Patient access programmes/patient assistance programmes: legal benefits and challenges for patients

Dovydas Gudžiūnas
Sorainen, Vilnius
dovydas.gudziunas@sorainen.com

A definition of PSPs

A general and the most popular understanding is that PSPs are a variety of actions that aim to provide solutions to patients beyond the medication itself. Overall, the main goal of a PSP is to increase the quality of the patient’s life by enhancing treatment adherence, improving clinical outcomes and facilitating the patient’s experience during their treatment.^[1]

The European Medicines Agency (EMA) describes PSPs in a narrower way. According to the EMA, a PSP ‘is an organised system where a marketing authorisation holder receives and collects information relating to the use of its medicinal products’. Examples of such programmes include post-authorisation patient support and disease management programmes, surveys of patients and healthcare professionals (HCPs), information gathering on patient compliance and compensation and reimbursement schemes.^[2]

Regardless of the theoretical definition, most PSPs share a common goal related to the need to increase the quality of the patient’s life and can be attributed to the one of the following categories:^[3]

• adherence PSP, which help patients to take their medications as prescribed and provide them with the necessary support;
• education PSPs, which help patients to learn about and understand their condition and provide general advice on how to manage their disease more effectively; and
• patient access programmes, which provide financial assistance or reimbursement in regard to the medicines required by patients.

Key principles of the regulatory framework and current practices

PSPs are relatively new; therefore, there are no general laws and regulations uniformly governing PSPs.^[4] In the majority of countries, PSPs fall into the so-called grey zone and are neither directly permitted nor prohibited. In such cases, PSPs are regulated in a fragmented way by other regulatory authorities, in regard to areas of law such as advertising, data protection, pharmacovigilance and anti-bribery.

Nevertheless, some specific principles of the regulatory framework for PSPs can be indicated that reflect current practices. At the outset, PSPs must be of a non-commercial nature. To achieve this, PSPs must be organised through HCPs/healthcare organisations (HCOs) and have obtained  their approval. Naturally, no advertising is allowed to take place in regard to PSPs. It is also important that the entire PSP implementation process  is transparent and the activities carried out by PSPs are well-documented and they are included in the company’s pharmacovigilance plan. The transparency principle also mandates that patients are provided with sufficient information, which is needed for the patient to decide whether to participate in the programme and, in the case of a positive decision, explicit consent must be obtained from the patient.^[5] In addition to the material requirements, PSPs are also subject to certain administrative rules that are important to be followed while implementing these programmes. For example, in regard to a PSP being carried out not by a pharmaceutical company, but by a third-party service provider, it must be ensured that a proper agreement is in place between these two entities. In such cases, special attention must be paid to employment laws to ensure that illegal staff leasing is avoided.^[6]

Even though the abovementioned principles represent a general regulatory framework for PSPs and reflect current market practices, some local industry initiatives foresee additional requirements for PSPs. In Canada, for instance, a local code of conduct states that PSPs must have clear objectives, timelines and scope, whereas in Australia, PSP providers must ensure that the clinical rationale for these programmes is documented.^[7] In conclusion, even though there are some general rules for the implementation of PSPs, currently the market lacks a clear legal framework governing PSPs.

Legal challenges for PSPs

Even though PSPs undoubtedly create enormous benefit for patients, HCPs and carers, PSPs face regulatory challenges, ranging from legal and compliance aspects to bioethical concerns. The key legal challenges for PSPs are presented and analysed below.

Transparency and accountability issues  

As a rule, PSPs are carried out with the help of HCOs and HCPs, who not only provide the required infrastructure and human resources for the implementation of such programmes, but most importantly they ensure that objectivity and neutrality is maintained in regard to the patients’ medical treatments. Therefore, only the administration related to PSPs, and not the original tasks carried out by an HCP (such as the patients’ enrolment in a PSP), should be carried out by pharmaceutical companies.^[8] Bearing this in mind, any interference between the patient and the HCP could place the medical treatment’s objectivity at stake. Naturally, the question arises as to whether pharmaceutical companies fully comply with these transparency principles.

When it comes to the HCP’s role in PSPs, it is important to ensure that during the entire lifecycle of the PSP that the HCP remains objective and neutral towards the patient’s treatment and eligibility for inclusion in the programme, otherwise it is impossible to guarantee the non-commercial nature of PSPs. It is evident that HCPs receive remuneration for their work in regard to PSPs. Naturally, transparency principles require that such remuneration reflects fair market value, is properly disclosed to the public and in no circumstances constitutes an inducement to recommend, prescribe, purchase, supply, sell or administer medicines.^[9] However, the current regulatory landscape does not impose an obligation in regard to the disclosure of PSP and related materials to the public. Without a disclosure obligation, it is hard to ensure the effective control of PSP implementation, which in turn leads to questions about accountability. The present findings confirm that transparency and accountability remain relevant matters in regards to the implementation of PSPs.

