Leadership Exchange: Small Size, Big Prize

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Leadership Exchange: Small Size, Big Prize
Leadership Exchange: Small Size, Big Prize

An esteemed panel of marketers from small biotech companies discusses the unique challenges and opportunities of bringing novel products to market. James Chase moderates the session

James Chase (MM&M): Each of your companies is built on a foundation of scientific discovery. What are the challenges of making the transition to a more commercial mindset?

Stefanie Nacar (Infinity Pharmaceuticals): When you're at a smaller company I think there is always the challenge to change the culture and get people comfortable with commercialization. Sometimes, to the scientists, it's like they're selling their drug out when they see commercial people come in. I think the hesitancy comes from a lack of awareness or education about why these certain functions are important. It's not just about trying to show activity in your compound, but how it could be clinically relevant once your drug is approved. You need to educate and share the story about why it matters.

Unfortunately, I think a lot of the smaller companies probably learn that the hard way, where they resist and resist, and then all of a sudden they come up with a challenging environment where it's either competitive and the data's not getting as much of a foothold as they would think. Or we all think that the results are fantastic but yet it's not being covered in the media. Or it's not getting the right placement at scientific congresses. And a lot of it has to do with building that foundation and that awareness and building that story to frame the data.

As a commercial person at a smaller organization that hasn't been through it before, I think you have to be of a certain personality where you have to be comfortable knocking on people's doors and saying, “This is where I could provide value. Let me be a part of this meeting. This is how this could be helpful, and this is why.”

Lori Horvat (Ariad Pharmaceuticals): I think what's interesting is a situation where scientists recognize the need to involve commercial but they don't recognize when is the right time. I remember last year our head of biology put together a meeting. He said, “We have this exciting preclinical asset that we've invested all these resources in and just want to make sure that you're bought in.” And after the course of the discussion, at the end of the day we killed it, because from a commercial perspective it really wasn't worth the investment and the resources.

He was crushed. I mean, the fact that he pulled us into a meeting to get our buy-in was good, but it was a little bit too late. That conversation probably should have occurred earlier. So I do think part of the battle is recognition of the value of commercial, but then the other part of the battle is involving us at the right time.

Paul Merrigan (Aegerion Pharmaceuticals): My situation's unique in that we were a commercial organization almost from the beginning. Our CEO is a sales and marketing guy, and came over and really bought an asset that was in a Phase III. So we have no research, but we have development. So instead of hearing that the product will sell itself, it was always the mentality that we all have to be in this together.

And so when you're building small, functions didn't exist yet. So everyone was always at the table all along the way to learn about what's coming down the line in commercialization and why we had to do certain things and the certain functions that you had mentioned.

Stefanie Nacar (Infinity Pharmaceuticals): Do you think having a CEO from a sales and marketing perspective changed the shift of the culture, or how that was embraced, or how that steered your strategy, or the support that you had?

Paul Merrigan (Aegerion Pharmaceuticals):

There's quite an advantage to having a scientist start a company and run it. But there's tremendous advantage of having a commercialization person do the same. So at the beginning, some of the things we learned were when you hire people there is specialization within specialization, for instance if you're a regulatory group whose primary goal and success and achievement has been getting approval but has no experience in the post-marketing world and has to learn promotional review, then that causes a learning issue internally.

James Chase (MM&M): Carolyn, you've worked with a number of smaller companies, both science-led and commercially minded. What are your observations?

Carolyn Morgan (LehmanMillet): Both have their pros and their cons. When we work with companies who have been started more from a scientific standpoint we find ourselves having to work a lot harder to help the organization see the benefits of commercial. They're very analytically driven, so they want to see results before they're willing to put in the investment. So we spend a lot of our time helping the commercial folks inside these organizations justify why you have to establish yourself as a brand.

On the flip side, commercially-charged organizations can act almost too risky. We had one that was going to go down with just a brochure and then release the rest of their materials at risk. We admire that and it's kind of cool… but we don't want to be the agency that got the letter.

James Chase (MM&M): Presumably, smaller companies are forced to build functions and develop processes on-the-fly. Has that been your experience, and can you share any examples?

