PNH Ukraine

Our organization unites people affected by an ultra-rare disease called PNH in Ukraine. Like many rare diseases, PNH does not have any treatment options available in Ukraine while there exists a safe and effective treatment in the world - one of the most expensive drugs ever existed called Soliris. The treatment is life-long and expensive; the government is understandably reluctant to cover it for its ill citizens, which is a common occurrence in LMICs; donors will not support it either; biopharmaceutical companies ignore Ukrainian market for its alleged small paying potential and risks; Ukraine is off the radars for PNH clinical trials as well. These people, mostly adults in their 20s and 30s, parents of young children, are condemned to die in their current situation. We fight. And we need help and visibility that the Elevate Prize promises.

We need a strong media campaign + a high impact media product to increase leverage in our hard negotiations with the pharma industry and government(s) for access to treatment for rare diseases patients in LMICs.

Some of our stories are good for Netflix series, seriously. Why not publicize them for a good cause?

I am a Ukrainian living with an ultra-rare disease PNH. Some years ago, I left Ukraine and managed to obtain life-saving treatment in Switzerland. Now I live a normal life while my fellow patients who stayed behind are dying back home. I want to help them to regain their future by creating legitimate options to access treatment for them in Ukraine.

We set up an organization of patients and doctors and collaborate with other patients' advocacy groups in Ukraine and globally to help, negotiate with and put pressure on the governments and biopharmaceutical industry to make treatments of rare diseases equally available in LMICs and HICs. The problem that we aim to address here is lack of access to adequate healthcare for people living with rare diseases in LMICs. We want to change this by:  

1) sharply increasing the visibility of the group and the problem; 

2) exposing the existing system failure that fails these people as equal human beings by depriving them of life-saving treatments for economic and corporate policy reasons;

3) creating public pressure on the pharma/biotech and the government to take on responsibility for saving these peoples' lives, now,

and becoming equal partners for them.

Our specific desired outcome is obtaining treatment for 23 PNH patients in Ukraine. 

Our overall goal is to raise visibility of the population living with rare diseases in LMICs and raising the patients to be equal partners in systemic negotiation with the pharmaceutical industry and national healthcare systems. Our strategy includes partnership, technical collaboration with both systemic stakeholders but also creating a blockbuster visibility token to show the world the fate of people with rare diseases (making around 5% of all population cumulatively) living in LMICs and reveal these people in their humanity, beauty and capacity and the way they are being "left behind". To give faces to numbers and notoriety to the faces.

It can be a film (documentary or fiction), book, blog or any other info product to be used in media and international fora such as WHA and GHF or else + workshops to present it + channels of media distribution. 

By doing so, we hope to change to our favor the power dynamic in the negotiations that our group and our partner groups lead with the pharmaceutical industry and governments to incite them to address the problem immediately and sustainably. 

In our work we use the approaches that have become classical for patients' populations - domestic and international advocacy, lobbying budget allocations with the governments to procure existing treatments, negotiating inclusion of severely ill among us in clinical trials or compassionate access programs for experimental compounds, etc. But the progress is less than modest on this path, stalled by the reluctance of the biopharmaceutical companies to provide access to any PNH products in Ukraine, approved or experimental, and lack of will and funds to procure the expensive treatment on the part of the government. Ruptured ties with the Russian market that is still seen in the Western business world as focal for Eastern Europe do not help either. 

The only effective mean that I see to change the power dynamic here is publicity. Media attention. If we cannot act up like early HIV activists did, we can and want to make a good film about us showing the existentially dire situation, in which rare disease patients in LMICs find themselves, and distribute it widely, raise the RD to the level of a lasting newsmaker and disrupt the BAU silence and low priority of RD in the global health policy landscape.

We are a very new organization and do not have all the necessary infrastructure and organizational capacity yet. Yet, we managed to:

- consolidate individual patients from all corners of Ukraine into a group and establish a mutual support network;

- contact all relevant biopharmaceutical companies and international players to assert our needs, expectations and offer our partnership and help;

- establish partnerships with other patients groups and collaborative contacts with the responsible agencies in the Ministry of Health of Ukraine;

- gain access to some international fora like Geneva Health Foum;

- see that the above is not enough to create change.

Not we need to raise the level of our activism to make ourselves seen, heard and funded. We hope to be able to set ourselves on this path with the help of Elevate Prize.

Another area of our interest is to look for or create alternative (other than the state) sources of funding for directly financing the procurement of drugs for our patients, first as pilot, then as practice if successful. 

These create a change to destigmatize and positively affect the situation of all people requiring (and not having) expensive healthcare in LMICs.