Personal data processing issues

One of the key requirements, relevant at the international level, is to obtain explicit and informed patient consent to process their personal data (PD) in regard to the activities carried out by PSPs.^[10] Before giving consent, patients must be informed about all the ways (including commercial) in which their PD will be processed and the grounds for PD processing.^[11] Even though this requirement is clear, its implementation is cumbersome. The main challenge arises when an industry needs to find the right balance between providing enough information to secure informed consent (as required by legal acts), while ensuring that plain language is used, as well as making sure that the language is simple and concise.

At the local level, PD processing issues vary depending on the jurisdiction. For example, in Italy, it is a requirement that third-party service providers are engaged for direct PD collection in regard to PSPs and pharmaceutical companies are only allowed to access aggregated PD in this context.^[12] This requirement helps to ensure that PSPs remain of a non-commercial nature and provides reliable protection in regard t patients’ PD. However, at the same time, while pharmaceutical companies remain responsible for the creation and implementation of such programmes, from the PD protection perspective, this requirement can be seen as preventing pharmaceutical companies from acting as data controllers in regard to the PD being processed as a result of PSP.^[13] Therefore, further legal guidance is required to fully and logically deal with these PD processing issues in the context of the activities carried out by PSPs.

The collection of real-world data

Since PSPs provide detailed health information on large patient populations, these programmes have become an effective way to collect real-world data (RWD). Compared to other RWD collection methods, PSPs can be tailored to the specific clinical needs of patients and, thus, they provide flexibility to pharmaceutical companies in regard to RWD collection.^[14] However, certain limitations in regard to data collected by PSPs raise concerns about whether this data is reliable and whether it can be trusted.^[15]

PSPs are not designed to investigate a scientific hypothesis, nor are they governed by any protocols, meaning that these programmes are not safety oriented.^[16] For example, RWD collected by PSPs does not provide comparative analysis in regard to the same dataset, as PSPs usually contain only one specific medicinal product per programme .^[18] One of the greatest disadvantages of RWD generated through PSPs is that in most cases such data is viewed as biased, since it is generated by private companies and without clear legal guidance.^[18] Examples of biased RWD include cases when data is collected from a small number of healthcare centres or in limited catchment areas.^[19] Cherry picking with regards to RWD is also recognised as a data reliability concern.^[20] Justified or not, these concerns reduce stakeholder trust in RWD collected via PSPs.

Even though there have been attempts to regulate the collection of RWD by PSPs,^[21] such initiatives are either not mandatory or very generic. Over the past few decades, the lack of proper guidance has led to a wide range of operational standards being applied in regard to RWD collection processes.^[22] Naturally, this raises a question about whether RWD collected during the operation of PSPs can be seen as reliable. To increase trust in RWD and make the relevant processes more uniform across the industry, some countries have adopted local guidance for RWD collection by PSPs. Even though these guidelines do not guarantee that RWD will be trusted by stakeholders, they set more general standards for such data, which helps to increase consistency and uniformity. A good example of such guidance is the real-world evidence framework by NICE in the UK, which provides in-depth guidance and tools for RWD collection.^[23]

Therefore, even though PSPs have the potential to generate quality RWD, there is still room for improvement, and both regulators and the industry have important roles to play in this regard. Specifically, regulators need to adopt a legal framework for reliable RWD collection by PSPs, whereas the industry should increase the use of transparent, qualified and reliable standards for verifiable RWD collection.

Conclusion

Although there is no uniform definition of a PSP, and such programmes cover a variety of different activities, starting from patient education and ending with financial support schemes, the common goal of all PSPs is to create tangible benefits for patients.  

Nevertheless, PSPs face legal challenges and, without appropriate solutions, these challenges could raise serious questions about the future of PSPs and their value, reducing patients’ overall trust in the pharmaceuticals industry. The primary issue that persists is the lack of a legal framework, which would govern PSPs and provide key legal principles for the implementation of these programmes. Even though there have been some attempts to regulate PSPs, such attempts have failed to ensure a consistent legal regime for these programmes (either because the laws are fragmented, or they are in the form of non-mandatory industry codes). Subsequently, the lack of clear guidance creates other issues related to transparency, accountability and personal data protection. Therefore, there are high hopes that regulators will adopt a proper legal framework for PSPs, which would serve as a remedy to the aforementioned issues.

In recent years PSPs have also become an effective tool for RWD collection due to certain advantages, such as flexibility, that PSPs can offer to the industry. However, a certain level of mistrust exists towards such RWD, which is mainly influenced by the lack of standardised RWD collection methods and a lack of mechanisms to ensure that such data is not biased. Therefore, once again, regulators need to take an active approach and adopt a legal framework for RWD collection by PSPs, which ensures that such RWD is trusted and used effectively.

Regardless of the legal challenges, PSPs remain an effective tool to bring healthcare closer to the patient. Through ongoing dialogue, innovation and a commitment to patient welfare, stakeholders can unlock the full potential of PSPs to transform healthcare delivery beyond the pill and improve patient outcomes for future generations.

Notes