WEBVTT
1
00:00:04.990 --> 00:00:08.029
Hi, my name is Paz Valanki. I'm a medical oncologist
2
00:00:08.089 --> 00:00:11.789
and I'm vice president of clinical developments at Precision for Medicine.
3
00:00:12.050 --> 00:00:16.089
We are a contract research organization that is focused on oncology,
4
00:00:16.179 --> 00:00:18.059
rare diseases, and autoimmune diseases.
5
00:00:19.760 --> 00:00:21.559
What is the AHRQ program?
6
00:00:22.019 --> 00:00:27.100
The AHRQ program, this is the Accelerating Rare Disease Cures program.
7
00:00:28.309 --> 00:00:32.509
This was launched in 2022, and it was launched within
8
00:00:32.649 --> 00:00:37.390
CDER at FDA, but it does collaborate extensively with other
9
00:00:37.490 --> 00:00:41.140
centers within FDA. And really, the goal has been to
10
00:00:41.259 --> 00:00:44.799
accelerate the development of safe and effective therapies for patients
11
00:00:44.820 --> 00:00:48.500
with high unmet need with rare diseases. And this has
12
00:00:48.560 --> 00:00:53.420
included a variety of programs and initiatives. They've had several
13
00:00:53.659 --> 00:00:58.880
successful pilot programs. They've had patient listening sessions that are
14
00:00:58.990 --> 00:01:02.439
within the office of the commissioner. And really, the focus
15
00:01:02.460 --> 00:01:08.260
here has been patient-centric drug development. There have been workshops
16
00:01:08.299 --> 00:01:12.120
that have been focusing on methodologies for novel clinical trial
17
00:01:12.159 --> 00:01:17.469
designs and using novel endpoints or biomarkers. Since we know
18
00:01:17.530 --> 00:01:20.670
that drug development for rare diseases can be quite complex
19
00:01:20.790 --> 00:01:24.530
and different than more common diseases, And oftentimes we need
20
00:01:24.549 --> 00:01:26.230
to take a different approach than some of our more
21
00:01:26.290 --> 00:01:27.549
conventional trial designs.
22
00:01:28.569 --> 00:01:30.569
What is the roadmap for ARC?
23
00:01:31.590 --> 00:01:36.459
CEDAR issued this five-year roadmap for the ARC program. And one,
24
00:01:36.480 --> 00:01:40.540
it defined what the program was. Two, it described how
25
00:01:40.560 --> 00:01:45.530
we're in this refined stage. There's a four-part implementation that
26
00:01:45.549 --> 00:01:48.890
they're trying to carry out for this program. And so
27
00:01:48.969 --> 00:01:52.810
now we're looking at pilot programs metrics to see how
28
00:01:52.890 --> 00:01:55.989
successful they may or may not have been. And then again,
29
00:01:56.090 --> 00:02:01.890
how do you continue to coordinate efforts across FDA? And
30
00:02:01.920 --> 00:02:05.579
so the roadmap, one, they talk about some of these initiatives.
31
00:02:05.620 --> 00:02:08.120
They talk about workshops that they have done. They talk
32
00:02:08.159 --> 00:02:14.259
about publications, including case studies that exemplify rare disease drug development.
33
00:02:15.009 --> 00:02:17.810
And then two, they talk about where things are going next.
34
00:02:18.849 --> 00:02:21.460
That I would say was there were not as many
35
00:02:21.500 --> 00:02:24.759
details as I would have liked regarding next steps, but
36
00:02:24.800 --> 00:02:27.650
I do think that this highlights that there really is
37
00:02:27.990 --> 00:02:34.689
ongoing momentum and focus on continuing to support drug development
38
00:02:34.930 --> 00:02:35.710
for rare diseases.
39
00:02:36.789 --> 00:02:40.169
How can precision medicine help small biotech companies?