Lori Horvat (Ariad Pharmaceuticals): We made a strategic decision to build an infrastructure in Europe. There were individuals that were hired to build from scratch an entire European launch plan in just six months, which was pretty miraculous. And here we are, on the fly, a year and a half into it, and we've never really taken the time, gone off site, and defined what is the role of global marketing versus the role of EU sales and marketing, versus what's the role of each individual country marketer? And that's just marketing. I could say the same for medical affairs, global health economics. I mean, I could go down the laundry list. We still haven't defined some of the processes that I think ultimately will be important for us to be successful in the long run. That's a challenge.

Stefanie Nacar (Infinity Pharmaceuticals): I think that it's a challenge, too. And a lot of times letting the different functions fall and thinking about what experience do I have, what experience do my colleague and I have, and it doesn't matter what function or what title you're in; it's about how can we best move this forward? You're forced to be comfortable with that in a smaller organization versus a larger organization, where from my experience, you are in your expertise, and your function, and your silo, and everyone has their responsibility and their area. So there is a lot of building processes on the fly.

Lori Horvat (Ariad Pharmaceuticals): I came from Bristol-Myers Squibb, from big pharma, and now I actually appreciate some of the processes and structures that big pharma has in place. I love being in a biotech and it's great that, you know, everybody rolls up their sleeves and gets it done. It's a fun environment to work in, but I feel like it has to be the right balance. So I think there is some beauty and some lessons learned for those who have been in a big company to kind of bring a little bit of that rigor, processes and structures.

Paul Merrigan (Aegerion Pharmaceuticals): Flying the plane and building it too is kind of exciting and scary, and it's all at the same time. You have comfort, in that, okay, it's still flying. But you're uncomfortable that “I don't know if I have wheels to land,” or “am I going up?”, or whatever. So I think that when we started it was like, boy, we need some processes. So wait, do we even have an SOP process to create an SOP? I mean, we're starting from scratch. And everything really needs a process ultimately at some point in time, or you don't get consistency, you don't have compliance, you can't expand, you can't transfer knowledge.

And, combined with every other commercial piece that you do, whatever playbook you put together, every marketing plan… there isn't a guide that says this is the right way to do it. So from the beginning, every system, whether it's sales, operations, distribution, each function has to create its own process on the fly. And it is hard sometimes to be able to come in and say, “Well, we're lucky we have phones, okay?” as opposed to “Do the phones work?” You're at that level in a startup company. But we get to shape it ourselves, and that's the fun part about it.  

James Chase (MM&M): So, the scenarios that each of you has just described… would you say they've been detrimental to your progress so far?

Lori Horvat (Ariad Pharmaceuticals): No, not at all. I think it's really when you're in launch mode. Once again, we're all focused on one prize, and so in the short term, get it done, launch, drive sales. But we're going to have to start to refine, make adjustments. And I think it's more in the longer term where I think we are going to have to have processes to be successful. So I don't think it was a detriment in the last year and a half, but moving forward we've got to lengthen that runway, we've got to be efficient, we've got to make the right choices globally.

Paul Merrigan (Aegerion Pharmaceuticals): My experience and training of a large global company was you would have a global strategic plan and you would roll it out. And it would give guidance on what were your critical success factors, your objectives, what each country should be considering for being launch-ready, etc.

And so when I joined Aegerion, that's what I started doing, because that's what I knew. And then I realized, well, who am I sending it to? Myself? No, I mean, that was like the joke. And then time started running out. Every year is different from the year before, from lessons learned.

Carolyn Morgan (LehmanMillet): I think that when you're thinking about a global organization, we have these conversations often with our clients… do you start in the US and then move outward, or do you start from a global structure? And it's a little bit of a crapshoot because it really does come down to investment of resources and really how you can allocate. And if you're not going to be going truly global at first, then to really invest that in that kind of scaling is a challenge for a lot of companies.

But I'm always a big believer in making sure that we're at least thinking that way and making sure that we've got a little bit of that long-term vision in play, or else we're going to end up with a plan that isn't really executable if it's only born from one location. It's rare that we find ourselves not working from a global scale in one capacity or another.

