Game Changers for Better and Affordable Medicines for Europe

Universal Access & Affordable Medicines Forum of the European Public Health Alliance, organized by the European Public Health Alliance (epha) under the auspices of the Belgian Ministry of Health 21.11. 2017

By Lauren Tuchman, ANME

This was a well-attended event filled with varying participants from a multitude of organizations. The atmosphere was lively, questions were raised, and the panels throughout the days were diverse, interactive, and the members actively engaged with the attendees. In the opening remarks from Yannis Nastis, (Policy Manager, Universal Access and Affordable Medicines, European Public Health Alliance [EPHA]), one point was made that was reiterated by many of the panelists of the day: Access to medicine is no longer just a topic and concern for the Ministry of Health; it is important that the Ministry of Finance become more involved and address public concerns.

For example, one such concern is that public is often paying twice for medicine —once as taxpayers for research and development (r&d) —and again once the developed drug is purchased by a private company and sold on the market/via the healthcare industry. Other important opening remarks were an open call to member states to use their leverage more, and the opinion that innovation should be incentivized.

Of note, the European Medical Agency (EMA) declined invitation to join the event, and therefore did offer commentary/discussion on any of the four panels.

Each panel was composed of 3-5 individuals, from varying vantage-points, companies, or advocacy groups, etc. All discussion panels were moderated by Dr. Deborah Cohen, Investigations Editor and British Medical Journal (BMJ).

The following were the four panels of the day:

1. Transparency in Pharmaceutical: Desirable and/or feasible?
2. Drug Development: Do the incentives work?
3. The Push for Accelerated Drug Approvals: speed and deregulation over patient safety and robust evidence?
4. Business Strategies: Anticompetitive practices & medicines’ shortages: what is the way out?

Below is an overview of some of the important opinions offered by the panelists from each of the four panels:

1. Transparency in Pharmaceutical: Desirable and/or feasible?

Panelists: Dr Andrew Hill, Senior Visiting Research Fellow, Department of Pharmacology and Therapeutics, Liverpool University; Dr Síle Lane, Head, International Campaigns and Policy, Sense about Science, UK; Francis Arickx, Head, Pharmaceutical Department, National Institute for Health and Disability Insurance (RIZIV INAMI), Belgium; Aldo Golja, Senior Policy Advisor, Pricing and Reimbursement-Coordinator of the BeNeLuxA Initiative on Pharmaceutical Policy, Ministry of Health, Welfare and Sport, The Netherlands; Boris Azais, Director, Public Policy for Europe and Canada, MSD

According to Dr. Andrew Hill, companies do not want the public to know the price for the raw product/material for the drug. And, additionally, it is very difficult to find such information because these prices are not readily accessible online. Dr. Hill recommends therefore, that the prices need to be clear across Europe —as there is a present divide and incongruency in pricing and transparency. Dr. Hill also commented that with respect to Hepatitis C, most countries across Europe are not on target for getting rid of disease and, furthermore, most countries do not understand how much they are overpaying. Dr. Hill recommended that the focus should be on lowering the price of generics because then it becomes harder and harder to justify the high prices for the name brands.

Dr. Síle Lane commented that half of all clinical trials have not been shared/published. This means that a vast amount of information is missing, and also that the information that is there is biased. Dr. Lane also made the point that when results are not shared, it is a big violation of the trust of the volunteers that sign up for such trials. Dr. Lane believes that a gold standard clinical trial transparency is achievable, and people are increasingly making investment decisions based on transparency. She also commented that in addition to the companies not publishing findings, the laws that are in place that state that companies must register clinical trial before they start, and then publish results up to one year after, are not being followed… and there are no repercussions for this.

Francis Arickx, (who is the head of the pharmaceutical department of a financially-sound payers organization), also advocated for transparency between member states. He believes the transparency should include: what money is being spent on, what price the states are willing to pay, and what healthcare budgeting and spending projects look like.

Aldo Golja, who has been instrumental in developing the BeNeLuxA initiative, raised to attention one potential problem that accompanies the increase of transparency. He believes that if transparent is increased for the sake of research and development (R&D), then there needs to be sustainable payment model…and this causes a problem because how could/does this benefit everyone? For example, how does this benefit the public, the patient, and the companies who are doing the research? He therefore recommends and increase in interaction and collaboration.

Boris Azais commented that within the last decade, healthcare expenditures have increased by 1.4%. He said it is clear that payers want to know that they are no overcharged and sellers want to know that they are not underpricing. Mr. Azais also pointed out that there is an equity problem among rich and lower economic countries. Additionally, he finds it important to have further clarification and transparency on what percentage is spent on pharmaceutical research vs. medical research — this means research on the drug vs. research on the disease or medical tools, equipment, etc.

2. Drug Development: Do the incentives work?

Panelists: Dyfrig Hughes, Professor of Pharmacoeconomics, School of Healthcare Sciences, Bangor University, Co-Director, Centre for Health Economics & Medicines Evaluation; Els Torreele, Executive Director, Access Campaign, Médecins Sans Frontières (MSF); Elizabeth Kuiper, Executive Director Public Affairs European Federation of Pharmaceutical Industries and Associations, EFPIA; Marcel Canoy, Advisor, Netherlands Authority for Consumers and Markets (ACM)

Dyfrig Hughes pointed out that gains in health are valued at ten times (10x) higher in people who are suffering from rare conditions. Gains, therefore, for orphan drugs are higher.

