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05 Oct 2016 Insight Blog

Insights on Access: A welcome from Chief Medical Officer, Ivo Timmermans

By Ivo Timmermans, Chief Medical Officer, Clinigen Group


Idis hosted its inaugural Insights on Access on May 14‐15 2014. In addition to senior Idis representatives, delegates in attendance included representatives from the pharmaceutical and biotech industries, patient organizations and related service providers. The forum was designed to provide the external delegates an exclusive opportunity to learn from Idis’ expertise in the Managed Access space. There were presentations both from delegates and Idis personnel, interspersed with panel discussions and a ‘break out’ discussion on Real‐World Data (RWD) collected during a Managed Access Programme (MAP).

RWD can be defined as data collected outside the controlled constraints of clinical trials for the purposes of evaluating what is happening in ‘normal’ practice. Idis’ definition of MAP RWD is, ‘any client‐requested parameters collected across the duration of a MAP’.  Idis has developed a bespoke system to collect such client‐requested data, in real‐time, directly from physicians participating in its MAPs.

Healthcare regulators and payors are increasingly interested in effectiveness, on top of the efficacy and safety measures established during clinical trials. RWD can shed light on epidemiology, burden of disease, treatment patterns, compliance and treatment outcomes. This document sets out to summarise the discussions assessing the benefits of RWD collection within the MAP setting, the challenges surrounding real‐world data collection during a MAP, and discusses possible solutions to these challenges.


The general consensus from the discussions was that there are evident benefits to be had for pharmaceutical and biotech companies through collecting RWD during a MAP. Consistent themes emerged from the delegate discussions, such as, RWD collected during a MAP could be useful in helping to control and mitigate post‐approval commitments, could potentially shed light on issues such as outcome measures, duration of treatment, quality of life and patient adherence. Furthermore, there was a common belief that the RWD generated during a MAP could also be used for HTA publications, to support reimbursement discussions, in regulatory submissions, to inform future
development plans, new indications, and commercialisation and marketing strategies.

“Real‐world data capture in this setting could assist in the control of post‐approval commitments”

For pharmaceutical and biotech companies specific benefits around collecting RWD during a MAP were identified, including developing a better understanding of patient characteristics as experienced in routine clinical practice, as well as understanding actual treatment patterns and outcomes. Such information was identified as being important in order to gain valuable insights into the therapeutic and competitive landscapes. A wide consensus was achieved around the criticality of having detailed planning (involving different teams on the sponsor side, and within Idis) to ensure that appropriate data is collected during a MAP in order to ensure that the maximum, relevant insight can be gained during the pre‐approval Managed Access phase, preparatory to launch. Concern was expressed that there may be too many different parties with an interest to make it possible to satisfy all stakeholders. A recommended way to mitigate this was for pharmaceutical companies to lead the discussion, rather than simply asking an open question of the regulators (as to what data should be collected), as this could prove unworkable.

Overall, RWD collection during a MAP was viewed by the delegates as representing a unique opportunity to gather additional data which could provide crucial clinical insights, protect patient safety, potentially drive down costs, and provide data which could be used for a range of purposes, both internally and externally.


The question of whether there is a role for local or regional RWD collection from a MAP was discussed at length. The general consensus was that some authorities would want local data, and that differences in treatment regimes means local data collection will be necessary. However, this regionalised data capture would not work in certain disease areas such as orphan conditions, as the small patient population would make local data collection challenging.

“Local data for local submissions will always be seen as positive, as differences in treatment regimes means local data is necessary”

With this localised data capture, comes the challenge of linking these pieces of fragmented information to create a comprehensive picture of that relevant patient population, as the data should be fully representative of the population under consideration.

Further issues identified surrounding the collection of localised MAP RWD included country‐specific differences around consent and confidentiality, so the infrastructure needed to capture regionalised RWD could be cumbersome (from regulatory and operational points of view), and potentially expensive.