Lori Horvat (Ariad Pharmaceuticals): We're talking right now about three small Cambridge-based biotechs who want to go it alone and global… we all want the big prize of being global entities. And that's a very different philosophical approach from 10 years ago where all small biotechs were partnering with big pharma to commercialize outside the US. It is fascinating.

Stefanie Nacar (Infinity Pharmaceuticals): I think innately it's a certain type of person, it's the underdog… small companies want to be able to do it and show that there is tremendous value. But when you look at what now needs to be done to get these compounds to patients, it's such a disheartening story to see smaller companies that have a very good compound to be able to treat patients but because of the nature of the business they're just not able to get that compound, that treatment to patients.

Where is the balance between the patients and between getting a product, finding value for your investors as a public company. And how do you think about letting go of some of that control for the greater good to get the drug to patients faster?

James Chase (MM&M): Does the collaboration that's required to get the product to patients always have to involve concessions to a big pharma company?

Stefanie Nacar (Infinity Pharmaceuticals): There's a lot of things that come into play, and I think every company's philosophy might be different. I mean, you could partner with a very large pharmaceutical company, and they just want to niche your drug to a certain indication because they might have other pipeline products going on that might end up competing. There are always other things in play. You just need to find the right partner that could collaborate well across the different functions and see the vision in a similar way.

Paul Merrigan (Aegerion Pharmaceuticals): It could be the opposite, too. I mean, particularly in a highly specialized area, a well-defined rare disease, if you provide this to a non-patient-centric, large pharmaceutical company, they won't know what to do with it, and it won't get to as many patients. And so this is a trade-off. It goes on both ends.

James Chase (MM&M): We've talked a lot about the issues, the challenges, the roadblocks of being small, but there is another side to it, that you guys are nimble and cutting-edge. Do you see opportunities in being small?

Lori Horvat (Ariad Pharmaceuticals): Yes, it's a huge advantage, we can react quicker. I mean, the amount of information we have at our fingertips and our ability to turn on a dime our strategy and our messaging is clearly a competitive advantage. Because in a large company there's just a lot more bureaucracy, processes take a lot longer.

If you want to bring something through a medical legal review process you might need a one-month lead time, whereas I can bring everybody together and get them in a room tomorrow, if it's urgent.

Stefanie Nacar (Infinity Pharmaceuticals): I completely agree. I think that my experience selling for a large pharmaceutical company and then also launching, you're almost slotting your compound into the sales force that you have and figuring out what physicians you're going to call on, and you have your approach. But we really have the opportunity to not say, “What do our customers need? How do they want to learn about this? How do they want to engage?” We could be creative.

You can think of it more as a customer engagement, not that you're pushing information out to get a prescription written. It's, how do patients need to feel about the drug in order to accept the treatment? How do physicians need to accept the information to feel comfortable and confident that this patient type is the right patient? You're looking at it from a completely different perspective.

Paul Merrigan (Aegerion Pharmaceuticals): You get in there for one quarter and you say, okay, what did we get right and what did we get wrong, and you end up getting more things wrong. But the things you got wrong, you can pivot from. You can incrementally grow within a quarter. If you go back and ask, “Did we follow the plan?” you would think we didn't do very well. But if you looked at it and said, “You didn't really follow your plan because you changed it as you learned, and because of that you've got to be successful,” and you were, then that was the right thing to do. In the end we have to change the world. We have to change the thinking and the perceptions of the product or the disease.

Lori Horvat (Ariad Pharmaceuticals): It's interesting because we all are one-product companies as well, right? So our people are 100% dedicated every single day to the one product. You're not competing with a sales representative that has three products in their bag.

And it's a lot easier to control the message when you have a lot fewer people. In an ideal world, all marketers would love to believe that everybody's on message, but that's just not the case. We know that ultimately people are going to adjust conversations to whichever direction that physician takes them, which is appropriate. But our ability to really keep people focused and driving toward one strategy and one goal gives us an advantage, because we're competing against Pfizer, Novartis and Bristol-Myers Squibb… all of these sales representatives have multiple products in their bag.

James Chase (MM&M): Are you experiencing the same rep access issues as the rest of the industry?

