Being a challenging and arduous operation, finding new therapies to treat cancer often is a time-consuming process. For some patients, marketed therapies are to no avail, while at the same moment new optional therapies are on the way to registration. Sometimes these therapies are just a few months away of being available – a period that can make the difference. An alternative to overcome this hurdle could be to participate in a Compassionate Use Program (CUP).
In some cases, it is in the best interest of the patient to have early access to certain medication before market authorization while not all new promising drugs are immediately commercially available after centralized marketing authorization is granted by the European Medicine Agency (EMA).
For compassionate reason, pharmaceutical companies are allowed to set up a program to make non-authorized medicinal products available for patients who may benefit from this treatment. Such a program is referred to as a Compassionate Use Program (CUP) or Expanded Access Program. A CUP is intended for patients who suffer from chronically, life threatening, or seriously debilitating disease who cannot be treated satisfactorily by an authorized medicinal product currently marketed in Europe and approved for the treatment in this indication. During such a program, the pharmaceutical company provides the medicine (free-of-charge) to individual patients on request by and under the responsibility of the treating physician. A CUP may also serve as a way to provide patients continued access to treatment following a pivotal clinical trial.
In 2017, we have been successful in assisting pharmaceutical companies with the execution of a CUP that is performed in over 50 hospitals in the Netherlands. All required processes are implemented in the company to provide expert guidance and corresponding tailored CUP services.
Compassionate Use Program versus clinical trials
It is important to realize that CUPs do not have a research rationale and data collection is often limited to safety data. Although CUPs provide pharmaceutical companies the means to collect additional safety data and expand the safety profile of the therapy, such programs cannot replace clinical trials for investigational purposes. Therefore, compassionate use should not slow down the continuation of clinical trials to provide essential information relative to the benefit/risk balance of a medicinal product. In addition, patients cannot participate in a CUP in case they are eligible for a clinical trial in the envisaged indication of this program.
The EMA provides guidelines on the compassionate use of medicinal products. In the European Union (EU), CUPs are coordinated and implemented by individual Member States (MS) and are governed by the legislation of the respective MS. The pharmaceutical company submits a compassionate use application to the National Competent Authority (NCA) of the relevant MS. After a CUP is approved and has been set up, the pharmaceutical company is obliged to ensure that all patients taking part in the CUP have access to the medicinal product until it is launched commercially on the market. In the Netherlands, the pharmaceutical company must submit the application for a CUP to the ‘College ter Beoordeling van Geneesmiddelen’ (Medicines Evaluation Board; CBG-MEB). A list of all CUPs approved by the MEB can be found here.
In contrast with the common practice in clinical trials, selection of participating hospitals is not generally driven by conventional criteria (Figure 1). A hospital may “self-select” suitable patients, may have little to no research experience and participation to a CUP may be approved as a result of subject self-referral. No marketing promotion by the sponsor is allowed.
The duration of a CUP typically depends on regulatory approvals and/or commercial availability. Consequently, all patients that are being treated in a CUP will migrate to the commercial medicinal product upon availability, provided that market authorization is granted for the defined indication of the target population of the CUP. In the EU, specific transition plans must be established prior to commercial availability, which must take into account the local regulations.
CUPs Expertise
The execution of a CUP requires specific expertise. We have a dedicated and flexible project team is assigned in order to guarantee clear oversight in the jungle of regulations. The services that are regarded to be of utmost value include the medical expert advice to the treating physicians by our in-house physicians, the instruction visits to hospitals by our oncology CRAs and our possibility to quickly scale up in case of unexpected increase of patient participation. In addition, the proficient evaluation from a medical scientific point of view to confirm the validity of the explicit, unsolicited requests from the treating physicians lead to short timelines and a fast increase of patient participation. SMS-oncology takes pride in efficiently implementing CUPs in daily clinical practice and being on top of requests and questions from hospitals, in order to contribute to the early access of promising medicinal products to patients.
Interested in what we can do for you? Contact us for more information on how we could support your CUP!
References
- American Cancer Society, Compassionate Drug Use
- CBG-MEB, Compassionate use programme
- European Committee, REGULATION (EC) No 726/2004 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL
- European Medicines Agency, Compassionate Use
- European Medicines Agency, GUIDELINE ON COMPASSIONATE USE OF MEDICINAL PRODUCTS, PURSUANT TO ARTICLE 83 OF REGULATION (EC) No 726/2004
- European Medicines Agency, Questions and answers on the compassionate use of medicines in the European Union
- Federal agency for medicines and health products, Guidance on compassionate use and medical need programs
- Heads of Medicines Agencies, Compassionate use program
- Medicines Evolution Board, Compassionate use programme
- U.S. Food and Drug Administration, Expanded Access (Compassionate Use)
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