40
00:02:40.870 --> 00:02:44.469
In our biotech company, we oftentimes are dealing with, again,
41
00:02:44.530 --> 00:02:48.199
patients or with programs that are focused on rare diseases
42
00:02:48.219 --> 00:02:52.800
and And again, we can't always be thinking about standard
43
00:02:52.919 --> 00:02:57.530
or conventional trial designs. Oftentimes when you're in early drug
44
00:02:57.550 --> 00:03:01.370
development or early phase drug development, you don't necessarily have
45
00:03:01.409 --> 00:03:05.430
to think about, will this endpoint that we're evaluating in
46
00:03:05.449 --> 00:03:08.949
the early phase setting be supportive of an approval in
47
00:03:09.189 --> 00:03:12.750
a later disease setting or in a later line of
48
00:03:12.949 --> 00:03:17.500
investigation rather? That being said, I think it's really important
49
00:03:17.539 --> 00:03:19.889
to think from the very beginning what the registrational pathway
50
00:03:19.930 --> 00:03:23.270
is going to look like. The AHRQ program does have
51
00:03:23.370 --> 00:03:29.500
some initiatives to encourage increased engagement between sponsors and the FDA.
52
00:03:30.099 --> 00:03:34.139
For example, there was this START pilot program. And again,
53
00:03:34.199 --> 00:03:38.180
this is when you have more complex design questions, and
54
00:03:38.219 --> 00:03:43.110
this allows for additional engagements between sponsors and FDA. I
55
00:03:43.150 --> 00:03:47.110
will say in this pilot program for CDER programs, this
56
00:03:47.129 --> 00:03:52.330
was primarily limited to neurodegenerative disease and metabolic disorders. And
57
00:03:52.370 --> 00:03:55.419
then for CBER, this was for cell and gene therapies
58
00:03:56.719 --> 00:03:59.639
in which there might be significant disability or death within
59
00:03:59.680 --> 00:04:02.879
the first decade of life. So they had this pilot program, again,
60
00:04:03.060 --> 00:04:05.240
to try to help sponsors that are trying to develop
61
00:04:05.879 --> 00:04:09.530
in a rare disease setting. But at that stage, it
62
00:04:09.590 --> 00:04:14.080
was limited to certain focus areas. So I would be
63
00:04:14.120 --> 00:04:17.319
very interested to see if they intend to expand this
64
00:04:17.360 --> 00:04:19.779
further and if this is something that would be of
65
00:04:19.860 --> 00:04:24.689
interest to our clients in our biotech company outside of
66
00:04:24.730 --> 00:04:25.610
those disease areas.
67
00:04:26.970 --> 00:04:30.449
What happens if natural history data is limited?
68
00:04:31.129 --> 00:04:33.850
No, that's a great question because on the one hand,
69
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as you mentioned, there is oftentimes a lack of natural
70
00:04:37.329 --> 00:04:40.879
history data. On the other hand, the AHRQ program does
71
00:04:40.920 --> 00:04:43.689
talk about how you can use natural history data data
72
00:04:43.769 --> 00:04:47.420
to help support for an approval. And they actually do
73
00:04:47.459 --> 00:04:50.399
provide some examples and case studies in which that has
74
00:04:50.420 --> 00:04:54.319
been used. I can think of two examples even. So
75
00:04:54.360 --> 00:04:57.519
if you go to the AHRQ website, they again have
76
00:04:57.579 --> 00:05:00.610
all of these different case studies that you can look at.
77
00:05:01.350 --> 00:05:04.790
In general, when we're thinking about substantial evidence of effectiveness
78
00:05:05.050 --> 00:05:08.769
and the FDA requirements to demonstrate substantial evidence of effectiveness,
79
00:05:09.769 --> 00:05:14.459
oftentimes we've historically thought about two adequate and well-controlled trials.
80
00:05:15.180 --> 00:05:18.639
That doesn't always apply. We don't usually follow that rule
81
00:05:18.680 --> 00:05:22.509
in oncology, for example. And for rare diseases, that's also
82
00:05:22.550 --> 00:05:27.129
very similar. Oftentimes, you're looking at one adequate and well-controlled
83
00:05:27.350 --> 00:05:32.189
trial and confirmatory evidence to help support substantial evidence of effectiveness.