Paul Merrigan (Aegerion Pharmaceuticals): Yes and no. I would say that it depends on what specialty. It is challenging. But when you get engaged in a rare disease, that's highly specialized, when it's mostly not about this product, it's about a patient in need and maybe this product can help them, it's engaging. And once you do that, you can get hours with people. But getting that foot in the door and being differentiated as a company—how you came to be, what you are all about—is a challenge. A lot of what you do at first is selling your company name.

We have the advantage of being tied to an academic specialist, so there are things you learn that people will respond to, because the academic people want the other academic people to win too, and that this is a partnership approach of doing this. So we figured out what are the buzzwords to get us in the door and differentiate us from other companies. But it's a big challenge.

Stefanie Nacar (Infinity Pharmaceuticals): Which brings up a really interesting conundrum; smaller companies have to be very selective about where they're going to invest. So where and how do you now get that information and that message to these customers in that way? What types of mediums are you going to use? How do you shift your focus and your tactics differently because of that? And then also within the budgetary constraints of really making everything that you do as impactful as possible, rather than large pharma, large budget, just going through your playbook of activities.

Paul Merrigan (Aegerion Pharmaceuticals): We try to take a different approach. If you can't get the physician, get the nurse, get anybody you can and say, “I'm here to help find if there are patients falling through the cracks. Do you have patients that look like this, that come like this? Anybody you know who's like 30 years of age and under who's had a heart attack? Do you know why they had a heart attack? Is it a genetic condition?”

And they might be like, “Well yeah, I remember this guy Jim.” So now I want to talk to your physician about Jim, because there could be something available for Jim now. So you got to come at it a different way. Because if we come in, I've got this product, so they don't know the disease, they don't know the product name, and they don't see reps, you're done, right at the beginning. So you got to be creative. And peer-to-peer is probably where you end up going, to get other people to kind of get the word out for you.

Stefanie Nacar (Infinity Pharmaceuticals): Carolyn, with all of the different companies that you work with, have you seen a shift in what companies are doing in different ways that they would be changing the focus, what might be ordinary typical things that they do but now because of this issue they're thinking about new creative ways of actually educating?

Carolyn Morgan (LehmanMillet): We are talking much more about non-manpower activities now, more than we ever have in the past in the US. So how do we reach those physicians when they're sitting at their desk? Or how do we approach them and gain access that way? Whereas before our strategies and our objectives and our marketing plans would always be the typical ones that you would find, now our non-manpower initiatives are always at the top of that list because that has just become such a challenge.

And making use of digital communications really has become the mainstay. When you were asking earlier, what is the difference between big pharma and small pharma, in terms of the nimbleness and the cool factor? For us what's great is that we're closer to the decision-makers, right? So a lot of times some of our bigger pharma clients still aren't doing things that these guys are, which is mind-boggling to me. Like they're not doing things, they're not back-end integrating, they're not thinking about how their rep using things.

It's kind of fascinating to me how, because of that slowness and that bureaucracy, that they can't get things going faster, whereas we're able to say, “Hey look, here's the case study,” again going back to the proof. “But hey, this is a really great tool we can use, what do you think about this?” These kinds of clients, these kinds of companies, because they have to be more efficient with less, they're more interested to try something, especially if they can see a return on investment for it. So we get to do more innovative things with companies like these than we can with the bigger companies.

James Chase (MM&M): We've talked about a lot of different stakeholders. Do you find you have to prioritize the stakeholders in terms of resources and focus? And what's your biggest priority … physicians, patients, payers, shareholders? Who are you answering to first?

Lori Horvat (Ariad Pharmaceuticals): I think we always prioritize our resources, and healthcare providers and physicians for us are our number one focus. I think patients have become our larger focus. When I think about Ariad and the past six years, and go back to our drug with Merck for soft-tissue sarcoma, I think our investment around patients and advocacy groups was probably much smaller than it is today.

So I think patients, and specifically working with advocacy groups, social media, really more in the disease education, getting patients to be champions and to ask their physicians the right questions—to me, that's where I've seen probably the biggest change in terms of how I've invested my resources at Ariad over the last six years. I think payers are obviously still extremely important customers.

