Patients who have run out of standard treatment options may get early access to investigational drugs. This project determines the ethical and societal conditions for a responsible design of services to facilitate early access.

This project maps the associated ethical and societal issues to assist policy makers in dealing with this new development and to help shape a socio-ethical design and set-up of the services envisioned by myTomorrows and other public or private initiatives to facilitate early access. The ethical framework will assist policy makers, physicians and pharmaceutical companies in handling that demand in a responsible, consistent and equitable way. Moreover it will critically evaluate and help shape an ethical design and set-up of the services envisioned by myTomorrows.

With the increasing popularity of the Right-to-Try movement in the US and the rise of commercial service providers in the Netherlands and elsewhere, it is imaginable that in Europe, as well, patients will increase pressure on pharmaceutical companies to obtain earlier access to new drugs. National healthcare systems allow for individual use of (so far) unapproved drugs in patients who cannot participate in clinical trials. Initiatives such as the company myTomorrows aim to make these medicines more accessible to patients and their physicians.

Ethical and societal issues

In this project the researchers will develop an ethical, practical framework for a responsible set-up of such services. Ethical and societal issues include:

• Potential benefits and risks for individuals patients;
• Shifting responsibilities among physicians, pharmaceutical companies and payers;
• Equal access to innovative new drugs;
• Potential adverse effects on current systems for drug research and development.

A key ethical issue identified by the researchers is that services like myTomorrows will have to gather outcome data as part of any expanded access program. Although investigational drugs are prescribed with a therapeutic aim, monitoring the clinical outcomes can contribute to more knowledge about the drugs’ ability to address unmet medical needs and their safety profiles. This might even expedite the registration process and help future generations of patients. However, it is difficult to envisage what such real-world data collection should look like (e.g. in the form of patient registries), given the existence of strict regulations for the gathering of research data and privacy concerns.

Obstacles

The research team has established that the most prominent obstacles to expanded access are:

• Low level of knowledge and experience among doctors and patients;
• Extra workload for health care professionals when making arrangements for expanded access;
• Unwillingness or lack of capacity on the part of the pharmaceutical company to supply the drug;
• Lack of appropriate financial arrangements.

Differences between countries

There are, however, large differences between the three countries studied in this project: the Netherlands, the USA and Turkey:

• In the Netherlands and the USA the use of early-phase investigational drugs is highly exceptional and rare. Not all medical specialists are aware of the options for early access and many do not have any experience with the process. Moreover, not all physicians have positive views on the use of unapproved drugs: it would not be evidence-based and it would foster false hope in patients who are seriously or terminally ill.
• In Turkey, on the other hand, physicians discuss options for expanded access with their patients by default, when standard treatment options have been exhausted. Some doctors prepare requests for expanded access on a daily or weekly basis.

The three countries also differ regarding the financial arrangements for early access:

• In the US pharmaceutical companies are allowed to charge only the direct costs (i.e. the production costs) of an investigational drug. However, most companies are not willing to disclose this information. They therefore prefer to supply the drug either at no cost to the patient, or - most likely, as many companies do not have the means or the production capacity to do so - not at all.
• In the Netherlands the use of investigational drugs is not reimbursed by health insurers and it is generally not accepted for patients to pay for their health services out of pocket.
• In Turkey a system of routine reimbursement of expanded access has been put in place by the state.

About myTomorrows

The new online platform myTomorrows connects drug developers to patients and their physicians worldwide to facilitate early access to unapproved investigational drugs. It is the first for-profit initiative seeking to make the existing regulatory routes of compassionate use and named-patients programmes more accessible to patients and their treating physicians. It does so by informing patients and physicians about opportunities for early access, handling the paper work – as a service for doctors – and helping to develop new models for funding early access and gathering data on its outcomes.

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