Saturday, 2 March 2013

EURORDIS: Sounds Like a Plan, Yann!

Last week I was invited by Yann Le Cam, CEO of EURORDIS, to some important rare disease policy events in Brussels. These events brought together a very passionate community of people including EC officials, MEPs, representatives from the European Medicines Agency, national officials, Pharma, researchers, patient groups and others. All these people are working on ways to improve access to orphan medicines and better treatment options for people in the EU with rare diseases.  I was really impressed with the way people from across the spectrum were all working together and making a difference. It felt like there was a great sense of camaraderie and real solidarity with everyone uniting against "the system" which I found surprising and inspiring. I found the experience genuinely moving and it was great to have the chance to meet so many smart people working in such an important and relevant area. 

Based on my recent experience of the issues with getting Vertex’s Kalydeco to patients in need, I was keen to go along and find out what is being done at the EU level to address some of the problems. As I see it, there are problems at almost every point of the drug development cycle from clinical trial design and marketing authorisation through to payers' cost-effectiveness reviews and post-marketing data collection. 

Why does it take so long after EMA Marketing Authorisation for drugs to reach patients? Why aren’t new treatments compared to current standard of care? Why wasn’t Bronchitol tested against hypertonic saline? Why do regulators insist new antibiotics are tested against placebo when hardly anyone with CF is not on at least one antibiotic? Why don’t NICE and other payers take proper account of quality of life, adherence and the effects of a new treatment on patients’ family members? Why don’t people get angry about their governments wasting hundreds of millions on branded drugs when therapeutically-equivalent, lower-cost generics will do the same job? Why do people object to paying high prices for genuine innovation? Why does it cost so much to bring a new medicine to market? These sorts of issues are common to all rare diseases and we stand a better chance of changing things if Pharma, patient groups, clinicians and others work together as everyone’s interest is served by improving access to rare disease medicines and the real problem is often government or State-controlled healthcare providers and health insurance systems.

A rare disease is any disease that affects a small percentage of the population. The European Union considers a disease as rare when it affects fewer than 1 in 2,000 citizens. Rare diseases are frequently chronic, progressive, degenerative and often life-threatening. Over 6,000 different rare diseases have been identified to date and 30 million people in Europe are affected. Rare diseases are also known as orphan diseases because they have traditionally been abandoned by governments and drug companies who cannot justify the investment in researching new treatments for such small groups of people. Despite their vast overall number, rare disease patients frequently lack access to diagnosis, treatment and the benefits of research.

EURORDIS is a non-governmental patient-driven alliance of patient organisations and individuals active in the field of rare diseases, dedicated to improving the quality of life of all people living with rare diseases in Europe. EURORDIS represents 561 rare disease organisations in 51 countries (including 25 EU Member States) covering more than 4,000 rare diseases.

In terms of EU rare disease policy, EURORDIS is clearly the prime mover and they have built a collaborative community of key stakeholders to shape and implement policy at the highest level. Yann Le Cam and his team have built this community and really drive it forward in a way that benefits patients while keeping key stakeholders on board. That is a wonderful thing. Overall I had a fascinating time over the Tuesday and Wednesday in Brussels and I hope to be able to build on my relationship with EURORDIS and the rare disease community as a whole.

The Big Picture 

There seems to be a realisation, in this community at least, that science is progressing in such a way that medicine is increasingly able to address whole new areas of disease and treatments are becoming more personalised. Treatments which only work on a small number of people are very expensive. We are moving into a world of ultra-orphan indications. It is clear that we will not be able to afford to pay for everything. So everyone is thinking very hard about what is funded and how to change the way the system is funded to maximise the benefit. In particular, there is increasing concern about the fact healthcare in the EU is largely “pay-as-you-go”/ unfunded in contrast to pensions which are generally funded, albeit poorly. Economists all agree that healthcare spending is going to become a far more significant proportion of total public expenditure in the future. Social/welfare commitments are seen as fundamental to the EU model so we had better find a way of paying for it! There is an increasing realisation that better healthcare would allow people to work more and for longer so spending in health will take pressure off social/welfare budgets. There should be more “fluidity” between health and welfare budgets. People, in this community at least, are starting to think about this in a more integrated way. 

