Seriously ill patients who have exhausted all treatment options often seek access to investigational drugs by way of government-sanctioned “expanded access programs”. Recently, the U.S. Food and Drug Administration (FDA) implemented new regulations to clarify and increase patient access to investigational drugs.
However, even with the new rules, this topic is more complex than it may seem on the surface. And it has particular relevance to people with rare diseases since most rare diseases have no approved treatment.
To explore the various sides of this issue, NORD recently co-hosted (with Idis Pharmaceuticals) a media briefing on this topic. Both NORD and Idis are providers of expanded access programs.
Representatives of FDA, patient organizations, industry, and a university bioethics program spoke from their various perspectives. They raised some fascinating points in this ongoing discussion. Some of the things the speakers said were:
- People with rare diseases are highly motivated to try investigational drugs because they have few treatment options. In fact, there is no approved drug for most rare diseases today.
- FDA considers access to investigational drugs for treatment purposes important and supports it as long as it doesn’t compromise the clinical trial process and gives appropriate consideration to patient safety.
- Expanded access programs must be set up in a way that is fair and consistent for all patients.
- The expense of making drugs available through EAPs can be significant, especially for small start-up companies.
- Companies may be reluctant to enter into such programs because they may fear that an adverse event could jeopardize their product’s approval.
- Traffic rules, created for the general good, may be suspended in an emergency, such as when an ambulance is speeding someone to a hospital. Many rare diseases should be considered emergencies.
Thank you for sharing this, Peter.
As you say it’s really a challenging situation, where faced with sure and near end of life, the balance between harm and benefit for the patient is no longer such an important issue. Ethical issues still play an important part, though.
The expenses might be covered by other sponsors than the small companies, but I think the risk involved in a failure or – worse – in significant adverse reactions may be the main reason keeping companies from put aside their reluctancy.
Problems and uncertainty notwithstanding I think in a growing enviroment of drug experimentation the use of drugs still “in the pipe” should be always considered as a last option, with the possible exception of paediatric use.
Of course at least preliminary but carefully conducted safety/efficacy tests should have been performed.
Would minutes or a detailed report of the media briefing be available?
Thanks, Flavio
As a patient advocate for the umbrella organisation for cancer patient organisations I encountered s slightly differnet problem, but related to the investigational drugs. Cancer drugs are registered mostly in large indications first. At a later stage and further investigation they might prove usefull in other, rare cancer as well or just outside the registered indication. An example is e.g. Sutent (sunitinib) registered for renal cell cancer, but also showing efficacy in paraganglioma. This is a rare cancer and a registration will most likely never be obtained. Would that mean that this drug will remain experimental for ever in this indication? Is it off label use? Who will be paying for this? And who determines of patients get access to these type of off label or, if you want to call it like that, experimnetal – drugs – for – ever?
Does anyone know how they deal with this issue in their own country?
Pauline Evers
I am very interested to create a reference center for rare diseases and orphan drugs and need some help. Would you be so kind to inform how to start? Thanks in advance.
I think any patient should be given the drugs when they ask for them. I have Fahr’s disease and right now, there is no treatment. Paula
write your comment here…Now that I am a parent and mother living with PRS I feel that I would do exactly what my parents did explore every possible way including experimental treatments because that is the only option they had. Nobody wh0 is diagnosed with a terminal illness or even worse is walking around undiagnosed and without any medical advice or for that matter compassion.
Faced with having a undiagnose my parents had to contact almost every child specialist that specialized in various deseases. I took every test including biopsy’s to rule out what I didn’t have. One very well know doctor “Lamb” a pediatric specialist, made my parents and me take off our shoes and socks to examine our feet aand ankles. why he never said. Scleroderma was just getting much need public awareness and my aunt saw a Donahue talk show about this illness. My aunt quickly noticed that that I had similar symptoms and reguested a transcript from the show which aired in 1975.
My mother contacted the Scleroderma Foundation who sent her information but little hope. They needed donations to find any drugs to help this deadly desease that was shunned by pub;ic because they were scared they could “catch it”. At the time childhood Scleroderma was not a separate type of the disease that was not in most cases deadly, my mother never forgot the personal compassionate members who “listened and treated her with dignity” unlike mist of the medical community. When she passed away in 2006, her probate lawyer told me she donated 20,000.00 dollars every year for over 35 years to the Scleroderma Foundation.
I feel it is the right of the family involved to make the choice about experimental drugs.