84
00:05:33.269 --> 00:05:37.069
So there are some case studies that they have published
85
00:05:37.189 --> 00:05:41.230
where there might be some natural history data, perhaps from
86
00:05:42.269 --> 00:05:46.009
a registry. Again, it could be a relatively small data set,
87
00:05:46.050 --> 00:05:49.930
but that can still be very helpful. And using that data,
88
00:05:49.990 --> 00:05:53.410
that can help provide confirmatory evidence to support an approval.
89
00:05:54.250 --> 00:05:57.110
So there was one example where they had, I think
90
00:05:57.129 --> 00:05:59.740
it was in Friedreich's ataxia, they had one adequate and
91
00:05:59.769 --> 00:06:03.939
well-controlled trial. They didn't think that that was sufficient for
92
00:06:03.980 --> 00:06:06.740
an approval. So they ended up doing an open label
93
00:06:06.819 --> 00:06:12.279
extension study. and doing propensity matching with natural history data
94
00:06:12.319 --> 00:06:16.209
from a registry, and that was used to generate confirmatory evidence.
95
00:06:17.009 --> 00:06:21.089
So that's an example of success with using natural history. Again,
96
00:06:21.829 --> 00:06:25.769
acknowledging that sometimes that natural history data isn't always readily available.
97
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How does this program ensure that the data is scientifically rigorous?
98
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You do need to generate kind of a body of
99
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evidence and have a totality of data that Sometimes when
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you're extending an indication for younger patients, for example, there
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might be opportunities for clinical pharmacology modeling. And so that
102
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might have been a scenario in which that was a possibility.
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I would have to go and look at that exact approval.
104
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But oftentimes it is evidence-based or there is some evidence
105
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to support that. I know, for example, when we were
106
00:07:01.769 --> 00:07:06.350
working on an approval for nasopharyngeal carcinoma, That's a pretty
107
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uncommon cancer in pediatric and adolescent patients. That being said,
108
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we had been encouraging the sponsor to try to enroll
109
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patients that were adolescents. And if we could generate some data,
110
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could that help support an approval in that population where
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maybe they don't have any other options? That being said,
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they weren't able to recruit any patients. But I think
113
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there's always this balance of FDA is always going to
114
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be focused on generating robust data. data to support an approval,
115
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but also there needs to be opportunities to provide therapies
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to patients that have high unmet need where there's maybe
117
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other supportive data to support expanding an indication, biological rationale,
118
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et cetera. So again, there needs to obviously be a
119
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balance of safety and efficacy and robust data generation, but
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also making it possible for other patients with unmet need
121
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to obtain access to a therapy.
122
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What are some practical strategies for small biotech companies?
123
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The core advice is still the same when you're developing
124
00:08:10.730 --> 00:08:13.910
for rare diseases as you are developing in other areas.
125
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We recommend early and frequent engagement with regulatory agencies. That's
126
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really helpful just to make sure that you're on the
127
00:08:21.509 --> 00:08:25.290
same page all the way from the pre-IND setting through
128
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the post-marketing setting. So I think those engagements are really important.
129
00:08:30.410 --> 00:08:32.889
I mentioned, again, that there are some pilot programs which
130
00:08:32.950 --> 00:08:35.690
are meant to enhance some of these formal meetings. So
131
00:08:35.950 --> 00:08:38.909
the START program, as I mentioned, that was a way
132
00:08:38.970 --> 00:08:43.039
that they were trying to augment some of those interactions.
133
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And so I think if there are opportunities there, there's
134
00:08:47.120 --> 00:08:52.059
also some other initiatives like the translational science team. And
135
00:08:52.080 --> 00:08:55.370
so there are other kind of mechanisms that FDA offer
136
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for that. sponsors to engage with if you're developing a
137
00:09:00.029 --> 00:09:03.049
novel biomarker or if you have a novel and innovative
138
00:09:03.090 --> 00:09:06.820
clinical trial design. So there are the formal meetings and
139
00:09:06.860 --> 00:09:11.480
then there are some additional interactions that the AHRQ program
140
00:09:11.620 --> 00:09:12.700
provides for.
