA bodies): as there are multiple levels of competences and evaluation criteria split between the EU and the member states during the different phases of drug development, industry wants to guarantee that it has a decisive say in the process early on. They want a detailed overview and a reserved place around the table during the market approval phase and the very important pricing and reimbursement stage. Adaptive licensing & parallel scientific advice are two of the main EU initiatives in this direction.
Early and “speedy” access to medicines: this is the prevailing theme in the EU legislative bodies at the moment. The Council is currently reviewing the existing mechanisms of early access such as conditional approval, compassionate use, and approval under exceptional circumstances. The risks are that very expensive medicines will enter the market faster on fewer reliable clinical trial data and a higher chance of unforeseen adverse effects.
Convergence of evidence requirements: in the spirit of “early dialogues”, industry pushes for a convergence of criteria in relation to market approval, the health technology assessment and the reimbursement phase employed by the different organisations in charge of these stages. Moreover, they highlight the primacy of the European Medicines Agency (EMA) as they do not wish to face additional hurdles from national regulatory authorities. This way they can predict what is coming up and how to tailor the different evaluation stages to their capacity.
Pricing & reimbursement decisions: this is probably the most important stage as it determines patients’ access to a treatment. Market approval is (or at least should be) based on scientific criteria in order to evaluate the efficacy, quality and safety of a drug. This makes it de facto more predictable i.e. more manageable. On the contrary, pricing & reimbursement is and has always been a political decision based on a different set of criteria.
HTA bodies: these are crucial for evaluating the added therapeutic value of new medicines. Therefore, HTA bodies are increasingly regarded as gatekeepers against the flood of me-too medicines that offer modest or no real therapeutic value but usually cost a fortune to public health systems in Europe and beyond. The more HTA’s role grows as part of the regulatory framework, the more they are targeted by pharma. This is due to the HTA’s distinct and instrumental role in introducing the concept of “therapeutic added value” into the market authorization procedure. This concept signals a higher threshold for the entry of new truly innovative medicines. The EMA –whose independence and capacity to control conflicts of interest are doubtful- is currently running pilot projects on adaptive licensing and parallel scientific advice that may a) threaten HTA bodies’ independence, b) weaken the regulatory framework, and c) lower safeguards.
Cost-effective, synergies between all stakeholders: emphasis is placed on cost-effectiveness against cost-benefit analyses. Moreover, industry and the EMA’s pilot projects aim to redesign the traditional licensing path with a view to putting their products earlier on the market while relying on fewer clinical data. It is worrisome that the language used in adaptive pathways (initially named adaptive licensing) widens the scope for the entry of new medicines referring to all medicines and not only those to treat unmet medical needs.
Views on early access vary considerably among EU member states as there are economic concerns especially for those where full reimbursement of treatments is foreseen. That is why, when it comes to HTA, they favour closer cooperation but wish to keep the final assessment on the added value strictly on a national level and oppose any efforts towards a single European HTA. In the meantime, the European Commission has announced that it will conduct a study to compare how member states implement the various early-access schemes-they want to make sure that there is no market distortion, referring to off-label use. The role of patient associations in this debate cannot be overlooked. They are often encouraged by pharmaceutical companies to put pressure on governments to demand early access to treatments as is the case with the seemingly private Dutch initiative presented via mytomorrows.com which is heavily propagated in the Council by the Netherlands.
There may be no new attention-grabbing legislation coming up but all of these initiatives and pilot projects need to be closely monitored as they influence ongoing debate and most importantly future legislation. On the whole, should pharma’s strategy be successful, it will result in an ever closer incestuous cooperation between the regulator and the regulated where any independent scrutiny of new medicines and their prices could be seriously weakened.