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European Pharmaceutical Contractor

Why Provide Early Market Access?

Simon Estcourt of Idis reflects on the implications and solutions posed by early market access and the importance of ethical communication

The growing focus on controlled access to pre-approved therapies is a reflection of patients’, physicians’ and the industry’s increased awareness of the important role that pharmaceutical and biotechnology companies can play in the management of access to their medicines for patients with unmet medical needs, often through ethical and regulated frameworks such as named patient programmes (NPPs).

NPPs are controlled and restricted access programmes set up by pharmaceutical and biotechnology companies, under which physicians and pharmacists can gain access to investigational therapies on a ‘named’ patient basis. They can provide access to medicines which are unavailable to patients for a variety of reasons: drugs may still be in clinical development; they may have medicinal value for a very small population (such as orphan drugs) but may never be approved; or they could be approved in one country but unavailable or discontinued in another.

GROWING ATTENTION ON ACCESS

Towards the end of last year, patient access to investigational drugs was the topic of a forum held in Washington, DC, where representatives from the US Food and Drug Administration (FDA), the pharmaceutical industry, advocacy groups, the medical profession and the bioethics community gathered to share their perspectives. It was clear from the forum that each situation in which early market access may be leveraged has its own unique dynamic and requires thoughtful consideration of patient needs, the company’s situation and regulatory guidelines. While the new FDA regulations provide an effective framework for access, many questions remain, including:

  • When should access be allowed – after Phase I trials when safety has been established or later in the trial process when efficacy data is available?
  • Who should get access? Must a patient be terminally ill? How do we define ‘terminally ill’?
  • In the case of a cancer drug, must the patient seeking access have the type of cancer the drug is being tested against in trials? What is considered ‘fair’when choosing which patients can get access and which cannot?
  • How do we appropriately balance the needs of desperately ill patients with safety concerns?
  • How do we ensure expanded access does not put clinical trial enrolment at risk?

Ultimately, the choice to offer expanded access – or not – is left up to the drug developer. However, for patients and their physicians the benefits are clear. NPPs (also known as expanded access programmes in the US) offer new, potentially life-saving treatment options when no other alternative is available – potentially providing one more chance at better treatment options or, in some cases, extending life. It also offers physicians the opportunity to utilise new medicines and stay at the forefront of medical advances. For pharmaceutical and biotechnology companies, NPPs can help to fulfil their vision of helping patients in need. They can also help address demand and build a network of physicians and knowledge about who, where, and in which patient populations the drug can be used effectively.

Global access management solutions delivered via the mechanism of NPPs provide pharmaceutical and biotechnology companies a controlled and ethical option for the provision of medicines for patients who need them the most. They provide both industry and physicians with information that will help them to understand the impact of a new drug, enabling more informed strategic decisions and pre-launch/pre-approval marketing plans, including the importance of appropriate health economic data and reimbursement options.

THE REGULATORY MAZE

Regulatory bodies cannot force companies to provide access. However, there are strict regulations in place to ensure companies do not use NPPs to market unapproved drugs and that there is ethical communication around such programmes.

Although drug approval is centralised via the European Medicines Agency (EMA), the drug reimbursement system is decentralised. Decisions regarding reimbursement take place within each respective EU country. This process often results in a delay in commercial launch as some countries can take between 12 and 18 months to establish reimbursement following EMA approval of a drug.

A report on patient access to cancer drugs in Europe found that the average time delay between marketing authorisation and effective market access over a three-year period varied significantly from country to country. For example, the average delay in France was 326 days. Other large European markets were demonstrating similar time delays with Spain taking an average of 282 days and Italy 335 days. From 20 new products approved by the EMA centralised procedure included in the report, the mean time for effective market access was a staggering 429 days (ranging from 224 to 739 days) (1).

What this may mean for patients is an excruciating wait for access to key medicines, which can potentially mean the difference between life and death for some patients requiring urgent medical attention. Understandably, patients may seek out alternative sources or suppliers of the medication. In a worst case scenario, this may mean exposure to counterfeit drugs through unreliable supply chains. In a bestcase scenario, patients will talk to their physician and become a ‘named’ patient in a named patient or expanded access programme, which will guarantee them a legitimate way of access and ensure that they receive their drug safely and reliably for as long as required.

National programmes regulating early access to medicines vary widely from one EU member state to another due to differences in national medical practices, resources available, funding of the project, hospital structures and national insurance systems. The terminology also differs but, broadly speaking, the programmes fall into one of two categories: either case-by-case situations known as NPPs, named patient supply or nominative importation, or authorisations for larger patient groups (or cohorts of patients). These are known as temporary authorisation for use, collective prescription, or general license.

Avenues for early access 

Named patient programmes (NPPs) can provide access at a number of stages throughout a product’s lifecycle:

  • During Phase III, for patients with an unmet medical need who do not meet a clinical trial’s inclusion criteria
  • Bridging the gap between the end of Phase III and receipt of marketing authorisation
  • Bridging the gap between approval and commercial launch
  • Throughout a staggered global launch while approval and reimbursement is being sought across countries worldwide

NPPs also provide an alternative route to patient access as part of a global commercialisation strategy where:

  • A formal launch is not planned
  • A sales infrastructure does not exist or is not practical
  • It is not commercially viable to seek marketing authorisation

KEY CONSIDERATIONS FOR
PROVIDING EARLY ACCESS

Companies must undertake a thorough evaluation of important questions, such as when to offer access and for which patients. Companies should also be mindful that the focus on early market access is likely to intensify and result in an increased number of requests for investigational drugs. The trend towards greater transparency of drug development pipelines and the accessibility of powerful social media tools has led to a more informed, empowered and vocal population of patients.

ADDRESSING THE UNMET NEED

Today, advocacy groups, patients and their physicians play an increasing role in gaining earlier access to pre-launch medicines. There is a growing demand for innovative treatments especially in oncology, infectious diseases and orphan diseases where patients have few therapeutic options or when medicines have shown significant efficacy in the early stages of drug development. Pharmaceutical and biotechnology companies are increasingly meeting these demands when appropriate by initiating NPPs as early as possible in order to establish access channels prior to launch for patients who have no alternative treatment options.

There is no doubt that the early access debate will continue to have broad implications for all those involved. As patients become more determined in their efforts to seek access, open dialogue and direct confrontation of the issues surrounding this topic must take place among a broad range of stakeholders including patient advocacy groups, physicians, pharmaceutical and biotechnology companies, regulatory bodies and policymakers. The objective must remain clear: defining and facilitating responsible, controlled access to investigational drugs in a manner that effectively balances the needs of all participants while best serving the needs of desperately ill patients.

Reference

  1. Wilking N et al, Comparator report on patient access to cancer drugs in Europe, 15 February 2009, http://www.comparatorreports.se/Comparator%20report%20on%20patient%20access%20to%20cancer%20drugs%20in%20Europe%2015%20feb%2009.pdf, accessed 14 July 2010

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Simon Estcourt is a Senior Vice President and Director at Idis, a leading consultancy firm that partners with pharmaceutical and biotechnology companies throughout the lifecycle of a medicine. Simon oversees a global team of business developers in the field of pre-launch access to medicines. His previous experience includes six years with Quintiles Transnational, where he worked in operational and business development roles, and in strategic partnerships with pharmaceutical and biotech companies to develop and implement successful outsourced commercialisation solutions. Prior to Quintiles, Simon spent eight years at Servier Laboratories in various commercial roles, ultimately heading up the UK sales force. Simon started his career at IBM in a specialist sales role after obtaining a Business and Politics degree from Aston University Business School, UK.
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