e Patients have adapted differently in their lives and have jobs that mostly take place indoors. So it's very difficult. There are also no interpretation aids in the sense of: what is normal light exposure or a target value? That has not been measured or standardized.
Therefore, I believe that what is measured with the PASS is still a good indication that
that there is effectiveness. For example, it is recorded how much the patient is outdoors
hold up. I was there when evaluating the PASS data in the Netherlands and was attended to by doctors
drawn in there. There it was six hours more per week than pre-treatment. So
I think that we have a kind of best evidence among the given, really very restrictive ones
Circumstances that cannot be changed either.
I also think that therapy regret is a patient-relevant endpoint in the case of EPP,
because patients can feel for themselves whether a drug is working or not, within minutes;
well, otherwise you just get burned. After all other therapies that have been tried before,
no patient asks anymore. There is no one to practice any other approach or afterwards
would ask because it just has no effectiveness. Therefore the PASS is not optimal, but
I believe that there are things there that could easily be used.
Prof. Hecken (chairman): Thank you, Dr. Barman. - Now I still have Mr Dürr
as a topic-related patient representative. - Mr Dürr, you have the floor.
Mr. Dürr: I am a patient and therefore a user of afamelanotide. I am now 51 years old. I had to
hide in the dark for so many years because I just couldn't get into the light. We have to be clear: it's about the light, not the warmth. That means, even at minus 10 degrees,
when the sun shines it hurts me. It just hurts my skin, it makes me terrible
Pain. If you overdo it, you lie in the dark for two or three days, you cool off
the hands, the face, you don't know how to suppress the pain in any way.
It's been a completely different life since I've been treated with afamelanotide. Otherwise you can do that
not introduce. You can do things that you could never have done in the light. I can with mine
Family go shopping, I can do anything a normal person does. I can mine
Coming to work, I can do my work, even though I have a job as a teenager
chose inside. It would be unthinkable to do a job that you can somehow use sunlight
should have done. So, as I said, it's a completely different life for me. I do not want it
I live near Stuttgart, I always go to Zurich to see Ms. Anna Minder. These are for
I really do quite a long trip, especially now, for example, in the time of the corona pandemic.
Traveling to Switzerland is not that great now, nor is it that easy. It costs too. As I said,
you have to get up pretty early. If you want to keep the appointment at 10 a.m. in Zurich,
you leave at 5 a.m. It's a 500km round trip. I have to
still make additional payments of 80 francs per implant. I have to take a vacation for this. Do that
I like everything very much, I don't care about anything, the main thing is that I get the medication and can my life
Go on living as in the last four or five years. I am now in my fifth year of treatment. Like me
enjoy this life meanwhile! As I said, I am only a layman, I can only describe it to you in an amateur manner. With all the medical things, I can't follow some things, but off
In my opinion, the patient's perspective, it has become a wonderful life. - So much for a little statement
from me. Thank you for the time.
Prof. Hecken (chairman): Thank you for this statement, Mr. Dürr. I allowed that
because it was very impressive and because I didn't want to underline the pharmaceutical company's opening statement about the blatant injustices. I wanted to point out
patient advocates usually have to ask questions, but it was impressive nonetheless, and
we log what you have shown. - Now Ms. Hauke, then Dr. Rose and then
I would also want to slowly bring this hearing to a final endpoint. - Frau Hauke, please.
Ms. Hauke: I am a patient representative in the self-help EPP. I thank the two clinicians because
they conveyed very well what the treatment is about. From my experience in the
many years and mother of a daughter with EPP: You have to understand: people with this disease
have that from birth. They learn very early what they cannot do. They are maximally adapted and
conditioned. Patients who come into treatment first have to learn about these new ones
Freedoms to deal with. What is a big problem - that's what Ms. Minder and Ms. Barman said - when the patients are treated with four implants, i.e. eight months, with it on
In the beginning it takes a little time before the drug works, they completely fall into their fears in winter
back and need a while with the new implant next year to get back to - -
I think that is also noticeable in the questionnaires. - Yeah, that's actually what I do
wanted to say. A year-round treatment like the FDA and how it is possible in Australia
would give many of our patients a better quality of life. - Thank you.
Prof. Hecken (chairman): Thank you, although we are in the relevant licensing context
act. That now has less to do with the G-BA's benefit assessment, but we're also taking that
note that we will also take this on record because we will of course - and I will say that now
without any cynicism - see the very considerable suffering and suffering of the patients. That is why I am now also giving Dr. Rose still has the word, regardless of whether or not he
asks a question or makes a case report.
But I also ask - I will also be doing this in this session - the patient representatives, in future the
to instruct topic-related patient representatives in the course of the training courses to the effect that
primarily not the formulation of wishes and expectations or the submission of case reports
or other things are in the foreground of such a hearing, but rather asking questions. But that is a job that the Patient Representation Unit has to take on. But as I said,
I am now allowing this in order not to stall the discussion. - Dr. Rose please.
(short-term clarification of technical difficulties)
Dr. Rose: Is it working now?
