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

Chief Executive Officer

Roger Legtenberg

After obtaining his PhD in physiology at the University Medical Center Nijmegen, Roger has worked over 15 years in various positions in the CRO industry. During the last six years, as CEO of PSR, he succesfully lead PSR’s specialisation & expansion into the orphan drug niche. Besides his role at PSR, he is also actively involved as member of the advice group orphan drugs and rare diseases of HollandBio (Dutch Biotech Association) and member of the board of the Association of Contract Research Organisations in the Netherlands (ACRON).

Team Lead Clinical Operations

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.”

Business Development Manager

Thijs op de Weegh

Thijs is our Business Development Manager, responsible for building and expanding PSR’s business globally.  Thijs has over 9 years experience as an entrepreneur in the Biotechnology and Pharmaceutical sector and a background in Biomedical sciences. During his experience as an entrepreneur he gained international commercial experience, strong customer focus and expertise in a variety of areas e.g. molecular diagnostics, small bio-techs. “Within PSR I am proud of the dedication and willingness within the teams to make the extra effort for the patients with a rare disease and their families, this mindset is what sets us apart!”

Team Lead HR & Sourcing

Matthijs Blommers

Matthijs is dedicated, as Team Lead HR & Sourcing, is responsible for all staffing related issues in the PSR Organisation. Ranging from recruiting new professionals and strengthening our project organisation to facilitating the (personal) development of existing employees, he and his department are dedicated to ensure the PSR organisation and its professionals have all the tools at hand to reach their, our goal(s): developing a cure for rare diseases.

Note from our CEO

For several decades, rare diseases were neglected by large drug development companies. Only a handful of small biotech companies had the drive and passion to search for treatments for these patients in need. Not because they wanted to make money, but because they cared about patients that had no alternatives, no hope.

Due to succesfull implementation of supportive legislations in the 90’s and 00’s, the number of new drugs developed for rare diseases has considerably increased. PSR has been very privileged to work with many companies & patient organizations for almost 20 years to significantly contribute to this success. However, the vast majority of the identified rare diseases still do not have any treatment available, which makes our passion and commitment to work in this exciting field even stronger.

With the progress made in rare diseases, also the debate on the long-term sustainability of the above mentioned system is increasing over time. I believe that preservation of the original purpose of the stimulating measures implemented is of crucial importance. If this will not be fostered, no equal chances for new treatments for rare diseases versus more common diseases will be applicable in the future.

In order to do so, a new business eco-system will be needed in which the rare disease patient is considered as the real customer. Initial steps have been taken towards such a new system, but there is still so much more to be achieved involving all stakeholders. Besides participation in several organizations and communities, we are also actively implementing new initiatives in order to involve patients in the developments of orphan drugs as early as possible, thereby achieving true patient-centricity.

I am very proud to lead and represent PSR, together with my colleagues who fully embraced the ultimate goal to make significant contributions to create a sustainable system with effective and accessible treatments for rare disease patients.

Roger Legtenberg, 1 Jan 2017

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.
Planetenweg 5
2132 HN Hoofddorp
The Netherlands
Tel: +31-23-55 63 220
Fax: +31-23-55 63 230
Email: info@psr-group.com
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