141
00:09:12.720 --> 00:09:16.950
Have a great day.
142
00:09:16.970 --> 00:09:18.250
Bye. Okay, bye-bye.
1
00:00:04.990 --> 00:00:08.029
Hi, my name is Paz Valanki. I'm a medical oncologist
2
00:00:08.089 --> 00:00:11.789
and I'm vice president of clinical developments at Precision for Medicine.
3
00:00:12.050 --> 00:00:16.089
We are a contract research organization that is focused on oncology,
4
00:00:16.179 --> 00:00:18.059
rare diseases, and autoimmune diseases.
5
00:00:19.760 --> 00:00:21.559
What is the AHRQ program?
6
00:00:22.019 --> 00:00:27.100
The AHRQ program, this is the Accelerating Rare Disease Cures program.
7
00:00:28.309 --> 00:00:32.509
This was launched in 2022, and it was launched within
8
00:00:32.649 --> 00:00:37.390
CDER at FDA, but it does collaborate extensively with other
9
00:00:37.490 --> 00:00:41.140
centers within FDA. And really, the goal has been to
10
00:00:41.259 --> 00:00:44.799
accelerate the development of safe and effective therapies for patients
11
00:00:44.820 --> 00:00:48.500
with high unmet need with rare diseases. And this has
12
00:00:48.560 --> 00:00:53.420
included a variety of programs and initiatives. They've had several
13
00:00:53.659 --> 00:00:58.880
successful pilot programs. They've had patient listening sessions that are
14
00:00:58.990 --> 00:01:02.439
within the office of the commissioner. And really, the focus
15
00:01:02.460 --> 00:01:08.260
here has been patient-centric drug development. There have been workshops
16
00:01:08.299 --> 00:01:12.120
that have been focusing on methodologies for novel clinical trial
17
00:01:12.159 --> 00:01:17.469
designs and using novel endpoints or biomarkers. Since we know
18
00:01:17.530 --> 00:01:20.670
that drug development for rare diseases can be quite complex
19
00:01:20.790 --> 00:01:24.530
and different than more common diseases, And oftentimes we need
20
00:01:24.549 --> 00:01:26.230
to take a different approach than some of our more
21
00:01:26.290 --> 00:01:27.549
conventional trial designs.
22
00:01:28.569 --> 00:01:30.569
What is the roadmap for ARC?
23
00:01:31.590 --> 00:01:36.459
CEDAR issued this five-year roadmap for the ARC program. And one,
24
00:01:36.480 --> 00:01:40.540
it defined what the program was. Two, it described how
25
00:01:40.560 --> 00:01:45.530
we're in this refined stage. There's a four-part implementation that
26
00:01:45.549 --> 00:01:48.890
they're trying to carry out for this program. And so
27
00:01:48.969 --> 00:01:52.810
now we're looking at pilot programs metrics to see how
28
00:01:52.890 --> 00:01:55.989
successful they may or may not have been. And then again,
29
00:01:56.090 --> 00:02:01.890
how do you continue to coordinate efforts across FDA? And
30
00:02:01.920 --> 00:02:05.579
so the roadmap, one, they talk about some of these initiatives.
31
00:02:05.620 --> 00:02:08.120
They talk about workshops that they have done. They talk
32
00:02:08.159 --> 00:02:14.259
about publications, including case studies that exemplify rare disease drug development.
33
00:02:15.009 --> 00:02:17.810
And then two, they talk about where things are going next.
34
00:02:18.849 --> 00:02:21.460
That I would say was there were not as many
35
00:02:21.500 --> 00:02:24.759
details as I would have liked regarding next steps, but
36
00:02:24.800 --> 00:02:27.650
I do think that this highlights that there really is
37
00:02:27.990 --> 00:02:34.689
ongoing momentum and focus on continuing to support drug development
38
00:02:34.930 --> 00:02:35.710
for rare diseases.
