Networking in Brussels

OIFE had several representatives in Brussels attending the 3rd EURORDIS Multi-Stakeholder Symposium on Improving Patients’ Access to Rare Disease Therapies ( in February 2019.

During the conference there were also opportunities to network. Here with representatives from Open App, Bindeweefsel.Be and Mereo.

What was interesting to know? Our Honorary Member Taco van Welzenis gives an overview in the following report titled “More therapies for rare diseases!”:

Relevance for OI
EURORDIS, an alliance of 851 rare disease organizations, including OIFE, was the organizer of this event. The purpose was to discuss how more new therapies can be made available to rare disease patients. Many stakeholders like pharma companies, policy makers, politicians and rare disease patients were present. The 500 “most common” rare diseases constitute 98% of rare disease patients, the other 6500 “ultra rare” diagnoses are just 2% of patients. At this moment 95% of rare disease patients do not have a therapy available for their condition.

EURORDIS has therefore set the ambitious goal to get 3 to 5 times more new therapies approved per year for 3 to 5 times less of the cost by 2025. In 1999 the EU Orphan Drug Regulation (ODR) came into force, under the ODR 143 new products have been approved so far. The ODR attracts development of new medicines to Europe, thereby speeding up availability to the European market of the resulting products. OI is a relatively rare diagnosis with several new drugs for it in the pipeline – and possibly more in the near future – so the question of availability of new therapies is fully relevant to our community.

New therapies do not always reach the patient
But why is this such an issue? That becomes clear when we look at the chain of events that has to take place before a new therapy becomes available; Investment – Research – Authorization (EU level) – Assessment (mostly supranational) – Price negotiation, Allowance and Reimbursement (national level). Many complications can arise along this chain. Sadly this means potential good therapies sometimes fail to reach the patient. It can be because no agreement can be reached about the price, (very unsatisfying!), or the number of patients is too small to statistically prove efficacy. When an effective therapy exists but is not available for patients this can give rise to – illegal – production of the medication by hospitals, mistrust between parties and most importantly, huge frustration for patients.

The chain of events to reach a new therapy
Investors only want to give money when they expect a good return on their investment, the same for pharma companies (industry), who have to make a profit. Tests have to be done for effectiveness, safety and dosage. Rare disease therapies are a niche market and often smaller companies with less economic resources specialize in them. In Europe a central body decides about market authorization of therapies, the European Medicine Agency (EMA). When a product fulfils some strict criteria EMA can give it “orphan drug designation”. This is highly desired because this also means that for the first years a company gets exclusive access to the EU market.

Next health technology assessment (HTA) bodies look how well the effect and safety have been proven, what the value is to the patient, what the side effects are and how this compares to the price that industry is asking. Often HTA assessment is done for several countries combined. Finally the national health care authority of each state negotiates with industry about the price and decide about allowance on the market and conditions for reimbursement at the national level. Besides the HTA assessment also the national healthcare budget plays a role here, which is both an economic and a political issue. Some of the wealthier states still spend relatively little on healthcare. A government can decide to prioritize care for rare diseases. Budgets and policies may change with each new government. Some people argue that rare disease therapies are very expensive, while some are indeed, most are not overly expensive. Because of the small number of patients the total costs amount to only 3-5 % of the total healthcare budget. Recently costs have received renewed attention because research into rare diseases also gives us therapies for common diseases, some of these innovative therapies are expensive which is a budget issue.

Possible solutions
The EU is supposed to function as a single market. But for medications this does not hold true. The reality is that we deal with some medium sized markets like France and Germany and over 20 small markets. This leads to higher prices and inequality within Europe when it comes to access. A real single market would seem the best way forward. If the EU could go to the negotiating table as one, representing perhaps hundreds or thousands of patients per rare diagnosis – it would be in a stronger position to negotiate a good deal with industry. EU wide transparency is easier for investors and pharma too, instead of negotiating 28 times about (ultra) small numbers of patients and dealing with many different HTA bodies. It would undoubtedly boost investment and therapy development. United the EU could also try to tackle some issues like industries trying to make huge profits with repurposing existing medications or massive increases in price once a drug has been proven effective.

In order to harmonize policies in Europe however, countries have to be prepared to give up some control over their budgets and policies, and it also demands economic solidarity between EU members. Patients with a rare disease have an equal right to care and new therapies. This point has been made at the EU level but it has not been translated into policy enough yet. If we adhere to this ethical standard it follows that we should be prepared to invest in rare disease therapies until the same level of care has been achieved as for more common conditions. Patient outcomes and all aspects of value should be taken into account when deciding about acceptable price levels. If we apply less strict rules for proof of effectiveness (not safety) at the point of market authorization that will speed up availability too. This can be done if the EU sets aside some money for evaluation of the effect at a later stage. As OIFE we can advocate for a fairer, more transparent sustainable system, encourage a Europe wide y, further cooperation between the national healthcare systems, challenge misconceptions about orphan drugs, support EURORDIS and take part as patient experts at as many levels of the process as possible.

Taco van Welzenis, OIFE Honorary Member

You can see a recording of the whole conference here:

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