Global healthcare's embrace of patient support programmes spurs regulatory action

The world healthcare sector, in response to the growing complexity of medications and diseases, is increasingly adopting patient support programmes (PSPs) to help patients manage their treatment and conditions, whether to take medication correctly, reduce complications or navigate the finances of medical coverage.

PSPs are now an essential tool for pharmacovigilance and the gathering of data to improve patient safety. Almost all large pharmaceutical companies provide some level of PSPs for medications that treat chronic conditions, and smaller and medium-sized companies have invested in these programmes. For those engaged in the treatment of rare diseases, PSPs are a mainstream aspect of their business.

Regulatory patchwork

However, the regulatory framework around the growth in PSPs remains patchwork and diffuse; for example, there is no uniform definition of PSPs at European level.

The European Medicines Agency (EMA), in its Guidelines on Good Pharmacovigilance Practice (GVP), defined a PSP as "an organised system where a marketing authorisation holder receives and collects information relating to the use of its medicinal products."  The agency's examples of PSPs include post-authorisation patient support and disease management programmes, surveys of patients and healthcare professionals, information gathering on patient compliance, or compensation and reimbursement schemes.

The EMA's definition is the same as the UK Association of the British Pharmaceutical Industry set out in its 2011 "Guidance notes for patient safety and pharmacovigilance in patient support programmes".

The Code of Conduct of Medicines for Europe, which is a non-profit, non-governmental organisation that represents industry associations and companies from across Europe, also defines PSP as “company-funded non-promotional arrangements that help consenting patients or carers, either directly or via their doctor, to better understand and/or manage their disease." 

Examples of PSPs that are given by Medicines for Europe include the provision of third-party nurses to assist individuals with drug administration, a patient helpline and periodic contact to check adherence to medication. It notes: "Selection and management of PSP vendors, and decision-making about PSPs, are typically within the company's medical function with significant support from pharmacovigilance, and must be independent of sales and marketing personnel.”

There are no EU laws and regulations uniformly governing the implementation of PSPs. However, many countries, regions and industry groups have local regulations that directly address, and often restrict, these programmes. There are also some main principles for marketing authorisation holders (MAH) to follow in order to carry out a PSP that is compliant with applicable healthcare laws, regulations and ethical provisions and more general regulations, such as anti-corruption laws. 

Programme models

From an operational perspective, the lack of regulation has allowed for the development of different "modalities" or ways of implementing PSPs, which will also depend on the type of product or device, the pathology and treatment, at the premises of health care organisation (HCO) or at the patient’s home. 

PSPs must be designed and implemented by compliance and medical affairs departments in order to avoid any commercial purpose and use. These departments can ensure that there is proper management of pharmacovigilance, data protection, communication of the programme and materials.

Specifically, the PSP must be included in the company’s pharmacovigilance plan. The compliance departments must also ensure that data collected during the PSP are used for the purposes of supporting patients only. Any other use needs to be agreed separately, in compliance with applicable law and deontological principles. PSP-related materials must also comply with the rules on advertising of medicinal products, which are contained in the Community code relating to medicinal products for human use (Directive 2001/83/EC) and in domestic regulations. Proper management is also required of materials, such as drugs and medical devices, and their disposal.

The approval by the treating physician or by the HCO is necessary in order to carry out a PSP, whether inside the HCO or at the patients’ home (and patients need to give express consent to this as well as to the treatment of their personal data). 

A PSP can be conducted by the MAH or the medical device (MD) company   directly or by a third party providing the qualified health care professional (HCPs)  support that is required by the programme.  Only doctors and qualified operators can support a patient and user in a PSP; some exemptions are applicable when the support only involves the assistance or training in the use of medical devices.

If the PSP is carried out with the support of an external provider, the MAH or the MD company will need to enter into a master service agreement to govern reciprocal obligations and responsibilities. Usually, the provider also drafts an operating manual that explains in details all the phases for the implementation of the PSP and to which all the parties involved shall abide.

Particular attention should be paid to the requirements of the service providers involved in PSPs: both to the type of service provided – mainly to ensure that there are functions in place that are suitable for guaranteeing compliance with obligations imposed on the MAH, such as pharmacovigilance – and to the protection of personal data.

When a service provider is involved, some jurisdictions recommend that the PSP model's compliance with employment laws is evaluated in order to avoid illicit staff leasing. Homecare PSPs can be carried out as long as the summary of product characteristics of the medicinal product involved expressly provides for the possibility to administer the drug at the patient’s domicile.

Pharmacovigilance good practice

When designing a PSP, it is essential to provide for the implementation of activities aimed at identifying, evaluating and preventing adverse effects or any other problem related to the use of the medicinal product (such as pharmacovigilance obligations).

The EMA’s good practice guidelines for pharmacovigilance addresses legal requirements applicable to MAHs for the management and reporting of safety data arising in PSPs that involve direct interactions with patients and caregivers for the purpose of helping to manage a patient’s medication and disease outcomes (for example, adherence, awareness and education) or providing HCPs with support for their patients.

When operating PSPs, MAHs can collect information relating to the use of its medicinal products, patients and carers and can report events that occurred simultaneously with the use of medicinal product and where there could be suspected adverse reactions.

The EMA’s guidelines specify that reports of serious or non-serious cases of adverse reactions originating within the PSP are considered as “solicited”; therefore, they must have a level of completeness in order to allow the MAH to decide on any reporting to the competent authorities. The pharmaceutical company will have to design the detection system with adequate diligence by providing, for example, a follow-up phase in the event that it deems that the report is not exhaustive.

If the PSP is carried out directly by a service provider, it would be important to identify the pharmacovigilance obligations and responsibilities in the master service agreement,  such as  indicating the modalities and timing for the exchange of information between the provider and the MAH.

Digital regulation

Regulatory intervention on PSPs is expected to increase in the near future and provide more guidance on the development and implementation of these programmes.

In particular, there is a need to regulate PSPs that involve the use of digital systems and products, which have become more common due to the Covid-19 emergency and the need to provide patients with remote services and maintain their involvement remotely. PSPs are using digital tools more frequently, such as mobile phone applications or devices connected to the network. For example, apps can allows patients to receive information and video-audio indications on their therapy, enter useful data for the management of the therapeutic path, and interact with the doctor or caregiver. 

The debate around the regulation of digital care and digital therapeutics products is shaping the development of PSPs and will involve – at least, for now – the need for a case-by-case assessment to ascertain if the medical device used qualifies under the new Medical Device Regulation and the guidelines of the European Commission MEDDEV 2.1/6.