39
00:02:36.789 --> 00:02:40.169
How can precision medicine help small biotech companies?
40
00:02:40.870 --> 00:02:44.469
In our biotech company, we oftentimes are dealing with, again,
41
00:02:44.530 --> 00:02:48.199
patients or with programs that are focused on rare diseases
42
00:02:48.219 --> 00:02:52.800
and And again, we can't always be thinking about standard
43
00:02:52.919 --> 00:02:57.530
or conventional trial designs. Oftentimes when you're in early drug
44
00:02:57.550 --> 00:03:01.370
development or early phase drug development, you don't necessarily have
45
00:03:01.409 --> 00:03:05.430
to think about, will this endpoint that we're evaluating in
46
00:03:05.449 --> 00:03:08.949
the early phase setting be supportive of an approval in
47
00:03:09.189 --> 00:03:12.750
a later disease setting or in a later line of
48
00:03:12.949 --> 00:03:17.500
investigation rather? That being said, I think it's really important
49
00:03:17.539 --> 00:03:19.889
to think from the very beginning what the registrational pathway
50
00:03:19.930 --> 00:03:23.270
is going to look like. The AHRQ program does have
51
00:03:23.370 --> 00:03:29.500
some initiatives to encourage increased engagement between sponsors and the FDA.
52
00:03:30.099 --> 00:03:34.139
For example, there was this START pilot program. And again,
53
00:03:34.199 --> 00:03:38.180
this is when you have more complex design questions, and
54
00:03:38.219 --> 00:03:43.110
this allows for additional engagements between sponsors and FDA. I
55
00:03:43.150 --> 00:03:47.110
will say in this pilot program for CDER programs, this
56
00:03:47.129 --> 00:03:52.330
was primarily limited to neurodegenerative disease and metabolic disorders. And
57
00:03:52.370 --> 00:03:55.419
then for CBER, this was for cell and gene therapies
58
00:03:56.719 --> 00:03:59.639
in which there might be significant disability or death within
59
00:03:59.680 --> 00:04:02.879
the first decade of life. So they had this pilot program, again,
60
00:04:03.060 --> 00:04:05.240
to try to help sponsors that are trying to develop
61
00:04:05.879 --> 00:04:09.530
in a rare disease setting. But at that stage, it
62
00:04:09.590 --> 00:04:14.080
was limited to certain focus areas. So I would be
63
00:04:14.120 --> 00:04:17.319
very interested to see if they intend to expand this
64
00:04:17.360 --> 00:04:19.779
further and if this is something that would be of
65
00:04:19.860 --> 00:04:24.689
interest to our clients in our biotech company outside of
66
00:04:24.730 --> 00:04:25.610
those disease areas.
67
00:04:26.970 --> 00:04:30.449
What happens if natural history data is limited?
68
00:04:31.129 --> 00:04:33.850
No, that's a great question because on the one hand,
69
00:04:34.550 --> 00:04:37.290
as you mentioned, there is oftentimes a lack of natural
70
00:04:37.329 --> 00:04:40.879
history data. On the other hand, the AHRQ program does
71
00:04:40.920 --> 00:04:43.689
talk about how you can use natural history data data
72
00:04:43.769 --> 00:04:47.420
to help support for an approval. And they actually do
73
00:04:47.459 --> 00:04:50.399
provide some examples and case studies in which that has
74
00:04:50.420 --> 00:04:54.319
been used. I can think of two examples even. So
75
00:04:54.360 --> 00:04:57.519
if you go to the AHRQ website, they again have
76
00:04:57.579 --> 00:05:00.610
all of these different case studies that you can look at.
77
00:05:01.350 --> 00:05:04.790
In general, when we're thinking about substantial evidence of effectiveness
78
00:05:05.050 --> 00:05:08.769
and the FDA requirements to demonstrate substantial evidence of effectiveness,
79
00:05:09.769 --> 00:05:14.459
oftentimes we've historically thought about two adequate and well-controlled trials.
