About me

I'm Alfonso Aguarón and I welcome you to my blog. Inspired by my own diagnosis of a Hodgkin's Lymphoma in 2008 I decided to get involved in patient advocacy.
A few years later, I keep commited to my desire of help and support patients. There's still a lot to do so, do we look for change in healthcare together? Let's go!

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Monday, October 5, 2015

European Reference Networks: The second chance

I remember when I first heard about the Cross-Border Healthcare Directive. By that time I thought that it was such a great opportunity to knock down barriers to access to care and also a good example of the so-called European spirit. What a naive dreamer I was! Two years after the deadline for transposition and implementation of the Directive, has just shown the true face of Europe in these times: an old cranky lady, without political will to build a more social and united Europe and member states serving only to their own interests. In the view of the "Evaluative study on the crossborder healthcare Directive" published by the European Comision, it seems obvious that the lack of information and the bureaucratic burden has turned the text into a nice declaration of intentions with no real outcomes for crossborder patients.

In the midst of this almost hopeless situation, there is still a ray of hope shaping as a second chance: European Reference Networks (ERNs).

What are ERNs? It is an European intend so the best specialists from across Europe could join their efforts to tackle complex or rare medical conditions that require highly specialised healthcare and a concentration of knowledge and resources.

So, what do ERNs bring to a patient in a practical way? Let us take an example. Let’s imagine that I’ve been diagnosed Castleman’s disease, a rare and poorly-understood inflammatory disorder that occurs in people of all ages, causes lymph node enlargement, and can result in multiple-organ dysfunction. This is an ultra-rare disease, with an incidence less than 1 in 100.000 people. It is likely that my doctor never treated a patient with the same condition or even my hospital lacks of the proper resources to treat me in a proper way. Thanks to ERNs my doctor could locate a reference center and a specialist who could either guide him to manage my disease or could recommend my transfer to that center in order to be treated adequately.

Which centers will be part of the ERNs? All those healthcare providers willing to be part of the network will be able to apply from January 2016 and they will have to meet the criteria established in the Commission Implementing Decision on setting out criteria for establishing and evaluating European Reference Networks and their Members and for facilitating the exchange of information and expertise on establishing and evaluating such Networks.

Will each disease has their own reference center? Let’s keep in mind that there are over 5.000 rare diseases, around 200 types of rare cancers as well as some other multifactorial complex diseases. The CID acknowledges that it might be difficult to reach the required minimum number of healthcare providers or Member States for some rare diseases or conditions due to a lack of expertise and it would therefore be a good idea to group healthcare providers that focus on related rare diseases or conditions in a thematic Network. Networks could also include providers of high technology services which usually require very high capital investment, such as laboratories, radiology services or nuclear medicine services.

Which criteria should a healthcare provider meet to be part of the ERNs? Admission criteria may vary based on several factors. Overall, they must meet the requirements established in the Commission Delegated Decision on setting out criteria and conditions that European Reference Networks and healthcare providers wishing to join a European Reference Network must fulfil.

When will ERNs begin to work? ERNs will start working in early 2017, as long as the centers in the network are big enough to enable the sharing of expertise and to improve access to care for patients across the Union. Otherwise, the Commission should ask member states to encourage their healthcare providers to join the proposed network.

While it is true there are still certain relevant aspects to solve, like who and when will “pay the bill”, it seems that ERNs are a more realistic approach to achieve an optimum care, an European shared-knowledge network and a better access approach. This still requires the involvement of member states and the pressure from all the stakeholders. Europe cannot afford to miss this second chance.
Saturday, August 1, 2015

Patients and their role in patient organisations

Summer time has come and I thought that it was a great opportunity to resume the normal activity of the blog. A few days ago I read a post in Celia Marin’s blog talking about an interesting topic which can be summed up in the new challenges of the professionalisation of patient organisations and how this can distance them from real patients.

Let’s start by saying that patient organisations (associations, networks, foundations, etc.) are quite different one from each other depending on their objectives, scope and organizational structure. You can find, for instance, from small volunteer-based support groups to big professionalised organisations; from local associations providing patient support to supranational foundations lobbying for treatment access; from Boards constituted by patients to HCP drive entities. Also you might find a wide variety of profiles in their driving-forces: patients, relatives, physicians and professionals of all areas, who can develop their work in a volunteer, part-time or full time paid basis.

Keeping in mind all these peculiarities of the advocacy community, let’s focus on the key issue: should it be mandatory to have patients involved in the driving-force of a patient organization? Even this is a hard question to answer with a yes or no, from my humble opinion, I would say no. Let’s go deeper into details.

Before I get started, don’t get me wrong: I think that patients and their inner circle can play an important role in patient organisations, basically because they are the ones experiencing the disease in their own flesh and this will certainly lead them to take some action in order to improve their lives and try to meet their uncovered needs. However, it is common to listen “only a patient can understand what another patient is going through”. Of course, there are many common feelings from patients suffering the same disease, but every journey is unique and, even it can be quite supportive for certain individuals, it shouldn’t be taken as the Holy Grail or we will fail into the error of proselytizing the role of the patient in the advocacy community.

