UK patients may benefit later this year from a new scheme granting early access to certain new medicines prior to formal approval. But one stumbling block is that the government will not provide funding for drugs used under the program.
Proposals for this Early Access Scheme were put forward late in 2009 by a working group co-chaired by the UK regulatory authority, the Medicines and Healthcare Products Regulatory Agency (MHRA), and the Association of the British Pharmaceutical Industries (ABPI). The proposals are now the subject of a 12-week public consultation.
The idea is simple: to better meet public health needs by allowing patients without treatment options to receive drugs in development that potentially could prolong their life, in advance of licensing approval.
As such, it's similar to FDA's Treatment IND regulation, which permits use of an investigational drug among some patients, granted preliminary evidence of efficacy exists and that the drug is for a serious or life-threatening disease, and patients are not eligible for clinical trials in progress.
One of Only a Few Formal, Pre-Approval Programs
Most countries, including the UK, have compassionate use programs permitting patients to obtain investigational drugs on an individual, named-patient basis. But only a few - including the U.S., France and Sweden - have programs to allow cohorts of patients, rather than individuals, to benefit from early access, and include a review process, albeit a curtailed one.
The proposed UK scheme will likewise "consider cohorts of patients, rather than individuals," explained Ian Hudson, MD, Director of Licensing at MHRA and a member of the working group that drew up the proposed Early Access framework.
The scheme also will contain compassionate-use type provisions, Hudson continued, targeting for example drugs that are highly promising, and treat, diagnose or prevent life-threatening, chronic or seriously debilitating illnesses associated with very significant morbidity.
Still, given that drugs eligible for the program should also represent a "significant advance in treatment in an area of unmet need," there aren't likely to be many authorizations under the scheme, according to the report outlining the proposals - just one or two per year. And it's not as if the access scheme will stretch that far upstream from formal approval anyway: eligible medicines should have completed Phase III trials and shown a positive benefit/risk profile, although "we didn't close the door to earlier approvals," Hudson clarified.
As such, this small handful of drugs is only likely to benefit from an early access scheme for a matter of months until formal approval is granted (or not).
The Cost Problem: Return To Payment Lottery?
Hudson is confident that many logistical details associated with the scheme will be relatively simple and quick to sort out. These include the MHRA review process, forecast to take no more than 75 days with one round of questions to applicants, data requirements for applications and information provision to local Primary Care Trusts that cover health care.
But the question of who pays for the drugs remains a thorny one, likely to be the subject of continued debate as well as a further limit on how widely drugs might be accessed under the program.
As now drafted, the proposals state that while industry will enjoy freedom of pricing for medicines supplied under the scheme, no additional government funding will be available for such products. That effectively leaves it up to individual PCTs to find the money to pay for such drugs. They will not be mandated to do so, however, as they are with products approved by the UK's cost-effectiveness watchdog, the National Institute of Clinical Excellence.
All of which spells a potential return to the reimbursement "lottery system" that NICE was set up a decade ago to eliminate. Before, a patient's access to a particular drug had depended on where he or she lived and local health care resources.
The funding issue "is the only real negative" associated with the scheme, noted working group member Matthew Speers, Managing Director, UK & Ireland, for UCB.
Overall, it's "a very good starting point," he said, adding that UCB and other, bigger pharmaceutical firms certainly will be looking at how they can use the program for various pipeline molecules. "But it isn't funded as industry was looking for." (There are other, existing mechanisms for companies to make drugs available, free of charge, to patients pre-approval, so companies are not about to offer to fund this program as well.)
A recent change to the French early access program, ATU (authorization temporaire d'utilization), which is social security-funded, requires that sponsor companies refund the difference between the drug price paid during temporary pre-approval period, and the price granted after approval, if the former is higher. This approach has not been proposed for the UK scheme, according to Hudson.
The UK proposal document acknowledges this potential problem - magnified in the current budgetary climate. But discussion in the report that the scheme will be limited to just a few medicines showing significant benefit, have been subject to a form of review and likely will be available under the scheme only temporarily is unlikely to assuage PCT managers responsible for that year's budget.
Inspiration From France
To use the early access program, sponsor companies will have to commit to submitting a formal application for their product within a defined time-frame that's "likely to be about a year," Hudson said in an interview.
This requirement for a formal licensing submission is one example of how the UK proposals have been inspired by France's ATU program.
One of the few formalized early access schemes in Europe, the ATU system has been running for 15 years and includes both a named-patient category (ATU nominative) and a patient-cohort category (ATU de cohorte). "Our proposals are indeed very similar to the French system, which we looked at very carefully," said Hudson.
According to Chantal Belorgey, Director of Pre-Licensing activities at France's drug regulatory agency AFSSAPS, the system remains popular even though the number of cohort ATUs being granted per year has been dropping since 2007. The agency granted just five in 2008 compared with 14 the year before. (The number of new drugs evaluated for named-patient ATUs, in contrast, rose from 55 to 64 over the same period.)
Belorgey attributes this to the higher administrative and data requirement burden associated with cohort ATUs, which are mainly granted only to end-of-Phase III products, and to the overall decline in the number of innovative drugs emerging from labs. AFSSAPS is currently trying to encourage companies to apply for cohort ATUs.
Early Access Schemes Remain National
UK venture capital veteran and businessman Sir David Cooksey had put forth the original scheme that eventually led to the UK proposals. He had suggested a European-wide and even global framework for earlier access to medicines. So far that isn't happening. "This is a national scheme, it's not being put in place on behalf of Europe," clarified Hudson. Likewise, ATU is independent of the European-level drug regulatory system.
Indeed, although there is scope within European legislation for the European Medicines Agency to issue an opinion on compassionate use, it has yet to do so. But the first cases in this area are under discussion, according to an EMEA spokeswoman, and the first EMEA opinion on compassionate use is expected "shortly." It's unclear what influence EMEA opinion will have on member states' decisions.
Meanwhile, another version of early access in the UK was initiated last year as part of the country's broader push towards promoting innovation and access. The Innovation Pass, to be implemented by NICE, will grant reimbursement for up to three years to certain highly novel drugs with insufficient data for a regular NICE approval (1 'The Pink Sheet,' Sept. 15, 2009).
The early access scheme unveiled in December "doesn't relate so directly to innovation," explained Hudson (although eligible drugs are more than likely to show some degree of innovation), "but is instead primarily driven by meeting a public health need."
- Melanie Senior ( 3 email@example.com )
This article also appeared in "The Pink Sheet" – Jan 18, 2010
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