How early access strategies affect market access
Peter Mansell explains why early access programs may become integral parts of comprehensive market access platforms
By on Oct 18, 2010Peter Mansell explains why early access programs may become integral parts of comprehensive market access platforms
Market access strategies usually focus on what happens at or around approval, launch and beyond, even if the planning process now has to start as early as possible in the drug discovery and development cycle.
But there is a way of giving patients access to new medicines before they hit the market, one that reflects increasing awareness of new therapeutic possibilities and the kind of pre-launch demand that spells headaches for agencies such as the UKs National Institute for Health and Clinical Excellence.
This is through named patient or, in the US context, expanded access programs.
They are distinct from clinical trials, although they may provide an alternative access route for patients who cannot take part in a trial.
Comprehensive market access
Early access programs can be set up in response to an unsolicited doctors request (Europe) or they may be a proactive initiative by the product sponsor (US).
The procedures vary from market to market, even within a nominally harmonized legal framework across the European Union.
Besides a straightforward compassionate-use rationale i.e., existing medicines fail to meet urgent patient need one argument for exploiting early access provisions is to discourage patients from resorting to unregulated channels, such as the Internet, with its growing risk of counterfeits, in cases where a product is already available in other markets.
At the same time, a procedure geared to exceptional circumstances can have ancillary benefits for the product sponsor, such as testing the ground for price points, educating doctors on appropriate product use, or identifying potential key opinion leaders.
In this respect, early access may increasingly come to represent an integral part of an ever more complex and comprehensive market access platform.
There are a number of situations in which named patient or expanded access programs may apply.
The product concerned may be stalled between marketing approval and launch, as the sponsor negotiates pricing and reimbursement approval.
Alternatively, it could be an orphan drug with limited commercial viability in certain markets.
Patients may be denied access due to exclusion criteria for clinical trials, or they may want to stay on the therapy once a trial has ended.
The overriding criterion is unmet need, taking into account what is already available locally or through clinical trials.
Drivers of early access
The scale of expanded access programs may range from single patients to the thousands served by US expanded access schemes in areas of high unmet need.
And demand is substantial.
UK-based strategic market access consultancy Idis says it dealt with some half a million requests for medicines last year, providing access to products in over 140 countries.
Speaking at eyeforpharmas Oncology Market Access Europe 2010 Summit in London recently, Mark Corbett, European account director for Idis, suggested an early access program should really be considered for any new drug coming to market.
Moreover, companies should be thinking about putting a scheme in place by the time their product is in Phase III trials.
Corbett ran through some of the drivers that are nudging early access programs towards the mainstream.
Top of the list is patients, who with the help of the Internet and social networking tools, are better informed and more vocal about drugs in development than ever before.
The globalization of the medical community has a similar effect.
As Corbett pointed out, 53% of attendees at last years annual meeting of the American Society of Clinical Oncology were from outside the US.
From the other side of the fence, increasing market access barriersbe they payer restrictions, health technology assessments (HTAs) or long delays for pricing and reimbursement approvalare narrowing the window of opportunity to reach patients through conventional means.
Pricing and reimbursement
Of course, early access programs are not without their complications, which is where consultants like Idis come in.
Apart from the regulatory and logistical challenges, there are considerations such as pricing and reimbursement.
According to Simon Estcourt, Idis global business director, with 75-80% of programs the company will charge for the drug.
Reimbursement may be available but sometimes the patient will have to pay out of pocket.
Supplying a drug free of charge may be ethically commendable and good PR, but it risks creating patient expectations that cannot be fulfilled once the product is out in the marketplace.
By a similar token, pricing for early access needs to be handled with care.
On the other hand, it can help to establish a precedent for the full-scale launch.
As Corbett noted, early access programs offer a tightly controlled supply mechanism that can also serve as a tool for market planning not only to gauge the responses of physicians and patients, educate on appropriate use and raise visibility pre-launch, but to illuminate patterns of demand.
Personalized medicine
Estcourt believes the shift towards personalized medicine will increase the need for market access solutions responsive to more uncertain patient demographics.
Targeted therapies will make for a much more limited marketplace, one characterized by pockets of demand, and you dont know where theyre coming from, he told the Institute of Clinical Researchs 31st Annual Conference in London.
In fact, Estcourt went further to suggest that early access programs can help pharmaceutical and biotechnology companies to innovate and change their model.
One way, he said, is by generating the kind of real-life patient data that will prove increasingly valuable as health technology assessors tighten their grip in the market access arena.
For more on HTAs and market access, see HTAs go global: What it means for market access.