It turns out that Pharma may be prepared to negotiate caps on general pharmaceutical budgets if it means more spending on orphan products. This is seen as a “re-distribution towards innovation” and something to be welcomed.  Similarly, everyone involved in the rare disease community should help to identify and highlight inefficiencies / waste in healthcare systems so as to force their governments to make savings where possible and create a greater degree of “headroom for innovation”.

There was a lot of talk of the need to mobilise “civil society” to force change. While EURORDIS is seen as a very effective voice for rare diseases in the Brussels village, there is an urgent requirement to mobilise local patient groups and other grass roots activity.

Tuesday 26th
Seminar on Faster Access to Medicines for Rare Disease Patients: “The Transparency Directive”

Why is it important? 

This measure aims to speed up the process of Health Technology Assessments and Pricing and Reimbursement decisions. It aims to remove duplication of work between the EMA and HTAs in individual countries and to bring greater transparency.

There is already a transparency directive in place but it is “soft” and poorly implemented and it is no longer compatible with the dynamics of the pharmaceutical market, particularly in relation to generics. The “new” Transparency Directive aims to bring greater transparency to the Pricing & Reimbursement (“P&R”) procedures in each Member State (e.g. the HTA reviews carried out by groups equivalent to NICE). According to EU officials, it is widely known that officials in several Member States are currently bribed to delay the entry of generics. A major objective is to stamp out corruption and save money for payers, particularly by accelerating generic substitution.  It is estimated that “late” transition to generics costs the EU more than €1bn per annum. Saving money for healthcare budgets in this way would release funds for the more expensive orphan drugs that will be an increasing feature of the market going forward.

The three elements are:

1. Time Limits: 180 days hard deadline for all P&R decisions but much shorter (30 days) for generics;
2. Statement of Reasons: national authorities must provide verifiable grounds for decisions; and
3. Remedies: legal remedies to be made available to applicants in the event of adverse decision.

The Directive also seeks to bring a clear separation of the Marketing Authorisation (MA) process and P&R processes in Member States and to eliminate duplication of work as far as possible given the fundamental rights of Member States to organise and fund their own healthcare systems (Article 168/7 Treaty on the Functioning of the European Union). The Directive aims to stop competent authorities in Member States from re-assessing aspects that have already been thoroughly assessed by the EMA as part of the MA process; to stop them “re-opening the science” or even re-evaluating the orphan designation.

Richard BergstrÖm, Director General of the European Federation of Pharmaceutical Industries and Associations (EFPIA) talked about how the pharma market had changed. Traditionally, pharma would consider their job done when their products got MA. Now there is a “4th hurdle” – the Pricing & Reimbursement hurdle. The real issue at the moment is access to medicines and that means who is going to pay, how much and when. The Transparency Directive is a necessary and important technical/procedural measure but it does not address the key issue. He said the current system in the EU is a mess and it doesn’t work for patients, payers or Pharma. It is too fragmented and this makes it very difficult for pharma. Pharma is open to talking about price and doing deals but there is currently nobody to call. For regulatory matters it is easy, you just go to the EMA. For P&R matters, there is no place to go. There needs to be a “front door” where people can go and have a sensible discussion.

It was great to listen to Richard BergstrÖm talk. He has an ability to cut through the procedural language and bureaucratic obstacles to get to the big issues, join all the dots together and say what really matters. It is incredibly refreshing to hear such integrated thinking and, as a patient, I found it extremely encouraging. It is mad that you get  labelled as a shill for the Pharma industry if you criticise government and market inefficiency when in fact your only objective is to get medicines to patients faster and encourage Pharma to take the risk of developing new treatments. The media in the UK is extremely "tabloid" in its approach to these issues and it is very unhelpful. I always feel politicians try to deflect attention from their own failings by seeking to paint Pharma as the bad guys and the media lap this up as it plays so neatly into the anti-capitalist narrative. Why don't people get more angry about their governments wasting hundreds of millions of Euros on branded drugs when generics will do just as well? Yet we balk at paying a decent price for genuine innovation (even though the total amounts represent a fraction of the wastage elsewhere). BergstrÖm  made a strong point when he said society wanted a "tap" of new drugs but they did not want to pay for the pipeline to supply the tap. I got a powerful sense that BergstrÖm  is an agent for positive change and I wish there were more people like him. 