A part from that, regardless of our organisation focus, there is always something to measure the actions of our organisations: the outcomes for patients. No matter if we’re focusing on providing psychological support in a one-on-one basis, developing educational tools about self-care, lobbying for better treatment access, funding clinical research or advocate for policy changes. At the end of the day what really matters is that patients are doing better than yesterday until we reach the point in which we won’t be longer needed, staying away from anecdotal or non-relevant results.  And for that, being a patient is not a must. What it is a must is commitment, liability, seriousness and hard work of every single person with an active role in our organisation, no matter whether they are volunteers, paid staff or trustees, no matter whether they are patients or not.

I know this is a controversial topic which has been raised in some forums in recent times and here it is my point of view. I would like to know yours. What do you think about it? I’m looking forward for your comments!

Thursday, April 9, 2015

When it is not just invisibility

We talk about invisible illnesses to refer to those diseases in which symptoms might not be apparent to other people's eyes. These diseases are usually chronic and have a variable impact in the quality of life of patients. It is complicated to strictly categorise all of them, but they include diseases such as diabetes, arthritis or heart diseases, to name just a few. According to some studies[1], approximately 96% of people who live with a chronic illness may look perfectly healthy but might suffer from symptoms such as fatigue, pain, dizziness or cognitive impairment. Many patient organisations around the world work hard to raise awareness with the goal of throwing light on this reality.

Most of these diseases are well known by the scientific community and there are lots of experts and many ongoing clinical trials aiming to get either a cure, either a proper management of the symptoms. Even a society used to ask for healthy and productive individuals knows, to some extent, about them and is usually aware of the constraints that patients may experience. But, what happens when some of this diseases are not only invisible to the eyes of society but also to the vast majority of the healthcare professionals and the pharmaceutical industry even when scientific evidence has already proved their existence?

In this regard, on the one hand, we refer to rare diseases, which are those with a low incidence, affecting a small number of patients. According to the European Organisation for Rare Diseases (EURORDIS), there are more than 6.000 rare diseases which have been recognised by the scientific community. Almost 245.000 people in Europe has been diagnosed a rare disease, which represents around 1% of the population[2]80% of rare diseases have a genetic origin and they are often chronical or life-threatening conditions. Given its nature, research on this field is more limited compared to other diseases with a higher prevalence. Ongoing clinical trials are usually based on the hypothesis that an orphan drug[3] might proportion a therapeutic benefit for the patient. While recognising that the investment from pharmaceutical industry in this field could clearly be improved, ad hoc joint working groups have been settled with research centers and patient organisations such as NORD in the U.S. and EURORIS in Europe in order to achieve significant results in this area. This underlines the importance of strong and empowered patient networks to advocate and raise awareness about these diseases which affect a large number of people. However, there’s still a long way to go in order to get optimal results.

Affect 1 in 2.000 people
United States
Affect less than 200.000 people
Affect less than 50.000 people
Affect less than 1.000 people

But this problem doesn’t affect only rare diseases. I’d like to mention here the concept of “emerging diseases”, not in its classic sense but to refer to those diseases which have been recently discovered based on scientific evidence, but which are not generally well known and managed by most of the healthcare professionals, research centers and the pharmaceutical industry. Diseases such as porphyrias, chronic fatigue syndrome (also known as myalgic encephalomyelitis and systemic exertion intolerance disease), Lyme disease or post-traumatic stress disorder, to name a few of them. The research on these diseases is limited to a reduced number of specialized research centers, the investment in research is very low and infradiagnosis due to the lack of training and awareness of non-expert clinicians is too frequent. Symptomatology is usually attributed psychosomatic disorders, hypochondria, anxiety, depression or psychiatric conditions. At this point the disease goes beyond invisibility, causing late diagnosis which can extend for months an even years and tens of visits to the doctor and referrals resulting in a tremendous impact on the integrity of the patients and their environment, not only in the physical dimension but also in the emotional, social, work and economic sphere.

What is the solution? When almost EVERYTHING remains to be done, the main driver of change is the patient and their environment. It is an arduous task along with the limitations of the disease itself. In my particular opinion, research investment, political pressure and recognition of the disease should be led by patients. Partnerships with the main experts should be established in a horizontal and direct way, without restrictions, in order to discover treatments, provide a proper support to patients and, ultimately, make the disease history. The efforts made to change the life of a single patient living with a disease that goes beyond invisibility is always worth. Because every patient is a real person. And it is our responsibility as advocates to make visible the invisible.

[1] American With Disabilities: 2002, Household Economic Studies - Current Population Reports, Erika Steinmetz, U.S. Department of Commerce, Economics and Statistics. U.S. Census Bureau https://www.census.gov/prod/2006pubs/p70-107.pdf
[2] About Rare Diseases -  European Organisation for Rare Diseases (EURORDIS) http://www.eurordis.org/about-rare-diseases
[3] Drugs that are not developed by the pharmaceutical industry for economic reasons but which respond to public health need. – Definition from Orphanet