Prof. Hecken (chairman): Yes! Yes! Who is Kurt? I am a little surprised now because woman
Dr. Barman here [editor. Note: in the chat of the video conference program] writes to a Kurt. I see,
Kurt is Dr. Rose. This is then the topic-related patient representative. Yes, okay. Thanks. So,
We note in the minutes that Kurt, who was addressed in this way by Ms. Barman, is the topic-related patient representative Dr. Kurt Rose is. - You're welcome.
Dr. Rose: I am also a patient with EPP. I can only join the two previous speakers as patient representatives, and I do not want to repeat that either. For me, taking is with one
really tremendous quality of life improvements. I also had the problem with the four
Implants, which has already been hinted at here, and would like to derive a question from this, how do you get it
I would like to know: Is there any indication of the number of implants in the
Year? And a second question that arises for me: Does it also apply to the new approvals
again comments on the age information? Because this conversation was or there were clues about
70 and the like, ... (acoustically incomprehensible) taken?
Prof. Hecken (chairman): Thank you, Mr. Rose. - Who are you directing this question to?
Dr. Rose: This is my first time here. It is not yet clear to me who will answer that
can. But these are the questions for me as a patient.
Prof. Hecken (chairman): Yes, well, we are asking questions to the pharmaceutical company and the clinical experts. So I ask the pharmaceutical company if you can
understood the question. I know the conversations that may be with regulators in Europe
conversations about admission frequency etc. pp. not. That also has for the benefit assessment
no effect, but we still like to discuss this question: Can you answer this question? Perhaps the clinical experts whose patient you seem to be obviously able to do this
Answer question? That is why I had just recorded that Kurt was the patient representative, Dr. Rose is, apparently, well known to those who appear here as clinical experts
is. We just heard from a patient representative that he was talking to a clinical expert here in
Treatment is. So who can give me an answer from the company or the clinical experts?
The G-BA does not answer any questions; because we don't know the facts. Against this background
it is slowly becoming idle. But here you go, Professor Ehlers.
Prof. Ehlers (Clinuvel): Thank you for your calm tour. - I think if Dr. Wolgen say something about it.
Dr. Wolgen (Clinuvel): Of course. - At the beginning, in 2014, when we discussed the admission requirements with the EMA, - I am now speaking to Dr. Rose in lay terms, in lay words -
came out, that is, a label that doctors appropriate a maximum of four implants per year
can prescribe, but with a maximum of six. But that depends on the decision between doctor and patient. So, the maximum limit is six, but the most common is four. I can
Tell you our position as a pharmaceutical company, with all countries, with all authorities in the
Countries where you have to negotiate how a product comes onto the market and so in Germany too,
a maximum number per treatment per year was determined in 2017. To stay within this treatment maximum, most clinicians have said we will only get four implants a year
deliver, although doctors are qualified to prescribe a maximum of six per patient. But we wanted them
Threshold not to cross the border in Germany. So that's limited.
Regarding your question of how it will look in the future: We are currently talking to the EMA about the patient
like in America and Australia to be able to give the therapy all year round. But that will also be of
depend on the discussions with the G-BA and the GKV.
Prof. Hecken (chairman): Thank you. - Are there any other questions?
Miss Dr. Minder (Stadtspital Waid und Triemli): I would like to say for the record: Dr. rose
is not being treated by me.
Prof. Hecken (chairman): Okay. - Then we'll take that on record. Then you know him
Miss Dr. Minder (Stadtspital Waid und Triemli): No, I don't know him either.
Prof. Ehlers (Clinuvel): I had reported in the chat, if we get to the end
Prof. Hecken (chairman): No, I just wanted to - - Oh, you don't know each other, but
Are you still writing to Kurt?
Miss Dr. Minder (Stadtspital Waid und Triemli): No, that was Ms. Barman.
Prof. Hecken (chairman): Yes, yes, good. OK.
Miss Dr. Barman-Aksözen (Stadtspital Waid und Triemli): I am a scientist and have given lectures in the German self-help EPP and was a member, so we know each other. This is
a very ... (acoustically incomprehensible)
Prof. Hecken (chairman): It doesn't matter. - I just took note of it,
but we've cleared it up now. - Professor Dr. Dr. Ehlers, you reported.
Prof. Ehlers (Clinuvel): Thank you very much. - I just wanted to thank you, despite
this special introduction by the company. The main points that we wanted to make are in the
written statement from the pharmaceutical company, and ask for your understanding.
Prof. Hecken (chairman): Thank you. - You don't need to ask for understanding, sir
Ehlers, you know that we handle every situation appropriately and, as I said, we do not allow ourselves to be influenced in our impartial and evidence-based decision-making. With this in mind, there is nothing to worry about. I thank myself. I take that as a final word, Mr. Ehlers,
or should another closing word be added? - That's not the case.
Thank you for this hearing, thank you for the comments that have been made
and finish this hearing. - We continue the meeting of the Medicines Subcommittee with one
Hopefully the hearing will proceed in a more conventional manner tomorrow morning, as stated in the agenda.
The session is over for today. I wish everyone involved a nice rest of the day. thanks
The hearing ends at 4:14 p.m.