Antonyia Parvanova, the brilliant outspoken MEP who is sponsoring the Transparency Directive and Cristian Busoi MEP who is also instrumental in this legislation, talked about an ambition to build a common market in pharmaceutical products - it is not one of the product categories for which a common market has been established under EU law. They see the Transparency Directive as critical to bringing clarity and transparency to the HTA/reimbursement process, eliminating corruption and saving substantial amounts of money by accelerating the transition to generics and getting new medicines to market faster. 

The emphasis is on showing the value / savings available to all parties rather than sanctions. Payers will save substantial amounts of money, pharma will be better able to invest and patients will get better access to treatments. 

There is a great deal of work going on around the total burden of rare diseases (i.e. health, welfare and social costs) so that we can see the true value of new medicines.

In terms of broader EC policy, Jaroslaw Waligora (Policy Officer, DG Health and Consumers) talked about the importance of patient registries in rare diseases. These are seen as crucial to effective research going forward. Currently there are hundreds of separate rare disease registries around the EU, not compatible with each other and hard to access. They need to be easier to aggregate and analyse. There are some initiatives that are helping (e.g. EPIRAR, EJA Joint Action, RD Connect) and there is a new EC initiative to establish a common platform (IT tools, software). This aims to bring greater compatibility, enable better data analysis and facilitate clinical trials. The disease-specific “European Reference Networks” made up of “Centres of Expertise” are an important part of this too.

Several audience members raised concerns about the Data Protection Directive and made the point that it raises barriers to patient registries which are so crucial to rare disease research. EURORDIS has released a clear statement on this issue (in defence of patient registries) and others with an interest in rare disease research should voice their concerns too. This issue is particularly important to CF and if patient registries are made impossible to operate as a result of the Data Protection Directive, as proposed, it will be a serious barrier to the development of new treatments.

There were about 100 people in the room including EC officials, MEPs, other officials from Member States, people from EMA, Eurordis, Pharma, Researchers and Patient Groups. The meeting was broadcast live over the web.

EURORDIS “Black Pearl” Gala Dinner 2013

This was a fundraising event with around 300 people in attendance. The aim was to explain the achievements to date of EURORDIS and the rare disease community but also to highlight the massive challenges ahead and the need for greater cross-border co-operation. They played some incredibly moving videos showing real life stories of people with rare diseases. Awards were presented to certain individuals and companies who had made a significant difference to the lives of those with rare diseases.

EURORDIS Awards 2013

Policy Maker Award: FranÇoise Grossetête, MEP
European Rare Disease Leadership Award: Dr Ruxandra Draghia-Akli
Volunteer Award: Lesley Greene
Scientific Award: Dr Ségolène Ayme
Patient Organisation Award: AlstrÖm UK
Company Awards: Celgene, Prosensa, Genzyme
Media Award: Andrew Jack
Lifetime Achievement Award: Former First Lady of Germany, Mrs Eva Luise KÖhler

I sat next to the wonderful Avril Daly who is a Board Member of EURORDIS, Head of Public Affairs for the Irish Charity Fighting Blindness and a leading figure in the Irish rare disease community. We talked about CF in Ireland and the work of EURORDIS. Avril’s energy and commitment is incredible and she is a true inspiration. Others on my table were Ken O’Reilly and others from Biogen Idec and Axel Van Der Mersch who is a major funder of EURORDIS. 

