Let’s be honest, at the end of the day, what we want is a drug that works for our children.
We are honored to share this guest post from Melissa Hogan, founder of Saving Case. Melissa started the organization after her third son, Case, was diagnosed with the rare disease Hunter Syndrome or MPS II. She is also the author of Calmer: Medical Events with Cognitively Impaired Children.
During my recent time at the World Orphan Drug Congress, I had the pleasure of a nice dinner and conversation with some operations folks from a larger pharmaceutical company, otherwise affectionately known in our crowd as “big pharma.”
Interestingly enough, while we talk about big pharma (I’m going to keep using that term even though it is not precisely defined) entering the rare disease space for MPS/ML, some of the companies more established in our space are definitely not “small” by pharma standards – Shire and Genzyme/Sanofi.
But, sometimes it’s the case that you can slander your family, but you’re not going to let anyone else do it, you know what I mean? Our patient populations have grown up with these companies in many respects, from when they were small and testing that one compound that gave us hope.
The conversations spurred some thought in me about what us MPS/ML (and maybe most ultra rare disease) families want from (big) pharma (or really, from pharma, period).
Let’s be honest, at the end of the day, what we want is a drug that works for our children. Period. If the science isn’t there, then it’s not a two way street in any event.
But if the science is there, if the pharma-patient relationship will likely be there in the future, what do we want now? What will we want then? I’ve come to a simple conclusion, and it involves four things.
We want the company to be honest with us, at least to the extent that it can in the face of pharma and public company communication guidelines. If there is a problem, we are big enough to handle it as well. We want to know the same information you are telling your investors. If it can be public, and it involves research that is applicable to our space, we’d like to be among the first to know, not stumble upon it accidentally in an investor call transcript. And if you can’t tell us anything, please tell us even that, kindly.
We want to know, especially from big pharma, that some might see as interlopers seeking to bleed the last of high profits from a new area now that their more common drugs are going generic, that entry into this area is not a fleeting thought of profits. We want to know that if the research is more difficult, if the science takes longer, if the competition gets heavier, that you are committed. We want to see understanding that commitment is not just offering us a product, it is helping to make our children’s lives better, a product being just part of that effort. Because it’s not commitment to us, it is commitment to our children.
We want to know that you care about our children, that you care what happens to them as people without respect to whether you are able to sell them a product or not. We are protective of them and we smell pretension a mile away and who would we be as parents to expose them to that? They are incredibly special people who have changed our lives and the lives of many around us, so we want to see you open up and allow them to change yours. You will be incredibly blessed by it.
This is especially applicable for those new companies that are coming in to hopefully serve new groups of patients in the rare disease space. I know sometimes in the realm of big/small pharma relationships, it seems as if big pharma is bringing huge capabilities to the table and it can be felt as if what “we” (big pharma) are doing to help “you” (the little guy). But we as parents feel as if we are doing a pretty bang up job caring for our children. Does a drug offer potential that is not already there? Absolutely. But it is just one piece of huge puzzle of what we do day-to-day in trying to make our children’s lives longer and better. We see it as a partnership, and expect to be treated as an equal partner in this process of trying to save the lives of our children. It’s not even necessarily “patient-centered” relationships as that term is now being bantered about so much, it is partnered relationships, with parents, physicians, payors, regulators, and pharma as equal partners. We’ll play our part, and we’ll even give you guidance as to how to play yours.
So I would welcome any responses as to whether I’m totally off base, whether we should expect more or something different, or whether I’m just playing the role of selfish parent who is simply asking too much. But respectfully, I ask these things on behalf of my son, because he cannot ask them for himself.
- 26 April 2012 at 8:04pm
- Beth Burnside
Insightful guest post about an ...