I think for us this year, because our label's much more niche, we have fewer barriers because we're not really competing against another drug for placement on formulary per se, because of the label that we have. But I think for me the priority is physicians. But where I see the trend or the change in the last six years is investing more in patients really on the disease education side, not necessarily product.

James Chase (MM&M): Do you think, again going back to the nimbleness, the size factor, do you think you're in a better position to deploy channels such as social media and to execute more effectively on those kinds of things?

Lori Horvat (Ariad Pharmaceuticals): Absolutely. I mean, social media's a prime example because we have a social media initiative with patients. Our competitors, it's not really a two-way communication. They have what they claim are social media sites, but there's no two-way dialogue. It's not really social media. So I think when I compared our three largest competitors we're really the only one that had a true social media campaign and a true two-way dialogue with patients. It's all disease-focused and not product-specific.

But I think why we feel it's important and why we think that we get a return on that investment is, we're educating patients on the right questions to ask their physicians. Because we know in the world of CML patients don't always get the appropriate testing to make sure that they're responding to their drug, that they're not responding to the particular mutations. So we're arming them with information about the disease to really enhance the dialogue that they have with the physician. So for us that is a return, because it's creating questions that maybe wouldn't come up in a normal dialogue.

Stefanie Nacar (Infinity Pharmaceuticals): I think that totally makes sense, and I think some of it too obviously depends on where you are on the developmental lifecycle and who you need to educate first. But I think one of the interesting things that I've begun to see working on different indications, is that in some of these diseases that are more chronic, or where patients are diagnosed and living longer with their disease, there's a different level of education or involvement that their family members and the patients themselves have.

And they tend to be a little bit more educated because they've had the time to think about the different types of treatments, what their options are if they want to be empowered in that way, versus maybe some of these other very challenging diseases where they get their diagnosis and, unfortunately, the outcome's just not very good. So I think again knowing who your patient is and who your customer is, I think it helps guide you in when to engage in these types of initiatives and to what extent.

James Chase (MM&M): Do you think your patient populations want to engage with you, and do you think it's your role to engage with them?

Stefanie Nacar (Infinity Pharmaceuticals): You know, I do think that we're part of the healthcare organization. We are investigating certain compounds and certain treatments, and our scientists I do believe understand a great deal about our particular pathway or our particular drug, more so than anybody else would because they're living it day in and day out. And I think it is important and a part of our responsibility to be able to make that information available.

I don't always think that the resources that are out there are always encompassing enough. And maybe because we are investing in the market research to understand where those gaps are and what information people are looking for, and I think if you're going to do that you need to be willing to then execute on those projects to fill that gap. And so if there is a gap that's existing, I think it is a part of our responsibility of getting these treatments to patients so that they have that education as well.

James Chase (MM&M):Paul, you recently added patient advocacy to your job title. So what are you guys doing in this area?

Paul Merrigan (Aegerion Pharmaceuticals): Well, again with most rare diseases, people know they have the disease. I don't want to say that in a negative way, but they own the disease. They say, I have CLM, I have Gaucher's disease, I have cystic fibrosis, whatever it is. And they know it, and they become part of a community. In our situation learning, we thought, okay, everybody who has homozygous familial hypercholesterolemia is out there and knows it. And they can all identify, because who couldn't diagnose this? And what we found was that there wasn't any community that exists. Nobody defined themselves as having homozygous FH.

Familial hypercholesterolemia, which is a less severe but one of the most common genetic disorders in the United States, is 90% under-diagnosed … because we might have it, but no one's told us. We just have high cholesterol that runs in the family, and no one knows what that means. But it runs in the family, we know it's inherited. So there was no community, there was no patient community to reach. So you can't do social media with a community that doesn't exist, and it's really hard to.

And if you reach out, no one calls themselves that. So one of the first things we did was try to help establish, along with others in the industry, the multi-sponsored way, and support and encourage some patients to create a community, to create a foundation. And there are also rare disease foundations, there are high cholesterol foundations, there are FH foundations now. So now there's an FH foundation but there's no HOFH, the homozygous foundation. So it's still a challenge for us in that sense to say we can go to that community.