Wednesday 27th 
EURORDIS “Round Table of Companies”:  Improving Access to Orphan Medicinal Products

Yann Le Cam gave a presentation summarising the latest policy initiatives and emerging thinking around rare diseases in Europe. Key themes included:

Evidence of EMA flexibility: when it comes to new orphan drugs

Adaptive/Progressive Licensing: the idea that you could start off with a licence in respect of a small population after a small trial but then gather more data and do progressively larger studies to build the evidence base and get a progressively broader licence over time. This would mean a constant cycle of data and a need for regulators and payers to become more comfortable making decisions on less evidence (at least initially)

Progressive Pricing: as the indication expands, pricing comes down.

Clinical Trials: trials need to anticipate the need for much better therapeutic value demonstration (HTA).

Payers: will always have their own views but they should not re-open the science as assessed by EMA.

Marketing Authorisation: EMA and others should share more information, more quickly with HTAs and Payers.

Value: what is value in orphan drugs? Greater focus on value-added for patients, physicians, payers, pharma, society as a whole.

FDA/EMA collaboration: move towards stronger collaboration in terms of scientific advice, protocol assistance, trial design, regulatory processes.

The two key initiatives being sponsored by the European Commission, namely CAVOMP and MOCA, are designed to improve access to orphan medicines and treatments for rare diseases. The emphasis is on creating a more efficient market and creating real value for all parties rather than thinking about the issues in terms of Corporate Social Responsibility which has been shown to be ineffective.

“CAVOMP”: Improving Informed Decisions Based on the Clinical Added Value of Orphan Medicinal Products

This is essentially a proposed methodology for removing duplication of work and sharing information more effectively (among regulators, national competent authorities, HTAs, payers, patient groups, clinicians, et al.) through the whole lifecycle of a new drug (Orphan Designation – Protocol Assistance – CHMP Opinion – Marketing Authorisation – HTA – Reimbursement). A key part of this is a permanent co-operation mechanism for HTAs building on the EUnetHTA. EUnetHTA is a network of government appointed organisations (from EU Member States, EEA and Accession countries) and a large number of relevant regional agencies and non-for-profit organisations that produce or contribute to HTA in Europe. See:

EUCERD made a formal recommendation to the EC in September 2012 and the EC is considering how to implement it and incorporate it into future policy.

“MOCA”: Mechanism of Coordinated Access to Orphan Medicinal Products

The objective of this work is to find common, non-regulatory approaches to ensure better, timely and equitable access to orphan medicines after their marketing authorisation.

A key aspect is better identifying the true value of new orphan drugs and moving to a pricing system based on value rather than cost. While it is acknowledged that payers will want visibility on cost construction as a quid pro quo for market exclusivity, there is a need to move towards a more rational value-based approach. This is called the Transparent Value Framework (“TVF”).

The main criteria under discussion as part of the TVF are:
a) Availability of alternative treatments / unmet medical need;
b) Relative effectiveness (clinical improvement, QoL, etc. vs side-effects) and societal impact;
c) Response rate; and
d) Certainty of evidence.

I took part in a "breakout” session to talk about this and I raised some points about the burden of treatment in diseases like CF: a) that new treatments which reduce the burden of treatment and which are likely to have a positive impact on adherence (with potential associated savings for payers) should be valued more highly; and (b) that it is important for clinical trials to be designed in a way that provides HTA’s (and clinicians/patients) with the information they need to assess the benefits of new treatments in comparison to current standard of care as it exists in reality rather than as perceived by the FDA / EMA. These points were well received, people felt they were relevant to rare diseases generally and a number of people came up to me afterwards to say what a useful discussion it was and that they were keen to talk more.  

The final report is due in March 2013 and the EC will consider how best to implement the proposals and how to factor them into future policy.

Research also shows a great inequality of access to medicines amongst Member States (particularly in the countries suffering financial hardship) and major inequalities in pricing. For example, the Czech Republic pays more for medicines than the UK.  As a result of “reference pricing” where certain countries in the EU insist on paying the same as certain other countries in the EU system (even if they are in financial distress and benefiting from special deals with Pharma), there is now a tendency for Pharma to refuse to supply certain markets so as to protect pricing power in other more important markets. There are discussions going on as part of the MOCA process around mechanisms to improve equality in these areas. “Differential Pricing” is seen by all (including Pharma) as a viable option. This would involve countries in the EU with lower GDP/capita being charged lower prices.

