Market Access Insight: Early Patient Access

Companies are increasingly receiving requests from physicians, KOLs and even patients throughout the world for early access to medicines that are still in clinical development.

For these patients, access to a drug or biologic outside the clinical trial or commercial setting can represent a new, and in many cases, life-saving treatment option.

This increasingly well-educated and empowered patient population coupled with greater transparency of pharmaceutical and biotechnology company pipelines is leading to patients and their advocates to seek access to unavailable medicines ever more frequently. Rather than waiting for them to be registered and launched in their country of origin, this group is increasingly looking for alternative routes to gain access.

This demand puts additional strain on an organisation’s existing resources that need to stay focused on progressing clinical development activities. Without an internal policy or framework for supporting early access requests, many challenges can arise.

Key challenges

• Defining an Appropriate Access Route
• Determining Access Criteria
• Identifying Qualified Resources
• Making Access Decisions
• Maintaining Stakeholder Relationships
• Forecasting Patient Demand

As a starting point, it is important that an organisation creates alignment of definition and terminology associated with these types of patient access programmes. Early Access, Named Patient, Compassionate Use, Expanded Access, Global Early Access are all used. Idis addresses this by using the umbrella term Managed Access Programmes (MAP) to define a patient access programme. It is paramount that there is a clear decision on the terminology that will be used within the internal organisation to ensure all stakeholders are aligned.

In order to design and implement a MAP, take time to identify key internal stakeholders who would need to have involvement and awareness of the programme.

This should be led by an internal programme sponsor or programme lead who would be responsible for deploying a core team of subject matter experts to help define the strategic goals and objectives for the programme. The relevant areas where stakeholders would be involved include; Clinical, Medical, Drug Safety, Contracts / Legal, Supply Chain, Quality Assurance, Procurement, Data Management, Finance, Compliance, Communications, Commercial, Marking Companies, Affiliates, Local Operating Companies, KOLs.

Of course, if the organisation is large, inclusion of global counterparts is essential. Working to harmonise across the organisation will ensure efficiency and alignment. In order to implement MAP, the strategic implementation framework below is an effective tool to ensure all processes relating to the programme are clearly defined and controlled.

Before the decision has been made to open up access via a MAP, it is also important to review overall early access considerations at a company-wide level.

• Company Structure – What are the entities that will be involved? What are the decision making processes?
• Corporate Vision – The organisation’s vision and strategic direction and is there a policy for managed access?
• Therapeutic Demand – Which therapy areas have or need to be identified where patient access will be a requirement?
• Corporate Policy – What are the internal policies and procedures and how will these need to be modified to include managed access?
• Risk Assessment – What are the risk benefits for these types of programmes?
• Corporate Governance – What governance processes are in place or will need to be determined for these programmes?
• Data Capture – What type of data capture / real world evidence would be insightful and useful?
• Global Regulations – Understanding of the regulations and compliance requirements for managing these programmes on a country by country basis.
• Stakeholder Engagement – Key stakeholder engagement and education. This would include KOLs, clinicians, the Local Operating Company (LOC), the marketing company (MC), the Regulatory Authorities, and the internal teams within the organisation.

At the programme implementation level, vital considerations include;

• Clinical Plan Alignment – Does the clinical development plan have any provision for a MAP to be implemented alongside trials?
• Lifecycle Access – Each stage of the development life cycle requires provision for a MAP – Phase II / Phase III / Ex-trial / Post-launch.
• Global Licensing – Is the drug globally unlicensed or licenced in one or more countries? This will impact the regulatory mechanism for access to be used.
• Patient Commitments – Are there clinical trials running / or ex-trial protocol commitments made to patients?
• Charging – Are you planning to charge for access and will there be changes to this policy after pricing and reimbursement decisions have been made?
• Treatment Duration – How long will a patient receive access?
• Drug Labelling – There are very specific requirements per country for supplying unlicensed drug therefore it is critical to ensure the drug is labelled to meet the regulatory requirements.
• Named Patient Regulations – Providing access via a MAP requires compliance with the named patient regulations. Each patient access request from a clinician requires competent authority approval to enable import of the drug. Each country has its own regulations for these types of programmes.

In summary, there are immense benefits for an organisation to define a clear strategy and prepare for implementation of an ethical and compliant MAP. The organisation will be able to respond quickly to a patient access request. All key stakeholders will be aligned in terms of their understanding and responsibilities.

Clearly defined policies and procedures for managed access will enable a standardised approach globally for companies to fulfill their social responsibility to help patients with unmet treatment needs, while protecting their clinical development programmes and corporate reputation.