Anavex Life Sciences Corp (AVXL)

[sokol]

SAS designation of Blarcamesine in Australia is a smart move. Will early access be expanded to Europe? See the below excerpts from 3 articles with references.

1.Early Access Programs
Governments worldwide have created provisions for granting access to drugs prior to approval for patients who have exhausted all alternative treatment options and do not match clinical trial entry criteria, these are so called Early Access Programmes (EAPs) or labelled Compassionate Use Schemes (CUSs). Some markets regulation allows patients to access drugs that are approved outside of the region, but not yet in their home countries. EAPs are governed by guidelines and legislation that vary by country, defining access criteria, data collection, supply and control of the drug distribution. Some countries (e.g. Canada and Australia) have well defined EAP, Special Access Program (SAP) and Special Access Scheme (SAS), respectively. EAPs can be put in place at any stage of development post-phase II and can run in parallel with phase III clinical trials, until market authorisation is granted. Reporting data about efficacy, safety and occurrence of adverse events to the responsible health authority are usually mandatory requirements. Mostly it is the treating physician that is responsible for initiating the request, monitoring and reporting any output coming for the utilisation of the unauthorised drug (in clinical trials, it is the sponsors responsibility). Regulations differ widely among countries, due to differences in national medical practices, resources available, product funding, hospital structures and national insurance systems.

While patients, hospitals and/or national insurance systems bear the costs in some countries, the sponsor is expected to provide Compassionate Use products free of charge. An important consideration is that if a drug is charged for, then the obtained price may be used as future benchmark for pricing and reimbursement committees.

Conclusions

Advances have been made in fast tracking medicines to patients with unmet needs. We have described some evolution by the regulators to create accelerated routes and health authorities allowing patients access to experimental or unapproved medicines. The developer needs to carefully evaluate these options before embarking on any of these routes, all while providing advantages to patients where certain limitations could apply.

Likewise, the decision to implement an EAP should be carefully considered and a sponsor should ask important questions such as when to offer access and for which patients, as there might also be many drawbacks tied to its implementation. Existing regulations do not force companies to offer access to drugs prior to approval or launch.

In addition to providing significant benefit to patients with unmet needs, EAPs can offer important benefits in terms of increased and earlier access to the sponsoring manufacturer. EAPs can be a part of a global market access strategy, generating development strategies that are increasingly innovative and global in scope.

https://www.huronconsultinggroup.com/insights/early-access-medicines-for-unmet-needs


2.Early access programs in Europe: a regulatory tool with pre-marketing impact

EAPs offer real marketing rewards

EAPs are a good means of testing the product in "real life" and convincing prescribers and patients of product efficacy before launch. One advantage is that "real life" data are obtained, reflecting a more clinically and ethnically diverse population than often encountered in clinical trials. This information gained pre-launch can also shape post-launch marketing messages. "Early Adopters" or brand advocates can be identified as a wider group of physicians gain experience with the drug, and physician loyalty may be developed. Furthermore, key opinion leaders often play a major role in discussions with regulatory agencies.

Equally with respect to patients, as well as providing them with access to a potentially life saving medicine, there is the opportunity for feedback, the development of patient loyalty and building relationship with patient advocacy groups and associations.

There is the potential for early revenues in countries where EAPs can be designed as "for-profit" programs, but whether or not a drug is made available free of charge, market penetration can be maximized pre-launch through an EAP, which can then translate to a successful launch and increased post-approval usage. In practice, a good rule of thumb is that first-year market penetration after employing an EAP strategy is equivalent to the second year market penetration of a regular launch.

Even when EAPs run at a loss or neutral returns in stand-alone financial terms, long-term benefits make them an attractive choice for companies. In fact, patient recruitment is generally faster for not-for-profit EAPs, so if these patients keep using the drug when it is sold post-launch, a not-for-profit scenario could in fact provide the most benefit to pharma/biotech firms.

3.Expanded access or compassionate use is the use of an unapproved drug or medical device under special forms of investigational new drug applications (IND) or IDE application for devices, outside of a clinical trial, by people with serious or life-threatening conditions who do not meet the enrollment criteria for the clinical trial in progress.

These programs go under various names, including early access, special access, or managed access program, compassionate use, compassionate access, named-patient access, temporary authorization for use, cohort access, and pre-approval access.

Europe
In Europe, the European Medicines Agency issued guidelines that members may follow. Each country has its own regulations, and they vary. In the UK, for example, the program is called "early access to medicine scheme" or EAMS and was established in 2014. If a company that wants to provide a drug under EAMS, it must submit its Phase I data to the Medicines and Healthcare products Regulatory Agency and apply for what is called a "promising innovative medicine" (PIM) designation. If that designation is approved, the data is reviewed, if that review is positive, the National Health Service is obligated to pay for people who fit the criteria to have access to the drug. As of 2016, governments also paid for early access to drugs in Austria, Germany, Greece, and Spain.

Companies sometimes make use of expanded programs in Europe even after they receive EMA approval to market a drug, because drugs also must go through regulatory processes in each member state, and in some countries this process can take nearly a year; companies can start making sales earlier under these programs.

https://en.m.wikipedia.org/wiki/Expanded_access