So let me go back to the original question of which stakeholder is the priority… of course, payers were extremely important at the beginning, preparing the waters and everything. But in a rare disease it's all reactive, because one payer might have one patient. You have to go where the patient is, not where the payer is all the time. But the majority of patients are covered by the big people and you have to help that. And that's where medical really is very important from an education standpoint in playing that.

And then our primary focus has been healthcare providers and to really understand which specialty is actually in a patient journey, these patients kind of stop in their referral path, and that's where they are, and why that is. And so that still is important. And when we close now, they ask, the conversation is, “Could you have a patient that has this disease?” And if you do, help them understand what it must be and get them diagnosed. If they then get them diagnosed, then they have the option to consider our therapy.

And then when you close on our therapy, it isn't just, say, start our therapy, go take it. It's, okay, “Here are the services that accompany this therapy, and here's what we need you to do with the patient. Because your patient probably has never been told they have a rare genetic disorder called homozygous… You yourself probably told them they have high cholesterol. They have to realize that they've had this since birth, they've had chronic exposure, and that they need to go on the therapy now, and it's very serious.”

Because one of the challenges we found out was, when the doctor just writes a prescription the patient thinks they just have that general garden variety of high cholesterol. And they take it, they get into some side effects, which we all have, and they stop right away—because no one's helped them realize how severe their disease is, and it's unnecessary. And so then the other end, this patient engagement group is to help on patient education, because you always have to come back and ask your physician for your medical advice, because we can't give medical advice. But they have to know what to ask their physician. And in coming back and really saying, these are the questions you need to be asking. Then on both ends, you're going to do this, the patient will do this, and they come together. And that's where you start seeing, successfully, people going on therapy and staying on therapy.

Stefanie Nacar (Infinity Pharmaceuticals): I think that helps to know who is the company, and who does the company want to be? So when you have these first product launches or a one-product company, so much of how you go about doing things really sets the company brand and what your company wants to stand for, and what do you want to be known for in the industry.

Carolyn Morgan (LehmanMillet): I think it's important, especially in rare diseases, these are such important things. To not make an effort to connect with the patient I think is a real miss, because these patients need these medicines. And I think it's an unfair burden on physicians to assume they should be on their own. And so a lot of times these physicians or their family members are the ones that are taking them. And we should say, “Is there something else that we can be doing, is there something else that we don't know about?”

And oftentimes it's through that passion of having the family member, or you yourself, you find it. And we have to make them, that's why we have to be where they're looking. We have to find opportunities to connect there and then give them the tools to have that conversation with the physician, and hope that the physician has heard of us and isn't going to shut that conversation down. So really it's got to be two-way. Because oftentimes you don't want to be driving patients into a physician's office and have the physician say, “I've never heard of that company” or “I've never heard of that drug”, and so “I would really recommend you go here.” Those are wasted dollars in investment, they're not doing anything. So it really is a combination of doing it on both sides of the fence to make sure you're really getting the best realization of what you're trying to achieve.

James Chase (MM&M): If you could do something differently, what would it be? Lori?

Lori Horvat (Ariad Pharmaceuticals): So hindsight's always 20/20 in the sense that obviously we developed a safety signal. We were very focused out of the gate around efficacy, and at the end of the day I still think that efficacy trumps with cancer. You need to make sure that when you select a cancer treatment that it's going to work. So efficacy trumps. I think though we'd spent very little time early on in the original launch talking about the safety profile, educating people about side effects, strategy to manage side effects to help retain patients on therapy. Because acquisition is only half the battle. The other half of the battle is maintaining a patient on therapy. And one of our early lessons learned in the first launch is, we spent so much time hammering efficacy, efficacy, efficacy that when a patient did get a side effect, physicians nine times out of 10—that's not the exact statistic—but nine times out of 10 they would actually take the patient off of therapy as opposed to just reducing. So that was a real lesson learned. Going into the second launch was making sure that we're really being proactive about the ability to adjust the dose and to help manage side effects.

But also with the second launch, not only does the dose reduction help manage side effects but it also helps to mitigate some of the risk that we potentially saw with the safety signal in ponatinib. So to me that's probably the biggest lesson learned, is not being 100% focused on acquisition but also focused on once you get a patient on therapy how do you retain them on therapy, and how do you educate around safety, adverse events, etc.

