Patient Summit USA

May 2, 2013 - May 3, 2013, Philadelphia

Understand the patient journey to build better adherence and engagement platforms

Patient Advocate Collaboration - Novel Methods for Rare Diseases

Louise Kelly talks to Dr. Bartholomew Tortella, Senior Director of Medical Affairs at Pfizer about rare disease clinical trials and the pivotal role patient advocacy groups can play in recruitment.



One of the biggest challenges in rare diseases is clinical trials patient recruitment. But with the rise in the sophistication of patient advocacy groups and hands-on approaches from innovative medical development teams, the numbers taking part in rare disease investigations are also set to increase. Big Pharma are lending a large funding hand as they realize how valuable this patient insight is in improving drug development for the market at large.

Medical affairs and clinical trials/development is quite different when considering rare diseases as opposed to more common ailments such as heart disease or diabetes, according to Dr. Bartholomew Tortella, Senior Director, Medical Affairs at Pfizer whose areas of expertise includes sickle cell disease (SCD) and hemophilia.  “You have to think about things far more from the patient perspective than the investigator,” he insists.

“For example, if you are running a diabetes trial, the most important thing is to line up the right investigators and the patients will come – as the availability is there.  This structure definitely needs to be in place with rare diseases but, because the investigators may only see two or three of these patients a month (or not at all if they have a stable disease), this approach is not enough to ensure clinical trials are carried out in a sufficient and expedient manner.”

Addressing these organizations with just the PA on board is not sufficient as the science of the subject would not be adequately covered.

Unless patients are organized in advocacy groups and are on a specific disease registry, there is a difficulty to identify and locate the patients. The fact that there are a small number of patients for each disease, scattered over large geographical area and around the globe, it requires developing new models for conducting clinical, and other studies, with small patient population.

Proactive approaches

Firstly, medical affairs need to reach out to patient organizations; Dr. Tortella uses the SCD Association of America – primarily a lay organization – as an example.  This outreach needs to be conducted as a collaborative effort between public affairs (PA) and the medical affairs team so that both physicians and lay people can be communicated with effectively.  “Addressing these organizations with just the PA on board is not sufficient as the science of the subject would not be adequately covered,” he says, adding, “However, they are just as necessary in the process to establish the right connection with a lay association – and to maintain the relationship.”

This on-going affiliation includes liaising with the advocacy press team in-house so that up to date information concerning drug trials and development is included in group circulars and on their online site.  But where the real difficulty lies is with patients that are not necessarily interested in joining these advocacy associations.  Then it’s time to approach the local community directly.  Dr. Tortella again uses the case of SCD, explaining that the disease is primarily linked to African Americans.  “If you have a site in Atlanta Georgia, for instance, you may need to approach churches and community centers– again public affairs and medical affairs hand in hand – asking them to inform their members about the opportunity and options they now have in treatment and research and encourage them to contact their physicians.”  

“If they are not joiners of the local chapter of the SCD association, how else are you going to reach the parents and patients? These are things that large pharmaceutical companies, often new to clinical research in rare diseases, don’t necessarily think of doing,” he says.

Furthermore, Dr. Tortella is adamant that when the services of a contract research organization (CRO) are employed, part of the contract negotiation has to include outreach to these local community and faith-based organizations.

Benefits for the future

Diseases such as SCD, that have no cure at all, are in the early stages of development with large pharma firms just starting to delve into the sickle cell arena. (Ordinarily, one or two small biotechs complete the phase 1 or phase 2 studies. The large expensive phase 3 study that leads to filing and approval is often taken over by a large pharma firm.) Yet with the more well-established diseases such as hemophilia, treatment is fully developed and mature as pharma companies have been investigating and perfecting the appropriate drugs/procedures for almost fifteen years.

The growing sophistication of the advocacy organizations is seen as a good news story as these groups are one of the main areas of education for those affected by the related disease. “How do you dig down and learn more about the disease and the treatment?  The internet can only provide book knowledge but the local organizations and support groups are particularly important for the more practical and in depth aspect of the disease state.”

Furthermore, a better relationship between manufacturer and organization allows the pharmaceutical company a better understanding of the medical needs of the patient community as they visit these organizations where they can communicate with patients and really start to understand the important points and attributes of the product being developed, insists Dr. Tortella.  “What are the important issues for a patient with SCD? How will the development of this medicine impact the lives of a patient with hemophilia? Unless you understand that, you couldn’t effectively put messaging together that would be meaningful for the patient,” he says.

This understanding also allows the drug manufacturers to look into the future and to distinguish what the impending needs of these patients will be.  They can incorporate this knowledge into their planning as scientists like Dr. Tortella use it for their medical development.


Dr. Tortella will be speaking at this year's Patient Summit USA. If you'd like more information on successfully implementing patient advocacy collaborations, or to find out more about the event visit the official website.


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Patient Summit USA

May 2, 2013 - May 3, 2013, Philadelphia

Understand the patient journey to build better adherence and engagement platforms

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