The other topic of the day was “National Measures”. Each Member State has been told to develop a national plan for the treatment of rare diseases and this includes improving access to new orphan medicines. A few countries have implemented their plans, notably France, some have developed but not yet implemented (e.g. UK) and others are still developing.

Another topic discussed at various points was the concept of the “right to reimbursement” and free movement within the EU. In other words, rare disease patients resident in one country should be free to travel to other countries in the EU and be entitled to the same level of treatment. This is seen as an important long term objective. There are increasingly cases of people travelling or even moving to places where they can get treatment which is not available in their home markets and even cases where parents of children with rare diseases cannot afford to move to a new country so they put the child up for adoption to go to families living in countries where treatment is available. France absorbs a lot of patients in this way and it is seen as a major issue in terms of equality and sustainability.

Eurordis will be making all the presentations and other materials available on their website in due course. A huge amount of ground was covered in much more detail than I go into here but I hope this gives a flavour of everything.

This is a great community of people. I had expected it to be a lot of hot air and grand talk of unrealistic ambitions but I was surprised at the level of camaraderie and the way people were cutting through to practical issues. It was also very striking how everyone from the EC, MEPs, Pharma and Patient Groups were genuinely collaborating with a shared goal of getting medicines to patients faster. It was quite inspiring and definitely something to build on. 

In addition to the brilliant team at EURORDIS, I met people from the European Commission, European Medicines Agency’s Committee for Orphan Medicinal Products, Fighting Blindness, Alliances Maladies Rare, the European Network for Ichthyosis, the Belgian National Health Insurance System and the following Pharma companies: PTC Therapeutics, Pfizer, Genzyme, BioMarin Europe, Bioden Idec and Celgene.

I attended these events as a representative of the CF Trust and I would like to thank them for part-funding my trip.


  1. nice experience. Nice and happy to read this. Thanks for your post

    custom dissertation writing service

  2. Great article.Thanks for your details and explanations..I want more information from your side..I Am working in erp development company in chennaishould you need for any other clarification please call in this number.044-6565 6523.

  3. Hi, nice blog really good and informative.... and I will be visiting often and telling my friends about this Hope to read more good articles. university essay writing service

  4. I think it was a truly nice experience. You have described this in an appealing way. View my personal/blog/work website, if you are interested to know about what we can do for your blog to become successful!

  5. I am happy as Dr. Benson sensitive treatment of Cystic fibrosis (CF) touched me deeply. I have suffered from Cystic fibrosis (CF) for twenty+ years after i have gone through surgery in 2012 and I have just figured out a Dr. which help me live a safer and less painful life after my family Doctor asked me to go for a second surgery. Don't ever lose hope. I am 41 and a mother of one now , Sure, it has been challenging, and there have been many ups and downs, but I went to internet got Dr. Benson contact I contacted him and he sent me the permanent cure to it,which I used for one month. and now i am having pretty normal life now. This is not the end of the world if you having Cystic fibrosis (CF). he can help you. The medication it taste natural. And most of all, I want to tell you that you need to learn to love yourself just the way you are- you are a special person that deserves love. Never never never give up!!!!
    Contact Dr Benson on

  6. I like this blog, saved to my bookmarks.I have got some important suggestions from it. I'm working in Fuzzy International private limited.

  7. Oh, that's super great, tanks for sharing your news, I really want to participate and I really want to be a good member of your community! Only if I don't go to my favourite site and don't spend all my time here. I just forget everything, when I'm here. But I'll try to manage it, even though that's so amazingly great source. Or we can play together. Don't you want to try?

  8. Thanks for your great information. Sign up and Register Your Events Today!!!!!!!
    Online Event Registration websites | Upcoming Events & Fests List