Durhane Wong-Rieger, Rare Diseases International: Patients should be the centre of attention

As a representative and advocate for patients worldwide through organisations like Rare Diseases International, what are the key objectives of patient advocates and experts?

I am pleased you have referred to both the patient ombudsmen and patient experts, as we are seeing a significant shift in attitudes towards patients. Ombudsmen are working to persuade decision-makers to implement changes that benefit patients. As advocates, we represent patients, provide expertise on their issues, and offer support. We operate within the system, assisting decision-makers in driving meaningful change.

Patients are increasingly becoming knowledgeable about their finances, medications, and healthcare systems. Unfortunately, they are also well-acquainted with the financial constraints of these services. Our goal is to play a more substantial role within the system. By doing so, we can help drive changes that will benefit not only the healthcare system but also the economy and the environment.

We must go beyond merely advocating for patients; we should also act as experts and partners who learn from the patients’ experiences. Our role involves guiding decision-makers on how to implement sustainable changes that benefit everyone.

In the traditional approach to treatment, patients are often seen as passive recipients rather than active participants. Why is it crucial to elevate their role in the process? Who should we involve at various stages of the decision-making process: during treatment, prescribing, or even reimbursement?

These are indeed challenging questions, but the answer is that patient involvement should occur at all stages to realize the benefits. Consider a typical doctor’s visit: As a patient, I receive recommendations from someone who knows me only superficially, while I have a deep understanding of my own condition. Consequently, some recommendations may be irrelevant or ineffective, especially if similar approaches have failed before. Without allowing patients to actively participate in decision-making, doctors might suggest ineffective treatments, leading to wasted resources. By treating patients as equal partners, doctors can gain valuable insights that enhance the effectiveness of the treatment.

You mentioned reimbursement. In healthcare, it is crucial for patients to clearly articulate their needs, but this often relies on information that may not always align with the best decisions for the healthcare system. For instance, a particular medication might be too costly, and with limited resources, we must decide how to allocate the funds effectively. If patients are not considered as active participants in their care, they may repeatedly insist on certain treatments without considering their overall impact. Only by treating patients as active partners can we determine where to begin, identify viable solutions, and choose the most effective methods based on both their needs and the system’s constraints.

Patients have the potential to contribute significantly, as evidenced by successful examples in Poland. When I request resources from authorities and hear that they are unaffordable, I counter by saying: ‘Yes, we can afford it. We simply need to stop wasting money on ineffective solutions that patients do not accept’.

Many tests are conducted, while we should do just one test instead. I can pinpoint exactly where money is being wasted and how it could be redirected to more impactful uses. The authorities should consult with us when making decisions. By doing so, they can better support not only individual patients but also entire patient groups, and hence make more effective use of the available resources. We need to influence policymakers and the legislative process. For example, I was approached by a patient group that went to court seeking access to treatment. This approach is inefficient, resulting in litigation costs, legal fees, and prolonged delays for services that are already available. These funds could be better utilized elsewhere.

Rare diseases present distinct challenges. What recommendations would you offer to authorities in Poland, and more broadly in our region, to enhance patient care and to ensure equitable access to optimal treatments?

I should clarify that I am not an expert on the Polish healthcare system. Our operations span diverse countries worldwide, including both impoverished and affluent nations. In my view, Poland is in a particularly favourable position, with one of the strongest healthcare systems in Central and Eastern Europe. The country’s health policy and its capacity to translate research into tangible benefits for patients are commendably advanced.

Poland’s population is nearly comparable to that of Canada, my home country. Given the relatively smaller number of patients, it can be challenging to accurately identify every rare disease. Thus, international collaboration is essential in the realm of rare diseases. Poland has the potential to emerge as a leader in Central and Eastern Europe by advancing clinical research, fostering partnerships with research and development companies, and offering a healthcare system that could benefit patients from neighbouring countries.

Therefore, I believe Poland should significantly invest in advanced technologies to deliver the highest standard of care for patients. While the country has already made substantial investments in fields such as artificial intelligence, genetics, and genomics, it is crucial to consider how to extend the availability of these technologies throughout the region.

PARTNER: ISW