PSR’s Mindset

Creating a true partnership with dedication to rare disease patients and our clients to make a positive impact on the lives of people with rare diseases is what drives PSR and its team. Both PSRs input on delivering new treatments for rare disease patients and our participation in fundraising activities exemplify our dedication to the rare disease field on a business as well as a societal level.

PSR is very privileged to work with many biotech and pharma pioneers over the last 16 years in assisting them with getting new therapies available for rare disease patients. We aim to accelerate the clinical development of orphan drugs and facilitate access to treatment for rare disease patients and their families.

We are proud to be experts in the regulatory and clinical development of orphan drugs, working closely with patients, companies, patient groups, regulators, key opinion leaders, government funders and other stakeholders.

WE CARE ABOUT RARE!

PSR’s Mindset

Creating a true partnership with dedication to rare disease patients and our clients to make a positive impact on the lives of people with rare diseases is what drives PSR and its team. Both PSRs input on delivering new treatments for rare disease patients and our participation in fundraising activities exemplify our dedication to the rare disease field on a business as well as a societal level.

PSR is very privileged to work with many biotech and pharma pioneers over the last 16 years in assisting them with getting new therapies available for rare disease patients. We aim to accelerate the clinical development of orphan drugs and facilitate access to treatment for rare disease patients and their families.

We are proud to be experts in the regulatory and clinical development of orphan drugs, working closely with patients, companies, patient groups, regulators, key opinion leaders, government funders and other stakeholders.

WE CARE ABOUT RARE!

Management Team

Managing Director

Christa van Kan

Christa van Kan has more than 20 years of experience in managing clinical trials in a broad range of diseases, including many orphan and paediatric indications. She is one of the co-founders and owners of PSR Orphan Experts. In her role of Team Lead Clinical Execution and hands-on Senior Project Manager, Christa has developed several innovative recruitment strategies for orphan drug trials. “What I love most about my work, is the fact that we’re often in close contact with patient groups, both for recruitment purposes and to use their input to make a clinical trial less burdensome for the patient and their families. I really enjoy finding ad-hoc solutions to all sorts of logistical challenges which often occur in orphan drug trials.”

Team Lead Data Management & Vendors

Margreet op ‘t Hof

Margreet is leading the Data Management Team and responsible for managing our vendors. She has over 9 years of experience in both sponsor and CRO environments, covering all aspects of data collection in clinical trials in multiple therapeutic areas, including rare diseases. She likes to work cross-functionally to find pragmatic and user-friendly solutions for data collection challenges.  “I believe that smart trial design and effective use of new technologies can truly improve the data quality, lower the patient burden and reduce workloads. Especially in Rare Disease trials (often small in terms of subject numbers but big in terms of complexity) every data-point counts and every effort should be made to collect it.”

Head of Rare Disease and RWE Research

Dr Alex Artyomenko

Dr. Alex Artyomenko currently serves as the Head of Rare Disease and RWE Research at Ergomed. Initially trained as a cardiologist, Alex managed acute care for ICU patients prior to joining the research field. Alex has nearly 20 years of clinical research experience, and has been involved in developing and implementing study designs and operational strategies for global studies and programs, involving many types of observational studies and therapeutic indications. Alex’s passion is research and development of new treatments for rare diseases, with a patient-centric approach to research. He is an active member of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), focusing on improving standards for observational research, and helping companies to strengthen the value of their products through Real-World Evidence.

Note from our Patient Engagement Officer

Mirosavljević
Patient Engagement Officer

  • Internationally Awarded Rare Disease Expert
  • Founder of the Patient Association “Zivot/Life”
  • Active in the Area of Raising Awareness of Rare Diseases, Fundraising and Lobbying
  • Involved in the Creation of New Legislation (Zoya’s Law) Concerning the Prevention and Diagnosis of Genetic and Rare Diseases. 

“PSR’s Patient Engagement Officer (PEO) will initially support the feasibility process by using her large patient advocacy network to connect to the relevant patient organizations and expert sites. Potential sites will be included in the feasibility process. 

During the preparation phase, the PEO will coordinate review of patient-facing documents (and possibly the study protocol) by the patient organization(s).

During the Recruitment phase, the PEO will stay in close contact with the patient community to increase trial awareness amongst the patients and to stimulate patient referrals. If possible, she will attend patient group meetings to present the study and/or answer questions about the study. She may also act as the central point-of-contact for patient organizations. “

Awards

  • Special European Black Pearl Award for Written Media, (Word for Life magazine), Eurordis European Organization for Rare Diseases (2018)
  • Special Annual Award for the Improvement of the Position of Patients with Rare Diseases, National Organization for Rare Diseases Serbia (2017)
  • American Chamber of Commerce Annual Leader in Change Awards, “Hero of the Year” (2015)

Science and Societal

Despite general acknowledgement of the benefits brought by innovative therapies for rare diseases, patients suffering from rare diseases continue facing difficulties in receiving a correct diagnosis and in getting timely access to adequate treatment.

This situation undermines the right to have equal benefit from the healthcare system between patients suffering from more common diseases versus patients suffering from rare diseases. This may lead to a negative discrimination among citizens which should be prevented on the basis of the principles of equity and solidarity (the core values of our society).

In this respect a joint effort with all stakeholders involved is necessary, taking into account, amongst others, the following aspects:

  • Engage in a constructive dialogue with all relevant stakeholders to define a shared and consistent approach to the needs of the rare diseases’ sector
  • Raising awareness around rare diseases, facilitating early diagnosis (including newborn screening) and limit the use of inappropriate treatments for patients.
  • Further optimize the use of patient registries to fill the gap of data collection and strengthen available scientific knowledge and understanding (including natural history studies).
  • Address ‘long-term’ policy environment (with continuous dialogue and political commitment) in order to achieve recognition of the needs of patients suffering from rare diseases & supporting the R&D infrastructure.
  • Jointly develop innovative reimbursement strategies & price models to ensure ‘long-term’ access to effective new treatments for rare diseases.
  • Achieve predictable and timely procedures by regulators to facilitate access to treatment for patients.
  • Enhance patient representation in policy, regulatory and industry processes.