Els Torreele stated that 70% of drugs that reach market have no added therapeutic value, and this is the same as the last several years. Ms. Torreele has suggested also that there have been few new antibiotics developed in the last twenty years. She also stated that there is more incentive to develop a life-long treatment rather than a cure! Mr. Torreele also pointed out that there is nothing in patentability that addresses unmet medical needs and health needs —and works towards making such affordable.

Elizabeth Kuiper, (Director Public Affairs European Federation of Pharmaceutical Industries and Associations), reiterated the point that the Ministry of Finance role is more important than it was in the past. Healthcare systems need to prepare for a different way of paying. She believes that competition is really important for pricing. She also acknowledged that pediatric pricing is really high.

Marcel Canoy discussed how ‘pharma' is incentivized to innovate in these areas where the companies have the best bargaining position. This is a problem because the incentive should be to invest in drugs that have the most value to society. While Mr. Canoy believes that the blame is on pharma, he also acknowledged that such companies are merely just “playing the system” — and the real problem is with the system.

3. The Push for Accelerated Drug Approvals: speed and deregulation over patient safety and robust evidence?

Panelists: Claudia Wild, Director, Ludwig Boltzmann Institute for Health Technology Assessment; Antonio Addis, Researcher, Department of Epidemiology, Regional Health Service, Rome; Simon Collins, Patient Advocate, HIV i-Base; Beate Wieseler, Head, Department of Drug Assessment, Institute for Quality and Efficiency in Health Care (IQWiG); Helen Lee, Unit B5 – Medicines: policy, authorisation and monitoring DG Santé, European Commission

Claudia Wild spoke that there is almost no data that suggests that fast approval is delivering better drugs than slower approval. And, such a thing is not working in favor of the European patients.

Antonio Addis added to this commenting that research can be very useful, and special measures should be put in place before the process is accelerated.

Simon Collins, (a patient advocate), made an important comment: What people are prepared to pay for something depends where they are in the timeline of their treatment, and the severity of the illness. Mr. Collins also pointed out that the surrogate market data is, on the whole, insufficient and lacking. He did, however, state that in the case of HIV, there is pretty good data with high clinical outcome(s). Mr. Collins also pointed out that sometimes data is driven by independent researchers, and he therefore advocates open-access to the patients on anything that involves their health — patients should be able to have vital information about the drugs that are being given to them and that they are consuming!

Beate Wieseler stated that data does not always exist so early on. She believes that patients are better off with evidence-based decision making. Ms. Wieseler also mentioned that we do not know what new drugs will do to and provide for our healthcare system and the effect this will have in the long run. And, furthermore, the follow-up data/post-approval data often is never given. Ms. Wieseler therefore suggests that it is best to spend money —and more specifically better manage money— that is used for the post-approval collection of data.

Helen Lee spoke about the unlikelihood that a full data-analysis could or would be available. She therefore believes that the regulator could be more efficient, and the bar could be raised— and not lowered—to approve only drugs with a certain standard of benefit. Another example of raising the bar would be changing the perspective on ‘time scope’ for example, in cases such as oncology: to look at the benefits achieved in months, not days.

*At this point an informal poll was taken in the room, and the overall feeling, by way of a show of hands, was that there is inadequate and lack of quantity and diversity of data!
Again, one concrete solution proposed and reiterated by Mr. Addis, was to look more into the specific disease, rather than specific drugs.

4. Business Strategies: Anticompetitive practices & medicines’ shortages: what is the way out?

Rainer Becker, Head of Unit for Antitrust Pharma and Health services, Directorate-General for Competition (DG COMP), European Commission; Marc-Alexander Mahl, President, Medicines for Europe; Aida Batista, Vice-President, European Association of Hospital Pharmacists (EAHP); Fernando Lamata, Senior Expert on Health Care Policies, Impeller of the Association for a Fair Access to Medicines, Spain; Vlad Voiculescu, Member, Romanian Health Observatory

Rainer Becker discussed how it is important to check for abuse of those companies in the dominant position. He also stated that a common ‘exclusionary practice’ is to squeeze out competition, and then the companies are of course at liberty to do what they like with pricing. Mr. Becker also pointed out that it is important to differentiate between the pricing and the profitability in the generic versus the originator market. This is because generics follow a different profitability mechanism. For a better example of this, one could look into what is going on in North America in the case of ‘bio-similars’.

Fernando Lamata also commented that he believes there is a large abuse of those in the dominant position.

Marc-Alexander Mahl commented that when a product goes off patent there is the opportunity to offer the generic price, yet the tenders do not generally open up. Mr. Mahl believes that this is a missed opportunity for healthcare systems, and something that could be taken advantage of on day 1 when the patent opens. He also commented that he would like to see the EC incorporate Supplementary Protection Certificate (SPC) waivers.

Vlad Volculescu added the commentary that there is a lot we do not know about the drug shortages, and, furthermore, there is no data on the impact of shortages on national budgets, and impact on hospitals, etc. Additionally, there is no data on what measures are being taken to curb shortages. Mr. Volculescu believes that there is a great need for early reporting mechanisms. And, ultimately, he questions why there is the case of such shortages in Europe? He believes better management and prevention of such is of great necessity.