Ten tips for industry when planning a CUP

Not all companies organise their Compassionate Use Programmes in the same way.  Below are some tips, based upon our experience.

  • Tip 1: Meet with patients’ representatives and clinicians before communicating important clinical trial results in scientific conferences or via press releases. Communicating on promising results of a new product to treat a severe or life-threatening disease will inevitably create hype in the patients’ community. Request to obtain the medicine on a compassionate use may follow this communication and the company needs to anticipate a demand. Communication is a corporate responsibility, and all consequences should be anticpated beforehand. By engaging an early dialogue with patients and clinicians, you can explain 1) when you think the manufacturing capacity might authorise you to open a Compassionate Use Programme, for how many patients, how it might evolve in the future, 2) which inclusion criteria you have in mind,  3) if the demand is too high, how you think you can pace down the programme, 4) should a marketing authorisation application be rejected, what will happen to the programme and to patients willing to continue treatment, etc.

  • Tip 2: A Compassionate Use Programme is a life-saving tool. Patients’ representatives who engage in a dialogue with a marketing authorisation applicant are not advocating for their own health, but for all patients. Information on compassionate use cannot be confidential. Equity in accessing a Compassionate Use Programme should be entire and complete. No one should be excluded from a Compassionate Use Programme due to a  lack of information. There is a balance to respect, between the ban on promotion of Compassionate Use Programmes and the necessity to ensure equity in accessing a programme, which needs to be discussed with regulators, patients’ representatives and clinicians.
  • Tip 3: Accept enrolling patients with all comorbidities and impairments. Even if a patient is suffering from a severe liver or renal impairment, it is he/she and the doctor to decide whether or not to participate in a Compassionate Use Programme. No company should be blamed for giving access to a potentially harmful product on a compassionate basis. The consent form is there to explain all risks, even though obtaining free and informed consent in a compassionate use situation is questionable for patients who are severely affected and may be close to death. Along the same lines, pregnant women, or patients expected to adhere poorly to the treatment, should also be given access. In all cases, collect data; this may complete your regulatory application dossier.
  • Tip 4: Senior management in the company or members of its governing bodies may request access to a medicine the company is developing on a compassionate use basis, for themselves, a relative or a friend. There should not be any privilege; all patients should be considered equally, with no distinction based on social-status, origin, gender, age, education, sexual orientation.
  • Tip 5: Refrain from using the compassionate use as a reward to clinical investigators who recruit large numbers of patients in your clinical trials. In a very competitive world, a company can be tempted to gain the favours of clinicians by offering them access to the medicine on a compassionate basis if they manage to enrol high numbers of patients. This practice is not official but exists, and is not recommended as it creates inequity in accessing the medicine on a compassionate basis.
  • Tip 6: Collect as much information as you can from the CUPs you run; data can include information on the toxicity profile of the product, detecting new adverse events not seen in patients enrolled in clinical trials, or evaluating the impact of risk minimisation measures if a risk management plan is in place. It can also provide indications on the efficacy: the percentage of prescriptions that are renewed is an indicator. When on a compassionate use, if there is no benefit for a given patient, his/her doctor may not renew the prescription. On the contrary, if the patient seems to benefit from it, the prescription is likely to be renewed.
  • Tip 7: If the demand is too high, do not propose biomedical criteria to select patients in greatest need. These criteria may end up arbitrary, will probably not be accepted and not adhered to. Instead, create a waiting list, target a reasonable waiting time (e.g. 5 weeks), monitor the product supply (the demand is rarely the same in all countries, and some supply can be transferred from countries where the demand is low to countries where it is higher), and communicate regularly with patients’ representatives and clinicians on the average waiting time by country.
  • Tip 8: When the CUP is running, meet at regular intervals with patients’ representatives and clinicians to discuss which adaptations are needed. The fact that sites cannot be selected (any request needs to be honoured, for any patient that meets the criteria for use of the drug), and also because the concept of Centre of Expertise is not yet established in all Member States, adds to the logistical effort and costs (principle of equal access and non-discrimination against patients).
  • Tip 9: If you obtain a marketing authorisation, do not stop the CUP immediately, but wait until a price and reimbursement decision or a coverage decision by the health care system is made, so that patients treated via the CUP do not interrupt treatment. You may decide not to enrol any new patient in the CUP and to continue providing the medicine only to those who entered the programme prior to the marketing authorisation. In any case, you should be clear about how you plan to end the programme, whatever the regulatory decision.
  • Tip 10: Be clear on what will happen to the CUP if a marketing authorisation is not granted, or if you withdraw the marketing authorisation application for a certain period, before submitting a new one at a later stage.

Page created: 30/10/2013
Page last updated: 07/11/2014
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