Paul Merrigan (Aegerion Pharmaceuticals): That's a really good point. We have a risk evaluation mitigation strategy (REMS) that's required in the US and then outside the US in Europe, a risk management plan. So before you can even talk about efficacy you have to certify people, sign them up in a club, that you've been trained on safety and risk. And I think at the beginning to a sales organization that was hard to deal with. It's like, well wait, usually you hit them with efficacy real hard and you come in with balance safety.

But here we said, “Well wait a second, in the end these are real, these are things that are real, and that people have to realize that they're manageable.” And whether it be dose individualization or monitoring, and these other things are in play here that allow the therapy to be effective. And then also emphasize efficacy, because no one's going to use it if it's not effective. So you're going to get that message. And the challenge for our product was never about the efficacy. We needed to sell and disclose the safety aspect of it.

And that was a training issue. It was, get comfortable with talking about safety first, and it's okay. It's still hard, though, that when someone has a safety issue, instead of reducing the dose they'll take them off the drug or change their diet in our case for certain reasons. That continues to be a challenge, but it's less so. But I'll say because this product was originally not moved forward by Bristol-Myers Squibb because of a safety signal, an issue, so it had a reputation that came along with it. We needed to also overcome that reputation from the day one because somebody's physician or the investigators from Bristol-Myers Squibb, well, this got killed, and so we took a real strong safety approach. But that scared a lot of patients.

When you have a REMS program and a boxed warning, and you go to the internet and you start seeing these things, many patients say, “No, that's too scary for me.” And so that's the challenge that we have to kind of help the physicians make sure that they position us appropriately with the risk-to-benefit ratio with their patients so that they don't get scared off on these things. It's quite a challenge.

Lori Horvat (Ariad Pharmaceuticals): We have a REMS program, and we talk about benefit/risk every single day, so it's the same challenge.

Stefanie Nacar (Infinity Pharmaceuticals): I completely agree with that because I experienced that elsewhere, it's very important. I think probably the biggest thing right now that I would say is the most impactful thing for a company at our stage that has not launched a product yet and doesn't have a commercial infrastructure, is you need to start your launch strategy early and get aligned on what it is you're trying to achieve and then how you're actually going to achieve it.

Because the focus so much within a company at our stage is getting to NDA and making sure that you're able to enroll your trials and get the data that you need, and then get the resources behind filing. But there's so much that needs to be done simultaneously as you're going along, and sometimes you're kind of forced, because of reduced internal resources, to focus on that.

But you can't forget about the other things. And if you wait too long you could do it, but you're going to end up needing to make sacrifices that maybe you would prefer not to, had you had the time to really consider it. So I would say that for us that's probably the biggest, most impactful thing.

James Chase (MM&M): Wise words. Any final comments?

Paul Merrigan (Aegerion Pharmaceuticals): The one thing I would really do differently is that we bought into what's in the literature. It's the prevalence, how this disease presents, and we thought that that's how it looked. And then when we went to the physicians, the specialists who wrote the textbooks, we realized some things were put in the literature 30 years ago, and since there's been no treatments, and it just keeps going, just lives on, and it's like legend... it's not really true, but everybody believes this.

So if we had a better understanding of that mentality going in, and if we didn't go in right away and say, “Oh, all the patients look like this.” And we realized that most of them don't, and that all these patients are already identified, and young, that most of them aren't even considered a diagnosis until they have a heart attack - and this country doesn't really focus on primary prevention, it's secondary prevention - and so they're then presenting to a whole different specialty.

So it's really about understanding the conditions of the market, and the patient journey, and the perceptions. And if you go to certain specialty who are experts in this, they'll say, “I have all the patients,” because they think they do because they live in their world. And then even in their own institution there are other patients. And they would say, “Well, why wouldn't they refer them to me, because I'm the expert?”

And so with those insights you start learning that it's, well, not the way you think, and it's not the way you expect in referrals, that you've got to learn into the market, and you've got to change right away. And if you don't, then I think you're in trouble. But if you're nimble and you can do that, that's the advantage. And then you just switch it. You say, “Hey, we learned something. Let's do it differently.”

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