1.                   Purpose

As a company active in the pharmaceutical industry, Arvelle Therapeutics International GmbH and Arvelle Group companies (hereinafter “Arvelle”) is determined to provide patients with the care and treatment they need and to develop new therapies that improve people’s lives. Rapid access to new potentially beneficial drug products may offer perspective to many patients.

Generally, Arvelle’s investigational therapies (therapies which are not yet approved by the relevant health authorities) are not available outside of clinical trials. Clinical trials are needed to generate the data required to evaluate investigational products’ safety and efficacy, and support regulatory approval by health authorities, ultimately resulting in new, approved medicines.

For this reason, Arvelle encourages patients to participate in clinical trials as much as possible, as clinical trials are critical in the scientific progress of healthcare all over the world.

However, Arvelle recognizes that for some patients participation to clinical trials is not possible. Additionally, investigational products are generally not available between the completion of clinical trials and regulatory approval or commercial availability. This is particularly challenging for patients for which no alternative or satisfactory treatment is available.

In the EU, there exist multiple mechanisms to provide investigational or unauthorized products before regulatory approval or commercial availability, or to grant access to unauthorized products to patients who cannot enroll in a clinical trial and for which all available treatment options have been exhausted or who have participated in a clinical trial which has ended and which would need continued access.  

Arvelle is committed to provide access to its investigational therapies in a manner that is consistent with its patient-focused values and compliant with all regulatory requirements.

2.                   General Information on early access mechanisms

Early access mechanisms take the form of specific programs such as “Compassionate Use”, “Expanded Access”, “Named Patient Supply”, “Special Access Schemes/Programs”, “Autorisations temporaires d’utilisation”, etc. (at Arvelle all referred to as “Managed Access Programmes” (MAPs)).

MAPs are governed by a variety of regulations and guidelines which differ from country to country. For country specific information on Arvelle’s existing MAPs in your jurisdiction, please contact Cenobamate@DurbinGlobal.com. MAPs can be either free of charge, or at certain prices set in accordance with applicable regulations.

In general, there exist two main forms of MAPs.

2.1.             Compassionate Use

Compassionate Use refers to programs that allow the use of an investigational medicine which is either undergoing clinical trials or which has entered the marketing authorization process to treat a group of patients who suffer from serious illnesses.

A company can make available its investigational products for compassionate reasons to a group of patients with a chronically or seriously debilitating disease or whose disease is considered to be life-threatening, and who cannot be treated satisfactorily by an authorized medicinal product.

Similar to clinical trials, Compassionate Use programs may have specific criteria that patients must meet to be included. Such programs also need to be approved by local health authorities.

If you have a query about Arvelle’s Compassionate Use programs, please contact Cenobamate@DurbinGlobal.com.

2.2.             Named patient access

A treatment may be approved and commercialized in one country but not available in the country of a (group of) specific patient(s). If a patient is located in a country where a drug product is not yet available and has a life-threatening illness, such patient may be able to access such drug products that are not available in its own country through a named patient program (NPP).

Access under an NPP requires an unsolicited request from the patient’s doctor formulated in accordance with his/her specification and for use by his or her individual patients on his or her direct personal responsibility.

This means that a doctor can prescribe a drug product which is not available in the patient’s country, but which is being commercialized in another country, after which such drug product can be specifically imported for the relevant patient.

If you have any query about potential access for named patients to Arvelle’s drug products, please contact Cenobamate@DurbinGlobal.com.

3.                   Arvelle’s criteria for establishing and discontinuing MAPs

Arvelle understands that early access to its investigational products can greatly improve patients’ lives. On the other hand, Arvelle needs to make sure that any access to its investigational products is provided ethically and fairly while minimizing the risks of harm to the individual patient and without jeopardizing the future broader access to the patient community. For that reason, decisions on such early access are of a very sensitive nature.

Therefore, Arvelle will consider each request carefully, but cannot guarantee that early access will be granted in any particular case. Whenever Arvelle receives a request for a MAP, it carefully considers the need and possibility for early access on the basis of clear pre-defined criteria.

  1. In order to qualify for an Arvelle MAP, the patient (i) must suffer from a chronically or seriously debilitating disease, and (ii) cannot be treated satisfactorily by an authorized medicinal product.
  2. Access to investigational products should generally be provided through clinical trials (as outlined above). Arvelle can only consider early access through MAPs if the patient does not qualify for enrolment in clinical trials.
  3. Arvelle places patients’ health and wellbeing first. It is important to remember that investigational medicinal products have not yet received regulatory approval. This means that early access to such products cannot be granted if there still exists insufficient clinical evidence of benefit from the use thereof, or if the treatment would pose or is anticipated to pose an unacceptable or excessive risk to the patient.
  4. Likewise, Arvelle can only consider requests from licensed doctors, as he/she has the expertise in treating the disease and understand the potential risks and benefits of the investigational medicinal product. 
  5. Arvelle can only commit to a MAP if sufficient supply of the required medicinal products to the patients enrolled in its clinical trials and MAPs is ensured. As a matter of principle, supply for clinical trials has priority, as clinical trials pave the way for regulatory approval and broad availability of the medicinal product. Arvelle shall also consider whether setting-up a MAP does not otherwise jeopardize the development program or delay regulatory approval of the investigational product.
  6. Arvelle can in principle only consider requests for MAPs relating to patients within the territory of Europe.
  7. MAPs in relation to Arvelle’s investigational products must at all time comply with the relevant (local and European) regulations.

Once marketing authorization is granted and the medicinal product is commercially available (and reimbursed by the local healthcare system), MAPs for such medicinal products will generally be discontinued, in compliance with local legislation and regulations, as the medicinal products can be accessed via the traditional channels.

If the development of any of its investigational medicinal products is discontinued e.g. because scientific evidence shows that the treatment is not sufficiently effective or safe, or regulatory approval is not granted by the relevant authorities and Arvelle determines not to further pursue regulatory approval, any MAPs for such medicinal products will be discontinued. In such event, Arvelle shall support patients and their physicians to help them safely transition to other treatments.

4.                   How physicians can access Arvelle’s MAPs?

Requests for access to unauthorized or not commercially available products must be made by the treating doctor of the specific patient.

For a consistent and efficient execution of its MAPs, Arvelle works closely with DURBIN PLC.

Please contact Cenobamate@DurbinGlobal.com for any requests relating to MAPs for Arvelle’s products.