As I’ve been speaking with various members of the HPS community, I’ve been getting a lot of the same questions. After we all get over the initial shock about the drug trial, just about everyone asks – what now?
It’s the best question anyone could ask. We are working through this, shedding our tears, and then standing back up, brushing ourselves off, and heading back to the battle. We will prevail.
So, what’s the plan?
As the NIH has a chance to do just what we’re doing – process this development and regroup – more specifics about what we need to do will become clearer. Some actions, however, are obvious to us.
Now, more than ever before, we need to be strong. Dr. Gahl and the researchers have told us they aren’t giving up on us, but just as this was a blow to us, it was also a blow to them. They do care about us and they are just as heart-broken as we are.
Because we’ve had difficulties with recruitment for this study, it’s up to us to prove to our researchers that while they regroup, we’re getting ready for the future. We have to show them that we as a patient community remain united and devoted to research. We haven’t fallen apart. We understand how science works. We will remain organized and strong in the best of times and the worst of times.
Some of you have asked whether we need to continue the Hundred People Search. The answer is a resounding YES! More than ever we need to keep up the momentum of the Hundred People Search.
First off, the Hundred People Search was never just about finding patients that might be interested in the protocol. It’s about so much more than that! It’s so important for people with HPS to know that they are affected. It allows them to advocate for themselves, anticipate potential future medical needs, plan accordingly, and take maximum advantage of preventative measures to stay healthy. Too often we at the Network have seen the fallout from cases where people didn’t know they had HPS, and as a result, had very bad outcomes.
Secondly, we couldn’t blame our researchers if they are now a bit gun shy about future drug trials. They are understandably going to ask whether we can really ever have enough people to participate. It’s not easy to find participants for studies like the Pirfenidone trial. I must confess, when we started I never imagined it would be so hard. People have to meet very specific medical criteria, can’t have any exempting factors – and they have to be willing and able to comply with what the trial requires. That isn’t so easy.
So what do we do? We need to even go beyond the Hundred People Search. We need to double the number of HPS’ers on our database.
Lastly, one day, when we have more to offer in the way of treatments, or even a cure, we don’t want to leave anyone behind. If we don’t know who they are, we’ll never be able to share information with them.
So, for the immediate future, the plan is outreach, outreach, outreach.
If you know anyone with HPS that isn’t registered with the Network, such as a cousin or a sibling, please encourage them to call the New York office, register, and be counted. Even if they never want to participate in any other way, we all need to stand up and be counted to let the medical world know we’re out here.
It’s the best question anyone could ask. We are working through this, shedding our tears, and then standing back up, brushing ourselves off, and heading back to the battle. We will prevail.
So, what’s the plan?
As the NIH has a chance to do just what we’re doing – process this development and regroup – more specifics about what we need to do will become clearer. Some actions, however, are obvious to us.
Now, more than ever before, we need to be strong. Dr. Gahl and the researchers have told us they aren’t giving up on us, but just as this was a blow to us, it was also a blow to them. They do care about us and they are just as heart-broken as we are.
Because we’ve had difficulties with recruitment for this study, it’s up to us to prove to our researchers that while they regroup, we’re getting ready for the future. We have to show them that we as a patient community remain united and devoted to research. We haven’t fallen apart. We understand how science works. We will remain organized and strong in the best of times and the worst of times.
Some of you have asked whether we need to continue the Hundred People Search. The answer is a resounding YES! More than ever we need to keep up the momentum of the Hundred People Search.
First off, the Hundred People Search was never just about finding patients that might be interested in the protocol. It’s about so much more than that! It’s so important for people with HPS to know that they are affected. It allows them to advocate for themselves, anticipate potential future medical needs, plan accordingly, and take maximum advantage of preventative measures to stay healthy. Too often we at the Network have seen the fallout from cases where people didn’t know they had HPS, and as a result, had very bad outcomes.
Secondly, we couldn’t blame our researchers if they are now a bit gun shy about future drug trials. They are understandably going to ask whether we can really ever have enough people to participate. It’s not easy to find participants for studies like the Pirfenidone trial. I must confess, when we started I never imagined it would be so hard. People have to meet very specific medical criteria, can’t have any exempting factors – and they have to be willing and able to comply with what the trial requires. That isn’t so easy.
So what do we do? We need to even go beyond the Hundred People Search. We need to double the number of HPS’ers on our database.
Lastly, one day, when we have more to offer in the way of treatments, or even a cure, we don’t want to leave anyone behind. If we don’t know who they are, we’ll never be able to share information with them.
So, for the immediate future, the plan is outreach, outreach, outreach.
If you know anyone with HPS that isn’t registered with the Network, such as a cousin or a sibling, please encourage them to call the New York office, register, and be counted. Even if they never want to participate in any other way, we all need to stand up and be counted to let the medical world know we’re out here.
Comments