The Rare Diseases Patient — At the Center of it All

The Rare Diseases Patient — At the Center of it All

There are 7000 rare diseases affecting about 350 million patients around the world. There are only about 400 treatments for these diseases.

The pharmaceutical industry is aware that the current rate of failure (attrition rate)  in the search for new treatments for rare and non rare diseases is unsustainable. At the same time, governments, patients, health systems, and others who pay for new treatments want drugs that make a real difference to the lives of those suffering from disease. Deciding what that difference (or benefit) looks like is not easy. A patient may define benefit differently to a pharmaceutical company, or even to a regulatory authority. In the effort to design clinical trials and drug development programs that can better demonstrate meaningful benefit, patients are being invited “to the party”. After all, who better to understand a disease than the patient who is suffering from it.

How does this impact patients with rare diseases? With the Billions of Dollars that are being spent on finding new potential treatments for rare diseases, there is  naturally a desire on the part of pharmaceutical companies, patient advocates, government agencies, and of course patients, to expedite access to effective treatments.  It is hoped that one way to make the process more effective is  to make the process more patient- centric.  What does this mean in practice for patients with rare diseases? It will mean that some patients will be consulted during the protocol design phase. It should mean that patient advocacy groups are consulted about how the patients experience their disease and what aspects of clinical trials may be unnecessary in relation to the benefits that patients need.

It appears that patients are willing to take on this role of advisor to the pharmaceutical industry, but they do need to be equipped to fulfill this role. How will patients access useful information about the drug development process? The internet is one way, but it is difficult to navigate the vast amount of information available without guidance from a trusted source.

The Rare Diseases Patients First! organization aims to be a trusted advisor to patients with rare diseases, and parents of children with rare diseases. A series of Webinars is being provided free of charge to anyone with a rare disease that wants to take part. The next one on the 23rd of April at 8 am PST will feature Dr. John Whyte from the FDA. Dr. John Whyte has worked at the Discovery Channel and understands how to engage people on the subjects of their health. He is in charge of patient advocacy and engagement at the FDA.

This is a very important Webinar because Dr. Whyte will be presenting on the frameworks that are being put in place for patient involvement in the drug development process.

Deciding who speaks for the patient is not as easy as it sounds. Patient advocacy organizations are going to take on an increasingly important role, but it is also important to ensure that we engage with real patients, who may not have time to be involved with patient advocacy organizations. This is why Rare Diseases Patients First! invites patients with rare diseases, and parents of children with rare diseases to the Webinars.


The Rare Diseases Patient at the Center of it All is a Webinar which will take place on the 23rd of April at 8 am PST/11 am EST/4 pm GMT by WEBEX. To receive the login details, email Dr. Speid as soon as possible  at lspeid@sndtm.com with RDPF! in the subject line.


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