There are many complexities (technical and otherwise) around the collection of RWD, but particularly so within the Managed Access setting. It was appreciated that RWD collected during a MAP would be unlikely to have statistical power, given the relatively small and heterogeneous patient populations typical of MAPs. Additionally, it was understood by all delegates that the purpose of a MAP is treatment, not research, and accordingly participating physicians could not be asked to collect more data than they would normally collect as part of the routine treatment of a patient.

Further perceived challenges of collecting RWD in the MAP setting raised by the participants included:

  • Concerns that the additional burden of data collection would deter physicians from participating
  • Patient‐reported outcomes might be difficult to gather
  • It may be perceived as the wrong place to collect data before fully understanding the drug, as MAPs are frequently run before a drug has received a marketing authorisation in any country
  • Could RWD captured at the time of the MAP reveal false safety‐signals?
  • Given that the purpose of a MAP is treatment, not research,  can the data desired be reconciled with the data that it is permissible to capture?
  • A concern that such data may not be of a quality robust enough to be put to practical use
  • The timelines for collecting and analysing RWD gathered in the MAP setting (whilst the marketing authorisation process is ongoing) raises the concern that it might not be timely enough for it to be deemed useful


Recognising the various challenges identified by the delegates around collecting RWD during a MAP, it is clear that any RWD collection during a MAP needs to be carefully planned, managed, deployed and communicated.

Firstly, considering the potential barrier of adding a further burden to participating physicians. This could be mitigated through a combination of careful RWD planning to minimise that extra burden upon physicians, through effective communication with physicians to ensure they understand the overarching benefits that the RWD collection may bring, and through appropriate incentivisation, where necessary.

Idis has already factored in the desirability to collect patient‐reported outcomes and QoL assessments, and future development of its MAP RWD system will incorporate such functionality.

Regarding concerns over the potential to pick up false safety signals, and whether a MAP is an appropriate setting in which to collect data, it is Idis’ belief that more data is always beneficial, even if that data is only used for internal guidance, and the ability to identify any potential signals is preferable sooner rather than later.

In terms of what data can be captured, and the quality of that data, even within the confines of being able to collect only routinely collected data, a wealth of useful data from the real patient population is available. Further, Idis’ RWD system is built to industry‐standards, and in a way to minimise data entry errors. Additionally, data collected will be reviewed internally and where errors or omissions are identified, Idis will reach out to the physician to ensure any missing/incomplete data is corrected. Notwithstanding that RWD collected during a MAP will lack the statistical power of data derived from clinical trials, and as such, the data is likely to be more useful as hypothesis‐generating, insightful, supplementary data, useful internally, and give insights into future studies and issues around compliance, adherence etc. To ensure sponsor expectations around data capture are managed appropriately it is critical that both sponsor teams, with guidance from Idis, communicate the full benefits, uses and limitations capturing and RWD.

With regard to the timeliness of data capture, Idis’ RWD system will allow real‐time extraction of data, meaning sponsors can extract data at multiple points of their choosing across the MAP. MAP enrolment generally peaks in the first year, and given the length of the marketing authorisation process in different countries, data collected during the first year of a MAP could still prove useful in many markets. Additionally, many of the benefits of RWD collection through a MAP relate to internal sponsor use, rather than external use.


RWD is becoming increasingly important for pharmaceutical and biotech companies. RWD in the MAP setting provides a unique opportunity to collect specific data from a patient population more closely reflecting that of routine clinical practice, providing insight into actual outcomes in a real‐life patient population, a better understanding of the patient journey, insight into patient adherence, quality of life etc.

There are many potential uses for such data, from being utilised for HTA publications, through supporting reimbursement decisions, regulatory applications, guiding future development plans as well as informing commercial and marketing strategies.

Clearly, a number of challenges, both technical and operational, exist, but none of these are insurmountable, and Idis is hopeful that its new ability to capture RWD from patients in the MAP setting will benefit its clients, patients, physicians alike.

To discuss your MAP real‐world data needs with Idis, please email us at: