Dan Zaksas

By Natasha Cowan, Content Marketing Manager at Fishawack Health

The pharmaceutical industry has set its sights on rare diseases with high unmet needs. However, as competition ramps up, how should pharma adapt its approach to succeed in this environment? Daniel Zaksas, PhD, Senior Vice President, Director of Scientific and Medical Affairs at Dudnyk, part of Fishawack Health, shares his learnings from a decade working in rare disease.

During the last 10 years, the pharmaceutical industry has ramped up its interest in rare disease. The number of new rare disease therapy approvals globally doubled from 29% in 2010 to 58% in 2018, according to research by Tufts CSDD.

A rare disease is defined as a medical condition that affects fewer than 200,000 people in the United States or fewer than 1 in 2,000 people in Europe. These diseases are defined by small patient populations and this leads to several challenges for drug developers. The market opportunity is far smaller than pharmaceutical companies have traditionally experienced when launching blockbuster drugs. Patient identification is also difficult, as is preparing healthcare professionals to diagnose patients with a disease they may not have heard of and may never come across during their careers. Additionally, as more companies target the same diseases, competition for these small groups of patients increases. 

Natasha Cowan

To successfully navigate this environment, the industry will need to alter its approach. We interviewed Daniel Zaksas, PhD, Senior Vice President, Director of Scientific and Medical Affairs at Dudnyk to learn his advice for launching a therapy in a small patient population.

Dudnyk joined Fishawack Health in 2019, adding extensive rare disease experience to the organisation’s medical, creative and consulting expertise.


Why did you want to work in rare disease? 

DZ: I think back to when I was a scientist and my work in the lab was driven by the sense of discovery and possibility. There was the sense that every day you have the chance to contribute something new and accomplish something that had never been done before. Working in rare diseases gives me that same feeling. Every educational campaign we develop, every therapy we help to launch, we can see it making a real difference. So, we have seen those real-world impacts that can help physicians solve their medical mysteries and help patients and their families – it’s extremely gratifying.


How is the rare disease space is evolving? 

DZ: The sheer number of diseases being addressed and therapies in development are pretty staggering compared to where we were even a decade ago. A third of the industry’s R&D pipeline is now in rare disease, and we’ve had almost 400 therapies approved exclusively in orphan indications. So, this is great news for patients who have waited for years or decades to be diagnosed or be treated, but there are also new challenges such as increased competition.  

Physicians are also now being asked to understand, diagnose, and treat many more diseases, including diseases that they might have heard mentioned once at a lecture in medical school. So, that’s a challenge. A decade ago, payers wouldn’t think twice about reimbursing some expensive therapy that only affected one or two covered lives, but now they’re facing a lot more pressure and, in turn, exerting more force to make sure pharma shows that these expensive therapies are providing a clear and measurable value.  


How do you educate physicians who not have seen a patient with the rare disease before?

DZ: The education part is not the challenge – the challenge is the motivation. Physicians have a great capacity and desire to learn new information. However, the material they choose to stay on top of must be that which is the most relevant to their practice.  

When you ask them to retain new information about the diagnosis of a rare disease, you have to communicate very clearly why retaining that information will make a real difference to the well-being of their patient.  

Ironically, what often motivates physicians to look for a rare disease is the knowledge that an effective therapy is either approved or in development for that disease. If establishing an obscure diagnosis is just an academic exercise, and they can’t really do anything about it, they’re less likely to retain any education you throw at them.


Some rare disease therapies are only prescribed in centres of excellence. How can pharma companies make this process easier? 

DZ: This is very relevant to gene therapies, which often require very precise delivery to specific sites on the body. So, the path to getting the treatment can involve several steps from genetic testing to evaluation, prescription, and administration. 

Success is about clarity of communication. Referral pathways must be clearly established between the diagnosing and prescribing physician. Also, there must be clarity about who is responsible for any follow-up, especially if the therapy is a single-dose treatment.  

Importantly, this whole process should be totally seamless for the patient and their family. We don’t want to add additional stress and expense to the challenges they already face, especially as they might be travelling a significant distance for the procedure.


In what other ways is launching a rare disease therapy different?

DZ: In many ways, the fundamentals of the launch are the same but there are a few elements that are different and critical for success. Diagnostic rates in rare disease are often completely abysmal. You have diagnostic delays that can be measured in years or even decades, and the referral patterns between physicians are often either absent or completely fragmented. Patients are wandering the healthcare system with no recourse, no resources, and often no hope. You can’t really launch a therapy into a landscape like that successfully unless you profoundly change those underlying conditions.    

As an agency, we think that success is contingent on building very strong relationships with the patients and earning their trust. You have to do that by evaluating the needs they have outside of strictly delivering the therapy itself. So, what do they need and how do we help fulfil those needs?  

The last thing we’ve learnt is that the basic patient support services used in traditional launches just don’t cut it in rare disease. The services have to be comprehensive, but also personalised and by making them personalised you start to build that trust that I mentioned, which eventually leads to brand loyalty.


How are you improving the relationship between healthcare professionals and rare disease patients?

DZ: The reality is that the relationship between doctor and patient in rare disease can be all over the map.  A big part of our mission as an agency is to help make those relationships as effective as possible through education and communication.

If patients are well informed, physicians may be ceding a lot of control over therapeutic decisions to the patient. But if you have poorly informed patients and poorly informed physicians, you get really disheartening results.  

So, for us as an agency, the healthcare professional/patient relationship is really at the heart of our philosophy. Shared understanding and empathy between patients and their physicians are foundational. That has to happen before you successfully educate the physician and patient and before you can ensure the patient receives the right therapy. We call this The Unifying Effect. 

It means getting them to speak the same language and communicate in the same way, sharing a common lexicon. But it’s more than that; it’s about being open about individual concerns and in complete agreement on the goals of therapy and the goals for the patient’s quality of life.  

I say that these relationship dynamics are different in rare disease, but they shouldn’t be. This model I described has been diligently worked on and refined over the past few years in rare disease. In reality, this should be the model of every healthcare professional/patient relationship across the board, whether the disease is rare or not.  


What advice do you have for designing a successful education programme for patients?

DZ: Patient education is highly dependent on the disease state in question. In many cases, diagnosed rare disease patients really seek connection with other patients like them. Education may well take the form of patients sharing their stories or experiences with each other.  

In some cases, it really helps for a pharma or biotech company to form strong relationships with advocacy groups because, unlike in the larger disease states, advocacy groups might be very much underfunded, and they might very much welcome industry involvement. Investment can empower them for the first time to provide the type of support they would like patients to receive, and gain the ability to disseminate that information effectively.  

Many of the rare diseases are familial, inherited, and in that case, you want to empower and equip family members to be educators, whether it’s for diagnosed relatives or for undiagnosed individuals who have a risk of developing the disease based on their genetics.  


Those discussions can be very sensitive, how do you handle that?

DZ: They are tricky, and the key is really understanding the audience. You must form the relationships with the patients, you must spend time with them and their families, you have to understand what the sensitivities are, what the trigger points are. That level of familiarity is necessary before you can create any materials or decide on a strategic direction. The intimate understanding is vital.


Do you have any examples of this working in practice?

DZ: We have had several outstanding experiences. For example, we’ve worked for a decade now in hereditary angioedema (HAE), including on the first product launched for prophylaxis to prevent debilitating and deadly attacks in this disease state.  

Historically the diagnostic delays were terrible. And without going into the details of the disease, its symptoms can be fairly nonspecific and resemble those of allergy or other conditions, so it took patients 12 years or longer to get an accurate diagnosis.  

We conducted some very successful education and engagement campaigns. Since HAE was a familial disorder, we encouraged patients to communicate and we also created tools for families to facilitate those conversations. We created campaigns for physicians to instil a sense of urgency to diagnose because now they had a real ability to change their patients’ experience with the disease. In the years since, diagnostic rates have gone up and diagnostic delays have gone down to under ten years. So, we have seen these techniques actually bear fruit.


That’s a huge difference. Dudnyk recently joined the larger Fishawack Health agency, has that changed your approach?

DZ: It means we can leverage the group’s strategic, scientific, and creative capabilities to greatly amplify The Unifying Effect. We can begin to build essential relationships earlier than ever. Then, we can use the full spectrum of healthcare communications to ensure that patients, families, caregivers, and healthcare providers are sharing perspectives and speaking in one language by the time a life-changing therapy becomes available.


Lastly,  if you had to give one piece of advice for a pharma company who’s launching a therapy in a small patient population what would that be?

DZ: My top piece of advice sounds really simple – get to know your patients, their families, and the doctors who treat them. And I don’t mean get to know them as patients or segments but get to know them as people and then treat them as such.

Figure out what they like, what makes them anxious, what makes their life difficult, and find ways to help them. Don’t stop at making sure patients can afford their therapy, help them to lead a more fulfilling life. Do this because it’s the right thing to do and ultimately your actions will be appreciated by the community, appreciated by the physicians, and by the patients themselves.


Fishawack Health is full-service, global healthcare communications group with over 800 medical experts, marketing professionals and strategic specialists. To find out more contact [email protected]