80
00:05:15.180 --> 00:05:18.639
That doesn't always apply. We don't usually follow that rule
81
00:05:18.680 --> 00:05:22.509
in oncology, for example. And for rare diseases, that's also
82
00:05:22.550 --> 00:05:27.129
very similar. Oftentimes, you're looking at one adequate and well-controlled
83
00:05:27.350 --> 00:05:32.189
trial and confirmatory evidence to help support substantial evidence of effectiveness.
84
00:05:33.269 --> 00:05:37.069
So there are some case studies that they have published
85
00:05:37.189 --> 00:05:41.230
where there might be some natural history data, perhaps from
86
00:05:42.269 --> 00:05:46.009
a registry. Again, it could be a relatively small data set,
87
00:05:46.050 --> 00:05:49.930
but that can still be very helpful. And using that data,
88
00:05:49.990 --> 00:05:53.410
that can help provide confirmatory evidence to support an approval.
89
00:05:54.250 --> 00:05:57.110
So there was one example where they had, I think
90
00:05:57.129 --> 00:05:59.740
it was in Friedreich's ataxia, they had one adequate and
91
00:05:59.769 --> 00:06:03.939
well-controlled trial. They didn't think that that was sufficient for
92
00:06:03.980 --> 00:06:06.740
an approval. So they ended up doing an open label
93
00:06:06.819 --> 00:06:12.279
extension study. and doing propensity matching with natural history data
94
00:06:12.319 --> 00:06:16.209
from a registry, and that was used to generate confirmatory evidence.
95
00:06:17.009 --> 00:06:21.089
So that's an example of success with using natural history. Again,
96
00:06:21.829 --> 00:06:25.769
acknowledging that sometimes that natural history data isn't always readily available.
97
00:06:26.379 --> 00:06:30.379
How does this program ensure that the data is scientifically rigorous?
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You do need to generate kind of a body of
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evidence and have a totality of data that Sometimes when
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you're extending an indication for younger patients, for example, there
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might be opportunities for clinical pharmacology modeling. And so that
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might have been a scenario in which that was a possibility.
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I would have to go and look at that exact approval.
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But oftentimes it is evidence-based or there is some evidence
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to support that. I know, for example, when we were
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working on an approval for nasopharyngeal carcinoma, That's a pretty
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uncommon cancer in pediatric and adolescent patients. That being said,
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we had been encouraging the sponsor to try to enroll
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patients that were adolescents. And if we could generate some data,
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could that help support an approval in that population where
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maybe they don't have any other options? That being said,
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they weren't able to recruit any patients. But I think
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there's always this balance of FDA is always going to
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be focused on generating robust data. data to support an approval,
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but also there needs to be opportunities to provide therapies
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to patients that have high unmet need where there's maybe
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other supportive data to support expanding an indication, biological rationale,
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et cetera. So again, there needs to obviously be a
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balance of safety and efficacy and robust data generation, but
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also making it possible for other patients with unmet need
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to obtain access to a therapy.
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What are some practical strategies for small biotech companies?
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The core advice is still the same when you're developing
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for rare diseases as you are developing in other areas.
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We recommend early and frequent engagement with regulatory agencies. That's
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really helpful just to make sure that you're on the
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same page all the way from the pre-IND setting through
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the post-marketing setting. So I think those engagements are really important.
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I mentioned, again, that there are some pilot programs which
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are meant to enhance some of these formal meetings. So
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the START program, as I mentioned, that was a way
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that they were trying to augment some of those interactions.
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And so I think if there are opportunities there, there's
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also some other initiatives like the translational science team. And
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so there are other kind of mechanisms that FDA offer
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for that. sponsors to engage with if you're developing a
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novel biomarker or if you have a novel and innovative
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clinical trial design. So there are the formal meetings and
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then there are some additional interactions that the AHRQ program
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provides for.
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Have a great day.
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Bye. Okay, bye-bye.
